Gene Editing InnovationEditas has nominated EDIT-401, an in vivo gene editor to be used in the treatment of atherosclerotic cardiovascular disease and heterozygous familial hypercholesterolemia.
Preclinical SuccessEDIT-401 showed potentially best-in-class LDL-C depletion (90% LDL-C reduction) in preclinical NHP and mice models compared to in vivo gene editors and RNA therapies.
Safety ProfileNo concerning adverse events were identified after treatment in both non-human primate or mouse models.