International Fanconi Anaemia Gene Therapy Working Group

“a real platform for the accelerated development of collaborative clinical studies.”


Reported in Nature: Molecular Therapy “Stem Cell Gene Therapy for Fanconi Anemia: Report from the 1st International Fanconi Anemia Gene Therapy Working Group Meeting” : Jakub Tolar.

Download the full paper here

Report from First Meeting

November 16th 2010 saw the first meeting of an International Fanconi Anaemia Gene Therapy Working Group, organised and funded by the Fanconi Hope Charitable Trust working in association with the British  Society for Gene Therapy and the Fanconi Anemia Research Fund (FARF). This meeting, hosted by Prof Adrian Thrasher at the Institute of Child Health and ably chaired by Prof Jakub Tolar from the University of Minnesota, brought together 14 leading Gene Therapy and Fanconi Anaemia experts from across the globe to create an action plan for gene therapy trials in Fanconi Anaemia. The need to run trials collaboratively stems from the very rare nature of the condition, since individual countries have insufficient patient bases.

After many hours of productive and intellectually stimulating discussion on topics such as vector design, transduction, trial design, ethics, safety and toxicology and finally application processes and centre selection the attendees agreed that 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 can be shared.

What happens next is of course key to the success of the project. Formal minutes with details of the action plan will be circulated shortly by Prof Tolar and it was agreed within the meeting that notwithstanding interim actions, the Group should reconvene formally in 12 months time. The Fanconi Anemia Research Fund suggested that the next meeting of the group be organised to coincide with its 23rd Annual Scientific Symposium in Barcelona in Octoober 2011.

Prof Thrasher, Chairman of the BSGT said afterwards that the meeting was “a real platform for the accelerated development of collaborative clinical studies.”

Fanconi Hope would like to thank all the participants from the UK, Europe and the US for lending their support to this ground-breaking project, to Prof Thrasher for hosting and Prof Tolar for chairing the meeting and to Bev Mayhew the Executive Director of FARF for her strong support and FARF representation at the meeting.

Attendees at the meeting

(in alphabetical order)

Dr Michael Antoniou
Head of Nuclear Biology Group
King’s College London School of Medicine
Department of Medical and Molecular Genetics, Guy’s Hospital

Dr Cynthia Bartholomä
Division of Translational Oncology
National Center for Tumor Diseases , Heidelberg
German Cancer Research Center

Dr Juan Bueren
Head of the Hematopoietic Gene Therapy Division.
CIEMAT, Madrid

Prof Marina Cavazzana-Calvo
Hopital Necker-Enfants Malades, Biotherapy Department, Paris

Mr Thomas Carroll
Chairman – Fanconi Hope Charitable Trust,
Consultant Neurosurgeon
Lead Clinician, Department of Neurosurgery, Royal Hallamshire Hospital, Sheffield,

Mr Bob Dalgleish
Secretary, Fanconi Hope Charitable Trust

Dr Anne Galy
Head of Immunology and Gene Therapy Group,

Prof Helmut Hanenberg
Department of Pediatric
Hematology & Oncology,
Children’s Hospital,
Heinrich Heine University
and Riley Hospital for Children, Indianapolis

Prof Hans-Peter Kiem
Professor of Medicine / Adjunct Professor of Pathology
University of Washington School of Medicine,

Mrs Bev Mayhew
Executive Director
Fanconi Anemia Research Fund

Prof Luigi Naldini
San Raffaele Telethon Institute for
Gene Therapy, Milan

Dr Raffaele Renella
Fellow in Pediatric Hematology/Oncology
Children’s Hospital – Dana-Farber Cancer Institute
Harvard Medical School, Boston

Dr Paula Rio
Gene Therapy Researcher

Dr Julian Sevilla
Gene Therapy Researcher

Prof Adrian Thrasher
Consultant in Paediatric Immunology
Wellcome Trust Senior Clinical Fellow
NIHR Senior Investigator
Centre for Immunodeficiency
Molecular Immunology Unit
Institute of Child Health

Prof Jakub Tolar
Division of Hematology-Oncology and Blood and Marrow Transplantation
University of Minnesota Medical School

Prof Els Verhoeyen
Ecole Normale Supérieure de Lyon
Unité de Virologie Humaine

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