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Approach may lead to new treatment approach for osteoarthritis, obesity

Researchers at Washington University School of Medicine in St. Louis found that gene therapy in mice helped build strength and significant muscle mass quickly, while reducing the severity of osteoarthritis. The gene therapy also prevented obesity, even when the mice were fed a high-fat diet.

Exercise and physical therapy often are recommended to help people who have arthritis. Both can strengthen muscle a benefit that also can reduce joint pain. But building muscle mass and strength can take many months and be difficult in the face of joint pain from osteoarthritis, particularly for older people who are overweight. A new study in mice at Washington University School of Medicine in St. Louis, however, suggests gene therapy one day may help those patients.

The research shows that gene therapy helped build significant muscle mass quickly and reduced the severity of osteoarthritis in the mice, even though they didnt exercise more. The therapy also staved off obesity, even when the mice ate an extremely high-fat diet.

The study is published online May 8 in the journal Science Advances.

Obesity is the most common risk factor for osteoarthritis, said senior investigator Farshid Guilak, PhD, the Mildred B. Simon Research Professor of Orthopaedic Surgery and director of research at Shriners Hospitals for Children St. Louis. Being overweight can hinder a persons ability to exercise and benefit fully from physical therapy. Weve identified here a way to use gene therapy to build muscle quickly. It had a profound effect in the mice and kept their weight in check, suggesting a similar approach may be effective against arthritis, particularly in cases of morbid obesity.

With the papers first author, Ruhang Tang, PhD, a senior scientist in Guilaks laboratory, Guilak and his research team gave 8-week-old mice a single injection each of a virus carrying a gene called follistatin. The gene works to block the activity of a protein in muscle that keeps muscle growth in check. This enabled the mice to gain significant muscle mass without exercising more than usual.

Even without additional exercise, and while continuing to eat a high-fat diet, the muscle mass of these super mice more than doubled, and their strength nearly doubled, too. The mice also had less cartilage damage related to osteoarthritis, lower numbers of inflammatory cells and proteins in their joints, fewer metabolic problems, and healthier hearts and blood vessels than littermates that did not receive the gene therapy. The mice also were significantly less sensitive to pain.

One worry was that some of the muscle growth prompted by the gene therapy might turn out to be harmful. The heart, for example, is a muscle, and a condition called cardiac hypertrophy, in which the hearts walls thicken, is not a good thing. But in these mice, heart function actually improved, as did cardiovascular health in general.

Longer-term studies will be needed to determine the safety of this type of gene therapy. But, if safe, the strategy could be particularly beneficial for patients with conditions such as muscular dystrophy that make it difficult to build new muscle.

In the meantime, Guilak, who also co-directs the Washington University Center for Regenerative Medicine and is a professor of biomedical engineering and of developmental biology, said more traditional methods of muscle strengthening, such as lifting weights or physical therapy, remain the first line of treatment for patients with osteoarthritis.

Something like this could take years to develop, but were excited about its prospects for reducing joint damage related to osteoarthritis, as well as possibly being useful in extreme cases of obesity, he said.

Tang R, Harasymowicz NS, Wu CL, Collins KH, Choi YR, Oswald SJ, Guilak F. Gene therapy for follistatin mitigates systemic metabolic inflammation and post-traumatic arthritis in high-fat diet-induced obesity. Science Advances, published online May 8, 2020.

This work was supported by the Shriners Hospitals for Children, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute on Aging and the Office of the Director of the National Institutes of Health (NIH). Grant numbers AR50245, AR48852, AG15768, AR48182, AG 46927, AR073752, OD10707, AR060719, AR057235. Additional funding was provided by the Arthritis Foundation and the Nancy Taylor Foundation for Chronic Diseases.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Gene therapy in mice builds muscle, reduces fat Washington University School of Medicine in St. Louis - Washington University School of Medicine in...

In 2013, a mysterious new flu virus hit the world. It was the H7N9 strain of bird flu, and by the time it infected 120 and killed 23, researchers still didnt know how it was transmitted or how to stop it. As the virus arrived in the dense Chinese province of Hunan, health officials began to fear the worst.

In the U.S., they called Dan Gibson. Gibson was Vice President of DNA Technology atSynthetic Genomics, the research institute founded in 2005 by genomics pioneers Craig Venter, Ph.D., and Nobel Laureate Hamilton Smith, M.D., shortly after the completion of the Human Genome Project. Among its research milestones, the institute created the first synthetic cell, designed in a computer and DNA-printed completely from scratch.

Gibson was given a download link to the DNA sequence of the bird flu virus and told to do one thing: design a vaccine.

Gibson is the namesake of the Gibson assembly method, the worlds leading branded method of joining DNA fragments together that he pioneered just four years before. The method lets you join DNA fragments together in a single step, and it proved to have a huge multiplying effect on the entire field ofsynthetic biology. So who better to ask for a DNA solution to the bird flu?

Dan Gibson is a pioneer in synthetic biology who can design vaccines against coronavirus and other pathogens in about an hour. Now, labs can put his methods to work on their own benchtops. Codex

In truth, Gibson had been preparing for this moment for years. His team was already collaborating with Novartis on a grant from the Biomedical Advanced Research and Development Authority (BARDA) on pandemic preparedness. The goal of that project was to create a vaccine against the H1N1 virus. BARDA designed some ambitious, forward-thinking exercises where project members were given an H1N1-type pandemic scenario, a DNA sequence, and a one-week deadline to manufacture a vaccine.

Even in 2013, Gibsons team had shown they could design a vaccine candidate within about 24 hours once given the virus sequence. But they knew that many of the things they were doing in the laboratory could be automated, which would enable others to pursue alternative vaccine candidates in parallel. They had been building a prototype machine that integrated all of their learnings and processes into a single box when H7N9 hit.

This is not a fire drill anymore, Gibson recalls thinking to himself. This is the real deal.

Gibsons team and its prototype system successfully developed an H7N9 vaccine, which Novartis made and the US government stockpiled. Fortunately, the virus did not become a pandemic. But the goal of putting their labs genius into a single box became the origin of a new company and a new product.

The genetic evolution of H7N9 bird virus in China, 2013. Like the novel coronavirus, the H7N9 bird virus probably originated in so-called wet markets. U.S. Centers for Disease Control

Codex DNA(formerly SGI DNA) was launched to make this technology available to researchers everywhere. Codex says that its flagship machine the BioXp 3200 system is the worlds first commercially available, fully automated gene synthesis platform. It synthesizes ready-to-use custom DNA overnight.

The same machine can be used to make DNA to create diagnostic tools, design and develop vaccines, or synthesize the cells to make those vaccines.

The instrument can make up to 32 genes at a time, and it can incorporate downstream processes like assembling the genes into the customers favorite host organism. It can also be used with newer cell-free methods of amplifying DNA to create micrograms of material.

Part of the special sauce in the BioXp system is its error correction process. The Codex team puts specific enzymes in the reagent mix that recognize and remove any unwanted mutations from the population. This allows Codex to synthesize extremely high-fidelity genes, which is critical to applications involving human therapies.

Its not only a research tool, but also a biomanufacturing tool across a wide range of applications. The system streamlines clinical research and allows for distributed manufacturing of personalized therapies, Gibson told me. Its the kind of tool thats needed in hospitals everywhere to not only make brand-new drugs locally, but also to realize the dream of individualized treatments for cancer, rare diseases, and other maladies that humans suffer.

In September, Codex closed a $25 Million Series A financing round to support the commercial launch of the BioXp system, and the company is now focused on making the system accessible to anyone in the research community who has a need for on-demand DNA synthesis across a variety of fields, including vaccine development, antibody drug discovery, personalized medicine, and beyond.

In particular, Gibson and his team are on a mission to get this platform to any researcher working on coronavirus to help in the effort to develop and make therapies and vaccines. There are now hundreds of BioXp systems around the world, with customers now doing in their lab with the BioXp system what Gibson alone was doing back in 2013.

In addition, Codex can provide researchers mostanything they might need to fight the SARS-CoV-2 virus, including positiveSynthetic RNA controlsfor tests, thefull synthetic genomefor vaccine and treatment development, and custom-order antigen panels and antibody librariesall of it made on the BioXp system.

The challenge with coronavirus, says Gibson, who is now the CTO of Codex, is that theres no defined process yet for how you go from your vaccine candidate to a final vaccine. Codexs customers and partners arent immune to that challenge, but the BioXp system is drastically speeding up the process of developing vaccine candidates.

Gibson says of one of his customers: He had ordered reagents from us and six days later he was injecting a vaccine candidate into mice.

All of this is light-years ahead of the current seasonal flu vaccine process, where you typically wait to receive a mucus sample in the mail, isolate the virus, inject it into chicken eggs, and then let them incubate. Thewhole vaccine selection and production processcan take six months, by which time the virus has mutated and your vaccine may not work that well.

Whats so amazing about synthetic biology is that if you have the DNA sequence, you can just download it and start synthesizing it right away, he says.

In a world where infections can travel as fast and far as a jet plane, well need biological defenses that can travel at the speed of the internet.

Follow me on twitter at@johncumbersand@synbiobeta. Subscribe to my weekly newsletters insynthetic biologyThank you toStephanie Michelsenfor additional research and reporting in this article. Im the founder ofSynBioBeta, and some of the companies that I write aboutincluding Codex DNAare sponsors of theSynBioBeta conferenceandweekly digestheres the full list of SynBioBeta sponsors.

Originally published on Forbes: https://www.forbes.com/sites/johncumbers/2020/05/14/a-coronavirus-vaccine-in-six-days/

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A Coronavirus Vaccine Candidate In Six Days? This Company Is Fast-Tracking Vaccine Development With The First Fully Automated Gene Synthesis Platform...

Siddhartha Mukherjee's "The Gene: An Intimate History" is a wonderful book to read at any time. During the COVID-19 pandemic, it may have even greater appeal.

The book is not just informative, it's also entertaining. And poetic. And personal. And, perhaps above all, accessible.

Mukherjee, the Pulitzer Prize-winning author of "The Emperor of All Maladies: A Biography of Cancer," makes an incredibly complex subject simple enough that even I can understand what he's saying. And he does it with a mixture of styles and approaches. He includes scientific descriptions. He reaches back to Aristotle and Plato for historical context. He tells compelling narrative stories about situations that led to advances in the understanding of what genes are and how they work. He discusses how genetic issues have played roles in his own life and family.

Within all this, he also portrays the ethical quagmires and quandaries that have emerged with advances in fields of medicine, psychiatry and human development as we've learned how to manipulate genes. Finally, he also clearly demonstrates how much more remains to be learned, and he ignites the imagination with the astounding array of possibilities -- inspiring as well as frightening -- that lie ahead.

"The Gene" is a masterful success on every one of the many levels of literary, historical, scientific and philosophical thought it engages. It is both deeply pleasurable to read and powerfully educational.

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Today's Recommended Read: "The Gene: An Intimate History" by Siddhartha Mukherjee - Chicago Daily Herald

We partner with clients in life sciences and medical technology hubs around the world who are developing medical devices and diagnostic tools enabled by optics.

By Optikos Corporation

Whether youre a start-up in the early concept stage, or a Fortune 500 looking to revamp a mature product, Optikos can help make your next medical device a reality. Where other optical consulting firms might offer only lens design services, Optikos has built its reputation on providing our clients with access to a broadly skilled team of optical problem solvers who provide solutions and continuity of support throughout your product development cycle.

Decades of service in the optics industry have given us aproven track recordof innovative and practical problem solvingthat serves the development needs of a diverse portfolio of clients.We know how to design products, assemblies and instruments that work for clients that range from medical start-ups to large institutions. The Optikos engineering team helps make our customers medical devices and diagnostic systems a reality while meeting high standards and requirements for performance and safety. Whether it is a unique microscopy system, gene sequencing platform, endoscopic instrument, or other type of optically based system.

Many of our clients turn to Optikos for additional engineering bandwidth or foraccess to the skills and experience their staff may not haveto develop an optically-based product or solution. When you need aflexible and scalable resource for engineering and assembly, we can work with you to quickly provide a powerful team during the most intense portions of a product development effort. We can then readily scale the team up or down according to the needs of the project.

Optikos clients are located worldwide, and our headquarters is in the greater Boston, MA area among the worlds top private- and public-sector healthcare innovators.The diverse nature of our clients and their applications requires our engineers to have the ability to adapt to the culture of an organization as well as to local and regional customs and regulations. Our teams have worked at the forefront of surgical and medical technologies, developing complicated designs that incorporate optics and photonics, opto-mechanics, software, electronics, and automation; and meet rigorous regulatory requirements, and we bring this experience to every new project we take on, no matter how large or small.

And when you transition to manufacturing, Optikos is well-positioned to help. Optikos leverages its world-renowned expertise in lens/sensor metrology and testing to ensure the performance and quality of what we design and manufacture. Well provide pilot production, test procedures, assembly instructions, and training to get you up and running quickly; well act on your behalf in identifying a manufacturer equipped to meet your needs; or well offer contract manufacturing at our facility. We do whatever is in our clients best interest.

Were ready to assist you in bringing new devices to market, including navigating strict regulations, clinical trials, and technological challenges associated with product prototype and production manufacturing. For more information, visit us online at optikos.com or reach out to us at sales@optikos.com.

Sponsored content by Optikos

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Optics makes amazing things possible in biological research and medicineand Optikos makes it happen - Mass Device

April 28, 2020

Rochester research into RNA structure and function provides key information for developing coronavirus treatments.

Viruses like the coronavirus that causes COVID-19 are able to unleash their fury because of a devious weapon: ribonucleic acid, also known as RNA.

A contingent of researchers at the University of Rochester study the RNA of viruses to better understand how RNAs work and how they are involved in diseases. As COVID-19 continues to spread around the globe, RNA research provides an important foundation for developing antiviral drugs, vaccines, and other therapeutics to disrupt the virus and stop infections.

The Universitys website is a way to find guidance and critical information during a rapidly changing situation.

