BFB 101
Alternative Names: BFB-101Latest Information Update: 16 May 2025
At a glance
- Originator BlackfinBio
- Class Gene therapies
- Mechanism of Action Gene transference
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Preclinical Paraplegia
Most Recent Events
- 28 Apr 2025 BlackfinBio plans a phase I/II trial for Hereditary Spastic Paraplegia (In infants, In children) (Intrathecal, Injection) in USA in August 2025 (NCT06948019)
- 28 Apr 2025 BFB 101 receives Orphan Drug status for Paraplegia (In children) in USA prior to April 2025
- 28 Apr 2025 The US FDA grants the rare pediatric disease designation (RPDD) for BFB-101 prior to April 2025