Clinical Stage LGMD Gene Therapy
Pipeline


Our Pipeline


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

Program Indication Gene Construct Preclinical Phase 1 IM Phase 1/2a ILP Phase 1/2a ILP
MYO-101¹ LGMD2E β-sarcoglycan scAAVrh.74.MHCK7.hSGCB 
MYO-102² LGMD2D α-sarcoglycan scAAVrh.74.tMCK.hSGCA
MYO-103 LGMD2C γ-sarcoglycan scAAVrh.74.MHCK7.hSGCG 
MYO-201³ LGMD2B Dysferlin rAAVrh.74.MHCK7.DYSF.DV
MYO-301 LGMD2L Anoctamin 5 AAVrh.74.tMCK.ANO5
Preclinical Phase 1 IM Phase 1/2a ILP Phase 1/2a ILP
MYO-101¹ for LGMD2E
MYO-102² for LGMD2D
MYO-103 for LGMD2C
MYO-201³ for LGMD2B
MYO-301 for LGMD2L
  1. For MYO-101, FDA granted permission to move directly to a systemic, intravenous (IV) delivery Phase 1/2a study
  2. For MYO-102, FDA required an initial intramuscular (IM) Phase 1 study and an Isolated Limb Perfusion (ILP) Phase 1/2a study to assess safety
  3. For MYO-201, FDA required an initial IM Phase 1 study to assess safety; dual vector design