Find out what to do if you or a close contact have symptoms or think you may have been exposed.

Understanding RNA structure and function helps us understand how to throw a therapeutic wrench into what the COVID-19 RNA doesmake new virus that can infect more of our cells and also the cells of other human beings, says Lynne Maquat, professor of biochemistry and biophysics at the University of Rochester Medical Center and the director of Rochesters Center for RNA Biology.

In the past few decades, as scientists came to realize that genetic material is largely regulated by the RNA it encodes, that most of our DNA produces RNA, and that RNA is not only a target but also a tool for disease therapies, the RNA research world has exploded, Maquat says. The University of Rochester understood this.

In 2007, Maquat founded the Center for RNA Biology as a means of conducting interdisciplinary research in the function, structure, and processing of RNAs. The center involves researchers from both the River Campus and the Medical Center, combining expertise in biology, chemistry, engineering, neurology, and pharmacology.

While much of the research across the University has been put on pause, labs that are involved in coronavirus research remain active.

Our strength as a university is our diversity of research expertise, combined with our highly collaborative nature, says Dragony Fu, an assistant professor of biology on the River Campus and a member of the Center for RNA Biology. We are surrounded by outstanding researchers who enhance our understanding of RNA biology, and a medical center that provides a translational aspect where the knowledge gained from RNA biology can be applied for therapeutics.

In mammals, such as humans, DNA contains genetic instructions that are transcribedor copiedinto RNA. While DNA remains in the cells nucleus, RNA carries the copies of genetic information to the rest of the cell by way of various combinations of amino acids, which it delivers to ribosomes. The ribosomes link the amino acids together to form proteins that then carry out functions within the human body.

Many diseases occur when these gene expressions go awry.

COVID-19, short for coronavirus disease 2019, is caused by the novel coronavirus SARS-CoV-2. Like many other viruses, SARS-CoV-2 is an RNA virus. This means that, unlike in humans and other mammals, the genetic material for SARS-CoV-2 is encoded in RNA. The viral RNA is sneaky: its features cause the protein synthesis machinery of our cells to mistake it for RNA produced by our own DNA.

While SARS-CoV-2 is a new coronavirus, it likely replicates and functions similar to related coronaviruses that infect animals and humans, says Douglas Anderson, an assistant professor of medicine in the Aab Cardiovascular Research Institute and a member of the Center for RNA Biology, who studies how RNA mutations can give rise to human disease.

A graphic created by the New York Times illustrates how the coronavirus that causes COVID-19 enters the body through the nose, mouth, or eyes and attaches to our cells. Once the virus is inside our cells, it releases its RNA. Our hijacked cells serve as virus factories, reading the viruss RNA and making long viral proteins to compromise the immune system. The virus assembles new copies of itself and spreads to more parts of the body andby way of saliva, sweat, and other bodily fluidsto other humans.

Once the virus is in our cells, the entire process of infection and re-infection depends on the viral RNA, Maquat says.

Researchers Douglas Anderson, Dragony Fu, and Lynne Maquat are among the scientists at the University of Rochester who study the RNA of viruses to better understand how RNAs work and how they are involved in diseases. (University of Rochester photos / Matt Wittmeyer / J. Adam Fenster)

Maquat has been studying RNA since 1972 and was part of the earliest wave of scientists to realize the important role RNA plays in human health and disease.

Our cells have a number of ways to combat viruses in what can be viewed as an arms race between host and virus. One of the weapons in our cells arsenal is an RNA surveillance mechanism Maquat discovered called nonsense-mediated mRNA decay (NMD).

Nonsense-mediated mRNA decay protects us from many genetic mutations that could cause disease if NMD was not active to destroy the RNA harboring the mutation, she says.

Maquats discovery has contributed to the development of drug therapies for genetic disorders such as cystic fibrosis, and may be useful in developing treatments for coronavirus.

NMD also helps us combat viral infections, which is why many viruses either inhibit or evade NMD, she adds. The genome of the virus COVID-19 is a positive-sense, single-stranded RNA. It is well known that other positive-sense, single-stranded RNA viruses evade NMD by having RNA structures that prevent NMD from degrading viral RNAs.

Maquats lab is currently collaborating with a lab at Harvard University to test how viral proteins can inhibit the NMD machinery.

Like Maquat, Fu studies fundamental aspects of RNAand has found that his research on proteins may, too, be applicable to coronavirus research.

Fus lab analyzes enzymes and proteins that modify the chemical structure of RNA and how these chemical modifications impact the function of RNA. A research group at the University of California, San Francisco, recently identified an interaction between a protein made by the SARS-CoV-9 virus and a protein Fu studies.

This is an intriguing result, and we are currently thinking of ways this interaction could affect the host cell, Fu says. There is emerging evidence that RNA-based viruses undergo RNA modification, so we could use this knowledge to identify key links between the host and pathogen for development of a coronavirus vaccine or treatment.

One of the reasons viruses are such a challenge is that they change and mutate in response to drugs.

Targeting viral RNA, or the proteins it produces, is key for treating this disease.

That means novel virus treatments and vaccines have to be created each time a new strain of virus presents itself. Armed with innovative research on the fundamentals of RNA, scientists are better able to develop and test therapeutics that directly target the RNAs and processes critical to a viruss life cycle.

The University of Rochester Medical Center, for instance, is currently participating in a clinical trial to evaluate the safety and efficacy of a potential coronavirus treatment called remdesivir, an antiviral drug particularly tailored to attack RNA viruses. The drug inhibits RNA polymerase, an enzyme responsible for copying a DNA sequence into an RNA sequence.

Anderson has found that alternative therapeutics, such as the gene-editing technology CRISPR, may additionally usher in a new approach to how we target and combat infectious diseases, he says.

For the past few years, Andersons lab has developed tools and delivery systems that use the RNA-targeting CRISPR-Cas13 to treat human genetic diseases that affect muscle function. CRISPR-Cas13 is like a molecular pair of scissors that can target specific RNAs for degradation, using small, programmable guide RNAs.

When the health crisis first became apparent in Wuhan, China, researchers in Andersons lab turned their focus toward developing a CRISPR-Cas13 therapeutic aimed at SARS-CoV-2. Applying the knowledge already available about coronavirus RNA replication, they designed single CRISPR guide RNAs capable of targeting every viral RNA that is made within a SARS-CoV-2 infected cell. Using a novel cloning method developed in Andersons lab, multiple CRISPR guide-RNAs could be packaged into a single therapeutic vector (a genetically engineered carrier) to target numerous viral RNA sites simultaneously. The multi-pronged targeting strategy could be used as a therapy to safeguard against virus-induced cell toxicity and prevent escape of viruses which may have undergone mutation.

Infectious viruses and pandemics seemingly come out of nowhere, which has made it hard to rapidly develop and screen traditional small molecule therapeutics or vaccines, Anderson says. There is a clear need to develop alternative targeted therapeutics, such as CRISPR-Cas13, which have the ability to be rapidly reprogrammed to target new emerging pandemics.

While many new treatments for the novel coronavirus are being considered, there is one thing that is certain, Maquat says: Targeting viral RNA, or the proteins it produces, is key for treating this disease.

Tags: Arts and Sciences, Center for RNA Biology, COVID-19, Department of Biochemistry and Biophysics, Department of Biology, Douglas Anderson, Dragony Fu, featured-post, Lynne Maquat, medical center

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COVID-19: What's RNA research got to do with it? - University of Rochester

Cryopreservation has become an indispensable step in the daily routine of scientific research as well as in a number of medical applications, ranging from assisted reproduction and transplantations to cell-based therapies and biomarker identification. It is hardly possible to picture todays scientific and medical advancements without this technique.The successful development and implementation of all the therapeutic and scientific discoveries involving cryopreservation relies on the correct and safe translation of the method from the laboratory to the clinical and manufacturing scale.

With the need to correctly use this technique, more research is focusing on optimizing cryopreservation methods and investigating what the long-term effects and consequences are on the physiology of the cryopreserved material.

An important part of cell therapy research is focused on adult stem cells (ASCs). ASCs can be derived from different sources such as peripheral blood, bone marrow or adipose tissue and display strong promises because of their capacity to differentiate into any cell type of the human body.In recent work3, the team of Michael Pepper at the Institute for Cellular and Molecular Medicine in Pretoria, South Africa, explored the effects of cryopreservation on the differentiation ability of adipose tissue-derived stem cells (ADSCs). After analyzing gene expression of key adipogenic genes and the degree of differentiating cells, characterized with high levels of CD36 and intracellular lipid droplets, the scientists reported that slow freeze cryopreservation of cells shortly after their isolation causes no alterations on their ability to differentiate. Pepper is convinced of the necessity to perform such analysis when cryopreserving important cell pools: It is critical to do a post-thaw analysis of cell function to determine how the cryopreservation may have affected the cells.His team is analyzing the effects of cryopreservation on other cell types largely used in cell-based therapies such as hematological stem cells and peripheral blood mononuclear cells (PBMCs). Although they didnt observe major alterations in terms of immunophenotyping or the post-thaw proliferation of the cells, Pepper expresses his concern that more subtle characteristics might be affected.

Correct cryopreservation of cells intended for therapeutic use is crucial. This is very important particularly as cells may persist for a long time in the recipient. This area of cell therapy research definitely requires more attention, Pepper says. Moreover, his words reflect on the need to evaluate not only the direct post-thaw recovery, but to look deeper into the late-onset effects cryopreservation might have and ensure that transplanted cells have preserved their therapeutic properties.

In contrast to slow freezing, vitrification relies on the fast freezing of the material by putting it in high concentration of cryoprotectant and in contact with liquid nitrogen. This method allows the direct transition of water from liquid to solid state without crystal formation. The highly concentrated cryoprotectant prevents ice formation and therefore there is no need for slow cooling.

Although vitrification has a great potential, there are a couple of parameters that are a point of concern. The quick and drastic freeze is possible thanks to the high concentration of cryoprotectant, but the latter is also associated with higher toxicity. In some cases, an additional limitation is the direct contact of the sample with liquid nitrogen which is a predisposition for viral or bacterial contamination.The team of Christiani Amorim at the Institute for Experimental and Clinical Research in Louvain, Belgium, is approaching the challenges of vitrification in the context of ovarian auto-transplantation. Ovarian auto-transplantation consists of preserving a piece of ovarian tissue with active follicles from the pre-therapeutic ovary of a cancer patient, as chemotherapy often has damaging effects on the reproductive organs. This tissue sample will be conserved and auto-transplanted onto the patients ovary when she has recovered and wishes to become pregnant.In their recent research4, the authors used stepped vitrification, in which the concentration of the cryoprotectant is gradually increased while simultaneously temperature decreases. This avoids ice crystal formation and also prevents cryoprotectant toxicity.Although stepped vitrification has previously given good results in bovine ovarian tissue5, this was not the case for human ovarian tissue. The scientists didnt detect normal follicles following thawing and linked this to high cryoprotectant toxicity. Indeed, they observed all signs of dimethyl sulfoxide (DMSO)-related cell membrane damage: significant organelle damage, cell membrane disintegration and apoptosis. These observations imply on the variability of outcomes that the method could give when applied to the same type of tissue but from a different organism.Amorim is positive about the future of their method and recognizes the need for further research on the topic: I can see a great potential in the stepped vitrification approach, but I also believe that there is a lot we still need to learn before thinking about using it as method of choice for human ovarian tissue cryopreservation. The high cryoprotectant concentration that should be applied in this approach is my first concern. () Our study clearly showed that 50% DMSO is too high, so we need to try lower concentrations or combine it with other cryoprotectants.

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Freezing Life: The Current Trends in Cryopreservation - Technology Networks

Ethiopia pardons more than 4,000 prisoners to help prevent coronavirus spread

Los Angeles offers free testing to all county residents

The Washington Post

All residents of Los Angeles County can access free coronavirus testing at city-run sites, Mayor Eric Garcetti (D) said on Wednesday. Previously, the city had only offered testing to residents with symptoms as well as essential workers and people who lived or worked in nursing homes and other kinds of institutional facilities. In an announcement on Twitter, Garcetti said that priority would still be given to front-line workers and anyone experiencing symptoms, including cough, fever or shortness of breath. But the move, which makes Los Angeles the first major city in the country to offer such widespread testing, allows individuals without symptoms to be tested. Health experts have repeatedly said that mass testing is necessary to determine how many people have contracted the virus and in particular, those who may not have experienced symptoms and then begin to reopen the economy. Testing is by appointment only and can be arranged at one of the citys 35 sites. Read more

Global coronavirus death toll surpasses 200,000, as world leaders commit to finding vaccine

By NBC News

The global coronavirus death toll surpassed 200,000 on Saturday, according to John Hopkins University data. The grim total was reached a day after presidents and prime ministers agreed to work together to develop new vaccines, tests and treatments at a virtual meeting with both the World Health Organization (WHO) and Bill & Melinda Gates Foundation. We will only halt COVID-19 through solidarity, said Dr. Tedros Adhanom Ghebreyesus, WHO Director-General. Countries, health partners, manufacturers, and the private sector must act together and ensure that the fruits of science and research can benefit everybody. As the U.S. coronavirus death tollpassed 51,000 people, according to an NBC News tally, President Donald Trump took no questions at his White House briefing on Friday, after widespread mockery for floating the idea that light, heat and disinfectants could be used to treat coronavirus patients.

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Germany to start first coronavirus vaccine trial

By DW

German Health Minister Jens Spahn has announced the first clinical trials of a coronavirus vaccine. The Paul Ehrlich Institute (PEI), the regulatory authority which helps develop and authorizes vaccines in Germany, has given the go-ahead for the first clinical trial of BNT162b1, a vaccine against the SARS-CoV-2 virus. It was developed by cancer researcher and immunologist Ugur Sahin and his team at pharmaceutical company BioNTech, and is based on their prior research into cancer immunology. Sahin previously taught at the University of Mainz before becoming the CEO of BioNTech. In a joint conference call on Wednesday with researchers from the Paul Ehrlich Institute, Sahin said BNT162b1 constitutes a so-called RNA vaccine. He explained that innocuous genetic information of the SARS-CoV-2 virus is transferred into human cells with the help of lipid nanoparticles, a non-viral gene delivery system. The cells then transform this genetic information into a protein, which should stimulate the bodys immune reaction to the novel coronavrius.

