Category Archives: Genetic Therapy

Synthetic biology-focused Twist Bioscience has teamed up with T cell therapy biotech Neogene Therapeutics to develop next-generation personalised T cell therapies, including chimeric antigen receptor T (CAR-T) cell and T cell receptor (TCR) therapies, for cancer.

According to the terms of the agreement, Twist co-founder and CEO Emily Leproust explains, Neogene will leverage Twists antibody Library of Libraries to create sequences of neo-antigens for future CAR-T therapies.

In addition, we will provide a synthetic TCR library with a very, very high degree of diversity to identify optimal sequences for [TCR] therapies, for two specific tumour targets, notes Leproust. We are thrilled that Neogene has partnered with Twist to leverage our platform in a broad-based way, both to create synthetic sequences as well as a synthetic DNA library fortherapeutic discovery, with the ultimate goal of creating a truly personalised cancer therapy.

In return, Twist will receive technology access fees, as well as milestone and royalty payments on preclinical, clinical and commercial milestones for any therapies resulting from the collaboration.

Neogene will apply its broad expertise on engineering T cell therapies and its proprietary technology to screen and identify specific T cell receptor genes from Twists synthetic libraries, explains CEO Carsten Linnemann.

Founded in 2018, Neogene is a pre-clinical biotech focused on developing pioneering, next-generation personalised T cell therapies for oncology indications. It relies on the cell therapy expertise of its two co-founders, Linnemann and immunologist Dr Ton Schumacher. They previously co-founded T-cell Factory, which was acquired by Kite Pharma in 2015.

Focused on neo-antigens, Neogens proprietary technology platform aims to identify TCR genes with specificity for neo-antigens from a tumour biopsy, explains Linnemann.

Since every cancer expresses a unique set of neo-antigens, Neogenes novel approach delivers a tailored set of TCR genes for each individual patient, adds Linnemann. These TCR genes are engineered into patient-derived T cells directing them towards neo-antigens in tumour cells, thereby providing a fully personalised engineered T cell therapy for cancer.

Currently, only two T cell therapies have been approved Novartiss Kymriah and Gileads Yescarta. These are both CAR-T therapies and are indicated for very small blood cancer patient populations.

Although these products and all the others currently in development are revolutionising cancer treatment, T cell therapies have many limitations. These include lengthy vein-to-vein times, variable potencies, high production costs and inability to create a product inventory, according to Linnemann.

However, Linnemann believes synthetic biology tools, like those offered by Twist, have the potential to further reduce timelines and costs of engineered cell therapies.

In addition, the promise of T cell therapies in blood cancers has not been repeated for solid tumours, where there remains a huge unmet need. But, Linnemann argues, synthetic gene libraries can provide a novel tool for the rapid discovery of TCR leads, therefore, expediting the development of TCR therapies for solid tumours.

Leproust agrees, writing in a statement: Putting our platforms together, we believe we will be able to expedite the identification and genetic engineering of TCR genes to create personalized T cell therapies for cancer, bringing new hope to address the current limitations of treatments available today.

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Twist and Neogene partner to develop next-gen T cell therapies - pharmaceutical-technology.com

From being a thing of science fiction to topping the priority list of every big pharma player, gene therapies have come a long way. With multiple approved products, the field of gene therapies has gained substantial momentum over the last couple of decades. The growing burden of genetic disorders and the rising evidence of gene therapys potential in curing these diseases has led to a worldwide interest in Gene Therapy.

Gene therapies have shown significant potential in the preclinical studies, as well as clinical trials. For instance, a study that was published in The New England Journal of Medicine showed that seven of the babies suffering from SCID, when treated with gene therapy, developed immune systems that can protect them against common childhood ailments. In fact, we now have gene therapies being developed for some of the more common indications, such as Alzheimers.

Get a complete list of the presentations,here.

The evolution of the regulatory landscape to facilitate the development of these therapies has further helped pharma companies to develop novel solutions. One of the Nature articles, published last year, talked extensively about how the release of standard guidelines has helped pharma players develop products in this space. Whatever be the reason, the fact that more than 10 gene therapies are already approved and close to 470 are in development, is already hinting towards a positive future.

A promising preclinical/discovery stage pipeline with diverse therapeutic reach does point towards increasing research efforts in this space.

While the development landscape is heavily crowded, the developers are adopting different approaches for developing novel gene therapies.

