EDIT-401(mu)

EDIT-401(mu) is a preclinical CRISPR-based gene editing candidate developed by Editas Medicine as a murine surrogate for their hereditary angioedema (HAE) program. It utilizes an engineered CRISPR nuclease (AsCas12a) delivered via lipid nanoparticles (LNPs) to specifically target and disrupt the *Klkb1* gene in hepatocytes. This knockout reduces the synthesis of prekallikrein, the precursor to plasma kallikrein. By lowering plasma kallikrein activity, the therapy aims to suppress the uncontrolled production of bradykinin, thereby preventing the inflammatory edema and swelling attacks associated with HAE. The (mu) version is specifically designed with guide RNAs targeting the mouse *Klkb1* sequence to demonstrate in vivo proof-of-concept and safety in animal models prior to clinical translation of the human candidate, EDIT-401.