EMD-101

EMD-101 is an investigational autologous hematopoietic stem cell therapy developed by Emendo Biotherapeutics for the treatment of severe congenital neutropenia (SCN). It utilizes a CRISPR-based, non-viral gene editing approach to specifically knock out the mutated allele of the ELANE gene in patient-derived hematopoietic stem cells while preserving the healthy allele. This monoallelic knockout strategy enables restoration of normal neutrophil differentiation and function. The edited cells are reinfused into the patient after thorough safety and potency assessment. EMD-101 is designed to address autosomal dominant mutations in ELANE, which account for a significant proportion of SCN cases, and has demonstrated proof-of-concept efficacy in preclinical studies with improved myeloid maturation and long-term engraftment. The therapy is currently in preclinical development, nearing clinical trials.