Category Archives: Gene therapy

Kriya Therapeutics cofounder and CEO Shankar Ramaswamy.

Over the past several years, breakthroughs in gene therapy have led to treatments for rare diseases that were deadly just a decade ago. Take Zolgensmain 2019, it was the first gene therapy approved by the FDA to treat spinal muscular atrophy, a rare genetic disease that affects the mobility of infants and children. But gene therapies have historically had two drawbacks: They are only used for rare diseases, and they carry a hefty price tag (treatment with Zolgensma costs $2.1 million).

Kriya Therapeutics is trying to overcome these obstacles by creating gene therapies for the massesand manufacturing them at a lower cost. On Wednesday, the startup announced that it had raised a $100 million Series B funding round to get it closer to this goal. The round was led by investors from Patient Square Capital and also involved investors from QVT, Dexcel Pharma, Foresite Capital, Bluebird Ventures, Transhuman Capital, Narya Capital, Amplo and the Juvenile Diabetes Research Foundation T1D Fund.

We think this is going to be an extraordinarily important therapeutic class that will revolutionize the treatment of many diseases, says Jim Momtazee, managing partner at Patient Square Capital.

The Silicon Valley-based company was founded in late 2019 by three pharmaceutical industry alums, including a former cofounder of Spark Therapeutics and the former president of United Therapeutics Corp. Shankar Ramaswamy, Kriyas CEO, was part of the foundational team at Roivant Sciences. The new round brings the companys total funding to $180 million; the company declined to reveal its valuation.

Kriyas main focus is its uniquely designed Adeno-associated virusesviruses that are harmless when they enter the body, but deliver instructions to cells that then pump out genes that are missing in some people with genetic diseases. Though the company still plans to develop treatments for rare diseases, what sets it apart is its focus on more common genetic diseases, like some forms of diabetes and obesity. So far, gene therapy has been really constrained in many respects from achieving its full potential, Ramaswamy says. We are believers in gene therapy being applied to rare diseases as well as prevalent diseases.

Ramaswamys goal is to build a company that can go from genetic target discovery to manufacturing and then full commercialization of new therapies, unlike a typical biotech startup that might partner with a large pharmaceutical company for the later stages of development (Ramaswamy says the company will be open to partnerships, but can bring a drug to commercialization on its own). Once the company discovers and develops new gene therapies, its 51,000-square-foot manufacturing facility in North Carolina can produce Adeno-associated viruses at scale to deliver the genes to patients in need. Ramaswamy says that capability will bring down the cost, with savings passed along to patients. I think the innovations that were delivering will make gene therapies much more affordable and accessible to patients, he says. We are very committed to not being a burden on the healthcare system.

The companys current pipeline of products are all preclinical, though Ramaswamy says that they plan to submit Investigational New Drug applications to the FDA for several products in late 2022 and early 2023. So far the company is developing gene therapies for type 1 diabetes, solid tumors and two eye conditions: geographic atrophy and uveitis. In the U.S., more than 3 million people combined have at least one of these conditions, meaning Kriya has a huge pool of potential customers. By comparison, there are fewer than 25,000 children and adults with spinal muscular atrophy in the country.

Ramaswamy says that the new capital will go toward continuing the companys explosive growthit now has 80 full-time employeesas well as refining its vector delivery platforms and manufacturing capabilities. In the future, the money will allow the company to continue to develop new gene therapies for diseases both common and rare. Were taking a very new approach, which is to think more broadly, Ramaswamy says.

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This Company Raised $100 Million To Bring Gene Therapy To The Masses - Forbes

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it will host a virtual manufacturing day for analysts and investors. The event will be webcast live on Tuesday, July 27, 2021, from 10:00 a.m. to 1:00 p.m. ET.

Topics of discussion will include the companys unique three-pillar approach to the manufacturing process, its manufacturing capabilities, the regulatory environment for gene therapy manufacturing, and the immunology of gene therapy. A question and answer session will follow each formal presentation.

