Meet Lottie
Hereditary tyrosinemia type 1 (HT1) is a rare inborn error of metabolism caused by a lack of the enzyme FAH that leads to the accumulation of tyrosine and its metabolites in the liver. HT1 affects approximately 1:100,000 live births and leads to cirrhosis, liver failure, hepatocellular carcinoma, and ultimately is fatal if untreated. Currently, HT1 is managed through chronic administration of the protective drug, nitisinone, which delays severe outcomes, but this management is associated with neurocognitive deficiencies. Liver transplantation is currently the only curative treatment available for HT1, which has its own life-long consequences.
Castle Creek is advancing preclinical development of LV-FAH, an in vivo investigational gene therapy for the treatment of HT1. LV-FAH is based on a lentiviral vector containing a functional copy of the human FAH gene which is administered directly to the patient through the portal vein. This therapy is designed to transduce hepatocytes and deliver the FAH enzyme that is deficient in these cells.
Castle Creek plans to submit an Investigational New Drug application for LV-FAH to the U.S. Food and Drug Administration for the treatment of HT1.
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Photo: Courtesy of the Pratt family. Used with permission
Castle Creek Biosciences’ product candidates are investigational. None of our investigational gene therapies have received marketing approval by the U.S. Food and Drug Administration nor any other regulatory agency
Designed to use lentiviral technology in vivo to deliver gene of interest to a patient’s cells