Just Gene Therapy is a not for profit organisation established to raise funds for gene therapy for cystic fibrosis.

Latest news

Wave 1

The Wave 1 clinical trial (CF gene delivered by liposomes (fat)) is going well. We were able to recruit all the patients needed (120) 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 three Safety Reviews by an independent external committee, with no problems noted. We have had extensive discussions with pharmaceutical companies with regards taking the product forward should the trial be successful. Although the 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.

Updates provided by Professor Eric Alton on behalf of the UK CF Gene Therapy Consortium, December 2013.





Wave 2

The 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 in a first human clinical study in early in 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.

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