Expert Interview
Discussing Sarepta’s Gene Therapy Pipeline in Treating Limb-Girdle Muscular Dystrophy
Ticker(s): SRPTA physiatrist or neurologist with experience in Limb-Girdle Muscular Dystrophy, and knowledge about the upcoming pipeline of Sarepta and its most recently presented data of SRP-9003 for LGMD Type 2E
Please tell us about your clinical experience. How many patients with Limb-Girdle Muscular Dystrophy do you see, and what is the choice of therapy for each type (LGMD2E, LGMD2D, LGMD2C, LGMD2B, LGMD2L and LGMD2A)?
Can you tell us more about the mechanism of action of SRP-9003 gene therapy, that delivers a gene that codes for the full-length beta-SG protein? Is the absence of this protein the cause of the disease?
Can you please discuss with us the main points of the data presented recently, where protein expression in muscle was sustained for 2 years following treatment in the low dose cohort, with mean beta-sarcoglycan expression of 54% at 24 months, compared to 36% at Day 60? How does it compare to what you’re seeing in clinic?
Added By: slingshot_insightsJust now, gene therapy GNT 0004(microdystrophin) in DMD, sponsored by Genethon, was suspended due to a serious adverse event that occurred in a patient. How likely is Sarepta’s 9001 Micro-dystrophin to show similar adverse events?
Can you talk about the differences in vector, muscle promoter and transgene, between the two?
Added By: slingshot_insightsHow likely is this event to affect Sarepta’s other gene therapy pipeline indications?
How would you rate the potential of the LGMD2A program that uses the AAVrh74 vector, and how do you assess the preclinical work on the CAPN-3 program in LGMD2A?
How likely would you be to use Sarepta's gene therapy for treating LGMD?
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