SERPINC1 gene therapy

SERPINC1 gene therapy is an experimental in vivo CRISPR-based gene-editing therapy being developed by Metagenomi Therapeutics for the treatment of inherited thrombophilias, specifically antithrombin III (ATIII) deficiency. The therapy utilizes a two-component delivery system consisting of an adeno-associated virus (AAV) vector carrying a human SERPINC1 donor gene template and a lipid nanoparticle (LNP) delivering a CRISPR nuclease and guide RNA. This system is designed to target and cut the endogenous albumin locus (specifically exon 1) in hepatocytes, facilitating the site-specific integration of the SERPINC1 gene under the control of the highly active endogenous albumin promoter. By permanently inserting the functional gene, the therapy aims to achieve stable, long-term expression of biologically active ATIII protein within the therapeutic range, potentially offering a one-time curative treatment that eliminates the need for lifelong anticoagulant therapy.

Other names

Antithrombin III gene therapySERPINC1 gene knock-in therapySERPINC-1 gene knock-in therapySERPINC 1 gene knock-in therapy