EMD-101, A Potential Autologous HSC-Based Therapy for ELANE-Related Severe Congenital Neutropenia

Time: 11:45 am
day: Day Two


  • EMD-101 is a patient-derived HSCs product with a specific knock-out of ELANE mutated allele, that reverts the block of myeloid differentiation, typical of severe congenital neutropenia (SCN)
  • The editing profile of our product undergoes a thorough genomic analysis to meet the highest safety standards
  • Our product maintains the core functions of neutrophils in vitro, in addition to efficient engraftment and multi-lineage differentiation in vivo