Sarepta Therapeutics Announces Preliminary Results in Clinical Trial Investigating Gene Therapy for LGMD2E

Today Sarepta Therapeutics announced preliminary data on the first three LGMD2E patients dosed in the MYO-101 Gene Therapy Trial. Muscle biopsies show that about 50% of muscle fibers show expression of beta-sarcoglycan, the protein which is missing or faulty in LGMD2E patients. Expression levels of the protein were about 30-40% of normal, a level that is predicted, based on mouse studies, to lead to functional improvement. Serum creatine kinase (CK) levels decreased by 90% in the patients. It is too early to determine if there are functional improvements, but early reports from family members indicate patients are doing activities they have not been able to before.

 

All patients received prednisone for 30 days to inhibit an immune reaction to the therapy. Two patients showed elevated liver enzymes upon when being tapered off the prednisone. They were put back on the prednisone temporarily until liver enzyme levels resolved, and now are no longer on prednisone with no lasting effects.

 

Sarepta also announced today that they will acquire Myonexus and continue development of gene therapies for LGMDs. Today’s announcements are great news for LGMD2A, as they will draw attention from the biotechnology industry and investors to rare muscular dystrophies. Furthermore, a highly similar treatment may be beneficial for LGMD2A patients.

 

C3 recognizes the potential for gene therapy to treat or cure LGMD2A. Click here to support gene therapy research for LGMD2A!