uniQure Secures FDA Agreement on Accelerated Approval Pathway for Huntington’s Disease Gene Therapy AMT-130

uniQure, FDA, Accelerated Approval, Huntington’s Disease, Gene Therapy, AMT-130, cUHDRS, Neurofilament Light Chain (NfL), Regenerative Medicine Advanced Therapy (RMAT)

Sarepta Therapeutics and Arrowhead Pharmaceuticals Enter $825M Global Licensing and Collaboration Agreement for Multiple siRNA Programs

Sarepta Therapeutics, Arrowhead Pharmaceuticals, siRNA programs, Global Licensing and Collaboration Agreement, Rare genetic diseases, Gene therapy, RNA interference