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PERSPECTIVES ON BIOPHARMA TRENDS

Therapeutic Development: AAV1-Follistatin (AAV1-FS344)

11/17/2016

 
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.
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Therapeutic Development: SAR 422459

3/4/2016

 
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.
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  • About
    • Team
    • Development Blog
    • White Papers
    • Contact Us
  • Services
    • Market Access and Forecasting
    • Data Management and Analytics
    • Global Pricing and Reimbursement
    • HEOR
    • Marketing and Messaging Analytics
    • Stakeholder Advisory Boards
  • Products
    • Indications
    • Therapeutics
  • BUSINESS DEVELOPMENT
    • RareValue >
      • RareValue Example
    • Out-License
    • On-Site Workshop