GeneRide-FAH

**GeneRide-FAH** is a preclinical AAV-based in vivo gene therapy developed by LogicBio Therapeutics for hereditary tyrosinemia type 1. It uses the company's GeneRide genome editing platform, which is designed to harness homologous recombination to insert a functional **fumarylacetoacetate hydrolase** gene into hepatocytes without using exogenous nucleases. The therapeutic intent is to restore hepatic FAH activity in patients with FAH deficiency, thereby correcting tyrosine metabolism and reducing accumulation of toxic metabolites. Preclinical mouse data reported liver repopulation by corrected hepatocytes and normalization of growth, liver function, and toxic metabolite levels. LogicBio subsequently nominated **LB-401** as a development candidate for hereditary tyrosinemia type 1 based on this platform program.