Castle Creek Biosciences, Inc., a late-clinical stage cell and gene therapy company focused on therapies for rare genetic diseases, has acquired Novavita Thera, Inc., a preclinical gene therapy company focused on rare liver and metabolic diseases. The acquisition expands Castle Creek’s technology platform by adding in vivo capabilities to its existing ex vivo approach, and broadens Castle Creek’s development pipeline beyond skin and connective tissue disorders to rare liver diseases.

With the acquisition of Novavita Thera, Castle Creek will initially develop a gene therapy for hereditary tyrosinemia type 1 (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 metabolites in the liver. HT1 affects approximately 1:100,000 live births and leads to cirrhosis, liver failure, hepatocellular carcinoma, and is ultimately fatal if untreated. Liver transplantation is currently the only curative treatment available for HT1.
 
The 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 (IND) application to the U.S. FDA for LV-FAH in HT1. Castle Creek also continues to progress several additional candidates targeting other rare liver and metabolic diseases and skin and connective tissue disorders.
 
In connection with the acquisition, Joseph Lillegard, MD, PhD, has joined Castle Creek as chief scientific officer. Dr. Lillegard is a board-certified pediatric and adult general, thoracic and fetal surgeon at the Children’s Hospital of Minnesota, and led the cell and gene therapy research lab at Mayo Clinic that discovered LV-FAH. Robert A. Kaiser, PhD, DABT, has also joined the company as vice president of preclinical development. Dr. Kaiser is a board-certified toxicologist with over a decade of experience designing, conducting, and reporting preclinical and IND-enabling studies.