January 20, 2014

UK Gene Therapy Update

The following information was recently sent to people involved with the UK Cystic Fibrosis Gene Therapy Consortium:

“The Wave 1 Clinical Trial (CF Gene delivered by liposomes (fat)) is going well. We were able to recruit all the patients needed from CF Centres across the UK. Approximately half the patients have received all the 12 doses of gene therapy or placebo given at monthly intervals. The remaining will be completing their dosing over the next few months,with the final dose in the final patient due around June. There will then be a period of data analysis, and we hope to present the findings at the North American CF Conference in October. There have been 3 safety reviews by an independent external committee, with noted. We have had extensive discussions with pharmaceutical companies with regards taking the product forward should the trial be successful.

Although wave 1 trial is very encouraging, whilst it has been in development, other promising new delivery methods have been considered. This has led to the development of a Wave 2 product, which seems to be a more effective way of delivering the correct gene.

This Wave 2 product (CF gene delivered by a virus) is now progressing rapidly. We have chosen the final form of the virus that we will take into CF Patients, have learnt how to make the large quantities that will be needed for the first clinical trial, and have undertaken preliminary safety studies which have not shown any unexpected problems. Following discussion with the regulatory agencies we are now embarking on a formal 16 month toxicology programme and aim to be the first human clinical study in early 2017. Again there has been considerable interest from pharmaceutical companies in taking this product forward, not least because the initial research has been so promising” – Professor Eric Alton, UK Cystic Fibrosis Gene Therapy Consortium

Further info is available here: http://justgenetherapy.org/cf/gene-therapy.htm


Join the conversation! 4 Comments

  1. http://justgenetherapy.org/ This site states “The Wave 2 product, which is already in the pipeline, has been demonstrated to be approximately 500 times more effective, so it is essential that this product is developed”

    Hopefully we get some good news flow from here but 2017 to start trials means we are a long way away from it being available if ultimately successful ……fingers crossed

    • Thanks for adding that link Vijay, hopefully gene therapy may help those with CF in 5-10 years, but at least in the meantime CFTR modulators and potentially other approaches are likely to help many people with CF

  2. Could you please tell me how long the trial for df508 and df508 might take thank you, cf adult/diabetic. Angela


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2. Gene Therapy