2020 Speakers
World Orphan Drug Congress USA will feature 350+ speakers from pharma, biotechs, government, regulators, patient groups & service providers.
Rebecca Abbott, Office Of Government Affairs, March of Dimes
Thomas Abbott, Head, Real World Data and Evidence, Astellas Pharma
Derek Adams, Chief Technology And Manufacturing Officer, bluebird bio
Sonalee Agarwal, Vice President Of Value And Evidence Strategy, Alnylam Pharmaceuticals
Jeonghoon Ahn, Director, National Evidence-based Healthcare Collaborating Agency
Kristin Anthony, President, Pten Hamartoma Tumor Syndrome Foundation
Anjan Aralihalli, Venture Partner, C.T.I. life Sciences Fund
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
Rasappa Arumugham, Chief Scientific Officer, Ocugen
Rania Ashraf, Associate Director Market Access, Janssen
Kagan Atikeler, Researcher, Turkish Medicines and Medical Devices Agency
Christopher Austin, Director, National Center For Advancing Translational Sciences (Ncats), National Institutes of Health (NIH)
Mark Baglin, Vice President Of Global Marketing, Alnylam Pharmaceuticals
Lakiea Bailey, Executive Director, Sickle Cell Consortium
Annette Bakker, President, Childrens Tumor Foundation
Ron Bartek, President, Director, And Founder, Friedreich's Ataxia Research Alliance
Shannon Baumann, Senior Director, Access Value And Evidence Strategy, bluebird bio
Allyson Berent, Chief Operating Officer, Genetx Biotherapeutics, Chief Science Officer, Foundation for Angelman Syndrome Therapeutics
Michelle Berg, President, Gmp Nucleic Acids, Aldevron
Diane Berry, Vice President, Global Health Policy And Government Affair, Sarepta Therapeutics
Luc Boileau, Chairman And Chief Executive Officer, INESSS
Vanessa Boulanger, Director Of Research Programs, NORD
Paula Boultbee, Chief Commercial Officer, Oncopeptides
Alfred Boyle, Senior Vice President Technical Operations, Alnylam Pharmaceuticals
Michael Bozik, President And Chief Executive Officer, Knopp Biosciences
Philip J Brooks, Program Director, National Center For Advancing Translational Sciences - Ncats, National Institutes of Health (NIH)
Michael Brudno, Scientific Director Of Centre For Computational Medicine, Hospital for Sick Children
Wilson Bryan, Director, Office Of Tissues And Advanced Therapies, Cber, FDA
Chuck Bucklar, Group Vice President, Commercial Operations North America, BioMarin Pharmaceutical Inc.
Anna Bucsics, Project Advisor, Mechanism of Coordinated Access to orphan medicinal products (MoCA)
Catherine Cancian, Vice President, Pharmaceutical Operations, GenSight Biologics
Casey Case, Senior Vice President Of Research And Development, 4D Molecular Therapeutics
Chelsea Catsburg, Engagement Manager, BluePrint Orphan
Rick Chapman, Director Of Health Economics, ICER
Lynsey Chediak, Project Lead, Precision Medicine, World Economic Forum
Alexander Cole, Executive Director, Global Head Epidemiology, Alexion Pharmaceuticals
Ellen Coleman, President And Chief Executive Officer, VOZ Advisors
Brett Cook, Senior Associate, F Prime Capital
Gavin Corcoran, Chief Research And Development Officer, Axovant Sciences
Benoit Coulombe, Director of Translational Proteomics at the Montreal Clinical Research Institute; Professor, University of Montreal; Founder,, Open for Rare
Anne Cropp, Chief Scientific Officer, Early Access Care
Pam Crowley Andrews, Co-Founder And Executive Director, Firefly Fund
Daniel Curran, Head, Rare Diseases Therapeutic Area Unit, Takeda
Omar Dabbous, Vice President, Health Economics And Outcomes Research, AveXis
Yong Dai, Chief Technology Officer, Prevail Therapeutics
Olivier Danos, Chief Scientific Officer, Regenxbio Inc
Santanu Das, MD, Huron Consulting Group
Khrystal K Davis, President, Texas Rare Alliance
Tom Defay, Senior Director, Research And Development, Strategy And Alliance Management, Alexion Pharmaceuticals
Ebrahim S Delpassand, Chairman And Chief Executive Officer, RadioMedix Inc.