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Webinar on COVID-19 and Mental Health: Interview with Dr. Seble Frehywot

By Liben Eabisa | TADIAS

Dr. Seble Frehywot, an Associate Professor of Global Health & Health Policy at George Washington University in Washington, D.C. and her colleague Dr. Yianna Vovides from Georgetown University will host an online forum next week on April 30th focusing on the COVID-19 pandemic and its impact on mental health. Dr. Seble who is also the Director of Global Health Equity On-Line Learning at George Washington University told Tadias that the virtual conference titled Peoples Webinar: Addressing COVID-19 By Addressing Mental Health is open to the public and available for viewing worldwide. Read more

Young and middle-aged people, barely sick with covid-19, are dying from strokes

By The Washington Post

Doctors sound alarm about patients in their 30s and 40s left debilitated or dead. Some didnt even know they were infected. Read more

CDC director warns second wave of coronavirus is likely to be even more devastating

By The Washington Post

Even as states move ahead with plans to reopen their economies, the director of the Centers for Disease Control and Prevention warned Tuesday that a second wave of the novel coronavirus will be far more dire because it is likely to coincide with the start of flu season. Theres a possibility that the assault of the virus on our nation next winter will actually be even more difficult than the one we just went through, CDC Director Robert Redfield said in an interview with The Washington Post. And when Ive said this to others, they kind of put their head back, they dont understand what I meanWere going to have the flu epidemic and the coronavirus epidemic at the same time, he said. Having two simultaneous respiratory outbreaks would put unimaginable strain on the health-care system, he said. The first wave of covid-19, the disease caused by the coronavirus, has already killed more than 42,000 people across the country. It has overwhelmed hospitals and revealed gaping shortages in test kits, ventilators and protective equipment for health-care workers.

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Americans at World Health Organization transmitted real-time information about coronavirus to Trump administration

By The Washington Post

More than a dozen U.S. researchers, physicians and public health experts, many of them from the Centers for Disease Control and Prevention, were working full time at the Geneva headquarters of the World Health Organization as the novel coronavirus emerged late last year and transmitted real-time information about its discovery and spread in China to the Trump administration, according to U.S. and international officials. A number of CDC staff members are regularly detailed to work at the WHO in Geneva as part of a rotation that has operated for years. Senior Trump-appointed health officials also consulted regularly at the highest levels with the WHO as the crisis unfolded, the officials said. The presence of so many U.S. officials undercuts President Trumps assertion that the WHOs failure to communicate the extent of the threat, born of a desire to protect China, is largely responsible for the rapid spread of the virus in the United States. Read more

In Ethiopia, Dire Dawa Emerges as Newest Coronavirus Hot Spot

By Africa News

The case count as of April 20 had reached 111 according to health minister Lia Tadesses update for today. Ethiopia crossed the 100 mark over the weekend. All three cases recorded over the last 24-hours were recorded in the chartered city of Dire Dawa with patients between the ages of 11 18. Two of them had travel history from Djibouti. Till date, Ethiopia has 90 patients in treatment centers. The death toll is still at three with 16 recoveries. A patient is in intensive care. Read more

COVID-19: Interview with Dr. Tsion Firew, an Ethiopian Doctor on the Frontline in NYC

Dr. Tsion Firew is Doctor of Emergency Medicine and Assistant Professor at Columbia University. She is also Special Advisor to the Ministry of Health in Ethiopia. (Courtesy photo)

By Liben Eabisa

In New York City, which has now become the global epicenter of the coronavirus pandemic, working as a medical professional means literally going to a war zone, says physician Tsion Firew, a Doctor of Emergency Medicine and Assistant Professor at Columbia University, who has just recovered from COVID-19 and returned to work a few days ago. Indeed the statistics coming out of New York are simply shocking with the state recording a sharp increase in death toll this months surpassing 10,000 and growing. According to The New York Times: The numbers brought into clearer focus the staggering toll the virus has already taken on the largest city in the United States, where deserted streets are haunted by the near-constant howl of ambulance sirens. Far more people have died in New York City, on a per-capita basis, than in Italy the hardest-hit country in Europe. At the heart of the solution both in the U.S. and around the world is more testing and adhering to social distancing rules until such time as a proper treatment and vaccine is discovered, says Dr. Tsion, who is also a Special Advisor to the Ministry of Health in Ethiopia. Dr. Tsion adds that at this moment we all as humanity have one enemy: the virus. And whats going to win the fight is solidarity. Listen to the interview

Ethiopia Opens Aid Transport Hub to Fight Covid-19

By AFP

Ethiopia and the United Nations on Tuesday opened a humanitarian transport hub at Addis Ababa airport to move supplies and aid workers across Africa to fight coronavirus. The arrangement, which relies on cargo services provided by Ethiopian Airlines, could also partially offset heavy losses Africas largest carrier is sustaining because of the pandemic. An initial shipment of 3 000 cubic metres of supplies most of it personal protective equipment for health workers will be distributed within the next week, said Steven Were Omamo, Ethiopia country director for the World Food Programme (WFP). This is a really important platform in the response to Covid-19, because what it does is it allows us to move with speed and efficiency to respond to the needs as they are unfolding, Omamo said, referring to the disease caused by the coronavirus. The Addis gateway is one of eight global humanitarian hubs set up to facilitate movement of aid to fight Covid-19, according to WFP.

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Covid-19: Ethiopia to buy life insurance for health workers

By TESFA-ALEM TEKLE | AFP

The Ethiopian government is due to buy life insurance for health professionals in direct contact with Covid-19 patients. Health minister Lia Tadesse said on Tuesday that the government last week reached an agreement with the Ethiopian Insurance Corporation but did not disclose the value of the cover. The two sides are expected to sign an agreement this week to effect the insurance grant. According to the ministry, the life insurance grant is aimed at encouraging health experts who are the most vulnerable to the deadly coronavirus. Members of the Rapid Response Team will also benefit.

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U.N. says Saudi deportations of Ethiopian migrants risks spreading coronavirus

By Reuters

The United Nations said on Monday that deportations of illegal migrant workers by Saudi Arabia to Ethiopia risked spreading the coronavirus and it urged Riyadh to suspend the practice for the time being.

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Ethiopias capital launches door-to-door Covid-19 screening

Getty Images

By TESFA-ALEM TEKLE | AFP

Ethiopias capital, Addis Ababa is due to begin a door-to-door mass Covid-19 screening across the city, Addis Ababa city administration has announced. City deputy Mayor, Takele Uma, on Saturday told local journalists that the mass screening and testing programme will be started Monday (April 13) first in districts which are identified as potentially most vulnerable to the spread of the highly infectious coronavirus. The aggressive city-wide screening measure intends to identify Covid-19 infected patients and thereby to arrest a potential virus spread within communities. He said, the mass screening will eventually be carried out in all 117 districts, locally known as woredas, of the city, which is home to an estimated 7 million inhabitants. According to the Mayor, the door-to-door mass Covid-19 screening will be conducted by more than 1,200 retired health professionals, who responded to governments call on the retired to join the national fight against the coronavirus pandemic.

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Worldwide deaths from the coronavirus hit 100,000

By The Associated Press

The worldwide death toll from the coronavirus has hit 100,000, according to the running tally kept by Johns Hopkins University. The sad milestone comes as Christians around the globe mark a Good Friday unlike any other in front of computer screens instead of in church pews. Meanwhile, some countries are tiptoeing toward reopening segments of their battered economies. Public health officials are warning people against violating the social distancing rules over Easter and allowing the virus to flare up again. Authorities are using roadblocks and other means to discourage travel.

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Ethiopia COVID-19 Response Team: Interview with Mike Endale

By Liben Eabisa | TADIAS

A network of technology professionals from the Ethiopian Diaspora known as the Ethiopia COVID-19 Response Team has been assisting the Ethiopian Ministry of Health since the nations first Coronavirus case was confirmed on March 13th. The COVID-19 Response Team has since grown into an army of more than a thousand volunteers. Mike Endale, a software developer based in Washington, D.C., is the main person behind the launch of this project. Read more

Ethiopia eyes replicating Chinas successes in applying traditional medicine to contain COVID-19

By CGTN Africa

The Ethiopian government on Thursday expressed its keen interest to replicate Chinas positive experience in terms of effectively applying traditional Chinese medicine to successfully contain the spread of COVID-19 pandemic in the East African country.

This came after high-level officials from the Ethiopian Ministry of Innovation and Technology (MoIT) as well as the Ethiopian Ministry of Health (MoH) held a video conference with Traditional Chinese Medicine (TCM) practitioners and researchers on ways of applying the TCM therapy towards controlling the spread of coronavirus pandemic in the country, the MoIT disclosed in a statement issued on Thursday.

China, in particular, has agreed to provide to Ethiopia the two types of Chinese traditional medicines that the country applied to successfully treat the first two stages of the novel coronavirus, a statement from the Ethiopian Ministry of Innovation and Technology read.

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WHO Director Slams Racist Comments About COVID-19 Vaccine Testing

The Director General of the World Health Organization, Dr. Tedros Adhanom Ghebreyesus, has angrily condemned recent comments made by scientists suggesting that a vaccine for COVID-19 should be tested in Africa as racist and a hangover from the colonial mentality. (Photo: WHO)

By BBC

The head of the World Health Organization (WHO) has condemned as racist the comments by two French doctors who suggested a vaccine for the coronavirus could be tested in Africa.

Africa cant and wont be a testing ground for any vaccine, said Director General Dr Tedros Adhanom Ghebreyesus.

The doctors remarks during a TV debate sparked outrage, and they were accused of treating Africans like human guinea pigs.

One of them later issued an apology.

When asked about the doctors suggestion during the WHOs coronavirus briefing, Dr Tedros became visibly angry, calling it a hangover from the colonial mentality.

It was a disgrace, appalling, to hear during the 21st Century, to hear from scientists, that kind of remark. We condemn this in the strongest terms possible, and we assure you that this will not happen, he said.

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Ethiopia declares state of emergency to curb spread of COVID-19

By Reuters

Ethiopias prime minister, Abiy Ahmed, on Wednesday declared a state of emergency in the country to help curb the spread of the new coronavirus, his office said on Twitter. Considering the gravity of the #COVID19, the government of Ethiopia has enacted a State of Emergency, Abiys office said.

Ethiopia virus cases hit 52, 9-month-old baby infected

By TESFA-ALEM TEKLE | AFP

Ethiopia on Tuesday reported eight new Covid-19 cases, the highest number recorded so far in one day since the country confirmed its first virus case on March 12. Among the new patients that tested positive for the virus were a 9-month-old infant and his mother who had travelled to Dubai recently. During the past 24 hours, we have done laboratory tests for a total of 264 people and eight out of them have been diagnosed with coronavirus, raising the total confirmed number of Covid-19 patients in Ethiopia to 52, said Health Minister Dr Lia Tadese. According to the Minister, seven of the newly confirmed patients had travel histories to various countries. They have been under forced-quarantine in different designated hotels in the capital, Addis Ababa. Five of the new patients including the 9-month-old baby and the mother came from Dubai while the two others came from Thailand and the United Kingdom, she said

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The coronavirus is infecting and killing black Americans at an alarmingly high rate

By The Washington Post

As the novel coronavirus sweeps across the United States, it appears to be infecting and killing black Americans at a disproportionately high rate, according to a Washington Post analysis of early data from jurisdictions across the country. The emerging stark racial disparity led the surgeon general Tuesday to acknowledge in personal terms the increased risk for African Americans amid growing demands that public-health officials release more data on the race of those who are sick, hospitalized and dying of a contagion that has killed more than 12,000 people in the United States. A Post analysis of what data is available and census demographics shows that counties that are majority-black have three times the rate of infections and almost six times the rate of deaths as counties where white residents are in the majority.

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In China, Wuhans lockdown officially ends after 11 weeks

After 11 weeks or 76 days Wuhans lockdown is officially over. On Wednesday, Chinese authorities allowed residents to travel in and out of the besieged city where the coronavirus outbreak was first reported in December. Many remnants of the months-long lockdown, however, remain. Wuhans 11 million residents will be able to leave only after receiving official authorization that they are healthy and havent recently been in contact with a coronavirus patient. To do so, the Chinese government is making use of its mandatory smartphone application that, along with other government surveillance, tracks the movement and health status of every person.

Read more

U.S. hospitals facing severe shortages of equipment and staff, watchdog says

By The Washington Post

As the official U.S. death toll approached 10,000, U.S. Surgeon General Jerome M. Adams warned that this will be the hardest and saddest week of most Americans lives.

Read more

Ethio-American Tech Company PhantomALERT Offers Free App to Track & Map COVID-19 Outbreak

By Tadias Staff

PhantomALERT, a Washington D.C.-based technology company announced, that its offering a free application service to track, report and map COVID-19 outbreak hotspots in real time. In a recent letter to the DC government as well as the Ethiopian Embassy in the U.S. the Ethiopian-American owned business, which was launched in 2007, explained that over the past few days, they have redesigned their application to be a dedicated coronavirus mapping, reporting and tracking application. The letter to the Ethiopian Embassy, shared with Tadias, noted that PhantomALERTs technology will enable the Ethiopian government (and all other countries across the world) to locate symptomatic patients, provide medical assistance and alert communities of hotspots for the purpose of slowing down the spread of the Coronavirus.

Read more

2nd COVID-19 death confirmed in Ethiopia

By Dr. Lia Tadesse (Minister, Ministry of Health, Ethiopia)

It is with great sadness that I announce the second death of a patient from #COVID19 in Ethiopia. The patient was admitted on April 2nd and was under strict medical follow up in the Intensive Care Unit. My sincere condolences to the family and loved ones.

