Research programme: RNA-interference therapeutics - BioMarin

Drug Profile

Research programme: RNA-interference therapeutics - BioMarin

Alternative Names: PRO 052; PRO 055; PRO 105; PRO 135; PRO 289

Latest Information Update: 10 Oct 2015

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At a glance

  • Originator Leiden University
  • Developer BioMarin Nederland
  • Class Antisense oligonucleotides
  • Mechanism of Action Antisense RNA modulators; Dystrophin stimulants
  • Orphan Drug Status

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

    Yes - Duchenne muscular dystrophy
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Duchenne muscular dystrophy; Huntington's disease; Muscular dystrophies
  • No development reported Spinal muscular atrophy
  • Discontinued Neuroblastoma

Most Recent Events

  • 29 Sep 2015 US PTAB confirms the priority of claims related to the use of exon 51 antisense oligonucleotides in the US application no. 14/198 992
  • 28 Apr 2015 Prosensa Therapeutics is now called BioMarin Nederland
  • 25 Feb 2015 Prosensa has been acquired by BioMarin Pharmaceutical
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