[Edaily Reporter Hyera Lee ] #ENCell Co., Ltd. announced on the 1st that it participated in the “2026 BIO International Convention (BIO USA),” the world’s largest biotech industry event held in San Diego, USA, and held discussions on global partnering for “EN001,” a candidate drug for Charcot-Marie-Tooth disease (CMT).

At the event, ENCell Co., Ltd. held business meetings with approximately 20 companies, including global pharmaceutical and biotech firms, companies specializing in rare diseases, and potential regional partners. The meetings covered EN001’s clinical development status, technical differentiation, commercialization potential in the CMT treatment market, and regional development collaboration strategies.
The company reported that overseas firms expressed strong interest in the encouraging reduction in ‘CMTNSv2 (Charcot-Marie-Tooth Neuropathy Score version 2)’ scores observed in EN001’s clinical results. CMTNSv2 is a key indicator for assessing neurological function and disease progression in CMT patients, and a decrease in this score is a critical indicator of potential clinical improvement.
EN001 is an allogeneic umbilical cord-derived mesenchymal stem cell therapy currently under development based on ENCell Co., Ltd.’s proprietary cell manufacturing platform, “ENCT (ENCell Technology).” ENCT is a platform technology designed to inhibit cell aging and enhance the secretion of regeneration-related factors. EN001 aims to restore nerve function by inducing the secretion of regenerative factors and myelin regeneration in damaged peripheral nerve sites.
CMT is a rare genetic disorder characterized by progressive muscle weakness, sensory loss, and gait disturbances due to peripheral nerve abnormalities. It is considered a prime example of an unmet medical need, as there are currently limited treatments capable of fundamentally inhibiting disease progression.
EN001 is currently undergoing a Phase 2a clinical trial in South Korea for patients with CMT1. Through this trial, ENCell Co., Ltd. is evaluating the safety, efficacy, and optimal dosage following repeated administration, and plans to explore opportunities for development partnerships, technology collaborations, and regional licensing in major markets such as the United States, Japan, and Europe.
EN001 has received Orphan Drug Designation (ODD) for the CMT indication from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Orphan Drug Designation provides a foundation for institutional support during clinical development, regulatory approval strategies, and commercialization, serving as a key factor supporting EN001’s potential for overseas development.
ENCell Co., Ltd. official stated, “At this year’s BIO USA, we were able to gauge the interest of overseas companies in EN001’s clinical data and its commercial potential as a treatment for CMT,” adding, “As a company dedicated to developing treatments for rare diseases, we will translate the clinical value and commercial potential of EN001 into tangible results.”