Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A(CMT1A) (Phase 1b: Open-label, Dose-escalation, Single-center; Phase 2a: Randomized, Double-blind, Placebo-controlled, Multicenter)

ENCell, a South Korean company, is testing EN001, donor stem cells from umbilical cord tissue (Wharton’s jelly), in about 27 adults with CMT1A. An open-label part finds the dose; a second part compares a low or high dose with placebo, given as two intravenous infusions four weeks apart.

The study is recruiting at three hospitals in Seoul and Gyeongju, and the registry expects it to finish at the end of 2026. Earlier results from a nine-person study came from a company press release, not a peer-reviewed paper.