Drug Profile
BB 301
Alternative Names: AXO-AAV-OPMD-Axovant Sciences; BB-301; Pabparna™Latest Information Update: 22 Apr 2024
Price :
$50
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At a glance
- Originator Benitec Biopharma
- Class Gene therapies; Small interfering RNA
- Mechanism of Action Poly(A) binding protein I replacements
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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
- 18 Apr 2024 Interim adverse events and efficacy data from a phase Ib/IIa trial in Oculopharyngeal muscular dystrophy released by Benitec Biopharma
- 30 Nov 2023 Phase-I/II clinical trials in Oculopharyngeal muscular dystrophy in USA (IM)
- 21 Sep 2023 Benitec Biopharma plans a phase Ib/IIa trial for Oculopharyngeal muscular dystrophy in USA and Canada (IM) in 2023