Scientific Advisory Board

Orchard's Scientific Advisory Board brings together the world's leading scientists and clinicians in gene therapy. Orchard is partnered with University College London; Great Ormond Street Hospital; the University of Manchester UK; Généthon France; the University of California Los Angeles and Boston Children's Hospital in the United States.

Alessandra Biffi

Alessandra Biffi

Alessandra Biffi

Director, Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Faculty of Pediatrics Harvard Medical School

Alessandra has been Principal Investigator for GSK's phase I/II clinical trial in MLD. Her research is dedicated at enhancing the efficacy of haematopoietic stem cell (HSC)-based therapeutic approaches for lysosomal storage diseases with severe nervous system involvement.

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Brian Bigger

Brian Bigger

Brian Bigger

Reader in Gene Therapy, Faculty of Medical and Human Sciences, University of Manchester

Brian developed the CD11b lentiviral vector for MPS IIIA. In 2006, he set up the Stem Cell & Neurotherapies laboratory at the University of Manchester to develop gene therapy and other treatments for lysosomal storage diseases such as MPS IIIA. He works closely with clinical colleagues Simon Jones and Rob Wynn at St Mary's Hospital and the Royal Manchester Children's Hospital to bring these treatments, and clinical outcome measures based on improved understanding of patients and lysosomal disease models, into medical practice.

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Bobby Gaspar

Bobby Gaspar

Bobby Gaspar

Professor of Paediatrics and Immunology; Consultant in Paediatric Immunology, UCL Institute of Child Health

Bobby is Professor of paediatrics and immunology at the UCL Institute of Child Health and Honorary Consultant in paediatric immunology at Great Ormond Street Hospital, London.

He has a special interest in the treatment of severe primary immune deficiencies, including bone marrow transplantation, gene and cell therapy. He has led multiple clinical trials that have shown that gene therapy can successfully correct the genetic defect in immune deficiencies.

Bobby is currently leading UK and European-wide initiatives for newborn screening in severe combined immune deficiency. He is Director of the Centre for Research in Rare Diseases in Children (CRRDC), a new academic and clinical facility that will open at Great Ormond Street in 2018.

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Simon Jones

Simon Jones

Simon Jones

Consultant in Paediatric Inborn Errors of Metabolism, Manchester Centre for Genomic Medicine, Honorary Senior Lecturer, University of Manchester

Simon acted as Principal Investigator and has been actively involved in many phase I–IV international multicentre trials of novel therapies for LSDs, including a phase III trial for MPS III with Genistein. He attended Edinburgh University Medical School and qualified with a BSc in Neurosciences.

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Donald B. Kohn

Donald B. Kohn

Donald B. Kohn

Professor, Departments of Microbiology, Immunology & Molecular Genetics; Pediatrics (Hematology/Oncology); and Molecular and Medical Pharmacology, University of California Los Angeles

Don is the Director of the Human Gene and Cell Therapy Program at UCLA. He has been an attending physician of paediatric bone marrow transplantation for more than 25 years and he has been a pioneer in the development of ex-vivo autologous gene therapies. He is also a member of the Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, as well as a former President of the American Society of Gene and Cell Therapy and the Clinical Immunology Society.

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Harry L. Malech

Harry L. Malech

Harry L. Malech

Chief, Genetic Immunotherapy Section and Deputy Chief, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National institutes of Health

Harry is an attending physician scientist involved in the care and study of children and adults with primary immune deficiencies for more than 35 years, with special focus on chronic granulomatous disease. He was one of the co-discoverers of the protein and genetic defects responsible for the p47phox deficient and p67phox deficient autosomal recessive forms of CGD and he conducted the earliest trials of gene therapy for CGD.  His program has pioneered allogeneic bone marrow transplant salvage therapy for CGD patients with life-threatening infections unresponsive to conventional therapy. He currently serves on the Medical Advisory Board of the Immune Deficiency Foundation, as a reviewer on Clinical Advisory Panels of the California Institute for Regenerative Medicine, and as member of the NIH Institutional Biosafety Committee which reviews all gene therapy protocols at the NIH Clinical Center. He is also a former President of the American Society of Gene and Cell Therapy.

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Adrian Thrasher

Adrian Thrasher

Adrian Thrasher

Wellcome Trust Principal Research Fellow, UCL Institute of Child Health; NIHR Emeritus Senior Investigator, Honorary Consultant Paediatric Immunologist, Great Ormond Street Hospital (GOSH) NHS Trust

Adrian is based at the UCL Institute of Child Health and GOSH. He has led multiple trials of gene therapy in monogenic bone marrow diseases including X-SCID, CGD, ADA-SCID and WAS, and has been co-investigator in gene therapy trials for retinal and metabolic disease. Adrian is a past president of the British Society of Gene and Cell Therapy, and has served on the board of the American and European societies. He has won several prestigious international awards for his work in clinical gene therapy.

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David Williams

David Williams

David Williams

President, Dana-Farber/Boston Children's Cancer and Blood Disorders Center; Chief, Division of Hematology/Oncology and Director of Clinical and Translational Research, Boston Children's Hospital; Associate Chair, Pediatric Oncology, Dana-Farber Cancer Institute; Leland Fikes Professor of Pediatrics, Harvard Medical School

Dr Williams was a Howard Hughes Medical Institute Investigator for 16 years and his laboratory has been continuously NIH funded since 1986. He has trained over 45 fellows and post-doctoral fellows and numerous residents and medical students in his laboratory, the majority of which are still in academic medicine. He is a member of the National Academy of Sciences (formerly the Institute of Medicine). Dr Williams was a Councilor for the American Society of Hematology (ASH) for four years, then served as President of ASH from 2014-2015. ASH is the world's largest professional society of haematologists and includes more than 15,000 haematologists who are committed to research and treatment of blood and blood-related diseases.

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Robert Wynn

Robert Wynn

Robert Wynn

Consultant Paediatric Haematologist, Director of Paediatric BMT Program, Royal Manchester Children's Hospital, Honorary Professor of Paediatric Haematology and Cell Therapy, University of Manchester, Department of Paediatric Haematology and BMT

Rob Wynn is Director of the Blood and Marrow Transplant Unit in the Royal Manchester Children's Hospital and Honorary Professor of Clinical Paediatric Haematology and Cellular Therapy. He grew up in Liverpool and undergraduate medical training was undertaken in Cambridge and The London, post graduate training in Newcastle and Edinburgh and Haematology and Transplant training in Manchester and Toronto. He is also an avid Liverpool fan!

Manchester has an interest and expertise in the transplant of children with metabolic disorders. The BMT Unit and its practice and development in this field have been enormously influenced by the late Professor Ed Wraith, a true pioneer in this field. The success of allogeneic cellular therapy in some disorders has recently led to a re-appraisal of gene therapy for currently treatment-refractory disorders.

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