The event will feature presentations from Taysha senior leaders:

Registration for this event is available through LifeSci Events. A live video webcast will be available in the Events & Media section of the Taysha corporate website. An archived version of the event will be available on the website for 60 days.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including our preclinical product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, the potential market opportunity for these product candidates, our corporate growth plans and our plans to establish a commercial-scale cGMP manufacturing facility to provide preclinical, clinical and commercial supply. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

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Taysha Gene Therapies to Host Manufacturing Day - Business Wire

UDG Healthcare company Ashfield has launched a new network offering an end-to-end approach for the commercialisation of cell and gene therapies.

The network, called EmerGENE, will enable Ashfield to support small and midsize biotech companies with the commercialisation of their discoveries, the agency said in a statement.

EmerGENE was created by a multidisciplinary team and combines experts from Ashfield Health, Ashfield Engage and Ashfield Advisory.

It aims to deliver expert-led guidance and services to biotech companies throughout their clinical to commercial journey.

The network will provide guidance on commercialisation strategic support, early clinical development, distribution and logistics, market access and patient and HCP engagement and support.

At Ashfield, we look to embed ourselves into our customers businesses and use our expertise to create tangible solutions which best meet their needs, said Amar Urhekar, global president at Ashfield Health.

EmerGENE is no different, and with over 1,200 cell and gene therapy clinical trials currently underway globally, and 1,000 different manufacturers exploring cell and gene therapies right now, its clear that there is demand for support in this space, he added.

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Ashfield launches 'end-to-end' cell and gene therapy commercialisation network - PMLiVE

Shape CEO Francois Vigneault. (Shape Photo)

Shape Therapeutics, a Seattle preclinical stage biotech company developing RNA editing and gene therapy technologies, has raised $112 million.

The companys RNA editing technologies are spun out of the lab of co-founder Prashant Mali, a bioengineer at the University of California, San Diego who is on Shapes scientific advisory board. Shape is also improving methods to deliver genetic material into cells.

We continue to harness the potential of RNA therapeutics to redefine the standard of care for genetic diseases, said CEO and co-founder Francois Vigneault, former VP of research at Juno Therapeutics, a Seattle flagship cell therapy company that was acquired by Celgene in 2018. Other Juno veterans at the company include Adrian Briggs, head of platform technologies, and David Huss, vice president and head of research.

The company is working on improving AAV vectors, a gene therapy delivery system that is the backbone of FDA-approved gene therapies for spinal muscular atrophy and a rare vision disease. Shape Therapeutics is developing AAV vectors that deliver genetic material directly to the nervous system or muscle, according to a news release. Its vectors can be used to deliver a variety of genetic payloads, including components of its RNA editing technology.

Shapes RNA editing technology could potentially be used to treat genetic conditions, and for other uses such as changing the amount of a key regulatory enzyme in the body.

The company envisions its technology being applied to a wide range of therapeutic areas, Vigneault said in the release. As examples, he named Parkinsons disease, Alzheimers disease, alpha-1 antitrypsin deficiency and Rett syndrome.

One challenge to RNA-editing approaches is that they can yield only low levels of corrected protein, according to Nature.

But one key advantage of RNA editing is that, unlike DNA editing, changing an RNA sequence does not result in permanent effects on the genome, as RNA is only transiently made in the body. The approach may also minimize the possibility that the body will have an immune reaction to the all-human RNA editing components. You really dont need heavy machinery to target RNA, Mali said in an interview with Nature.

The companys scientific board also includes Don Cleveland, who in 2018 was awarded a Breakthrough Prize in Life Sciences for his work on RNA therapies in animal models of Huntingtons disease and Amyotrophic lateral sclerosis (ALS), also called Lou Gehrigs disease.

Shape, founded in 2018, will be jostling with other companies working to improve AAV vectors and gene therapies including Affinia Therapeutics and Dyno Therapeutics. Biotech companies involved in the development of RNA-based therapeutics include Beam Therapeutics, Locana and Korro Bio.

