Research programme: RNA based therapeutics - ProQR Therapeutics

Drug Profile

Research programme: RNA based therapeutics - ProQR Therapeutics

Alternative Names: QR 110; QR-313; QRX 021; 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: 05 Jun 2017

Price : $50

At a glance

  • Originator ProQR Therapeutics
  • Class Antidementias; Eye disorder therapies; Oligonucleotides; 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 - Leber congenital amaurosis
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Epidermolysis bullosa; Leber congenital amaurosis
  • Research Alzheimer's disease; Corneal disorders; Cystic fibrosis; Friedreich's ataxia; Huntington's disease; Usher syndromes

Most Recent Events

  • 31 May 2017 Research programme: RNA based therapeutics - ProQR Therapeutics (QR 110) receives Fast Track designation for Leber congenital amaurosis [Intravitreous,Injection] in Netherlands
  • 27 Apr 2017 US FDA approves IND application for QR 110 in Leber congenital amaurosis
  • 28 Feb 2017 Updated pharmacodynamics data from a preclinical trial in Leber congenital amaurosis released by ProQR Therapeutics
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