BIO-Europe Spring Presenting Companies 2023
Emendo Biotherapeutics is a next generation gene editing company with a diverse therapeutic pipeline based on proprietary nucleases. Our nuclease technology combines a discovery pipeline and cutting-edge protein-engineering capabilities, supported by extensive computational and machine learning tools. The resulting panel of novel nucleases is the OMNI(TM) series used in our clinical pipeline and available for licensing. OMNITM nucleases overcome key challenges in the field: they are variable in size and compatible with all delivery modalities (including AAV); diverse PAM recognition allows for 86% genome coverage; OMNI(TM) 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 the core of our lead program for ELANE-dependent Severe Congenital Neutropenia (SCN), a life-threatening immunodeficiency disease. SCN is a dominant negative genetic disease usually caused by heterozygous toxic gain of function mutations in ELANE. The therapeutic approach is based on monoallelic knockout in patient-derived hematopoietic stem cells (HSCs). We are completing the IND submission for this program, with a first-in-human study planned for the second half of 2023. A different therapeutic approach is taken for Familial Hypercholesterolemia (FH). FH is characterized by elevation in low‐density lipoprotein cholesterol (LDL‐C), leading to atherosclerosis and coronary disease. The majority of genetically confirmed FH is caused by LDLR haploinsufficiency resulting in reduced LDL-C uptake. Emendo biotherapeutics’ unique editing strategy utilizes our OMNI-A2 novel nuclease to upregulate LDLR expression, without the need to knock-in genes. Additional OMNITM nucleases are used for ophthalmology and immune-oncology advanced pre-clinical stage programs.