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; APOC3-GalNAc ASO; Apolipoprotein C-III modulators - WaVe Life Sciences; WVE 3972 01

Latest Information Update: 13 Apr 2018

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At a glance

  • Originator WaVe life Sciences
  • Developer Pfizer; University of Dundee; University of Massachusetts Medical School; WaVe life Sciences
  • Class Anti-inflammatories; Antidementias; Apolipoprotein therapeutics; Eye disorder therapies; Hepatoprotectants; Neuroprotectants; Nucleic acids; Skin disorder therapies
  • Mechanism of Action Apolipoprotein C-III modulators; Dystrophin expression modulators; 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.

  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Amyotrophic lateral sclerosis; Dementia; Duchenne muscular dystrophy
  • Research Epidermolysis bullosa; Eye-Disorders; Inflammatory bowel diseases; Liver disorders; Metabolic disorders; Myotonic dystrophy; Skin disorders; Spinal muscular atrophy; Spinocerebellar degeneration

Most Recent Events

  • 10 Apr 2018 Wave Life Sciences and Deep Genomics agree to co-promote and co-develop therapeutics for Neuromuscular disorders
  • 20 Feb 2018 Wave Life Sciences plans to initiate clinical development for next Duchenne muscular dystrophy programme (targeting exon 53) in Q1 2019
  • 20 Feb 2018 Wave Life Sciences grants Takeda an option to co-develop and co-promote WVE 3972 01 for Amyotrophic lateral sclerosis and frontotemporal Dementia and nucleic acid therapies for Spinocerebellar ataxia type 3
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