Read more

The Next Coronavirus Test Will Tell You If You Are Now Immune. And Its Fast.

People line up in their cars at the COVID-19 testing area at Roseland Community Hospital on April 3, 2020, in Chicago. (E. Jason Wambsgans / Chicago Tribune)

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LA Becomes First City in US to Offers Free COVID-19 Testing to All Residents at Tadias Magazine - Tadias Magazine

Mutations present in our DNA from birth can cause us to die at a younger age.

By Amanda HeidtApr. 24, 2020 , 12:40 PM

Scientists have discovered a handful of ultrarare mutations present in our cells from birth that likely shave years off a persons life. Each of these DNA variants, most likely inherited from our parents, can reduce life span by as much as 6 months, the researchers estimate. And different combinations can dictate how long people live before developing age-related diseases such as cancer, diabetes, and dementia.

A persons genes dont set a specific natural life spandiet and many other factors play large roles, toobut studies have shown that DNA variants can influence the aging process. Biologists chalk up less than one-third of that influence to the genes we inherit. The source of other age-influencing DNA variation is environmental: Sun damage, chemical exposure, and other insults that create thousands of random mutations. Each cells collection of these environmental mutations differs, and most dont greatly impact a persons life span.

Hunting for these rare mutations, which are found in less than one in every 10,000 people, required a group effort. Harvard University geneticist Vadim Gladyshev, a senior co-author in the new study, partnered with academic colleagues and a biotech company called Gero LLC to scour the UK Biobank, a public database containing the genotypes of about 500,000 volunteers.

Using more than 40,000 of these genotypes, the team looked for correlations between small changes in DNA and health conditions, a so-called genomewide association study. Specifically, the variants they were targeting knock out genes entirely, depriving all the cells in the body of certain proteins.

On average, each person is born with six ultrarare variants that can decrease life span and health span, the amount of time people live before developing serious diseases, the team reports this month in eLife. The more mutations, the more likely a person was to develop an age-related illness at a younger age or die. The exact combination matters, Gladyshev says, but in general, each mutation decreases life span by 6 months and health span by 2 months.

The results build on what is already known about aging: Family genes matter. But rather than studying the common mutations found in especially long-lived people, researchers can now target rarer variants present in everyone. Gladyshev hopes this information can be used in clinical trials to categorize participants by their mutations in addition to things like gender and actual age.

He admits the findings are potentially controversial, as they minimize the perceived contribution to aging of environmental somatic mutations acquired throughout life. Somatic mutations live in a larger universe of age-related changes influenced by lifestyle, he says, adding that changes to hormone and gene expression also come with age. They [all] contribute to the aging process, but on their own they do not cause it.

Jan Vijg, a geneticist at the Albert Einstein College of Medicine who studies the role of somatic mutations in aging, agrees, though he adds that somatic mutations can still cause diseases such as skin cancer that decrease life span.

Alexis Battle, a biomedical engineer at the Johns Hopkins University School of Medicine, points to an important caveat, however: The new research only looked at the exome, the 1% of the genome that actively builds the proteins that direct our cells. The rest is largely a black box, although growing evidence shows it can affect gene expression. Both Battle and Vijg agree that this DNA could be even more important in aging than the regions targeted by Gladyshev and his colleagues.

Going forward, Gladyshev would like to repeat his analysis on DNA from centenarians: those that live to be older than 100. Most of the previous research focused on what these people have that makes them long-lived, he says. But [we want to look at] the oppositeits what they dont have.

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Rare 'killer' mutations present at birth could be taking years off your life - Science Magazine

Global Gene Therapy for Inherited Genetic Disorders Market: Overview

Rapid advances in mammalian DNA sequencing technologies over the past several years have enabled the identification of the aberrant genes responsible for a vast spectrum of genetic disorders. Gene therapy as a novel approach inarguably holds profound potential in finding universal therapeutic alternatives to treating inherited genetic disorders. Gene therapy for inherited genetic disorders entails introducing a functional copy of the defective gene to make up for the missing function, and can be accomplished using in vivo or ex vivo gene transfer.

Gene therapy for inherited genetic disorders has generated groundswell of interest in the research fraternity in finding cure for or in treatment of Mendelian genetic error causing rare diseases. Particularly, gene therapy in recent years has held promising potential in the treatment of a range of recessive gene disorders most notably sickle cell anemia, hemophilia, muscular dystrophy, cystic fibrosis, and other monogenic disorders. The axes of developments in the gene therapy for inherited genetic disorders market have been in the U.S., Europe, China, and Australia.

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Global Gene Therapy for Inherited Genetic Disorders Market: Notable Developments

Growing body of clinical studies done on mice models have unrivalled troves of preclinical data, which bodes well for the effectiveness of gene therapy for inherited genetic disorders. New approaches in the gene therapy for inherited genetic disorders market are being adopted to bring progress in this direction. In this regard, Salmeterol, a medicine approved for asthma, has shone a new light. The vasodilator to be used along with gene therapy has shown potential in increasing the effectiveness of the therapy for Glycogen storage disease type II (Pompe disease).

A team of investigator led by the researcher at Duke University Medical School discussed the preclinical data recently at 2019 annual meeting of the American Society of Gene & Cell Therapy. The preclinical data showed that the Asthma medicine reduces the accumulation of toxic glycogen accumulated in lysosome. The researchers concluded that it holds potential as an adjunctive therapy, and building on that may pave way for novel approaches on gene therapy for inherited genetic disorders.

Efforts to translate the findings of clinical research on gene therapy for inherited disorders to make the therapy a part of standard treatment has caught momentum in recent times. In this regard, vectors containing non-viral vectors have attracted the attention of scientists. A team of researchers at Fred Hutchinson Cancer Research Center in 2019 found that gold nanoparticles enable them to deliver gene-editing tools to blood stem cells in lab models. This might, they opined, pave way for more practicaland accessiblegene therapies for inherited disorders, notably for treating life-threatening blood disorders. Gene therapies were mediated by CRISPR. In the coming years they hope to collaborate with companies with commercial interest to develop the therapy for patient populations.

Some of the bigplayerseyeing promising stakes in the gene therapy for inherited genetic disorders market areSpark Therapeutics Inc., Orchard Therapeutics, Novartis AG, bluebird bio Inc., and BioMarin Pharmaceutical.

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Global Gene Therapy for Inherited Genetic Disorders Market: Key Drivers

Since 2000, scores of clinical trials involving patients with inherited genetic disorders have raised hopes of the medical fraternity of the potential of gene therapies. Thus far, more than 5000 clinical trials on gene therapy have been conducted, especially for hard-to-treat diseases. Diseases such as inherited blindness and leukemia have seen the efficacy and safety of gene therapies. Advances in bioengineering are expected to invigorate pre-clinical pipelines. In the not-so-distant future, success of more protocols will catalyze the prospects of the gene therapy for inherited genetic disorders market.

Further, advances have been made in viral and non-viral vectors with the purpose of making gene transfer more efficient, thereby boosting the gene therapy for inherited genetic disorders market. Particularly, new approaches emerged with the aim of making vectors more powerful.

Global Gene Therapy for Inherited Genetic Disorders Market: Regional Assessment

On the regional front, Asia Pacific bears considerable potential in the gene therapy for inherited disorders market. Of note, numerous strategic alliances have shifted their focus on the region, particularly China. The North America market has also been rising at a promising pace, driven by several gene-therapy tools and related drugs in the final stages of clinical trials. Favorable reimbursement models has also encouraged research into the gene therapy for inherited disorders.

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Gene Therapy for Inherited Genetic Disorders Market Key Companies and Analysis Top Trends by 2028 Cole Reports - Cole of Duty

Easy-to-understand genetic counseling reports will lead to more appropriate follow-up and better health outcomes

NEW HAVEN, Conn., April 16, 2020 /PRNewswire-PRWeb/ -- My Gene Counsel, a digital health company that provides innovative genetic counseling solutions, today announced it is teaming up with UConn Health's Neag Comprehensive Cancer Center's Hereditary Cancer Program to ensure that UConn's patients who have undergone genetic testing for cancer predisposition have access to timely and accurate genetic counseling information through the online delivery of My Gene Counsel's Living Lab Reports. Together, My Gene Counsel and UConn Health will use state-of-the-art digital tools to efficiently communicate up-to-date genomic information that will drive an improved standard of care.

By supplementing its current genetic counseling services with My Gene Counsel's digital counseling tools, UConn Health will serve as a leader in this space with the ability to better support patients over time in the post-test period. Each incoming patient will receive an electronic Living Lab Report sponsored by UConn Health and the Linda Clemens Breast Cancer Foundation that is personalized to the individual's genetic test results and outlines the most frequently asked questions and answers on topics related to disease risk, medical management options, relevance to family, emotional support, and available resources. This information is supported by tools to enhance understanding, such as hover dictionary and graphics.

"Genetics can be overwhelming and confusing, so when a patient leaves my office, I worry about how much information they have retained," said Connor Linehan, MS, LGC, a board-certified genetic counselor for the Hereditary Cancer Program at UConn Health. "Our goal, in partnering with My Gene Counsel, is to increase patient understanding in the hopes that better comprehension equals appropriate medical follow-up and better health outcomes. The addition of a user-friendly genetic counseling report that patients can review before and after their appointment and over time will be invaluable to empower them to make informed decisions about their healthcare."

The Living Lab Reports are written and continuously updated by a network of top certified genetic counselors and medical experts and are vetted by patient advocates. In addition to delivering complex genomic information in a way that patients can understand, the reports will update and notify patients automatically by text and/or email as My Gene Counsel adds new information to reflect changes in disease risk, medical management options, variant reclassification, and relevant clinical trials.

"I encourage patients to contact us over the years as information about hereditary cancer changes over time," said Jennifer Stroop, MS, CGC, LGC, a board-certified genetic counselor for the Hereditary Cancer Program at UConn Health. "However, this is not always easy. We are very excited to now be able to offer our patients a reference tool with continuing updates and notifications. With these continued touchpoints for engagement and retention, we will be able to meet the expressed need to help our patients feel more connected and supported in the long term."

My Gene Counsel's wraparound solution, available at UConn in May 2020, will enable the responsible return of results, engage and update patients, and integrate data into UConn's health care system. Living Lab Reports will be personalized by gene and variant and provided for all results, whether testing is negative or identifies a variant of clinical or uncertain significance.

"We are excited to partner with UConn Health, a forward-thinking health system on the cutting-edge of hereditary cancer and precision medicine," said Ellen Matloff, MS, CGC, president and CEO of My Gene Counsel. "Their dedication to improving health through education, innovation, and patient-centered clinical care beyond the initial genetic test aligns seamlessly with our own ideals."

More than 600 people undergo genetic counseling and testing each year as part of the Neag Comprehensive Cancer Center's Hereditary Cancer Program, which is staffed by two genetic counselors. The expanding volume of patients and limited bandwidth led the team to proactively seek out a technical solution that could help solve the challenge of monitoring critical clinical updates and research and recontacting patients.

Story continues

"It is essential for UConn Health, as a major university center, to keep pace with the growing demand for up-to-date genomic information," said Susan Tanenbaum, MD, medical director of the Neag Comprehensive Cancer Center. "The integration with My Gene Counsel is a giant step towards UConn Health assuming a position of global leadership in genomics and personalized medicine."

About My Gene Counsel: My Gene Counsel bridges the gap between genetic testing and precision medicine by addressing one of the most critical pain points in the industry lack of accurate and timely genetic counseling information for patients and providers. Their Living Lab Reports deliver genetic counseling information that updates as new clinical information emerges, guidelines evolve, and genetic variants are reclassified. Founded by certified genetic counselors with 30+ years of clinical experience, My Gene Counsel empowers partners to efficiently deliver on the promise of precision medicine. For more information, visit http://www.mygenecounsel.com.

About UConn Health: UConn Health is Connecticut's only public academic medical center. Based on a 206-acre campus in Farmington, UConn Health has a three-part mission: research, teaching and patient care. Home to the UConn School of Medicine, School of Dental Medicine and UConn John Dempsey Hospital with nearly 5,000 employees supporting nearly 1,000 students, over 800,000 annual patient visits, and innovative scientific research contributing to the advancement of medicine. For more information, visit http://www.health.uconn.edu.

SOURCE My Gene Counsel

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My Gene Counsel Partners with UConn Health to Provide Expanded Genetic Counseling Services - Yahoo Finance

1. In this case study involving one generally well infant with coronavirus disease 2019 (COVID-19), various parts of the isolation room were found to be contaminated with PCR-detectable SARS-CoV-2 on day 2 of admission.

2. Despite close physical contact with the infant during feeding, all three items of personal protective equipment worn by the healthcare worker were found to be negative for the virus.

Evidence Rating Level: 4 (Below Average)

Study Rundown: Severe acute respiratory syndrome-coronavirus 2 (SARS-CoV-2) is suspected to spread primarily through droplets and direct contact, but it is unknown whether airborne and fomite transmission are also causes for concern. Additionally, there is no data available regarding the risk of transmission from asymptomatic or pauci-symptomatic infants and children. This case study of an asymptomatic 6-month-old infant with COVID-19 revealed that, by day 2 of admission, the bedding, cot rail, and table in the isolation room all carried detectable amounts of the virus. While the viral load of the bedding was higher than that of the railing, the table, situated one meter from the patient, was found to have nearly the same concentration of viral particles as the bedding. Because viral load in the environment ought to fall with increasing distance from the source for droplet transmission, this unusual finding supports the possibility of contamination via indirect contact when the healthcare worker transferred items such as baby formula and baby wipes between the patient and the table. While virus viability was not assessed, these results reaffirm the importance of hand hygiene and social distancing to prevent unwitting spread by asymptomatic or presymptomatic carriers.

