Solid Bioscence: New Hope For Muscular Dystrophy Treatment

(RTTNews) - Shares of Solid Biosciences Inc. (SLDB) are currently up 8% after the company said the U.S. Food and Drug Administration has lifted the clinical hold on SGT-001, the treatment of Duchenne muscular dystrophy.

In its letter, the FDA acknowledged that the company satisfactorily addressed all clinical hold questions. Solid has begun activities to resume the clinical trial and plans to reinitiate enrollment as quickly as possible.

"We believe SGT-001 has the potential to offer significant benefit to patients with DMD, regardless of their age or stage of disease," said Ilan Ganot, Founder and Chief Executive Officer of Solid Biosciences. "We are pleased to have been able to provide the FDA with a comprehensive response resulting in the removal of the clinical hold so we can continue development of this important potential treatment."

The FDA had placed a clinical hold on IGNITE DMD, the company's Phase I/II clinical trial for its investigational microdystrophin gene transfer, following the company's report of a Serious Adverse Event in the first patient dosed with SGT-001. The event was characterized by a decrease in platelet count followed by a reduction in red blood cell count, transient renal impairment and evidence of complement activation.

"Gene therapy has the potential to dramatically change the course of DMD and may offer long-term benefit for those who suffer from this devastating disease," said Barry Byrne, M.D., Ph.D., Director, University of Florida Powell Gene Therapy Center and Professor, Pediatrics and Molecular Genetics & Microbiology at the University of Florida College of Medicine.

SLDB is currently trading at $28.69, up $2.19 or 8.27%, on the Nasdaq.