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BB 301

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Drug Profile

BB 301

Alternative Names: AXO-AAV-OPMD-Axovant Sciences; BB-301; Pabparna™

Latest Information Update: 18 Mar 2026

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At a glance

  • Originator Benitec Biopharma
  • Class Anti-inflammatories; Gene therapies; Small interfering RNA
  • Mechanism of Action Gene transference; Poly(A)-binding protein I expression inhibitors; Poly(A)-binding protein I replacements; RNA interference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Oculopharyngeal muscular dystrophy
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Oculopharyngeal muscular dystrophy

Most Recent Events

  • 09 Mar 2026 Efficacy and adverse events data from the phase Ib/IIa trial in Oculopharyngeal muscular dystrophy released by Benitec Biopharma
  • 12 Feb 2026 Benitec Biopharma plans to meet with the US FDA in mid-2026 to confirm the BB 301 pivotal study design
  • 12 Feb 2026 Efficacy data from the phase Ib/IIa trial in Oculopharyngeal muscular dystrophy released by Benitec Biopharma

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