EN001

EN001 is an investigational allogeneic cell therapy consisting of early-passage mesenchymal stem cells (MSCs) derived from Wharton’s Jelly of the umbilical cord. Developed by ENCell, the therapy is designed to treat degenerative neuromuscular and musculoskeletal disorders, including Duchenne muscular dystrophy (DMD), Charcot-Marie-Tooth disease (CMT), and sarcopenia. EN001 exerts its therapeutic effects primarily through paracrine mechanisms, secreting trophic factors such as XCL1 (lymphotactin) and MMP-1 to promote anti-apoptotic and anti-fibrotic pathways, respectively. By utilizing early-passage cells (typically passage 3), EN001 aims to maintain high differentiation potential and migratory capacity while minimizing cellular senescence and immunogenicity. Clinical studies have demonstrated the safety and tolerability of single intravenous administrations in pediatric DMD patients and adult CMT patients, with ongoing trials evaluating repeated dosing and long-term efficacy.

Other names

Wharton's jelly-derived mesenchymal stem cellsWJ-MSCsallogeneic early-passage mesenchymal stem cells