HITI-hF8-ex22-26 gene therapy

HITI-hF8-ex22-26 is an experimental gene therapy candidate designed for the treatment of Hemophilia A, specifically targeting the common intron 22 inversion mutation. The therapy employs Homology-Independent Targeted Integration (HITI), a CRISPR/Cas9-based genome editing technique, to precisely integrate a functional human Factor VIII (hF8) exon 22-26 cassette into intron 21 of the endogenous F8 gene. This site-specific integration is intended to bypass the inversion mutation and restore the production of full-length, functional Factor VIII protein. Delivery of the CRISPR components (Cas9 and sgRNA) and the donor DNA has been demonstrated using both plasmid-based hydrodynamic injection and non-viral virus-like particles (VLPs). Preclinical data indicate successful restoration of FVIII activity in mouse models and robust editing efficiency in human cell lines.

Other names

HITI-mediated integration of hFVIII exons 22–26