Click here to read the study in Annals of Internal Medicine

Relevant Reading: A Well Infant With Coronavirus Disease 2019 With High Viral Load

In-Depth [case study]: In this case study, a 6-month-old male was admitted to KK Womens and Childrens Hospital in Singapore after both his parents developed fever and sore throat within three days of each other. Upon arrival, the infant was afebrile and in no respiratory distress, but real-time reverse transcription polymerase chain reaction (rRT-PCR) testing of a nasopharyngeal specimen confirmed COVID-19 infection with very high viral load and cycle threshold (Ct) values of 15.6 and 13.7 for the N gene and Orf1ab gene, respectively. Ct values of <36 were considered positive, with lower values corresponding to higher viral load. On day 2 of admission, nasopharynx Ct values for the N gene and Orf1ab gene were 18.8 and 18.6, respectively, while urine and stool samples continued to test negative. After the infant was carried and fed within a span of 15 minutes, synthetic fiber flocked swabs with Universal Transport Medium were run over nearly 100% of the infants bedding, the cot rail, and a table located 1 meter away from the bed as well as the healthcare workers face shield, N95 mask, and waterproof gown. All three PPE samples were found to be negative for SARS-nCov-2, but the RdRp gene Ct values for the bedding, rail, and table were 28.7, 33.3, and 29.7, respectively.

Image: PD

2020 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced without expressed written consent from 2 Minute Medicine, Inc. Inquire about licensing here. No article should be construed as medical advice and is not intended as such by the authors or by 2 Minute Medicine, Inc.

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SARS-CoV-2 detected on various surroundings of asymptomatic COVID-19 positive infant - 2 Minute Medicine

Today, a new personalized cancer treatment was approved for NHS use in England.

But this was no ordinary drug approval. Larotrectinib (Vitrakvi) is a highly innovative new treatment that, unlike most cancer drugs, is designed to target specific changes in cancer cells DNA rather than where the cancer is growing in the body.

This means patients with various types of cancer may be able to benefit.

Its the first drug licensed in Europe that works in this way. And its been called revolutionary by the head of NHS England, Simon Stevens.

But drugs like larotrectinib also pose unique and complex challenges for the NHS, which have been exacerbated by the COVID-19 pandemic.

The latest decision

Larotrectinib was originally rejected for funding on the NHS in England back in January. Since then, the manufacturer and NHS England have negotiated a new price for the drug, which has made it possible for it to go into the Cancer Drugs Fund (CDF).

Because the drug is so innovative, its been more challenging than usual for NICE (the body that recommends whether the NHS should pay for new medicines in England) to answer key questions about how the treatment should be used and how effective it is.

This uncertainty is why the drug was initially rejected. Its taken a lot of work from all sidesthe NHS, NICE and the drugs manufacturerto overcome these challenges.

But now the drugs been approved for the Cancer Drugs Fund, the NHS will pay for it for a time-limited period, while more data is collectedincluding from NHS patientsto help resolve these questions. And if this data shows the drug is cost effective for the NHS, it will become available on a permanent basis.

Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, said: These cutting-edge therapies can be used to treat tumors with often rare genetic mutations regardless of where in the body the tumor originated.

The clinical evidence is usually based on extremely small sample sizes, requiring novel approaches to testing them in clinical trials and translation into models of assessment for potential value in NHS practice.

Drugs in the CDF are also usually made available to patients in Wales and Northern Ireland, through bespoke funding routes in those nations. Scotland has a separate system for appraising new drugs and larotrectinib hasnt been considered there yet.

Why it matters

Larotrectinib will be used to treat people whose tumors test positive for a particular genetic change, called an NTRK fusion, and who have run out of other treatment options.

This is hugely significant for these patients, but according to Professor Peter Johnson, national clinical director for cancer at NHS England, its also a great example of how the NHS can bring to bear its recent investment in genomic (genetic) testing in England to improve cancer treatment.

Johnson says work in this area has been building at a national level, ever since Cancer Research UK started our Stratified Medicine programme in 2010.

And while the NHS has offered genomic tests to help guide decisions on patients treatment for many years, NHS England has been working to coordinate and expand genomic testing services in seven hubs in England. This aims to make access to these tests more consistent across the country and help to speed up the introduction of new targeted drugs like larotrectinib in the future.

According to Johnson, whats come together are the development of the genomic laboratory hubs across England, and the first targeted drugs coming through from research that are effective across multiple tumor types. This is an exciting convergence of these two strands of work.

Testing challenges in rollout

But despite this convergence, there are still challenges to overcome before larotrectinib will be available to patients. And as weve previously explained, chief among these is making sure the NHS can test patients appropriately to see who might benefit from the drug.

The NHS intends to test up to 100,000 patients a year for this genetic change eventually. But its not in a position to make the test that widely available yet.

To balance this, its going to stagger the roll out of larotrectinib, beginning with people with rare cancers where the genetic change is most common, and children and young people.

According to NICE, between 600700 people in England have solid tumors with NTRK gene fusions. And a proportion of these people with no satisfactory treatment options will be eligible for treatment within the first year that its available on the CDF.

The impact of coronavirus

Rolling out a treatment like larotrectinib would be challenging at the best of times, but the COVID-19 pandemic makes things even more complicated and means it will take longer to scale up testing to the wider population.

Because of the crisis equipment and people have been diverted from Genomic Laboratory Hubs to support testing for coronavirus, says Johnson.

As soon as we can, we will introduce the capacity to continue rolling this out in a phased way to a much wider population of people for whom conventional treatment has not been successful.

And while the NHS is under a huge amount of strain with COVID-19, Johnson believes its vital that the NHS continues to assess and introduce new treatments.

We will get through the coronavirus crisis and are planning to put cancer services back on a firm footing in the future. Having new therapies coming through and the diagnostics to find out who could benefit is as important as it has ever been.

Provided byCancer Research UK

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What does the new personalized cancer medicine approved in England mean for patients?. - Brinkwire

DUBLIN, Aug. 10, 2020 /PRNewswire/ -- The "Cell & Gene Therapy Market - Global Outlook and Forecast 2020-2025" report has been added to ResearchAndMarkets.com's offering.

In-depth Analysis and Data-driven Insights on the Impact of COVID-19 Included

The study considers the present scenario of the cell and gene therapy market and its market dynamics for the period 2019-2025. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market.

Key Questions Answered

1. What is the cell and gene therapy market size and growth rate during the forecast period?2. What are the factors impacting the growth of the cell and gene therapy market share?3. How is the growth of the healthcare segment affecting the growth of the cell and gene therapy market?4. Who are the leading vendors in the cell and gene therapy market, and what are their market shares?5. Which product type/ end-user type/region is generating the largest revenue in the Asia-Pacific region?

The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.9% during the period 2019-2025

The global cell and gene therapy market is one of the fastest-growing segments in the regenerative medicine market. The market is expected to grow at a faster pace during the forecast period. The demand can be attributed to the growing prevalence of several chronic diseases such as cancer, cartilage related problems, wounds, diabetic foot ulcer, genetic disorders, and other rare diseases across the globe.

The prevalence of cancer and diabetes is increasing in the global population, which is influencing the growth of the market. There is a large unmet need in the treatment available, which is filled by cell and gene therapies. The market is growing due to the increased availability of funding from various public and private institutions. Besides, there is increased support from regulatory bodies for product approval. Several governments are creating awareness of cell and gene therapies in the population.

Cell and Gene Therapy Market Segmentation

The global cell and gene therapy market research report includes a detailed segmentation by product, disease, end-user, and geography.

In 2019, the cell therapy segment accounted for a market share of over 53% in the global cell and gene therapy market. The segment is expected to grow at a steady rate during the forecast period due to the increase in the target population and the rise in the number of countries preferring cell therapies in their patients. Increased therapeutic benefits are attracting several countries to invest in this technology and conduct a high number of clinical trials. However, the lack of advanced infrastructure in developing countries is hindering the growth of the segment.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

Segmentation by Product

Segmentation by Disease

Segmentation by End-user

Insights by Geography

In 2019, North America accounted for a share of over 60% of the global cell and gene therapy market. There are more than 530 regenerative medicine companies, including cell and gene therapy manufacturing developers. The number of products approved in North America grew significantly in 2019, with developers filed for marketing authorization for 10+ regenerative medicines, many of which we expect to be approved in 2020. Within the next 1-2 years, the number of approved gene therapies is expected to double.

The US and Canada are the major contributors to the cell and gene therapy market in North America. Regulatory bodies are supporting several investigational products, fast track approvals, RMAT designation for the faster approval of the product into the market. The alliance for regenerative medicine and Medicare and Medicaid is working together to bring the structured reimbursement channels for cell and gene therapies.

Segmentation by Geography

Insights by Vendors

The global cell and gene therapy market is highly dynamic and characterized by the presence of several global, regional, and local vendors offering a wide range of therapies. Dendreon, Gilead Sciences, Novartis, Organogenesis, Osiris Therapeutics, Vericel, Amgen, and Spark Therapeutics are the leading players in the market with significant shares.

Vendors such as NuVasive, APAC Biotech, Nipro, Orthocell, bluebird bio, J-TEC, and Terumo are the other prominent players in the market with a presence, especially in the cell therapy market. Most leading players are focusing on implementing strategies such as product launches and approvals, marketing and promotional activities, acquisitions, increased R&D investments, and strengthening their distribution networks to enhance their share and presence in the market.

Prominent Vendors

Other Prominent Vendors

Market Dynamics

Opportunities & Trends

Growth Enablers

Growth Restraints

For more information about this report visit https://www.researchandmarkets.com/r/is5t27

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Research and Markets Laura Wood, Senior Manager [emailprotected]

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Global Cell and Gene Therapy Market, Forecast to 2025 by Product, Disease, End-user and Region - COVID-19 Updated - PRNewswire

LEXINGTON, Mass., Aug. 10, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio or the Company), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, today reported financial results for the quarter ended June 30, 2020, provided a business update and announced the U.S. Food and Drug Administration (FDA) has cleared the Companys Investigational New Drug (IND) application for LB-001 for the treatment of methylmalonic acidemia in pediatric patients. LogicBio released a separate press release this morning providing further details on the planned Phase 1/2 clinical design for LB-001.

We are thrilled to have received clearance to move forward with this first-in-human clinical trial with our lead product candidate, LB-001, for the treatment of methylmalonic acidemia, a life-threatening congenital genetic disease with no current therapeutic treatment options. This represents a significant milestone in our goal of bringing a treatment to MMA patients as well as for our GeneRide platform. We have maintained continuous dialogue with the centers of excellence that are planned to participate in the Phase 1/2 clinical trial, and we look forward to activating these sites as quickly as possible, said Fred Chereau, CEO of LogicBio. We have instituted systems attempting to mitigate COVID-19 dynamics on our study start-up process and, based on our best estimates, we plan to enroll our first patient in early 2021.

Commenting on the Next Generation Capsid Program, Mr. Chereau said, We are very excited about the recent advances in our novel capsid program, which has generated liver-tropic capsids intended for use in gene editing technologies such as GeneRide and other gene therapy approaches. We are focused on executing across all of our programs and look forward to sharing further details on our novel capsids in early 2021.

Appointment of Daniel Gruskin, M.D. to SVP, Head of Clinical Development

Daniel Gruskin, M.D. was appointed as SVP, head of clinical development in August 2020. Dr. Gruskin has served as interim head of clinical development of LogicBio since June 2020. In April 2020, Dr. Gruskin started consulting with the Company as a special advisor. Previously, Dr. Gruskin served in roles of increasing responsibility at Sanofi Genzyme, most recently as vice president, head of global medical affairs, rare disease, in which capacity he oversaw medical affairs, life cycle management, scientific affairs and other medical and development activities related to metabolic, rare and/or genetic diseases. Prior to his role at Sanofi Genzyme, Dr. Gruskin served as assistant professor, human genetics and pediatrics at Emory University School of Medicine, where he was also the chief of the genetics section at Childrens Healthcare of Atlanta.

Daniel has been instrumental in leading LB-001 clinical development efforts including getting the IND cleared. His deep experience in genetic medicines and metabolic diseases will serve LogicBio well as we look to execute on our goals for both the GeneRide and Next Generation Capsid platforms in search of transformative medicines, said Mr. Chereau.

Anticipated Milestones for 2020 and 2021:

Second Quarter 2020 Financial Results

Three Months Ended June 30, 2020 and 2019

About LogicBio Therapeutics

LogicBio Therapeuticsis dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms.

LogicBios proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration with Takeda to research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.

LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples. Top-tier capsid candidates from this effort demonstrated significant improvements over benchmark AAVs currently in clinical development. LogicBio is developing these highly potent vectors for internal development candidates and potentially for business development collaborations.

LogicBio is headquartered inLexington, Mass. For more information, please visitwww.logicbio.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the federal securities laws, including those related to the Companys plans to initiate, advance and complete its planned SUNRISE Phase 1/2 clinical trial of LB-001 in MMA; the timing, progress and results of the Companys research and development activities, including those related to the GeneRide technology platform and Next Generation Capsid Program; its plans for LB-301 in Crigler-Najjar; and the sufficiency of its cash and cash equivalents to fund operating expenses and capital expenditure requirements. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. In particular, the impact of the COVID-19 pandemic on the Companys ability to progress with its research, development, manufacturing and regulatory efforts, including the Companys plans to initiate, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Companys common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the United States and in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on March 16, 2020 with the SEC, the Companys Quarterly Report on Form 10-Q filed on May 11, 2020, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Contacts:

Investors:Brian LuqueAssociate Director, Investor Relationsbluque@logicbio.com951-206-1200

Media:Stephanie SimonTen Bridge CommunicationsStephanie@tenbridgecommunications.com617-581-9333

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LogicBio Therapeutics Reports Second Quarter 2020 Financial Results and Provides Business UpdatesFDA Clears IND Application for LB-001 for the...

About a month ago, Medical News Today started a series aiming to bring together the more encouraging research that emerges around COVID-19. We continue with this Special Feature that focuses on an incoming vaccine and other potential treatments for this new coronavirus and the disease it causes.

