On August 21, 2018, Audentes Therapeutics, Inc. (Nasdaq: BOLD ) announced that the U.S. FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to AT132, its treatment for X-linked Myotubular Myopathy (XLMTM).
Audentes is a biotechnology company that develops gene therapy products for patients for rare diseases.
Audentes has pursued a number of advanced regulatory pathways for AT132, including:
- Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, Fast Track and Orphan Drug designations by the FDA
- Priority Medicines (PRIME) and Orphan Drug designations by the European Medicines Agency (EMA)