Research programme: gene therapies - Genethon/Helixmith
Alternative Names: AAV vector containing modified U7 snRNA gene for Duchenne muscular dystrophy - Genethon; Gene therapy for Duchenne muscular dystrophy - Genethon; SMN1 gene therapy for spinal muscular atrophy - Genethon; VM 101 - Helixmith; VM 102; VM 103; VM 104; VM 105Latest Information Update: 28 Dec 2021
At a glance
- Originator ViroMed Co Ltd
- Developer Genethon; Helixmith
- Class Gene therapies
- Mechanism of Action Dystrophin expression stimulants; Gene transference
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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
- No development reported Duchenne muscular dystrophy; Immunological disorders; Metabolic disorders; Musculoskeletal disorders; Spinal muscular atrophy
Most Recent Events
- 28 Dec 2021 No recent reports of development identified for preclinical development in Duchenne muscular dystrophy in France (Parenteral)
- 28 Dec 2021 No recent reports of development identified for preclinical development in Immunological-disorders in France (Parenteral)
- 28 Dec 2021 No recent reports of development identified for preclinical development in Metabolic-disorders in France (Parenteral)