Program
Collaborator
Lead Optimization
Pre-Clinical
Ind-Enabling
Phase 1-3
hamatology

Hematology

Lead Optimization

Pre-Clinical

Ind-Enabling

Phase 1-3

EMD-101 Severe Congenital Neutropenia

University of Washington

Ind-Enabling

Collaborator:University of Washington

Ophthalmology

Ophthalmology

Lead Optimization

Pre-Clinical

Ind-Enabling

Phase 1-3

EMD-201 Retinitis Pigmentosa

Columbia University

Ind-Enabling

Collaborator:Columbia University

EMD-202 Cone-Rod Dystrophy

Proprietary

Ind-Enabling

Collaborator:Proprietary

EMD-203 Macular Dystrophy

Proprietary

Ind-Enabling

Collaborator:Proprietary

Immuno-oncology

Immuno-oncology

Lead Optimization

Pre-Clinical

Ind-Enabling

Phase 1-3

CAR-T Cells

Confidential

Ind-Enabling

Collaborator:Confidential

NK Cells

Proprietary

Ind-Enabling

Collaborator:Proprietary

Liver

Liver

Lead Optimization

Pre-Clinical

Ind-Enabling

Phase 1-3

EMD-301 Familial Hypercholesterolemia

Proprietary

Ind-Enabling

Collaborator:Proprietary

EMD-302 Inborn Errors of Metabolism

Proprietary

Ind-Enabling

Collaborator:Proprietary

Emendo is focused on bringing innovation to disease indications where there has been little to no progress. We tackle the “incurable” with our CRISPR-based technology platform to bring to bear the highest precision in gene editing to treat the most challenging diseases.

Our novel approach to engineering nucleases creates a custom fit design for each disease, allowing us to achieve single-allele editing strategies for the severest indications, such as Severe Congenital Neutropenia (SCN), a debilitating disease caused by mutations in the neutrophil elastase gene ELANE. Our successful knockdown of the single ELANE allele for SCN demonstrates the incredible therapeutic potential of Emendo’s approach, opening up a wide range of options for gene editing therapies where traditional CRISPR nucleases have previously been ineffective.

close