2018 Speakers

Prof. Dr. Annemieke Aartsma-Rus is a professor of translational genetics at the Leiden University Medical Center. She played an important role in the development of the antisense mediated exon skipping therapy for Duchenne muscular dystrophy during her PhD research (2000-2004). Her work currently...

Founder and CEO of Lysogene, Ms. Aiach is also the mother of a child with MPS IIIA. She has a strong business background starting her career with Arthur Andersen specializing in audit and transaction services. Her entrepreneurial experience includes founding and running a financial business...

Dennis has a finance background and worked in the investment banking industry for 9 years before joining myTomorrows. Dennis is based New York where he has been developing many cross-Atlantic relationships in the life-sciences industry over the last 6 years.

Luca Alberici is Chief Business Officer of MolMed since April 2015. He holds responsibility for Business Development, Intellectual Property, Project Management and is the former responsible for Market Access. He joined MolMed in 2006 and worked with growing responsibilities in R&D, contributing ...

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 2016 white paper...

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...

Denis Belyakov CEO of Russian Union of patients’ and patients’ organizations. Denis leads the cross-functional, umbrella, interregional patient’s organization responsible for patients’ support and access to treatment for different rare diseases.  This includes not only the end-result of successful...

Marian Bendik obtained his master's degree in Biochemistry from the University of Chemistry and Technology, Prague in 2006. He then worked as an immunoassay research scientist in academia and later in industry. Afterwards, he occupied different process and manufacturing expert roles with...

Medical Doctor with more than 15 years of clinical and pharmaceutical industry experience. After several years working as physician he moved to pharma organization. Worked in different positions in sales and medical affairs and covered different therapeutic areas:...

Meriem Bouslouk (PhD in dentistry, MSc International Health) joined the Pharmaceuticals Department of Germany’s Federal Joint Committee (G-BA) in 2011, and has worked on the implementation of AMNOG, the Act on the Reform of the Market for Medicinal Products, from the start, principally in the field ...

Meriem Bouslouk-Marx (PhD in dentistry, MSc International Health) joined the Pharmaceuticals Department of Germany’s top health policy body, the Federal Joint Committee (G-BA), in 2011 and has worked on the implementation of AMNOG, the Act on the Reform of the Market for Medicinal Products, from...

Dr. Jacoline Bouvy is a health economist specialising in the interface between marketing authorisation and health technology assessment (HTA) of medicines. She works at the National Institute for Health and Care Excellence (NICE) in London within the Science, Policy & Research team where she...

Agnese is a health economist graduated at Catholic University of Sacred Heart in Rome. Since the beginning of her work in AIFA, she has dealt with the monitoring of pharmaceutical consumption and expenditure and with the yearly publication of National Report on medicines’ use in Italy. Her...

Nathalie Cartier obtained her Medical degree from the University René Descartes in Paris where she completed her residency and specialized in Pediatrics. She became Research Director at INSERM and associate Professor at University Descartes.  Her research interest focuses on the development of gene ...

Dr. Maria Chatzou, CEO of Lifebit, is a biotech innovator and a proud geek, expert in bioinformatics, medical informatics and high performance computing. She is also a passionate entrepreneur and has founded two companies, Innovation Forum Barcelona and, Techstars-backed Lifebit.Prior to...

James Christie (BSc, MBA) is the SVP Manufacturing and Supply Chain for MeiraGTx.  Prior to MeiraGTx, he was Head of Manufacture, Innovation and Project Director for the Medicines Manufacturing Industry Partnership (MMIP), employed by Association of the British Pharmaceutical Industry (ABPI). ...

Laura has 20+ years of experience working with medical device and biotech companies developing new markets and launching innovative products.  Companies include Eli Lilly, Guidant, FoxHollow Technologies, and ProFibrix. She has an MBA and a degree in Industrial Engineering.

Emily set up the Duchenne Children’s Trust in 2012 after her son was diagnosed with Duchenne. Before that she was a reporter and anchor for Channel 4 News and CNN International. The Duchenne Children's Trust raised £3.5million in its first three years, to spend on clinical trials, research, and ...

