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So far, those affected have mainly been treated with regular injections of coagulation factors or replacement substances. Gene therapy now enables a one-off treatment in which the defective gene is introduced into the liver cells with the help of harmless viruses. In future, patients will be able to produce the missing coagulation factor themselves. Studies have shown good long-term results, particularly in people with haemophilia B. The therapy requires close medical supervision, but offers the chance of a life without regular medication. With the authorisation, the UKL can also treat patients from other institutions - an important step for care throughout Saxony.
Press release Leipzig University Hospital from 11 March 2025
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