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BioMarin Announces 6 Presentations at American Society of Gene and Cell Therapy (ASGCT) Virtual 2021 Annual Meeting
Research Findings are Part of the Largest and Longest Development Program for any Gene Therapy in Hemophilia A and Advance the Scientific Understanding of
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"Research Findings are Part of the Largest and Longest Development Program for any Gene Therapy in Hemophilia A and Advance the Scientific Understanding of AAV5 Gene Therapy\n\n\nSAN RAFAEL, Calif., May 12, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced three oral and three poster presentations on valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A at the American Society of Gene and Cell Therapy (ASGCT) Virtual 2021 Annual Meeting being held May 11-14. These six presentations advance the scientific understanding of the potential of valoctocogene roxaparvovec to treat people with hemophilia A. \n\n \n \n \n \n \n \n\n \n\"BioMarin is committed to furthering our scientific understanding of gene therapy through our experience developing valoctocogene roxaparvovec. This foundation of knowledge will help to inform the development of other gene therapies in our pipeline. The studies presented at ASGCT illustrate our continued quest to define who responds to treatment, why, and for how long. Vector catabolism remains the leading hypothesis as to why Factor VIII expression evolves over time, highlighting pathways to improve patient outcomes,\" said Lon Cardon, Ph.D., Senior Vice President, Chief Scientific Strategy Officer at BioMarin. \"Our presentations at ASGCT extend our nearly decade-long experience in AAV scientific and clinical research to improve our understanding of the critical features of vectors, promoters, pharmacologic agents, and manufacturing technologies to help us deliver potentially transformative treatments to people with rare genetic disease.\"\nThe oral presentation, Investigating Mechanisms of Variability of AAV5-hFVIII-SQ Expression in Mice, provide new insights into inter-individual differences in Factor VIII expression of regulatory molecules involved in transduction, transcription and protein folding/secretion, as well as advance the understanding of factors that may impact variability in transgene expression. BioMarin is conducting multiple ongoing in vitro and in vivo studies to further investigate the mechanistic drivers of AAV5 gene therapy variability. \nThe oral presentation, Long-Term Expression Comparison of Adeno-Associated Virus (AAV) Vector Produced in HEK293 vs Sf Cell Lines, compares the durability of tr...