Recent Advances in CRISPR Gene Editing Technology in 2025 In 2025, CRISPR gene editing technology has moved from the realm of science fiction to become a real-world product, already delivering tangible benefits to patients around the globe. Below are brief overviews of key projects showcasing major successes across various medical fields. Casgevy – Treatment for Sickle Cell Disease and β-Thalassemia Casgevy represents a revolutionary treatment based on editing a patient’s own stem cells. More than 90% of patients experience no crises and do not require blood transfusions for at least one year after a single transplant. This drug has already been approved by the FDA and EMA, confirming its safety and efficacy. Casgevy opens a new era in the treatment of inherited blood disorders. NTLA-2002 – Fighting Hereditary Angioedema NTLA-2002 is an innovative therapy that significantly reduces the frequency of hereditary angioedema attacks. A single infusion reduces the number of attacks by approximately 95% over six months. The drug is currently in Phase II trials, transitioning into Phase III, which demonstrates its promising effectiveness and future potential. VERVE-102 – Cholesterol Reduction and Slowing Aging VERVE-102 offers a unique solution for lowering LDL cholesterol without the need for daily medication. A single infusion reduces cholesterol levels by 65% as early as day 28. This project also targets slowing aging processes, opening new horizons in preventive medicine. CRISPR-TIL – Immunotherapy for Colorectal Cancer CRISPR-TIL is an example of successful gene editing application in oncology. In a patient with colorectal cancer, edited T-cells completely eradicated tumors, and remission has lasted for two years. This marks a significant step toward personalized and highly effective cancer treatments. NTLA-2001 – Treatment for Transthyretin Amyloidosis NTLA-2001 shows a significant reduction in the pathological protein TTR—by more than 80%—after a single dose, with effects lasting up to 18 months. Patients demonstrate improved physical endurance, making this drug a promising tool in the fight against amyloidosis. Personalized CRISPR for CPS1 Deficiency A unique case of CRISPR use involved correcting a rare mutation in an infant with CPS1 deficiency. Ammonia levels normalized, and the child developed normally. This is an example of compassionate use—single-dose treatment that saved a patient’s life and health. EBT-101 – Breakthrough in HIV Treatment EBT-101 aims to remove HIV DNA from the body. After treatment, the virus remains undetectable for months without the need for antiretroviral drugs, paving the way for functional HIV cure strategies. HG202 – Therapy for Age-Related Macular Degeneration HG202 is administered directly into the eye and helps preserve vision in age-related macular degeneration. The drug entered Phase I/IIa clinical trials in 2025 and has already demonstrated potential in combating one of the leading causes of blindness in older adults. These projects illustrate how rapidly the field of gene editing is advancing and transforming medicine, making treatments more precise, effective, and accessible. In the coming years, CRISPR technologies promise to become an integral part of healthcare, improving the quality of life for millions worldwide.
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