Overall, gene therapies hold immense potential in overcoming some of the key unmet needs of the patients. Also, given the estimated market size of close to $12 billion by 2030, this remains a highly lucrative space for drug developers.

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Gene Therapy Market: A Strong Pipeline with $12 Billion Opportunity by 2030 | Roots Analysis - The Think Curiouser

EDMONTON, AB, Oct. 27, 2020 /CNW/ -Entos Pharmaceuticals (Entos), a Canadian biotechnology company developing genetic medicines with its breakthrough Fusogenix nucleic acid delivery platform, is pleased to announce its work with Applied Pharmaceutical Innovation in enhancing its Alberta-based vaccine production capacity.

Applied Pharmaceutical Innovation is a Canadian not-for-profit institute focused on providing translational capacity for life sciences companies. With a broad expertise and a network of over 11 institutions and centers of excellence, API provides capacity for companies at all stages of the drug development pathway. API's engagement with Entos started in May 2020, providing the rapidly growing team at Entos with enhanced space as they expanded their presence in Edmonton with the rapid development of their Fusogenix DNA vaccine candidates for SARS-CoV-2.

Entos is pleased to announce that API has now begun local GMP production of inputs for Entos clinical trial manufacturing, adding supply chain resiliency to Entos' manufacturing platform components for the clinical and commercial production of its SARS-CoV-2 vaccine. This is the first step as Entos works with API to secure enhanced Canadian-based capacity including fill and finish for the local and global market as part of API's broader security of supply facility development initiative.

"We're thrilled to be working with API on this." said John Lewis, CEO, Entos Pharmaceuticals. "Combined with our Fusogenix genetic medicines platform, we are developing a robust, cost effective pathway to produce millions of doses of our COVID-19 DNA vaccine as well as future vaccines and therapeutics."

"We see Entos as a company with a very bright future," said Andrew MacIsaac, CEO, Applied Pharmaceutical Innovation, "Working with them to enhance their efforts addressing the COVID-19 crisis is a very meaningful investment of energy and effort. The potential for our region and Canada, as we secure manufacturing resiliency with them, is tremendous."

API is in the process of establishing a 40,000 sq. ft manufacturing plant in Edmonton that includes active pharmaceutical ingredient production and fill-finish capacity for sterile products to secure gaps in the Canadian pharmaceutical supply chain.

About Entos Pharmaceuticals, Inc.Entos develops next generation nucleic acid-based therapies using its Fusogenix genetic medicines delivery platform. Fusogenix is a proteo-lipid vehicle (PLV) formulation that uses a novel mechanism of action to deliver nucleic acid, intact and unmodified, directly into the cytosol of target cells. The technology applies a wide range of therapeutic types including gene therapy, mRNA, miRNA, RNAi, and CRISPR. For more information, visit http://www.entospharma.com.

About Applied Pharmaceutical InnovationApplied Pharmaceutical Innovation (API) is a federally incorporated Canadian not-for-profit institute based in Edmonton, Alberta. API provides a wide range of services for industry and innovators who are pursuing the commercial development of life sciences discoveries. From GMP manufacturing to assays and analysis, API provide comprehensive pre-clinical and clinical development for companies on both a contract and capacity building basis.

SOURCE Entos Pharmaceuticals

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Entos Pharmaceuticals Working with Applied Pharmaceutical Innovation to Enable In-house Development and Manufacturing Capability - BioSpace

Luke Timmerman interviews Sana Biotechnology CEO Steve Harr at the GeekWire Summit this week.

Sana Biotechnology CEO Steve Harr shed more light on one of most secretive, heavily funded startups in Seattle and the global biotech industry detailing its plans to create tools that replace and repair human body cells, with the potential to treat various diseases and create new medicines.

Harr spoke with biotechnology journalist Luke Timmerman, founder of The Timmerman Report, this week at the GeekWire Summit. Sana raised more than $700 million this summer in one of the largest venture financing deals in the life sciences industry and one of the biggest rounds on record in Seattle.

Founded in 2019, the 250-person company has an ambitious goal of both repairing cells in the body (gene therapy) and also replacing damaged cells (cell therapy). Its led by several former executives from Juno Therapeutics, another Seattle biotech company that went public in 2014 and sold to Celgene for $9 billion in 2018.

Sana has kept a relatively low profile since launching. It is competing with much larger entities that have deeper pockets and more robust logistics capabilities. But Harr said a startup such as Sana has a key differentiator.

