New gene treatment can lower the risk of bleeding in people with hemophilia: Research

A study involving UCL researchers found that a single gene therapy injection can dramatically reduce the bleeding risk faced by people with haemophilia B.

For the paper, published in the New England Journal of Medicine, experts from UCL, Royal Free Hospital and biotechnology company Freeline Therapeutics trialled and continue to evaluate a new type of adeno-associated virus (AAV) gene therapy candidate, called FLT180a, to treat severe and moderately severe cases of the condition.