A quick read of whose wallets got thicker in the biopharma industry,from largest to smallest.

D3 Bio

Industry-veteran George Chen left a seven-year career at AstraZeneca to launch his ownShanghai-based biotech, D3 Bio, with the support ofbig-nameinvestorslikeBoyu Capital, Matrix Partners China,SequoiaCapital China, Temasek and Wuxi AppTecs Corporate Venture Fund. At this point D3 is keeping its pipeline close to the chest.But Chensays its the approach thatsunique. The company will startwith insights from clinical development and a market assessment of unmet needs and use that to guide the clinical development path. D3 will use this$200 million Series Ato build out an R&D team focused on precision medicine in the realms of immunology and oncology.

Metagenomi

Unlocking the power of microbial evolution,Metagenomimines the worlds natural microbial environment to rapidly develop effective cures to treat incurable genetic diseases. A recent$65 million Series Awill help accelerate the expansion of its gene editing systems for therapies in oncology and genetic-related diseases.This means developing a vast database of gene editing capabilities to enable unprecedented therapeutic approaches. Working with visionary investors, such as Leaps by Bayer and Humboldt Fund, will allow us to deliver on our promise to partners and fuel the development of our own pipeline of innovative curative medicines, Thomas said in a statement.

AliveCor

AI companydisguisedas a medical device companyAliveCorpicked up$65 millionto ramp up speed on their remote cardiology platform. Amidst a pandemic, telehealth appointments have been increasingly necessary.AliveCorsECG will be strengthened withcardiologicaltelehealthservices as well as with detection and condition management services. To date, its products have served more than one million customers globally, recording over 85 million ECGs.AliveCorsKardiaMobiledevice is FDA-cleared and the most clinically validated personal ECG solution in the world.

Nereid Therapeutics

Birthed fromthe work of Clifford P.Brangwynne, Ph.D., Nereid hopes to translate the therapeutic promise of biomolecular condensates from physics to physicians.The biotech will take the$50 million Series Afunding andBrangwynnesproprietary technology enabling precise measurement, interrogationand control of phase separation in cells to develop their drug discovery platform. The platform holds potential to enable completely new approaches to discovering and developing therapeutics across a wide variety of diseases, focusing first on cancers and neurodegenerative disordersaffected by phase transitions.

KiraPharmaceuticals

Backed by$46 million in financingfrom biotech entrepreneur Peter Wirth and others,Kira launchedwith a mission of pioneering a new generation of complement-targeted therapies to treat immune-mediated diseases. With the financing in hand, Kira is aiming to have three assets in the clinic within the next 18 months. The companys most advanced program, P014, is a first-in-class biologic drug with a unique mechanism of action designed to inhibit both upstream and downstream complement targets.Former Sienna Biopharmaceuticals CEO Frederick Beddingfield will be at the helm.

Adagio Medical

Adagio is singing joyfully to the tune of a $42.5 million Series Eto supportthe commercialization of itsiCLASsystem.iCLASis Adagios intelligent Continuous Lesion Ablation Systempursuing both an Investigational Device Exemption trial and a European VT CE-Mark trial.Cardiac ablation is a large and growing market that faces significant challenges including disappointing clinical outcomes, long procedure times and unsatisfactory profitability for providers, saidTuan Huynh, ofArrowMark, one of the Series E investors joining Adagios board of directors. We believe Adagio represents a unique opportunity to transform ablation therapy and look forward to partnering with Adagios management team to support the companys growth and commitment to addressing challenges faced by physicians and their patients.

IniPharm

Founded in 2018 with a focus on liver disease,IniPharmbrought in$35 million with a Series Afinancing roundto take its lead program through to IND filing and into clinical trials.Theprogram targets the HSD17B13 gene, which according to CEO Brian Farmer, confers pretty amazing protection against liver disease.It doesnt appear to actually prevent the diseases causation, but slows progression to more serious illness by preventing inflammation, fibrosis and cirrhosis of the liver, which are the dangerous effects of liver disease.The potential for therapies that effectively target HSD17B13 activity is significant because it is linked to a broad spectrum of liver and related diseases, said Farmer.

InterVennBiosciences

InterVennlooks to ramp up their ability to discover biomarkersand design clinical trials with the help of a little AI. Funds from a$34 million Series Bwill expand its precision medicine platformfor cancer detection.InterVennstech platform targets carbohydrates known as glycans, looking for aberrant glycosylation of certain proteins, which are implicated in a variety of disease states, including inflammation.The companysVOCALprojectis evaluating a blood test to determine ifan ovarian tumor is benign or malignant.It is also conducting research into colorectal cancer and kidney cancer, hunting for clinically actionable biomarkers that can be used for diagnosis, prognosis, and detection of cancer recurrence, as well as predictive tests to help choose appropriate drugs.

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Biopharma Money on the Move PharmaLive - PharmaLive

Nov. 16, 2020 12:30 UTC

- Highly innovative genome editing technology platform accelerates and broadens ElevateBios cell and gene therapy enabling technologies

- LifeEDIT Therapeutics to develop its own pipeline of potentially life-transforming therapeutics

- LifeEDIT Therapeutics to leverage its novel gene editing platform with strategic partners including ElevateBio portfolio companies

CAMBRIDGE, Mass. & RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)-- ElevateBio, a Cambridge-based cell and gene therapy holding company, and AgBiome, a leader in developing innovative products from the Earth's microbial communities, today announced that LifeEDIT Therapeutics has joined ElevateBios growing portfolio of therapeutic, technology and manufacturing companies. LifeEDIT Therapeutics combines a highly innovative genome editing platform, derived from AgBiomes massive proprietary microbial library, with ElevateBios proven expertise in the discovery and development of new cell and gene therapies. LifeEDIT will continue to develop internal cell and gene therapies while further strengthening its platform of diverse genome-editing enzymes and provide gene editing expertise to strategic partners including ElevateBio portfolio companies. AgBiome retains rights for gene editing in agriculture, animal health, and diagnostics.

Genome editing technologies have revolutionized the way we develop cell and gene therapies and regenerative medicines, said Mitchell Finer, Ph.D., President, ElevateBio BaseCamp, and newly appointed Chief Executive Officer, LifeEDIT Therapeutics. However, in order to realize the promise of, and democratize, these highly innovative therapeutic approaches, the field needs to access novel RNA-guided nucleases and base editors that offer greater specificity and broader genome coverage, which LifeEDIT can provide. LifeEDITs genome editing platform is one of the most versatile in the field and was the natural fit as we continue to build a world leading cell and gene therapy offering.

Members of ElevateBio will join the newly formed LifeEDIT Therapeutics management team, which will continue to benefit from its existing visionary scientific leadership and research team. The combined executive team will include:

Over the last 18 months we have built a truly unique platform of numerous RNA-guided nucleases with diverse PAM requirements, for which weve been able to show functional activity, said Tedd Elich, Ph.D., Chief Scientific Officer, LifeEDIT Therapeutics. The wide range of gene editing enzymes across our platform increases our ability to target any genomic sequence of interest and will allow us to tackle some of the most challenging diseases, bringing desperately needed, potentially curative, therapies to patients in need.

"AgBiome's GENESISTM platform is built on our microbial collection, their complete genome sequences, and our industry-best data science platform to identify new useful functions, said Eric Ward Co-Chief Executive Officer, AgBiome. This unique resource formed the basis for the many genome editing technologies that are now part of LifeEDIT Therapeutics. We look forward to continuing to collaborate with the LifeEDIT team as they build a world-class pipeline of clinical candidates and bring a broad array of genome editing technologies to innovators across the biotechnology industry."

About Genome Editing and LifeEDIT Therapeutics Platform

Genome editing technologies have revolutionized the way cell and gene therapies and regenerative medicines are discovered and developed by allowing genetic material to be removed, added, or altered at specific locations in the genome. While these technologies are in widespread use experimentally, enzymes that offer broader coverage and greater specificity are needed for creating novel cell and gene therapies.

To meet the need for better genome editing approaches, LifeEDIT Therapeutics has built one of the worlds largest and most diverse arrays of novel RNA-guided nucleases (RGNs) and base editors that are active in mammalian cells. These RGNs were developed using AgBiomes proprietary collection of more than 90,000 microbes and their complete genomes. LifeEDIT Therapeutics is investigating these proprietary RGNs, which are sourced exclusively from non-pathogenic organisms, to develop new gene editing tools with higher fidelity, novel functionality, reduced immune response risk, and easier delivery. LifeEDIT Therapeutics nuclease collection also has a broad range of Protospacer Adjacent Motifs (PAMs) short sequences that must follow the targeted DNA sequence in order for the enzyme to make cuts that offer unprecedented access to genomic loci of interest. The LifeEDIT Therapeutics RGNs offer flexible editing options which encompass knock-out and knock-in capabilities, transcriptional regulation, and base editing when coupled with its proprietary deaminases.

LifeEDIT Therapeutics next generation editing systems will propel the development of novel human therapeutics by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies. In addition to developing its own pipeline of cell and gene therapies, LifeEDIT Therapeutics will continue to build its platform of novel nucleases, provide gene editing expertise to strategic partners and ElevateBios portfolio companies, and form other third-party partnerships to discover and develop new therapies.

About ElevateBio

ElevateBio, LLC, is a Cambridge-based creator and operator of a portfolio of innovative cell and gene therapy companies. It begins with an environment where scientific inventors can transform their visions for cell and gene therapies into reality for patients with devastating and life-threatening diseases. Working with leading academic researchers, medical centers, and corporate partners, ElevateBios team of scientists, drug developers, and company builders are creating a portfolio of therapeutics companies that are changing the face of cell and gene therapy and regenerative medicine. Core to ElevateBios vision is BaseCamp, a centralized state-of-the-art innovation and manufacturing center, providing fully integrated capabilities, including basic and translational research, process development, clinical development, cGMP manufacturing, and regulatory affairs across multiple cell and gene therapy and regenerative medicine technology platforms. ElevateBio portfolio companies, as well as select strategic partners, are supported by ElevateBio BaseCamp in the advancement of novel cell and gene therapies.

ElevateBios investors include F2 Ventures, MPM Capital, EcoR1 Capital, Redmile Group, Samsara BioCapital, The Invus Group, Emerson Collective, Surveyor Capital (A Citadel company), EDBI, and Vertex Ventures.

ElevateBio is headquartered in Cambridge, Mass, with ElevateBio BaseCamp located in Waltham, Mass. For more information, please visit http://www.elevate.bio.

About AgBiome

AgBiome partners with the microbial world to improve our planet. AgBiome discovers and develops innovative biological and trait products for crop protection. The proprietary GENESIS discovery platform efficiently captures diverse, unique microbes for agriculturally relevant applications, and screens them with industry-best assays for insect, disease, and nematode control. Through its commercial subsidiary, AgBiome develops and sells proprietary crop protection solutions. The first of these, Howler, is a revolutionary fungicide for disease control in a broad variety of crops. AgBiome and Genective recently formed a strategic partnership to establish a new leader in insect traits, a market with over $5 billion in annual opportunities. AgBiome has a global R&D collaboration with Elanco Animal Health Incorporated (NYSE: ELAN), to develop nutritional health products for swine. AgBiomes investors include Polaris Partners, ARCH Venture Partners, Fidelity Investments Inc., UTIMCO, Pontifax AgTech, Innotech Advisors, Syngenta Ventures, Leaps by Bayer, and Novozymes. For more information, visit http://agbiome.com.

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ElevateBio and AgBiome Announce LifeEDIT Will Join ElevateBio's Portfolio of Innovative Cell and Gene Therapy Companies - BioSpace

Johns Hopkins Medicine researchers report that new experiments with genetically engineered mice have found clear connections among a range of autism types and abnormalities in brain cells whose chemical output forges platonic (non-sexual) feelings of love and sociability.

The findings, the researchers say, could eventually fuel the development of autism therapies that target disease symptoms spurred on by abnormalities in parvocellular oxytocin neurons, which are brain cells in the hypothalamus of mammals.

A report on the experiments was published online Oct. 27 in Neuron.

The investigators pursued evidence of the connections because of long-known variations in forms and symptoms of autism spectrum disorders, and because those with Fragile X -- an inherited disorder that occurs in one in 4,000 males and one in 6,000 females -- frequently is characterized by the inability to form close social bonds.