Eelko Den Breejen, Associate Director, Market Access, Biogen
Lisa Dilworth, Vice President, Rare Orphan and Pediatric Diseases, Synteract
Scott Dorfman, Chief Executive Officer, Odylia Therapeutics
Debbie Drell, Director Of Membership, National Organization for Rare Disorders (NORD)
Andrew Ecob, Vice President Of Real World Evidence Strategy, PAREXEL
Sean Ekins, Founder And Chief Executive Officer, Collobrations Pharmaceuticals
Rafael Escandon, Vice President Of Development Operations, BridgeBio
Mark Farmery, Vice President Of Business Development, Neurovive Pharmaceutical
Lisa Feng, Senior Director, Policy And Advocacy, Cystic Fibrosis Foundation
Vivian Fernandez, Director Of Patient Advocacy, Regenxbio Inc
Emily Fields, Senior Manager, Patient Advocacy, bluebird bio
Amanda Finlayson, Therapeutic Strategy Lead, ClinEdge
Kelly Folkers, Senior Research Associate, Division Of Medical Ethics,, NYU Langone Medical Center
Chad Forinash, Senior Director, Pharmacy And Clinical Services, PharmaCord
Romina Foster-Bonds, Programs Director, CureDuchenne
Danielle Friend, Director Of Science And Regulatory Affairs, Biotechnology Industry Organization
Jon Fryzek, Practice Director, Epidemiology, EpidStrategies, A Division of ToxStrategies
Andrea Furia-Helms, Director, Patient Affairs Staff, U.S. Food and Drug Administration (FDA)
Pat Furlong, Founding President And Chief Executive Officer, Parent Project Muscular Dystrophy
Kamilla Gaitova, Head Of Center For Health Economics And Health Technology Assessment, Republican Center for Health Development, Kazakhstan
Mark Galbraith, Head Of Quality Control And Analytical Sciences, Spark Therapeutics
Pamela Gavin, Chief Strategy Officer, National Organization for Rare Disorders (NORD)
Barbara Goodman, President And Chief Operating Officer, Cures Within Reach
Joana Granado, External Innovation Director, Actelion - A Janssen Pharmaceutical Company of Johnson&Johnson
Amy Gray, Chief Executive Officer, Charcot-Marie-Tooth Association
Scott Gray, Chief Executive Officer, Clincierge
Bernard Grimm, Director, EuropaBio
Mike Hale, Vice President And Chief Statistical Scientist, Takeda Pharmaceuticals
Marianne Hamilton Lopez, Research Director, Duke Margolis Center for Health Policy
Patrick Haslett, Executive Director of Clinical Research and Biology, Alnylam Pharmaceuticals
Robert Haynes, Senior Director, Global Product And Market Planning, Regenxbio Inc
Jennifer Helfer, Director, Global Patient Advocacy Lead, bluebird bio
Andrew Hobbs, MD, Huron Consulting Group
Melissa Hogan, Founder And President, Project Alive
Kui Huang, Rare Disease Epidemiology Lead Strategist, Pfizer
Kevin Huang, President, Chinese Organization for Rare Disorders
Adam Hutchings, Managing Director, Dolon
Gavin Jones, Director Of Rare Disease, OPEN Health
Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical
Sujay Kango, Chief Commercial Officer, Acceleron Pharma
Rashida Karmali, Chief Executive Officer, Tactical Therapeutics Inc
Vikram Karnani, Executive Vice President, Chief Commercial Officer, Horizon Therapeutics
Thomas Kassberg, Chief Business Officer And Executive Vice President, Ultragenyx Pharmaceutical
Ken Kengatharan, Managing Partner, Atheneos Ventures And Chief Executive Officer, Atheneos Ventures
Uday Khire, Chief Executive Officer, AlloMek
Sean Khozin, Global Head Of Data Strategy, Johnson & Johnson
Maya Khurana, Director, Huron Consulting Group
Therese Kinsella, Founder And Chief Scientific Officer, ATXA Therapeutics
Prashant Kohli, Vice President Commercial Operations, Grace Therapeutics
Jonathan Kornstein, Executive Director, Program Strategy, Premier Research
Mark Kroes, Chief Executive Officer, Global Data Support
Akshay Kumar, Partner, Partners4Access
Salvatore La Rosa, Chief Scientific Officer, Children's Tumor Foundation
Steven Laffoon, President, Wylder Nation
Tom Lambdin, Staff, Ben's Friends
Janet Lynch Lambert, Chief Executive Officer, Alliance for Regenerative Medicine
Jim Lang, Chief Executive Officer, EVERSANA
Janice Lansita, Practice Director, Biopharmaceuticals and Pharmaceuticals, EpidStrategies, A Division of ToxStrategies
