EmendoBio is a gene editing company with a diverse therapeutic pipeline based on proprietary nucleases. Our nuclease technology combines a discovery pipeline, cutting-edge protein-engineering capabilities, and extensive computational tools. The resulting panel of 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. Moreover, diverse PAM recognition allows for 86% genome coverage. OMNI nucleases are engineered to be hyper-active and super specific thus eliminating off-target effects. OMNI-A1, an allele specific editor, is used in our program for ELANE-dependent Severe Congenital Neutropenia (SCN), a dominant negative genetic disease. Our 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 pre-clinical stage programs.
Company Type:
Privately Funded Company
Company Website:
Company HQ State:
Delaware
Company HQ Country:
United States
Year Founded:
2016
Main Therapeutic Focus:
Gene/Cell Therapy
Lead Product in Development:
EMD-101 - Severe Congenital Neutropenia
Development Phase of Primary Product:
Pre-Clinical
Number Of Unlicensed Products (For Which You Are Seeking Partners):
Not Provided
Speaker
BIO DOUBLE HELIX SPONSORS
BIO HELIX SPONSORS
