Limb Girdle Muscular Dystrophy (LGMD) Global Pipeline (Gene Therapy)

Last Update: June 19, 2023

Purchase the complete database for non-gene therapies (7 listed), discontinued programs (5 listed), and corresponding clinical trial information (NCT identifiers, initiation dates, no. of trial participants, primary outcomes, clinical trial status) and notes.

Gene Therapy

Company/Sponsor Candidate MOA Indication Phase
Sarepta TherapeuticsSRP-9003scAAVrh74 delivering hSGCB gene under MHCK7 promoterLGMD2E / LGMD R4 Beta-sarcoglycan-relatedI/II
Sarepta TherapeuticsSRP-9004scAAVrh74 delivering SGCA gene under tMCK promoterLGMD2D / LGMD R3 Alpha-sarcoglycan-relatedI/II
Atamyo TherapeuticsATA-100/GNT0006AAV delivering FKRP geneLGMD2I / LGMD R9 Dystroglycan-relatedI/II
Sarepta TherapeuticsSRP-6004rAAVrh74 delivering DYSF gene under MHCK7 promoterLGMD2B / LGMD R2 Dysferlin-relatedI
AskBio (Bayer)LION-101rAAV9 delivering FKRP geneLGMD2I / LGMD R9 Dystroglycan-relatedI/II
Atamyo TherapeuticsATA-200/GNT0007AAV1 delivering SGCG geneLGMD2C / LGMD R5 Gamma-sarcoglycan-relatedPreclin
Sarepta TherapeuticsSRP-9005AAVrh74 delivering SGCG gene under MHCK7 promoterLGMD2C / LGMD R5 Gamma-sarcoglycan-relatedPreclin
Sarepta TherapeuticsSRP-9006AAVrh74 delivering ANO5 gene under tMCK promoterLGMD2L / LGMD R12 Anoctamin5-relatedPreclin
Atamyo TherapeuticsATA-300/GNT0008AAV delivering CAPN3 geneLGMD2A / LGMD R1 Calpain3-relatedPreclin
Sarepta TherapeuticsCalpain 3 (CAPN-3)AAVrh74 delivering CAPN3 gene under tMCK promoterLGMD2A / LGMD R1 Calpain3-relatedPreclin
Atamyo Therapeutics-AAV delivering DYSF geneLGMD2B / LGMD R2 Dysferlin-relatedPreclin
Atamyo Therapeutics-AAV delivering SGCA geneLGMD2D / LGMD R3 Alpha-sarcoglycan-relatedPreclin
Cure Rare Disease-Gene replacement therapyLGMD2G / R7 Telethonin-relatedPreclin
Cure Rare Disease-Gene replacement therapyLGMD2I / R9 Dystroglycan-relatedPreclin
Children’s National Hospital -AAV delivering hASM (secreted)LGMD2B / LGMD R2 Dysferlin-relatedPreclin
Children’s National Hospital -rAAV delivering TCAPLGMD2G / LGMD R7 Telethonin-relatedDisc