Sarepta acquires rights to sixth LGMD gene therapy

Published Date
Jenny Sharpe
AAV virus

Sarepta Therapeutics announced today that it has signed an agreement with the Research Institute at Nationwide Children’s Hospital (Ohio, USA) to develop a gene therapy for LGMD type 2A. This will be the sixth LGMD gene therapy that the company is developing, with three of them already in clinical trials. This new gene therapy aims to replace the CAPN3 gene, which is faulty in people with LGMD 2A.

For more information read Sarepta’s press release.

If you have any questions about this news story or any other LGMD research, please contact the MDUK Research Line on 020 7803 4813 or email

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