Dan Tagle is associate director for special initiatives at NCATS. He also recently served as acting director of the NCATS Office of Grants Management and Scientific Review and currently serves as executive secretary to the NCATS Advisory Council and Cures Acceleration Network Review Board. Prior to ...

Cecile De Coster has joined bluebird bio in 2017 as Director, Regulatory Sciences. At bluebird bio, she is responsible for leading the global regulatory strategy for a gene therapy medicinal product developed for a rare pediatric disorder. Before joining bluebird bio, Cecile worked as an Associate...

Dr De Kimpe is co-founder and SVP R&D of Auxesia Orion, a start-up committed to develop and commercialise orphan drugs through an integrated disease management strategy. Dr de Kimpe has over 25 years experience at different stages of development from research to registration in a variety of...

Michele De Luca, MD is Director of the Centre for Regenerative Medicine “Stefano Ferrari”, University of Modena and Reggio Emilia, and Scientific Director of Holostem Terapie Avanzate S.r.l. Michele De Luca is internationally recognized as a leading scientist in epithelial stem cell biology aimed...

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...

As Vice President Rare and Orphan Diseases, Lisa Dilworth regularly consults with Synteract clients in rare and orphan diseases on study design, eligibility criteria, appropriate patient populations, country/site mix, end point selection, and overall program strategy.  Lisa has more than 18 years...

Scott Dorfman is president and CEO of Odylia Therapeutics, a non-profit biotech company whose mission is to facilitate the translation of rare disease with proof-of-concept from the lab into clinical trials. Prior to Odylia, Mr. Dorfman founded and was Chairman, & CEO of Innotrac Corporation...

Cinzia Dorigo, Executive Director Clinical Development, Rare Disease and General Medicine, PharmD, leads INC Research’s Rare Disease Consortium focused on an effective strategic implementation for successful delivery of rare and ultra-rare disease studies. She brings 17 years’ experience in rare...

Cynthia Dukes brings over 35 years of diversified clinical experience encompassing 29 years of clinical research experience in vaccines, oncology and infectious disease.  Cynthia began her clinical research experience as the Clinical Team Lead at Baylor College of Medicine’s Vaccine Treatment and...

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...

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

As Head of Alliance Managment she was responsible for the execution of the first strategic Alliance of Telethon/OSR with GSK managing gene therapy projects at different stage of development ranging from preclinical, to clinical, to Registration & market access. With her capability to work in a...

Benjamin is head of international government affairs, public policy and patient engagement with Vertex Pharmaceuticals. In his role Ben supports reimbursement strategies and  rare diseases policy development relevant to Vertex’ portfolio of transformative medicines for cystic fibrosis. Having...

Pamela Gavin sets the strategic direction for NORD and oversees the implementation of the programs and services that support the rare disease community. Before NORD, Pam worked to execute healthcare programs aimed at improving safety and delivery. For the federal government, she implemented a new...

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...

Tim is a social entrepreneur passionate about delivering the next generation of therapeutics to rare disease patients in need. He is the Founder and Chief Executive of Healx and the Co-Founder and Chair of the Cambridge Rare Disease Network (CRDN), a charity aiming to foster dialogue and awareness...

Dr. Femida Gwadry‐Sridhar is the Co‐Founder and CEO of Pulse Infoframe Inc. She has her PhD in Research Methodology, Health Economics, McMaster University. She is a pharmacist, epidemiologist and methodologist with over 25 years of experience in clinical trials, disease registries, knowledge...

Annie Hubert is Senior Director, EU Section and Public Policy at the Alliance for Regenerative Medicine.  The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to...

Lynne Hughes has worked in clinical research for more than 32 years and has lived and worked during this time period in both the US and in Europe. She has been with IQVIA for 21 years and is currently Vice President and Global Head of CNS Medical Strategy. Lynne has worked exclusively in neurology...

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...

Dr. Incerti oversees Global Medical Affairs in the 4 Therapeutic Areas of Sanofi Genzyme, a Global Business Unit of Sanofi Corporation: Oncology, Immunology, Multiple Sclerosis and Rare Diseases.He is member of the Sanofi Genzyme Portfolio Management Committee, member of the Sanofi Research...

