Research programme: gene therapies for CNS disorders - Voyager Therapeutics

Drug Profile

Research programme: gene therapies for CNS disorders - Voyager Therapeutics

Alternative Names: AAV Frataxin; AAV-delivered gene therapies for CNS disorders - Voyager Therapeutics; AAVrh.10-vectored PHF1; ALS SOD1; Amyotrophic lateral sclerosis gene therapy - Voyager Therapeutics; Anti-Tau Antibody; 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: 25 May 2018

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At a glance

  • Originator Voyager Therapeutics
  • Class Analgesics; Antidementias; Gene therapies; Monoclonal antibodies; Neuroprotectants
  • 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.

  • 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

  • 18 May 2018 Pharmacodynamics and adverse events data from a preclinical trial in Friedreich's ataxia released by Voyager Therapeutics
  • 17 May 2018 Pharmacodynamics and safety data from preclinical studies in Amyotrophic lateral sclerosis, and Huntington's disease released by Voyager Therapeutics
  • 10 May 2018 Voyager Therapeutics plans clinical development in Amyotrophic lateral sclerosis, Huntington's disease and Friedreich's ataxia
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