Derailed gene therapy study reports 3rd death as safety and durability issues cloud a booming field
A third patient has died in Audentes’ pivotal gene therapy trial for X-linked myotubular myopathy, adding fresh concerns for the field right on the heels of BioMarin’s surprise slapdown at the FDA.
The biotech, bought out by Astellas just 8 months ago for $3 billion, says the third patient died from gastrointestinal bleeding. And like the two others who died, this person was on the high dose.
According to Audentes:
Keep reading Endpoints with a free subscription
Unlock this story instantly and join 117,600+ biopharma pros reading Endpoints daily — and it's free.