Research programme: calpainopathy gene therapy - Genethon
Latest Information Update: 28 Nov 2023
At a glance
- Originator Genethon
- Class Gene therapies
- Mechanism of Action Calpain replacements; 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 dystrophies
Most Recent Events
- 28 Nov 2023 No recent reports of development identified for preclinical development in Limb-girdle-muscular-dystrophies in France (Parenteral)
- 22 Oct 2019 Preclinical trials in Limb girdle muscular dystrophies in France (Parenteral) (Genethon pipeline, October 2019)