Adverse Event Concerns With Solid Biosciences' Candidate DMD Gene Therapy

The presence for the first time of cardiopulmonary insufficiency/respiratory symptoms in an adverse event report may be what led the US Food and Drug Administration to put Solid Biosciences Inc.’ candidate DMD gene therapy, SGT-001, on clinical hold for the second time.

The US FDA halted Solid’s Phase I/II IGNITE DMD study of its gene therapy because of a single patient report of a serious adverse event which included complement activation, thrombocytopenia, a decrease in red blood cell count, acute kidney injury and cardio-pulmonary insufficiency