International FA Gene Therapy Working Group – 3rd Meeting: 2012

The 3rd International Fanconi Anaemia Gene Therapy Working Group Meeting, co-sponsored and organised by Fanconi Hope and FARF, and chaired by Prof Jakub Tolar, was held on Oct. 25, 2012 in Versailles, France and included 19 representatives from the UK, US, France, Spain, Italy and Germany. The UK was represented by Prof Bobby Gaspar from the Molecular Immunology Unit at the Institute of Child Health in London. Also attending from the UK were representatives from Fanconi Hope.

Whereas the previous two meetings were about preparing the groundwork for coordinated clinical trials, this year’s meeting included reports of 3 imminent trials. Hans-Peter Kiem was able to report that his trial in Seattle was now starting. Juan Bueren from Madrid reported that a European-consortium-based trial was in the advanced stages of preparation, having secured an EU grant of 5M Euros. This multi-site trial, which includes Spain, France, Germany and the UK has now been press released as of 26/1/13. Helmut Hanenberg described preparations for trials in Indianapolis.

Fulvio Mavilio, the Scientific Director of Biotech firm Genethon, announced that a new lentivirus production method was being established that would lead to much larger quantities of lentiviruses and much lower cost.

Thomas Carroll presented on the results of a short survey of perceptions of gene therapy in FA-affected individuals & families. The survey questions were devised by Thomas Carroll and the questionnaire itself produced by FARF.

As with previous meetings it is expected that Prof Jakub Tolar, the Chairman, will publish a full report on the meeting in due course. His reports of the first two meetings were published in Nature:Molecular Therapy and Human Gene Therapy respectively. The 4th Annual Meeting has now been set for Sept 5th 2013 in Boston, USA. 

Full list of Attendees:



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