Research programme: stereopure nucleic acid therapeutics - WaVe Life Sciences

Drug Profile

Research programme: stereopure nucleic acid therapeutics - WaVe Life Sciences

Alternative Names: Apolipoprotein C-III modulators - WaVe Life Sciences; Exon 51 modulators - WaVe Life Sciences; WVE 120101; WVE 120102

Latest Information Update: 27 Jan 2017

Price : $50

At a glance

  • Originator WaVe life Sciences
  • Developer Pfizer; WaVe life Sciences
  • Class Antisense elements; Apolipoprotein therapeutics; Nucleic acids
  • Mechanism of Action Apolipoprotein C-III modulators; Dystrophin expression stimulants; Gene expression modulators; RNA interference; Smad7 protein modulators; Survival of motor neuron 2 protein expression modulators
  • Orphan Drug Status

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

    Yes - Huntington's disease
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Duchenne muscular dystrophy
  • Research Amyotrophic lateral sclerosis; Epidermolysis bullosa; Eye disorders; Huntington's disease; Inflammatory bowel diseases; Liver disorders; Metabolic disorders; Myotonic dystrophy; Skin disorders; Spinal muscular atrophy

Most Recent Events

  • 06 Jan 2017 WAVE intends to refile its IND application for WVE 120101 and to file an IND application for WVE 120102 for Huntington’s disease in the first half of 2017
  • 06 Jan 2017 WAVE Life Sciences announces intention to submit Clinical Trial Application (CTA) in Europe in the first half of 2017
  • 09 Nov 2016 WaVe Life Sciences plans a phase Ib/IIa trial for Huntington's disease for both WVE 120101 and WVE 120102
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