Charcot-Marie-Tooth Association Strategy To Accelerate Research (CMTA-STAR) Alliance Partner ENCell, Co., Ltd. (ENCell) announced that it will conduct a Phase 1b/2a clinical trial of its investigational stem cell therapy EN001 for people with CMT1A, following approval of a modification application to the Korea Food and Drug Administration. The study is underway at Samsung Medical Center in Seoul, South Korea.
ENCell also reported results from an earlier Phase 1a trial, which focused on safety outcomes. While the company previously reported that EN001 has received Orphan Drug Designation (ODD) from the FDA, the current trial is only open in South Korea.
According to the company, EN001 is a mesenchymal (pronounced meh-ZEN-kime-uhl) stem cell therapy built on its ENCT platform. ENCell says the therapy is designed to target damaged nerves, release regenerative signals, and promote peripheral nerve remyelination. This approach is distinct, the company believes, because most treatments in development focus on slowing degeneration rather than rebuilding myelin.
Read ENCell’s full announcement here.
Read ENCell’s ODD announcement here.
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