Just Gene Therapy has been established by Rosie Barnes in conjunction with Professor Eric Alton and the UK CF Gene Therapy Consortium (see www.cfgenetherapy.org.uk) to raise funds for the continuation of the gene therapy research programme for cystic fibrosis (CF).
There has been considerable research into gene therapy for CF since the faulty gene which causes CF was identified in 1989. It has now reached the stage where a potentially effective product is in a double blind randomised multi-dose trial. Other gene therapy products are almost certain to be needed, to improve efficacy and to benefit more patients.
Results of the Wave 1 clinical trial (CF gene delivered by liposomes (fat)) have now been published in The Lancet. Gene therapy was shown to give a significant, if modest, benefit to patients. More details, incuding a link to the article, can be found on the Consortium's website.
“The Wave 2 product (CF gene delivered by a virus) is now progressing rapidly. … 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.”
—Professor Eric Alton, UK CF Gene Therapy Consortium
For more information, see the UK CF Gene Therapy Consortium site page.
To ensure more progress can be made, further funding will be needed to take Wave 2 through to clinical trials.
There are several ways to give to Just Gene Therapy, including donating online. Donations of any size are welcome, from individuals, charitable trusts or companies.
You can also make your own JustGiving page for online sponsorship (ensure you mention Just Gene Therapy).
Giorgia Arnold is cycling 365 miles at an altitude of 4,500m in aid of Just Gene Therapy. Find out more on our Events page.
A meeting is being arranged for the autumn, when Professor Alton will give updates on the results of the Wave 1 trial, along with the progress and future of Wave 2. Please check back here for more details in the next few weeks.
Previous events include the launch in June 2012 at the Stone House, St John’s, South East London, and talks by Professor Alton in September and November (watch the video in our Events section).