Monthly Archives: July 2021

July 19, 2021 -- There are lots of unfounded fears about the COVID-19 vaccines floating around, and one of the most pervasive is the idea that these new shots aren't really vaccines, but that they will somehow change your genes or insert themselves into the DNA of your cells.

You may see people posting on social media about the vaccines being a kind of gene therapy, and they're partly right, but in the end this idea often misses some important details about how the vaccines work. They can't change your genes, and they don't stay in your body for more than a few days.

But plenty of people have distorted the way the vaccines work into something that could sound sinister. For example, in January, the Weston A. Price Foundation, a group that discourages vaccination, hosted a podcast where David Martin, PhD, described by FactCheck.org as a "financial analyst and self-help entrepreneur," called the vaccines gene therapy.

"a vaccine is supposed to trigger immunity. It's not supposed to trigger you to make a toxin," Martin said. "It's not a vaccination."

Except that these shots are vaccines, according to the US Food and Drug Administration (FDA), and they don't cause you to make a toxin.

So where did this idea get started?

"Like many rumors, there's sort of an element of truth," says Beth Thielen, MD, PhD, a pediatric infectious disease specialist at the University of Minnesota Medical School.

But the truth is that the vaccines involve sound science that sounds complicated to most people not educated in the field.

The vaccines made by Pfizer and Moderna use tiny oily envelopes called lipid nanoparticles to slip a single strand of genetic material called messenger RNA (mRNA) into our cells.

The Johnson & Johnson vaccine is slightly different. It uses double-stranded DNA inserted into a common, but inert virus called an adenovirus. This DNA also contains the instructions for building the spike protein. Once inside the cell, these instructions are read and translated into mRNA.

These bits of mRNA go into the jellied liquid called cytoplasm that makes up the body of our cells.

"Where they join about 200,000 other pieces of messenger RNA that are also sitting in every cell's cytoplasm, because our cells make proteins and enzymes all the time," says Paul Offit, MD, director of the vaccine education center at Children's Hospital of Philadelphia.

The mRNA chains are basically work orders that spell out the instructions for making the spike proteins that stud the outside of the coronavirus that cases COVID-19. The virus uses its spikes to dock onto our cells and infect them.

It's one of the viruses' most recognizable features. Our cells read this mRNA and use them to assemble the spikes. The spikes migrate to the outside of our cells where they are recognized and remembered by our immune system.

These spikes, by themselves, are not dangerous. They can't make anyone sick. They are essentially mug shots that help the body recognize and fight off the real perpetrator when it comes along.

The mRNA chains from the vaccines only last for a couple of days before they break down and the pieces are swept away by the body's normal waste disposal system.

Messenger RNA is genetic material, so in that sense, the vaccines are genetically based therapy.

But the FDA classifies them as vaccines, not gene therapy.

"I think people hear that and they think 'Oh my God, You're going to alter my DNA," Offit says. "That's not possible."

For the vaccines to alter a person's genes, Offit explains, the mRNA instructions would have to enter the cell's control center, the nucleus. The nucleus is walled off from the rest of the cell by its own membrane. To get past that membrane, the mRNA would have to have an enzyme called a nuclear access signal, Offit says, "which it doesn't have."

Even if it could get into the nucleus, the single strand of mRNA would have to be translated back into a double stranded DNA.

HIV, the virus that causes AIDS, can do this. It uses an enzyme like reverse transcriptase to insert itself into our chromosomes. The mRNA in the vaccines lacks this enzyme, so it can't turn back into DNA.

The DNA adenovirus used in the Johnson & Johnson vaccine does enter the nucleus of our cells, but it never integrates into our chromosomes.

Even after those two steps, there's a third firewall between the vaccines and our genes: Another enzyme, called an integrase, would be needed to stitch the new DNA into the DNA of our cells. That's also not in the vaccines.

"So the chances are zero that that can happen," Offit says.

One way to think about mRNA is to imagine if a friend wanted to make a delicious salad that you have the recipe for, Thielen says.

"You'd go to your cookbook, you'd copy the recipe on a note card and give it to them," she says. They can make the salad, but they don't have the cookbook, the original cookbook. You didn't change the cookbook, you just gave them a Post-it note or something that's temporary and that's meant to be," she says.

It's true that these are some of the first vaccines to work this way, but the technology was years in the making. The science was given a final push by billions in funding that was made available through Operation Warp Speed.

The vaccines have now been given to millions of people. They are some of the most effective in the world at preventing severe outcomes from COVID infections. So far, they are holding up well against all the viral variants.

While very rare side effects have been linked to the vaccines, so far, the FDA has determined that the benefit from taking one far outweighs these rare risks for most people.

"I've been astonished, actually,at how well it seems to be working. And so, I think it is very exciting from a vaccine development standpoint that we have new tools in our armamentarium to make new vaccines," Thielen says.

But there's still more to learn.

"I think we need to do dedicated studies of this platform to really understand how long the protection lasts and how well does it adapt to other vaccine targets like RSV. I think that remains to be seen," Thielen says.