David Lapidus, President, Lapidus Data
Kimberly Leblanc, Associate Director Of Research Operations Undiagnosed Diseases Network Coordinating Center, Harvard Medical School
Joseph Lee, Executive Director, Analytical Development - Gene Therapy, PTC Therapeutics
Michael Lehmicke, Director, Science And Industry Affairs, Alliance for Regenerative Medicine
Clement Leong, Chief Executive Officer And Managing Director, Stamford Pharmaceuticals
Friedhelm Leverkus, Director Health Technology Assessment And Outcomes Research, Pfizer
Paul Levesque, Global President, Rare Disease, Pfizer
Jeremy Levin, Chairman of the Board, Directors and Chief Executive Officer, Ovid Therapeutics
Edmund Lim, abc, We CARE Journey
Marius George Linguraru, Principal Investigator, Children's National Medical Center
Donald Lo, Director, Therapeutic Development Branch, Division Of Pre-Clinical Innovation, National Center for Advancing Translational Sciences - NCATS
Robert Lodder, President, Biospherics.net
Robert Long, Executive Director, Uplifting Athletes
Jarrod Longcor, Chief Business Officer, Cellectar Biosciences Inc
Pamela Mace, President, Fibromuscular Dysplasia Society of America
Sandy Macrae, President And Chief Executive Officer, Sangamo Therapeutics
Peter Marks, Director, Center For Biologics Evaluation And Research (Cber), U.S. Food and Drug Administration
Paolo Martini, Chief Scientific Officer, Rare Diseases, Moderna Inc.
Allison May Rosen, Representative, Firefly Fund
Janet Maynard, Director, Office Of Orphan Products Development, Food And Drug Administration, U.S. Food and Drug Administration (FDA)
Mary Mcgowan, Executive Director, The Myositis Association
Jenn Mcnary, Consultant, Duchenne Parent Advocate
Eileen Mcneill, Programme Manager - Disease Foundations, Oxford University Structural Genomics Consortium
Pooja Merchant, Head Of Patient Partnership, Engagement And Oncology, Bayer US LLC
Parag Meswani, Senior Vice President Of Commercial Strategy And Operations, Axovant Sciences
Annastasiah Mudiwa Mhaka, Co-Founder, Alliance for Artificial Intelligence in Healthcare
Sheila Mikhail, President And Co-Founder, Asklepios BioPharmaceutical Inc
Randy Milby, Chief Executive Officer, Hillstream Biopharma
Timothy Miller, Co-Founder, President And Chief Scientific Officer, Abeona Therapeutics
Debra Miller, Founder And President, CureDuchenne
James Miskin, Chief Technical Officer, Oxford Biomedica
Samarendra Mohanty, Founder And President, NanoScope Technologies
Michelle Muscat, Co-Founder and President, National Alliance for Rare Diseases Support - Malta
Nuriya Musina, Head Of Development, Federal State Budgetary Institution, The Center For Healthcare Quality Assessme, Ministry of Health Russian Federation
Joseph Musumeci, President, BluePrint Orphan
Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
Lauren Neighbours, Neighbours, Senior Director, Regulatory Affairs, Optum Digital Research Network, Optum
Jay Newman, Senior Vice President & General Manager, Head of US Commercial, Spark Therapeutics
Scott Nickerson, Senior Vice President, Global Quality, Moderna Therapeutics
Jan Nielsen, Division President, Patient Solutions, AssistRx Patient Solutions
Yukiko Nishimura, President, ASrid Japan
Neena Nizar, President, The Jansens Foundation
Sara Nochur, Senior Vice President Regulatory Affairs, Alnylam Pharaceuticals
Lynn O'Connor Vos, CEO, Muscular Dystrophy Association
Daniel O'Mahony, Partner, Seroba Life Sciences
Nawal Ouzren, Chief Executive Officer, Sensorion Pharma
Arrigo Paciello, Assessor, AIFA
Cindy Padgett, Vice President, PharmaCord
Nick Page, Chief Operations Officer, Oxford Biomedica
Palani Palaniappan, Head Of Technical Operations And Andover Site, Sarepta Therapeutics
Jim Palma, Executive Director, TargetCancer Foundation
Francis Pang, Vice President Global Market Access, Orchard Therapeutics
Art Pappas, Managing Partner, Pappas Capital
Anne Pariser, Director, Office Of Rare Diseases Research, National Center For Advancing Translational Sciences - Ncats, National Institutes of Health (NIH)
Detlev Parow, Head Pharmaceutical Department, D.A.K.
