Drug Profile

Research programme: RNA splicing modifiers - Skyhawk Therapeutics/Vertex Pharmaceuticals

Latest Information Update: 28 Jan 2025

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At a glance

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

28 Jan 2025 No recent reports of development identified for research development in Unspecified in USA
22 Dec 2020 Skyhawk Therapeutics and Vertex Pharmaceuticals agree to co-develop RNA splicing modulators in USA for unspecified indication
22 Dec 2020 Early research in Unspecified in USA (unspecified route)

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