The $112 million Series B financing round was co-led by Decheng Capital and Breton Capital, with participation from Willett Advisors, and continued participation from New Enterprise Associates, and Mission BioCapital. The financing builds on $35.5 million in Series A financing in 2019 with participation from CureDuchenne Ventures, the investment arm of CureDuchenne, a nonprofit dedicated to finding cures for Duchenne muscular dystrophy.

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Seattle-based Shape Therapeutics raises $112M to develop RNA-editing and gene therapies - GeekWire

RESEARCH TRIANGLE PARK Kriya Therapeuticsis poised to revolutionize gene therapies for highly serious diseases like diabetes and severe obesity after landing a whopping $100 million in capital.

Thats on top of $80.5 million raised last year.

The biotech startup, with headquarters in Durham and Palo Alto, secured the Series B financing from Patient Square Capital.

Existing institutional investors also participated in this round, including QVT, Dexcel Pharma, Foresite Capital, Bluebird Ventures, Transhuman Capital, Narya Capital, Amplo and JDRF T1D Fund. New investors included Woodline Partners LP, CAM Capital, Hongkou, Alumni Ventures and others.

The company said proceeds from the financing would be used to further develop Kriyas core technology platforms, expand its therapeutic pipeline and advance its current programs in metabolic disease, ophthalmology and oncology.

Gene therapy startup in Triangle lands $100 million in new funding

Meanwhile, Jim Momtazee, managing partner of Patient Square Capital, will join Kriyas board of directors.

In recent years, we have seen the promise of gene therapy become a reality for the treatment of a number of devastating diseases, said Shankar Ramaswamy, M.D., Kriya co-founder and chief executive officer.

However, the field has been constrained by critical limitations in manufacturing technology, vector design capabilities and cost. Kriya was formed with the mission of revolutionizing how gene therapies are designed, developed and produced by fully integrating advanced manufacturing technologies, computational tools and development capabilities within a single company.

Founded in 2019, Kriya is the brainchild of Ramaswamy, former chief business officer for Axovant Gene Therapies; Fraser Wright, co-founder of Sparks Therapeutics; and Roger Jeffs, the former United Therapeutics CEO who has deep rootsinNorth Carolina.

At present, the company is developing its SIRVE (System for Intelligent Rational Vector Engineering) platform for de novo vector design, sequence modification and data analysis.

It is also developing STRIPE (System to Realize Improved Production Efficiency), a proprietary high-efficiency manufacturing platform integrating advances in cell line technology and upstream and downstream process to achieve exponential reductions in production costs at scale.

STRIPE is being developed at Kriyas 51,000-square-foot manufacturing facility in Research Triangle Park.

The companys full cGMP manufacturing infrastructure is expected to be online this year.

(C) N.C. Biotech Center

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Durham startup's $100M fundraiser could help lead to revolution in gene therapies - WRAL Tech Wire

Botond Roska, M.D., Ph.D., world-renowned scientist and Director at IOB and scientific co-founder of Affinia Therapeutics, will oversee collaboration

Collaboration will expand upon companys leading capsid library approach to broaden the reach of gene therapy beyond rare diseases

WALTHAM, Mass., July 20, 2021 (GLOBE NEWSWIRE) -- Affinia Therapeutics, an innovative gene therapy company with a proprietary platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies for rare and non-rare diseases, today announced a multi-year sponsored research collaboration with the Institute of Molecular and Clinical Ophthalmology Basel (IOB) focused on the central nervous system (CNS). The company will collaborate with IOB to develop novel, next-generation, rationally designed promoters that can enhance gene expression, a key limitation to date in the field. The company has option rights to acquire exclusive licenses to the resulting promoters from the collaboration.

The collaboration with IOB will expand upon Affinia Therapeutics platform of novel capsids by applying its proprietary capsid discovery approach to promoter design. Key features of that approach include rational in silico design of a barcoded library of synthetic promoters, high throughput screening in non-human primates, and large dataset analytics to determine structure-activity relationships. By applying this unique approach, the collaboration is intended to improve upon promoter technologies that optimize when, where, and how much a gene is expressed.

