These companies have a known or expressed interest in LGMD2I.
- Antibody therapy (PF-06252616): Monoclonal antibody that blocks myostatin, a naturally occurring protein in muscles that helps restrict muscle growth.
Phase 1/2: Clinical trial extension in LGMD2I
Phase 2: Recruitment completed in DMD
- Gene therapy (PF-06939926): AAV9 carrying a mini-dystrophin gene under the control of a human muscle specific promoter.
Phase I: Recruiting in DMD
Start-up company with a preclinical program in LGMD2I. The technology is a combination gene therapy that is expected to deliver a corrective FKRP gene and stimulate an increase of the muscle mass.
Data in animal model for LGMD2I
No clinical programs
Its “mission is to positively transform lives through the development of pioneering gene therapy treatments for LGMDs”, using rh.74 AAV as a vector to deliver the corresponding corrective gene (see individual programs).
- MYO-101 in LGMD2E (SGCB gene)
Phase ½ IV: Starting soon
- MYO-102 in LGMD2D (SGCA gene)
Phase 1/2 ILP: Completed enrollment
- MYO-201 in LGMD2B (DYSF gene)
Phase 1 IM: Completed enrollment
- MYO-103 in LGMD2C (SGCG gene)
- MYO-301 in LGMD2L (ANO5 gene)
Genzyme’s mission is to discover and deliver transformative therapies for patients with rare and special unmet medical needs. Genzyme Rare Diseases R&D is currently focused on select disease areas including inherited metabolic, muscle, pulmonary, CNS, renal and hematological diseases. For many years, Sanofi Genzyme has been an active partner in important and thoughtful projects in muscular dystrophies encompassing diagnosis, registry, and therapy.
- Protein-based therapy: Lumizyme for Pompe disease
Its “mission is to bring innovative gene therapy products to patients living with serious, life-threatening rare diseases” using AAV8 as a vector to deliver the corresponding corrective gene (see individual programs).
- AT132 in X-Linked Myotubular Myopathy (MTM1 gene)
Phase 1/2 (called ASPIRO): Recruiting
- AT982 in Pompe disease (GAA gene)
Phase 1/2 : Starting soon
The goal of Italfarmaco research activity is to provide physicians and patients with more effective and safer drugs aimed at improving health and ensuring a better quality of life to patient populations.
Givinostat in DMD
- Phase 3 : Recruiting in DMD across North America and Europe