Research programme: CFTR mRNA therapeutics - Translate Bio
Latest Information Update: 17 Sep 2021
At a glance
- Originator Translate Bio
- Class Antifibrotics; RNA
- Mechanism of Action Cystic fibrosis transmembrane conductance regulator stimulants
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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 Yes
Highest Development Phases
- Preclinical Cystic fibrosis