The publication suggests good results for the treatment of severe cases.
Researchers said eliminating the need for hemophilia patients to regularly inject their missing proteins is an important step in improving their quality of life. Photo: Shutterstock.
This is a gene therapy injection that can significantly reduce the risk of bleeding faced by people with a type of hemophilia B.
The researchers indicated that this is a clinical trial conducted on 10 patients, the results of which have been published in The New England Journal of Medicine, including researchers from the University College of London-UCL, the Royal Free Hospital and the Biotechnology Company. Freeline Therapeutics tested a new type Candidate for gene therapy with an adeno-associated virus (AAV) called FLT180A for the treatment of severe and moderately severe cases of hemophilia B.
The medical literature defines hemophilia B have a bleeding disorder Rare and inherited genes causing low levels factor IX protein (fix), needed for blood clotting Those that help stop or stop bleeding.
Scientists show that the gene responsible for this fix protein production is located on the X chromosome, so severe form of hemophilia B is much more common in men.
Currently, patients with hemophilia B must be routinely injected – usually weekly – with recombinant FIX, that is, routine replacement therapy to prevent excessive bleeding. Despite advances in treatment, patients may continue to experience joint damage.
This multicentre phase I/II clinical trial, called B-AMAZE, and a related long-term follow-up study found that single treatment with FLT180a resulted in sustained production of FIX protein from the liver in nine out of ten patients. Four different dosage levels, eliminating the need for regular replacement therapy.
17 male patients 18 years of age and older were screened, 10 of whom with severe or moderately severe hemophilia B participated in the 26-week FLT180a trial. All of them are also enrolled in a long-term follow-up study to assess the safety and durability of FIX expression for 15 years.
“Eliminating the need for hemophilia patients to regularly inject their missing proteins is an important step in improving their quality of life,” said lead author Pratima Choudhary.
AAV gene therapy works by using an envelope of proteins found on the virus’s outer covering, to deliver a working copy of a gene directly to the patient’s tissue to prevent it from working properly. Newly synthesized proteins are released into the blood, and a single infusion can achieve long-lasting effects.
The patients had to take immunosuppressive drugs for several weeks or months to prevent their immune systems from rejecting the therapy, and all reported known side effects.
Source consulted here.