"Autism is defined by impaired social behaviors, but not all social behaviors are the same," says Gl Dlen, M.D., Ph.D., associate professor of neuroscience at the Johns Hopkins University School of Medicine. "People with autism generally have less difficulty with developing very close, family bonds than with friendships. Our experiments provide evidence that these two types of affection are encoded by different types of oxytocin neurons, and that disruption of one of these types of neurons is responsible for the characteristic social impairments seen in autism."

For more than a century, Dlen says, scientists have known there are two types of neurons in the hypothalamus. The neurons release the so-called "love hormone" oxytocin, which induces contractions during childbirth, reduces stress and fosters bonding among animals across mammalian species, including humans.

A magnocellular oxytocin neuron, which is one type of oxytocin-releasing neuron, releases huge quantities of oxytocin to the brain and body -- as much as 500 times or more than is released by parvocellular oxytocin neurons, which limit their scope and avoid flooding the body with all-consuming feelings of love.

As their name suggests, magnocellular oxytocin neurons are larger than other neurons and can send their arm-like axons beyond the blood-brain barrier. Among their functions, magnocellular oxytocin neurons stir filial love -- what Dlen calls "mad love" -- and bonding between infants and mothers, and between sexual partners.

Dlen's research shows that parvocellular oxytocin neurons, which comes from the Greek word "parvo" or "small" -- also encode social behaviors, but a different kind than the magnocellular neurons encode. While magnocellular oxytocin neurons encode social behaviors related to reproduction (pair bonding and parental bonding), parvocellular oxytocin neurons encode social behaviors related to what Dlen calls "love in moderation," or the platonic love that is important to communities (friends and colleagues).

To study if and how autism symptoms are associated with disruptions in either or both of magnocellular and parvocellular neurons, Dlen and her team first genetically engineered mice to glow a fluorescent light in all oxytocin neurons, magno and parvo. Then, knowing that magnocellular neurons project their axons and chemicals beyond the blood/brain barrier, the research team used dyes that stay within the barrier to mark only the parvocellular neurons -- which are rarer and harder to detect, as well as smaller in size.

Next, Dlen enlisted the help of Johns Hopkins scientist Loyal Goff, Ph.D., an expert in charting the genetic profile of individual cells. The technique, called single cell sequencing, specifically reads an individual cell's RNA -- a genetic cousin to DNA -- which indicates how the cell's genetic code is being read and which proteins are being produced. The way our genetic code is read makes one cell type different from another.

"This study is a comprehensive characterization of two types of closely-related neurons involved in the regulation of social behavior," says Goff, assistant professor of genetic medicine at the Johns Hopkins University School of Medicine. "One of the things that makes this study so unique is the multi-modal aspect of this characterization; relating anatomical, morphological, electrophysiological, transcriptional, genetic, and behavioral features to fully define the relevant and important differences between these two types of neurons."

The research team used single cell sequencing and other gene-tracking tools and techniques to ensure that the subpopulations of magnocellular and parvocellular neurons were, indeed, distinct, so that they could genetically alter each group to determine if a change would induce autism-like behaviors in mice. What the researchers measured included how much the mice liked their social interactions and how much they preferred things associated with those social interactions (such as bedding).

To re-create a model of autism in mice, the scientists turned to the FMR1 gene, which is linked to Fragile X, an inherited disorder characterized by intellectual disability, but also one of the most commonly identified causes of autism, occurring in about five percent of people with the condition.

In humans, the FMR1 gene is silenced through a cellular process that adds chemicals called methyl groups to the gene. This same process does not occur in mice, so to replicate the FMR1 gene abnormality, the scientists genetically engineered the mice to have no functioning FMR1 gene either throughout the brain or only in parvocellular neurons.

The researchers studied how mice without FMR1 valued the rewards from forming a social bond with an adult female mouse serving as a surrogate parent. These mice learned to like bedding associated with the surrogate parent, but not bedding associated with social interactions with peer mice -- evidence that mutations in genes that cause autism selectively disrupt platonic love, but spare filial love.

When the scientists deleted the FMR1 gene in parvocellular cells only, not magnocellular cells, the mice had the same reaction: intact affinity for things associated with their surrogate parent, compared with things associated with peer mice. The scientists found no such preference in mice lacking FMR1 in oxytocin magnocellular cells.

In a further set of experiments to pin down the specificity of their findings with the oxytocin-producing neurons, the scientists studied how certain genes linked to risk for autism were turned on or off, or expressed, among the two types of oxytocin neurons. They found that significantly more autism risk genes had higher expression levels in parvocellular neurons compared with magnocellular neurons. However, when the scientists looked at genes for schizophrenia, Alzheimer's disease and diabetes, there were no such differences in gene expression between the two oxytocin neuron types.

"This tells us that the difference we are seeing between the two types of oxytocin neurons relates to the disease that is characterized by impaired social behaviors, but not diseases where this behavior is not a defining symptom," says Dlen.

She also notes, "What may be happening in the brain is that even though all brain cells may carry a particular mutation associated with autism, some neurons are more vulnerable to the symptoms related to social bonding."

Dlen plans to conduct similar studies on genes associated with other types of autism. She says her work may indicate that drugs currently being tested for autism -- such as intranasal oxytocin -- could prove ineffective because the treatments target magnocellular neurons, which the new study indicates is not central to the disease. Instead, she says, their evidence suggests that parvocellular oxytocin neurons should be the focus of drug development for autism.

Reference:Connie Jiang, Loyal A. Goff, Gl Dlen et al. Parallel Social Information Processing Circuits Are Differentially Impacted in Autism. Neuron, 2020; DOI: 10.1016/j.neuron.2020.10.002

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Mouse Studies Link Some Autism to Brain Cells That Guide Sociability and Platonic Love - Technology Networks

This month, Insights & Outcomes is mindful of the mental health implications of COVID-19, the moments when cells act like portfolio managers, and a missive from a Milky Way magnetar.

As always, you can find more science and medicine research news on YaleNews Science & Technology and Health & Medicinepages.

Magnetars, a type of neutron star believed to have an extremely powerful magnetic field, could be the source of some fast radio bursts (FRBs), according to a new study in the journal Nature. FRBs are extremely bright, fast radio emissions that can release more energy in a fraction of a second than the Sun generates over many years. Astronomers discovered the existence of FRBs a decade ago and continue to debate the cause of the signals.

This is the first evidence of an astrophysical source for one FRB, tying it to a galactic neutron star with a large magnetic field and providing evidence that at least some FRBs are consistent with extragalactic magnetars, based on the brightness of this event, said co-author Laura Newburgh, an assistant professor of physics at Yale. Newburgh developed new analysis and measurement information that helped establish the brightness of an FRB emanating from a nearby magnetar located in the Milky Way. The Canadian Hydrogen Intensity Mapping Experiment, a collaboration of 50 scientists, produced the research.

Age, sex, and underlying medical issues have been recognized as major risk factors for an adverse outcome from COVID-19 infection. Now Yale psychiatrists say doctors should also consider another factor that increases risk of death in the pandemic a patients mental health. A new study shows that patients with psychiatric disorders admitted to Yale New Haven Hospital for treatment of COVID-19 were significantly more likely to die than those without a diagnosed mental health disorder. The higher mortality rate held even after controlling for other risk factors such age, sex of the patient, and pre-existing health conditions. The authors theorized that psychiatric disorders such as depression may have a harmful effect on patients immune system response to infection. We need to consider the health of the mind as well as the body when considering treatment options for people diagnosed with COVID-19, said John Krystal, chair of the Department of Psychiatry and senior author of the study. Luming Li, assistant professor of psychiatry, was lead author of the study published in thejournal JAMA Network Open.

In times of stability, cell populations act like investors with large portfolios. They hedge their bets by diversifying receptors on the surfaces of individual cells, preparing the population for sudden swings in the environment. But how can these populations respond quickly to unanticipated changes when the process that dictates composition of those receptors the regulating activity of genes is typically so time consuming?

A new study of E. coli bacteria by Yale scientists shows that when receiving new environmental signals, the diversity of cellular portfolios is reduced 10-fold, allowing the cell population to adjust to changing circumstances.

Essentially, cells instantly stopped hedging their bets and adjusted their sensitivity to focus on following the present signal, effectively consolidating assets into a winning portfolio. The mechanism we found enables a population to very rapidly switch from a bet-hedging mode to an exploitation mode, said Yales Thierry Emonet,professor of molecular, cellular, and developmental biology and of physicsand co-senior author of the study. Before this study, all mechanisms reported to do so also involved gene expression, which is orders of magnitude slower.Keita Kamino is first author of the study, published in the journal Science Advances. The research was conducted in the labs of Emonet and co-senior author Thomas S. Shimizu, group leader at the AMOLF Institute.

Sidi Chen,assistant professor in the Department of Genetics and the Systems Biology Institute and member of the Yale Cancer Center, received a $50,000 research grant from the Alliance for Cancer Gene Therapy (ACGT) to advance a versatile, scalable technology for targeting difficult-to-treat cancers. The technology Chen developed is called MAEGI Multiplexed Activation of Endogenous Genes as an Immunotherapy which leveragesthe natural power of the immune system to fight tumors.

The ACGT Scientific Advisory Council finds Dr. Chens MAEGI technology to be unique and exciting because it simultaneously targets multiple differences and activates multiple immune system responses, said Kevin Honeycutt, CEO and president of ACGT. It has proven to be very effective in animal models. We believe our support will enable its advancement into the clinic where it would have major, life-saving impact on pancreatic and other difficult-to-treat cancers, such as melanoma, glioblastoma and triple negative breast cancer.

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Insights & Outcomes: Cellular bet hedgers and a message from a magnetar - Yale News

LEXINGTON, Mass., Nov. 03, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, today announced the appointment of Mariana Nacht, Ph.D., as chief scientific officer, effective Nov. 30, 2020, and the promotion of Kyle Chiang, Ph.D., to chief operating officer, effective Nov. 2, 2020.

Dr. Nacht brings more than 20 years of experience in both large and small biotech companies to her role at LogicBio. Most recently, she served as CSO and was a founding executive team member of Cereius, where she led a small internal research team and a group of collaborators to develop radiolabeled proteins for the treatment of brain metastases. Before that, she served as CSO of Vivid Biosciences, a functional precision medicine company, where she was also a founding executive team member. Dr. Nacht has also served in key scientific roles at Padlock Therapeutics (acquired by Bristol Myer Squibb in 2014) and Avila Therapeutics, a platform company that developed covalent irreversible inhibitors and was acquired by Celgene in 2012. Earlier in her career, she spent a decade working at Genzyme (now Sanofi Genzyme), where she led anti-angiogenesis and oncology target discovery efforts. Dr. Nacht received her B.S. in biology from Tufts University and her Ph.D. from the University of Pennsylvania.

We are proud to expand our leadership team as we prepare to launch our first clinical trial in pediatric patients with methylmalonic acidemia (MMA) and continue to advance both our GeneRide and Next Generation Capsid platforms, said Fred Chereau, CEO of LogicBio. Mariana brings terrific expertise in novel therapeutic platforms as well as deep experience in building and leading strong scientific teams to her position as CSO. Were thrilled to welcome her to LogicBio as we move into this exciting next phase of progress. Im also delighted to have Kyle promoted to our core leadership team. He has provided essential guidance on pipeline strategy and program development from the early days of LogicBio and he will continue to be an important voice in shaping our future growth.

I am very enthusiastic about the potential for the GeneRide platform to transform care for pediatric patients with rare genetic diseases, Dr. Nacht said. We all enter this field to make a difference for patients, and I am excited to be joining LogicBio just as LB-001, our lead program for children with MMA, is about to enter the clinic with the Phase 1/2 SUNRISE trial. Beyond LB-001, I look forward to further advancing LogicBios pipeline with the goal of bringing more durable and transformational therapies to people living with devastating genetic diseases.

Dr. Chiang was the second employee at LogicBio and has held positions of increasing responsibility since joining the team as director of translational science in 2016. Most recently, he served as vice president, product strategy, where he led LB-001 through early regulatory interactions and managed LogicBios collaboration with the Childrens Medical Research Institute to develop more potent and more easily manufacturable AAV capsids for gene therapy and genome editing applications. Before joining LogicBio, Dr. Chiang led aTyr Pharmas ATYR1940 program from discovery through early clinical development for patients with facioscapulohumeral muscular dystrophy. Dr. Chiang received his B.S. in biochemistry and cell biology from the University of California, San Diego and his Ph.D. in macromolecular cellular structure and chemistry from the Scripps Research Institute.

