Clinical Stage LGMD Gene Therapy

Our Pipeline

Intended as one-time, systemic administrations of corrective gene delivered using rh.74 AAV

  1. For LGMD2E, FDA granted permission to move directly to a systemic, intravenous (IV) delivery Phase 1/2a study.
  2. For LGMD2D and LGMD2B, FDA required an initial intramuscular (IM) Phase 1 study to assess safety.
  3. For LGMD2D, Completion of an Isolated Limb Perfusion (ILP) Phase 1/2a was required before progressing to systemic, IV delivery Phase 1/2a study.