Drug Profile

Research programme: gene editing therapeutics - GenEdit/Sarepta Therapeutics

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 No

28 Jan 2025 No recent reports of development identified for preclinical development in Neuromuscular-disorders in USA (Parenteral)
31 Dec 2020 Sarepta Therapeutics and GenEdit signs a research collaboration and option agreement for gene editing therapeutics in Neuromuscular disorders in December 2020
31 Dec 2020 Preclinical trials in Neuromuscular disorders in USA (Parenteral) as of December 2020

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