LogicBio also announced today that Bryan Yoon, Esq., the companys chief administrative officer, general counsel and corporate secretary, will be departing from the company effective Nov. 6, 2020. I want to thank Bryan for his contributions to LogicBio and we wish him the best in his next challenge, Mr. Chereau said.

AboutLogicBioTherapeuticsLogicBio 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 withTakedato 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 in Lexington, Mass. For more information, please visit http://www.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 timing, progress and results of the Companys strategic directives and its research and development activities, including those related to LB-001 and its pipeline. 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:Matthew LaneGilmartin Investor Relationmatt@gilmartinir.com

Media:Stephanie SimonTenBridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

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LogicBio Therapeutics Announces Appointment of Veteran Biotech Executive Mariana Nacht, Ph.D., as Chief Scientific Officer and Kyle Chiang, Ph.D.,...

Understanding genetic disease in mixed-breed and purebred dogs and cats can bring about more effective treatments and better client service, says clinical geneticist and general practitioner Dr. Jerold Bell.

If we understand the genetic background of our patients, were better positioned to prevent, to mitigate, or to alter the expression of genetic disease, allowing our patients to be healthier in their lifetimes as well as to breed healthier dogs and cats, Dr. Bell said.

An adjunct professor at the Cummings School of Veterinary Medicine at Tufts University, Dr. Bell spoke about genetic diseases during the AVMA Virtual Convention 2020 this August. In addition to his teaching duties, Dr. Bell works as a solo practitioner, and he sees dogs and cats all day long and sees genetic disease in our patients all day long.

He explained that common genetic disorders are caused by ancient disease liability genes that preceded breed formation. Since these mutations occurred long before the separation of breeds, these diseases are seen across all breeds and in mixed breeds.

The most common hereditary diseases in dogs are allergies, followed by hip and elbow dysplasia; inherited cancers such as lymphoma, hemangiosarcoma, mast cell tumor, and osteosarcoma; patella luxation; nonstruvite bladder stones; hypothyroidism; mitral valve disease; inflammatory bowel disease; diabetes mellitus; retained testicles; and umbilical hernias.

In cats, the most prevalent genetic diseases are inflammatory cystitis, then feline urological syndrome, diabetes mellitus, lymphoplasmocytic gingivostomatitis, nonstruvite bladder stones, allergies, eosinophilic skin disease, and inflammatory bowel disease.

Disease is not a function of homozygosity, which happens when identical DNA sequences for a particular gene are inherited from both biological parents, nor is it a consequence of inbreeding. Rather, Dr. Bell explained, hereditary diseases are a result of the accumulation and propagation of specific disease liability genes. Breed-related deleterious genes accumulate in various ways, including direct selection for disease-associated phenotypes, linkage to selected traits, carriage by popular sires, genetic drift, andmost importantlythe absence of selection against deleterious phenotypes.

If we dont select for healthy parents to produce offspring, then we have no expectation of health in those offspring, Dr. Bell said. Not selecting for health is selecting for disease, and we need to understand that and pass that on to our breeder clients.

On the topic of disease and extreme phenotypes, Dr. Bell said brachycephalic obstructive airway syndrome is frequently diagnosed at veterinary clinics on account of the popularity of certain brachycephalic dog breeds, namely Pugs, French Bulldogs, and Bulldogs. Most breed standards do not call for the expression of extreme phenotypes, he said, nor do they select for the most extreme size or the most extreme brachycephalic trait.

Moderation away from extremes that cause disease should be the guiding principle in breeding, Dr. Bell noted, and in judging dog shows.

Common genetic diseases seen in mixed-breed dogs and cats occur randomly because of dispersed ancient liability genes, according to Dr. Bell. Uncommon and breed-specific recessive or complexly inherited disease is far less likely to occur in mixed-breed individuals.

Dr. Bell said designer-bred dogs and cats often have inherited diseases common in random-bred populations. They can also inherit disease liability genes shared by the parent breeds or parent species. So if youre breeding short-statured breeds together, it wouldnt be surprising to see patellar luxation, or in smaller toy size breeds, to see mitral valve disease, he said.

Hereditary disease manifests as a result of anatomical mismatch between parent breeds. We see a lot of this in dental disease, where we see crowding of teeth and malocclusions and misplaced teeth, Dr. Bell continued. Even in the musculoskeletal, if you breed two breeds with different body types together, we may see degenerative joint disease and poor joints. All of these things, all need to be monitored.

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Making sense of genetic disease in dogs and cats - American Veterinary Medical Association

Vertex Announces European Commission Approval for KALYDECO (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants With Cystic Fibrosis as Early as Four Months of Age

- Approval provides opportunity to treat the underlying cause of cystic fibrosis earlier than ever before in Europe -

LONDON 4 November 2020 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the EuropeanCommission has granted approval of thelabel extension for KALYDECO (ivacaftor) granules to include the treatment of infants with cystic fibrosis (CF) ages 4 months and older and weighing at least 5 kg who have the R117H mutation or one of the following gating (class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

Our very first CFTR modulator, KALYDECO, was first approved eight years ago, for certain CF patients ages 6 years and older. With todays approval, babies as young as 4 months are eligible and we believe early treatment is important in managing CF, said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. Todays approval is a testament to our commitment to keep going until all people with CF have a treatment option.

The label update is based on data from a cohort in the 24-week Phase 3 open-label safety study (ARRIVAL) consisting of six children with CF ages four months to less than six months who have eligible gating mutations.

KALYDECO (ivacaftor) will be now available to additional eligible patients in Germany and will be available shortly in countries that have entered into innovative long-term reimbursement agreements with Vertex, including the UK, Denmark and the Republic of Ireland. In all other countries, Vertex will work closely with relevant authorities in Europe to secure access for eligible patients.

KALYDECO (ivacaftor) is already approved in Europe for people with CF ages 6 months and older weighing at least 5 kg who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, R117H, S1251N, S1255P, S549N or S549R.

About Cystic Fibrosis

Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes one from each parent to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

About KALYDECO (ivacaftor)

Ivacaftor is the first medicine to treat the underlying cause of CF in people with specific mutations in theCFTRgene. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 inCambridge, Mass.,Vertex's global headquarters is now located inBoston'sInnovation Districtand its international headquarters is inLondon. Additionally, the company has research and development sites and commercial offices in North America,Europe,AustraliaandLatin America.Vertexis consistently recognized as one of the industry's top places to work, including11 consecutive years onScience magazine'sTop Employers listand a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visitwww.vrtx.comor follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special Note Regarding Forward-looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Reshma Kewalramani in this press release, and statements regarding the eligible patient population in Europe, our expectations regarding the timing of access to KALYDECO for eligible patients four months of age and older across countries in Europe, and our plans to secure access to KALYDECO for additional eligible patients four months of age and older in Europe. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, risks related to commercializing KALYDECO in Europe, and other risks listed under Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

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Study May Lead to Biomarkers for Tailoring COVID-19 Vaccines and Therapeutics

Potential Development of Human Monoclonal Antibody Therapeutics to SARS-CoV-2

Results from the PRECISION Study Expected in First Half of 2021

CHATHAM, N.J., Oct. 15, 2020 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced that the first participant was enrolled in the observational PRECISION study (TNX-C002), to examine the immune responses to COVID-19 in healthy volunteers who have recovered from COVID-19 or were asymptomatic. The research is part of an ongoing collaboration between Columbia University and Tonix that focuses on T cell and antibody responses to SARS-CoV-2 (CoV-2), the virus that causes COVID-19. The research encompasses two projects. The study led by Dr. Ilya Trakht, Ph.D., Associate Research Scientist at Columbia University Vagelos College of Physicians and Surgeons, has the potential to lead to the isolation and characterization of therapeutically relevant fully human monoclonal antibodies to SARS-CoV-2. The study led by Dr. Sergei Rudchenko, Ph.D., Assistant Professor of Medical Sciences at Columbia University Vagelos College of Physicians and Surgeons, is designed to generate DNA aptamer-based anti-idiotypes to selected monoclonal antibodies identified in Dr. Ilya Trakhts study. Such aptamers have the potential to identify biomarkers for protective CoV-2 immunity and may lead to accelerated precision medicine-driven vaccines designed to protect against COVID-19.

Data from the PRECISION study may help guide research to determine which vaccine or therapeutic is appropriate on an individual basis, said Seth Lederman, M.D., President and Chief Executive Officer of Tonix Pharmaceuticals. This work may also guide the selection of appropriate participants for clinical trials of COVID-19 vaccines, including future human trials using Tonixs TNX-1800, a live replicating, attenuated COVID-19 vaccine candidate designed to confer durable T cell immunity.

About TNX-1800

TNX-1800 is a live modified horsepox virus vaccine for percutaneous administration that is designed to express the Spike protein of the SARS-CoV-2 virus that causes COVID-19 and to elicit a predominant T cell response. Horsepox and vaccinia are closely related orthopoxviruses that are believed to share a common ancestor. Live replicating orthopoxviruses, like vaccinia or horsepox, can be engineered to express foreign genes and have been explored as platforms for vaccine development because they possess; (1) large packaging capacity for exogenous DNA inserts, (2) precise virus-specific control of exogenous gene insert expression, (3) lack of persistence or genomic integration in the host, (4) strong immunogenicity as a vaccine, (5) ability to rapidly generate vector/insert constructs, (6) readily manufacturable at scale, and (7) ability to provide direct antigen presentation. Relative to vaccinia, horsepox has substantially decreased virulence in mice1. Horsepox-based vaccines are designed to be single dose, vial-sparing vaccines, which can be manufactured on conventional cell culturing systems, with the potential for mass scale production.

1Noyce RS, et al. (2018) PLoS One. 13(1):e0188453

About Tonix Pharmaceuticals Holding Corp.

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing small molecules and biologics to treat and prevent human disease and alleviate suffering. Tonixs portfolio is primarily composed of central nervous system (CNS) and immunology product candidates. The immunology portfolio includes vaccines to prevent infectious diseases and biologics to address immunosuppression, cancer and autoimmune diseases. The CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonixs lead vaccine candidate, TNX-1800*, is a live replicating vaccine based on the horsepox viral vector platform to protect against COVID-19, primarily by eliciting a T cell response. Tonix expects data from animal studies of TNX-1800 in the fourth quarter of this year. TNX-801*, live horsepox virus vaccine for percutaneous administration, is in development to protect against smallpox and monkeypox and serves as the vector platform on which TNX-1800 is based. Tonix is also developing TNX-2300* and TNX-2600*, live replicating vaccine candidates for the prevention of COVID-19, but using bovine parainfluenza as the vector. Tonixs lead CNS candidate, TNX-102 SL**, is in Phase 3 development for the management of fibromyalgia. The Company expects topline data in the Phase 3 RELIEF study in the fourth quarter of 2020. Tonix is also currently enrolling participants in the Phase 3 RALLY study for the management of fibromyalgia using TNX-102 SL, and the results are expected in second half of 2021. TNX-102 SL is also in development for agitation in Alzheimers disease and alcohol use disorder (AUD). Both programs are Phase 2 ready, and the AAD program has FDA Fast Track designation. Tonixs programs for treating addiction conditions also include TNX-1300* (T172R/G173Q double-mutant cocaine esterase 200 mg, i.v. solution), which is in Phase 2 development for the treatment of life-threatening cocaine intoxication and has FDA Breakthrough Therapy designation. TNX-601 CR** (tianeptine oxalate controlled-release tablets) is another CNS program, currently in Phase 1 development as a daytime treatment for depression while TNX-1900**, intranasal oxytocin, is in development as a non-addictive treatment for migraine and cranio-facial pain. Tonixs preclinical pipeline includes TNX-1600** (triple reuptake inhibitor), a new molecular entity being developed as a treatment for PTSD; TNX-1500* (anti-CD154), a monoclonal antibody being developed to prevent and treat organ transplant rejection and autoimmune conditions; and TNX-1700* (rTFF2), a biologic being developed to treat gastric and pancreatic cancers.

*TNX-1800, TNX-801, TNX-2300, TNX-2600, TNX-1300, TNX-1500 and TNX-1700 are investigational new biologics and have not been approved for any indication.

**TNX-102 SL, TNX-601 CR, TNX-1600 and TNX-1900 are investigational new drugs and have not been approved for any indication.

This press release and further information about Tonix can be found at http://www.tonixpharma.com.

Forward Looking Statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as anticipate, believe, forecast, estimate, expect, and intend, among others. These forward-looking statements are based on Tonix's current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties caused by the global COVID-19 pandemic; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2019, as filed with the Securities and Exchange Commission (the SEC) on March 24, 2020, and periodic reports filed with the SEC on or after the date thereof. All of Tonix's forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.

