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VX 809 -gene therapy

My son is 6 year old and he have a cistic fibrose.- fenotype Delta F 508 in both allels. When we can to expect the gene therapy of product VX 809 in Europe as part of nesessery medicine therapy.
Thank you for this question.
VX809 is in early phase of study for safety checks. It is a small chemical molecule and not gene therapy based. The safety studies will take about 1 year and will, if they show no adverse effects, be followed by some phase II studies in people with CF. This will try to figure out if VX809 moves F508del to the cell membrane and can be activated by a second drg-VX-770. Gene therapy is also at this stage using a liposome (fatty globule) called GL-67 and the ‘normal CF gene’. If either or both work, the time frame for clinic availability is 5-7 years as the regulatory process for new drugs is very rigorous. I hope this helps as it is difficult to be precise about times.

Best wishes

Stuart Elborn
The answer is edited by: Prof Stuart Elborn