Sarepta Therapeutics Executes Licensing Settlement for Gene Therapy Program from Nationwide Children’s Medical center to Deal with Limb-Girdle Muscular Dystrophy Variety 2A

Limb-girdle muscular dystrophy kind 2A is the most frequent form of LGMD, accounting for a third of LGMD diagnoses

Sarepta’s unmatched portfolio of investigational gene therapies for LGMD presents the potential to handle six LGMD subtypes, which jointly signify much more than 70% of all identified LGMDs

CAMBRIDGE, Mass., Aug. 04, 2021 (World NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the chief in precision genetic medication for rare diseases, nowadays announced that on completion of a range of preclinical and basic safety scientific tests, it experienced executed an unique license agreement for an investigational gene treatment applicant, calpain 3 (CAPN-3), to treat Limb-girdle muscular dystrophy variety 2A (LGMD2A), developed by the Abigail Wexner Investigate Institute at Nationwide Children’s Hospital (Nationwide Children’s).

LGMDs characterize a group of distinctive genetic neuromuscular health conditions with a usually widespread set of signs, together with progressive, debilitating weak spot and squandering that begins in muscle mass all-around the hips and shoulders right before progressing to muscles in the arms and legs. Lots of LGMD sub-kinds are appreciably existence-restricting and often life-ending health conditions. Also identified as calpainopathy, LGMD2A is induced by mutations in the CAPN-3 gene and is the most common variety of LGMD, accounting for virtually a third of scenarios.

“Treatment ideas for LGMD2A are at this time constrained to actual physical treatment, assistive devices and surgical treatment for issues. We’re excited about the opportunity to transform patient care for this appreciably lifestyle-restricting illness by advancing the CAPN-3 method subsequent comprehensive pre-clinical function by the group at Nationwide Children’s. Preclinical investigation carried out to date has delivered early evidence of strategy for CAPN-3 in LGMD2A and supports even further development,” explained Louise Rodino-Klapac, Sarepta’s govt vice president and chief scientific officer. “We intend to establish off the information we have acquired from our guide investigational gene transfer programs for Duchenne muscular dystrophy and LGMD2E, as the CAPN-3 software also makes use of the AAVrh74 vector to tackle yet another perfectly-characterized genetic disease. Sarepta’s motivation and study investment in LGMD is unparalleled and we keep on to operate towards advancing all of our LGMD plans as immediately as achievable.”

Like SRP-9001, Sarepta’s guide investigational gene transfer treatment for Duchenne muscular dystrophy, and the Company’s five other LGMD programs, the LGMD2A software takes advantage of the AAVrh74 vector, developed to systematically and robustly deliver treatment to skeletal muscle mass, including the diaphragm, building it an great prospect to address muscle mass sickness.

The preclinical do the job on the CAPN-3 method in LGMD2A has been led by Zarife Sahenk, M.D., Ph.D., attending neurologist at Nationwide Children’s, Director of Clinical and Experimental Neuromuscular Pathology at The Investigate Institute at Nationwide Children’s and Professor of Pediatrics, Pathology and Neurology at The Ohio Point out University School of Medication.

About Limb-girdle Muscular Dystrophy
Limb-girdle muscular dystrophies are genetic diseases that trigger progressive, debilitating weak spot and throwing away that starts in muscle tissue all over the hips and shoulders in advance of progressing to muscular tissues in the arms and legs. Sarepta’s 6 LGMD gene treatment systems in development contain LGMD2E, LGMD2D, LGMD2C, LGMD2B, LGMD2L and LGMD2A, which with each other symbolize more than 70 per cent of recognised LGMD scenarios.

About Sarepta Therapeutics
Sarepta is on an urgent mission: engineer precision genetic medication for scarce health conditions that devastate lives and minimize futures shorter. We maintain leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we now have more than 40 applications in a variety of levels of advancement. Our large pipeline is pushed by our multi-system Precision Genetic Drugs Motor in gene treatment, RNA and gene enhancing. For a lot more details, make sure you go to or follow us on Twitter, LinkedIn, Instagram and Facebook.

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Forward-Searching Statements
This push release consists of “ahead-on the lookout statements.” Any statements contained in this press release that are not statements of historical reality may be considered to be forward-wanting statements. Terms this kind of as “thinks,” “anticipates,” “options,” “expects,” “will,” “intends,” “likely,” “feasible” and similar expressions are intended to discover forward-hunting statements. These forward-wanting statements consist of statements pertaining to the possible positive aspects of the licensing agreement the structure of the AAVrh74 vector to systematically and robustly provide cure to skeletal muscle, including the diaphragm, generating it an great candidate to address muscle mass sickness the opportunity of our portfolio of investigational gene therapies for LGMD to handle six LGMD subtypes, which with each other depict much more than 70% of all regarded LGMDs and our prepare to continue on to advance all of our LGMD programs as promptly as probable.

These forward-hunting statements involve challenges and uncertainties, quite a few of which are past our manage. Identified hazard aspects involve, amongst others: the envisioned rewards and chances relevant to the licensing agreement may perhaps not be realized or may possibly just take longer to know than expected because of to troubles and uncertainties inherent in solution exploration and enhancement. In individual, routines underneath the license could not outcome in any practical treatment options suitable for commercialization thanks to a variety of factors, which includes any lack of ability of the get-togethers to perform their commitments and obligations under the settlement accomplishment in preclinical trials does not ensure that later on scientific trials will be effective Sarepta may well not be equipped to execute on its enterprise ideas and ambitions, together with meeting its envisioned or planned regulatory milestones and timelines, scientific development options, and bringing its product or service candidates to current market, owing to a range of causes, many of which may well be outside of Sarepta’s management, including feasible constraints of firm financial and other sources, manufacturing limits that may possibly not be anticipated or settled for in a timely fashion, regulatory, courtroom or company decisions, this kind of as selections by the United States Patent and Trademark Business office with regard to patents that go over Sarepta’s products candidates and the COVID-19 pandemic even if Sarepta’s courses result in new commercialized items, Sarepta could not attain the predicted revenues from the sale of these goods if the precise variety of clients residing with LGMD2A is scaled-down than believed, Sarepta’s earnings and capability to achieve profitability may well be adversely influenced and individuals threats discovered underneath the heading “Risk Factors” in Sarepta’s most recent Yearly Report on Kind 10-K for the calendar year ended December 31, 2020, and most new Quarterly Report on Type 10-Q submitted with the Securities and Trade Fee (SEC) as properly as other SEC filings produced by the Business which you are inspired to review.

Any of the foregoing hazards could materially and adversely have an effect on the Company’s organization, effects of functions and the investing rate of Sarepta’s prevalent stock. For a thorough description of threats and uncertainties Sarepta faces, you are encouraged to assessment the SEC filings produced by Sarepta. We warning traders not to put significant reliance on the forward-searching statements contained in this push launch. Sarepta does not undertake any obligation to publicly update its forward-looking statements based mostly on activities or situations following the date hereof.

Resource: Sarepta Therapeutics, Inc.

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