Category Archives: Gene Medicine

An Auburn University research team from the College of Veterinary Medicine and the College of Agriculture recently became the first to map a high-quality genome assembly of the blue catfish.

The genome, which was published in the journal GigaScience, is essential for genetic improvement using gene-editing or genome-assisted selection and will aid in the genetic enhancement of better catfish breeds for the multimillion-dollar catfish farming industry.

Catfish farming is the largest aquaculture industry in the U.S., accounting for approximately 70% of the nations total aquaculture output. Mississippi, Alabama, Arkansas and Texas account for the great majority of total U.S. freshwater catfish production, with Alabama ranking second only behind Mississippi. The primary fish utilized for farming purposes is a hybrid produced by breeding male blue catfish with female channel catfish.

The hybrid catfish is superior in growth and disease resistance, according to Xu Wang, assistant professor of comparative genomics in animal health in the College of Veterinary Medicines Department of Pathobiology and adjunct faculty investigator with the HudsonAlpha Institute for Biotechnology, who is one of the leaders of the project.

Faster growth means more profit. Originally, farmed fish were primarily channel catfish, but three major bacterial pathogens resulted in a 40% loss of production and annual economic damage of over $100 million in the U.S. industry alone. The hybrid mix of the blue and channel catfish has improved disease resistance and reduced mortality by half.

Even so, Wang says there is a critical need for further genetic improvement using genomic methods.

The channel catfish genome was mapped in 2016 by John Lius lab at Auburn [now at Syracuse University], but the blue catfish genome was not available until we published it, Wang added. Our high-quality blue catfish genome addresses the urgent needs to achieve the long-term goal of improving growth, feed utilization, stress and disease resistance and reproduction.

Wang served as senior author of the GigaScience paper, assisted by Haolong Wang (no relation), a doctoral student in biomedical sciences supported by both an Auburn Presidential Graduate Research Fellowship and a College of Veterinary Medicine Deans Fellowship. The veterinary researchers collaborated closely with a team from the College of Agricultures School of Fisheries, Aquaculture and Aquatic Sciences led by Professor Rex Dunham, an internationally recognized authority in the genetic enhancement and gene editing of catfish.

This is a fantastic step forward, Dunham said of the mapping of the blue catfish genome. There have been many genetic enhancement projects related to gene transfer and gene editing that were not possible for blue catfish without it. As a result, we could not do parallel work with what we are doing with channel catfish. Since a hybrid between channel and blue is the best genetic type for the catfish industry, that also put limitations on what we could do with these tools to improve the hybrid.

That roadblock is now gone. Having the blue catfish genome available opens a huge treasure chest of markers we can use for other approaches, such as marker assisted selection, and also gives us many more tools to distinguish and track different genetic types of blue catfish. Thanks to this research, we are much less limited than before.

Read more:
Auburn University researchers first to map blue catfish genome - Office of Communications and Marketing

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LAS VEGAS, Nevada (KVVU) -- A mysterious illness has turned one local familys life upside down. Their baby is fighting to survive after a rare diagnosis.

Josette Gentile told FOX5 her daughter Isla was a dream baby for the first few months of her life, but she became concerned when the infant wasnt able to hold her head up.

Her eyes just didnt focus like a usual baby does at four months old, Gentile shared.

That started months of testing. Doctors were stumped as to the diagnosis.

Every test kept coming back normal, just a little bit off but something was obviously wrong, recalled Gentile.

Things got worse, Isla was not eating and had no energy.

I took her to the ER. They did a bunch of tests and said everything was normal. Sent us home again and two days later Im like, I dont care what that doctor said, I know something is wrong with my baby. Took her to Summerlin Childrens Hospital where they took us very seriously and turns out she had a bladder infection that had turned to sepsis, Gentile explained.

Doctors said something was also wrong with her brain.

They life flighted us to Childrens Primary Hospital in Salt Lake City, said Gentile.

A team of doctors came together to solve the mystery: what was making Isla so sick?

One of her genes has two mutations. Its her FDXR gene. Only 35 people in the world have this mutation. Her specific mutation, the location in the gene and everything, she is the only one in the world known to have it, Gentile relayed.