We have one competitive advantage: we can make faster and better decisions, he said. We get there because we have better people, we have greater focus, and we have better communication.

Read on for key takeaways from the conversation.

How Sana started:Harr and his former colleagues at Juno learned a lot about engineering cells and manipulating genes during their startup journey. Juno was among a handful of U.S. companies making cutting-edge cancer immunotherapy treatments.

But they also knew there was more opportunity in a nascent industry of gene and cell therapy.

We wanted to build the transformative or winning company of this next era, of the next 20 years, Harr said. To do that, we had to break the model of what biotech is, which is typically taking an idea and figuring out where to apply it best.

Sana instead is trying to build the platform that can engineer cells and fix them, much like building a computer.

There are a whole host of component parts that go into it, Harr said. We have to aggregate the right technologies.

Harr said too many biotech companies sell solutions in search of problems. He likened it to someone showing up with a tiny screwdriver and looking for a loose screw to fix. Harr sees Sana more as a toolbox that can help build the right medicine for the right patient.

Sana is targeting various disease areas, including cancer, diabetes, genetic disorders, and more. They are relatively diverse, but there are some really fundamental underlying platform and strategy principles that drive each of those, Harr said.

Sanas secret sauce:One key focus for the company is reimagining the delivery system for these therapies how to get DNA, RNA, proteins, etc. into a cell. Ultimately at the core, what were trying to do is really improve delivery, and really figure out how to hide cells from the immune system, Harr said.

Hiding re-engineered or replaced cells from ones immune system is important to prevent the possibility of the body rejecting the new cells.

Harr also talked about delivering therapy via injection, with the body becoming the bioreactor. Its similar to technology built by Moderna and others. You deliver the tools to enable your body to make its own medicine, Harr said.

Manufacturing:Sana is also aiming to innovate how gene and cell therapies are produced and distributed at scale. They are typically expensive Timmerman said CAR T-cell immunotherapies for cancer ran in the $300,000-to-$400,000 range per patient. Figuring out manufacturing costs at scale and making it less than current alternative methods of care for patients will be key to the strength of Sanas business. Harr added that you have to do it in a way thats constructive for the system.

Headcount: Sana employs 250 people spread across offices in Seattle, the Bay Area, and Cambridge, Mass. Having three outposts helps the company attract the best talent, Harr said. One advantage to raising so much capital is being able to hire the best folks. Last month Sana added top scientists Ed Rebar and Terry Fry to the executive team.

If you really hire one of the true world leaders in something, it is pretty amazing how quickly teams form around them, Harr noted.

Money matters: Having more than $700 million in the bank helps Sana in various other ways. Harr said a lot of biotech companies often run what amount to experiments to justify raising more capital. We have the privilege of running experiments to find truth as fast as you want to, he said. And then we want to have the balance sheet, technologies, and people to be able to grapple with whatever the truth is.

Timeline: Harr said the company is on track with its original strategy but does not plan on selling medicine in the next two years. It will progress with multiple medicines in parallel, not one at a time, Harr said.

Leadership advice:During the pandemic and remote work, Harr said hes started to reach out to four-to-six people at Sana each week that he wouldnt normally talk with. He holds half-hour meetings to chat about what they are working on, and what leadership could do to make their life or job better. I found that to be just such an invigorating way to learn whats going on, he said.

[The full interview with Harr, and other GeekWire Summit sessions, are available on-demand exclusively to attendees of the virtual event.Learn more and register here.]

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Sana CEO reveals details about stealthy gene therapy startup that has raised more than $700M - GeekWire

-Collaboration combines Solids differentiated microdystrophin construct and Ultragenyxs HeLa PCL manufacturing platform for use with AAV8 and variants-

-Solid receives $40 million upfront via equity investment at a premium; up to $255 million in milestones plus royalty payments-

-Solid retains exclusive rights to all other uses of its microdystrophins, including its existing SGT-001 program-

NOVATO, Calif. and CAMBRIDGE, Mass., Oct. 23, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, and Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today announced a strategic collaboration and license agreement to focus on the development and commercialization of new gene therapies for Duchenne. The parties will collaborate to develop products that combine Solids differentiated microdystrophin construct, Ultragenyxs HeLa producer cell line (PCL) manufacturing platform, and AAV8 variants. The collaboration also brings together Solids expertise in muscle biology and Ultragenyxs expertise in bringing novel therapies to patients with rare diseases.

