2017 SPEAKERS

Dr. Acquadro is actively involved in scientific communication, in the development of collaborative projects, and in the new developments and audit of MRT databases (PROQOLID, PROlabels, PMR and Outcomes Insite). Dr. Acquadro received her MD from the University of Grenoble (France) in 1990. Her...

Senior Adviser at Norwegian Medicines Agency HTA-analyst and reimbursement decision maker for the National Insurance Scheme Leading a working group on very small populations with highly severe disease Former adviser at Ministry og Health and Care services  Co-writer on the...

Dr. Ardigò is an MD (specialized in Internal Medicine) and obtained a PhD at the University of Parma (Italy) and a post-doctoral fellowship at Stanford University (California, US). He is author of more than 40 indexed papers. He joined Chiesi in 2010, where acted as Clinical Lead in the...

Ségolène Aymé is Emeritus Director of Research at the French Institute of Health and Medical Research (INSERM). She now acts as expert in residence for rare diseases at the Brain and Spine Institute in Paris, and serves as Editor-in-Chief of the Orphanet Journal of Rare Diseases. She was the...

Michelle joined the company in June of 2015. In her role as VP, Patient Advocacy, she serves as the point of contact for families and foundations seeking information on Abeona’s pipeline of programs, partners with communities to incorporate priorities and input into program development, focuses on...

Dr Stella Blackburn is currently Vice President, Global Head of Risk Management at Quintiles.  Following medical training at Cambridge University and Guy’s hospital, Stella worked in hospital medicine before joining the pharmaceutical industry.  There, she spent 11 years in pharmacovigilance and...

Jane Blyth is a Scientific Director at Complete HealthVizion, a medical communications agency within the McCann Health network.  Jane has a clinical nursing background and over 20 years’ experience in the pharmaceutical industry, including clinical research, publication management and strategic...

Nadia is a seasoned rare disease advocate, born with 3 debilitating rare diseases complicated by several associated conditions. Of course that didn’t stop her! She served as the chairperson for Rare NJ, a rare disease advocacy coalition and was the Executive Director for the Rare Disease United...

Nicole founded Global Genes in 2008, with the goal of helping families affected by rare disease connect with tools, resources, and much needed support, helping eliminate the challenges of rare disease. She has successfully launched and developed Global Genes to a multi-million dollar non profit...

As the Director of Research Programs, Vanessa is responsible for overseeing natural history studies and research grant initiatives for NORD. She has over 10 years of research and program development experience and was most recently with the Dana-Farber Cancer Institute.  Vanessa received her...

Dr. Bryan became our Senior Vice President of Business Development in March 2017. Prior to joining Sucampo, he served as SVP BD of Lupin Inc., where he had responsibility for business development for the brand division, since 2015. From 2005 to 2015, Dr. Bryan was Vice President Business...

Emily is External Strategy Manager, Gene Therapy in GSK’s Rare Diseases Unit. She works as a scientist looking at developing external relationships and partnerships in the field, as well as providing scientific guidance and input to the internal gene therapy strategy and pipeline. Emily has a...

David Dasberg is the Managing Director of Facio Therapies and Facio Intellectual Property. He has over 20 years' experience with the development and commercialization of (bio)pharmaceutical products. As an independent consultant, David performed a feasibility study commissioned by the Dutch...

Francesco De Rubertis is Partner and co-founder of medicxi. Prior to this, he was with Index Ventures for 18 years, having joined the firm in 1997 to launch its life sciences practice. While at Index life sciences, he spearheaded the creation and adoption of the asset-centric investment strategy...

Dr. Dellamano, founder and President of ValueVector, was Head of Strategic Pricing at the global Headquarters of the Swiss pharmaceutical giant Hoffmann-La Roche from 1996 to 2003. Before heading the Strategic Pricing function at Hoffmann-La Roche, Dr. Dellamano served as Global Controller of the...

Debbie DrellDirector of Membership ServicesNational Organization for Rare Disorders (NORD) As the Director of Membership, Debbie is responsible for overseeing NORD’s Membership programs, which support the collective and individual needs of rare disease patient organizations, patients, and patient...

Experienced health economist with strong institutional background and in depth knowledge of market access and public affairs activities in the pharmaceutical sector. With a great deal of experience in consultancy activities at international level related to healthcare systems...