The mitochondrial disease means Islas body cannot produce enough energy to function properly.

She has regressed to almost like a newborn, shared gentile.

Islas family, mom, dad Alejandro Ledesma, and 3-year-old sister Sage have dropped everything to focus on her care.

Its just flipped our lives completely upside down. This is our 21st day in the hospital, said Gentile.

There is no cure and no treatment. Doctors put Isla on a regimen of vitamins in hopes of boosting her energy.

What that is going to do is just make her more comfortable, her mother explained.

As the family is temporarily living in Salt Lake City, the Las Vegas community has stepped in to help. A fundraising page has raised thousands so far to help with their bills.

It has taken honestly a lot of stress off of us so we only have to worry about being here and keep her here as comfortably as we can, said Gentile.

It just makes you not feel alone in such a terrible time in your life, added Ledesma.

The disease will continue to get worse until Islas body can no longer handle it. The family plans to come back to Vegas if and when Isla is stable enough to travel.

Read more here:
Las Vegas baby diagnosed with rare genetic mutation - KVOA Tucson News

Imagine a patient with a rare genetic disorder that makes their arms and legs have imprecise and slow movements. For years, the patient has faced serious restrictions in day-to-day life. They tried several treatments, but all have failed to ease the symptoms.

Now imagine a university team discovering a therapy that could tackle this condition, with a solution that lies in the patients own body. The patients blood would be collected, some key cells would be separated in a laboratory, gene-editing techniques would be applied, and personalised medicine, produced with specialised equipment, would be injected back into the patients body.

A biological process would then be triggered in which all faulty genes would be corrected, reducing the diseases severity, perhaps correcting it all together. The modification would be restricted to the patient and would not be passed on to their children, since it would not affect reproductive cells.

Our story has a catch, though: the blood cells needed for the personalised medicine are very fragile and do not live very long outside the human body. This means theres little time to take the blood to the specialised laboratory, transport the cells to the production facility, and take the medicine back to the hospital where the patient is.

But what if all these production steps were quickly performed in the same place that is, in the hospital?

Read more: The human body has 37 trillion cells. If we can work out what they all do, the results could revolutionise healthcare

Our story is ceasing to be just imagination because this way of producing medicines in the hospital is actually emerging. Its what specialists call point-of-care manufacture. And there are several notable examples of it already in use.

For instance, a medicine for multiple myeloma (a type of bone marrow cancer) is being produced in the Hospital Clinic in Barcelona, Spain. Products for severe burns are being manufactured in Lausanne University Hospital in Switzerland.

At the University of Colorado in the US, researchers are developing a therapy for hard-to-treat lymphoma, a type of blood cancer. In the UK, an NHS Blood and Transplant laboratory is investigating the manufacture of red blood cells which, if successful, could be carried out in hospitals and other clinical settings for the treatment of cardiac diseases.

These illnesses might not have been treated if the medicines had needed to be frozen and transported over long distances, instead of being made in the hospital.

Given that these therapies have such a short shelf life and will need to be produced at the patients bedside, there are many things we need to consider before we can deploy them on a wider scale. For example, what measures should hospitals, companies, and regulators take to adopt this model and make it work? This is what our research team has been investigating.

Its vital that the same safe and high-quality production methods are used in different hospitals so that all patients receive the best possible care. This is why regulatory agencies in the UK are already proposing new ways of managing this model.

For example, it has been suggested that to begin with, manufacturers could oversee the medicines production in several different hospitals from a central site. They could also be responsible for providing training and quality control in the hospitals that have rolled out point-of-care manufacture to ensure that the products are safe and high-quality.

But just because a new policy has been made, doesnt mean it will be successfully implemented. This will mean hospitals and companies will need to change how they operate for these new technologies to be implemented safely and efficiently.

Our research, in collaboration with the Medicines and Healthcare Products Regulatory Agency (MHRA) and several public and private sector organisations has also looked at what benefits and challenges there may be in implementing this innovative approach to the production of medicines.

