Transformational therapy cures bleeding disorder haemophilia B

pharmafile | July 21, 2022 | News story | Business Services  

Researchers have found that a single injection of a new gene therapy, called FLT180a, removed the need for people to inject themselves weekly with clotting factors.

The study was led by experts from UCL, the Royal Free Hospital in London, and biotechnology company Freeline Therapeutics. The medical team says that the majority of adults with haemophilia could be cured in the next three years.

Experts found that a single treatment with FLT180a led to sustained production of the protein from the liver in nine out of ten patients with severe or moderately severe haemophilia. This removed the need for their regular injections by correcting their genetic fault, and it has long-lasting effects.

Lead author Professor Pratima Chowdary, from UCL, said: “Removing the need for haemophilia patients to regularly inject themselves with the missing protein is an important step in improving their quality of life.

“The long-term follow up study will monitor the patients for durability of expression and surveillance for late effects.”

Patients on the trial had to take immune suppressing drugs over several weeks to several months, to prevent their immune systems from rejecting the therapy.

Freeline co-founder Professor Amit Nathwani, from UCL, who co-authored the study, said: “Gene therapy is still a young field that pushes the boundaries of science for people with severe genetic diseases.”

Nathwani commented that the new trial adds to “the growing body of evidence that gene therapy has the potential to free patients from the challenges of having to adhere to lifelong therapy or could provide treatment where none exists today.”

Clive Smith, chairman of the Haemophilia Society, said: “This initial data is promising, but we continue to monitor gene therapy trials closely and cautiously, as with all new treatments.

“If they are shown to be safe and effective, NICE and the NHS must work together to make these innovative treatments available.”

Lina Adams

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