Sarepta Therapeutics to Present Results from its Gene Therapy and RNA Platforms at the 2021 Annual MDA Clinical and Scientific Conference

-- Ten abstracts, including four podium presentations, reflect Sarepta’s ongoing commitment to advancing genetic medicine for rare neuromuscular disease and facilitating greater understanding of these devastating conditions --

CAMBRIDGE, Mass., March 15, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the Muscular Dystrophy Association (MDA) Annual Clinical and Scientific Conference, which will take place virtually March 15-18, 2021. Among the research that will be presented:

• New, two- and one-year data including expression data from muscle biopsies taken two years post-treatment, from Study 9003-101 of SRP-9003, an investigational gene therapy for the treatment of limb-girdle muscular dystrophy (LGMD) type 2E/R4;
• Data from Part 1 of Study 9001-102, an ongoing clinical trial of SRP-9001, Sarepta’s investigational gene therapy for Duchenne muscular dystrophy and pre-clinical approaches to the challenge of pre-existing antibodies; and,
• An analysis of time to loss of ambulation in patients taking eteplirsen, a phosphorodiamidate morpholino oligomer (PMO) for the treatment of Duchenne, compared to standard of care.
  

All posters are available on-demand throughout the Congress beginning on Monday, March 15, 2021 at 6:00 a.m. ET. Podium presentations will take place on Thursday, March 18, 2021. The full MDA 2021 Virtual Congress program is available here:  https://mdaconference.org.

Podium Presentations:

Title	Program	Date, Time
Micro-dystrophin Gene Therapy Delivery and Therapeutic Plasma Exchange in Non-Human Primates	SRP-9001	Thurs., March 18, 2021 10:30-10:45 AM ET
A Randomized, Double-Blind, Placebo-Controlled, Gene-Delivery Clinical Trial of rAAVrh74.MHCK7.micro-dystrophin for Duchenne Muscular Dystrophy	SRP-9001	Thurs., March 18, 2021 3:30-3:45 PM ET
Safety, β-sarcoglycan Expression, and Functional Outcomes from Systemic Gene Transfer of rAAVrh74.MHCK7.SGCB in Limb Girdle Muscular Dystrophy Type 2E/R4	SRP-9003	Thurs., March 18, 2021 4:30-4:45 PM ET
Delay in Duchenne Muscular Dystrophy Progression with Eteplirsen: Longer Time to Loss of Ambulation Versus Standard of Care	Eteplirsen	Thurs., March 18, 2021