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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Translational Pioneer Award 2017

New for 2017, Cell & Gene Therapy Insights and Phacilitate presented the Translational Pioneer award at the Cell and Gene Therapy Leaders Forum in Miami, USA. The award recognizes the exceptional contribution made by an individual scientist to the translation of cell and gene therapies into the clinic.
We received over 50 nominations for the award and with the help of our esteemed judging panel – Dr Maria Grazia Roncarolo, Dr Olivier Danos and Dr Joanne Kurtzberg we selected this year’s deserved winner.
We were delighted to present the inaugural Translational Pioneer Award 2017 to Prof. Carl June in recognition of his outstanding contribution to the field of immuno-oncology.
“Dr. June’s pioneering research led to the development of novel and effective immunotherapies for patients with leukemia. His work opened a new frontier for the treatment of hematological malignancies.” commented Chris Mason, Senior Editor.

Sponsoring the award provides a great opportunity to demonstrate your company’s full commitment to supporting translation of cell and gene therapies on a global basis. To discuss options for the 2018 Award, contact Nicola McCall at Cell & Gene Therapy Insights (, T: +44 (0)1732 463215)

Carl June


We are delighted to be working with a panel of world class experts in the field to help us select a worthy winner from our nominee shortlist.


OLIVIER DANOS, Senior Vice President, Gene Therapy, Biogen Idec, USA

Olivier is a pioneer in the field of gene therapy, and has dedicated his career to advancing the use of this technology to develop life-saving therapies for patients. Olivier joins Biogen Idec from Kadmon Pharmaceuticals, where he served as SVP, Molecular Medicine, Synthetic Biology and Gene Regulation since 2011. In this role, he was instrumental in assembling a gene therapy program and a technology platform for the development of controllable gene expression systems. Prior to Kadmon, Olivier acted as the director of the Gene Therapy Consortium of the University College of London and led a gene therapy research team at the Necker Hospital – Enfants Malades in Paris.
Olivier also served as chief scientific officer at Genethon and senior director of research at Somatix Therapy Corporation. He has held several senior roles at the French National Centre for Scientific Research(CNRS) and at the Institut Pasteur in Paris. Olivier is the former president and a founding member of the European Society of Gene and Cell Therapy.
Olivier received a Master’s in Genetics and Molecular Biology at University of Paris Orsay, and his Ph.D. in Biology at the Pasteur Institute and University of Paris Diderot.


DR JOANNE KURTZBERG, Director, Carolinas Cord Blood Bank, Duke University, USA

Dr Joanne Kurtzberg is an internationally renowned expert in pediatric hemtaolgoy/oncology, pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation and novel applications of cord blood in the emerging fields of cellular therapies and regenerative medicine. Dr Kurtzberg pioneered the use of umbilical cord blood as an alternative stem cell source for unrelated hematopoietic stem cell transplantation (HSCT). Over the last two decades she has established an internationally known pediatric transplant program at Duke University, which treats children with cancer, blood disorders, immune deficiencies, hemoglobinopathies and inherited metabolic diseases. In 2010 Joanne established the Julian Robertson Cell and Translational Therapy Program (CT2) at Duke. CT2 focuses on translational studies from bench to bedside with a focus on bringing cellular therpaies in regenerative medicine to the clinic. Recent areas of investigation in CT2 include the use of autologous cord blood in children with neonatal brain injury and cerebral palsy, as well as preclinical studies manufacturing oligodendrocyte-like cells from cord blood to treat patients with acquired and genetic brain diseases. Studies of autologous bone marrow ALDHbright cells in adults with stroke, radiation-induced brain injury are also underway. Plans are ongoing to extend this work to the use of autologous cord blood in the study of young children with autism specrum disorder.

Prof. Chris Mason, AvroBio

PROF. CHRIS MASON, Chief Science Officer, AVROBIO, USA

Prof. Chris Mason is a clinician-scientist with over 20 years of cell and gene therapy experience including Full Professor of Regenerative Medicine Bioprocessing at University College London. He has a multidisciplinary track record spanning R&D, clinical medicine, bioprocessing, regulation, healthcare economics, reimbursement and commercialization. He is co-founder of the London Regenerative Medicine Network, trustee of the UK Stem Cell Foundation and on the UK-Israel Science Council. Current SAB appointments include; public and private companies, the UK Cell Therapy Catapult and the Canadian Centre for the Commercialization of Regenerative Medicine. Prof. Mason is a general spokesperson for the cell and gene therapy sector including frequent newspaper, radio and TV interviews plus social media including @Prof_ChrisMason on Twitter. He is Senior Editor of Cell and Gene Therapy Insights

Maria Grazia Roncarolo

DR MARIA GRAZIA RONCAROLO, Co-director of the Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, USA

Dr. Roncarolo has made outstanding contributions to translational research in the fields of immunology and gene therapy. Her translational research studies have led to greater understanding of the mechanisms underlying immune tolerance and have been of paramount importance to the development of novel therapies for patients with genetic and acquired diseases of the hematopoietic and immune systems.

She contributed to the elucidation of the mechanisms underlying the induction and breaking of tolerance in bone marrow/organ transplantation/autoimmune diseases and gene therapy. Specifically, she discovered a new subset of human T regulatory cells with immune regulatory and suppressor functions, named Type 1 (Tr1) cells. She established that these cells were present in tolerant patients following hematopoietic stem cell transplantation. Subsequently, she isolated these cells from both mice and man, demonstrating that they are responsible for induction and maintenance of tolerance to allo- and self-antigens and food- and environmental- antigens. Recently, she discovered that the surface molecules CD49b and LAG3 are specific biomarkers for this subset of T regulatory cells, which allows for their isolation for therapeutic purposes and in vivo tracking in patients. She was the principal investigator of the first clinical trial using these ex-vivo generated donor-derived Tr1 cells to prevent the occurrence of severe graft-versus-host disease in leukemia patients.

Dr. Roncarolo was the principal investigator for the first lentiviral-vector based gene therapy trial for patients with Wiskott-Aldrich syndrome, which demonstrated safety and efficacy of this therapy. In addition, she was the principal investigator for the first successful gene therapy trial for Severe Combined Immunodeficiency (SCID) patients lacking adenosine deaminase (ADA), a purine metabolism disorder that results in severe immunodeficiency and death. In this trial she introduced a new therapeutic conditioning regimen for the host, which favored the outgrowth of the gene corrected cells, resulting in the most successful clinical outcome for this formerly non-treatable genetic disease. Based on these results, gene therapy for ADA-SCID has obtained Orphan drug status from both the FDA and EMEA and recently has received European Commission approval to market under the name of Strimvelis. This is the first stem cell gene therapy product, which received market authorization.

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