Contacts

Jessica Morris (corporate)Tonix Pharmaceuticalsinvestor.relations@tonixpharma.com(212) 688-9421

Travis Kruse (media)Russo Partnerstravis.kruse@russopartnersllc.com(212) 845-4272

Peter Vozzo (investors)Westwickepeter.vozzo@westwicke.com(443) 213-0505

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Tonix Pharmaceuticals Enrolls First Participant in the PRECISION Study, an Observational Study to Facilitate Development of Precision Medicine...

In July, we exited the Stay-at-Home Economy phase of the COVID-19 pandemic and entered a chapter we call the Re-Opening Economy a transitional stage that seeks to balance progress toward economic normalcy with limiting the virus' spread. Many businesses and schools have since carefully welcomed back customers, employees, and students, often instituting new policies requiring social distancing and wearing personal protective equipment (PPE). However, even for the world's top medical professionals and policymakers, there exist many unknowns associated with re-opening, and the risk of resurgent outbreaks remains high. Amid this uncertainty, a handful of groundbreaking technologies like Cloud Computing, Genomics, the Internet of Things (IoT), and Telemedicine are playing critical roles in promoting safety while maintaining flexibility. Ultimately, they could prove key to advancing to the final stage of this global crisis: The New Normal Economy.

Sandwiched between the two extremes of the largely shut-down Stay-at-Home Economy and a fully opened New Normal Economy, the Re-Opening Economy is an uncertain, transitional phase. Without a widely distributed COVID-19 vaccine, future outbreaks remain a looming threat, while the economic and emotional tolls of the Stay-at-Home Economy create mounting pressure to resume in-person learning and work.

Government officials and business leaders are re-opening in phases, hoping to balance these competing forces by gradually resuming in-person interactions. Supportive policies can include enforcing mandatory mask-wearing, encouraging outdoor dining, reducing office-capacity, and alternating schedules for students' and employees' in-person attendance. Over time, these policies can loosen or tighten depending on their success.

Yet despite these efforts to prevent new outbreaks, weddings, college campuses, and Wall Street trading floors were recently responsible for a handful of localized COVID-19 outbreaks. Responses to these spreader-events are often steps backward; employees and students are sent home, while public gatherings are further reined in. Therefore, the Re-Opening Economy is not a linear path to the New Normal Economy. Policies and social norms are changing on the fly, while we adapt and learn from this deadly virus.

Navigating the tenuous environment requires an approach rooted in two critical goals: 1) Maximizing the safety of stakeholders like employees, students, and customers, and 2) Minimizing the impact future outbreaks may have on business and society by improving flexibility and resilience. We have identified multiple themes that we believe play an essential role in achieving these goals, including Cloud Computing, Genomics, the Internet of Things, and Telemedicine. In our view, the successful and widespread implementation of the technologies could accelerate our progress towards overcoming this pandemic.

Cloud Computing

Cloud Computing broadly describes the digital infrastructure that gives us access to applications, files, and data over the internet. Whereas in the past this functionality was constrained to on-premises desktops and servers, the cloud brings this information to any number of connected devices around the world. Over the past decade, many enterprises already began shifting their IT to cloud-based platforms. But ensuring business continuity amid global shutdowns brought work-from-home to the forefront, solidifying Cloud Computing as an essential pillar of the Stay-at-Home Economy.

Many services-based industries, for example, saw successful rapid implementations of cloud-based solutions, resulting in surprising business resilience throughout the pandemic, despite offices widely remaining closed. Employees used videoconferencing to connect with clients, remote server connections to access data and files, internal chat platforms to communicate with team members, and specialized browser-based software to manage business operations

While re-opening efforts suggest the work-from-home trend could be reversing, both employer and employee preferences indicate its likely here to stay. On the enterprise side, many companies simply do not want to take the risk of bringing workers back into their offices before a vaccine is readily available. Major employers like Google (NASDAQ:GOOG) (NASDAQ:GOOGL), Amazon (NASDAQ:AMZN), and Uber (BER) announced that they will not require employees to return to their corporate offices until 2021. And an even greater fundamental shift is underway at firms like Facebook (NASDAQ:FB), Twitter (NYSE:TWTR), and Square (NYSE:SQ), where employees can now opt to work from home indefinitely.1 For employees, work-from-home affords them greater flexibility, and tellingly, a recent survey found that 40% of employees indicated a preference to work from home for 3+ days a week versus just 14% prior to the pandemic.2

As we face continued re-opening uncertainties and preferences indicate a hesitation to return to the office, work-from-home increasingly appears to be a long-term trend that should benefit companies across the Cloud Computing ecosystem.

Genomics

Genomics is the study of the order, structure, and function of genetic material. It gives us information that can prove valuable in developing diagnostics, treatments, and/or cures for diseases and other afflictions. Over the last two decades, the cost of sequencing a whole human genome dropped by 99.9%, paving the way for a massive collection of genetic data across populations, organisms, and pathogens.3 But it doesn't end there: more data means more possibilities to analyze and apply it. In recent years, hundreds of genomics-derived drugs and treatments entered clinical trials in the United States. Many of these have the potential to mitigate previously incurable conditions like cancer, assorted heart, eye, lung, and liver-related ailments, and rare genetic diseases.4

Genomic sub-sciences proved essential in lessening the impact of the pandemic at its onset. Modern genomic sequencing and analysis allowed researchers to identify COVID-19 as a novel pathogen in less than 10 days, leading to the prompt development of diagnostics which today inform our mitigation strategies. For comparison, the virus behind 2003's SARS outbreak took over three months to sequence.

Extending the impact of modern sequencing, genomic data has proved invaluable in kickstarting treatment and vaccine development. There are more than 529 COVID-19 treatments and vaccines in development with 35 in clinical trials as of September 29th, 2020. Notably, one of the eight candidates in phase III trials is an RNA-based vaccine. This type of vaccine seeks to use portions of viral genomes to induce immune responses without actually injecting pathogens.5

Until there is a vaccine that is effective, widely distributed, and embraced, massive testing and diagnostics will likely remain the predominant mitigation strategy. While country-level efforts for the most part reflected this sentiment from the get-go, private organizations are increasingly embracing their testing procedures as a path to re-opening. Major League Baseball, for example, tests players and staff every two days. The league has conducted well over 140,000 tests since its season began in July, successfully preventing multiple team-specific infections from escalating to a league-wide outbreak.6 Following the success of this approach, companies and universities are likely to institute similar testing policies to protect students, employees, and the communities they reside in. Implementing testing on such a broad scale would likely entail administering millions, if not billions of tests, benefiting genomics and biotech companies with exposure to the diagnostics value chain.

Internet of Things

The internet of things refers to the growing array of internet-connected devices, including smart home appliances, autonomous vehicles, wearables, smart factories, and next-generation infrastructure. Prior to the COVID-19 pandemic, consumers demonstrated a growing preference for smart devices, increasingly buying TVs with internet connections and watches sophisticated enough to supplement their smartphones. But as the pandemic unfolded, technologists realized internet-connected devices could potentially help identify symptoms and enforce social distancing.

Popular wearable health trackers, like the Apple Watch or Fitbit, continually collect heart rate and blood oxygen data from their users. Researchers theorized that measuring this data across thousands of subjects, could uncover population health patterns attributable to COVID-19. Preliminary results from a Fitbit study of 100,000 participants add credence to this idea. In 50% of instances where a positive test followed, Fitbit data detected COVID-19 one day before subjects noticed symptoms.7 The study implies that wearables may help with earlier identification of infection and can therefore recommend formal testing and/or quarantine to prevent the virus from spreading more broadly.

Beyond wearables, other IoT devices can help enforce social distancing and detect symptoms. For example, temperature monitors are increasingly used in airports, schools, and other heavily-trafficked areas to identify those with elevated temperatures. The connected nature of these devices enables data to be transmitted and centralized, offering health officials greater visibility of population-level health. Additionally, proximity sensors can help monitor adherence to social distancing guidelines, while near-field communication chips can play a part in alerting individuals if they encounter infected persons.

Beyond wearables and other sensor-equipped devices, there are a handful of use-cases for internet-connected devices that could prove invaluable to the re-opening process. These include drones and robots that can also help facilitate social distancing and limit person-to-person contact.

Telemedicine

Telemedicine connects doctors and patients over the internet, enabling virtual consultations and remote monitoring of patients' health. Prior to COVID-19, these technologies saw limited adoption as deeply engrained stakeholder practices proved difficult to disrupt. Yet as the pandemic unfolded, providers, patients, and payers quickly realized the benefits of telemedicine. By connecting with doctors through videoconferencing services and mobile health applications, patients could avoid the pandemic-related dangers of in-person visits and reduce costs by $19-121 per visit.8 From February to April 2020, telemedicine usage surged more than 30x, with related medical claims soaring from 0.4% to 13%.9

We anticipate telemedicine playing a continued essential role in the Re-Opening Economy given the flexibility and safety it offers. In addition, telemedicine represents just one facet of health care's digital transformation that should see broader acceleration as health systems seek to improve patient outcomes and improve efficiency.

Though the Re-Opening Economy roughly began in July, the lack of a vaccine and continued flare-ups of COVID-19 around the world suggest that this transitional phase is likely to remain in place for an extended time frame. The new norms and policies introduced to combat the virus will force economies and societies to adapt and may have lasting impacts beyond the current pandemic. Work-from-home could become a standard option for employees. IoT implementation in buildings and public spaces could become a part of standard health and security practices. Frequent diagnostics and telemedicine could become default approaches to medicine and health care.

And while the world will recover, it will not return to the Pre-COVID Economy of yesteryear. Such potential for permanent change and disruption is why we refer to the next phase of our recovery from the COVID-19 pandemic as the "New Normal Economy." We expect that many of the themes and technologies that enabled the Stay-at-Home Economy, facilitated the Re-Opening Economy, and could feature prominently in the New Normal Economy, will play vital roles in our lives moving forward.

CLOU: The Global X Cloud Computing ETF seeks to invest in companies positioned to benefit from the increased adoption of cloud computing technology, including companies whose principal business is in offering computing Software-as-a-Service (SaaS), Platform-as-a-Service (PaaS), Infrastructure-as-a-Service (IaaS), managed server storage space and data center real estate investment trusts, and/or cloud and edge computing infrastructure and hardware.

SNSR: The Global X Internet of Things ETF seeks to invest in companies that stand to potentially benefit from the broader adoption of the Internet of Things (IoT), as enabled by technologies such as WiFi, 5G telecommunications infrastructure, and fiber optics. This includes the development and manufacturing of semiconductors and sensors, integrated products and solutions, and applications serving smart grids, smart homes, connected cars, and the industrial internet.

GNOM: The Global X Genomics & Biotechnology ETF seeks to invest in companies that potentially stand to benefit from further advances in the field of genomic science, such as companies involved in gene editing, genomic sequencing, genetic medicine/therapy, computational genomics, and biotechnology.

EDOC: The Global X Telemedicine & Digital Health ETF seeks to invest in companies positioned to benefit from further advances in the field of telemedicine and digital health. This includes companies involved in Telemedicine, Health Care Analytics, Connected Health Care Devices, and Administrative Digitization.

1. Employee data from Bloomberg, accessed on 8/31/2020.

2. Iometrics, Global Workplace Analytics, Global Work From Home Experience Survey, 2020.

3. NIH, National Human Genome Research Institute, 2018.

4. NHGI, 2018.

5. Global X ETFs, Milken Institute, Aug 21, 2020

6. Major League Baseball, "Latest MLB COVID-19 Test Results," Sep 25, 2020.

7. Fitbit, "Early Findings from Fitbit COVID-19 Study Suggest Fitbit Devices Can Identify Signs of Disease at Its Earliest Stages," Aug 19, 2020.

8. The American Journal of Emergency Medicine, "On-demand synchronous audio video telemedicine visits are cost effective," Aug 7, 2018.

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Thematic companies may have limited product lines, markets, financial resources or personnel. They typically engage in significant amounts of spending on research and development, capital expenditures and mergers and acquisitions.

The risks related to investing in cloud computing companies include disruption in service caused by hardware or software failure, interruptions or delays in service by third-party data center hosting facilities and maintenance providers, security breaches involving certain private, sensitive, proprietary and confidential information managed and transmitted by cloud computing companies, and privacy concerns and laws, evolving Internet regulation and other foreign or domestic regulations that may limit or otherwise affect the operations of such companies.

