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BFB-101 Gene Therapy for SPG47
NCT06948019Phase 2Not yet recruiting
- Sponsor
- BlackfinBio
- Start
- Aug 2025
- Primary completion
- Aug 2030
- Modality
- Gene Editing, Gene Silencing, AAV Vectors
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of BFB-101 in Hereditary Spastic Paraplegia Type 47 (HSP47), sponsored by BlackfinBio. Primary endpoint: Incidence of unanticipated treatment-related toxicities, Grade 3 or higher at 60 months. Planned start Aug 2025; status: not yet recruiting.
02
Drugs
BFB-101
03
Conditions
Hereditary Spastic Paraplegia Type 47 (HSP47)
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