Research programme: RNA based therapeutics - ProQR Therapeutics

Drug Profile

Research programme: RNA based therapeutics - ProQR Therapeutics

Alternative Names: QR-313; 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: 11 Jul 2017

$50 / €47 *
* $ price applies to USA, Canada and Latin America. € price applies to the rest of the world. Final gross prices may vary according to local VAT.

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

Highest Development Phases

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

Most Recent Events

  • 05 Jul 2017 Research programme: RNA based therapeutics - ProQR Therapeutics receives Orphan Drug status for Retinitis pigmentosa in European Union
  • 05 Jul 2017 Research programme: RNA based therapeutics - ProQR Therapeutics receives Orphan Drug status for Retinitis pigmentosa in USA
  • 04 Jul 2017 Research programme: RNA based therapeutics - ProQR Therapeutics is available for licensing as of 04 Jul 2017. http://www.proqr.com/
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