In a recent publication, we put forward several steps that need to be taken by regulators, hospital staff, and companies to make the production of personalised therapies in hospitals a reality. First, trusts, clinical centres and hospital staff will need to investigate how best to make therapy production happen in medical wards. They will also need to identify any issues such as staff training and data management which may stop this from happening.

Companies already developing these advanced treatments can also supply hospitals with manufacturing equipment and production system know-how, making it easier to start developing personalised therapies in hospitals with as little disruption to day-to-day operations as possible. Regulators may need to provide guidance for different therapies to ensure quality control and patient safety.

Now, let us return to our patients story. After receiving the therapy produced in the hospital, the patient goes on to live a healthy life and have a child that is diagnosed with the same genetic condition. But now, the way to receive treatment is much clearer.

The child will be treated in a specialised hospital where certified equipment and trained staff are available for producing and delivering an enhanced version of the personalised therapy. With more experience and better infrastructure in place, the child will receive a treatment that yields faster outcomes with fewer side effects.

But this will only be possible if everyone including hospital staff, manufacturers, scientists and policymakers work together to ensure point-of-care manufacture is successfully rolled out.

Excerpt from:
Personalised medicine made in hospitals can revolutionise the way diseases are treated the challenge now will be implementing it - The Conversation...

DNA replication and repair happens thousands of times a day in the human body and most of the time, people dont notice when things go wrong thanks to the work of Replication protein A (RPA), the guardian of the genome. Scientists previously believed this protein hero responsible for repairing damaged DNA in human cells worked alone, but a new study by Penn State College of Medicine researchers showed that RPA works with an ally called the WAS protein (WASp) to save the day and prevent potential cancers from developing.

August 9, 2022Penn State College of Medicine News

The researchers discovered these findings after observing that patients with Wiskott-Aldrich syndrome (WAS) a genetic disorder that causes a deficiency of WASp not only had suppressed immune system function, but in some cases, also developed cancer.

Dr. Yatin Vyas, professor and chair of the Department of Pediatrics at Penn State College of Medicine and pediatrician-in-chief at Penn State Health Childrens Hospital, conducted prior research which revealed that WASp functions within an apparatus that is designed to prevent cancer formation. As a result, some cancer patients had tumor cells with a WASp gene mutation. These observations led him to hypothesize that WASp might play a direct role in DNA damage repair.

Replication protein A (RPA) forms a complex with WASp at replication forks (red) within the nucleus (blue) of a human cell during DNA replication stress.

WAS is very rare less than 10 out of every 1 million boys has the condition, said Vyas, who is also the Childrens Miracle Network and Four Diamonds Endowed Chair. Knowing that children with WAS were developing cancers and also observing WASp mutations in tumor cells of cancer patients, we decided to investigate whether WASp plays a role in DNA replication and repair.

The researchers conducted protein-protein binding experiments with purified human WASp and RPA and discovered that WASp forms a complex with RPA. Further tests revealed that WASp directs RPA to the site where single DNA strands are broken and need to be repaired. According to Vyas, without the complex, DNA repair happens by secondary mechanisms, which can lead to cancer. This novel function of WASp is conserved through evolution, from yeast to humans. The results of the study were published in Nature Communications.

In the future, Vyas and colleagues will continue to study how their observations about this RPA-WASp complex formation can be applied to treating cancer patients. Vyas said it is possible that gene therapy or stem cell therapy could restore WASp function and may prevent further tumor growth and spread. He also mentioned the possibility of using WASp dysfunction as a biomarker for identifying patients at risk for autoimmune diseases and cancers.

This complex weve discovered plays a critical role in preventing the development of cancers during DNA replication, said Vyas. Translating this discovery from bench to bedside could mean that someday we have another tool for predicting and treating cancers and autoimmune diseases.

Seong-Su Han, Kuo-Kuang Wen of Penn State College of Medicine and formerly of the University of Iowa Stead Family Childrens Hospital; Mara Garca-Rubio and Andrs Aguilera of University of Seville-CSIC-University Pablo de Olavide; Marc Wold of University of Iowa Carver College of Medicine; and Wojciech Niedzwiedz of the Institute of Cancer Research also contributed to this research. The authors declare no conflicts of interest.

