Sarepta has expanded its presence in gene therapy by licensing CNS-targeted gene therapy programs from Lacerta. This includes exclusive rights to Lacerta’s candidate for Pompe disease. Lacerta uses proprietary adeno-associated virus (AAV) vector technologies to develop CNS-targeted treatments for lysosomal storage diseases, including Pompe disease. This increases Sarepta’s pipeline to 11 gene therapy programs.
This also gives Sarepta access to incredible talent from places like University of Florida, Nationwide Children’s Hospital, CHOP/University of Pennsylvania and Weill Medical College of Cornell.
Tagged: gene therapy