Ms. Begonya Nafría (F), MsC, Patient Advocacy Manager at Institut de Recerca Sant Joan de Déu and Fundació Sant Joan de Déu. Experience in coordinating and managing projects in clinical research on rare diseases. Coordinator of YPAG (Young Persons’ Advisory Group)-KIDS Barcelona and RareCommons...

Davide Finocchiaro is a Pharmacist graduated at the University of Catania and  with a masters in Health management earned at the Bocconi University in Milan. He has more than 10 years of experience in the pharmaceutical industry. He has a particular experience and interest for Market...

Rob works within the global business development team consulting with pharmaceutical and biotech companies to identify and implement strategic solutions to meet their global access needs. With more than 13 years experience in managed access programmes; he has worked across a range of therapeutic...

Kelly Franchetti has nearly two decades of direct patient experience, gained in both hospital and industry settings including CROs, healthcaretechnology, patient research and hands-on patient care across multiple therapeutic areas. Ms. Franchetti has been involved in the development and moderation...

Professor Bobby Gaspar is Professor of Paediatrics and Immunology at UCL Great Ormond Street Institute of Child Health and Honorary Consultant Paediatric Immunologist at Great Ormond Street Hospital for Children NHS Foundation Trust.   He initially trained in paediatrics and then became interested...

Rory is the Senior Director of Regulatory Affairs at Mapi, he has 25 years of experience in the heathcare Industry to devise and execute tailored regulatory solutions for clients. Expertise provided in EU Scientific Advice pathways, parallel SA-HTA meetings, Orphan Designations, Paediatric...

Rachel is a co-founder of Caribou Biosciences and has been President and CEO since its inception. She has a research background in CRISPR-Cas biology, and is also a co-founder of Intellia Therapeutics. In 2014, she was named by Forbes Magazine to the "30 Under 30" list in Science and...

Patrick is Executive Director World Wide Health Economics and Outcomes Research (WWHEOR) Markets at Bristol Myers Squibb. He leads the team responsible for supporting country HTA submissions and access. He is responsible for all countries outside of the US. Patrick also leads WWHEOR HTA Policy...

Wills Hughes-Wilson is Chief Patient Access Officer at Sobi and a member of the company’s global Executive Management team.  She joined the company in 2012. Hughes-Wilson leads the cross-functional, international team responsible for Sobi’s patient-access-centred approach across the company and its ...

Adam is Managing Director of Dolon - an innovative, strategic market access consultancy specialising in rare diseases. A health economist by training, he has over 15 years’ market access experience in consulting and the pharmaceutical industry, including roles at Aventis, GlaxoSmithKline and Baxter ...

Edmund Jessop has been practising public health for the NHS in England since 1981 in various jobs at district, regional and national levels. Since 2002, he has been medical adviser to the team which plans, funds and monitors services for patients with very rare disease. This has included decision...

Edmund Jessop has been practising public health for the NHS in England since 1981 in various jobs at district, regional and national levels. Since 2002, he has been medical adviser to the team which plans, funds and monitors services for patients with very rare disease. This has included decision...

Dr Kengatharan is a co-founder of several San Francisco bay area based biopharmaceutical companies. He is currently Chairman of Renexxion. Previously he was President & CEO of Armetheon and prior to that President & CSO of Altheos. He is the former Vice President,  Pre-Clinical R&D of...

Alastair Kent OBE is the Director of Genetic Alliance UK – the national charity of over 180 patient organisations, supporting all those affected by genetic conditions. Genetic Alliance UK’s mission is to promote the development of the scientific understanding of genetics and the part that genetic...

Dr Sven Kili is the VP and Development Head for the Cell and Gene Therapy division of GSK where he leads the teams developing ex-vivo Gene Therapies for a variety of genetic disorders. They are currently developing solutions for a number of diseases including ADA-SCID; WAS; Metachromatic...

Dr. Kishimoto is the Chief Scientific Officer of Selecta Biosciences, a biotechnology company developing synthetic vaccines based on a novel self-assembling nanoparticle technology.  Prior to joining Selecta, Dr. Kishimoto was Vice President of Research at Momenta Pharmaceuticals where he led...

