Drug Profile

EN 001

Alternative Names: EN-001

Latest Information Update: 04 Aug 2025

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At a glance

Originator ENCell
Class Mesenchymal stem cell therapies; Neuroprotectants
Mechanism of Action Cell replacements

Orphan Drug Status

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

Yes - Charcot-Marie-Tooth disease
New Molecular Entity No

Highest Development Phases

Phase I Charcot-Marie-Tooth disease; Duchenne muscular dystrophy

Most Recent Events

04 Aug 2025 Phase I development for Charcot-Marie-Tooth disease is ongoing South Korea (IV)
04 Aug 2025 Phase I development for Duchenne muscular dystrophy (In adolescents, In children) is ongoing South Korea (IV)
07 Mar 2025 EN 001 receives Orphan Drug status for Charcot-Marie-Tooth disease in USA

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