With this series, we aim to remind our readers that while COVID-19 causes great sorrow and loss around the world, the resulting global emergency has also meant that scientists are working at an unprecedented pace. They are making progress that is easy to overlook among the worrying numbers of new cases and deaths.

Two recent MNT articles COVID-19: 5 reasons to be cautiously hopeful and COVID-19: Physical distancing, drug trials offer hope looked at the latest developments in potential treatments, vaccines, and the outcomes of infection control measures during the pandemic.

We continue our series with this third Special Feature, which continues to monitor progress in the areas mentioned above.

Stay informed with live updates on the current COVID-19 outbreak and visit our coronavirus hub for more advice on prevention and treatment.

We focus on a vaccine that some researchers believe may be available by the fall and round up expert opinions on this promising development. We also cover an app-based social tracing system that could help create intelligent physical distancing instead of national lockdowns.

We previously reported that the World Health Organization (WHO) have launched a global megatrial that involves testing four potential treatments for COVID-19. Remdesivir, initially developed to treat Ebola, was one of those four potential treatments.

Now, scientists from the University of Alberta in Edmonton, Canada, say that remdesivir is showing promise in in vitro experiments.

The same team had previously demonstrated that remdesivir effectively combatted another coronavirus, MERS-CoV. It did so by blocking polymerases, which are enzymes that allow the virus to replicate.

Study co-author Prof. Matthias Gtte explains, If you target the polymerase, the virus cannot spread, so its a very logical target for treatment.

He continues to report the results of the teams new experiments: We obtained almost identical results as we reported previously with MERS, so we see that remdesivir is a very potent inhibitor for coronavirus polymerases.

Prof. Gtte goes on to explain, These coronavirus polymerases are sloppy, and they get fooled, so the inhibitor gets incorporated many times, and the virus can no longer replicate.

Still, the author cautions, Weve got to be patient and wait for the results of the randomized clinical trials.

Another hopeful finding comes from researchers from Cornell University in Ithaca, NY. These scientists also started their research efforts by drawing parallels with other coronaviruses, such as SARS-CoV and MERS-CoV.

Namely, they looked at the spike protein that coronaviruses have and zoomed in further on the fusion peptides these are short-chain amino acids that the spike proteins contain.

Whats really interesting about SARS-CoV and MERS-CoV, and this new virus, SARS-CoV-2, is this particular part of the protein, the fusion peptide, is almost exactly the same in those three viruses, explains study co-author Prof. Susan Daniel.

The new study found that calcium ions enable fusion peptides to help coronaviruses penetrate healthy cells through a process called membrane fusion. This offers a potential target for a new antiviral treatment.

The team has already secured funding to start developing an antibody that could stop this process by targeting SARS-CoV-2s fusion peptide.

Blocking the fusion step is significant because the fusion machinery doesnt evolve and change as fast as other parts of the protein do. Its been built to do a particular thing, which is to merge these two membranes together. So if you can develop antiviral strategies to reduce that efficiency, you could have potentially very broadly-acting treatments.

Prof. Susan Daniel

Sarah Gilbert, a professor of vaccinology at Oxford Universitys Jenner Institute in the United Kingdom, and her team may soon be closing in on a vaccine for SARS-CoV-2.

The approach uses a harmless chimpanzee virus to carry the fragment of SARS-CoV-2 that is required for immunity, explains Ian Jones, Professor of virology at the University of Reading, U.K.

Colin Butter, an associate professor of bioveterinary science at the University of Lincoln in the UK, explains: Professor Gilberts team [] have made a recombinant vaccine against the SARS-CoV-2 virus by taking a virus that is entirely harmless to humans, the Chimp Adenovirus designated ChAdOx1, and inserting into it the spike protein gene from the [new] coronavirus.

Prof. Gilbert believes that the vaccine will be available for general use by the fall, which could prevent a potential second wave of the new coronavirus.

That is just about possible if everything goes perfectly, Prof. Gilbert told The Times in an interview. The researchers are set to put the new vaccine into human trials in the next 2 weeks.

The researcher explains that during the pandemic, scientists can fast-track the process through which the vaccine reaches the population by doing many of the necessary steps in parallel.

First, there is the need to manufacture the vaccine for clinical studies under tightly controlled conditions, certified and qualified we need ethical approval and regulatory approval. Then, the clinical trial can start with 500 people in phase I.

This is always in healthy adults aged about 18 to 55, and usually the primary read-out from a phase 1 study is safety, Prof. Gilbert explains. Then we can do phase 2, looking at a wider age range; in this case, we are going to increase the age range, 55 to 70 plus. We are looking at safety in the older age group, [and] we expect to see weaker immune responses.

The researcher explains that she and her team plan to spread their studies across different countries so that they can reduce the time it takes to test the vaccine.

[I]ts vital we go fast before a high proportion [of the population] become infected. But it also means we are going to need to do studies in different countries because the amount of virus transmission is affected by the lockdowns.

The vaccine could get approval under emergency use legislation, meaning that in an emergency situation, if the regulators agree, its possible to use a vaccine earlier than in normal circumstances, Prof. Gilbert adds.

It is worth noting that other experts have expressed concern over Prof. Gilberts estimates.

Prof. David Salisbury, for example, says, [I]t is not just the availability of the first dose that we need to focus on. We need to know by when there will be sufficient doses to protect all of the at-risk population, probably with two doses, and that means industrial-scale manufacturing that governments do not have.

The approach in itself, however, is viable, and the research group benefits from a lot of credibility in the scientific community. The approach has been extensively tested in other situations, so there is indeed a good chance it will work as designed, says Prof. Jones.

The [research] group has a long history of success in this area, adds Dr. Butter. On the basis of this prior experience, it would be reasonable to assume that the vaccine would induce antibody and cellular immune responses, both of which may be important in controlling the virus in an individual.

Any final roll-out will almost certainly need a level of manufacturing the [U.K.] does not readily have, so transfer to and liaison with an external manufacturer may also need to be tackled. But the roadmap is clear, lets hope they get there.

Prof. Ian Jones

Tissue plasminogen activator (tPA) is a drug designed and approved to prevent blood clots in people who have had a stroke, pulmonary embolism, or heart attack.

Now, a new trial to test its benefits for relieving acute respiratory distress syndrome (ARDS) in people with COVID-19 is underway.

TPA acts as an anticoagulant. This means it prevents blood clotting by breaking down fibrin. Fibrin can form plugs in the airways and contributes to small clots in the blood vessels of the lungs.

In patients with COVID-19, these small microfibrin plugs in the air sacs lead to ARDS. As a result, these patients require ventilators to be able to breathe.

Were hearing anecdotally that a subset of patients with COVID-19-induced ARDS are clotting abnormally around their catheters and [intravenous] lines, explains Dr. Michael B. Yaffe, Ph.D., an acute care surgeon at the Beth Israel Deaconess Medical Center (BIDMC) in Boston, MA.

Dr. Yaffe is also the senior author of the study that proposed repurposing tPA to treat COVID-19 complications.

We suspect these patients with aggressive clotting will show the most benefit from tPA treatment, and this new clinical trial will reveal whether thats the case, says Dr. Yaffe.

The scientists have started to recruit some of the COVID-19 patients admitted to the BIDMC for the trial. The team also hopes to find biomarkers that can help identify patients who are most likely to benefit from the treatment.

If effective and safe for the treatment of ARDS in patients with COVID-19, tPA could save lives by reducing recovery time and freeing up more ventilators for other patients in need.

Christopher D. Barrett, clinical trial investigator

Another finding that may help relieve the pressure on public health systems is a mobile app-based contact tracing system.

The authors of the new project explain that such a system could help reduce the rate at which the virus spreads while also mitigating some harmful effects of a full national lockdown.

Dr. David Bonsall senior researcher at Oxford Universitys Nuffield Department of Medicine, clinician at Oxfords John Radcliffe Hospital both in the UK, and co-lead of the project explains how the system works.

He says, The mobile app concept weve mathematically modeled is simple and doesnt need to track your location. It uses a low energy version of Bluetooth to log a memory of all the app users with whom you have come into close proximity over the last few days.

If you then [contract the virus], these people are alerted instantly and anonymously and advised to go home and self-isolate. If app users decide to share additional data, they could support health services to identify trends and target interventions to reach those most in need.

The findings could pave the way for intelligent [physical] distancing, avoiding the social and economic effects of full lockdowns.

For live updates on the latest developments regarding the novel coronavirus and COVID-19, click here.

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COVID-19: Vaccine may be ready by fall and other reasons for hope - Medical News Today

$26.5 million total near-term payments to BridgeBio, plus participation in long-term value creation of up to $505 million in milestone payments, tiered double-digit royalty payments and an equity interest in LianBio.

BridgeBio CEO and founder Neil Kumar, Ph.D., has been appointed to LianBios board of directors.

PALO ALTO, Calif., Aug. 11, 2020 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (NASDAQ: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases and cancers with clear genetic drivers, today announced that it is partnering with Shanghai-based LianBio, a new company founded by Perceptive Advisors, to expand its global reach into China, the second-largest pharmaceutical market in the world. The partnership marks the first major expansion of BridgeBios pipeline into Asian markets.

This strategic relationship will initially focus on two of BridgeBios targeted oncology drug candidates, FGFR inhibitor infigratinib, currently in Phase 3 development for FGFR-driven tumors and Phase 1-ready SHP2 inhibitor BBP-398, for tumors driven by RAS and receptor tyrosine kinase mutations. The agreement also provides LianBio with preferential future access in the territory to more than 20 drug development candidates currently owned or controlled by BridgeBio. This collaboration is designed to advance and accelerate BridgeBios programs in China and other major Asian markets, allowing BridgeBio to quickly bring innovation to large numbers of patients with high unmet need.

Tremendous patient need and a fast-developing healthcare infrastructure make China a strategic priority. We are eager to not only expand late-stage therapies to the broader patient population there, but also to accelerate our clinical development efforts in Asia and better understand and address the needs of patients there early. We are grateful to be deepening our relationship with Perceptive Advisors through this agreement with LianBio and look forward to a lasting partnership focused on expanding our reach to patients, said BridgeBio CEO and founder Neil Kumar, Ph.D.

We value our relationship with BridgeBio and are happy to be enabling the entry of important programs to LianBios territories, said Adam Stone, CIO of Perceptive Advisors. BridgeBio and its affiliate companies exemplify the commitment to science-driven, precision medicine that we believe is a key driver to innovation in healthcare. We are excited about this opportunity to leverage their promising pipeline and LianBios local expertise to accelerate both global development and local access to leading edge therapeutics.

Under the terms of the agreements, LianBio will receive commercial rights in China and selected Asian markets and participate in clinical development activities for infigratinib (housed in BridgeBio affiliate QED) and BBP-398 (housed in BridgeBio affiliate Navire). BridgeBios near-term economics includes a total of $26.5 million in upfront and milestone payments. BridgeBio will receive up to $505 million in future milestone payments, tiered royalty payments from single- to double-digits on net sales of both products in licensed territories. Additionally, BridgeBio will increase its equity interest via investment in LianBio and BridgeBio CEO Neil Kumar has been appointed to the LianBio board of directors.

LianBio is participating in the ongoing Phase 3 study of infigratinib in first line cholangiocarcinoma (PROOF) in mainland China and further plans to initiate a Phase 2a study of infigratinib in gastric cancer and other FGFR-driven tumors. Additionally, LianBio will contribute to clinical development of BBP-398 in combination with various agents in solid tumors such as non-small cell lung cancer (NSCLC), colorectal and pancreatic cancer, in mainland China and other major Asian markets.

About BridgeBio PharmaBridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBios pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, please visitwww.bridgebio.com.

About LianBio

LianBios mission is to catalyze the development and accelerate availability of paradigm-shifting medicines to patients in China and major Asian markets through partnerships that provide access to the best science-driven therapeutic discoveries. LianBio collaborates with world-class partners across a diverse array of therapeutic and geographic areas to build out a pipeline based on disease relevance and the ability to impact patients with transformative mechanisms and precision-based therapeutics. For more information, please visit http://www.lianbio.com.

About Perceptive Advisors

Founded in 1999, Perceptive Advisors is a leading life sciences focused investment firm with over $7billion of regulatory assets under management as of June 30, 2020. Since inception, Perceptive Advisors has focused on supporting progress in the life sciences industry by identifying opportunities and directing financial resources toward the most promising technologies in modern healthcare. For more information, please visitwww.perceptivelife.com.

About QED Therapeutics

QED Therapeutics, an affiliate of BridgeBio Pharma, is a biotechnology company focused on precision medicine for FGFR-driven diseases. Its lead investigational candidate is infigratinib (BGJ398), an orally administered, FGFR1-3 selective tyrosine kinase inhibitor that has shown activity that it believes to be meaningful in clinical measures, such as overall response rate, in patients with chemotherapy-refractory cholangiocarcinoma with FGFR2 fusions and advanced urothelial carcinoma with FGFR3 genomic alterations. QED intends to submit a New Drug Application (NDA) with the United States Food and Drug Administration for second and later-line cholangiocarcinoma in 2020. QED Therapeutics is also evaluating infigratinib in clinical studies for the treatment of achondroplasia. QED plans to conduct further clinical trials to evaluate the potential for infigratinib to treat patients with other FGFR-driven tumor types and rare disorders. For more information, please visit http://www.qedtx.com.

About Navire Pharma

Navire Pharma, an affiliate of BridgeBio Pharma, and in collaboration with the Institute for Applied Cancer Science at MD Anderson, is developing inhibitors of SHP2 as targeted therapeutics for the treatment of multiple cancers. Together with patients and physicians, the company aims to bring safe, effective treatments to market as quickly as possible. For more information, please visit http://www.navirepharma.com.

