Drug Profile
Research programme: gene therapies - 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; CNS-Directed GBA1 Gene Therapy - Voyager Therapeutics; Friedreich’s ataxia gene therapy - Voyager Therapeutics; GBA1 gene replacement therapy - Voyager Therapeutics; Tau silencing gene therapy - Voyager Therapeutics; VY FXN01; VY SMN101; VY SOD101; VY SOD102; VY-NAV01Latest Information Update: 05 Mar 2024
Price :
$50
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At a glance
- Originator Voyager Therapeutics
- Class Analgesics; Antidementias; Gene therapies; Neuroprotectants; Small interfering RNA
- Mechanism of Action Gene transference; Nav1.7 voltage-gated sodium channel inhibitors; Tau protein expression inhibitors; Tau protein inhibitors
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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; CNS disorders; Friedreich's ataxia
- No development reported Dementia; Huntington's disease; Pain; Spinal muscular atrophy