Investing in securities engaged in the social media industry include disruption in service caused by hardware or software failure; interruptions or delays in service by third-parties; security breaches involving certain private, sensitive, proprietary and confidential information managed and transmitted by social media companies; and privacy concerns and laws, evolving Internet regulation and other foreign or domestic regulations that may limit or otherwise affect the operations of such companies.

Information Technology companies can be affected by rapid product obsolescence, and intense industry competition. Risks include disruption in service caused by hardware or software failure; interruptions or delays in service by third-parties; security breaches involving certain private, sensitive, proprietary and confidential information managed and transmitted; and privacy concerns and laws, evolving Internet regulation and other foreign or domestic regulations that may limit or otherwise affect the operations. Healthcare, Pharmaceutical, Biotechnology and Medical Device companies can be affected by government regulations, expiring patents, rapid product obsolescence, and intense industry competition.

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Tools developed to probe the virome could aid in variety of research

Researchers at Washington University School of Medicine in St. Louis have received an $8.5 million grant to study the role of gut viruses in inflammatory bowel disease. Tools developed in the course of the project could accelerate research into other roles of the virome in health and disease.

The communities of bacteria that live in our digestive tracts help digest food and produce vitamins, protect against pathogens, and promote the healthy functioning of our immune system. But alongside gut bacteria thrives a vast community of viruses, and we know little about their impact on health and disease.

Efforts to study the gut viral community known as the virome have been hindered by a lack of tools to analyze viral diversity. Researchers at Washington University School of Medicine in St. Louis have received an $8.5 million grant from the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health (NIH) to develop the tools needed to study the role of the virome in inflammatory bowel disease. Once developed, such tools could be applied widely, opening up new avenues of research into the role of the virome in normal physiology and development, as well as diseases such as diabetes, AIDS and cancer.

The virome has been linked to a number of conditions inflammatory bowel disease, malnutrition, graft-versus-host disease and there is also some evidence that the virome supports human health in some ways, said principal investigator David Wang, PhD, a professor of molecular microbiology, and of pathology and immunology. But the problem that plagues virome studies is that people find an association, and then they cant pursue it. Once you find an association, the next step is to see what happens when you introduce the virus to an animal. Does it cause the disease? Make it worse? But there are no tools to carry this out.

Tools to analyze the viral community are relatively scarce partly because viruses are more diverse than bacteria. All bacteria carry certain basic housekeeping genes necessary for life, notably the 16S ribosomal RNA gene. Scientists use this universal gene to screen mixed communities of unidentified bacteria by pulling out all the 16S ribosomal RNA genes and using the sequences to classify the bacteria into families. There is no equivalent universal gene among viruses.

Wang and colleagues previously have discovered differences between the viromes of people with inflammatory bowel disease and healthy people. Inflammatory bowel disease is caused by chronic inflammation in the digestive tract and characterized by persistent diarrhea and abdominal pain. The researchers found that people with the condition carry more Caudovirales, a group of viruses that infects bacteria, and anelloviruses, a family of viruses that infects human cells, in their intestines. But they do not yet know what, if anything, the presence of these viruses means.

The new grant will allow the researchers to follow a group of people with inflammatory bowel disease over time, along with healthy members of their households for comparison. Inflammatory bowel disease tends to be cyclical, flaring up and then resolving again and again. By taking repeated stool samples and analyzing the genetic material of the viruses in such samples, the researchers will be able to see how the makeup of the gut viral community changes over the course of the disease, and gauge whether any particular groups of viruses become more abundant during flare-ups or resolutions. They also will assess what effect treatment has on the virome.

Such analysis will require the development of computational tools to identify the viruses by their genetic sequences, classify them into family groups, identify potential genes within viral sequences, and propose functions of the genes.

With the tools we have now, more than half the sequences cant be classified because they are not similar enough to known sequences, Wang said. We frequently cant even tell whether weve found a virus that infects bacteria or human cells.

Wang and colleagues also will develop ways to cultivate viruses so they can study them. As nonliving things, viruses require a living cell to multiply, which makes cultivation in the lab tricky. To grow viruses that infect human cells, researchers must first culture human cells and then infect them with viruses. But the majority of the viruses in the intestinal tract are likely to infect bacteria, not human cells. Such viruses known as bacteriophages, Latin for bacteria eaters are even more complicated: Researchers must first identify the correct bacterial species from among the thousands in our intestines, culture that species, and then attempt to grow the virus within the bacterial culture.

In previous work, we established the first culture system for a gut virus, Wang said. Were relying on our experience there to try to culture more of these novel viruses. Some of these might actually grow in a quite straightforward way, its just that no one has tried yet. And once we have the viruses, then we can use them to start doing experiments in animal models of inflammatory bowel disease.

The impact of the gut bacterial community on human health is a hot topic of study, with a possible role in health conditions ranging from autoimmunity to heart disease to psychiatric illnesses. The virome may prove to be equally consequential if only we can find a way to investigate it.

This isnt a typical grant, because part of its goal is to build resources that will then be available to the scientific community, Wang said. Creating tools is unsexy and usually unfundable. But we have to build these tools before we can answer the exciting questions.

Along with Wang, the research team includes Michael S. Diamond, MD, PhD, the Herbert S. Gasser Professor of Medicine, and Scott Handley, PhD, an associate professor of pathology and immunology, both at Washington University; Thaddeus Stappenbeck, MD, PhD, of the Cleveland Clinic; and collaborators at Cambridge University in the United Kingdom, San Diego State University in California, and Flinders University in Australia.

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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Role of gut viruses in inflammatory bowel disease is focus of $8.5 million grant - Washington University School of Medicine in St. Louis

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces two presentations at the European Society for Immunodeficiencies (ESID) 2020 Meeting to be held virtually October 14-17, 2020. An oral presentation will provide an update on data from the Phase 1/2 clinical trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). An e-poster will highlight preclinical study data on RP-L401 for Infantile Malignant Osteopetrosis (IMO).

Additional presentation details can be found below:

Oral Presentation

Title: A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1 Session Title: TreatmentPresenter: Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los AngelesSession Date: Friday, October 16, 2020Session Time: 10:45 a.m. 12:01 p.m. CESTLecture Time: 11:45 a.m. CESTLocation: Hall D

This session will be followed by a Q&A from 12:01 p.m. to 12:30 p.m. CEST

E-Poster

Title: Preclinical Efficacy and Safety of EFS.HTCIRG1-LV Supports IMO Gene Therapy Clinical Trial InitiationPresenter: Ilana Moscatelli, Ph.D., Associate Researcher, Division of Molecular Medicine and Gene Therapy, Lund University, Sweden

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I (less than 2% normal expression) are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other Agency of the State of California.

About Infantile Malignant Osteopetrosis

Infantile Malignant Osteopetrosis (IMO) is a rare, severe autosomal recessive disorder caused by mutations in the TCIRG1 gene, which is critical for the process of bone resorption. Mutations in TCIRG1 interfere with the function of osteoclasts, cells which are essential for normal bone remodeling and growth, leading to skeletal malformations, including fractures and cranial deformities which cause neurologic abnormalities including vision and hearing loss. Patients often have endocrine abnormalities and progressive, frequently fatal bone marrow failure. As a result, death is common within the first decade of life. IMO has an estimated incidence of 1 in 200,000. The only treatment option currently available for IMO is an allogenic bone marrow transplant (HSCT), which allows for the restoration of bone resorption by donor-derived osteoclasts which originate from hematopoietic cells. Long-term survival rates are lower in IMO than those associated with HSCT for many other non-malignant hematologic disorders; severe HSCT-related complications are frequent. There is an urgent need for additional treatment options.

RP-L401 was in-licensed from Lund University and Medizinische Hochschule Hannover.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding when clinical trial sites will resume normal business operations, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-Q for the quarter ended June 30, 2020, filed August 5, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Two Presentations at the European Society for Immunodeficiencies 2020 Meeting - Business Wire

Last week geneticist Dr Charles Steward shared with us his experiences of searching for a genetic cause for his childrens rare neurological diseases. Here he gives us a deeper look at how genomic medicine is evolving and the barriers that are preventing it from reaching its full potential.

Through his work at the Wellcome Sanger Institute and Congenica, and involvement with the 100,000 Genomes Project, Charlie has been well placed to see the development of genomics over the years and the promises that it will bring to healthcare. However, he says there is always the danger of overpromising the benefits and at the same time, underdelivering.

I saw this, in particular, with the finishing of the human genome, says Charlie. At that time, we thought this would be the holy grail for medicine. Many of the promises we made were not forthcoming, in particular to the pharma industry.

In reality, finishing the human genome was the first step of what is a long journey.

Unsurprisingly, in many respects, the human genome turned out to be a lot more complex than was originally thought.

While there may be just under 20,000 confirmed protein coding genes, it turns out that much of the genome outside of these genes is also important in regulating how the genome is controlled. For example, we know that not all genes are expressed in all tissues and that not all genes are expressed during all developmental stages.

Now, however, the field is changing with respect to genomic medicine.

This is essential information for the pharma industry to know about, says Charlie. If you are developing a drug, for example, for infantile epilepsy, then you need to know if your drug target is expressed in the brain and also during early development.

Likewise, some patients may have a different version of a gene with respect to people on whom a drug has been tested, which means that drug might not work.

While the ability to specifically target the genome for therapy for all patients is not yet a reality, the time will come when it will be.

Already, we know of some types of epilepsy where the underlying genome can inform on specific therapy, says Charlie.

For example, people who have epilepsy caused by mutations in the gene SCN1A should not be prescribed sodium channel blockers, as this can make their epilepsy worse. Another example is pyridoxine-dependent epilepsy, which can result in severe developmental regression, caused by mutations in the gene ALDH7A1. Yet, this can be treated simply by administering a type of vitamin B6.

While it is a very rare form of epilepsy, it is such a cheap and harmless medication that it is often used as a first-line therapy for infants in intensive care who have no genetic diagnosis.

This is where the future of medicine lies where we are able to read a patients genome and then direct care and therapy based upon that.

Genomics also means we will be able to stratify patients, based upon their genomic makeup, to make clinical trials much more efficient and targeted.

Currently, the approach is akin to throwing darts in the dark, where a clinical trial may use the same drug across a whole range of patients, each with a very different genomic makeup, in the hope that a drug will work, says Charlie.

For example, this approach was taken with the International Collaborative Infantile Spasms Study (ICISS) clinical trial that my daughter was on, where therapy was administered to patients with no idea of the underlying genome.

While the clinical trial was successful in stopping seizures in around 72% of patients, which is an amazing result, nearly one third of patients did not respond. This is indicative of patients having different underlying aetiologies.

In such epilepsy studies, where every seizure causes catastrophic brain malfunctioning, time is of the essence to prevent severe and irreversible developmental regression.

Had clinicians known that this cohort was not going to respond to the clinical trial, perhaps because of their genomic makeup, a different treatment pathway could have been chosen, says Charlie.

On one hand, unfortunately this means that many drugs are doomed to failure. On the other hand, many drugs that have been developed so far, but have not made it into the clinic, are worth revisiting, to see if their effectiveness can be improved with better genomic insight.

However, there are still hurdles to overcome to understand completely how a patients genome impacts their health.

Enhancing sequencing

Luckily there have been some encouraging developments in sequencing technology in recent years.

Currently, the most common way of looking at genomes in these settings is by using short-read technology.

The human genome is too long to be sequenced as one continuous string by current technology so short-read sequencing breaks DNA into short fragments that are amplified and then sequenced to produce reads of around 150 nucleotides in length. Bioinformatic techniques are then used to piece together the reads into a continuous genomic sequence by aligning them to the reference human genome.

That works really well most of the time, says Charlie, but if a patient has a region of the genome thats deleted, or expanded, its very difficult for short-read technology to understand that. If you look at a region that is repeated or deleted, by, for example, 10,000 nucleotides in length and youve got a tiny read, you cant match that to the genome with any confidence.

To address these issues, scientists and clinicians are starting to look at long-read sequencing.

These technologies directly sequence single molecules of DNA in real time, often without the need for amplification. This allows for much lengthier reads.

Through this, we are able to more clearly resolve large changes in the genome, says Charlie. Thats going to be really helpful, because we know that large changes in the human genome are responsible for a lot of developmental disorders such as epilepsy and autism.

