Solid Bio's comeback hits a snag

In February, Solid Biosciences reported disappointing results for a gene therapy administered to patients with Duchenne muscular dystrophy. Solid Bio decided to push forward, hoping a higher dose would prove more effective. But on Monday night, the biotech disclosed another possible setback: serious side effects in the first patient treated at the higher gene therapy dose.

Shortly after dosing with Solid Bio’s gene therapy, the patient was diagnosed with a serious gastrointestinal infection deemed unrelated to the gene therapy. More potentially worrisome, however, were reports of a “non-serious” drop in the patient’s platelet count and “serious” increases in certain liver enzymes suggestive of liver injury — both deemed related to the gene therapy, Solid Bio said.

All of the side effects resolved with treatment and the patient has resumed normal activities, the company said, adding that additional patients are being enrolled and treated in its Duchenne muscular dystrophy study.

A rival gene therapy under development by Sarepta Therapeutics has, so far, shown more positive results without any concerning safety issues.