Drugs / BFB-101
BFB-101
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT06948019 | Aug 2025 → Aug 2030 expected | hereditary spastic paraplegia 47, hereditary spastic paraplegia 50, hereditary spastic paraplegia 51, hereditary spastic paraplegia 52 +2 | BlackfinBio Ltd | Not yet recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 2
| Date | Issuer | Release |
|---|---|---|
| 2025-04-28 | BlackfinBio Ltd | Regulatory BlackfinBio Announces FDA Clearance to Initiate Phase 1/2 Clinical Trial for Novel AAV Gene Therapy BFB-101 to Treat the Rare Neurological Disease Hereditary Spastic Paraplegia, Type 47 (SPG47) blackfin.bio ↗
BlackfinBio Ltd, a clinical stage gene therapy company focused on the development of treatments for rare neurological diseases, announces today that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for a Phase 1/2 clinical trial of its novel adeno-associated virus (AAV) gene therapy, BFB-101, in children with Hereditary Spastic Paraplegia, Type 47 (SPG47). |
| 2025-03-18 | BlackfinBio Ltd | Regulatory BlackfinBio Ltd Announces Submission of IND Application to the U.S. FDA for Novel AAV Gene Therapy to Treat Hereditary Spastic Paraplegia, Type 47 (SPG47) blackfin.bio ↗
The IND submission marks a significant milestone for BlackfinBio and the SPG47 community. |
All press releases naming this drug 2 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | BFB-101 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06948019 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “an adeno-associated virus serotype 9 (AAV9) vector encoding a codon-optimized full-length human AP4B1 cDNA” NCT06948019 ↗ |
| Route | Other | “delivered as a single dose by intra-cisterna magna injection” NCT06948019 ↗ |
| Target | AP4B1 | “an expression cassette of AP4B1 driven by CBh promoter.” NCT06948019 ↗ |