Research programme: small molecule therapeutics - Vertex
Latest Information Update: 28 Jan 2025
At a glance
- Originator Vertex Pharmaceuticals
- Class Small molecules
- Mechanism of Action Alpha 1-antitrypsin modulators
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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
- Available For Licensing Yes - Alpha 1-antitrypsin deficiency
Highest Development Phases
- No development reported Alpha 1-antitrypsin deficiency
Most Recent Events
- 28 Jan 2025 No recent reports of development identified for preclinical development in Alpha-1-antitrypsin-deficiency in USA
- 30 Jan 2023 Small molecule therapeutics is still in preclinical trials for Alpha-1-antitrypsin-deficiency in USA (Vertex Pharmaceuticals pipeline, January 2023)
- 28 Nov 2022 No recent reports of development identified for preclinical development in Alpha-1-antitrypsin-deficiency in USA