International Fanconi Anaemia Gene Therapy Working Group

The International Fanconi Anaemia Gene Therapy Working Group, organised and funded jointly by the Fanconi Hope Charitable Trust and the Fanconi Anemia Research Fund (FARF) was set up to bring together leading Gene Therapy and Fanconi Anaemia experts from across the globe to create an action plan for gene therapy trials in Fanconi Anaemia. The group is chaired by Prof Jakub Tolar from the University of Minnesota.

The need to run trials collaboratively stems from the very rare nature of the condition, since individual countries have insufficient patient bases.

At the First International Fanconi Anaemia Gene Therapy Working Group Meeting , held in London on November 16th 2010 and  hosted by Prof Adrian Thrasher, Chairman of the British Society of Gene Therapy discussed topics such as vector design, transduction, trial design, ethics, safety and toxicology and finally application processes and centre selection. The attendees agreed  there was a basis for moving to an internationally coordinated set of gene therapy trials such that data from clinical trials set up under different regulatory systems in different countries could be shared. The meeting was reported in Nature: Molecular Therapy “Stem Cell Gene Therapy for Fanconi Anemia: Report for the 1st International Fanconi Anemia Gene Therapy Working Group Meeting”: Jakub Tolar

The second meeting, with increased attendance, was held in Barcelona on 24th Oct 2011. Prof Tolar, the Chairman concluded that the progress made through the activities of the International FA Gene Therapy Working Group had caused an acceleration in the movement from the concept to the reality of world scale collaborative clinical gene therapy trials for Fanconi Anaemia. This second meeting was reported in Human Gene Therapy in Feb 2012. Gene Therapy for Fanconi Anemia: One Step Closer to the Clinic – Human Gene Therapy, Volume 23, Issue 2, p.141-144 (2012). 

You can find more details of the 2nd and subsequent meetings here.

Read more about the current Gene Therapy activity for Fanconi Anaemia in a 2020 update on the FARF Website 

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