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

Read the full 656 word article

User Sign In

Article Purchase

This article may not be distributed to non-subscribers

PURCHASE THIS ARTICLE FOR LIMITED ONE-TIME DISTRIBUTION AND WEBSITE POSTING $995.00 USD

PURCHASE