Anja Kladar is a Croatian physiotherapist, bachelor of economics and project manager for EU funds with more than 10 years of experience in the civil sector, especially in the field of rare diseases, trying to help and increase the quality of life of the patients. During the years, Mrs. Kladar...

Maryna Kolochavina, PharmD, PMP, Project Director, RWLP Operations, Europe and Asia Pacific, has over 12 years’ experience in worldwide clinical research and observational studies in various roles within the CRO and Pharma Industry. Broad experience in post-marketing research, project...

Yann Le Cam is a patient advocate who has dedicated 25 years of professional and personal commitment to health and medical research non-governmental organisations in France, Europe and the United States in the fields of cancer, HIV/AIDS and rare diseases.He has three daughters, the eldest of...

Following a Ph.D. in Biochemistry and Molecular Biology from the University of Zürich Switzerland, Dr Linden held faculty positions at Mount Sinai School of Medicine in New York and King’s College London, engaged in the field of AAV biology and AAV-mediated gene therapy.  Most recently Michael...

Douglas Lundin, PhD, is chief economist at TLV (Swedish Dental and Pharmaceutical Reimbursement Agency). He has had that position since 2011. Before that he worked at the Swedish Competition Authority.  

Tiziana Magni has gained more than 3 years of experience in the Global Pricing & Market Access team within Chiesi Group headquarters, in Parma. As part of her activity, she is mainly focused on the pricing and market access for Advanced Therapy Medicinal Products (ATMPs) and for Orphan Drugs...

Mapi is the only Global CRO specializing in late phase research. We also provide strategic consulting and related services in Epidemiology and Product-Related Risk Management . This includes market forecasting, health burden and economic assessments, pharmacovigilance and risk management plans, and ...

Abeona Therapeutics (NASDAQ: ABEO) is clinical stage biopharmaceutical company focused on delivering gene therapy products for severe and life-threatening rare diseases. Our lead indications are for children with the rare genetic metabolic and dermatologic disorders, Sanfilippo syndrome, Batten...

Dr. Ana Mingorance is a specialist in genetic epilepsy syndromes and an independent consultant in CNS and orphan drug development. Ana has close relationships with patient groups and has acted as a scientist-in-residence, scientific advisor, and patient advocate. She is the author of the eBook...

Executive Director, Strategic Development, Rare Diseases Juliet Moritz has worked in clinical research for more than 25 years, and her extensive background covers the spectrum from single-site studies to large, multinational trials. She joined Premier Research in 2016 to specialize in rare...

Since 1980, Arnold Munnich has made every efforts to conciliate the clinical and molecular approaches of genetic diseases in children. His efforts have resulted in the founding of the Department of Genetics, Hôpital des Enfants-Malades, Paris, which brings together i) a Clinical Research INSERM...

Dr. Murphy’s professional career has spanned 25 years and his positions within the pharmaceutical industry emphasize the integration of medical and scientific acumen with operational excellence. He is board-certified in psychiatry and has a doctorate in pharmacology, with training at Tulane...

Alexander Natz is the Secretary General of the European Confederation of Pharmaceutical Entrepreneurs (www.eucope.org) in Brussels and works as a lawyer in the healthcare boutique law firm NOVACOS in Düsseldorf (www.novacos-law.com). He advises innovative pharmaceutical and biotech...

Sandra Nestler-Parr has extensive experience in the global biotech and pharmaceutical sector, holding senior international medical and market access roles in industry and consulting. She focuses on advising clients on issues relating to market access, pricing, outcomes research and healthcare...

Judith Ng-Cashin is a senior physician executive with more than 20 years experience in medicine discovery and development, portfolio strategy, marketed product support, medical governance, clinical research, bench science and clinical medicine.Judi currently serves as Chief Scientific Officer at...

Jane Nunn is the Head of Operations at Complete HealthVizion, a medical communications agency within the McCann Health network.  Jane has 20 years’ experience in medical communications and brings a strong scientific background to her role having been awarded her PhD in the field of genetic...

Tara O’Meara is the Vice President of Clinical Development Operations at bluebird bio.  Tara oversees a team responsible for the execution and analysis of bluebird bio’s lentiviral vector based gene therapy development programs.  Currently bluebird bio has 4 active programs (Phase 1/2 through 3)...

