On Tuesday, BridgeBio Pharma, Inc. (NASDAQ:BBIO) released topline results from the Phase 1/2 open-label ADventure study of BBP-631, an investigational adeno-associated virus (AAV) 5 gene therapy, for congenital adrenal hyperplasia (CAH).
CAH is a rare, inherited disorder that affects the adrenal glands and causes a hormone imbalance. The adrenal glands are on top of the kidneys and produce hormones that help the body function.
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To date, key results from the study include:
Increased endogenous cortisol production was achieved in all patients at higher doses.
At the two highest dose ...
