Vertex expands partnership with MNGHA to advance gene therapy care in Saudi Arabia

The announcement was made at the Riyadh Global Medical Biotechnology Summit (RGMBS), building on a collaboration established after Saudi Arabia’s first gene therapy infusion in 2024.

SAUDI ARABIA—Vertex Pharmaceuticals and the Ministry of National Guard Health Affairs (MNGHA) have renewed and expanded their strategic partnership to advance gene therapy care and clinical capabilities in the Kingdom.

The announcement was made at the Riyadh Global Medical Biotechnology Summit (RGMBS), building on a collaboration established after Saudi Arabia’s first gene therapy infusion in 2024.

The renewed two-year partnership will focus on patient care, clinical expertise, knowledge exchange, and the development of healthcare capabilities in Saudi Arabia.

Following approval by the Saudi Food and Drug Authority (SFDA), the collaboration has expanded access to a one-time functional cure for eligible patients with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

It has also supported the treatment of additional eligible patients in the Kingdom.

Clinical education and research

As part of the renewed partnership, MNGHA and Vertex will organize a third joint clinical Preceptorship program.

The initiative follows two previous programs and will give healthcare professionals opportunities to exchange scientific knowledge, review clinical protocols, and develop the skills needed to deliver cell and gene therapies safely and effectively.

The two organizations will also continue working on clinical research, including participation in clinical trials and the generation of real-world evidence.

These activities support objectives outlined in a 2024 memorandum of understanding involving the Ministry of Health, the Ministry of Investment of Saudi Arabia, and the Ministry of Industry and Mineral Resources.

The agreement also seeks to support the development of local scientific talent and strengthen Saudi Arabia’s clinical research capabilities.

Strengthening local expertise

H.E. Dr. Bandar Al Knawy, CEO of MNGHA, said the renewal marks two years since Saudi Arabia’s first gene therapy infusion and demonstrates progress in building advanced biotechnology capabilities.

He said the partnership supports the objectives of Saudi Vision 2030 and the National Biotechnology Strategy by combining patient care with clinical education, knowledge exchange, and healthcare workforce development.

Hisham Hagar, Executive Country Manager for Vertex Pharmaceuticals in the GCC region, said the partnership has expanded beyond treatment delivery to include clinical education, research, and real-world evidence generation.

He added that Vertex will continue working with MNGHA through initiatives such as the third Preceptorship program.

Dr. Mohsen AlZahrani, Assistant Professor and Consultant of Hematology and Stem Cell Transplantation at King Abdulaziz Medical City, MNGHA, said gene therapy requires close coordination among multidisciplinary teams.

He added that the collaboration has helped strengthen the expertise needed to deliver advanced gene therapies safely.

Dr. Mohammed Essa, Deputy Chairman and Section Head of Pediatric Hematology and Oncology and Pediatric Stem Cell Transplantation and Cellular Therapy at King Abdullah Specialist Children Hospital, MNGHA, said hereditary blood disorders place significant emotional, physical, and financial pressures on young patients and their families.

He noted that gene therapy can provide patients with an alternative to lifelong management of these conditions

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