[Edaily Reporter Shin Ha-yeon ] #ENCell Co., Ltd., a company specializing in cell and gene therapies (CGT), is accelerating its Phase 2a clinical trial for “EN001,” a candidate drug for Charcot-Marie-Tooth disease type 1A (CMT1A).

ENCell Co., Ltd. announced on the 8th that the Phase 2a clinical trial for EN001 has officially entered the multi-center, simultaneous administration phase.

Three institutions—Samsung Seoul Hospital, Kangdong Kyung Hee University Hospital, and Dongguk University Gyeongju Hospital—are participating in this clinical trial. Currently, patient enrollment and administration of the investigational drug are proceeding sequentially at each institution, and the company expects the pace of the trial to accelerate now that all institutions have entered the dosing phase.

The EN001 Phase 2a clinical trial was designed to evaluate the safety and efficacy of repeated dosing in patients with CMT1A. The trial is divided into a placebo group, a low-dose group, and a high-dose group to compare the therapeutic effect relative to the placebo and assess dose-response relationships. The primary endpoint is the change in the CMT Neuropathy Scale (CMTNS v2) at Week 24.

CMT1A is a rare genetic disorder characterized by peripheral nerve damage, leading to muscle weakness, sensory loss, and gait disturbances. Currently, there are no treatments that fundamentally halt the progression of the disease, so management focuses on rehabilitation and symptom relief.

EN001 is an allogeneic umbilical cord-derived mesenchymal stem cell (MSC) therapeutic candidate manufactured using ENCell Co., Ltd.’s proprietary cell culture platform technology, “ENCT (ENCell Technology).” Classified as an advanced biopharmaceutical, it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the CMT indication.

ENCell Co., Ltd. views this Phase 2a clinical trial as a step toward securing the evidence needed to design a subsequent confirmatory clinical trial and pursue conditional marketing authorization. Based on the clinical data, the company plans to confirm the safety, efficacy, and optimal dosage of EN001 and accelerate efforts toward expedited review and conditional approval in the future.

ENCell Co., Ltd. official stated, “Patient enrollment and administration of the investigational drug are proceeding smoothly as planned at three clinical trial sites,” adding, “While prioritizing the quality of the clinical trial and the safety of participants, we will focus on confirming the safety, efficacy, and optimal dosage of EN001.”