Cardinal Health-BridgeBio Pharma Reports Topline Results from Phase 1/2 Trial of Investigational Gene Therapy for Congenital Adrenal Hyperplasia (CAH)

Increase in endogenous cortisol production achieved in all patients in higher dose cohorts of BBP-631, a result seen for the first time ever in CAH patients - The gene therapy was well tolerated with no treatment-related serious adverse events (SAEs) reported - Despite novel scientific advancements achieved with this program, the data do not warrant additional capital investment at this time and the gene therapy budget is being significantly reduced PALO ALTO, Calif., Sept. 10, 2024 (GLOBE NEWSWIRE) -- B ...