Castle Creek Biosciences

Small molecules

LV-FAH Clin0 hereditary tyrosinemia type 1 (ODD); CCB-003 RD Ehlers-Danlos syndrome; CCB-005 RD Ehlers-Danlos syndrome; CCB-001 RD osteogenesis imperfecta; LV-PAH RD phenylketonuria; undisclosed RD hepatic/metabolic diseases

Castle Creek Biosciences is developing gene therapies for a broad range of genetic diseases. There are many ways to deliver gene therapies: Castle Creek is utilizing a lentiviral (LV) delivery platform to develop and commercialize both ex vivo and in vivo therapies for genetic diseases. Castle Creek’s expertise is in using self-inactivating LVs for ex vivo gene therapy of dystrophic epidermolysis bullosa, a rare skin and connective tissue disorder. Now, Castle Creek is focusing on in vivo gene therapies beginning with inborn errors of metabolism of the liver. With their dual LV vector delivery platform, Castle Creek is establishing their pipeline in pursuit of multiple indications.