Drug Profile

EN 001

Alternative Names: EN-001

Latest Information Update: 15 Sep 2026

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At a glance

Originator ENCell
Class Mesenchymal stem cell therapies
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/II Charcot-Marie-Tooth disease
Phase I Duchenne muscular dystrophy

Most Recent Events

11 Sep 2026 ENCell plans a phase I/II trial for Sarcopenia in South Korea in March 2027 (IV) (NCT07808905)
26 May 2026 Phase-I/II clinical trials in Charcot-Marie-Tooth disease in South Korea (IV) (NCT06328712)
01 Nov 2025 ENCell completes a phase I clinical trials in Charcot-Marie-Tooth disease in South Korea (IV) (NCT06328712)

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