Medscape Medical News

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Chance That COVID-19 Vaccines Are Gene Therapy? 'Zero' - WebMD

Media Advisory

Friday, July 16, 2021

Results provide hope for children with aromatic L-amino acid decarboxylase deficiency.

A new study published in Nature Communications suggests that gene therapy delivered into the brain may be safe and effective in treating aromatic L-amino acid decarboxylase (AADC) deficiency. AADC deficiency is a rare neurological disorder that develops in infancy and leads to near absent levels of certain brain chemicals, serotonin and dopamine, that are critical for movement, behavior, and sleep. Children with the disorder have severe developmental, mood dysfunction including irritability, and motor disabilities including problems with talking and walking as well as sleep disturbances. Worldwide there have been approximately 135 cases of this disease reported.

In the study, led by Krystof Bankiewicz, M.D., Ph.D., professor of neurological surgery at Ohio State College of Medicine in Columbus, and his colleagues, seven children received infusions of the DDC gene that was packaged in an adenovirus for delivery into brain cells. The DDC gene is incorporated into the cells DNA and provides instructions for the cell to make AADC, the enzyme that is necessary to produce serotonin and dopamine. The research team used magnetic resonance imaging to guide the accurate placement of the gene therapy into two specific areas of the midbrain.

Positron emission tomography (PET) scans performed three and 24 months after the surgery revealed that the gene therapy led to the production of dopamine in the deep brain structures involved in motor control. In addition, levels of a dopamine metabolite significantly increased in the spinal fluid.

The therapy resulted in clinical improvement of symptoms. Oculogyric crises, abnormal upward movements of the eyeballs, often with involuntary movements of the head, neck and body, that can last for hours and are a hallmark of the disease, completely went away in 6 of 7 participants. In some of the children, improvement was seen as early as nine days after treatment. One participant continued to experience oculogyric crises, but they were less frequent and severe.

All of the children exhibited improvements in movement and motor function. Following the surgery, parents of a majority of participants reported their children were sleeping better and mood disturbances, including irritability, had improved. Progress was also observed in feeding behavior, the ability to sit independently, and in speaking. Two of the children were able to walk with support within 18 months after receiving the gene therapy.

The gene therapy was well tolerated by all participants and no adverse side effects were reported. At three to four weeks following surgery, all participants exhibited irritability, sleep problems, and involuntary movements, but those effects were temporary. One of the children died unexpectedly seven months after the surgery. The cause of death was unknown but assessed to be due to the underlying primary disease.

Jill Morris, Ph.D., program director, NIHs National Institute of Neurological Disorders and Stroke (NINDS). To arrange an interview, please contact nindspressteam@ninds.nih.gov

Pearson TS et al., Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons, Nature Communications, July 12, 2021. https://doi.org/10.1038/s41467-021-24524-8

This study was supported by NINDS (R01NS094292, NS073514-01).

The NINDS NINDS is the nations leading funder of research on the brain and nervous system.The mission of NINDS is to seek fundamental knowledge about the brain and nervous system and to use that knowledge to reduce the burden of neurological disease.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

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funded study finds gene therapy may restore missing enzyme in rare disease - National Institutes of Health

ByGina Wadas

Viruses are experts at infiltrating the body, as the SARS-CoV-2 virus (and resulting COVID-19 pandemic) have amply demonstrated. But their efficiency in targeting specific and isolated cells also make them useful drug delivery vehicles, known as viral vectors.

Viral vectors are modified viruses that can act as couriers to transport therapeutic "packages" to specific diseased cells. These packages contain instructions with modified or designed DNA or RNA to correct or supplement a faulty or missing gene. For instance, the Johnson & Johnson COVID-19 vaccine uses viral vectors to transport modified genetic material from the SARS-CoV-2 virus to cells, generating an immune response.

Though viral vector-based gene therapies are among the most advanced treatments for many congenital and acquired diseases, producing them is complex and costly.

"One of the major challenges in viral vector gene therapy is how to improve the quality, purity, and cost of the manufactured viral vectors, so that we can use the smallest possible effective dose, reduce immune side effects, and lower the cost of treatments," said Hai-Quan Mao, associate director and core faculty member of the Institute for NanoBioTechnology. He is also a professor in the departments of Materials Science and Engineering and Biomedical Engineering and a core faculty member at the Translational Tissue Engineering Center.

Hai-Quan Mao

Associate director, Institute for NanoBioTechnology

To address this challenge, Mao and his team are teaming up with Nolan Sutherland, senior scientist at bluebird bio, a Cambridge, Massachusetts-based biotechnology company that develops gene therapies. The partnership started about two years ago when Yizong Hu, a biomedical engineering PhD student under the mentorship of Mao, was at an annual meeting for the American Society for Gene and Cell Therapy presenting his research on a new particle assembly technology. Sutherland heard the presentation and approached Hu to discuss the technology and its application to the production of lentiviral vectors, which are made from a family of viruses that infect people by reverse transcription of their RNA into DNA in their host cells' genome.

