Research programme: gene therapies for methylmalonic acidemia - Genespire
Latest Information Update: 25 May 2024
At a glance
- Originator Genespire
- Class Gene therapies
- Mechanism of Action Gene transference; Methylmalonyl CoA mutase replacements
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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 Methylmalonic acidaemia