Speakers
2020 Key Speakers
Program Director, Office of Rare Diseases Research
The Advisory Board
We’re proud to work alongside a stellar line up of Orphan Drug and Gene Therapy Professionals who have helped us curate the most up to date and relevant program for you.
200 World Leading Speakers
2019 Speakers
Bringing together industry leaders from across the orphan drug and cell and gene therapy industries.
Annemieke Aartsma Rus, Professor of Translational Genetics, Department of Human Genetics, Leiden University Medical Center
Karen Aiach, Chief Executive Officer, Lysogene
Dennis Akkaya, Corporate Development, myTomorrows
Luca Alberici, Chief Business Officer, MolMed
Sofie Alverlind, Project Manager, Swedish Association of Local Authorities and Regions
Einar Andreassen, Senior Adviser, Norwegian Medicines Agency
Aris Angelis, Research Officer, Medical Technology Research Group, London School of Economics and Political Science
Diego Ardigo, R&D Rare Diseases Unit Head, CHIESI Farmaceutici Spa
Francis Arickx, Head of the Directorate Reimbursement of Medicines and Pharmaceutical Policy,, National Institute for Health and Disability Insurance RIZIV-INAMI
Dr Segolene Ayme, Founder of Orphanet & Rare Disease Expert in Residence, Inserm
Jasmin Barman, Scientific Advisor, SGP
Denis Belyakov, Executive Director, Union of Patients? And Patients? Organizations With Rare Diseases
Marian Bendik, Head of Gene Therapy, Process Development, Shire
Annika Bergman, Pricing and Market Access Europe, Sarepta International Holdings GmbH
Dr Michael Binks, VP, Rare Disease Clinical Research, Pfizer
Catalin Bobirca, Medical Franchise Head, Rare Diseases, Oncology Region Europe, Novartis
Mathieu Boudes, Coordinator of PARADIGM, European Patients' Forum
Dr Meriem Bouslouk, Orphan drug policy adviser for Germany
Meriem Bouslouk, Orphan drug policy adviser for Germany
Jacoline Bouvy, Senior Scientific Adviser, National Insitute for Health and Care Excellence
Sophia Brodin, Deputy Head of Department, Department of Value Based Pricing, The Dental and Pharmaceutical Benefits Agency T.L.V.
Nicholas Brooke, Executive Director, Patient Focused Medicines Development
Virginie Bros-Facer, Scientific Director, EURORDIS
Anna Bucsics, Project Advisor, Mechanism of Coordinated Access to orphan medicinal products (MoCA)
Dr Andreas Busch, Chief Scientific Officer, Head of Research and Development, Shire Pharmaceuticals
Clara Campàs, Partner, Asabys Partners
Agnese Cangini, Health Economist, AIFA
Nathalie Cartier, Director, Institut National De La sante Et De La Recherche Medicale
Fleur Chandler, Therapy Area Head of Respiratory Value Evidence and Outcomes, GlaxoSmithKline Biologicals
Maria Chatzou, Co-founder & Senior Advisor, Innovation Forum
James Christie, SVP Manufacturing and Supply Chain, MeiraGTx
Gabriel Cohn, Vice President Of Clinical Development Lead, Avrobio
Laura Cox, UK Country Lead and Business Development, myTomorrows
Emily Crossley, Co-founder, Duchenne U.K.
