BridgeBio Pharma Inc. (BBIO) said that it will no longer be pursuing development of BBP-631 for Congenital Adrenal Hyperplasia. The company is actively seeking partnership opportunities to support future development of BBP-631 or next-generation gene therapies for the treatment of Congenital Adrenal Hyperplasia, a very prevalent genetic disease that still has significant unmet need, with more than 75,000 cases estimated in the United States and European Union.
The company announced results from the Phase 1/2 open-label ADventure study investigating BBP-631, the Company's investigational adeno-associated virus (AAV) 5 gene therapy, for the treatment of congenital adrenal hyperplasia or CAH.
The company noted that the trial did not meet the threshold to warrant additional capital investment at this time, it will be reducing the gene therapy budget more than $50 million.
The key results from the study included increased endogenous cortisol production was achieved in all patients at higher doses. BBP-631 has been well tolerated with only mild to moderate treatment-emergent adverse events (TEAEs) and no treatment-related serious adverse events reported.
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May 01, 2026 15:54 ET Central banks dominated the economics news flow this week with almost all major ones announcing their latest policy decisions and many boosted expectations for a rate hike in June. In other news, several countries released the preliminary data for first quarter economic growth. In the U.S., comments by Fed Chair Jerome Powell were also in focus as his term ends this month.