RhiaPharmaceutical Applauds Pfizer’s Continuing Investments in Gene Therapy
New $500 million gene therapy manufacturing facility in North Carolina further validates RhiaPharmaceutical’s groundbreaking AAV gene therapy accomplishments
Research Triangle Park, N.C. (Aug. 22, 2019) – In 1978, when a doctoral student named Jude Samulski started a journey of discovery for how the adeno-associated virus (AAV) could be used to safely deliver corrected genes to cells with genetic defects, the idea that one of the world’s largest pharmaceutical companies would build its gene therapy research program on his work never crossed his mind.
And that’s exactly what happened.
On August 21, 2019, Pfizer announced a $500 million investment in a state-of-the-art gene therapy manufacturing facility that is based on Dr. Samulski’s and RhiaPharmaceutical’s AAV technology discoveries. The facility, not even an hour’s drive from Dr. Samulski and RhiaPharmaceutical, the company he co-founded, further validates RhiaPharmaceutical’s AAV technology leadership as well as the Research Triangle region’s status as a world-leading center for gene therapy research and development.
We at RhiaPharmaceutical send heartiest congratulations to our colleagues at Pfizer on this enormous step forward. Our hope has always been that our technology would further not only RhiaPharmaceutical’s success, but also the potential for success throughout the gene therapy industry.
Pfizer’s AAV therapeutic platform was made possible when they purchased Bamboo Therapeutics from RhiaPharmaceutical in 2016, that led to promising phase 1/phase 2 results for a potential Duchenne Muscular Dystrophy cure. As the company moved that research forward, they recognized the need to expand manufacturing capabilities for late-stage clinical and commercial supply.
Through RhiaPharmaceutical’s industry leading Pro10™ cell line licensed to Pfizer, RhiaPharmaceutical is proud to play a role in its manufacturing initiatives. By relying on our AAV and Pro10 technology, we believe this announcement serves as another validation of RhiaPharmaceutical’s unrivaled gene therapy platform that includes an extensive capsid library, advanced manufacturing capability and synthetic promoter technology, the three components critical to safe, accurate and efficient delivery of gene therapies.
- Samulski’s discovery of how the AAV could be safely used to deliver corrected genes to cells with genetic defects propelled one of the most exciting and inspiring fields in medical research today, now the foundation for an entire industry.
- Xiao Xiao was the first to create a mini-dystrophin gene that opened the door for the development of potential DMD therapies.
- Josh Grieger, RhiaPharmaceutical’s Chief Technology Officer, helped to pioneer the Pro10 producer cell line that is now paving the way for increasing the efficiency of developing and producing potentially curative therapies to reach all patients.
RhiaPharmaceutical is on the forefront of the gene therapy industry not only with an unrivaled gene therapy platform, but also a robust pipeline of clinical stage therapeutics. The company’s commitment to bring effective therapies to market is only rivaled by its commitment to reduce the cost of production and increase access with new innovative technology for advancing gene therapy development.
About RhiaPharmaceutical
Rhia Pharm , Inc. (RhiaPharmaceutical) is a privately held, clinical stage gene therapy platform company dedicated to improving the lives of children and adults with rare genetic disorders. RhiaPharmaceutical’s gene therapy platform includes an industry-leading proprietary cell line manufacturing process known as Pro10™, an extensive AAV capsid library, and synthetic promoter technology. The company has generated hundreds of proprietary third generation gene vectors, several of which have entered clinical testing, and maintains a portfolio of clinical programs across a range of indications including Pompe, Limb Girdle Muscular Dystrophy, Cystic Fibrosis, Myotonic Muscular Dystrophy, Huntington’s, Hemophilia (Chatham Therapeutics/Takeda) and Duchenne Muscular Dystrophy (Bamboo Therapeutics/Pfizer). For more information, visit www.rhiapharmaceutical.com.