Avril Daly, CEO, Retina International
Tagle Danilo, Associate Director for Special Initiatives, National Center for Advancing Translational Sciences
Cecile De Coster, Director Regulatory Science, Bluebird Bio
Dr Sjef De Kimpe, Co-founder and SVP R&D, Auxesia Orion
Professor Michele De Luca, Scientist, Professor, University of Modena e Reggio Emilia
Laura De Rosa, Collaborator & Co-author, University of Modena e Reggio Emilia
Dr Renato Dellamano, President, Medical Marketing Economics
Lisa Dilworth, Vice President Rare and Orphan Diseases, Synteract
Scott Dorfman, President & CEO, Odylia Therapeutics
Cinzia Dorigo, Executive Director Clinical Development, Syneos Health
Dr Cynthia Dukes, Vice President, Drug Development, Rare Disease, ICON plc
Rudy Dupree, Senior Project Manager, EUnetHTA WP4, EUnetHTA
Jami Ellis, Executive Director of Commercial Development, Horizon Pharma
Erik Emerson, Executive Vice President, Commercial Operations, Mezzion Pharma
Van Zyl Engelbrecht, Clinical Project Director EU, UBC
Karen Facey, Senior Research Fellow, University of Edinburgh
Kelly Franchetti, RN, CCRN, CEN VP, Global Patient Insights & Engagement, Commercialisation & Outcomes, ICON plc
Pedro Franco, Director for Europe for Global Regulatory and Scientific Policy, Merck Serono
Michela Gabaldo, Head of Alliance Management, Fondazione Telethon
Benjamin Gannon, Head of International Government Affairs, Public Policy & Patient Engagement, Vertex Pharmaceuticals
Dr Caridad Pontes García, Pharmacotherapeutic Harmonization, Catalan Health Service
Pamela Gavin, Chief Strategy Officer, National Organization for Rare Disorders
Josie Godfrey, Director, JG Zebra Consulting
Rory Graham, BPharm, MBA Senior Director, Regulatory Affairs, Europe & International Region, ICON plc
Dr Tim Guilliams, CEO, Healx
Dr Femida Gwadry-Sridhar, Founder and CEO, Pulse Infoframe Inc
Denis Horgan, Executive Director, EAPM (European Alliance for Personalised Medicine)
Annie Hubert, Director, European Public Policy, Alliance for Regenerative Medicine
Lynne Hughes, Vice President & Head, Centre of Excellence, Neurology, Therapeutic & Speciality Business Development, IQVIA
Wills Hughes-Wilson, Head of Patient Access & Commercial Planning, Mereo BioPharma
Adam Hutchings, Managing Director, Dolon
Dr Carlo Incerti, SVP, Head of Global Medical Affairs, CMO, Genzyme, A Sanofi Company
Edmund Jessop, Medical Adviser, National Health Service
Bodil Jonason, Vice President, Marketing and Sales, Inceptua
M Ken Kengatharan, Managing Partner, Atheneos Ventures and CEO, Auxesia Orion
Alastair Kent Obe, Former Director, Genetic Alliance UK, Retired
Diane Kleinermans, Advisor to the Ministry of Health and Social Affairs, Belgian Federal Government
Nicolas Koebel, Head of Program & Portfolio Management, Orchard Therapeutics
Maryna Kolochavina, Project Director, Real World & Late Phase, Syneos Health
Daan Kranenburg, VP Commercial Operations Europe, bluebird bio
Jennifer Lambe, Senior Project Manager, ICON plc
Dr Greg Larosa, Senior Vice President & Head of Scientific Strategy, Rare Disease RU, Pfizer
Yann Le Cam, Chief Executive Officer, EURORDIS
Bastiaan Leewis, MSAT Manager Industrialization, MeiraGTx
Sune Lindgaard, Chief of Business Intelligence, Amgros, Denmark
Yolanda Ludena, Foundation twenty-nine
Alastair Mackinnon, CMO, Mereo BioPharma
Dr William Maier, PhD, MPH Chief Scientific Officer & Head, Rare Disease Research, Commercialisation and Outcomes, ICON plc
Marc Martinell, CEO, Minoryx Therapeutics
Camille Metais, Senior Regulatory Affairs Director, Alexion Pharma LLC
Ana Mingorance, Chief Development Officer, Loulou Foundation
Eva Molero, CEO, Synapse Research Management Partners
Nathalie Moll, Director General, EFPIA
Arlete Monteiro, IT Director, Ministry Of Health Of Portugal
Dr Marisol Montolio Del Olmo, Scientific Director, Duchenne Parent Project Spain
Dmitry Mordvintsev, Head of Specialty Care BU, Genzyme
