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

Latest Information Update: 14 Aug 2017

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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; Apolipoprotein therapeutics; Eye disorder therapies; Neuroprotectants; Nucleic acids; Skin disorder therapies
  • Mechanism of Action Apolipoprotein C-III 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

  • Research Amyotrophic lateral sclerosis; Dementia; Epidermolysis bullosa; Eye disorders; Inflammatory bowel diseases; Liver disorders; Metabolic disorders; Myotonic dystrophy; Skin disorders; Spinal muscular atrophy

Most Recent Events

  • 10 Aug 2017 Early Research in Dementia in USA (Parenteral)
  • 09 Aug 2017 WaVe life Sciences plans clinical trials for Amyotrophic lateral sclerosis and Frontotemporal dementia in late 2018
  • 08 Jun 2017 ReadCoor and WAVE Life Sciences initiate a research collaboration to charecterise and advance WAVE’s stereopure nucleic acid candidates
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