Most current technology actually focuses on looking at the bits of the genome that produce proteins. As mentioned, this accounts for roughly 1-2% of the genome, so theres 98% that were not actually looking at yet because we dont know what it does.

Its likely to contain what we call control regions parts of the genome that control whether a gene is turned on or off. We need to start finding technologies that help us to understand these regions more fully.

At the same time as trying to understand what those regions do, researchers may also be able to measure to what degree a gene is being expressed.

If you can see that you have a gene thats being massively over-expressed or under-expressed in a patient, with respect to a normal person, it may also be an indicator that theres something wrong, says Charlie.

There are lots of technologies already out there, but we still need to wait for them to be included in general clinical practice. Its still difficult to interpret a lot of these results. From a research point of view, you can make all sorts of guesses and hypotheses, but that cant really apply in a clinical setting.

Clearly, there are still challenges in integrating genomic medicine into everyday healthcare, yet great strides are already being made. For example, we are beginning to see genomic medicine being used to treat disorders caused by a single faulty gene, so the ability to replace the gene (or the affected part of the gene) should help.

One such example uses the adeno-associated virus (AAV), a small non-pathogenic virus that lives in humans (the way gut bacteria do) and travels freely around the body, including crossing the blood-brain barrier unimpeded.

Scientists are removing the virus DNA and replacing it with a normal copy of a faulty gene in the specific genetic nervous system disease.The virus then travels and delivers the normal gene to the cells making them functional again. This has recently led to an approved therapy for spinal muscular atrophy.

Such technologies have immense potential to bring relief to patients with severe genomic disorders, although there remain ethical concerns about how these techniques could be used.

The future will only see more of these technologies being embraced and it is clear that the pharma and genomics industry must start talking to each other again in earnest, Charlie says. We are entering the age where genomic medicine is no longer a pipe dream but actually becoming a reality.

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The future of genomic medicine: can it fulfil its promises? - - pharmaphorum

Locusts have a reputation of biblical proportions.

Certain species of grasshoppers that are typically solitary and harmless can suddenly swarm and consume entire crops, including plants that support livestock. Large swarms can destroy livelihoods for farmers and entire communities food supply.

That is why researchers fromTexas A&M AgriLife,Baylor College of Medicine,Arizona State University,Washington University in St. LouisandUniversity of California, Davis, have created the Behavioral Plasticity Research Institute (BPRI).

The institute will work to understand the mechanisms behind locust swarms and migration, and use this knowledge to develop effective methods to limit the destruction the swarms can leave behind.

The cross-institutional, multi-disciplinary effort is led byHojun Song, associate professor, and Spencer Behmer, professor, both in the Texas A&M College of Agriculture and Life SciencesDepartment of Entomology;Fabrizio Gabbiani, professor of neuroscience, and Dr. Herman Dierick, associate professor of molecular and human genetics, Baylor; and Arianne Cease, associate professor of sustainability and director of the Global Locust Initiative at Arizona State University.

The team with wide-ranging expertise also includesGregory Sword, professor, Texas A&M Department of Entomology; Erez Lieberman, assistant professor, and Chenghang Zong, assistant professor, Baylor; Rick Overson, senior scientist, Arizona State; Stephen Richards, project scientist, Earth BioGenome Project, UC Davis; andBarani Raman, professor, Washington University.

A$12.5 million, five-year National Science Foundationgrant provides funding for the BPRI. The institute is one of four inaugural Biology Integration Institutes established by the NSF this year to work on broad problems in biology.

Using cutting-edge technologies in research projects spanning from molecules to landscapes, the BPRI will greatly enhance our understanding of how grasshoppers transform into locusts a phenomenon called locust phase polyphenism and develop innovative solutions to manage locust plagues, Gabbiani said. With a commitment to improving diversity, inclusion and equity, the institute will train the next generation of integrative biologists who can efficiently navigate across disciplines to reach this goal.

The institute will communicate groundbreaking research to the general public and the scientific community. By partnering with the Global Locust Initiative hosted at Arizona State, the institute plans to translate its scientific findings to real-world management with a goal of improving global food system sustainability.

Currently, when locusts outbreak, they can affect one in 10 people globally. The impact and the benefits to society that might come from this institute are pretty enormous.

The phenomenon BPRI will study, locust phase polyphenism, is a prime example of how distinct phenotypes can arise from environmental and other cues, rather than only genetic information. In the case of grasshopper species that are considered to be locusts, typically harmless insects can change their behavior in response to certain environmental and sensory cues to become a cohesive swarm.

The changes locusts undergo belong to a broad scientific concept known as phenotypic plasticity, the ability of organisms to change in response to their environment. Phenotypic plasticity is common in nature. But, to fully understand its mechanisms, maintenance and evolution, biological integration is needed, Song said. This work also illuminates how gene expression patterns and epigenetic regulation are linked to shifts in behavior, physiology and ecology that result in outbreaks, collective movement and mass migration.

So, the team expects this work to eventually lend itself to more than just insects. The information learned will help to understand how environment influences genetic makeup to shape behavior across all animals.

Currently, when locusts outbreak, they can affect one in 10 people globally, Behmer said. The impact and the benefits to society that might come from this institute are pretty enormous.

To better understand the scope of locusts impact, one must consider the situation of places affected by swarms, Sword said.

When we have disasters in developed countries, we have mechanisms in place to get people support and relief they need, Sword said. But in a country dependent on small-holder subsistence agricultural operations, a locust swarm can literally take away a familys entire source of income and food for the year.

To gain a fuller understanding of the problem and provide individualized solutions, the institute will involve people with diverse backgrounds.

Every step of the way, we will ask the question of whether we are being inclusive and hearing all the perspectives, Song said. We need to work across subdisciplines and try to get at the big picture rather than focusing on little slices. I believe that by bringing all these people together, within the next five or 10 years, we can make amazing changes.

Researchers with the BPRI plan to carry out 10 integrative research activities. The projects will use three locust and three non-swarming grasshopper species with varying degrees of plasticity. The researchers will work with genomes, tissue-specific and time-resolved transcriptomes and epigenomes, as well as CRISPR/Cas9 and reverse genetics tools to understand the functional genetics of locust phase polyphenism, all considered within an evolutionary framework.

Well be studying the factors that nudge individual locusts to join a larger group and the changes that follow, Raman said. Given the reports of massive, destructive locust swarms in many African and Asian countries this year, this is indeed a timely investigation of a well-reported, but not yet fully understood phenomenon.

In essence, the institute will aim to solve problems humans have faced for thousands of years due to locusts, Song said.

Weve had the locust problem for millennia, Song said. But, we still struggle to control these pests. I believe that the discoveries made through the BPRI will fundamentally transform our understanding of why and how locusts swarm, which will ultimately translate into sustainable management practices.

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New Institute To Study Behavioral Plasticity In Locusts - Texas A&M Today - Texas A&M University Today

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Tessera Therapeutics, a biotechnology company pioneering a new approach in genetic medicine known as Gene Writing, announced today the appointment of Howard Liang, Ph.D., as President and Chief Financial Officer. The company also expanded its executive bench with newly promoted talent and hires: Madhusudan Peshwa, Ph.D., as Chief Technology Officer for Cell Therapy; Bill Querbes, Ph.D., as Senior Vice President, Therapeutic Discovery & Translational Sciences; Cecilia Cotta-Ramusino, Ph.D. as Senior Vice President, Platform Development; Vikram Ranade, Ph.D., as Senior Vice President, Corporate Development; David Pollard, Ph.D., as Head of Bioprocess, and Steve Garbacz as Head of Finance.

These additions represent the latest leadership expansion for the company, following the appointments of Elliott Sigal, M.D., Ph.D., and Mary Rozenman, Ph.D., to the Board of Directors in June, and the appointments of David Davidson, M.D., as Chief Medical and Development Officer, Hari Pujar, Ph.D., as Chief Operating Officer, and Lin Guey, Ph.D., as Senior Vice President of Rare Diseases Program Strategy and Operations in March. Tessera also announced the successful completion of $230 million Series B financing in January.

Outstanding people are the lifeblood of great companies and Im thrilled to welcome these accomplished individuals to the Tessera leadership team, said Dr. Geoffrey von Maltzahn, CEO and Co-Founder of Tessera and General Partner, Flagship Pioneering. Howards track record in both strategy and finance at BeiGene and in the capital markets will play a key role in guiding Tessera to new territory in Gene Writing. I am excited to be working with him, and our other new senior leaders, each of whom will be instrumental in expanding the limits of how we discover life-changing medicines.

Howard Liang, Ph.D., President and Chief Financial OfficerHoward Liang joined Tessera in 2021 as President and Chief Financial Officer. Dr. Liang brings nearly three decades of combined experience in management, financing, strategy, and research in the biotechnology and pharmaceutical industries and investment research on Wall Street. Prior to joining Tessera, he was Chief Financial Officer and Chief Strategy Officer at BeiGene for six years, where he was a member of the senior team that led the companys growth from a research organization with fewer than 200 employees to a fully integrated global biotechnology company with more than 6,000 employees on five continents. At BeiGene, he led the companys IPOs on NASDAQ and the Hong Kong Stock Exchange and its ongoing effort to list on the Shanghai Stock Exchange, raising more than $8 billion to date through equity and alternative financings, and overseeing the growth of the companys market capitalization from less than $300 million to more than $30 billion during his tenure. Prior to BeiGene, Dr. Liang spent 10 years at Leerink Partners, where he was Managing Director and Head of Biotechnology Equity Research. His prior investment research experience included positions at A.G. Edwards, JMP Securities, and Prudential Securities, covering biotechnology, and major and specialty pharmaceutical sectors. He started his career in R&D at Abbott Laboratories, where he was a Senior Scientist and member of an industry-leading structure-based drug discovery team. Dr. Liang is a member of the Hong Kong Stock Exchange Biotech Advisory Panel. He was named a member of the All-America Research Team by Institutional Investor magazine and Best of the Street by The Wall Street Journal. As a scientist, he authored 14 papers, including 6 in Nature, Science, and Proceedings of the National Academy of Sciences, and a review in the Journal of Molecular Biology. He received his Ph.D. in Biochemistry and Molecular Biology and M.B.A. from the University of Chicago and his B.S. in Chemistry from Peking University.

Tessera is developing a first-of-its-kind technology with the potential to cure diseases across multiple categories by writing in the code of life itself, said Dr. Howard Liang. I look forward to helping the company realize the full breadth of Gene Writings potential.

Madhusudan Peshwa, Ph.D., Chief Technology Officer for Cell TherapyDr. Peshwa joined Tessera in May 2021 and is responsible for developing the strategy and executing the operating plan encompassing the design, development, and manufacture of Tesseras proprietary mobile gene element engineered cell therapy product portfolio. Recently, in March 2020, Dr. Peshwa was inducted into the College of Fellows at the American Institute for Medical and Biological Engineering (AIMBE), in recognition of Lifetime contributions in Regenerative Medicine to the advancements in the field of cell & gene therapies.

Prior to joining Tessera, Dr. Peshwa was CTO at Mana Therapeutics, an immunotherapy company focused on the development of allogeneic, multi-tumor-antigen-targeted, non-engineered, T-cell immunotherapies with additional oversight of Quality Assurance and Quality Control functions. Previously, Dr. Peshwa was CTO and Global Head of R&D for the Cell and Gene Therapies business at GE Healthcare (GEHC), with responsibilities that include GEHCs CGT product and service portfolio to enable and accelerate the development of robust, scalable, industrialized manufacturing and delivery of cell and gene therapies. Prior to these roles, Dr. Peshwa held various executive positions at MaxCyte, Inc., NewNeural LLC, and Dendreon Corporation. At MaxCyte, as CSO and EVP, Cellular Therapies, Dr. Peshwa was responsible for leading the development and commercialization of ex vivo cell loading platform technology. Additionally, he also established MaxCytes proprietary therapeutic product portfolio with lead program being a non-viral mRNA engineered CAR Immunotherapy (CARMA) with one-day manufacturing process under company sponsored IND for treatment of solid cancers; and additional collaborative programs under CRADA Agreement with Investigators at NIAID and NHLBI, for ex vivo gene correction in autologous hematopoietic stem cells, as cell therapy for potential treatment of monogenic diseases. As Vice President of Process Sciences and Manufacturing, at Dendreon Corporation, Dr. Peshwa was responsible for leading the CMC and GMP manufacturing for Provenge (Sipuleucel-T), an autologous cellular immunotherapy product for treatment of prostate cancer, the first ever active cellular immunotherapy product approved by the US FDA.