This research was supported in part by the National Institutes of Health, the ICR Intramural Grant and Cancer Research UK Programme, the European Research Council and the Spanish Ministry of Science and Innovation grant, the University of Iowa Dance Marathon research award, the Research Bridge Award from the Carver College of Medicine University of Iowa and endowments from the Mary Joy & Jerre Stead Foundation and from Four Diamonds and Childrens Miracle Network. The content is solely the responsibility of the authors and does not necessarily represent the official views of the study sponsors.

Read the full manuscript in Nature Communications.

If you're having trouble accessing this content, or would like it in another format, please email Penn State Health Marketing & Communications.

Link:
'Guardian of the Genome' and the 'WASp' team up to repair DNA damage - Penn State Health News

A yearslong effort by Novartis to repurpose a rare disease drug for cancer has come up short in a late-stage trial for a third time, closing off an opportunity for the Swiss pharmaceutical company to seek an expanded approval.

On Monday, Novartis revealedthe medicine, called canakinumab, failed to show a benefit versus placebo in a large Phase 3 study testing the drug in lung cancer patients following surgery to remove their tumors. Full results were not disclosed, but Novartis acknowledged the study did not meet its primary goal.

The outcome follows negative findings from two other Phase 3 lung cancer trials of canakinumab, which is sold as Ilaris for several fever syndromes and uncommon forms of arthritis.

Novartis quest to prove canakinumab might have broader potential was launched by a 10,000-person study called CANTOS, which in 2017 suggested treatment with the drug reduced heart risk as well as lowered the incidence and lethality of a common type of lung cancer.

The Food and Drug Administration in 2018 rejected Novartis pitch to secure canakinumabs approval as a treatment for reducing cardiovascular events like heart attacks and strokes. But the drugmaker pressed on in lung cancer, running a series of trials called CANOPY, the most recent of which was dubbed CANOPY-A.

We made an investment in the CANOPY program based on signals of reduced lung cancer incidence and mortality observed in the CANTOS study, said Jeff Legos, Novartis head of oncology and hematology development, in a statement.

While we are disappointed CANOPY-A did not show the benefit we hoped for, every trial generates scientific evidence that supports future research and development, he added.

CANOPY-A enrolled 1,382 patients with non-small cell lung cancer who had their tumor removed via surgery. Following their procedure, participants were randomized to receive either canakinumab or placebo and followed for several years.

However, treatment with Novartis drug did not extend disease-free survival, a metric designed to assess how long patients live without their disease returning. No unexpected safety signals were reported, according to Novartis, which will present its full findings at an upcoming medical meeting.

An antibody drug, canakinumab binds to a cytokine protein called IL-1. The protein helps control inflammatory signaling, and blocking it was thought to potentially suppress pro-tumor inflammation.

Novartis is still running one more trial of canakinumab in lung cancer, a Phase 2 study called CANOPY-N thats testing treatment before surgery rather than following it. Researchers at Memorial Sloan Kettering are running another study with Novartis assistance.

But CANOPY-A was Novartis clearest path to asking the FDA for an expanded approval in lung cancer and unlocking a market opportunity the company estimated could be worth between $1 billion and $2 billion in peak annual sales.

Read the original:
Novartis bid to repurpose rare disease drug for cancer falls short in third trial - BioPharma Dive

Abnormal SOD1 protein detected in human spinal cord tissue (dark spots) Trist et al. 2022.

Normally, the protein superoxide dismutase 1 (SOD1) protects cells, but a mutation in its gene is thought to make the protein toxic; this toxic protein form is associated with hereditary forms of ALS. Abnormal mutant SOD1 is only found in regions of the spinal cord where nerve cells die, implicating this abnormal protein in cell death.

Previous investigations into the role of toxic forms of SOD1 protein largely focussed on mutant forms of the protein and were primarily conducted using animal and cellular models of ALS.

The study, led by a team from the University of Sydneys Brain and Mind Centre, advances our understanding of the causes of motor neurone disease by studying this abnormal protein in post-mortem tissues from patients with ALS.

