KFSHRC Achieves Breakthrough in Gene Therapy for Hemophilia A with One-Time Treatment Success
Key Insights
King Faisal Specialist Hospital & Research Centre successfully treated eight hemophilia A (search) patients with one-time gene therapy (search), eliminating the need for preventive treatment and significantly improving quality of life.
The hospital became the first in Saudi Arabia to locally produce CAR-T cells in 2024, marking a major step toward national self-sufficiency in advanced cancer therapies.
KFSHRC now conducts 52.4% of all active clinical trials nationwide and recorded 701 active research proposals by the end of 2024, representing an 8% increase over the previous year.
King Faisal Specialist Hospital & Research Centre (KFSHRC) has achieved a significant breakthrough in hemophilia care, successfully using one-time gene therapy (search) to restore clotting function in eight patients with hemophilia A (search). This landmark treatment eliminated the need for preventive therapy and significantly improved patients' quality of life, marking a major advancement in the treatment of this rare bleeding disorder.
The breakthrough represents part of KFSHRC's broader expansion into gene therapy (search) and advanced therapeutics. In 2024, the hospital became the first institution in Saudi Arabia to locally produce CAR-T cells, a critical step toward achieving national self-sufficiency in advanced cancer therapies and reducing treatment costs.
Leading Clinical Research in Saudi Arabia
KFSHRC has established itself as the Kingdom's premier clinical trials hub, conducting 52.4% of all active clinical trials nationwide according to the Saudi National Institute of Health. By the end of 2024, the hospital recorded 701 active research proposals, representing an 8% increase over the previous year. In Riyadh alone, research approvals rose by 20%, demonstrating growing alignment between scientific discovery and clinical care delivery.
This growth reflects the sustained success of KFSHRC's Clinical Trials Transformation Initiative, launched in 2021 to build an integrated, patient-centered research ecosystem. The initiative has positioned the hospital as a key driver of Saudi Arabia's biotechnology transformation under Vision 2030.
Revolutionary Cancer Treatment Outcomes
KFSHRC's Oncology Centre of Excellence has demonstrated exceptional clinical outcomes, treating 25% of all cancer cases in Saudi Arabia in 2023. The center achieved an overall recovery rate of 50% and an outstanding 90% recovery rate for leukemia (search) patients.
As one of the few global centers offering CAR-T cell therapy (search), KFSHRC has transformed blood cancer treatment by reprogramming patients' immune cells. Since 2021, over 100 patients have been treated with this innovative therapy, including the hospital's first case involving a 13-year-old leukemia (search) patient. In 2023, 59 CAR-T cell therapies were administered, representing a 62% increase from the previous year.
Pediatric Oncology Excellence
The hospital's pediatric oncology program, based at the King Fahd National Center for Children's Cancer (search) (KFNCCC), has achieved remarkable success with a 92% recovery rate for acute lymphoblastic leukemia (search) (ALL). KFSHRC has completed over 3,000 pediatric stem cell transplants and achieved a 97% five-year survival rate for pediatric renal tumors.
These achievements have enabled the localization of critical treatments, reducing the need for patients to seek treatment abroad and making advanced therapies more accessible to Saudi families.
Advanced Genomic Medicine and CRISPR Technology
KFSHRC is advancing genomic medicine through CRISPR-Cas9 gene editing and personalized cancer therapies. The hospital has developed the Kingdom's first genetically engineered mice for rare disease research, supporting its commitment to advancing precision medicine.
Gene therapy (search) trials for hemophilia A (search) and paroxysmal nocturnal hemoglobinuria (search) (PNH) have demonstrated improved outcomes while reducing treatment costs, showcasing the potential for gene therapy to transform care for rare diseases.
Alignment with National Vision
These advancements align closely with Saudi Arabia's Vision 2030 and National Biotechnology Strategy, which designate biotechnology as a national priority sector. The strategy aims to localize therapeutic development, accelerate innovation, and establish global leadership in biotechnology by 2040.
KFSHRC has also invested in public engagement, launching awareness campaigns in high-traffic public venues to promote clinical trials literacy and reduce misconceptions around participation, supporting broader adoption of innovative treatments.
The hospital's achievements have earned international recognition, with KFSHRC ranked first in the Middle East and Africa and 15th globally among the world's top 250 Academic Medical Centres for the third consecutive year. It has also been recognized as the most valuable healthcare brand in the Kingdom and the Middle East according to the 2025 Brand Finance rankings.