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Research programme: antisense drug therapies - GlaxoSmithKline/Ionis Pharmaceuticals/ProQR Therapeutics

Drug Profile

Research programme: antisense drug therapies - GlaxoSmithKline/Ionis Pharmaceuticals/ProQR Therapeutics

Alternative Names: ISIS-AATRx; ISIS-GSK5Rx; ISIS-RHO-2.5Rx; QR 1123

Latest Information Update: 28 Jan 2019

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At a glance

  • Originator Isis Pharmaceuticals
  • Developer Ionis Pharmaceuticals; Isis Pharmaceuticals
  • Class Antisense oligonucleotides; Eye disorder therapies
  • Mechanism of Action Alpha 1 adrenergic receptor modulators; Alpha 1 antitrypsin modulators; RNA inhibitors; RNA interference
  • Orphan Drug Status

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

  • New Molecular Entity Yes

Highest Development Phases

  • No development reported Alpha 1-antitrypsin deficiency; Blindness; Eye disorders; Infections; Retinitis pigmentosa

Most Recent Events

  • 28 Jan 2019 No recent reports of development identified for preclinical development in Retinitis-pigmentosa in USA
  • 29 Oct 2018 ProQR Therapeutics in-licenses QR 1123 from Ionis Pharmaceuticals
  • 29 Oct 2018 ProQR Therapeutics plans to files an IND application with the US FDA for Retinitis pigmentosa
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