We have shown for the first time that mechanisms of disease long hypothesised to occur in animal and cellular models are present in patients with motor neurone disease, says lead author Dr Benjamin Trist from the Brain and Mind Centre, Faculty of Medicine and Health.

This is a significant milestone in our understanding of ALS and motor neurone disease more broadly.

In related experiments, Professor Double and her team are also currently studying how abnormal SOD1 interacts with other disease-linked proteins in motor neurone disease. This work is in press and will be published in Acta Neuropathologica Communications.

The rest is here:
Abnormal protein could be a common link between all forms of motor neurone disease - University of Sydney

SHANGHAI, Aug. 6, 2022 /PRNewswire/ -- Epigenic Therapeutics Co., Ltd., a frontier biotechnology company dedicated to developing next generation gene editing therapy utilizing regulation of epigenetic genome for wide variety of diseases, today announced it has secured $20 million in Series Angel and Pre-A funding. Series Pre-A funding is jointly invested by Morningside Venture Capital, Kingray Capital, Trinity Innovation Fundand TigerYeah Capital. Angel investor FountainBridge Capital is also participating.

Proceeds of financing will be used to validate advances of the Company's proprietary epigenetic editing in non-human primates, expand expertise and capabilities, and sponsor early-stage clinical investigations.

Epigenetic modification is a natural andheritable gene regulation mechanism in the human body without altering the underlying DNA sequence. Leveraging company's proprietary and patented technology platform, scientists are able to harness endogenous epigenetic gene regulation pathway to precisely and efficiently deliver medicine to target cells and tissues, and achieve potent and durable therapeutic impact. Epigenic Therapeutics has gathered highly talented scientists and industry veterans to direct discovery and development.

"Epigenetic editing is an emerging and highly differentiated gene editing technology." Said Bob Zhang, co-founder and CEO of Epigenic Therapeutics, "along with our scientific co-founders and advisers, we are able to expand our understanding of precise regulation of epigenetic genome, and unlock its potential as medicine for many diseases. With the funding, we will continue expanding our team and capabilities, validate the technology platform in animal model, and accelerate our leading product from discovery to clinical development."

"Epigenic Therapeutics is uniquely positioned in various gene editing therapy developers. We are thrilled to invest in Epigenic Therapeutics and we believe this company has solid foundation to further explore and develop precise genome medicine to benefit many patients." Commented by Michael Xue, Managing Director of Morningside Venture Capital.

About Epigenic Therapeutics' Technology PlatformEpigenic Therapeutics' proprietary technology platform employs its own artificial intelligence (AI) algorithms to explore and obtain an optimized CRISPR-Cascomponent to regulate target gene(s) or govern the expression of one or multiple gene(s) at once without changing the sequence of the DNA. Among peer technologies,our platform is capable to overcome the potential risk rising from DNA cleavage including but not limited to off-target effect, short half-life and challengingpatientcompliance issues. Combing a patented lipid nanoparticle (LNP) medicine delivery system, Epigenic Therapeutics'platform has been proven to precisely and efficiently deliver medicine to target cells and tissuesex vivoandin vivoin ocular, neurodegeneration, metabolic, and rare disease models.

About Epigenic TherapeuticsEpigenic Therapeutics is a frontier biotechnology company dedicated to developing next generation gene editing therapy utilizing regulation of epigenetic genome for a variety of diseases. Founded in 2021 by leading scientists focused on discovering gene editing technologies and developing gene editing therapies, the company has multiple product candidates in the pipeline, including treatment for ocular, neurodegeneration, metabolic, and rare diseases. For more information, visit http://www.epigenictx.com

About Morningside Venture CapitalMorningside Ventures was founded in 1986 by the Chan Family of Hong Kong. Since its establishment, Morningside has been focusing on trends of the forefront life science and healthcare industries over the world, spreading its business scope and investment footprint over North America, Europe and Greater China. Morningside comprises a group of investment professionals who are entrepreneurial, have deep industry knowledge and profound experience in venture capital management. For more information, please visit http://www.morningside.com

About Kingray CapitalKingray Capital was founded in 2018, focusing on investment opportunities in the fields of information security, new energy, industrial intelligence, medical and health care and enterprise services. Kingray Capital is committed to helping high-tech enterprises grow rapidly and creating long-term and stable investment returns for investors.