Mareike Ostertag has extensive experience in the pharmaceutical industry. In over 16 years with Novartis, Dr. Ostertag held different roles with increasing responsibility within the company as well as with external industry- and non-industry partners. Roles included Technical Marketing, Global...

Samantha Parker MBA is Lysogene’s Chief Patient Access Officer. Samantha is responsible for providing global patient access strategic leadership.  Samantha has specialised in rare disease research and public health for about 20 years. She has focused on expanding the expert disease community of...

Kay Parkinson was the mother of two children who were diagnosed with the ultra rare disease Alström Syndrome when they were aged 18 & 15, having had four previous mis-diagnosis. Both children died following heart and heart/kidney transplantations aged 25 and 29 respectively.She qualified as a...

David Pearce is President of Research for Sanford Health.  He completed his undergraduate Bachelor of Science Degree with honors in biological sciences at Wolverhampton Polytechnic in 1986.  He gained his PhD in 1990 at the University of Bath, UK, and did postdoctoral training at the University of...

Mathew Pletcher currently serves as the Head of Rare Disease - Discovery for Roche, directing early drug development and research activities for rare neuromuscular, neurodevelopmental, and ophthamological diseases including spinal muscular atrophy, Duchenne muscular dystrophy, myotonic...

Dr Krzysztof Potempa is a translation-minded neuroscientist with over ten years of experience in advanced analytics of neuroscience datasets gained in academic (The Francis Crick Institute) and pharmaceutical (Lundbeck) settings. He received his B.Sc. in Biochemistry from the University of Georgia...

As the Chief Executive Officer of Genethon, Frédéric Revah has more than twenty years’ experience in the pharmaceutical and biotech industries as well as in academic research. Immediately prior to joining Genethon in January 2010, he had been Chief Executive Officer at Sepal Pharma...

Mr. Reynolds has over 30 years experience in launching  software companies in the UKI, EMEA and US. Since 2013 he has been responsible for OpenApp’s  rare disease registry business. In 2015 he set up the agreement with Vertex to run the 4,000 patient phase IV clinical trial for Orkambi in...

Diana has played a leading role at Action Duchenne for the past six years and was appointed as CEO last year, based on her knowledge and invaluable contribution to the Duchenne community. She has worked closely with hundreds of international families and young people living with Duchenne and other...

An experienced executive with a track record in successfully building and managing clinical programs, Marc’s expertise encompasses all aspects of drug development in different clinical indications from pre-clinical development to post marketing surveillance. At TVM Capital Life Science, he focuses...

Paul is Executive Director in the Office of the Chief Patient Officer at MSD, a global healthcare company with a 125-year history of working to make a difference to people’s lives.  He has held posts in clinical research and medical affairs in both the UK and the USA.  He has a background in...

Subhasis Roy is Managing Partner and co-founder of Sirius Healthcare Partners, a healthcare advisory boutique. He has over 22 years of investment banking and healthcare advisory experience. He is a strategic advisor to numerous emerging private and listed healthcare companies and healthcare...

Carina Schey has many years of both pharmaceutical industry and clinical pharmacy experience. She is a consultant health economist based in Switzerland, currently pursuing research through the University of Groningen, PharmacoEconomics and PharmacoEpidemiology Unit in the Netherlands.  The primary...

Mr. Schliebner is a clinical scientist by training, with a 20-year background in clinical drug development.  His expertise is focused on providing strategic, scientific and operational guidance to clients on clinical trial design, competitive landscape analysis, investigator selection, advocacy ...

Sophie is Managing Partner at Partners4Access, a global consultancy 100% focused on orphan drug access. We support the biotechnology and pharmaceutical industry along their launch journey to help secure successful price, reimbursement and access for orphan drugs. Partners4Access has solid...

Matthias Schönermark is one of the most reputable strategy consultants in Germany. He is a trained head and neck surgeon with a Ph.D. in molecular oncology. In 1998, he left his job as an Associate Professor at Hannover Medical School for a position as project leader and manager at The Boston...

Dr. Raghuram Selvaraju, Ph.D., MBA, currently serves as a Managing Director and Senior Healthcare Analyst at Rodman & Renshaw Research, a unit of H.C. Wainwright & Co. Earlier, he started his sell-side research analyst career with Rodman & Renshaw in 2005. Prior to rejoining Rodman, he...

