PALO ALTO, Calif. — BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, announced that updated data from CANaspire, its Phase 1/2 clinical trial of BBP-812, an investigational intravenous (IV) adeno-associated virus serotype 9 (AAV9) gene therapy for the treatment of Canavan disease, will be presented at the 2023 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, taking place in Los Angeles, CA on May 16 – 20, 2023.
Additionally, members of BridgeBio leadership, including Neil Kumar, CEO and founder, will be presenting on multiple panels discussing drug development and accelerated approval for gene therapy.
Oral presentation details:
Initial biomarker and clinical findings from the CANaspire Canavan disease gene therapy trial: Exploration of connections between NAA and disease severity
Presenter: Florian Eichler, M.D., director of the leukodystrophy service at Massachusetts General Hospital, Center for Rare Neurological Disease and lead CANaspire investigator
Session date/ time: Saturday, May 20 at 10:15 am – 12:00 pm PT
Presentation time: 11:15 am – 11:30 am PT
Location: Concourse Hall 150 & 151, Los Angeles Convention Center
Session title: Gene and cell therapy trials in progress
Abstract number: 358
Additional panel details:
Accelerated approval for cell and gene therapies
Date & time: Wednesday, May 17 at 9:00 am – 9:45 am PT
Panelist: Adora Ndu, Pharm.D., J.D., chief regulatory and interim legal officer of BridgeBio
Location: Room 515 AB, Los Angeles Convention Center
Venture capital in the gene and cell therapy space
Date & time: Friday, May 19 at 8:00 am – 9:45 am PT
Co-chair: Eric David, M.D., J.D., CEO of BridgeBio Gene Therapy
Panelist: Neil Kumar, Ph.D., founder and CEO of BridgeBio
Location: Concourse Hall 152 & 153, Los Angeles Convention Center
Evolution of genetic medicines: Navigating the challenges and synergies among therapeutic modalities
Date & time: Friday, May 19 at 10:15 am – 12:30 pm PT
Panelist: Eric David, M.D., J.D., CEO of BridgeBio Gene Therapy
Location: Room 411, Los Angeles Convention Center
Accelerate your drug development program in rare disease with robust natural history data: An evolving space
Session title: How to design a natural history study that regulators and clinicians support
Date & time: Friday, May 19 at 10:15 am – 12:00 pm PT
Panelist: Adam Shaywitz, M.D., Ph.D., chief medical officer at BridgeBio Gene Therapy
Location: Room 408 AB, Los Angeles Convention Center
About BridgeBio Pharma, Inc.
BridgeBio Pharma Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers, and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.