
FDA slams clinical hold on LogicBio's gene editing drug after clotting cases in child patients
Another day, another AAV-delivered gene therapy running into roadblocks with the FDA.
The agency has placed a clinical hold on LogicBio’s AAV-delivered gene editing therapy LB-001 after two adolescent patients in an early trial for the drug were diagnosed with thrombotic microangiopathy (TMA), a clinical syndrome defined by the formation of tiny clots in blood vessels, the company said Wednesday.
The biotech is trying LB-001 in a Phase I/II trial dubbed SUNRISE to treat children with the rare metabolic disease methylmalonic acidemia, which results in patients not being able to break down certain fats and proteins.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.