Research programme: oculopharyngeal muscular dystrophy therapeutics - Benitec/Institut de Myologie/Royal Holloway University of London

Drug Profile

Research programme: oculopharyngeal muscular dystrophy therapeutics - Benitec/Institut de Myologie/Royal Holloway University of London

Alternative Names: BB-301; Pabparna™

Latest Information Update: 29 Nov 2017

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At a glance

  • Originator Benitec Ltd; University of London
  • Developer Benitec Biopharma
  • Class Small interfering RNA
  • Mechanism of Action Gene silencing; 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

  • Preclinical Oculopharyngeal muscular dystrophy

Most Recent Events

  • 27 Nov 2017 Benitec Biopharma applies for Orphan Drug status in Oculopharyngeal muscular dystrophy in USA
  • 17 Jan 2017 BB 301 receives Orphan Drug status for Oculopharyngeal muscular dystrophy in European Union
  • 17 Jan 2017 Benitec Biopharma plans a phase I/II trial for in Oculopharyngeal muscular dystrophy in second half of 2018
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