Drug Profile
Research programme: dysferlin gene therapies - Genethon
Alternative Names: AAV1 dysferlin gene therapy - Genethon; Adenoviral-associated vector-mediated dysferlin gene therapy - Genethon; Dystrophy-associated fer-1-like protein therapy - Genethon; Gene therapy for dysferlinopathies - Genethon; Girdle muscular dystrophy 2B gene therapy - Genethon; Miyoshi myopathy gene therapy - GenethonLatest Information Update: 28 Sep 2021
Price :
$50
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At a glance
- Originator Genethon
- Class Gene therapies
- Mechanism of Action 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 Limb girdle muscular dystrophy type 2B
Most Recent Events
- 28 Sep 2021 No recent reports of development identified for preclinical development in Limb girdle Muscular-dystrophy type 2B in France (Parenteral)
- 18 Sep 2019 Preclinical development is ongoing in France (Genethon pipeline, September 2019)
- 25 Feb 2016 Preclinical development is ongoing in France