Charles River, Gazi University Partner On HTC Gene Therapy Research, New CDMO Collaboration Targets Rare Phosphate Disorder

Charles River Laboratories and Gazi University Faculty of Medicine have entered into a gene therapy CDMO collaboration to support research into hyperphosphatemic tumoral calcinosis (HTC), a rare inherited disorder marked by elevated phosphate levels and painful calcium deposits around major joints. The partnership will focus on supplying plasmid DNA for adeno-associated virus (AAV) production and conducting in-vitro efficacy studies aimed at advancing early-stage gene therapy development.

HTC is caused by genetic defects that disrupt phosphate regulation, leading to inflammation, mobility challenges, and reduced quality of life. Researchers at Gazi University have identified the GALNT3 gene as a key driver of the disease. Through this collaboration, Charles River will provide ready-made, research-grade AAV plasmids that are animal component–free and royalty-free, enabling faster experimental progress while lowering development costs.

“We are excited to build a strong collaboration with Gazi University,” said Kerstin Dolph, Corporate Senior Vice President, Global Manufacturing, Charles River. “Leveraging cell and gene therapy CDMO expertise will enable the Faculty of Medicine to test and ultimately transform ground-breaking concepts into real-world therapies.”

The plasmids offered are designed as off-the-shelf tools, eliminating the need for lengthy custom manufacturing at the early research stage. This approach is expected to help academic investigators rapidly move from discovery toward proof-of-concept studies in gene therapy programs.

“We are very happy to enter this strategic collaboration, bringing together academic innovation and CDMO expertise to accelerate the development of advanced therapies,” commented Prof. Fatih Ezgü, MD, Professor of Pediatrics and Head of the Department of Pediatric Genetics at Gazi University Faculty of Medicine. “This partnership marks a significant milestone in our mission to pioneer transformative therapies for rare diseases, and we look forward to the progress we can achieve together.”

Charles River highlighted that the project reflects its broader commitment to accelerating advanced therapeutics, supported by its integrated portfolio spanning discovery services, safety assessment, biologics testing, and manufacturing of plasmid DNA, viral vectors, and cell therapies from preclinical work through commercial scale.