Castle Creek Acquires Novavita Thera to Expand Innovative Cell and Gene Therapy Platform

Cell and gene therapy biotech Castle Creek Biosciences has acquired Novavita Thera, a preclinical gene therapy company focused on rare liver and metabolic diseases.

Photo: Matthew Gantz, president and CEO of Castle Creek Biosciences

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.

“This acquisition is a significant inflection point for Castle Creek and positions us to expand our research and development efforts using a versatile, dual technology platform that will accelerate the discovery of disease-modifying and potentially curative therapies for people living with rare diseases,” said Matthew Gantz, president and CEO of Castle Creek Biosciences. “The ability to leverage both ex vivo and in vivo based approaches is a distinct advantage that few cell and gene therapy companies can offer. We are now in position to pursue new indications for devastating rare diseases, while also advancing our ongoing pivotal clinical trial in recessive dystrophic epidermolysis bullosa (RDEB).”

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.

Castle Creek will advance the development of LV-FAH, a potential therapy based on a lentiviral vector containing a functional copy of the human FAH gene that is administered directly to the patient through the portal vein. 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. Food and Drug Administration for LV-FAH in HT1. Castle Creek will also continue 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, has joined Castle Creek as chief scientific officer. 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 has also joined the company as vice president of preclinical development. Kaiser is a board-certified toxicologist with more than a decade of experience designing, conducting, and reporting preclinical and IND-enabling studies. Lillegard and Kaiser will be the company leads for Castle Creek’s recently announced research collaboration with Mayo Clinic to advance discovery and development of investigational gene therapy candidates for the treatment of osteogenesis imperfecta and classical Ehlers-Danlos syndrome.

Author: Rare Daily Staff