NHS to Offer Groundbreaking Gene Therapy for Sickle Cell Disease

Quibim Secures $50M Series A to Advance AI-Powered Imaging for Precision Medicine

Apollo Hospitals Unveils Advanced Oncology Facility in Chennai

Beckman Coulter’s Alzheimer’s Blood Test Receives FDA Breakthrough Device Designation

ANGLE Announces Breakthrough Dual CTC-DNA and ctDNA Analysis with Illumina’s NGS Solution

Diatech Pharmacogenetics and Merck Expand Collaboration to Enhance RAS Biomarker Testing in MEA Region

DCG Unveils AI-Powered Video-Reporting for Personalized Chemotherapy Selection

FDA Approves Monthly Maintenance Dosing for LEQEMBI® in Alzheimer’s Treatment

FDA Approves Enhertu for HR-Positive, HER2-Low and HER2-Ultralow Metastatic Breast Cancer

CrisprBits Unveils State-of-the-Art CRISPR Gene Editing Lab in Bengaluru

Takara Bio Acquires Curio Bioscience, Advancing Leadership in Spatial Genomics

Tempus AI Launches FDA-Approved xT CDx for Comprehensive Solid Tumor Profiling

FDA Accepts Eisai’s Biologics License Application for LEQEMBI® Subcutaneous Autoinjector for Alzheimer’s Disease

Paige’s FullFocus™ Digital Pathology Viewer Receives FDA 510(k) Clearance for Expanded Compatibility

Grifols, MJFF Launch Chronos-PD to Identify Early Parkinson’s Biomarkers

Home
/
update
/
details

NHS to Offer Groundbreaking Gene Therapy for Sickle Cell Disease

NextEdge Admin

31 Jan 2025

1 min 53 sec

Vertex Pharmaceuticals,CRISPR Therapeutics,Sickle cell disease

Britain’s National Health Service (NHS) will offer a groundbreaking gene therapy to treat sickle cell disease, as announced by the National Institute for Health and Care Excellence (NICE) on Friday. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, the treatment costs approximately £1.65 million ($2.1 million) per patient.

The therapy was approved by Britain’s Medicines and Healthcare products Regulatory Agency (MHRA) in 2023, making it the first regulator in the world to authorize its use. NICE, responsible for evaluating medical technologies for NHS adoption, has determined that around 50 patients per year will be eligible for the treatment.

Sickle cell disease is a lifelong and severe genetic disorder caused by mutations in the haemoglobin-producing genes, leading to abnormal red blood cells that hinder oxygen transport throughout the body. The new therapy utilizes CRISPR gene-editing technology to modify stem cells extracted from a patient’s bone marrow. These edited cells are then reintroduced into the patient to correct the genetic defect.

A similar gene-editing treatment was approved for NHS use in August to treat beta thalassemia, another rare blood disorder. This advancement marks a significant step forward in precision medicine and gene therapy.