Gene therapy has proved efficiency to address the root cause. It has been used in other LGMDs proving its efficiency.
It addresses the root cause, by replacing the faulty gene with a new efficient one allowing the production of ANO5 protein.
One single injection is enough to replace the faulty gene in most muscle cells.
Strong proof-of-concept already exists
- Clinical success on Spinal Muscular Atrophy, Hemophilia B, some retinal diseases.
- Clinical trials on LGMD have demonstrated biological correction: SGCB, SGCG, SGCA, FKRP.
- The same platform can be adapted to many LGMD subtypes
Skeletal muscle is highly accessible to gene delivery
Modern AAV (adeno-associated virus) vectors have demonstrated the ability to reach 94% of the muscles.
One single treatment can potentially provide years of benefit, unlike drugs that require daily administration.