Castle Creek Biosciences’ in vivo investigational gene therapy technology is designed to use a lentiviral vector (LV) to deliver functional copies of genes that are deficient in a patient’s own liver cells due to genetic diseases. Initially, this therapy is being developed for HT1, a rare inborn error of metabolism caused by a lack of the enzyme fumarylacetoacetate hydrolase (FAH) which leads to accumulation of tyrosine and its toxic metabolites in the liver.
Our in vivo technology approach uses a LV that is packaged with a functional copy of the therapeutic gene (transgene) and is designed to be infused through the portal vein. Foundational preclinical research1 has shown the vector transduces the target (diseased) liver cells, called hepatocytes, and integrates the transgene into the DNA of the diseased cells where it expresses the therapeutic protein to correct the cells. These corrected hepatocytes continue to expand in number to replace the diseased cells in the liver.
1Clara T Nicolas, Caitlin J VanLith, Kari L Allen, Raymond D Hickey, Zeji Du, Lori G Hillin, Rebekah M Guthman, William J Cao, Aditya Bhagwate, Daniel O’Brien, Jean-Pierre Kocher, Robert A Kaiser, Stephen J Russell, Joseph B Lillegard. In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions. bioRxiv 2021.01.02.425079; doi: https://doi.org/10.1101/2021.01.02.425079.
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.
