BridgeBio Pharma drops gene therapy program for AAV

11 September 2024

California, USA-based biotech BridgeBio Pharma (Nasdaq: BBIO) saw its shares dip 4% to $29.60 yesterday on disappointing clinical results and the company’s decision to drop an early-stage candidate.

BridgeBio announced top-line results from the Phase I/II open-label ADventure study investigating BBP-631, its investigational adeno-associated virus (AAV) 5 gene therapy, for the treatment of congenital adrenal hyperplasia (CAH).

Most patients achieved ≥50% reduction in 17-hydroxyprogesterone (substrate of 21-hydroxylase), with a maximum reduction of 95%, and no treatment-related SAEs were reported. Despite trends in the data, the program did not meet the threshold for additional capital investment, said BridgeBio.

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