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I just read from Frank that the CF Foundation has removed the Gene Therapy from the Drug Pipeline. I looked at it 2 weeks ago before Abby's appt and it was still there. Very Sad news!! :'(
Hi Jenny,
After a rocky couples of years on the funding front, the UK Gene Therapy Consortium has just started a Phase 2 clinical trial of a gene therapy using a lipid vector. Work also continues to develop a "wave 2" product using a lenti-viral vector. The phase 2 trial for the "wave 1" product should complete in 2014. You can find more information about the phase 2 trial here: http://www.cfgenetherapy.org.uk/clinical/multidose.html
Hi Jenny,
The UK Gene Therapy Consortium is just starting a Phase 2 gene therapy trial using a liposome vector. The trial will complete in 2014. You can read more about it here.
Well it's about time. Haven't seen any progress on that front in years. I've thought for a while that the pipeline needed some updating. I mean according to the CFF, the new form of Tobi (TIP) is only approved in Canada and Chile.
Someone posted about this in the praying for success with VX809/770 facebook group and I mentioned that genetic therapy research is still occuring elsewhere in the world, in europe and in australia, possibly elsewhere. I've been following the UK group that maddiesfamily mentioned, I think that is one of the largest groups and also one of the first to do large human trials.
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