[Edaily Reporter Shin Ha-yeon] #ENCell, a company specializing in cell and gene therapies, is accelerating the development of its Charcot-Marie-Tooth (CMT) treatment 'EN001' by presenting clinical data at a global academic conference.

ENCell announced on the 16th that it presented the combined results of the Phase 1a and 1b clinical trials for its CMT treatment EN001 at the '2026 PNS Annual Meeting,' held in Maastricht, Netherlands, from the 13th to the 16th.

The PNS Annual Meeting is a leading international academic event in the field of peripheral nerve disorders, serving as a platform for researchers and clinical experts worldwide to share the latest research findings and development trends.

EN001 is an allogeneic umbilical cord-derived mesenchymal stem cell-based therapy currently under development for patients with CMT Type 1. CMT is a hereditary peripheral nerve disorder and a representative rare disease characterized by progressive deterioration of muscle strength, sensory loss, and gait disturbances.

In this presentation, safety and exploratory efficacy data from 18 CMT Type 1 patients, including results from the recently completed Phase 1b clinical trial, were disclosed.

According to the company, no dose-limiting toxicities (DLTs), infusion-related reactions (IRRs), or serious adverse events (SAEs) were observed in the EN001 treatment group, confirming its favorable safety profile.

In the exploratory efficacy evaluation, signs of improvement were observed in the 'CMTNSv2' score, which indicates the severity of CMT. The integrated analysis showed an average reduction of 6.2 points from a baseline score of approximately 18 points at the time of maximum response.

CMT is known to be a progressive disease in which the CMTNSv2 score worsens by approximately 0.5 points annually over its natural course; typically, even a change of 1 to 2 points is considered clinically significant. The company explained that these results represent exploratory data demonstrating the potential to delay disease progression over the long term.

An E-Ncell representative stated, “The PNS Annual Conference is a major international conference in the field of peripheral nerve disorders. The selection of the EN001-CMT clinical results for presentation will serve as an important opportunity to share our development status and clinical potential with global experts, thereby attracting attention in the global market.” "Given that we have confirmed the favorable safety profile and exploratory efficacy signals of EN001, including the recently completed Phase 1b clinical trial, we will accelerate subsequent clinical development to provide a new treatment option for CMT patients."