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...

Bodil JonasonVP Marketing & Sales, Inceptua With more than 20 years in Pharma, managing specialty and orphan drugs products in international senior marketing and sales positions, Bodil has a strong track record of leading businesses and teams launching products. Currently, she has taken on a...

Daria Julkowska is a Scientific Coordinator at the French National Research Agency (ANR) where she is responsible for the management of several European and international funding programmes. Among others, since 2010 she is involved in E-Rare, the ERA-Net for Research programmes on rare...

Panos Kanavos (BSc, MSc (Oxon), MSc (LSE), PhD) is Associate Professor in International Health Policy in the Department of Social Policy, London School of Economics (LSE), Programme Director of the Medical Technology Research Group (MTRG) at LSE Health and Deputy Director at LSE Health. Panos has...

Vikram KarnaniHorizon Pharma plcExecutive Vice President, Chief Commercial Officer Vikram joined Horizon Pharma in July 2014 and has been in his current position since February 2018.   Prior to being promoted to Chief Commercial Officer, Vikram led the Rheumatology Business Unit, including the...

M. (Ken) Kengatharan, CEO, Auxesia Orion and Managing General Partner, Atheneos Ventures Dr Kengatharan is a co-founder of several San Francisco bay area based biopharmaceutical companies where he held responsibilities in R&D and corporate development. He is currently President & CEO of...

Diane Kleinermans works as advisor to the Minister of Public Health and Social Affairs since 2015. She is responsible of dossiers related to clinical trials. Diane has a medical background and a broad experience in various fields. She started her career as GP in Brussels but rapidly...

Nicolas is a co-founder of Orchard Therapeutics and joined the company in October 2015, as the first founding member of Orchard's management team. Nicolas brings extensive experience in Commercial and Market Access Strategy and Business Operations. Prior to joining Orchard Therapeutics, he...

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...

Dr. Nerissa Kreher is an accomplished physician executive and pediatric endocrinologist with strong biotech experience in Clinical Development and Medical Affairs. Her expertise is in the development of medications for rare diseases spanning multiple therapeutic areas. Equipped with an MBA and an...

Dr. Alex Kuta joined uniQure in January, 2017 as Senior Vice President Regulatory Affairs. He joined uniQure from EMD Serono where he served as Vice President of Research & Development Global Regulatory Affairs and where he was a member of the U.S. Leadership Team.  In this role, he was...

Jennifer Lambe is a Senior Project Manager in Language Services at ICON plc, Patient Centered Sciences, Commercialisation & Outcomes.Jennifer joined Mapi in 2004 as a Project Manager. Her work entails the management of Linguistic Validation projects in a wide range of disease areas including...

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...

After receiving his Ph.D. in Biochemistry and Molecular Biology at the University of Zürich, Switzerland, Prof. Linden trained in Molecular Virology at Cornell University Medical College, N.Y. After his postdoctoral training he joined the faculty at Mount Sinai School of Medicine in New York, where ...

Mapi (now part of ICON) 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...

Ana Mingorance, PhD, is the Chief Development Officer of the Loulou Foundation, the Scientific Director of the Dravet Syndrome Foundation Spain, and an independent consultant in genetic epilepsy syndromes and orphan drug development. A neuroscientist by training, Ana first worked in drug discovery...

Eva Molero, Founder & CEO of Synapse Research Management Partners, a company specialised in creating value in collaborative innovative partnerships. University degree in Law by University of Barcelona, Master Degree in Communications Management by University of Toulouse. She developed her...

Marisol Montolio is Biologist and PhD in Neuroscience at the University of Barcelona. In 2006, she moved to Barcelona's Biomedical Research Park where she specialized in the identification of drugs for neurological diseases, including genetic and degenerative diseases.Subsequently, she...

Dirk brings extensive industry experience to Blue Matter. His background spans 25+ years, with senior commercial roles in leading biopharmaceutical companies such as Shire, Roche / Genentech, and Amgen. His specialties include commercial strategy development, lifecycle management, and...