Dirk Moritz, Principal, Blue Matter Consulting
Dr William Motel, Regulatory Affairs Director, IQVIA
Karl-Johan Myren, Head of Patient Access, Alexion Pharmaceuticals
Dr Alexander Natz, Secretary General, EUCOPE
Daniel O'Connor, Medical Assessor, Medicines and Healthcare Products Regulatory Agency
Sam Oliver, Joint Managing Director, pH Associates
Tuyen Ong, Chief Development Officer, Nightstar Therapeutics
Dr Emanuele Ostuni, Head of Europe, Cell and Gene Therapy, Novartis
Romina Oxborough, Director of Clinigen Consulting, Clinigen Group
Dr Pan Pantziarka, Program Director, Anticancer Fund
Samantha Parker, Chief Patient Access Officer, Lysogene
Andrew Parker, Executive Vice President and Chief Science Officer, Zealand Pharma
Doug Paul, partner, Medical Marketing Economics
Tapani Piha, Head Of Unit, European Commission
Sarah Pitluck, Head, Global Pricing & Reimbursement, Spark Therapeutics
Silvia Ragno, Senior Director External Asset Lead, Takeda
Dr Sam Rasty, Chief Operating Officer, Homology Medicines
Kirsty Reid, Senior Manager Science Policy and Animal Welfare, EFPIA
Owen Roberts, Chief Executive Officer, M4K Pharma
Dr Paul Robinson, Patient Engagement Lead, Europe, Merck Sharp and Dohme
Angi Robinson, Executive Director, Strategic Development, Rare Disease & Pediatrics, Premier Research
Gregory Robinson, Chief Scientific Officer, Nightstar Therapeutics
Dr Jerome Rossert, Vice President, General Medicine, Global Clinical Development, Vertex Pharmaceuticals
Delphine Roulland, Senior Manager Public Affairs chez, EUCOPE
Jude Samulski, Director, Professor of Pharmacology, University of North Carolina at Chapel Hill, Gene Therapy Center
Luca Sangiori, Head of Medical Genetics and Rare Orthopedic Diseases, Rizzoli Orthopaedic Institute
Reto Schaberl, Head Business Development, Ewopharma AG
Scott Schliebner, Vice President, Scientific Affairs, Rare Diseases, PRA Health Sciences
Gabriele Schlosser-Weber, Clinical assessor, Bfarm
Dr Yvonne Schmidt, Scientific Advisor, Pharmaceuticals Department, G-BA
Sophie Schmitz, Managing Partner, Partners4Access
Ad Schuurman, Senior Advisor International Affairs, National Health Care Institute (ZIN), the Netherlands
Raghuram Selvaraju, Managing Director, Senior Analyst, Healthcare, Rodman & Renshaw
Daniela Settesoldi, Pharmaceutical Expenditure, Expenditure monitoring, AIFA
Nick Sireau, Chairman, AKU Society
David Sourdive, Executive Vice President Technical Operations, Cellectis
Marcio Souza, Chief Operating Officer, PTC Therapeutics
Leo Strican, Head of Access Value & Evidence Strategy, Europe, Bluebird Bio
Rick Thompson, CEO, Findacure
Dr Josep Torrent-Farnell, Medicines Department, Catalan Health Service
Marie Trad, Executive Medical Director, IQVIA
Dr Giuliana Vallanti, Director Of Development, Quality Control And Qualified Person, Molmed SpA
Inneke Van De Vijver, Advisor, N.I.H.D.I.
Dr Sander Van Deventer, Chief Scientific Officer And General Manager, uniQure
Dr Andreas Vogel, Head of European Regulatory, Bluebird Bio
Till Voigtlander, Vice-President of the Board of Members States on ERNs, Medical University of Vienna
Dr Birgitte Volck, Senior Vice President, Head of R&D Rare Diseases, GlaxoSmithKline
Lucia Von Bredow, Public Affairs & Patient Policy Manager, Clinigen Group
Misha Votruba, Director, Gradus R.S.J. Life Sciences
Darren Walsh, Senior Director - Market Access & Govt. Affairs – EU, Orchard Therapeutics
Dr Mary Wang, Global Partnerships & Project Manager, Fondazione Telethon
Dr Richard Wenzel, Principal, Charles River Associates
Magdalena Wladysiuk, Owner And Vice President, HTA Consulting
Pawel Wozniak, Managing Director, Komtur Polska
Entela Xoxi, Former AIFA, Universita Cattolica Del Sacro Cuore
Stefano Zancan, Head Clinical Development and Operations, Fondazione Telethon
Martine Zimmermann, Senior Vice President, Head of Global Regulatory Affairs, Alexion Pharmaceuticals
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