LNP-CRISPR-Cas9

LNP-CRISPR-Cas9 is an experimental gene-editing therapeutic platform that utilizes lipid nanoparticles (LNPs) to deliver CRISPR-Cas9 components, typically consisting of Cas9 messenger RNA (mRNA) and a single guide RNA (sgRNA). In the context of treating hereditary tyrosinemia type 1 (HT1), the platform is designed to target and knock down the *Hpd* (4-hydroxyphenylpyruvate dioxygenase) gene in hepatocytes. The delivery system employs ionizable cationic lipids, such as the experimental formulation 306-O10S or the clinically validated ALC-0315, to facilitate intracellular uptake and endosomal release of the gene-editing machinery. This therapy can be administered through systemic in vivo injection or via an ex vivo approach where primary hepatocytes are isolated, edited, and subsequently transplanted back into the liver to engraft and repopulate the organ. The goal is to achieve permanent genetic disruption of the *Hpd* gene to rescue the disease phenotype in *Fah*-deficient models.

Other names

LNP-delivered CRISPR-Cas9LNP-mediated CRISPR-Cas9LNP-enabled gene editing cell therapy