In addition to his broad industry experience, Dr. Peshwa has served as Principal Investigator / Co-Investigator on multiple grant-funded research studies, is an inventor of six issued US patents in the field of cell therapy, and has served in various consultative, advisory, and board capacities to industry, government, not-for-profit, and financial organizations. Dr. Peshwa earned his Ph.D. in Chemical Engineering from the University of Minnesota and his B.Tech. in Chemical Engineering from the Indian Institute of Technology in Kanpur, India.

Tesseras Gene Writing platform represents an opportunity to drive a fundamental change in our ability to treat disease, said Dr. Madhusudan Peshwa. I look forward to joining the executive team to help move Tesseras bold mission forward.

Bill Querbes, Ph.D., Senior Vice President, Therapeutic Discovery & Translational SciencesBill Querbes joined Tessera in April of 2021 as Senior Vice President of Therapeutic Discovery and Translational Sciences. He brings a strong background in genetic medicine and a passion for rare disease drug development with over 15 years of experience leading cross-functional teams from early discovery through clinical trials.

Before joining Tessera, Dr. Querbes held the position of Vice President and Fabry Program Lead at AVROBIO. Prior to this role, as Senior Director at Synlogic, he led clinical program teams in PKU and urea cycle disorders. Earlier in his career he spent 12 years at Alnylam Pharmaceuticals where he made important contributions to the maturation of both the siRNA delivery platforms and therapeutic pipeline. Dr. Querbes led the discovery and early clinical development of GIVLAARI (givosiran) for the treatment of acute hepatic porphyria, which was the first FDA approved RNAi therapeutic utilizing GalNAc conjugate technology.

He holds a B.S. in Biology from SUNY Geneseo and a Ph.D. from Brown University.

Cecilia Cotta-Ramusino, Ph.D., Senior Vice President, Platform DevelopmentCecilia Cotta-Ramusino joined Tessera in 2019 as the Head of Platform Development. She drives the discovery and optimization of novel Gene Writers, enabling their translation into gene therapy tools. Dr. Cotta-Ramusino has spent more than 20 years in academia and biotech, working in the areas of gene editing, cell engineering, and DNA damage. Dr. Cotta-Ramusino was the first employee at insitro where she was the Head of Functional Genomics. Prior to insitro, she was one of the first scientists hired at Editas, the first CRISPR-based therapeutic company, where she helped to define and shape the vision of the Editas platform. She spearheaded numerous academic collaborations devoted to platform optimization and led the development of a T cell gene therapy treatment aiming to treat an immunodeficiency disease. She conducted her postdoc in Steve Elledges lab at Harvard Medical School where she performed whole genome high-throughput screens in mammalian cells using siRNA/shRNA to identify novel components of the DNA damage response. Dr. Cotta-Ramusino obtained her Ph.D. in genetics at University of Milan, Italy and has been principal author and co-author on several publications in high impact factor journals, such as Science, Nature, Nature Communications and Molecular Cell. She has invented several foundational patents in all of the early-stage companies in which she has worked.

Vikram Ranade, Ph.D., Senior Vice President, Corporate DevelopmentDr. Ranade joined Tessera in 2020 as the Head of Corporate Development. In this role, he drives corporate strategy, business development, and investor relations for Tessera.

Dr. Ranade was previously at McKinsey & Company, where he was an Associate Partner in the healthcare practice. At McKinsey, he worked with large biopharma and early-stage biotech companies on strategy, M&A, and R&D topics. He led diligence efforts for more than $15B in completed deals and advised on clinical strategy for more than 20 programs. Dr, Ranade also co-led McKinseys Center for Asset Optimization, which focuses on clinical-stage asset development strategy. He holds a Ph.D. in Genetics and Development from Columbia University, where he studied transcriptional regulation of developmentally important genes at the molecular level. He has a B.S. in biochemistry from Brandeis University, where he was awarded highest honors for his research on DNA damage repair pathways.

David Pollard, Ph.D., Head of BioprocessDavid Pollard has over 25 years of bioprocess development for a range therapeutics including novel mAbs, peptides, anti infectives, biocatalysts and more recently cell and gene therapies. During his career at Merck & Co. Inc, Dr. Pollard led early and late stage CMC teams, providing contributions to multiple INDs & BLAs for Biologics & Vaccines. Dr. Pollard also led an innovation team that co-developed the state-of-the-art ambr250 high throughput bioreactor system and also pioneered lights out automated continuous mAb production. More recently Dr. Pollard pursued processing for personalized neoantigen T cell therapies and helped create corporate research for the technology provider Sartorius. Dr. Pollard will help Tessera drive digital workflows and high throughput automation to accelerate sustainable gene therapy process development.

Steve Garbacz, Head of FinanceSteve Garbacz joined Tessera in 2021 as the Head of Finance and is responsible for financial reporting, planning, taxes, and treasury. Garbacz has more than 25 years of experience in financial management for a range of companies, including Biogen, Epizyme, Spero, and Anika. He has a passion for building scalable financial organizations leveraging new technology, and drove successful IPOs at Epizyme and Spero. At Anika, Garbacz was a key leader in acquiring and integrating two private companies. Garbacz has a B.S. in Economics from George Mason University and an MBA in Finance from the Leonard Stern School of Business at New York University.

For more information about Tessera, including how Gene Writing works, partnership opportunities, and job openings, visit http://www.tesseratherapeutics.com.

About Tessera TherapeuticsTessera Therapeutics is an early-stage life sciences company pioneering Gene Writing, a new biotechnology designed to offer scientists and clinicians the ability to write small and large therapeutic messages into the genome, thereby curing diseases at their source. Gene Writing holds the potential to become a new category in genetic medicine, building upon recent breakthroughs in gene therapy and gene editing while eliminating important limitations in their reach, utilization, and efficacy. Tessera Therapeutics was founded by Flagship Pioneering, a life sciences innovation enterprise that conceives, resources, and develops first-in-category bioplatform companies to transform human health and sustainability.

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Leading Gene Writing Company Tessera Therapeutics Announces Pivotal Expansion of Leadership Team - Business Wire

WASHINGTON, May 24, 2021 /PRNewswire/ --An article published in Experimental Biology and Medicine (Volume 246, Issue 10, May, 2021), describes a new treatment strategy for glioma.The study, led by Dr. Julun Yang, in the Department of Pathology at the 920th Hospital of the Joint Logistics Support Force of PLA in Kunming (China), reports that targeting RAS, a gene that promotes tumorigenesis in multiple cancers, enhances antitumor activity against glioma xenografts.

Gliomas are the most common type of central nervous system tumor in humans, with approximately 250,000 cases reported annually worldwide. Current treatment options include aggressive surgical resection, radiation therapy and chemotherapy.Nonetheless, the survival rate for patients is poor, and new treatments are needed.RAS genes play a critical role in regulating cell proliferation, differentiation, migration, apoptosis and senescence.Overexpression of and mutations in RAS genes promote tumorigenesis in numerous types of cancers, including gliomas.Nevertheless, there are no therapies targeting RAS approved for use in patients.

In the current study, Dr. Yang and colleagues used a gene therapy approach to deliver antibodies that inhibit RAS to glioma cells and tumors.Adenovirus delivery of a RAS antibody gene to glioma cells resulted in anti-RAS antibody expression, decreased growth and proliferation, and increased cell death.Intravenous delivery using cytokine-induced killer cells resulted in expression of high levels of RAS antibodies in tumors and low levels in normal organs.Furthermore, tumor volume was reduced and accompanied by decreased cell proliferation and expression of anti-apoptotic genes as well as increased cell death and expression of pro-apoptotic genes.Collectively, these results suggest that gene therapy targeting RAS may be a safe and effective treatment for glioma and other RAS-driven cancers.

Dr. Steven R. Goodman, Editor-in-Chief of Experimental Biology & Medicine, said, "Dr. Yang and colleagues have performed in vitro and in vivo studies that provide compelling evidence that the delivery of anti-p21Ras scFv by recombinant adenovirus and cytokine-induced killer cells may be an effective therapy against gliomas and, by extension, other Ras-driven cancers."

Experimental Biology and Medicine is a global journal dedicated to the publication of multidisciplinary and interdisciplinary research in the biomedical sciences. The journal was first established in 1903. Experimental Biology and Medicine is the journal of the Society of Experimental Biology and Medicine. To learn about the benefits of society membership, visit http://www.sebm.org. If you are interested in publishing in the journal, please visit http://ebm.sagepub.com.

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Approval provides opportunity to treat the underlying cause of CF earlier than ever before in Canada

TORONTO, Aug. 25, 2021 /CNW/ - Vertex Pharmaceuticals Incorporated (Canada) (Nasdaq: VRTX) today announced that Health Canada has granted Marketing Authorization for PrKALYDECO (ivacaftor) for use in children with cystic fibrosis (CF) as young as four months of age who have at least one of the following gating mutations in their cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

Vertex Pharmaceuticals Incorporated (Canada) Logo (CNW Group/Vertex Pharmaceuticals Incorporated (Canada))

"With today's approval, children in Canada as young as 4 months now have a medicine to treat the underlying cause of their disease," said Nia Tatsis, Executive Vice President and Chief Regulatory and Quality Officer, Vertex Pharmaceuticals. "This is another step in our goal to develop medicines to treat people living with CF as early in life as possible."

The label update is based on data from a cohort in the 24-week Phase 3 open-label safety study (ARRIVAL) consisting of six children with CF ages four months to less than six months who have eligible gating mutations.

PrKALYDECO (ivacaftor) is now approved for additional eligible patients in Canada, and Vertex will work with payers to secure access for this new patient population.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 80,000 people globally. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes one from each parent to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

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About KALYDECO (ivacaftor)

Ivacaftor is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Nia Tatsis in this press release, and statements regarding the availability of KALYDECO to additional eligible patients in Canada and Vertex's work with payers to secure access for the new patient population. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at http://www.sec.gov and available through the company's website at http://www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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Scientists at Washington University School of Medicine (WUSM) in St. Louis have spent some years investigating the links between circadian rhythm and Alzheimers, and have recently been making some real inroads. Following a 2018 study demonstrating how disrupted sleep can accelerate the buildup of toxic plaques associated with the disease, the team has now identified a protein implicated in the progression of the disease that appears highly regulated by the circadian rhythm, helping them join the dots and providing a potential new therapeutic target.

In their previous research, the WUSM team set out to explore how disruptions to our natural sleep cycles, or circadian rhythm, may accelerate the accumulation of amyloid plaques in the brain, which are strongly linked to Alzheimers disease. Through studies on humans and in mice, the team was able to show a strong correlation between the two, and now through follow up work, the team has identified a brain protein that appears to play a role in this relationship.

The brain protein in question is called YKL-40 and for years has served as a biomarker for Alzheimers, as high levels of it have been found in the cerebrospinal fluid of those suffering from the disease and these levels rise as the disease progresses. The researchers were screening for genes that are regulated by the circadian rhythm, and were intrigued to see the gene for this brain protein pop up.

The gene for YKL-40 came up as highly regulated by clock genes, says Erik Musiek, senior author. That was really interesting because it is a well-known biomarker for Alzheimers.

From there, the team investigated this connection between YKL-40 and Alzheimers, which is characterized by chronic inflammation, by exploring how much of the protein is made under inflammatory conditions both with and without a key circadian gene. Indeed, this demonstrated that the circadian rhythm controls how much YKL-40 is produced.

If you have inflammation in the morning, you might get lots of YKL-40; if you get inflammation in the evening, when the clocks in a different phase, you might get less YKL-40, Musiek says.

Next up, the team worked with mice prone to developing amyloid plaques, and genetically modified one group of them to be lacking the gene for YKL-40. As the mice reached old age, the team analyzed their brains and found that those without the YKL-40 protein exhibited around half the amyloid plaques of the control group.

Digging deeper into the reasons why, the team found that the mice lacking the YKL-40 gene featured more microglia, which are immune cells that surround amyloid plaques and prevent them from spreading. Essentially, this meant that those mice had more hungry immune cells prepared to gobble up the amyloid.

This YKL-40 protein probably serves as a modulator of the level of microglial activation in the brain, Musiek says. When you get rid of the protein, it appears the microglia are more activated to eat up the amyloid. Its a subtle thing, a tweak in the system, but it seems to be enough to substantially reduce the total amyloid burden.

The team also examined this idea in human subjects, drawing on genetic data on 778 subjects from aging and dementia studies and finding only a quarter of them featured a genetic variant that lowers levels of YKL-40, and that cognitive function declined 16 percent more slowly in that group.

