International FA Gene Therapy Working Group – 2nd Meeting: 2011

The Working Group Members

The 2nd Meeting of the International FA Gene Therapy Working Group was held at the FARF 23rd Annual Scientific Symposium in Barcelona on 20th October, 2011 with the meeting co-sponsored by Fanconi Hope, FARF, and the Spanish group, the Centre for Biomedical Network Research on Rare Diseases (CIBERER).

The meeting was again ably chaired by Prof Jakub Tolar of the University of Minnesota

Prof Jakub Tolar

Progress Statement for the International FA Gene Therapy Working Group

This has been formally written up in a paper published 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). Available here


With the support from the Fanconi Anemia Research Fund and Fanconi Hope Charitable Trust, two dozen scientists and clinicians convened in Barcelona on November 22, 2011, for the second meeting of the FA Gene Therapy International Work Group. This International Work Group has been established with the goal of coordinating the best available knowledge in gene therapy with the best format of clinical trial for Fanconi anemia.


The following is a synopsis of Professor Tolar’s summary of the proceedings of the meeting as presented at the conclusion of the Working Group Meeting and in the full FARF Research Symposium the following day. (Text approved by Prof Tolar)

The progress made through the activities of the International FA Gene Therapy Working Group has caused an acceleration in the movement from the concept to the reality of world scale collaborative clinical gene therapy trials for Fanconi Anaemia.

In the first meeting the best intellectuals & experts in FA were brought to the table to seek common ground in efforts to accelerate the transition from gene therapy research into clinical trials on FA patients.

Without collaborative efforts there will be flashes of brilliance with nothing practical coming out of them.  People have realised that nobody is smart enough to do this alone and there is significant, even exponential value in combining forces.

Through the agreements reached within the Working Group, all the links in the chain and all the building blocks were put in place and care was taken to ensure that this would produce interpretable readout to be shared amongst the community.

In this second meeting, the group were able to move this process forwards, demonstrating that the first coordinated GT trials will be starting in the very near future.

The group is now looking to the second generation of FA GT trials, ensuring that the necessary funding mechanisms are identified and in place.

For affected families, for whom gene therapy has held promise for the last 20 to 25 years but failed to deliver, we can now say that the gene therapy community is close to answering this with solid action, with a system that is not going to fail.

The collaborative activities of the International FA Gene Therapy Working Group have now moved us into a new era and the challenge to all is to move to something genuinely useful to the patient.

Attendee list:

Juan Bueren,  Spain

Pamela Becker,  US

D Wade Clapp, US

Thomas Carroll,  UK

Robert Dalgleish,  UK

Dave Frohnmayer,  US

Lynn Frohnmayer,  US

Eva Guinan, US

Helmut Hanenberg,   US

Hans-Peter Kiem,  US

Deane Marchbein,  US

Bev Mayhew, US,

Stephen Meyn  US

Susana Navarro, Spain

Peg Padden, US

Pankaj Qasba, US

Raffaele Renella, US

Paula Rio,  Spain

Manfred Schmidt, Germany

Jordi Surralles, Spain

Adrian Thrasher, UK

Jakub Tolar, US

Els Verhoeyen, France



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