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Also known as AAV9 BBP-812
BBP-812 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1) | EXPERIMENTAL | Participants will receive a single intravenous (IV) infusion of low-dose BBP-812 on Day 0 in the dose-finding phase of the study. |
| Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2) | EXPERIMENTAL | Participants will receive a single IV infusion of high-dose BBP-812 on Day 0 in the dose-finding phase of the study. |
| Enrollment Expansion Phase: BBP-812 | EXPERIMENTAL | Participants will receive a single IV infusion of BBP-812 at the selected dose from the dose-finding phase on Day 0 in expansion phase of the study. |
| Name | Type | Description |
|---|---|---|
| AAV9 BBP-812 | BIOLOGICAL | Sterile solution for injection for 1-time use via volumetric infusion pump |
Key Inclusion Criteria: * Maximum age for inclusion is 30 months. * Participant has stable health in the opinion of the investigator and as confirmed by medical history and laboratory studies with no acute or chronic hematologic, renal, liver, immunologic, or neurologic disease (other than Canavan ...
BBP-812 is an investigational gene therapy being developed by BridgeBio Pharma, Inc. for Canavan Disease, a rare inherited neurological disorder. It is designed as an AAV9-based therapy and is currently in Phase 1 clinical development. BBP-812 has received several FDA designations, including RMAT, Orphan Drug, Rare Pediatric Disease, and Fast Track.
BBP-812 is being developed as a potential treatment for Canavan Disease, a rare pediatric neurodegenerative condition. It is an investigational gene therapy and has not been approved for any indication. The Phase 1 CANaspire trial is evaluating its use in participants with Canavan Disease.
BBP-812 is being developed by BridgeBio Pharma, Inc., which trades on the Nasdaq under the ticker BBIO. The company is conducting clinical development of this gene therapy for Canavan Disease.
BBP-812 is in Phase 1 clinical development. It is an investigational therapy and has not been approved by the FDA. The ongoing Phase 1 trial is currently recruiting participants.
BBP-812 is being evaluated in a single Phase 1 clinical trial, NCT04998396, titled "A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)." The trial is currently recruiting and has an estimated enrollment of 26 participants. It is conducted in the United States.
Yes, BBP-812 is also known as AAV9 BBP-812. The alternative name reflects the AAV9 viral vector used in this gene therapy. Both names refer to the same investigational product being developed by BridgeBio Pharma for Canavan Disease.