International FA Gene Therapy Working Group Meetings 2011 onwards

Meeting  Date   Venue  Agenda Attendees   Details
 8  16 Nov 17 Heidelberg, Germany  tbc  tbc
 7  7 Oct 16 Ciemat, Madrid Agenda


Thanks to Dr Juan Bueren and his team in Ciemat for hosting the 7th Annual International Gene Therapy Working Group Meeting held on 7th October 2016 in Madrid.The Working Group brings together world experts in Fanconi Anaemia, Gene and Cell Therapy, Gene Editing, Academic Research and Vector and Cell Manufacture to accelerate the move from research into clinical trials for improved treatments for FA. Thanks also to Rocket Pharma for providing industry sponsorship for the event
 6  2 Sept 15 Guy’s Hospital, London
 5  16 Oct 14 Milan
FA gene therapy in Seattle – now and in 3 years Jennifer Adair
FA gene therapy in Madrid – now and in 3 years Juan Bueren
FA gene therapy in London – now and in 3 years Claire Booth
FA gene therapy in Indianapolis – now and in 3 years Helmut Hanenberg
Vector platforms – top models, real and imagined Fulvio Mavilio
Editing and recoding of FA genes – tools and carpenters Mark Osborn
Off-target effects – decision-making modules Manfred Schmidt
New envelopes – basic understanding or applied work? Els Verhoyen
Inflammation in FA – can we redirect and reshape it? Carlo Dufour
FA GT for HNSCC – seek and destroy a shape-shifting target Tim Osborn
Gene therapy in Hannover – now and in 3 years Christian Kratz
Gene therapy in Milan – now and in 3 years Luigi Naldini, Alessandra Biffi
How would FA patients and their families decide now (knowing what has been presented here today and knowing that they do not have the luxury of waiting till 2025 to see all the evidence) between gene therapy and bone marrow transplant? Bob Dalgleish, Thomas Carroll


  • Jakub Tolar – US/Minnesota (Working Group Chairman)Laura Hays – US/Oregon (FARF Executive Director)
  • Jennifer Adair – US/Seattle
  • Tim Osborn, – US/Boston
  • Juan Bueren – Spain/Madrid
  • Susana Navarro – Spain/Madrid
  • Paula Rio – Spain/Madrid
  • Julian Sevilla – Spain/Madrid
  • Claire Booth – UK/London, University College London Institute of Child Health
  • Helmut Hanenberg – US/Indianapolis & Germany
  • Mark Osborn – US/Minnesota
  • Manfred Schmidt – Germany
  • Els Verhoyen – France
  • Carlo Dufour – Italy
  • Christian Kratz – Germany
  • Luigi Naldini, – Italy
  • Alessandra Biffi – Italy
  • Thomas Carroll – UK (Fanconi Hope)
  • Bob Dalgleish, – UK (Fanconi Hope)
 4  5 Sep 2013 Boston, USA  Agenda The emphasis for the talks was on coordination among centres and consensus of the FA gene therapy field.  The presentation structure proposed in regards to each individual topic on the agenda was

  • Slide 1: What has been the challenge, historically?
  • Slide 2: How has it been approached and (at least in part) solved?
  • Slide 3: Where are we now?
  • Slide 4: How do we prepare for where we want to be in one year?
  • Slide 5: How do we prepare for where we want to be in 2020?
 3  25 Oct 2012 Versailles, France


Jakub Tolar, Chair, University of Minnesota, Minneapolis

Juan Bueren, CIEMAT, Madrid

Nathalie Cartier, National Institute of Health and Medical Research, Paris

Marina Cavazzana-Calvo, Hôpital Necker-Enfants Malades, Paris

Carlo Dufour, G. Gaslini Children’s Hospital, Genova

Bobby Gaspar, Institute of Child Health in London

Guillermo Guenechea, CIEMAT, Madrid

Helmut Hanenberg, Indiana University School of Medicine, Indianapolis

Zoltan Ivics, Max Delbrück Center for Molecular Biology, Berlin

Hans-Peter Kiem, Fred Hutchinson Cancer Research Center, Seattle

Fulvio Mavillo, Università degli Studi di Modena e Reggio Emilia, Moderna

Mark Osborn, University of Minnesota, Minneapolis

Paula Rio, CIEMAT, Madrid

Jose Segovia, CIEMAT, Madrid

Julian Sevilla, CIEMAT, Madrid

Els Verhoeyen, ENS de Lyon, Lyon

Christoff von Kalle, German Cancer Research Center, Heidelberg

Dave Frohnmayer, Fanconi Anemia Research Fund (virtual attendee)

Thomas Carroll, Fanconi Hope Charitable Trust

Bob Dalgleish, Fanconi Hope Charitable Trust

The meeting, again chaired by Prof Jakub Tolar, 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.

2  20 Nov 2011  Barcelona  



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

This meeting was co-sponsored by Fanconi Hope, FARF, and the Spanish group, the Centre for Biomedical Network Research on Rare Diseases (CIBERER). and again ably chaired by Prof Jakub Tolar of the University of Minnesota.

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



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