William C. Motel, Ph.D.Associate Director of Regulatory Affairs, Orphan Drug UnitIQVIA William delivers expert advice and strategic support to biopharmaceutical & biotechnology companies to assist them with the development and valuation of their assets. At IQVIA, he provides leadership to the...

Karl-Johan (Kalle) is Head of Patient Access at Wilson Therapeutics as of April 2018. In his role, he coordinates and leads the global pricing and patient access activities for WTX101, an investigational compound in phase III. Prior to joining Wilson Therapeutics, Kalle headed up the global Health...

Dr. Alexander Natz, LL.M. (Duke) 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...

Tuyen has served as chief development officer of Nightstar since August 2017. Prior to Nightstar, Tuyen served as chief medical officer at PTC Therapeutics Inc. (“PTC”) where he was responsible for advancing the company’s pipeline of product candidates for the treatment of rare diseases. Prior to...

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...

Prior to joining Zealand, Andrew was general partner and Scientific director of Eclosion2 & Cie SCPC in Switzerland. CEO of Arisgen SA, an Eclosion2 portfolio company developing an oral peptide drug delivery technology. Andrew has more than 20 years’ experience in international...

Ms. Pitluck is the Head of Global Pricing & Reimbursement (P&R) at Spark Therapeutics, Inc., a fully integrated gene therapy company committed to meeting the needs of patients living with genetic diseases.  She has been involved with healthcare market access issues including...

Silvia has more than 20 years international experience in the biopharmaceutical industry and began her career in academy gaining a PhD in Immunology followed by a postdoctoral in infectious diseases at the Medical Research Council in London.  She then decided to join a start-up biotech company...

Sam Rasty joined Homology Medicines in January 2016 as Chief Operating Officer and helped launch the company along with former management colleagues from Shire Pharmaceuticals as a next-generation gene editing and gene therapy biotech start-up in April 2016.  He was previously Vice President and...

Kirsty is the Senior Manager for Science Policy and Animal Welfare at EFPIA. Kirsty coordinates activities within EFPIA on the IMI-PARADIGM project which EFPIA co-leads and she is involved in supporting the implementation of EFPIA's strategy on Patient Engagement. In addition she focuses on...

Owen is the Chief Executive officer of both Nobelex Biotech Inc and M4K Pharma. Prior to founding M4K Pharma in 2017 and Nobelex in 2014, he was a co-founder of Affinium Pharmaceuticals, a privately held, clinical stage, antibiotic company.  At Affinium, he was the CFO and led the company’s...

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...

Greg has served as our chief scientific officer since August 2016. Prior to Nightstar, Greg was chief scientific officer at Agilis Biotherapeutics LLC, a company focused on developing gene therapies for rare CNS disorders. Prior to Agilis, he was a member of the discovery research group and served...

Jerome Rossert is Vice President General Medicine, Global Clinical Development at Vertex Pharmaceuticals. In his role, Jerome supports the clinical development of programs targeting rare diseases, such as a gene editing program that Vertex Pharmaceuticals is developing in collaboration with CRISPR...

Dr Luca Sangiorgi helds a Medical degree from the University of Bologna, a PhD in Clinical Genetics at “La Sapienza” University of Rome and a Master Degree in Research Promotion and Governance in Hospital Trusts and Local Health Units at the University of Modena and Reggio Emilia.He has been...

Reto Andreas Schaberl joined Ewopharma 8 years ago and is heading the company’s business development and specialty pharma business and is as well responsible for market access in 15 CEE countries and Switzerland. He has 15 years experiences in developing and setting up new businesses, in particular ...

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...

Yvonne Schmidt is a scientific advisor at the Federal Joint Committee (G-BA) in Berlin, Germany.She studied biology at the Free University of Berlin and the Jagiellonian University of Kraków, and obtained her PhD at the University Hospital Charité Berlin. Following three years of postdoctoral...

