Charles River and Gazi University Partner on Gene Therapy Development

Charles River Laboratories International and Gazi University Faculty of Medicine have entered into a strategic collaboration aimed at advancing gene therapy research for rare genetic diseases. Under the agreement, Charles River will act as a contract development and manufacturing organization (CDMO), supplying plasmid DNA for adeno-associated virus (AAV) production and supporting in vitro efficacy studies.

The collaboration brings together Charles River’s global expertise in cell and gene therapy manufacturing with Gazi University’s academic research capabilities. By providing research-grade AAV plasmids, Charles River will support early-stage gene therapy development and help translate laboratory discoveries into potential clinical therapies.

A key focus of the partnership is hyperphosphatemic tumoral calcinosis (HTC), a rare inherited disorder characterized by abnormally high levels of phosphate in the blood. The condition leads to painful, non-cancerous calcium phosphate deposits in soft tissues, particularly around major joints such as the hips, shoulders, and elbows. These deposits can cause chronic pain, inflammation, and significant limitations in mobility, severely impacting patients’ quality of life. HTC results from genetic defects that disrupt phosphate regulation, and researchers at Gazi University have identified the GALNT3 gene as a primary driver of the disease.

As part of the agreement, Charles River will supply off-the-shelf, research-grade AAV plasmids to Gazi University. These plasmids are pre-manufactured, animal component–free, and royalty-free, offering a ready-to-use solution for academic and early development programs. The approach is designed to reduce development timelines and costs, enabling researchers to focus on evaluating therapeutic concepts rather than building manufacturing infrastructure from scratch.

Charles River executives noted that the collaboration reflects the company’s commitment to supporting innovative academic research through its CDMO capabilities. By combining advanced manufacturing expertise with university-led discovery, the partnership aims to accelerate the transformation of promising gene therapy concepts into viable treatment candidates.

Gazi University also emphasized the strategic importance of the agreement. Faculty leaders highlighted the value of integrating academic innovation with industry-grade development and manufacturing expertise to advance therapies for rare diseases. The collaboration is seen as a significant step toward developing transformative gene therapies for conditions like HTC, where treatment options are currently limited.

Overall, the partnership underscores a growing trend of closer collaboration between academia and industry in the gene therapy space, with the shared goal of accelerating progress from discovery to real-world patient impact.

Comments (0)
Add Comment