If your circadian clock is not quite right for years and years you routinely suffer from disrupted sleep at night and napping during the day the cumulative effect of chronic dysregulation could influence inflammatory pathways such that you accumulate more amyloid plaques, says Musiek. We hope that a better understanding of how the circadian clock affects YKL-40 could lead to a new strategy for reducing amyloid in the brain.

The research was published in the journal Science Translational Medicine.

Source: Washington University School of Medicine in St. Louis

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Biomarker of Alzheimer's found to be regulated by sleep cycles - New Atlas

LEXINGTON, Mass., Dec. 14, 2020 /PRNewswire/ --LogicBio Therapeutics Inc. (Nasdaq: LOGC), a clinical stage genetic medicines company developing therapies based on advanced gene editing technology and next-generation synthetic capsids, today announced that Daphne Karydas and Jeff Goater have been appointed to the Company's board of directors.

"We are excited to welcome Daphne and Jeff. They both bring an impressive depth of experience and proven leadership in the areas of corporate finance, global strategic planning, mergers and acquisitions and strategic partnerships in the biopharma sector," said Frederic Chereau, LogicBio president and CEO. "As we plan for many major developments at LogicBio including initiation of our phase 1/2 SUNRISE clinical trial for LB-001, we believe their expertise will play a central role in guiding our corporate strategic planning and help us reach new levels of momentum in all operational areas."

Ms. Karydas is chief financial officer at Syndax Pharmaceuticals, a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies. Previously, she served as senior vice president of corporate financial planning & analysis and strategy at Allergan plc, where she oversaw financial and business strategy through the company's acquisition by Abbvie in May 2020. Prior to joining Allergan, Ms. Karydas spent over 17 years in asset management and investment banking focused on the bio-pharmaceutical sector. She served as a senior healthcare analyst at J.P. Morgan Asset Management and a portfolio manager and senior healthcare analyst at The Boston Company Asset Management and was a vice president at Goldman Sachs Asset Management and a member of Goldman Sachs' healthcare investment banking team. She began her career as a project chemical engineer at Merck & Co. and earned a B.S. and M.S. in chemical engineering from the Massachusetts Institute of Technology and an M.B.A. from Harvard Business School.

Mr. Goater is chief executive officer at Surface Oncology, an immuno-oncology company developing next-generation antibody therapies. Previously he was chief financial officer of Voyager Therapeutics, helping to guide the company through an initial public offering and establishment of a strategic partnership with Sanofi Genzyme. For almost ten years he was an investment banker, most recently at Evercore Partners where he was an advisor on more than $100 billion in strategic transactions in the biopharma industry. He began his career as a research scientist and earned master's degrees in microbiology/immunology, pathology and business administration from the University of Rochester.

"I am especially excited to be joining the LogicBio board of directors as the Company plans for a range of promising product development milestones and business opportunities based on the potential of the platform," said Ms. Karydas. "I look forward to working with the team to expand the Company's progress and target new opportunities in the rapidly emerging gene editing space."

"In recent years, LogicBio has made significant progress both in advancing its pipeline and in positioning the Company for many promising opportunities in research and business development," said Mr. Goater, adding, "I look forward to joining with the other members of the board in helping the Company achieve its mission of bringing innovative therapies to patients with rare diseases around the world."

"As we welcome Daphne and Jeff to our board, we also are very grateful to Erez Chimovits and Daniel O'Connell, who are stepping down from our board, for their service during several formative and active years for our Company," Mr.Chereau added.

About LogicBio Therapeutics

LogicBio Therapeuticsis dedicated to extending the reach of genetic medicine with pioneering platforms. LogicBio's 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 in the phase 1/2 SUNRISE clinical trial 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 have 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.

Forward Looking Statements

This press release contains "forward-looking" statements within the meaning of the federal securities laws. These are not statements of historical facts and are based on management's 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 Company's plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Company's current and future research and development activities and preclinical studies and potential future clinical trials. These risks are discussed in the Company's filings with theU.S. Securities and Exchange Commission(SEC), including, without limitation, the Company's Annual Report on Form 10-K filed onMarch 16, 2020, the Company's Quarterly Report on Form 10-Q filed on May 11, 2020, and the Company's subsequent filings with theSEC. 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.

Media contact:Jenna UrbanBerry & Company Public Relationsjurban@berrypr.com212 253 8881

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LogicBio Therapeutics names Daphne Karydas and Jeff Goater to Board of Directors - BioSpace

SAN JOSE, Calif., Nov. 4, 2020 /PRNewswire/ --Thermo Fisher Scientific, the world leader in serving science, and the Biopharmaceutical Analysis Training Laboratory (BATL) at Northeastern University, have entered into a collaborative agreement in a bid to advance analytical capabilities and drive innovation across the biopharmaceutical industry in areas including personalized medicine, monoclonal antibodies and gene and cell therapies.

BATL's globally-recognized technical training programs for regulators, industry personnel and academics, complemented by Thermo Fisher's leading ultra-high performance liquid chromatography-mass spectrometry (UHPLC-MS) technology, will facilitate the development of cutting-edge analytical solutions and delivery of training designed to meet the current needs of the industry. The implementation of the Thermo Scientific Orbitrap Exploris 240 mass spectrometer and Thermo Scientific Vanquish Duo UHPLC System within BATL's state-of-the-art training facility will offer the accuracy, precision and reliability required to analyze the complex molecules defining the medicines of the future. Utilizing the power of these analytical systems, researchers at BATL will be in a position to develop robust, business need-driven, chromatography-based workflows for the streamlined analysis of biotherapeutics and share these methods across the industry to drive analytical advancements.

"In the last few decades we've seen the development of novel biotherapies, such as gene therapies and personalized medicines, to treat a myriad of diseases, and this level of innovation within the biopharmaceutical industry continues to grow," said Eric Grumbach, director, biopharma/pharma, chromatography and mass spectrometry, Thermo Fisher Scientific. "With new therapies, there comes a need for forward-looking analytical techniques and methods to deliver the sensitivity and accuracy required to ensure the safety and efficacy of drugs. Our collaboration with BATL will provide biopharmaceutical organizations with the analytical solutions required to answer groundbreaking scientific questions, while still meeting regulations and optimizing their operations."

Jared Auclair, associate dean of professional programs and graduate affairs and director of the Biopharmaceutical Analysis Training Laboratory said,"Our progressive training programs focus on the science- and risk-based evaluation of therapeutics, allowing scientists to reduce the regulatory burden and deliver potentially lifesaving drugs to patients more quickly. This collaboration with Thermo Fisher not only provides us with the analytical power we need from our instruments, but also allows us to drive the development of valuable biopharmaceutical characterization and monitoring workflows to address the ever- evolving analysis needs within the industry."

BATL has implemented the Orbitrap Exploris 240 mass spectrometer because of the system's proven ability to meet the requirements of late-stage biopharmaceutical developers for superior application flexibility, improved accuracy, operational simplicity and rapid turnaround times. The Orbitrap Exploris 240 mass spectrometer is a natural choice for BATL, which typically works across an extensive range of samples of varying characteristics, due to the system's ability to deliver consistent results regardless of sample complexity. Additionally, BATL selected the Vanquish Duo UHPLC System for its ability to be operated in tandem mode, eliminating wait time for column equilibration, thereby further accelerating biopharmaceutical analyses.

About Thermo Fisher Scientific

Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue exceeding $25 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, improving patient diagnostics and therapies or increasing productivity in their laboratories, we are here to support them. Our global team of more than 75,000 colleagues delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services and Patheon. For more information, please visit http://www.thermofisher.com.

Media Contact Information:Laura BrightThermo Fisher Scientific+1 562-335-8318laura.bright@thermofisher.com

Janice FoleyBioStrata+1 617-823-5555jfoley@biostratamarketing.com

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By taking advantage of a natural process of gene silencing, a new gene therapy approach appears to prevent the toxicity todorsal root ganglion(DRG) a specific cluster of sensory neurons seen in non-human primates during gene therapy studies for neurological disorders, researchers report.

The approach successfully protected the primates DRG from excessive activity of the introduced gene known as a transgene and subsequent toxicity, without affecting the transgenes activity elsewhere in the nervous system.

DRG inflammation and toxicity was observed in non-human primates after spinal canal injection of AVXS-101 approved asZolgensma when given as an intravenous infusion prompting a partial hold of a Phase 1/2 trial (NCT03381729) to allow for further investigation.

That trial, calledSTRONG, is testing intrathecal (spinal canal) injection of this gene therapy in children with spinal muscular atrophy (SMA) between 6 months and 5 years old.

The study, MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates, was published in the journal Science Translational Medicine.

We believe that this new approach could improve safety in genetherapyuniversally, as well as in SMA, Juliette Hordeaux, PhD, the studys first author at University of Pennsylvanias Perelman School of Medicine (Penn Medicine) said in a press release.

This approach could be used to design other gene therapy [carriers] to repress transgene [activity] in the cell types that are affected by the toxicity and not others, which is critical, because you need [such activity] everywhere else to effectively treat the disorder, added Hordeaux, who is also senior director of translational research for Penn Medicines gene therapy program.

Gene therapy works to deliver a functional version of a gene to correct or replace a faulty gene within specific cells in the body. Most therapy approaches today use a modified and harmlessadeno-associated virus(AAV) as a carrier for the working gene, a vehicle to transport it to a target cell.

But previous studies, including primate work by Penn researchers, showed that AAV-based gene therapies targeting the central nervous system (CNS; brain and spinal cord) can damage the DRG, a cluster of neurons of the spinal nerve that bring sensory information from the periphery to the spinal cord.

Notably, DRG toxicity was observed in studies regardless of the therapys route of administration (directly into the bloodstream or into the spinal canal). No reports of such toxicity in humans, including children treated in STRONG, are known.

Conventional immunosuppressive regimens were ineffective in preventing this toxicity, strongly indicating that an excessive immune response was not its cause. This led Hordeaux and her Penn colleagues to evaluate whether the damage was related to excessive levels of the transgenes product, which could cause cellular stress.

To test their idea, they took advantage of RNAi, a natural process of gene silencing, in which microRNA (miR) molecules bind to a specific messenger RNA (mRNA), targeting it for destruction and preventing the production of that protein. (mRNA is the molecule derived from DNA and used as a template for protein production.)

Since the miR183 complex is specifically produced in sensory tissues and organs such as dorsal root ganglion, the researchers introduced miR183s sequence targets at the endof the transgene sequence in an AVV. With this, any mRNA produced from the transgene would be destroyed in DRG neurons by the naturally present miR183, preventing protein production in these cells.

Researchers then compared the effects of administering AAV with a transgene containing or not containing miR183s sequence targets into the cerebrospinal fluid (the fluid that bathes the brain and spinal cord) of non-human primates.

Introducing miR183s sequence targets in the transgene were found to significantly reduce its mRNA levels and subsequent toxicity in DRG neurons, without affecting the transgenesmRNA levels elsewhere in the primates brain.

Steroids given to primates treated with unmodified AAVs, in contrast, failed to alleviate DRG damage, despite their known anti-inflammatory and immunosuppressive effects. This ineffectiveness was consistent with the proposal that immune system activity does not mediate this neuronal toxicity, the researchers wrote.

We were concerned about the DRG [toxicity] that was observed in most of our [non-human primate] studies, said James M. Wilson, MD, PhD, the studys senior author, and gene therapy program director and a professor of medicine and pediatrics at Penn Medicine.

This modified [viral] vectorshows great promise to reduce DRGtoxicityand should facilitate the development of safer AAV-based gene therapies for many CNS diseases, Wilson added.

Novartis gene therapy Zolgensma, when given directly into the bloodstream, is currently available for use in newborns and toddlers up to age 2 with any type of SMA in the U.S. and Japan, and to those with almost all types who weigh up to 21 kilograms (about 46 pounds) inEurope.

To meet aU.S. Food and Drug Administration(FDA) request for a pivotal confirmatory study of the gene therapys use with older SMA patients, who would be treated via intrathecal (IT) injection, Novartisplans to launch a new AVXS-101 IT trial.

This administration route is favored for those beyond toddler age, as it is thought to better target themotor neuronsdamaged by the disease.

According to the company, this IT trial cannot include U.S. sites until the hold on STRONG is lifted.

Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.

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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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Intrathecal Gene Therapy Use Might Be Safer With 'Silencing' Step - SMA News Today