Linn Parrish, Head Of Csr Innovation And Stakeholder Engagement, Corporate Communications And Public Affairs, Takeda
Doug Paul, Vice President And Partner, MME
Steven Peskin, Associate Clinical Professor Of Medicine, Rutgers Rwj Medical School, Executive Medical Director Population Health And Transform, Horizon Blue Cross Blue Shield - New Jersey
Dmitry Petrov, Head of Medical Writing and Pharmacovigilance, ICRC-Weyer GmbH
Areejit Phukan, Director, Analytics And Forecasting, Insmed Inc
Andres Pichon-Riviere, Executive Director Of The Institute For Clinical Effectiveness And Health Policy (Iecs), Director Of The Health Technology, University Of Buenos Aires
Oresta Piniazhko, Director, Hta Department, Ministry of Health of Ukraine
Sarah Pitluck, Head, Global Pricing And Reimbursement, Spark Therapeutics
Nader Z Pourhassan, President And Chief Executive Officer, CytoDyn, Inc.
Leslie Powell, Director of Policy and Advocacy, Cystic Fibrosis Foundation
Mayo Pujols, Vice President, Head Of Global Cell And Gene Technical Development And Manufacturing, Novartis
Anthony Quinn, President And Chief Executive Officer, Aeglea Biotherapeutics
Harsha Rajasimha, Founder And Chairman, IndoUSrare
Shankar Ramaswamy, Chief Business Officer, Axovant
Karlyne Reilly, Senior Associate Scientist And Section Head, National Cancer Institute
Frederic Revah, Chief Executive Officer, Genethon
John Reynders, Vice President, Data Sciences, Genomics, And Bioinformatics, Alexion Pharmaceticals
Marisa Ricci, Vice President Patient Diagnosis Programs And U.S. Field Medical Engagement, Ultragenyx Pharmaceutical
Trevor Richter, Director, Common Drug Review And Optimal Use, CADTH
Shawn Ritchie, Chief Executive Officer, Med-Life Discoveries
Juan Roman, Head Of Market Access And Pricing, Acceleron Pharmaceuticals, Inc.
Kieran Rooney, Vice President, Strategic Alliances and Licensing, Amryt Pharmaceuticals
Luke Rosen, Head Of Patient Engagement And Digital Health Experience, Ovid Therapeutics
Ruth Rostron, Vice President Of Project Management, Inceptua Medicines Access
Seth Rotberg, Co Founder And President, Our Odyssey
John Rother, Chief Executive Officer, National Coalition On Health Care
Mark Rothera, President And Chief Executive Officer, Orchard Therapeutics
Delphine Roulland, Director Government Affairs And Public Policy, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
David Russell, Head Of External Innovation, Actelion-Janssen
Leo Russo, Senior Director, Epidemiology Worldwide Safety And Regulatory, Pfizer
Peter Saltonstall, Chief Executive Officer, National Organization for Rare Disorders (NORD)
Abby Sandler, Executive Director, Mypart, Center For Cancer Research,, National Institutes of Health
Scott Schliebner, Vice President, Scientific Affairs, Rare Diseases, PRA Health Sciences
Sophie Schmitz, Managing Partner, Partners4Access
Jeffrey Schwartz, Managing Director, Bain Capital Life Sciences
Denise Scots Knight, Chief Executive Officer And Co-Founder, Mereo BioPharma
Jim Shaffer, Cbo, Eiger Biopharmaceuticals
Lesha Shah, Co-Chair, Pediatric Gene Therapy And Medical Ethics Working Group, Assistant Professor Of Psychiatry, Icahn School Of Medicine, The Mount Sinai Hospital
Nataliya Shandarovska, Patient Advocate, National Alliance for Rare Diseases Support - Malta
Panna Sharma, President And Chief Executive Officer, Lantern Pharma
Adam Shaywitz, Chief Medical Officer, Rare Disease Drug Development, BridgeBio Pharma