Sophie is President and Managing Partner at Partners4Access (P4A), the global access experts for orphan drugs, cell and gene therapy. She brings a wealth of commercial and access experience across orphan and cell and gene therapies to the benefit of clients. She plays an active role in advising...

Ad Schuurman MSc MBA, Senior Advisor International Affairs with the National Health Care Institute (ZIN) in the Netherlands, President of the Medicine Evaluation Committee (MEDEV) in Brussels and Senior Medical Advisor with the European Medicines Agency (EMA) in London. Ad Schuurman manages...

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...

Daniela is a biologist and health economist graduated at Catholic University of Sacred Heart in Rome. Her responsibility covers the monitoring of pharmaceutical consumption and expenditure at national and regional level in Italy, the monitoring of consumption and accessibility of orphan drugs, the...

Nick Sireau PhD is Chair and CEO of the AKU Society, a medical charity that works to find a cure for and support patients with AKU (also called Black Bone Disease), an ultra rare disease that affects his two sons (www.akusociety.org).  The AKU Society is a fast-growing patient movement, with formal ...

David Sourdive, Ph.D., is a co-founder of Cellectis and joined the Board of Directors in 2000. Dr. Sourdive holds the position of Executive Vice President, Technical Operations, with the mission to develop the Company’s industrial and technological basis as well as to deploy its...

Kári Stefánsson, M.D., Dr. Med. founded deCODE in 1996. Dr. Stefánsson was a professor of Neurology, Neuropathology and Neuroscience at Harvard University and Director of Neuropathology at Beth Israel Hospital in Boston, Massachusetts from 1993-1997. From 1983 to 1993, he held faculty positions in...

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...

Dr. Josep Torrent-Farnell. PhD in Medicine and Surgery and Bachelor of Pharmacy, specialist in internal medicine and clinical pharmacology. Head of the medicine division of the CatSalut, of the Generalitat de Catalunya (May 2016). Full Professor of the Department of Clinical and Therapeutic...

Dr Marie Trad, M.D.Executive Medical Director, Strategic Drug Development, IQVIADr Trad is a Senior Neurologist and Neuroradiologist with focused expertise in multiple sclerosis (MS), Parkinson’s Disease (PD), Amyotrophic Lateral Sclerosis (ALS) and other Neuro-orphan diseases. She has 29 years of...

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...

Giuliana Vallanti is Development and Quality Control Director and Qualified Person in MolMed. She joined MolMed in 2005 and worked with growing responsibilities in Development  and Quality Control units, contributing to the development of processes and assays in cell and  gene therapy field. She...

Dr. Sander van Deventer joined uniQure as Chief Scientific Officer, General Manager, Amsterdam in August, 2017.  Dr. van Deventer was a co-founder of uniQure's predecessor Amsterdam Molecular Therapeutics (AMT) in 1998 and Board member since 2010.  Dr. van Deventer is currently Operating...

Andreas Vogel, Ph.D., is Senior Director, Regulatory Science at bluebird bio, a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies. A developmental biologist by training, he joined the pharmaceutical...

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 ...

Mary Wang, PhD is the Global Partnerships & Project Manager at Fondazione Telethon, Italy.Mary has been coordinating efforts in developing tools to link and share rare disease biobank data internationally since 2014, in the context of RD-Connect. She also supports EuroBioBank coordination;...

Dr. Richard Wenzel is a principal in the Life Sciences Practice at CRA’s Munich office with more than four years of consulting experience. He specializes in strategy and launch preparation of innovative therapies and devices, developing fact-based commercial and business models with measurable...

After graduating from the University of Rome La Sapienza in 2001 with highest honours, she obtained a PhD in Pharmacology, Pharmacognosy and Toxicology (in 2005), a postgraduate diploma (specialization) in Pharmacology (in 2009) and two Master’s Degree in Drug Development (in 2010) and Health...

Stefano has been working in  clinical research  for more than 20 years, mainly in GSK but also in Novartis and Aptuit, managing a portfolio of clinical projects and  leading a group of scientists accountable for the end to end operational management of the assigned clinical studies from concept...

Martine Zimmermann, Senior Vice President Head 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...