On September 28, 2020, Sarepta Therapeutics, Inc. (NASDAQ:SRPT) (http://www.sarepta.com), a leader in precision genetic medicine for rare diseases, announced positive results from the ongoing study of SRP-9003 (rAAVrh74.MHCK7.hSGCB), the Company’s investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). Results included 18-month functional data from three clinical trial participants in the low-dose cohort and 6-month functional data from three participants in the high-dose cohort. SRP-9003 is in development for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4), a devastating monogenic neuromuscular disease caused by a lack of beta-sarcoglycan proteins. SRP-9003 is a gene construct that transduces skeletal and cardiac muscle, delivering a gene that codes for the full-length beta-sarcoglycan protein (https://en.wikipedia.org/wiki/Sarcoglycan), the absence of which is the sole cause of the progressive degeneration and a shortened lifespan characterized by the disease. “There are currently no approved treatments for people with LGMD2E--a disease that causes significant disability in children and often leads to early mortality. It’s very encouraging that we continue to see consistent, positive data from our investigational gene therapy SRP-9003 across several measures, as we know the community needs more options,” said Louise Rodino-Klapac, PhD, Senior Vice President of Gene Therapy, Sarepta Therapeutics. “The improvements in functional measures at 18-months and 6- months in participants from both cohorts who received SRP-9003 are distinctly different from what an age-matched, natural history group would predict with LGMD2E.
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