Research programme: gene therapies - Genethon/ViroMedAlternative Names: AAV vector containing modified U7 snRNA gene for Duchenne muscular dystrophy - Genethon; AAV vector transferred UGT1A1 gene - Genethon; Gene therapy for Crigler-Najjar syndrome - Genethon; Gene therapy for Duchenne muscular dystrophy - Genethon; Gene therapy for Leber's hereditary optic neuropathy (LHON) - Genethon; Gene therapy for myotubular myopathy - Genethon/Wake Forest University School of Medicine; SMN1 gene therapy for spinal muscular atrophy - Genethon; VM 101 - ViroMed; VM 102; VM 103; VM 104; VM 105
Latest Information Update: 22 Jun 2015
At a glance
- Originator ViroMed Co Ltd
- Developer Genethon; ViroMed; Wake Forest University School of Medicine
- Class Gene therapies
- Mechanism of Action Dystrophin expression stimulants; Gene transference
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 Congenital structural myopathies; Duchenne muscular dystrophy; Eye disorders; Immunological disorders; Liver disorders; Metabolic disorders; Musculoskeletal disorders; Spinal muscular atrophy