- Renewed collaboration and expanded clinical education initiatives will strengthen local expertise and support continued innovation in gene therapy.
- The collaboration has resulted in the initiation of treatment for a number of patients living with sickle cell disease and transfusion-dependent beta thalassemia.
The Ministry of National Guard Health Affairs (MNGHA) and Vertex Pharmaceuticals, a global biotechnology leader, today announced the renewal and expansion of their strategic partnership, which began following the administration of the first gene therapy in Saudi Arabia in 2024. The announcement was made during the Riyadh Global Medical Biotechnology Summit and reflects a shared commitment to improving patient care, strengthening clinical expertise, and advancing healthcare capabilities in the Kingdom through continued innovation and knowledge exchange.
Since receiving approval from the Saudi Food and Drug Authority (SFDA), this landmark collaboration has expanded access to the one-time gene therapy treatment and enabled the treatment of additional eligible patients living with sickle cell disease and transfusion-dependent beta thalassemia in Saudi Arabia.
As part of the renewed two-year partnership, MNGHA and Vertex announced plans to launch the third edition of their joint Global Advanced Gene Therapy Center Training Program, following the significant success of the previous two editions. The educational program aims to facilitate the exchange of scientific knowledge and clinical protocols and prepare the next generation of Saudi healthcare professionals to deliver advanced cell and gene therapies with the highest standards of safety and efficiency.
In addition to healthcare professional education, MNGHA and Vertex will continue to collaborate on clinical research initiatives, including participation in clinical trials and the generation of real-world evidence. These joint efforts contribute to the key objectives of the landmark Memorandum of Understanding signed in 2024 between the Ministry of Health, the Ministry of Investment, and the Ministry of Industry and Mineral Resources, supporting the development of local scientific expertise and strengthening clinical research capabilities in the Kingdom.
Commenting on the partnership, H.E. Dr. Bandar Al-Knawy, Chief Executive Officer of MNGHA, said:
“Marking two years since the administration of the first gene therapy in the Kingdom, alongside the renewal of our partnership with Vertex, represents a significant milestone in our journey. This summit provides an ideal platform to showcase the joint efforts of MNGHA and Vertex in translating the ambitious goals of Saudi Vision 2030 and the National Biotechnology Strategy into tangible outcomes that positively impact patients. Through the renewal of our partnership, we remain committed to maintaining the Kingdom’s position at the forefront of global biotechnology while ensuring sustainable, long-term access to innovations that make a meaningful difference in patients’ lives.”
Hisham Hajar, Regional Executive Director, Gulf Region, Vertex, said:
“Our partnership with MNGHA continues to evolve beyond treatment delivery to encompass clinical education, scientific knowledge exchange, participation in clinical trials, generation of real-world evidence, and initiatives that help strengthen local healthcare capabilities. We look forward to deepening our collaboration through initiatives such as the third edition of the joint Global Advanced Gene Therapy Center Training Program, contributing to further establishing the Kingdom as a leading hub for advanced therapies.”
Dr. Mohsen Al-Zahrani, Assistant Professor, Consultant in Hematology and Stem Cell Transplantation, and Head of the Adult Stem Cell Transplantation and Cellular Therapy Section at King Abdulaziz Medical City – MNGHA, Riyadh, said:
“Gene therapy represents one of the most complex clinical pathways in modern medicine, requiring seamless integration across multiple multidisciplinary teams. The continued development of the partnership between MNGHA and Vertex has contributed to strengthening multidisciplinary expertise and supporting the efficient and safe delivery of advanced gene therapies.”
Dr. Mohammed Issa, Deputy Head of Pediatric Hematology and Oncology and Head of Pediatric Stem Cell Transplantation and Cellular Therapy at King Abdullah Specialized Children’s Hospital – MNGHA, Riyadh, added:
“Inherited blood disorders place a significant psychological, physical, and financial burden on young patients and their families. With gene therapy, we are providing potentially curative medical care, helping children return to a more normal childhood and alleviating a lifelong burden on their families.”