BridgeBio Pharma Forward-Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as anticipates, believes, estimates, expects, intends, may, plans, projects, seeks, should, will, and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to BridgeBios anticipated receipt of future milestone and/or royalty payments from LianBio, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, the success of clinical trials, regulatory filings, approvals and/or sales of infigratinib and BBP-398 in China and other major Asian markets, as well as those risks set forth in the Risk Factors section of BridgeBio Pharmas most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q and BridgeBio Pharmas other SEC filings. Moreover, BridgeBio Pharma operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Contact:Grace RauhBridgeBio Pharma, Inc.Grace.rauh@bridgebio.com(917) 232-5478

Source: BridgeBio Pharma, Inc.

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BridgeBio Pharma Expands Reach Into China and Other Major Asian Markets Through Strategic Collaboration With Perceptive Advisors-Founded Company,...

Patients can pick from a growing array of treatments, from same-day- results surgery to game-changing immunotherapy, to fight the most common type of cancer.

BY By Beth Fand Incollingo and Kristie L. Kahl

In an interview with CURE, Dr. Jeremy Brauer, a dermatologist at NYU Langone Health in New York City and a spokesperson for the Skin Cancer Foundation, reviewed the latest strategies for treating these cancers, which include squamous cell and basal cell carcinomas, as well as the less common but more aggressive melanoma.

CURE: What surgical strategies are used to treat skin cancers?

Brauer: The intervention with the highest cure rate for surgical procedures is Mohs micrographic surgery. This is ideal for nonmelanoma skin cancers specifically, but there also is growing interest and use in certain melanomas, depending on the skin cancer and its location.

(Tissue is removed and tested a little at a time until the area being treated is free of cancerous cells).

Theres no leaving the office and waiting a week for your results; everything is done on-site in real-time. We process the tissue, and a Mohs surgeon also functions as the pathologist to read the slides and map out where the tumor is or isnt. The benefit of this is that it allows for tissue conservation, (especially on) the tip of the nose and ears. Also, it has a higher cure rate with the good cosmetic outcome because you are sparing tissue.

Another surgical intervention is a standard excision. You excise, take a big piece of skin and put stitches in, just like you would with Mohs, but its not a staged procedure. In general, its just one procedure.

Could you describe some of the nonsurgical therapies?

Nonsurgical treatments include electrodesiccation and curettage. These tend to be reserved for individuals who have a superficial basal cell carcinoma or what we call in situ squamous cell carcinoma, where the lesion is very superficial and on the uppermost part of the skin. Here, we use a sharp tool called a curette to scrape the area of involvement, but we also use electrodesiccation to burn the surrounding skin. This is often repeated in series a few times to remove the majority, if not all, of the tumor.

The goal here is to avoid having to cut and sew. The drawback is that you cant evaluate the tissue under the microscope because youre scraping and burning the remaining tumor cells.Another nonsurgical option is radiation therapy. This is sometimes used in conjunction with surgical treatment if it is determined that the subtype of skin cancer warrants it.

If involvement of the nerves is found during the course of Mohs surgery or when the specimen comes back after incision, we refer the individual to receive concurrent radiation therapy.

Radiation therapy is also good for (skin cancer that is not being treated with surgery). In certain instances, the individual might not able to tolerate (surgery) or declines the procedure. Similarly, certain tumors that dont heal well may be better candidates for a nonsurgical option.

Which medical options do you consider the most exciting?

I really do believe immunotherapy and targeted therapies for metastatic disease have been game changers. We consider immunotherapy an option for melanoma. Its been a game changer for ... survival in advanced cancers. That has definitely prolonged life for many individuals who unfortunately otherwise would not have fared as well.

Another interesting and promising area is targeted therapy. Here, were looking at the identification within the tumor of a mutation, specifically in a gene or pathway, and then targeting that gene or pathway (with medication). This has also led to increased survival rates and really allowed for a change in the way we approach some of the more advanced tumors.

That said, early intervention is key. Once weve detected these skin cancers, early intervention results in very high cure rates and, hopefully, prevents some of these local tumors from becoming metastatic or advanced.

What is on the horizon for patients with skin cancer?

Patients can be encouraged by the fact that medicine, technology and innovation are all moving at a very fast pace. More immunotherapy and targeted therapies will be made available to individuals with metastatic melanoma and advanced squamous cell carcinoma.

Also, right now, a biopsy is invasive. There is numbing and taking a blade to the skin. But there are other imaging techniques, and as our ability to detect skin cancer becomes better and greater, well begin to see additional noninvasive biopsy techniques.

How can patients become empowered to be their own best advocates when making treatment decisions?

It starts with education. Knowledge is power. A great resource is a board-certified dermatologist, who can discuss in detail the diagnosis and expectations. Also, reputable websites like that of the Skin Cancer Foundation offer information. Within dermatology, the American Academy of Dermatology and other societies can be great resources.

Going back to prevention, people need to understand that skin cancer is serious. You can die from skin cancer. But the good news is that these are preventable tumors and cancers. You have to take action to prevent it.

How do you do that? (It takes) appropriate use of UVA- or UVB-spectrum sunscreen, sun-protected behaviors when outdoors and screening, not just seeing a dermatologist once a year but also doing monthly self-skin examinations. Look for something new, unusual or changing and bring that to the attention of a dermatologist. Early detection results in early intervention, which results in very high cure rates.

More here:
Speaking Out: Taking Action Against Skin Cancer - Curetoday.com

Keeping blood sugar in balance can be a challenge, especially for people with type 1 diabetes who rely on intensive insulin therapy to prevent blood sugar from going too high. AtBaylor College of Medicine, the group ofDr. Yong Xuand his colleagues from other institutions study glucose-sensing neurons in the brain and they have identified a novel neural feedback system in a small brain region that contributes to keeping blood sugar in balance.

Glucose-sensing neurons sense fluctuations in blood sugar levels and respond by rapidly decreasing or increasing their firing activities. This response can trigger changes in behavior to increase glucose levels. For instance, the animals may begin eating, said Xu, professor ofpediatrics-nutrition at Baylor and theUSDA-ARS Childrens Nutrition Research Center(CNRC) at Baylor andTexas Childrens Hospital. Glucose-sensing neurons also can affect the production of hormones such as glucagon that can directly regulate glucose production or uptake by peripheral tissues. Its a feedback system that keeps the balance of blood glucose.

Glucose-sensing neurons are found in several brain regions. Xu and his colleagues focused on neurons located in a small area called the ventrolateral subdivision of the ventromedial hypothalamic nucleus (vlVMH). Many neurons in this region express estrogen receptor-alpha and respond to glucose fluctuations in the blood, but their functions in glucose metabolism had not been specifically investigated.

The researchers found that neurons in the vlVMH nucleus of murine brains had unique characteristics.

First, Xu and his colleagues were surprised that, while in other VMH subdivisions about half of the neurons were glucose-sensing, in the ventrolateral subdivision all the estrogen receptor-alpha neurons were glucose-sensing. Just this fact makes this group of neurons quite unique, said Xu, who also is professor ofmolecular and cellular biologyat Baylor.

They also found that, although all the neurons in this area sense glucose, they do not respond to changes in glucose level in the same way. About half of the neurons are glucose-excited their firing activity increases when they sense high glucose levels and decreases when glucose levels are low. In contrast, the other half of the neurons are glucose-inhibited they decrease firing when glucose is high and increase it when glucose is low.

We wondered why these neurons responded in opposite ways to the same glucose challenge, Xu said.

The researchers combined genetic profiling, pharmacological, electrophysiological and CRISPR gene-editing approaches to look into this question. They investigated the ion channels that each type of glucose-sensing neuron uses to respond to glucose levels. Ion channels are large molecules spanning across the cell membranes of neurons. The channels control the traffic of ions electrically charged atoms or molecules in and out of neurons, a process that is crucial for regulating neuronal firing activities.

The researchers found that glucose-excited neurons use a KATPion channel, but the glucose-inhibited neurons used a different ion channel called Ano4.

The KATPion channel is well known in our field, but the role of Ano4 ion channel in glucose sensing has never been reported. We have identified a new ion channel that is important for glucose-inhibited neurons, Xu said.

In addition, Xu and colleagues identified the neuronal circuits that are involved when glucose-excited and glucose-inhibited neurons respond to low blood glucose levels. They discovered that the circuits were different glucose-excited neurons project neuronal connections to a brain region that is different from the one reached by glucose-inhibited neurons.

Using optogenetics, a combination of genetic modifications and light to activate specific neuronal circuits, the researchers showed in mice that when glucose-inhibited neurons responded to low glucose levels, they activated a particular circuit, and the result was an increase of blood glucose. On the other hand, when glucose-excited neurons responded to low blood glucose, they inhibited a different circuit, but the result also was an increase in blood glucose levels.

When the mice were hypoglycemic, these two circuits were regulated in an opposite manner one was excited while the other was inhibited but the outcome was the same, bringing blood glucose to normal levels, Xu said. This forms a perfect feedback system to regulate blood glucose levels.

Interestingly, all the neurons in this important group express estrogen receptor-alpha, a well-known mediator of the ovarian hormone, estrogen. In the future, Xu and colleagues want to investigate whether estrogen plays a role in the glucose-sensing process and whether there are gender differences in the functions of these neurons on glucose balance.

Would you like to know more about this work? Find it in the journalNature Communications.

Other contributors to this work include Yanlin He, Pingwen Xu, Chunmei Wang, Yan Xia, Meng Yu, Yongjie Yang, Kaifan Yu, Xing Cai, Na Qu, Kenji Saito, Julia Wang, Ilirjana Hyseni, Matthew Robertson, Badrajee Piyarathna, Min Gao, Sohaib A. Khan, Feng Liu, Rui Chen, Cristian Coarfa, Zhongming Zhao, Qingchun Tong and Zheng Sun. The authors are affiliated with one or more of the following institutions: Baylor College of Medicine, University of Cincinnati, the University of Texas Health Science Center at San Antonio and the University of Texas Health Science Center at Houston.

This work was supported by grants from the NIH (R01 DK114279 and R21NS108091, R01ES027544/DK111436, R01DK100697, R00DK107008 and K01 DK119471), John S. Dunn Foundation and Clifford Elder White Graham Endowed Fund and USDA/CRIS (3092-5-001-059). Further support was provided by American Diabetes Association (1-17-PDF-138 and 1-15-BS-184) and American Heart Association awards (17GRNT32960003 and 19CDA34660335). Single cell transcriptome profiling was conducted at the Single Cell Genomics Core at BCM that is partially supported by shared instrument grant from NIH (S10OD018033, S10OD023469, S10OD025240) and data were analyzed by the BCM Multi-Omics Data Analysis Core (P01DK113954). This work also was partially supported by the Cancer Prevention and Research Institute of Texas (CPRIT, RP170005 and RP180734) and the NCI Cancer Center Support Grant (P30CA125123).

Excerpt from:
Unique sugar-sensing neurons work together to prevent severe hypoglycemia in mice. - ScienceBlog.com

June 9, 2020

Despite the uncertainty created by the COVID-19 pandemic, companies across the BioHealth Capital Region (BHCR) continue to hunt for talented life science professionals to advance their technologies, produce their commercial products for the patients that need them, and for many they are also supporting COVID-19 vaccine and diagnostic efforts that the whole world is counting on.

If you are looking for biotech jobs in Maryland or across the BioHealth capital region, now is a great time to start your search. Three of the top companies listed are located in Frederick, Maryland which has become an advanced BioManufacturing hub within the region and is seeing continued growth in this sector.

Heres a roundup of the top companies hiring for biotech jobs in Maryland and are hiring across a wide array of functions and experience levels.

Emergent BioSolutions, with multiple facilities in the BioHealth Capital Region, is hiring for dozens of full-time positions to propel its existing pipeline forward while also playing a leading role in the global fight against the coronavirus.

Emergent is looking to hire top talent for a variety of positions across the company that can be found on their Career Page. More specifically, they are seeing a great influx of hiring for several critical biomanufacturing roles out of their Bayview facility in Baltimore, including:

The companys Talent Acquisition team recently held a virtual recruiting event through BioBuzz to spotlight their career opportunities, showcase their mission and culture and share why Emergent is the leading biotech company in Maryland for career growth and opportunity, even in the times of COVID-19. During this event, job seekers learned more about open positions and the work culture at the companys Emergent Bayview facility in Baltimore, which is designated as a Center for Innovation in Advanced Development and Manufacturing (CIADM) by BARDA and the U.S. Department of Health and Human Services (HHS).

Emergent BioSolutions has a 22-year history of providing specialty products and services that address public health threats. It has approximately 1,800 employees across the globe dedicated to the companys mission to protect and enhance life. About 700 employees are located across five locations in Maryland. In Baltimore, Emergents Bayview facility is one of only three centers in the U.S. designated by the Department of Health and Human Services (HHS) as a CIADM designed to rapidly develop and manufacture medical countermeasures in response to public health emergencies like the COVID-19 pandemic.

You can read more about whats happening at Emergent by following the links below:

In late August 2017, Gilead Life Sciences acquired Kite Pharma for $12B. By October 2019, the FDA approved Kites CAR-T cancer therapy, YESCARTA, making it one of the first autologous cell therapies ever approved by the FDA for cancer.

Kite Pharma announced their initial expansion to Gaithersburg, Maryland in May 2018 with the lease of a 26,000 sq facility to support the work of a new Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI) to develop adoptive cell therapies targeting patient-specific tumor neoantigens.

Additional expansion news came in April 2019 with the announcement of their plans to open a new state-of-the-art commercial manufacturing facility in Frederick County (Urbana) that will produce innovative cell therapies for people with cancer, including Yescarta, starting in 2021.

Since the announcement, the company has made significant progress on construction and has been actively hiring top talent to bring the new cell therapy manufacturing facility online and produce its innovative cancer therapies when it opens.

Currently, the company has twenty (20) open positions at both the Frederick and Gaithersburg sites that range from Manufacturing, Quality, Process Engineering and more

To learn more, visit Kite Pharmas Frederick County, Maryland job page here.