Under the terms of the collaboration, Solid granted Ultragenyx an exclusive license for any pharmaceutical product that expresses Solids proprietary microdystrophin construct from AAV8 and variants thereof in clade E for use in the treatment of Duchenne and other diseases resulting from lack of functional dystrophin, including Becker muscular dystrophy. Ultragenyx has made a $40 million investment in Solid and has agreed to pay up to $255 million in cumulative milestone payments per product upon achievement of specified milestone events, and tiered royalties on worldwide net sales at low double digit to mid-teens percentages. Upon achievement of proof-of-concept, Solid has the right to opt-in to co-fund collaboration programs in return for participation in a profit share or increased royalty payments.

We believe that Solids microdystrophin is best-in-class with its unique neuronal nitric oxide synthase binding domain, said Emil D. Kakkis, MD, PhD, Chief Executive Officer and President of Ultragenyx. By using an AAV8 variant validated in prior human and other studies combined with our scalable, efficient HeLa producer cell line platform, we believe we can leverage our mutual strengths to develop a high-quality AAV-based treatment alternative for Duchenne.

Ultragenyx has a demonstrated track record of success in developing and commercializing innovative therapies for rare diseases, said Ilan Ganot, Co-Founder, President and Chief Executive Officer at Solid Biosciences. We believe it is the partner of choice for exploring new gene therapy opportunities for patients with Duchenne.

Solids proprietary microdystrophin construct has exhibited functional benefit in preclinical models. In preclinical studies, animals expressing a microdystrophin capable of restoring neuronal nitric oxide synthase (nNOS) resisted fatigue better than those expressing a microdystrophin that does not. Patients dosed with Solids proprietary microdystrophin construct at the 2E14 vg/kg dose in Solids ongoing IGNITE DMD clinical trial have also preliminarily demonstrated nNOS activity and function, further validating these preclinical results. Solid expects to dose the next patient in the IGNITE DMD clinical trial, using SGT-001 produced using its improved HSV manufacturing process, in the first quarter of 2021.

Ultragenyx intends to use its AAV-based HeLa PCL platform including HeLa 3.0 improvements for the development of product candidates. The platform enables large 2,000 liter commercial-scale AAV-based gene therapy product manufacturing. The PCL platform yields high-quality product from a highly reproducible, highly scalable, and less expensive process a distinct vantage in higher dose indications like Duchenne. The capsid planned is an AAV8 variant with a favorable immunological profile that has been used successfully in the large scale 2,000 liter production process.

About Ultragenyx

Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyxs strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency and ensuring majority access to its therapies for patients who can benefit.

Ultragenyx currently has three AAV gene therapies in clinical development, including DTX201 that uses an AAV8 variant in the HeLa PCL platform and that is partnered with Bayer, who has released positive Phase 1/2 data in Hemophilia A. The companys other clinical AAV8 gene therapies, DTX301 and DTX401, are in Phase 1/2 studies for ornithine transcarbamylase (OTC) deficiency and glycogen storage disease type Ia (GSDIa), respectively. An investigational new drug (IND) application is expected by the end of 2020 for a fourth AAV gene therapy for Wilson disease, which will also use the HeLa PCL AAV manufacturing platform.

For more information on Ultragenyx, please visit the companys website at http://www.ultragenyx.com.

About Solid Biosciences

Solid Biosciences is a life sciences company focused on advancing transformative treatments to improve the lives of patients living with Duchenne. Disease-focused and founded by a family directly impacted by Duchenne, our mandate is simple yet comprehensive work to address the disease at its core by correcting the underlying mutation that causes Duchenne with our lead gene therapy candidate, SGT-001.

Solids SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne. Duchenne is caused by mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including nNOS. Data from Solids preclinical program suggests that SGT-001 has the potential to slow or stop the progression of Duchenne, regardless of genetic mutation or disease stage.

SGT-001 is based on pioneering research in dystrophin biology by Dr. Jeffrey Chamberlain of the University of Washington and Dr. Dongsheng Duan of the University of Missouri. SGT-001 has been granted Rare Pediatric Disease Designation, or RPDD, and Fast Track Designation in the United States and Orphan Drug Designations in both the United States and European Union.

For more information, please visit http://www.solidbio.com.

Ultragenyx Forward-Looking Statements

Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions,the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the effects from the COVID-19 pandemic on the companys clinical activities, business and operating results, uncertainty and potential delays related to clinical drug development, smaller than anticipated market opportunities for the companys products and product candidates, manufacturing risks, competition from other therapies or products, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the companys future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyxs products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with theSecurities and Exchange CommissiononJuly 30, 2020, and its subsequent periodic reports filed with theSecurities and Exchange Commission.

