Drug Profile

EMU 003

Alternative Names: EMU003

Latest Information Update: 26 Sep 2025

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At a glance

Originator Emugen Therapeutics
Class Antiepileptic drugs
Mechanism of Action RNA splicing modulators; SCN1A protein expression modulators

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

13 Aug 2025 Emugen Therapeutics in-licenses TfR1 CapX capsid technology from Apertura Gene Therapy
13 Aug 2025 EMU 003 is covered by patent protection (Emugen Therapeutics pipeline, August 2025)
13 Aug 2025 Early research in Dravet syndrome in USA (unspecified route)

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