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: 14 Nov 2016

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
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

  • 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

  • 09 Nov 2016 WaVe Life Sciences plans a phase Ib/IIa trial for Huntington's disease for both WVE 120101 and WVE 120102
  • 15 Aug 2016 Preclinical data released by WaVe Life Sciences
  • 21 Jun 2016 Research programme: stereopure nucleic acid therapeutics - WVE 120101 receives Orphan Drug status for Huntington's disease in USA
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