About Trinity Innovation FundTrinity Innovation Fund ("TIF") is dedicated to investing on biomedical innovations. Our limited partners (LPs) include renowned biopharmaceutical companies and investment institutions. Embedded in our name, TRINITY represents the basic philosophy as "Triad of scientists, managers and investors, let professionals do their own jobs". As investor, TIF helps scientists to transform research outcomes, managers to develop corporates. Together, we turn Innovation into Cure. Leveraging on our profound industry knowledge and resources, we are committed to accelerating growth of our portfolio companies via strategy optimization, recruitment of key positions, partnering and more.

About TigerYeah CapitalTigerYeah Capital, an independent venture capital institution under Tigermed, was founded in 2014.TigerYeah Capital focuses on equity investment in the early and growing medical and health field. The management team has deep industrial background, extensive industrial resources and rich investment experience. Since its inception, TigerYeah Capital whose investment portfolio covers medical devices, biomedicine, CRO and health food has completed nearly 100 projects with the investment of 1.5 billion yuan. Through empowering the invested enterprises, TigerYeah Capital values the development of China and the global medical and health industry and makes contribution to public health.

About FountainBridge CapitalFountainbridge Capital is an avant-garde and emerging venture capital focusing on early-stage innovations. Starting even from ideas or concepts, Fountainbridge works closely with entrepreneurs and researchers to set up new companies and translate innovation into market products. Under the guidance of deep research, Fountainbridge has made outstanding investments in cutting-edge technology including semiconductor, cloud computing, bio-tech and green energy, and consumer innovation like new retailing, overseas brand and novel consumer-electronics. Being the first investor of most portfolios, Fountainbridge is the founder and also the co-founder of start-ups. With a robust ecosystem built, Fountainbridge helps in growth strategy, top industrial experts'recruitment, patent application, legal counseling, and continuous fundraising. Many of Fountainbridge portfolios has become market leaders.

Contact

[emailprotected][emailprotected][emailprotected]

SOURCE Epigenic Therapeutics

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Epigenic Therapeutics Raises $20 Million in Series Angel and Pre-A Funding to Advance Next Generation Gene Editing Therapy - PR Newswire

The Food and Drug Administration has halted testing of a preclinical cancer medicine from Beam Therapeutics, the biotechnology company announced Monday.

Beam, a high-profile developer of a gene editing technique known as base editing, said in a short statement that the FDA put its request to start human trials of the experimental treatment on clinical hold.

Beam didnt say why the FDA paused its application. The biotech was informed of the agencys decision via an email on Friday, and expects to provide an update pending discussion with the FDA. The regulator will provide Beam with a formal letter within 30 days.

Company shares fell by more than 10% in pre-market trading Monday.

Beam is the leading developer of base editing, an approach borne out of research from the labs of Harvard University gene editing specialist David Liu. Unlike the first generation of CRISPR editing, which cuts both strands of DNA, base editing is designed to change single DNA letters without causing a double-stranded break, a method thats thought to carry fewer risks.

Beam was formed five years ago to turn the approach into human medicines and has since received significant financial support. The company raised $180 million in an initial public offering in February 2020 and in January got $300 million upfront from Pfizer in a wide-ranging research deal. The biotech had $1.2 billion in cash on its balance sheet at the end of the first quarter.

The company has already been cleared by U.S. regulators to start a study of BEAM-101, a drug for sickle cell disease, and expects to start enrolling patients in that trial later this year. Verve Therapeutics also recently began clinical testing of a heart disease drug that uses Beams base editing technology.

BEAM-201, an experimental treatment for leukemia and lymphoma, was expected to follow this year along with a second sickle cell drug called BEAM-102.

Verves treatment is an infusion of a drug that performs base editing inside the body. Beams two most advanced programs, including the cancer drug now on hold, genetically modify cells outside the body.

