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Also known as AAV BBP-631
BBP-631 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Dose Level 1 | EXPERIMENTAL | BBP-631 lowest dose, administered once, intravenously (IV) |
| Dose Level 2 | EXPERIMENTAL | BBP-631 middle dose, administered once, IV |
| Dose Level 3 | EXPERIMENTAL | BBP-631, high dose, administered once, IV |
| Dose Level 4 | EXPERIMENTAL | BBP-631, highest dose, administered once, IV |
| Name | Type | Description |
|---|---|---|
| AAV BBP-631 | BIOLOGICAL | intravenous |
Key Inclusion Criteria 1. Adult male and non-pregnant females with classic CAH (simple virilizing or salt-wasting) due to 21-OHD 2. Screening/baseline 17-OHP levels \> 5-10 × ULN and \< 40 × ULN (upper limit of normal) 3. Stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maint...
BBP-631 is an investigational gene therapy being developed for classic congenital adrenal hyperplasia (CAH), an endocrine disorder. It is designed as a one-time treatment approach for adults with this condition. BBP-631 has not been approved by any regulatory agency and remains in clinical development.
BBP-631 is developed by BridgeBio Pharma, Inc., which trades on the Nasdaq under the ticker BBIO. BridgeBio is a biopharmaceutical company focused on genetic diseases and has advanced BBP-631 into clinical testing for congenital adrenal hyperplasia.
BBP-631 is in Phase 1 clinical development. The only trial to date is a Phase 1 study that is currently active but not recruiting. There are no completed trials for BBP-631, and the program remains investigational.
BBP-631 is being evaluated in a single Phase 1 trial, NCT04783181, titled A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH). The trial is active but not recruiting, enrolled 8 participants, and is conducted in the United States. It is controlled and includes adults 18 years and older.
Yes, BBP-631 is also known as AAV BBP-631. The alternative name reflects its gene therapy modality, which uses an adeno-associated virus (AAV) vector. Both names refer to the same investigational product developed by BridgeBio Pharma for congenital adrenal hyperplasia.