Argininosuccinic aciduria gene therapy - Insmed
Latest Information Update: 18 May 2023
At a glance
- Originator Insmed
- 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
- Preclinical Argininosuccinic aciduria
Most Recent Events
- 12 May 2023 Preclinical trials in Argininosuccinic aciduria in USA (Parenteral) prior to May 2023 (Insmed pipeline, May 2023)