BEAM-201 is meant to overcome some of the limitations of personalized cancer cell therapies from Bristol Myers Squibb, Novartis and Gilead, which are approved to treat certain leukemias and lymphomas. The treatment uses cells from donors, rather than patients themselves, and silences multiple genes simultaneously an approach Beam claims could make those cells more durable. Several developers of so-called off-the-shelf cell therapies have struggled to prove their drugs are as long-lasting as personalized treatments, however.

The drug is the latest gene-based medicine, meanwhile, to be slowed by regulators. The FDA has recently paused testing of a number of gene replacement or gene editing therapies, wary of potential safety concerns.

This is obviously negative for the stock and reiterates a high level of scrutiny from the regulators on novel technologies like gene/base editing, wrote RBC Capital Markets analyst Luca Issi in a research note. However, we also note that the [application] was submitted at the end of June, so we assume no patient has been dosed, and it is possible that the hold is simply procedural in nature.

Beam is seeking to treat patients with either relapsed or refractory T cell acute lymphoblastic leukemia or T cell lymphoblastic lymphoma.

Read more from the original source:
FDA halts testing of Beam's base editing cancer therapy - BioPharma Dive

OHSU researchers will receive a grant to helpadvance a first-of-its-kind method to turn an individuals skin cell into an egg, with the potential to produce viable embryos. (OHSU/Christine Torres Hicks)

Scientists at Oregon Health & Science University have received significant philanthropic support to advance a first-of-its-kind method to turn an individuals skin cell into an egg, with the potential to produce viable embryos.

The technique, initially demonstrated in mice, could eventually provide a new avenue for child-bearing among couples unable to produce viable eggs of their own.

Paula Amato, M.D., professor of obstetrics and gynecology in the OHSU School of Medicine, andShoukhrat Mitalipov, Ph.D., director of the OHSU Center for Embryonic Cell and Gene Therapy. (OHSU/Christine Torres Hicks)

Even though the proof of concept in mice shows promise, significant challenges remain to be resolved before the technique could be ready for clinical trials under strict ethical and scientific oversight. Even then, Congress currently precludes the Food and Drug Administration from providing oversight for clinical trials involving genetic modification of human embryos.

Shoukhrat Mitalipov, Ph.D., (OHSU)

It will take probably a decade before we can say were ready, said Shoukhrat Mitalipov, Ph.D., director of the OHSU Center for Embryonic Cell and Gene Therapy. The science behind it is complex, but we think were on the right path.

This type of research is not funded by the National Institutes of Health, so it depends on philanthropic support. For this project, Open Philanthropy awarded $4 million over three years through the OHSU Foundation.

Paula Amato, M.D. (OHSU)

Paula Amato, M.D., professor of obstetrics and gynecology in the OHSU School of Medicine, sees the potential for an enormous benefit to families struggling to have children if the technique proves successful.

Age-related decline in fertility remains an intractable problem in our field, especially as women are delaying childbearing, said Amato, who is the principal investigator for the grant award.

The technique holds promise for helping families to have genetically related children, a cohort that includes women unable to produce viable eggs because of age or other causes, including previous treatment for cancer. It also raises the possibility of men in same-sex relationships having children genetically related to both partners.

The skin cell can come from somebody who doesnt have any eggs themselves, Amato said. The biggest implication is for female, age-related infertility. It can also come from women with premature ovarian insufficiency due to cancer treatment or genetic conditions, or from men who would be able to produce a genetically related child with a male partner.

The award from Open Philanthropy will enable OHSU researchers to develop the technique in early human embryos using eggs and sperm from research donors. As with other groundbreaking research at OHSU including a gene-editing discovery that generated worldwide attention in 2017 none of the early embryos will be allowed to develop past the early blastocyst stage.

Researchers will build on a study in mice published this January in the journal Communications Biology.

The study demonstrated that it is possible to produce normal eggs by transplanting skin-cell nuclei into donor eggs from which the nuclei have been removed. Known as somatic cell nuclear transfer, the technique was famously used in 1997 to clone a sheep in Scotland named Dolly. In contrast to a direct clone of one parent, the mouse study published earlier this year required OHSU and collaborating scientists to cut the donor DNA in half and then fertilize the resulting egg with sperm to generate a viable embryo with chromosomes from both parents.

