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Sarepta Therapeutics to Showcase Data from its Gene Therapy and


GlobeNewswire Inc | Sep 14, 2021 08:30AM EDT

September 14, 2021

CAMBRIDGE, Mass., Sept. 14, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the World Muscle Society 2021 Virtual Congress (WMS 2021), taking place Sept. 20-24, 2021. This years presentations at WMS 2021 highlight scientific leadership and innovation from across Sareptas deep, multi-platform portfolio and reflect the Companys continued commitment advancing life-changing therapies to those with rare genetic diseases.

Presented research will include data from the Companys gene therapy and RNA platforms, in addition to new research into the prevalence of pre-existing antibodies to the AAVrh74 vector, which is used in several of Sareptas gene transfer therapy programs. All posters are available on-demand throughout the Congress beginning on Monday, Sept. 20, 2021 at 7:00 am E.T. The full WMS 2021 program is available at https://www.wms2021.com/page/programme.

Poster Presentations

Poster # Title??EP.096? Micro-dystrophin gene therapy delivery and therapeutic plasma exchange in nonhuman primates?EP.139? Phase 1/2a trial of SRP-9001 in patients with Duchenne muscular dystrophy: 3-year safety and functionaloutcomes (SRP-9001-101)?EP.149? Delay in Duchenne muscular dystrophy progression with eteplirsen: Longer time to loss of ambulationversus standard of careEP.150? Casimersenin patients with Duchenne muscular dystrophy amenable to exon 45 skipping: Interim resultsfrom the Phase 3 ESSENCE trial?EP.151? Evaluation of total binding antibodies against rAAVrh74 in patients with Duchenne muscular dystrophy? ENDEAVOR: A gene delivery study to evaluate the safety of andEP.152? expression from SRP-9001 in Duchennemuscular dystrophy (SRP-9001-103) ? Safety,?-sarcoglycanexpression, and functional outcomes fromEP.185? systemic gene transfer of rAAVrh74.MHCK7.SGCB in limbgirdle muscular dystrophy type 2E/R4? A Phase 2 clinical trial evaluating the safety and efficacy ofEP.254 SRP-9001 for treating patients with Duchennemuscular dystrophy (SRP-9001-102) ? Safety, tolerability, and pharmacokinetics of eteplirsen in patientsLBP.22 6-48 months old with Duchenne muscular dystrophy amenable to exon 51 skipping

Presentations will be archived on the events and presentations page in the Investor Relations section of www.sarepta.com for one year following their presentation at WMS 2021.

About Sarepta TherapeuticsSarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Internet Posting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Investor Contact: Ian Estepan, 617-274-4052iestepan@sarepta.com

Media Contact: Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com







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