Shots:

• The 1st patient has been dosed in P-I/II (ADventure) trial evaluating the safety, tolerability & PD of BBP-631 in patients with CAH
• The 1EPs of the study are safety, change from baseline in endogenous cortisol levels, steroid biomarkers for hydroxyprogesterone (17-OHP) levels & androstenedione (A4) levels. Preclinical PoC data showed the potential to provide efficient & persistent delivery of functional 21-hydroxylase (21-OH) enzyme to the adrenal gland
• BridgeBio’s portfolio also includes a clinical-stage program for Canavan disease & preclinical programs for classic galactosemia, TMC1 hearing loss & 3 others. The results are expected in H2’22 for CAH & Canavan disease

Ref: Globe Newswire | Image: Bridgebio