The UK’s National Health Service (NHS) has made history by introducing the world’s first gene-editing therapy for sickle cell disease. Known as Crizanex-V, the therapy is designed to provide a potential cure for thousands of patients suffering from this debilitating condition.
The treatment was officially launched on February 10, 2025, at King’s College Hospital in London. Dr. Jennifer Allen, a leading hematologist overseeing the rollout, described the development as a “transformational leap in sickle cell care.” She explained that the therapy utilizes CRISPR-Cas9 technology to correct the genetic mutation responsible for the disease, effectively allowing patients to produce normal red blood cells.
“This is a once-in-a-lifetime breakthrough,” Dr. Allen said. “For patients who have endured painful crises, frequent hospitalizations, and organ damage, this offers a path to lasting relief.”
The therapy, priced at £1.65 million per patient, will initially be offered to around 100 eligible individuals annually under a specialized NHS program. Patients will undergo a single infusion of Crizanex-V, followed by close monitoring for six months.
One of the first recipients, 28-year-old Alisha Thomas from Birmingham, shared her hope: “I’ve lived with sickle cell my entire life. Knowing this could cure me feels like a miracle.”
The therapy has been developed by Vertex Pharmaceuticals in partnership with CRISPR Therapeutics. Clinical trials have shown that over 90% of participants achieved sustained remission, a groundbreaking outcome for a disease that has been historically managed rather than cured.
Professor David Roberts, an expert in hematology at Oxford University, emphasized the significance: “This therapy doesn’t just treat symptoms; it addresses the root cause of sickle cell disease. It’s a revolution in genetic medicine.”
The NHS has pledged to expand access to Crizanex-V across the UK by 2026, aiming to reduce the annual cost of managing sickle cell complications, which currently exceeds £100 million.
“This is a new dawn for sickle cell care,” said Amanda Pritchard, NHS Chief Executive. “The NHS is proud to lead the world in offering this life-changing therapy to those in need.”
This therapy marks a significant milestone in the fight against genetic diseases, opening doors to further advancements in personalized medicine.