Drug Profile
BB 301
Alternative Names: AXO-AAV-OPMD-Axovant Sciences; BB-301; Pabparna™Latest Information Update: 06 Dec 2023
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
- 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
- 08 Aug 2023 AXO AAV OPMD is still in preclinical phase for Oculopharyngeal muscular dystrophy in Australia (IM)