Rachel Sher, Vice President Policy And Regulatory Affairs, NORD
Alex Sherman, Director, Center For Innovation And Bioinformatics, Harvard Medical School
Gerard Short, Senior Vice President, Clinical And Regulatory, Freeline Therapeutics
Alison Skrinar, Vice President, Clinical Outcomes Research And Evaluation, Ultragenyx Pharmaceutical
Kristin Smedley, President, Curing Retinal Blindness Foundation
Bob Smith, Senior Vice President, Global Gene Therapy Business, Pfizer
Meagan Spychala, Assistant Vice President Of Patient Engagement And Program Strategy, Rho, Inc
Kim Stephens, President, Project Alive
Marshall Summar, Division Chief Of Genetics And Metabolism, Children's National Medical Center
Weiyong Sun, Senior Director, Specialty Medicine Search, Evaluation And Global Business Development, Daiichi Sankyo Co Ltd
Thayer Surette, Deputy Director, Partnership to Improve Patient Care (PIPC)
Stephanie Tagliatela, Co-Founder And Chief Scientific Officer, Encoded Therapeutics
Jan Thirkettle, Chief Development Officer, Freeline Therapeutics
Becky Thompson, Director Integrated Solutions, PAREXEL
Dan Tierno, Strategic Implementation Manager, Global Data Sciences And Analytics, Bayer US LLC
Cynthia Tifft, Deputy Clinical Director, Office Of The Clinical Director, National Human Genome Research, National Human Genome Research Institute
Brigette Tippin Davis, Senior Vice President, R&D, Ambry Genetics
Vladimir Tomov, Chairman, National Alliance of People with Rare Diseases - Bulgaria
Ngeh Toyang, Chief Executive Officer, Flavocure Biotech
Sara Traigle Van Geertruyden, Executive Director, Partnership to Improve Patient Care (PIPC)
Evert Jan Van Lente, Director Eu-Affairs, AOK Bundesverdand
Carole Spangler Vaughn, Chief Executive Officer, Emtora Biosciences
Jodie Vento, Chair, Access Committee, National Society Of Genetic Counselors, Manager, Center For Rare Disease Therapy, Childrens Hospital of Pittsburgh
Terry Jo Vetters Bichell, Founder And Director, COMBINEDBrain
Kim Warren, Head Of Operations, Avrobio
Melissa Wasserstein, Chief, Division Of Pediatric Genetic Medicine, Department Of Pediatrics,, Montefiore Medical Center
Monica Weldon, President and Chief Executive Officer, Bridge The Gap Syngap Education And Research Foundation
Samuel Agyei Wiafe, Founder Executive Director, Rare Disease Ghana Initiative
Terry Wilcox, Co-Founder And Executive Director, Patients Rising & Patients Rising
Durhane Wong-Rieger, President And Chief Executive Officer, Canadian Organization For Rare Disorders
Janet Woodcock, Director Of Drug Evaluation And Research Center, Food and Drug Administration (FDA)
Jonathan Woodring, Executive Vice President And General Manager, IPM.ai
William Xiang, Director, Strategy And Operations, XW Laboratories
Zheng Yang, Head Of Technology And Data Innovation, Customer Value, Boehringer Ingelheim
Richard R Yang, Chief Executive Officer And Founder, Reflection Biotechnologies
Dimitar Yonchev, Patient Advocate Representative, Pro Retina Deutschland e.V.
David Young, Chief Executive Officer, Processa Pharmaceuticals, Inc.
Timothy Yu, Attending Physician, Investigator, Division Of Genetics And Genomics, Boston Children's Hospital
Wyatt Yue, Principal Investigator, Associate Professor, Lead Scientist, Rare Disease Research, Oxford University Structural Genomics Consortium
Ran Zheng, Chief Technology Officer, Orchard Therapeutics
Organiser
