BB 301
Alternative Names: AXO-AAV-OPMD-Axovant Sciences; BB-301; Pabparna™Latest Information Update: 02 Apr 2025
At a glance
- Originator Benitec Biopharma
- Class Anti-inflammatories; Gene therapies; Small interfering RNA
- Mechanism of Action Poly(A)-binding protein I expression inhibitors; Poly(A)-binding protein I replacements; RNA interference
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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
- Phase I/II Oculopharyngeal muscular dystrophy
Most Recent Events
- 19 Mar 2025 Efficacy and adverse events data from the phase Ib/IIa trial in Oculopharyngeal muscular dystrophy released by Benitec Biopharma
- 14 Nov 2024 Interim efficacy and adverse events data from the phase Ib/IIa trial in Oculopharyngeal muscular dystrophy released by Benitec Biopharma
- 15 Jul 2024 Updated interim adverse events and efficacy data from a phase Ib/IIa trial in Oculopharyngeal muscular dystrophy released by Benitec Biopharma