Drugs / BBP-812
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT04998396 | Sep 2021 → Oct 2026 expected | Canavan disease | Aspa Therapeutics | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 8
| Date | Issuer | Release |
|---|---|---|
| 2024-10-24 | Eidos Therapeutics, a BridgeBio company | Results bridgebio shares positive data from high dose cohort of phase 1/2 canaspire study of gene therapy bbp-812 for canavan disease at esgct 2024 bridgebio.com ↗
“We continue to be encouraged by the results children are achieving in the CANaspire trial of BBP-812,” said Dr. Eichler. |
| 2024-09-10 | Eidos Therapeutics, a BridgeBio company | Regulatory bridgebio receives fda’s regenerative medicine advanced therapy (rmat) designation for bbp-812 canavan disease gene therapy program bridgebio.com ↗
BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases, today announced that the United States Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BBP-812, an investigational intravenous (IV) adeno-associated virus serotype 9 (AAV9) gene therapy for the treatment of Canavan disease. |
| 2023-08-03 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma reports second quarter 2023 financial results and business update bridgebio.com ↗
Following treatment, the N-acetylaspartate (NAA) levels of CANaspire participants were consistent with levels seen in individuals with milder Canavan disease based on findings from the Company’s natural history study and reports in the scientific literature. |
| 2023-05-22 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma presents updated encouraging clinical and biomarker data from its bbp-812 canavan disease gene therapy program at the 2023 american society of gene and cell therapy (asgct) annual meeting bridgebio.com ↗ |
| 2022-10-13 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma presents updated positive data from its bbp-812 canavan disease gene therapy program at the 51st annual meeting of the child neurology society bridgebio.com ↗ |
| 2022-06-22 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma announces early positive data for bbp-812, its investigational aav9 gene therapy for canavan disease bridgebio.com ↗ |
| 2021-05-14 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma receives fda fast track designation for investigational gene therapy for congenital adrenal hyperplasia bridgebio.com ↗
The preclinical findings demonstrate safety through IV administration, which will provide superior biodistribution to deep brain regions and avoid potentially invasive brain surgery. |
| 2019-10-22 | Eidos Therapeutics, a BridgeBio company | Results bridgebio pharma gene therapy subsidiaries present data demonstrating potential in two rare disease indications at the european society of gene and cell therapy conference bridgebio.com ↗
Preclinical data shows promise for gene therapy candidates for congenital adrenal hyperplasia due to 21-hydroxylase deficiency (BBP-631) and Canavan disease (BBP-812) |
All press releases naming this drug 10 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | BBP-812 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04998396 ↗ |
| Action | Restore | “to restore ASPA expression” NCT04998396 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “BBP-812, an investigational AAV9-based gene therapy” NCT04998396 ↗ |
| Route | Intravenous | “single intravenous (IV) infusion” NCT04998396 ↗ |
| Target | ASPA | “engineered to deliver the aspartoacylase (ASPA) transgene” NCT04998396 ↗ |