Gazi University in Ankara has officially launched the production process for Türkiye's first local and national gene therapy research product. The project, which targets rare genetic diseases, is being developed under the Research Universities Support Program (ADEP) with the support of the Council of Higher Education (YÖK).

The production process is a joint effort between the Gazi University Faculty of Medicine's Pediatric Metabolism and Pediatric Genetics departments and the Professor Dr. Alev Hasanoğlu Phase-1 Clinical Research Center. The goal is to develop an AAV-based (Adeno-associated virus) therapy that delivers correct genetic material to human cells, adhering to international standards for eventual global use.

Gazi University Rector professor Dr. Uğur Ünal explained that the research process has reached a critical stage. He noted that the center is currently monitoring the treatment of many patients from both Türkiye and abroad.

"In the 100th year of Gazi University, I hope for discoveries that will provide a cure for our patients and children who suffer from these conditions,” Ünal said. He reminded that the clinic was previously honored with an award by President Recep Tayyip Erdoğan. "I believe we will make significant progress in gene therapy that will improve the quality of life for children, not only in Türkiye but across the entire world.”

Rector Ünal highlighted that the agreement signed with a leading American firm, Charles River, represents a significant leap forward in this research journey.

Professor Dr. Fatih Ezgü, head of the Pediatric Genetics Department and deputy director of the Phase-1 Center, explained that the unit was established five years ago to research the safety and efficacy of new drugs. Currently, the center is conducting 40 different advanced treatment studies, 12 of which are specifically focused on gene therapy.

"We said back then that we would successfully implement these treatments in our country, and we have achieved that,” Ezgü said. "Now, we are starting the first stage of producing an investigational gene therapy for a genetic disease that currently has no cure worldwide.”

The specific disease targeted by this first product is Familial Hyperphosphatemic Tumoral Calcinosis, a condition involving abnormal calcium deposits in various body tissues. Ezgü emphasized that the design of the therapy is entirely original to Gazi University. While certain components are sourced from international partners, the final production will occur entirely at the university.

The infrastructure being built is intended to be a platform for other genetic disorders. Ezgü noted that the product is designed to meet the requirements of international health authorities for global approval.

"The process will begin with cellular-level studies, followed by human clinical research,” Ezgü explained. "If we prove the product is safe and beneficial, it will be made available to children worldwide.”

He also emphasized the specialized nature of these treatments, noting that they require rigorous training for the medical teams involved. "For instance, we perform intracranial gene therapy. Our teams undergo very strict training. Because of this, we are one of only four or five centers in the world capable of applying these Phase-1 methods at such an advanced level.”

The center has already become a global hub, with patients from 12 different countries currently receiving treatment. Recently, the center began accepting Palestinian and Jordanian patients who were unable to access research medications in Israel due to the ongoing conflict.