23 Jan Emendo Biotherapeutics
Rachel Diamant, Chief Business Officer
April 13 | 12:15pm | Dark Horse Consulting Ballroom
New York, NY
Emendo Biotherapeutics is a next-generation gene editing company with a therapeutic pipeline based on proprietary nucleases. Our nuclease technology combines a discovery pipeline and protein-engineering capabilities, supported by extensive computational and machine learning tools. The resulting panel of novel nucleases is the OMNI series. OMNI nucleases overcome key challenges in the field as they are variable in size and compatible with all delivery modalities (including AAV), diverse PAM recognition allows for 86% genome coverage. Furthermore, the OMNI nucleases are engineered to be hyper-active and super specific thus eliminating off-target effects and enabling allele specific editing. OMNI-A1, an allele specific editor, is at the base of our lead program for ELANE-dependent Severe Congenital Neutropenia (SCN), a dominant negative genetic disease. The therapeutic approach to Familial hypercholesterolemia (FH) utilizes OMNI-A2 to upregulate the expression of the LDLR gene. Additional OMNI nucleases are used for ophthalmology and immune-oncology advanced preclinical-stage programs.