The gene therapy field today has very few promoters for CNS applications, and even those promoters are limited in their control of expression levels and cellular specificity. In partnership with IOB, our team will build upon our leading gene therapy platform and develop promoters that significantly improve expression control in the CNS, said Charlie Albright, Ph.D., chief scientific officer of Affinia Therapeutics.

Story continues

The collaboration with Affinia Therapeutics and IOB will build on IOBs proof of principle research in the retina using pre-clinical models that have successfully translated to humans. In collaboration with Affinia Therapeutics, IOB will initially focus on extending this work to the cortex, with the potential to expand more broadly in the CNS and other tissues over time.

By focusing on developing rationally designed synthetic promoters, we aim to fill a void in the field that will address the well-known limitations in regulating protein expression in the CNS, said Botond Roska, M.D., Ph.D., Director at IOB and scientific co-founder of Affinia Therapeutics. I am excited to lead the team and to apply our extensive knowledge of synthetic promoters to the cortex. With this, IOB will also expand its research strategy.

Founded in 2019, Affinia Therapeutics proprietary capsid discovery platform originates from work done by Luk Vandenberghe, Ph.D., associate professor at Massachusetts Eye and Ear and Harvard Medical School and a co-inventor of AAV9.

About IOBAt the Institute of Molecular and Clinical Ophthalmology Basel (IOB), basic researchers and clinicians work hand in hand to advance the understanding of vision and its diseases, and to develop new therapies for vision loss. IOB started its operations in 2018. The Institute is constituted as a foundation, granting academic freedom to its scientists. Founding partners are the University Hospital Basel, the University of Basel and Novartis. The Canton of Basel-Stadt has granted the institute substantial financial support.

About Affinia TherapeuticsAffinia Therapeutics purpose is to develop gene therapies that can have a transformative impact on people affected by devastating rare and non-rare diseases. Our proprietary platform enables us to methodically engineer novel AAV capsids and gene therapies with potentially improved tissue tropism, cell specificity, safety, and yields. With our innovative science, we are working to broaden the reach of life-changing gene therapies to meaningful numbers of patients with an initial focus on nervous system and muscle diseases with significant unmet need. For more information, visit http://www.affiniatx.com.

Contact Information

Investors:investors@affiniatx.com

Media Affinia Therapeutics:media@affiniatx.com

Media IOB:sandra.schluechter@iob.ch

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Affinia Therapeutics Announces Collaboration with the Institute of Molecular and Clinical Ophthalmology Basel (IOB) To Rationally Design Novel CNS...

By delivering a viral payload of gene silencers directly to the heart, scientists have developed a new strategy for regenerating cardiac muscle after damage from a heart attack. Described in a paper published Wednesday in Science Translational Medicine, the approach led to new cell growth and improved heart function in pigs.

The findings, though preliminary, indicate its possible to prod cardiac muscle cells into regenerating, at least long enough to stave off some of the worst after-effects of a heart attack. If the gene therapy bears out as safe and effective in further testing, it might someday be used to address the root cause of heart failure one of the leading causes of death in the U.S.

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Gene therapy to aid recovery after a heart attack passes a key test - STAT - STAT

The facility will equip AGC Biologics with significant additional capacity: adding 622,000 square feet of operations and office space within six buildings. It is 16 miles from AGC Biologics large-scale stainless steel mammalian facility in Boulder.

AGC Biologics says the new facility will help it expand its end-to-end Cell and Gene Therapy (C>) offering, ensuring security of supply for current and future C> customers.It adds that it plans to hire a 'significant percentage' of the current staff employed by Novartis Gene Therapies at the site

Parent company AGC acquired Italys MolMed in 2020 and has since boosted investment in cell and gene therapy technologies: presenting itself as one of a few CDMOs in the world offering end-to-end services in the space.

