Research programme: stereopure nucleic acid therapeutics - WaVe Life Sciences

Drug Profile

Research programme: stereopure nucleic acid therapeutics - WaVe Life Sciences

Alternative Names: ApoC-III modulators - WAVE Life Sciences; Apolipoprotein C-III modulators - WaVe Life Sciences; Exon 51 modulators - WaVe Life Sciences; WVE 120101; WVE 120102; WVE-210201

Latest Information Update: 12 May 2017

Price : $50

At a glance

  • Originator WaVe life Sciences
  • Developer Pfizer; University of Dundee; University of Massachusetts Medical School; University of Oxford; 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; Huntington's disease
  • Research Amyotrophic lateral sclerosis; Epidermolysis bullosa; Eye disorders; Inflammatory bowel diseases; Liver disorders; Metabolic disorders; Myotonic dystrophy; Skin disorders; Spinal muscular atrophy

Most Recent Events

  • 16 Mar 2017 Preclinical trials in Huntington's disease in Japan (Parenteral) before March 2017
  • 16 Mar 2017 Preclinical trials in Huntington's disease in USA (Parenteral) before March 2017
  • 16 Mar 2017 WAVE Life Sciences plans a clinical trial for Duchenne muscular dystrophy (Second-line therapy or greater) (Parenteral)
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