Research programme: gene therapies for CNS disorders - Voyager Therapeutics

Drug Profile

Research programme: gene therapies for CNS disorders - Voyager Therapeutics

Alternative Names: AAV-delivered gene therapies for CNS disorders - Voyager Therapeutics; Amyotrophic lateral sclerosis gene therapy - Voyager Therapeutics; Friedreich’s ataxia gene therapy - Voyager Therapeutics; Huntington's disease gene therapy - Voyager Therapeutics; VY FXN01; VY HTT01; VY SMN101; VY SOD101; VY-NAV01; VY-TAU01

Latest Information Update: 16 Dec 2016

Price : $50

At a glance

  • Originator Voyager Therapeutics
  • Class Analgesics; Gene therapies; Monoclonal antibodies
  • Mechanism of Action Gene transference; Nav1.7-voltage-gated-sodium-channel-inhibitors; Tau protein inhibitors
  • Orphan Drug Status

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

  • 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 No

Highest Development Phases

  • Preclinical Alzheimer's disease; Amyotrophic lateral sclerosis; Dementia; Friedreich's ataxia; Huntington's disease; Pain; Spinal muscular atrophy

Most Recent Events

  • 20 Oct 2016 Preclinical trials in Alzheimer's disease, Dementia and Pain in USA (Voyager Therapeutics' pipeline, October 2016)
  • 15 Sep 2016 Voyager Therapeutics licenses Gene therapy capsids from the California Institute of Technology
  • 12 Sep 2016 Voyager Therapeutics and CHDI enter research collaboration to advance Huntington's disease gene therapy
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