Solid's Duchenne trial on hold
The FDA has put a hard stop on a Solid Biosciences clinical trial after a patient suffered serious kidney and blood-related injuries. The experimental gene therapy, called SGT-001, is being tested in patients with Duchenne muscular dystrophy. This is the third time Solid has had to halt this particular gene therapy, STAT’s Adam Feuerstein writes.
Six patients in total have been dosed with the inactivated virus, which contains a version of the dystrophin gene that’s lacking in patients. Two other companies — Pfizer and Sarepta — are also working on Duchenne gene therapies. Pfizer, too, has experienced similar kidney toxicity with its Duchenne drug, as well as immune system over-activation, but the trial is still active.
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