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.
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