The process involves implanting the skin cell nuclei into a donor egg, and then allowing the egg to discard half its skin cell chromosomes a process similar to meiosis, when cells divide to produce sperm or egg cells. This results in a haploid egg with a single set of chromosomes with precisely half the chromosomes of the diploid skin cell with two sets of chromosomes. At just the right phase of the cell cycle, the new egg is combined with sperm chromosomes through in vitro fertilization.

An embryo then develops with the correct diploid number of chromosomes from each parent.

We had to show in the mouse that this hypothesis works, Mitalipov said. Open Philanthropy saw the implications for fertility with a new way of looking into this. The key is inducing haploidy.

Read more:
OHSU advancing first-of-its-kind strategy to overcome infertility - OHSU News

Wilmington, Delaware, United States, Transparency Market Research Inc.: Gene therapy is one of the best treatment options for most chronic diseases. It involves inserting a functional copy of a gene into a defective cell. Gene therapy is useful in the treatment of cancers, inherited disorders, cardiovascular diseases, and infectious pathogen neurological disorders.

Read Report Overview https://www.transparencymarketresearch.com/viral-vectors-manufacturing-market.html

Viral or non-viral vector methods are used in efficient transfer of therapeutic gene into the target cells. Viral vectors used in gene therapy include adenovirus, lentivirus, retrovirus, and adeno-associated viral (AAV). Non-viral vectors generally depend on delivery of plasmid DNA.

Development of quality vectors in terms of formulation, physical size, cost, and delivery function is quite challenging. To minimize this problem, manufacturers use various approaches such as development of cell line culture, current good manufacturing practices, cell culture system, and expression systems that are used in the development of vectors. This is projected to boost the growth of the global viral vectors manufacturing market.

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Additionally, increase in the number of gene therapy candidates due to rapid development of diseases and rise in funding for gene therapies are expected to fuel the growth of the global viral vectors manufacturing market. The Alliance for Cancer Gene Therapy (ACGT) is a public charity foundation in the U.S. which funds for advancement in cancer gene therapies from laboratory to clinical trials. However, high cost of gene therapies and possible mutagenesis restrain the market.

The global viral vectors manufacturing market can be segmented based on type, disease, application, and region. In terms of type, the global market can be divided into adenoviral vectors, retroviral vectors, adeno-associated viral vectors, and others. The retroviral vectors segment dominated the global viral vectors manufacturing market due to ease of application in major target diseases such as cancer and genetic disorders. Based on disease, the global viral vectors manufacturing market can be classified into cancers, infectious diseases, genetic disorders, and other diseases.

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The genetic disorders segment is anticipated to dominate the market due to increase in research activities on various genetic disorders such as sickle cell anemia, hemophilia A and B, and Huntingtons disease, and a strong gene therapy pipeline in the last phase of drug development. In terms of application, the global market can be bifurcated into gene therapy and vaccinology. The gene therapy segment is expected to account for the largest share of the market due to increase in the number of gene therapy clinical trials conducted for chronic diseases such as cancer, cardiovascular diseases, and neurodegenerative diseases globally.

Geographically, the global viral vectors manufacturing market can be segmented into North America, Europe, Latin America, Asia Pacific, and Middle East & Africa. Each region can be divide into specific countries/sub-regions such as the U.S., Canada, the U.K., Germany, Brazil, China, India, and GCC Countries. North America dominated the global viral vectors manufacturing market because of increase in research activities, large number of regenerative medicine companies, rise in prevalence of target diseases, and availability of funds. Asia Pacific is expected to be the most attractive market during the forecast period due to increase in health awareness and demand for advanced medical technology.

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Key players operating in the global viral vectors manufacturing market are Lonza, Merck, Oxford BioMedica, CGT Catapult, Cobra Biologics, uniQure, FUJIFILM Diosynth Biotechnologies, Kaneka Eurogentec, and Spark Therapeutics, among others. These players adopt various strategies such as collaborations, agreements, partnerships, and launch of new products to gain competitive advantage in the market.

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Viral Vectors Manufacturing Market: Increase in the Number of Gene Therapy Candidates due to Rapid Development of Diseases to Drive the Market -...