EMD-101 / Emendo Biotherap - LARVOL DELTA

EMD-101, an autologous, allele-specific gene-edited hematopoietic stem cell product, for treating ELANE-mediated severe congenital neutropenia (ESGCT 2022) - No abstract available

Hematological Disorders • Neutropenia • ELANE

Safe and Efficient Engraftment of CRISPR-Based ELANE Mono-Allelic Knocked out HSCs in Mice: Evidence for a Novel Treatment for ELANE Neutropenia (ASH 2021) - "Emendo Biotherapeutics has developed a novel CRISPR-based (OMNI A1) ex-vivo gene editing strategy that involves specific excision of disease-causing ELANE mutant allele...These data show that edited cells can be engrafted successfully, maintain their excision profile and populate the bone marrow. This study also supports the safety of the OMNI A1 therapeutic composition."

Preclinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • Transplantation • CD19 • CD33 • CD34 • ELANE • PTPRC