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BridgeBio Pharma Announces Dosing of First Patient in Phase 1/2 Trial of Investigational Gene Therapy for Canavan Disease
-If successful, BridgeBio's gene therapy could be the first approved therapeutic option for children born with Canavan disease, a devastating and
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"-If successful, BridgeBio's gene therapy could be the first approved therapeutic option for children born with Canavan disease, a devastating and life-threatening condition\n -The Canavan disease program is part of BridgeBio's growing gene therapy portfolio, which includes clinical candidates forcongenital adrenal hyperplasia (CAH) and classic galactosemia (severe GALT deficiency), and preclinical programs forTMC1 hearing loss, tuberous sclerosis, cystinuria, and a genetic dilated cardiomyopathy\n\n\nPALO ALTO, Calif., Nov. 3, 2021 /PRNewswire/ -- BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the first patient has been dosed in CANaspire, its Phase 1/2 clinical trial of BBP-812, an investigational adeno-associated virus (AAV) 9 gene therapy for the treatment of Canavan disease. Canavan disease is an ultra-rare and fatal disease that affects approximately 1,000 children in the United States and European Union. There are currently no approved therapies for the condition.\n\n \n \n \n \n \n \n\n \n\"Dosing the first patient in our Canavan disease trial is a significant achievement for our gene therapy team and we would not be here without the support of our collaborative partners in the patient, medical and scientific communities,\" said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy. \"We are committed to the Canavan community and will work to advance this trial and the other programs in our gene therapy portfolio because we believe deeply in the potential of gene therapy to improve and save lives.\" \n\n \"Based on the efficacy and safety data we've observed in our preclinical studies, we are hopeful that our investigational gene therapy can become a meaningful treatment option for children living with Canavan. Right now, these children only have access to supportive care because there are currently no approved therapies to treat this devastating disease,\" added Adam Shaywitz, M.D., Ph.D., chief medical officer at BridgeBio Gene Therapy.\n\n The Phase 1/2 open-label study is designed to evaluate the safety, tolerability, and pharmacodynamic activity of the company's AAV9 gene therapy, BBP-812, in pediatric patients with Canavan disease. In the initial dose-finding phase of the study, each patient will receive a single intra...