AAV1-FS344 is a gene therapy technology being developed by Milo Biotechnology for the treatment of Duchenne Muscular Dystrophy as well as other muscle diseases including Becker Muscular Dystrophy and Inclusion Body Myositis. Successful AAV1-FS344 delivery leads to the production of follistatin, a TGF-β inhibitor.

Updated: February 2, 2017

SAR 422459 is a drug candidate being developed by Sanofi and Oxford BioMedica for the treatment of Stargardt disease (STGD1). The primary cause of Stargardt disease is the buildup of toxic vitamin A aggregates due to a genetic defect. SAR 422459 aims to correct this by using a gene-based approach to insert a healthy copy of the gene into the retina.