Drug Profile

Research programme: gene editing therapies - Moderna Therapeutics/Vertex Pharmaceuticals

Latest Information Update: 28 Oct 2024

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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 No

28 Oct 2024 No recent reports of development identified for research development in Cystic-fibrosis in USA (Parenteral)
02 Nov 2021 Vertex Pharmaceuticals plans to submit Investigational New Drug application for gene editing therapies in 2022
16 Sep 2020 Early research in Cystic fibrosis in USA (Parenteral), prior to September 2020

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