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 year’s presentations at WMS 2021 highlight scientific leadership and innovation from across Sarepta’s deep, multi-platform portfolio and reflect the Company’s continued commitment advancing life-changing therapies to those with rare genetic diseases.

Presented research will include data from the Company’s 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 Sarepta’s 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 functional outcomes (SRP-9001-101)​
EP.149​ Delay in Duchenne muscular dystrophy progression with eteplirsen: Longer time to loss of ambulation versus standard of care
EP.150​ Casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: Interim results from the Phase 3 ESSENCE trial ​
EP.151​ Evaluation of total binding antibodies against rAAVrh74 in patients with Duchenne muscular dystrophy ​
EP.152​ ENDEAVOR: A gene delivery study to evaluate the safety of and expression from SRP-9001 in Duchenne muscular dystrophy (SRP-9001-103)​
EP.185 ​ Safety, β-sarcoglycan expression, and functional outcomes from systemic gene transfer of rAAVrh74.MHCK7.SGCB in limb girdle muscular dystrophy type 2E/R4​
EP.254 A Phase 2 clinical trial evaluating the safety and efficacy of SRP-9001 for treating patients with Duchenne muscular dystrophy (SRP-9001-102) ​
LBP.22 Safety, tolerability, and pharmacokinetics of eteplirsen in patients 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 visit www.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 at www.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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