Solid Biosciences Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding whether the collaboration will yield any viable product candidates, potential milestone payments or royalty payments in connection with the collaboration, the potential benefits of the collaboration, the safety or potential efficacy of SGT-001 and other statements containing the words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, would, working and similar expressions. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with each partys ability to perform its obligations under the collaboration, the Companys ability to resume and/or continue IGNITE DMD on the timeline expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; obtain and maintain the necessary approval from investigational review boards at IGNITE DMD clinical trial sites and the IGNITE DMD independent data safety monitoring board; enroll patients in IGNITE DMD; continue to advance SGT-001 in clinical trials; replicate in clinical trials positive results found in preclinical studies and earlier stages of clinical development; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; successfully optimize and scale its manufacturing process; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop DMD/Duchenne treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-001, achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Companys actual results to differ from those contained in the forward-looking statements, see the Risk Factors section, as well as discussions of potential risks, uncertainties and other important factors, in the Companys most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Companys views as of the date hereof and should not be relied upon as representing the Companys views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

Contacts:

Ultragenyx Joshua Higa(415) 475-6370

Solid BiosciencesInvestor Contact:David CareyFINN Partners212-867-1768David.Carey@finnpartners.com

Media Contact:Erich SandovalFINN Partners917-497-2867Erich.Sandoval@finnpartners.com

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Ultragenyx and Solid Biosciences Announce Strategic Collaboration to Develop and Commercialize New Gene Therapies for Duchenne Muscular Dystrophy -...

Paris-based SparingVision, a genomic medicine company focused on ocular diseases, raised 44.5 million (approximately $52.2 million) in a financing round. Funds will be used to advance the development of the companys treatment for a genetic eye disorder that can lead to vision loss.

SparingVision is developing SPVN06 for the mutation-agnostic treatment of retinitis pigmentosa, the most common inherited retinal degeneration that affects about two million people globally. There is currently no approved treatment to treat all genetic forms of this rare retinal disease that leads to blindness, the company said. According to SparingVision, SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy consisting of one neurotrophic factor and one oxidative stress reducing enzyme which, acting synergistically, aim to slow or stop the degeneration of photoreceptors. Loss of photoreceptors leads to blindness in retinitis pigmentosa. In June, the European Commission granted Orphan Drug designation to SPVN06

In addition to advancing its gene therapy treatment, funds from the financing round will be used to support SparingVisions GMP activities, including the manufacturing of a first clinical batch of the product, as well as regulatory activities. Funds will also be used to begin human trials of the gene therapy, which are set to begin in 2021.

Gene therapy has already been approved by regulatory to approve a type of genetic blindness. Spark Therapeutics, now a division of Roche, won regulatory approval for Luxturna (voretigene neparvovec), a gene therapy for a rare, genetic form of blindness. Luxturna is approved for the treatment of pediatric and adult patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. The disease can lead to vision loss and may cause complete blindness in certain patients. The approval marked the first time the U.S. Food and Drug Administration approved a directly administered gene therapy that targets a disease caused by mutations in a specific gene.

In addition to developing its gene therapy for retinitis pigmentosa, SparingVision said it intends to establish a toehold in the United States and will expand its management team.

The financing round was led by 4BIO Capital and UPMC Enterprises. It was supported by Jeito Capital and Ysios Capital. Current investors Bpifrance and Foundation Fighting Blindness also participated in the round. Torreya Capital, LLC served as exclusive placement agent for the offering.

In addition to the financing, Stphane Boissel, who currently serves as chairman of the board of directors, was named chief executive officer of the company. He takes over from Florence Allouche, a cofounder of the company. Boissel, who previously served as head of corporate strategy at Sangamo Therapeutics, said the support SparingVision received in the financing round demonstrates the excitement about the potential of SPVN06.

With its singular mutation-agnostic approach, SPVN06 could have a much broader commercial potential than most gene therapy products for RP currently in development and will be used as an anchor to build an economically-viable portfolio of therapies in the field of ophthalmology. Our shareholders, both new and existing, are all long-term, strategic and patient-centric investors that share our vision and we are excited to be working with them to achieve our goals, Boissel said in a statement.

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SparingVision Nabs 44.5 Million to Support Gene Therapy, Adds New CEO - BioSpace