Bruno Sepodes holds a PhD in Pharmacy (Pharmacology) by the University of Lisbon. Currently a Professor of Pharmacology and Pharmacotherapy at the Faculty of Pharmacy of the University of Lisbon, he develops his research in Pharmacology and Translational Medicine. Presently Prof. Sepodes is the...

Mathieu Simon, MD, has served as Executive Vice-President, Chief Executive Officer of Cellectis Therapeutics since 2012 and as Chief Operating Officer of Cellctis since 2013. Dr. Mathieu Simon is also Member of Cellectis, Board of Directors since 2013 and sits at the board of Vaximm a Swiss-based...

Andrea is currently Chief Medical Officer, R&D Head and co-founder of Orchard Therapeutics, a late clinical stage potential of ex-vivo autologous haematopoietic stem cell gene therapy to restore normal gene function in severe and life-threatening inherited disorders. Prior to joining Orchard...

Heide is a Director of Epidemiology and a GSK Fellow in the RWE & Epidemiology department at GlaxoSmithKline.  She is based in the UK, and has been working as an Epidemiologist in the pharmaceutical industry since 1999, providing Real World Evidence and observational research to inform decision ...

Andrew Storey has over 30 years in the biopharmaceutical industry providing leadership for Regulatory Affairs.  He is presently Vice President, Global Regulatory Strategy for AbbVie and was a VP with global responsibilities for the previous 14 years at Cangene. Prior to that he held several...

Dr Rick Thompson joined Findacure in 2015, after completing his PhD in Evolutionary Biology at the University of Cambridge. He has recently moved to the position of CEO, after working as Head of Research. He is responsible for the running and development of the charity, as well as their scientific...

uExecutive Medical Director, Medical & Scientific Drug Development Lead, Neurology Center of Excellence.u27 years of experience in CNS: 11 in hospital/university settings and 16 years in the pharmaceutical industry of which 9 years in Quintiles.uPhase II–IV clinical trial experience in...

Sheela Upadhyaya is currently the Associate Director of the Highly Specialised Technology program at NICE and is responsible for running the program to evaluate medicines and technologies for rare and ultra-rare conditions for commissioning in the NHS.Prior to joining NICE, she was responsible for...

Birgitte Volck, MD, PhD is, by 1 June 2016, Head of R&D, Rare diseases at GSK. From 2012-2016 she was Chief Medical Officer and head of Development, leading Sobi’s portfolio of products and product candidates in rare diseases and speciality areas across hemophilia, inflammation, neonatology and ...

Julie Walters, founder, is the driving force behind Raremark and leads on product strategy. She has a first-class degree in molecular genetics from King’s College London and a background in popular journalism, including time as a news editor on TV’s Good Morning Britain. Outside Raremark, Julie is ...

As Managing Partner, Rob is responsible for delivering Cello Health’s public affairs offering across Europe, the US and the rest of the world. His particular areas of expertise include rare disease policy, consensus building with community stakeholders and the development of innovative finance...

Dr. Christina Waters has been responsible for leading medical research in a broad range of organizations ranging from academia and disease – focused non-profits to biotech and large pharmaceutical companies.   Her broad range of experience in basic, translational and clinical research and business...

Diane has over 18 years of Pharmaceutical and Biotech experience and has held a variety of positions involved in clinical development and commercialization of drug products. She is currently Director Global Project Management at BioMarin, based in the USA. She holds a B.Sc. (hons) in Biochemistry...

Dr. Stephen Wright has served as our Research and Development Director since January 2004 and as a Director since March 2005. Dr. Wright has more than 20 years of experience in drug development. Prior to joining our company, Dr. Wright was Senior Vice President of Clinical Research &...

Dr. Stephen Yoo is the Chief Medical Officer of REGENXBIO. Prior to joining REGENXBIO, he was Medical Science Director of Clinical Development at AstraZeneca and Group Director of Clinical Development at MedImmune, AstraZeneca’s global biologics research and development arm. In these roles, he led...

Martine Zimmermann, Senior Vice PresidentHead of Global Regulatory Affairs, ALEXION PHARMA GMBH Martine is global head of regulatory affairs at Alexion Pharmaceuticals.  She is based in Zurich, Switzerland. She has over 20 years of combined R&D and global regulatory strategy experience.  She...