Industry Partner News: Sarepta Announces that Jerry Mendell, M.D. Presented Positive Preliminary Results from the First Children Dosed in Gene Therapy Micro-dystrophin Trial to Treat DMD Patients

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Dear Duchenne Community,

Today Sarepta Therapeutics held its first ever Research and Development Day, an opportunity to gain insight into Sarepta’s pipeline, its collaborations, and its progress toward its mission of bringing a better life to those living with Duchenne muscular dystrophy, Limb-girdle muscular dystrophy and other rare diseases.

Multiple speakers shared information regarding their work in Duchenne muscular dystrophy today. From these presentations a notion of Sarepta’s full body of work is emerging. The good news is that we are making progress toward our goal of developing therapeutic options for 100% of the individuals with Duchenne. Speakers included (in order of appearance):

  • Doug Ingram, President & Chief Executive Officer, Sarepta Therapeutics
  • Gilmore O’Neill, M.B., M.M.SC., Chief Medical Officer, Sarepta Therapeutics
  • Kevin M. Flanigan, M.D., Director, Center for Gene Therapy, Nationwide Children’s Hospital
  • Serge Braun, Pharm.D., Ph.D., Chief Scientific Officer, AFM-Téléthon; Director Neuromuscular Strategy, Genethon; President, Genosafe SAS; Member, French National Academy of Pharmacy
  • Jerry R. Mendell, M.D., Curran-Peters Chair of Pediatric Research, Center for Gene Therapy, Nationwide Children’s Hospital
  • Louise Rodino-Klapac, Ph.D., Vice President, Gene Therapy, Sarepta Therapeutics; Chief Scientific Officer, Myonexus Therapeutics
  • Charles A. Gersbach, Ph.D., Associate Professor of Biomedical Engineering, Director, Center for Biomolecular and Tissue Engineering, Duke University
  • Gunnar J. Hanson, Ph.D., Senior Director, Research Chemistry, Sarepta Therapeutics
  • Marco Passini, Ph.D., Senior Director, Biology, Sarepta Therapeutics

Notably, Drs. Jerry Mendell and Louise Rodino-Klapac presented positive preliminary results from the first three children dosed in the Phase 1/2a gene therapy micro-dystrophin trial to treat patients with Duchenne muscular dystrophy. Dr. Mendell shared the following:

  • Biopsies performed at Day 90 showed robust micro-dystrophin expression in muscle measured by all methods and observed in all three patients
  • Significant decrease in levels of serum creatine kinase (CK), an enzyme biomarker strongly associated with muscle damage caused by Duchenne muscular dystrophy
  • No serious adverse events (SAEs) observed

We should remember that these are preliminary results involving three patients; much more work
remains to be done. Nevertheless, this data is encouraging and gives us confidence that we are on the right track. 

Regarding the micro-dystrophin gene therapy program, we want to pause and acknowledge every person who has helped to bring this work forward including Dr. Jerry Mendell, Dr. Louise Rodino-Klapac, Pat Furlong, and Parent Project Muscular Dystrophy, who from the earliest days supported this work. We also want to thank the individuals with Duchenne and their families who participated in our clinical trials. Their courage and dedication benefits all living with Duchenne.

Great science is born from great questions, and we know that this community has enormous insight to share. If you have questions related to today’s news, please reach out to your advocacy partners. We will be offering a conference call with them in the near future to discuss community questions. Advocacy groups are instrumental in helping digest movement and change, and they can be a fantastic conduit for ongoing conversation with the community. If you would like to receive future updates from Sarepta, please register on www.duchenne.com/connect.

Thank you to the community for allowing us to engage in this fight with you. We are humbled and inspired by your spirit daily and will remain dedicated to finding meaningful solutions.

Sincerely,

Douglas S. Ingram
President & Chief Executive Officer
Sarepta Therapeutics, Inc.

To read Sarepta's full Press Release, please click here.