Sutherland thought that the Mao team's approach might help streamline transfection, a key step in producing viral vectors. During transfection, a polymer solution is combined with a mixture of DNA plasmids to form transfection particles, a cumbersome procedure involving complicated solution blending and strictly timed dosing.

Mao, Hu, and Yining Zhu, also a biomedical engineering PhD student, developed a more effective and shelf-stable formulation of DNA particles in a ready-to-dose form. They also discovered that size-controlled sub-micron particles are most effective in transfecting cells and producing viral vectors. This production method is based on the team's years of experience in controlling transfection vehicle characteristics to enhance performances and stability.

The team members validated their findings with Sutherland at bluebird bio using that company's bioreactor. They compared the new method with the industry standard, and the results showed improved vector production yield, shelf stability, handling stability, and quality control of the transfection process.

"With the drastic increase in demand for lentiviral vector-based cell therapy products ... this new technology will greatly improve the production quality, consistency, and yield of our therapeutic LVVs," Sutherland said.

The team reported its findings in Nano Letters and is scaling up production with an eye to transferring the technology to the marketplace.

"This work represents a great example how we can partner with corporate collaborators to accelerate the translation of discoveries on the bench to the industry. This type of collaboration with industry provides us opportunities to identify the technical gaps in the engineering solutions that we develop, and fine tune them to better address the real-world problems in a more targeted fashion," Mao said.

According to Sutherland, the partnership with Mao and his team has "allowed bluebird to pursue high risk/reward innovation in a space outside of its core expertise. The team has a keen eye for application to industry which has made the partnership incredibly productive."

Team members say that this new particle engineering technology will find a wide range of applications in the manufacture of a variety of viral vectors for gene and cell therapy applications.

Also contributing to the project are Jordan Green, professor in the Department of Biomedical Engineering and associate member at the INBT, and Sashank Reddy, assistant professor of plastic and reconstructive surgery at Johns Hopkins Medicine, medical director at Johns Hopkins Technology Ventures, and affiliate faculty member at the INBT.

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Researchers partner with industry to create better gene therapy tools - The Hub at Johns Hopkins

Scientists from Ohio State University have developed a novel method for delivering gene therapy to specific regions of the brain. Now, they have evidence from a small clinical trial in children that the treatment could address a rare, inherited neurodegenerative disease. And they believe their technique could eventually be used to treat more common brain diseases, like Alzheimers and Parkinsons.

The Ohio State team developed the gene therapy to treat aromatic L-amino acid decarboxylase (AADC) deficiency, which hampers the bodys ability to make dopamine and serotonin and results in developmental delays and a range of motor and behavioral symptoms. The gene therapy uses a viral vector to carry DNA-expressing AADC to the brain.

In seven children with AADC deficiency, the gene therapy boosted the metabolism of dopamine, the researchers reported in Nature Communications. Within three months, six of the children were no longer experiencing oculogyric crises (OGC), which are eye spasms that commonly occur in children with the disorder. After a year, six of the participants had normal head control, the researchers said.

During the trial, the Ohio State team delivered the gene therapy directly to the midbrain, monitoring the spread of the DNA in real time using MRI imaging. They reported dramatic improvements in several symptoms beyond OGC, including sleep disturbances and irritable mood, according to the paper. Improvements in motor function took longer to emerge, they wrote, but were markedly better than what would normally be expected to occur in the natural course of AADC deficiency for patients in this age range (49 years) who have severe motor impairment.

RELATED: Taking gene therapy to the masses with innovations in diabetes, Alzheimer's and more

The ability to deliver gene therapy directly to the brainand monitor its effects with advanced imagingcould enhance efforts to treat a range of neurodegenerative disorders, said Krystof Bankiewicz, M.D., Ph.D., neurological surgery professor at the Ohio State College of Medicine, in a statement.

In fact, a handful of companies are working toward that goal. They include Lexeo Therapeutics, which is developing a gene therapy to deliver the APOE2 gene into the central nervous system in the hopes that it will slow the development of Alzheimers in people with two copies of the the high-risk APOE4 variant.

Another company examining brain-delivered gene therapy is VectorY, which recently raised $38 million to advance its work. VectorY is using viruses to carry genes into the brain that can encode therapeutic antibodies. It is using the newly raised funding to complete preclinical work on therapies for amyotrophic lateral sclerosis and Alzheimers.

Based on the success of their early-stage trial in AADC deficiency, the Ohio State researchers are planning clinical trials of their brain-delivered gene therapy in other incurable, debilitating diseases, they said.

The gene therapy approach described here represents many years of careful work to develop and to understand effective ways to deliver gene therapy to the brain, they wrote in the study. This work provides a framework for the treatment of other human CNS genetic diseases, and iterative refinement of the individual components of this approach will facilitate broader application.

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Gene therapy delivered to the brain shows promise in children with rare neurodegenerative disease - FierceBiotech

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.

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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...