BioFactura is a growing biotech company in Frederick, Maryland that develops and commercializes high-value biosimilars using its patented StableFast Biomanufacturing Platform. The platform has shown to be the optimal system for bringing these drugs to market with faster, lower cost, superior-quality manufacturing.

The companys current hiring effort was sparked by a recently awarded contract with the Biomedical Advanced Research and Development Authority (BARDA) valued at up to $67.4 million for the advanced development of its Smallpox Biodefense Therapeutic as a potential medical countermeasure (MCM) for the treatment of smallpox.Its current and past programs include biodefense drugs against smallpox and Ebola, novel medicines for cancer, and low-cost/high-quality biosimilars for autoimmune and infectious diseases.

For over 10 years, the company has been advancing life-saving medicines from the research bench to the patient using its innovative drug development and manufacturing technologies. Lead by founder, Darryl Sampey, PhD, and Dr. Jeffrey Hausfeld, BioFactura has fostered a culture of curiosity, innovation and love of science and engineering that is attracting top talent to join the company and be part of the mission.

We are poised to bend the cost curve in health care by creating generic versions of very expensive biologics. We have a real opportunity to do well and do goodby increasing patient access to great medications. Our technology has a niche focus on biosimilars that are high value and notoriously difficult for others to make, stated Dr. Jeffrey Hausfeld, Chairman of the Board, Chief Medical Officer at BioFactura at a 2019 Fireside Chat.

Current openings include:

Viela Bio, a clinical stage biotechnology company pioneering treatments for autoimmune and inflammatory diseases, has been growing rapidly since it spun out of AstraZeneca in 2018.

In just over two years, the company went public with a successful $150M IPO, had its Biologics License Application (BLA) for its lead product candidate, inebilizumab, accepted for review by the FDA and just recently announced the pricing of an underwritten public offering of 3,600,000 shares of its common stock. In addition, Viela recently announced positive interim Phase 1b data from a study with VIB7734, its novel anti-ILT7 therapy, in patients with cutaneous lupus erythematosus (CLE).

Viela Bio is currently hiring for a number of positions to progress its pipeline, in addition to supporting the potential commercialization of inebilizumab, which has PDUFA (The Prescription Drug User Fee Act) date set for mid-June.

Viela Bio currently has eight (8) job openings; job descriptions for these positions as well as information on the companys culture can be found here on their careers page.

Altimmune is a clinical-stage biopharmaceutical company focused on developing treatments for liver disease and immune-modulating therapies. The companys diverse pipeline includes next-generation peptide therapeutics for NASH (ALT-801) and chronic hepatitis B (HepTcell), conjugated immunostimulants for the treatment of cancer (ALT-702) and intranasal vaccines (NasoVAX and NasoShield).

The company has recently been in the news for its effort to develop a COVID-19 vaccine. Progressing its existing pipeline and developing a potential vaccine for COVID-19 has been driving Altimmunes current hiring push, which has been making progress attracting and hiring the talent it needs.

According to Altimmunes CEO Vipin Garg, We have been extremely busy and have hired a number of new positions in the last 3 months in product development, quality, regulatory and finance. Interestingly, we have made several senior-level hires (including a new board member) using video technology without ever meeting the candidates face-to-face. We are delighted to have new team members to support our efforts on COVID-19.

The company is currently seeking to fill two positions, including an Associate Director/Director for Product Development. Details on Altimmunes current open positions can be found on their Careers page.

Candidates can also email hr@altimmune.com

Catalent is a leading partner in helping pharmaceutical, biotech, and consumer health innovators develop, deliver, and supply superior treatments that improve peoples lives. The companys operating facilities remain open around the globe during the pandemic and it is continuing to deliver to its customers and their patients.

Catalent is working with several customers on multiple COVID-related antivirals, vaccines, diagnostics, and treatments for symptoms and effects of the COVID-19, including Johnson & Johnsons lead vaccine candidate, Arcturus Therapeutics mRNA-based vaccine, Humanigens GM-CSF monoclonal antibody, and Ennaid Therapeutics antiviral treatment targeted at COVID-19. Catalent is also involved in helping to support the delivery of over 2 million COVID-19 test kits monthly.

Catalents Maryland sites have over 270 openings for full-time roles across various functions including Quality, Manufacturing, Process Development, Product Development, IT, Accounting, Project Management, HR, Engineering, and more.

As Catalent navigates the COVID-19 pandemic, it will continue to keep its patient-first mindset top of mind and do everything it possibly can to keep its people safe and healthy.

Learn more about working in Gene Therapy at Catalent, and visit Gene_Therapy_Careers to explore opportunities in Maryland.

GSK is a global company delivering billions of healthcare products every year, and several of those products are manufactured right here in Maryland.

Maryland is home to one of GSKs Global Vaccine Research Centers, as well as Rockville Biopharm where they make and supply key medicines like Benlysta for patients.

We discover, make and market healthcare products; and we aim to bring them to as many people as possible. Find out how we make this happen and what career opportunities there could be for you from research and development through to supplying products to those who need them.

As a business, we are supporting global efforts to tackle the COVID-19 pandemic and protecting the safety of our people by following local government advice and guidance. We are still hiring to ensure that we can continue to supply our medicines, vaccines, and products to our patients and consumers all over the world. You can learn more about our recruitment practices here. Some of our roles are inundated with applications at this time, so response times may be slower than usual. We appreciate your patience, stay safe, and look after yourselves.

According to GSK Rockville there are 65 Positions in between Biopharm (35 openings mostly production/manufacturing) and Vaccines (30 openings mostly R&D).

Related articles on GSK:

More information about GSK careers.

Meso Scale Diagnostics, LLC. (MSD), based in Rockville, Maryland, is a global leader that develops, manufactures, and markets instruments and assays for array-based biological measurements for research use in life sciences and for biodefense applications. MSD is the worlds leading provider of highly sensitive multiplex immunoassays.

Founded in 1995, MSDs proprietary MULTIARRAY technology enhances medical research and drug development by enabling researchers to profile many biomarkers simultaneously in a single sample without compromising assay performance. MSDs technology has been widely adopted by researchers in pharmaceutical companies, government institutions, universities, and clinical laboratories worldwide for its high sensitivity, excellent reproducibility, and wide dynamic range. Throughout its history, MSD has continued to evolve its technology platform to enable researchers to solve complex biological questions and, as the Company looks toward the future, it is expanding into clinical applications and the emerging fields of personalized medicine and companion diagnostics.

According to their career site; MSD has 57 Positions in Maryland between Rockville and Gaithersburg facilities.

More information about MSD can be found at http://www.mesoscale.com.

Stay tuned to BioBuzz for more Career Resources and news on the latest employers with biotech jobs in Maryland and across the Biohealth Capital Region.

Over the past 8 years, Chris has grown BioBuzz into a respected brand that is recognized for its community building, networking events and news stories about the local biotech industry. In addition, he runs a Recruiting and Marketing Agency that helps companies attract top talent through a blended model that combines employer branding and marketing services together with a high powered recruiting solution.

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The Top Biotech Companies That are Hiring June 2020 - BioBuzz

June 9, 2020

This has certainly been a year unlike any other for BioHealth Capital Region (BHCR) life science companies of all sizes and kinds. Just a few months into 2020, the coronavirus pandemic swept the region, seriously disrupting, albeit temporarily, the best laid plans of aspiring founders, entrepreneurs, emerging startups, pre-clinical and more established, larger biohealth organizations in Virginia, Washington, D.C. and Maryland.

No one has escaped the reach of COVID-19 in the BHCR, the rest of the U.S., or around the globe. While this industry continues to be uniquely positioned to weather a storm like this, many were still impacted via delayed clinical trials and having to adjust to near 100% remote work for non-bench role employees. Some areas like Travel Medicine have been hit hard, as most are canceling their 2020 trips. Sadly, even when daily life resembles a new normal, Travel Medicine is likely to have a longer recovery as many organizations have adapted to virtual meetings that will potentially reduce future travel significantly.

The regional life science ecosystem was prepared and pivoted rapidly to not only ensure continued operations, but also to redirect resources, talent and technologies to creating testing, developing COVID-19 therapies and progressing SARS-CoV-2 vaccine candidates.

The BHCR life science community has been remarkably resilient and innovative during this time, for certain, despite myriad challenges caused by COVID-19 across VC funding, the supply chain, and clinical trials, to name just a few.

Despite all of the tumult and turmoil, VC funding deals are still getting done in the BHCR, though perhaps currently on a slightly slower pace and scale. Well see what the second half holds on the VC funding front, but with signs of a slow, steady, and careful reopening afoot in the region, its possible that funding deals will pick back up in the latter half of 2020.

With that, lets take a look at funding deals that were announced in the BHCR in Q1/Q2 of 2020, the year of the pandemic.

Immunomic Therapeutics Incorporated (ITI), located in Rockville, Maryland recently announced it had closed on $61.3M in financing, exceeding its initial fundraising projections by over $11M. The Korean investment group HLB Co., LTD led the financing round, which is just the latest accomplishment for the clinical-stage biotechnology company that launched in 2006.

Read more about CEO Bill Hearls plans for ITI in our recent BioBuzz story, Marylands Immunomic Therapeutics Exceeds Fundraising Goal, Envisions Future IPO.

LifeSprout, a privately-held regenerative medicine company located in Baltimore, Maryland, recently announced the closing of a $28.5M Series A financing round, marking a significant milestone in its history. The round was led by Redmile Group, LLC, with new institutional investors Nexus Management, LP, Emerald Development Managers, LP, and Baltimores Abell Foundation joining the investment group.

You can read more about LifeSprouts story and plans in our recent featured story, Baltimore, Marylands LifeSprout Closes $28.5M Series-A, Looks Toward the Future.

Integrated Biotherapeutics subsidiary IBT Vaccines, which is located in Rockville, Maryland received $3.9M in funding as part of a larger $48M investment in eight companies by Novo Holdings REPAIR Impact Fund.

The funding will be used to advance its advance the development of the IBT-V02 vaccine for Staphylococcus aureus induced recurrent skin and skin structure infections (SSSI), according to the companys press release in early January 2020. At the time of the announcement, the vaccine was in the late clinical development stage and is potentially a first-to-market, multivalent vaccine for antibiotic resistant Staphylococcus aureus.

Vigene Biosciences, which just recently opened its new headquarters in Rockville, Maryland, announced in late March that it had secured institutional investment from Signet Healthcare Partners (Signet), a New York-based growth equity firm specializing in healthcare investments.

Vigene is a leading provider of viral vector products and services used in gene therapy. According to Washington Business Journal reporting, the round was close to $3M.

We wrote about Vigenes new HQ grand opening and its remarkable relationships with the families it serves. You can read Maryland Biotech Opens New Gene Therapy Contract Manufacturing Facility To Support Rapid Growth to learn more.

College Park, Marylands Pathotrak announced it had closed a seed round of $1.2M in mid-May 2020. The company has developed a rapid food test for salmonella and E. coli in food. The companys new test has the potential to cut down food safety test result turnaround times by nearly one full day, which could have a significant positive financial impact on the food industry while improving food safety for companies and consumers.

The Maryland Momentum Fund contributed $150K, the Dingman Angels, which is a program within the Robert H. Smith School of Business at the University of Maryland, invested $310K, and a global life sciences company with a food safety division also invested in pathOtrack.

You can read more here about Pathtracks novel test and the company in a story BioBuzz recently published.

ARMR Systems, a Maryland BioPark-affiliated startup, received seed money from local funding sources to support its hemorrhagic control systems designed to increase the survivability of battlefield trauma when advanced medical support is not immediately available.

The seed funding included a $350,000 joint investment from the University of Maryland, Baltimore, and the Maryland Momentum Fund and also included funding from the Tamiami Angel Fund.

ARMR Systems was featured in the BioBuzz story, Baltimores Top 10 BioHealth Startup CEOs.

There were several large HealthTech investments in this first half of the year that include:

Timonium, Marylands b.well Connected Health secured $16M in Series A financing to back its patient-facing health management tool. The round was led by UnityPoint Health Ventures, with participation from ThedaCare and Well Ventures.

B.wells management tool integrates data, insights, and partners into a single customized solution that helps people take control of their healthcare experience, according to the companys website. The company is located at The Grid, which is located at the Lion Brothers Building one block south of the University of Maryland Biopark. The company will use the Series A funding to scale as it enters a growth and hiring phase.

Bethesda, Marylands Aledade, a data analytics and healthcare practice workflow company, closed a $64M Series C round led by OMERS Growth Equity. California Medical Association, Echo Health Ventures, and Meritech Capital and GV also contributed to the round. Aledade has created a network of approximately 550 doctor-led Accountable Care Organizations (AOC) that utilize an outcome-based rather than a fee for service business model. The company plans to use the Series C funding to expand its U.S. footprint and AOC practice network.

In the first half of 2020, the resilient nature of the BioHealth Capital Region has been on full display, yet a sobering reality exists: we still dont have a COVID-19 vaccine. And while many companies around the world and region work day and night to create one, the region must remain diligent and smart in how it continues to weather this storm. The second half of 2020 will be interesting to watch, as the region slowly and carefully reopens to a new COVID-19 normal.

If the transition to the post COVID-19 environment goes well, will VC firms come off the sidelines in droves, releasing a flood of new funding? Will companies move forward with M&A thats been held close and delayed? Will a BioHealth Capital Region life science company be the first to get COVID-19 vaccine approved and manufactured? Its difficult to answer any of these questions, but it is certain that Q3/Q4 of 2020 will be fascinating to watch.

Steve has over 20 years experience in copywriting, developing brand messaging and creating marketing strategies across a wide range of industries, including the biopharmaceutical, senior living, commercial real estate, IT and renewable energy sectors, among others. He is currently the Principal/Owner of StoryCore, a Frederick, Maryland-based content creation and execution consultancy focused on telling the unique stories of Maryland organizations.

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2020 Life Science Venture Capital Funding Roundup for Q1/Q2 in the BioHealth Capital Region - BioBuzz