Solid reports another DMD gene therapy setback

Solid Biosciences Inc. (NASDAQ:SLDB) reported lower-than-expected microdystrophin protein levels in liver biopsies from the first three patients treated with its microdystrophin gene transfer therapy, SGT-001, in the Phase I/II IGNITE DMD trial to treat Duchenne muscular dystrophy.

In two patients treated with 5x10¹³ vg/kg SGT-001 -- the trial protocol's lowest dose -- three-month biopsy data showed that microdystrophin protein was undetectable via western blot, and detected in fibers via immunofluorescence at very low levels. In the third patient, microdystrophin expression was detected below the 5% level of quantification by western blot and in 10% of fibers via immunofluorescence...