In February, AGC Biologics also announced an expansion at the Center of Excellence for Cell and Gene Therapy in Milan, Italy: with increased capacities and viral vector suspension capabilities.

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AGC Biologics to acquire cell and gene therapy manufacturing facility in Longmont, Colorado - BioPharma-Reporter.com

CRISPR gene editing has had a big decade. The technology, which earned two of its discovers a Nobel Prize in 2020, can target and edit genes more easily than its predecessors. Still, as tantalizing (and controversial) as the technologys been over the years, its mostly been developed in the lab.

Thats changing now as a growing number of clinical trials are beginning to test gene therapies in humans.

Early CRISPR trials have focused on hereditary blindness and diseases of the blood, including cancer, sickle cell anemia, and beta thalassemia.

Although cutting-edge, the therapies can be costly and intense. In one trial for sickle cell anemia, doctors remove cells from the body, edit them in a dish, and then infuse them back into the patient. In another trial, practitioners inject the gene editing system directly into target tissues in the eye.

Such approaches wont work as readily for other diseases. So researchers and doctors are looking for a general delivery method, like any other medication. Now, a clinical trial from University College London (UCL) has taken a step in that direction.

Participants in the trial suffer from a condition called hereditary transthyretin amyloidosis, in which a mutated gene produces a malformed protein (transthyretin) that builds up in and damages the heart and nerves. The disease is eventually fatal.

Patients received a single infusion of a CRISPR-based therapy into their bloodstream. Blood carried the therapy to the liver, where it switched off the mutated gene and curtailed production of the errant protein. Though the Phase 1 trial was small, the approach had strong results relative to existing options. And it hints at the possibility other genetic diseases may be treated in a similar fashion in the future.

The University of California, Berkeleys Jennifer Doudna, who shared the Nobel Prize for CRISPR, cofounded Intellia, the company that, alongside fellow biotech company Regeneron, developed the treatment (NTLA-2001) used in the UCL trial.

This is a major milestone for patients, Doudna said. While these are early data, they show us that we can overcome one of the biggest challenges with applying CRISPR clinically so far, which is being able to deliver it systemically and get it to the right place.

The therapy is made up of three parts. A tiny bubble of fat, called a lipid nanoparticle, carries a payload of CRISPR machinery: a strand of guide RNA and a sequence of mRNA coding for the Cas9 protein.

Billions of these CRISPR-carrying nanoparticles are infused into the bloodstream, making their way to the liver, the source of the dysfunctional protein. The mRNA instructs the cells to produce the Cas9 protein (CRISPRs genetic scissors) which then links up with the guide RNA, seeks out the target gene, and snips it.

The cell repairs the DNA at the site of the break, but imperfectly, switching the gene off and shutting down production of the problematic protein.

Interim trial results, reported in the New England Journal of Medicine last weekend, were very encouraging. The trials six patients, who received either a low or high dose, reported no serious side effects. Meanwhile, production of the target protein declined by up to 96 percent (and an average of 87 percent) in those given the high dose.

The disease, which affects some 50,000 people worldwide, was untreatable until recently.

Existing drugs, approved by the FDA in 2018, silence the mRNA that produces the malformed thyretin protein, instead of altering its gene. They reduce protein production some 80 percent and keep people alive longer, but dont work for everyone and require ongoing treatment.

The CRISPR approach, if successful, would be a one-time treatment. That is, by targeting the genes themselves, the protein is permanently silenced.

Patrick Doherty, a trial participant, told NPR he jumped at the opportunity.

Doherty, an avid trekker and hiker, was diagnosed with transthyretin amyloidosiswhich had killed his fatherafter noticing symptoms, like tingling fingers and toes and breathlessness on walks.

Its [a] terrible prognosis, he said. This is a condition that deteriorates very rapidly. Its just dreadful.

Doherty started feeling better a few weeks after the treatment and said improvements have continued.

A one-hit wonder, he called it. A two-hour process, and thats it for the rest of your life.

Although the results are promising, theres reason to temper expectations.

