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-411; QR-421; QR-421a; QRX 021; QRX 1011; QRX 203; QRX 411; QRX 421; QRX 504; QRX 604; QRX 704; QRX 812; QRX 911; Therapeutics for genetic disorders - ProQR

Latest Information Update: 15 May 2018

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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; Leber congenital amaurosis; Retinitis pigmentosa
  • New Molecular Entity Yes
  • Available For Licensing Yes

Highest Development Phases

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

Most Recent Events

  • 12 Jun 2018 ProQR plans the phase I/II WINGS trial for QR 313 in Epidermolysis bullosa in the first half of 2018
  • 09 May 2018 Preclinical trials in Usher syndromes in Netherlands (Ophthalmic)
  • 12 Feb 2018 ProQR Therapeutics plans a phase I/II trial for Usher syndrome type 2A in 2018
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