Research programme: antisense drug therapies - GlaxoSmithKline/Ionis Pharmaceuticals

Drug Profile

Research programme: antisense drug therapies - GlaxoSmithKline/Ionis Pharmaceuticals

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

Latest Information Update: 24 Dec 2015

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At a glance

  • Originator Isis Pharmaceuticals
  • Developer Ionis Pharmaceuticals; Isis Pharmaceuticals
  • Class Antisense oligonucleotides
  • 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

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

Most Recent Events

  • 18 Dec 2015 Isis Pharmaceuticals is now called Ionis Pharmaceuticals
  • 15 Oct 2015 No recent reports of development identified - Preclinical for Alpha 1-antitrypsin deficiency in USA (unspecified route)
  • 15 Nov 2013 Preclinical trials in Eye disorders in USA (unspecified route)
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