The trial, as noted was small, and focused on safety. Future work will further test safety and efficacy in larger groups, whichas is apparent from recent experience with covidcan reveal rare side effects or prove disappointing despite early success.

Researchers will likely also look out for off-target snips in the liver or other cells. A benefit of this approach, however, is the cells break down the mRNA after theyve made the Cas9 protein. In other words, the gene-editing system doesnt persist long.

It also remains to be seen whether the approach would work as well in other diseases. The liver was a prime target for the trial because it greedily soaks up foreign substances. Other organs and tissues may not be as amenable to a general infusion of the therapy.

Finally, the treatment may come with a hefty price tag, perhaps running into the hundreds of thousands of dollars according to Bloomergs Sam Fazeli.

Not one person in my field is doing a victory lap, even around their laboratory bench,Fyodor Urnov, a University of California, Berkeley gene editing expert, told US Today. Were all slightly blue because were all holding our breath.

If the trial does prove successful, however, researchers will want to know if they can reach any organ or target tissue with a general infusion. And can genes also be edited in vivo? Instead of merely knocking out a faulty gene, can we safely correct it?

In the future, when the kinks have been worked out and the science more thoroughly proven, Urnov said, gene editing could help millions of people around the world with genetic conditions. And this trial, it seems, is a notable step in that direction.

Image Credit: NIH

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Breakthrough CRISPR Gene Therapy Could Be a 'One and Done' Injection - Singularity Hub

Viralgen, the gene therapy CDMO coinedby Bayers Asklepios BioPharmaceutical, is wading into commercial waters.

The Spanish contractor on Wednesday said itcut the ribbon on the first $83 million piece of itsnew factory in the Basque Country, Spain. Viralgen, which specializes in adeno-associated virus (AAV) vectorsthe engineered viruses used to deliver gene therapieshas been helping customers with preclinical through phase 2 projects since 2018.

With the new plant, located in the same San Sebastian technology park as Viralgens existing facility, the company will now be able to tackle projects from pre-clinical stages all the way through commercial medicines, it said.

Thanks to the new plant, Viralgen has expanded its capacity fivefold. The company expects the site will release its first commercial-grade 2,000-liter batches by the middle of next year. Throughout 2021, the company will work to validate its equipment and score certifications.

RELATED:With $71M investment, French CDMO Yposkesi set to double capacity and become a viral vector force

Viralgen has invested more than 70 million($83 million) for the first phase of its manufacturing upgrade. Itplans to drop a total of 120million ($142 million) as it works to buildtwo more buildings there. The company will recruit130 workers to staff thefacility and expects its headcount at the site to surpass 250 by 2022.

Viral vectors are needed for many next-gen drugs, from CAR-T cell therapies and gene therapies to certain COVID-19 vaccinesand thats causing big problems for the industry. The vector shortage is already upon us, and its set to worsen in the coming years unless regulators, biopharmas and contract manufacturers move fast to address shortfalls, GlobalData said in a recent report.

RELATED:CDMO Vibalogics, speeding toward U.S. commercial plant, pumps $50M into viral vector capacity

Answering the call, Viralgen'splant will boastthe distinction of being one of the "largest" AAV factories in the world once it's complete, Viralgen says on its website. The company says it will use the facility to help "democratize" access to gene therapies.

Columbus Venture Partners and Asklepios, also known as AskBio, founded Viralgen in 2017 in a bid to tackle mounting production demands in the gene therapy arena. As of last fall, both Viralgen and AskBio now fly the Bayer banner.

In October, Bayer struck a deal to acquire AskBioand Viralgen by extensionfor $2 billion upfront, plus another $2 billion in milestones. The deal will allow Bayer to tap into AskBios AAV gene therapy platform and a pipeline of clinical-phase treatments for Parkinsons disease, Pompe disease and congestive heart failure.

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Bayer's gene therapy CDMO Viralgen cuts ribbon on first phase of $142M viral vector expansion - FiercePharma