ENCell, a South Korean company focused on cell and gene therapies, announced in May 2026 (as reported by Korean outlet The Bio News) that the first patient has been dosed in a Phase 2a clinical trial of EN001, a treatment candidate for Charcot-Marie-Tooth disease Type 1A.  

This is a significant step forward: unlike the Phase 1 stage, which focused primarily on safety, this trial is designed to evaluate whether EN001 actually works. 

This phase 2a study focuses on efficacy. The trial will enroll 21 patients across three major university hospitals in South Korea, beginning with Samsung Medical Center. 

What Is EN001? 

EN001 is a cell therapy, meaning the treatment itself is made of living cells rather than a traditional pill or an injection of genetic material. The cells used to make EN001 come from umbilical cord tissue donated at birth, and ENCell has developed a specialized manufacturing process to prepare them for use as a medicine. 

The therapy is designed to promote remyelination, which is the repair of the protective myelin sheath around nerve fibers that deteriorates in CMT1A, and to support functional recovery of damaged peripheral nerves. 

What makes EN001 notable is its mechanism: rather than targeting a specific gene mutation, it works by delivering cells that secrete nerve health factors, encouraging the nervous system to repair itself. Prior results from a Phase 1b clinical trial showed a favorable safety profile. 

How the Trial Is Structured 

The Phase 2a trial uses a placebo-controlled design with three cohorts: placebo, low-dose, and high-dose. Researchers will track changes in the CMT Neuropathy Score over 24 weeks, a validated clinical measure of nerve function and disease severity. The goal is to establish both whether EN001 works and the optimal dose, which will be critical for advancing to a larger Phase 2b or Phase 3 trial. 

Why This Matters for CMT Patients 

CMT1A is the most common subtype of CMT, and right now there is no approved treatment that addresses the underlying disease. EN001 represents a different kind of approach than the small molecule and gene therapy candidates also in development. If validated, it could offer an option for patients regardless of their specific genetic variant within CMT1A. 

ENCell also shared that it is expanding its development pipeline beyond CMT into other muscle and nerve disorders, including sarcopenia, which reflects growing confidence in the underlying cell therapy platform. 

The CMT Research Foundation will continue to monitor EN001’s progress and share updates as trial results become available.