Unique Gene Therapy to Become Available for Patients in England.

The Ukrainian National Health Service of England has reached an agreement with Vertex Pharmaceuticals Inc. to make a new gene editing therapy available to patients. This technology, known as CRISPR, can be used to treat patients with a severe form of beta-thalassemia. Under the agreement, patients will be able to receive treatment through Casgevy infusion, which will make precise changes to their DNA and free them from the need for constant blood transfusions. This could bring hope to over 460 patients in England.

The drug usually costs $2.1 million, but it will now be available at a reduced price through the Innovative Medicines Fund. It is important to note that while studies have shown positive results, the long-term effect of the therapy still requires further study.

AstraZeneca Plc and other pharmaceutical companies have expressed concerns about the drug cost evaluation system in the UK. They are worried that it may limit patients' access to new drugs and reduce investments in the country.

This is a significant achievement in the treatment of genetic diseases and opens up new opportunities for patients who previously had no effective treatment options.