BridgeBio Pharma Seeks Partner To Develop Gene Therapy For Inherited Condition

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 ...