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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; QRX 021; QRX 1011; QRX 203; QRX 411; QRX 604; QRX 704; QRX 812; QRX 911; QRX-036; QRX-042; QRX-052; Therapeutics for genetic disorders - ProQR; Trident®

Latest Information Update: 23 May 2023

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

Highest Development Phases

  • No development reported Alzheimer's disease; Cystic fibrosis; Friedreich's ataxia; Huntington's disease; Mucopolysaccharidosis I; Stargardt disease; Usher syndromes

Most Recent Events

  • 31 Dec 2022 ProQR Therapeutics has patent protection for antisense oligonucleotides that are applicable for nucleotide-specific pseudouridylation in South Africa
  • 28 Jun 2022 No recent reports of development identified for preclinical development in Usher syndromes in Netherlands (Ophthalmic)
  • 28 Oct 2021 No recent reports of development identified for preclinical development in Mucopolysaccharidosis-I in Netherlands
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