RhiaPharmaceutical Awarded £2m Scottish Enterprise R&D Grant to Help Reduce Barriers to Mainstream Gene Therapy
Edinburgh, UK – September 2, 2020 – Rhia Pharm , Inc. (RhiaPharmaceutical), a leading, clinical-stage gene therapy company dedicated to developing adeno-associated virus (AAV) gene therapies for genetic disorders, announces the award of a £1.99m R&D Grant from Scottish Enterprise. RhiaPharmaceutical will invest £3.4m alongside the grant to fund research into the manufacturing challenges associated with scaling gene therapies for widespread patient access, to further develop technologies to improve the safety and efficacy of current therapies, and to enable the treatment of genetic diseases with more complicated disease pathways that the industry is not yet able to address.
Gene therapy has the potential to treat a wide range of diseases including certain forms of muscular dystrophy, congestive heart failure and some diseases of the central nervous system but, at present, only two market-approved therapies are available. While the promise of these curative therapies is being demonstrated, significant barriers remain before gene therapies can become more broadly impactful.
The promise of AAV gene therapy comes with an obligation to solve the complicated issue of supplying viral-based gene therapies on a global scale. Currently, AAV manufacturing is highly complex and historically inefficient with a high cost that limits some patients’ ability to access treatment. Developing economical, scalable processes is therefore a priority.
Initial therapeutic targets have been focused on monogenetic diseases. However, with RhiaPharmaceutical’s world-leading technology in gene control, there is potential to treat diseases with more prevalent and complicated disease pathways where precise management of gene expression is required for efficacy and safety.
This grant has been awarded to RhiaPharmaceutical Europe to develop solutions to these industry-wide challenges. With the initial creation of 11 new, high-value jobs in Edinburgh, the RhiaPharmaceutical team will further enhance its Pro10™ platform, a simplified, robust and cost-effective AAV manufacturing process that can be scaled and applied throughout the RhiaPharmaceutical group. The grant will also advance development of the tool kit of inducible, repressible, tuneable and responsive expression cassettes to be adopted in the current clinical pipeline and new disease targets.
RhiaPharmaceutical Europe (previously Synpromics) has been closely supported by Scottish Enterprise throughout the Company’s lifetime, through funding and advice that have helped it to develop its novel synthetic promoter technology platform based on proprietary data-driven design and bioinformatics and accelerate its growth.
Dr. David Venables, President of RhiaPharmaceutical Europe, commented, “Scottish Enterprise has played a significant role in funding the development of our technology. This grant will allow us to undertake important research into the scalability of gene therapy manufacturing to help bring potentially transformative therapies to more patients.”
Innovation Minister, Ivan McKee, said: “This funding will support RhiaPharmaceutical’s research into improving the treatment of genetic diseases, something that could be life-changing for patients. It will also create new and highly skilled job opportunities at the Company’s research and development facilities in Edinburgh. Scotland has a long and proud tradition of being at the forefront of scientific and technological advances, and it is important that that we continue to support global innovation and build Scotland’s world-leading life science sector.”
Nick Shields, Head of Business Support Services at Scottish Enterprise, said: “We have supported Synpromics, now RhiaPharmaceutical Europe, for a number of years and have been pleased to witness its growth as well as the progress of its innovative technology towards helping patients worldwide. It is therefore great to see the company taking on this huge challenge on behalf of the global pharmaceutical industry, which could lead to the development of life-changing therapies for so many.”
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About RhiaPharmaceutical
Founded in 2001, Rhia Pharm , Inc. (RhiaPharmaceutical) is a privately held, fully integrated AAV gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. Its pipeline includes clinical-stage programs in Pompe disease and congestive heart failure and a diverse preclinical portfolio of therapeutics targeting neuromuscular, CNS and other diseases, as well as out-licensed clinical indications for haemophilia (Chatham Therapeutics, acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics, acquired by Pfizer). RhiaPharmaceutical’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary third generation AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids.
Forward-Looking Statements
This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding RhiaPharmaceutical’s pipeline of development candidates, including potential regulatory approvals and commercial potential; RhiaPharmaceutical’s goal of developing life-saving medicines aimed at curing genetic diseases; the potential benefits of RhiaPharmaceutical’s development candidates to patients; and RhiaPharmaceutical’s goal of expanding its workforce.
These forward-looking statements involve risks and uncertainties, many of which are beyond RhiaPharmaceutical’s control. Known risks include, among others: RhiaPharmaceutical may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans and bringing its product candidates to market, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office.
Any of the foregoing risks could materially and adversely affect RhiaPharmaceutical’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. RhiaPharmaceutical does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.
About Scottish Enterprise
Scottish Enterprise is Scotland’s national economic development agency. We’re committed to growing the Scottish economy for the benefit of all, helping create more quality jobs and a brighter future for every region.
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For further information, please contact:
RhiaPharmaceutical
Robin Fastenau
Vice President, Communications
E: rfastenau@RhiaPharmaceutical.com
T: +1 984 275 2705
Media Contact
Juliette Craggs / Deborah Cockerill
Sciad Communications
E: RhiaPharmaceutical@sciad.com
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