Research programme: RNA based therapeutics - ProQR Therapeutics

Drug Profile

Research programme: RNA based therapeutics - ProQR Therapeutics

Alternative Names: Axiomer® EONs; Axiomer® editing oligo nucleotides; QR-313; QR-411; QR-421; QRX 021; QRX 1011; QRX 203; QRX 313; QRX 411; QRX 421; QRX 504; QRX 604; QRX 704; QRX 812; QRX 911; Therapeutics for genetic disorders - ProQR

Latest Information Update: 30 Nov 2017

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At a glance

  • Originator ProQR Therapeutics
  • Class Antidementias; Antifibrotics; Eye disorder therapies; Neuropsychotherapeutics; Oligonucleotides; Skin disorder therapies; Small interfering RNA
  • Mechanism of Action RNA interference
  • Orphan Drug Status

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

    Yes - Usher syndromes; Retinitis pigmentosa; Epidermolysis bullosa; Leber congenital amaurosis
  • New Molecular Entity Yes
  • Available For Licensing Yes

Highest Development Phases

  • Preclinical Epidermolysis bullosa; Mucopolysaccharidosis I
  • Research Alzheimer's disease; Corneal disorders; Cystic fibrosis; Friedreich's ataxia; Huntington's disease; Stargardt disease; Usher syndromes

Most Recent Events

  • 29 Nov 2017 QR-313 receives Orphan Drug status for Epidermolysis bullosa in European Union
  • 30 Sep 2017 Preclinical trials in Mucopolysaccharidosis I in Netherlands (unspecified route)
  • 19 Sep 2017 QR 313 receives Orphan Drug status for Epidermolysis bullosa in USA
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