Mohamed Abou El-Enein is a junior professor for clinical development of re­generative therapies and head of the clinical development platform at Charité Medical University Berlin. He serves as a senior teaching assistant in clinical research at Harvard T.H. Chan School of Public Health and instructor in trans­lational medicine and regulatory science at the Berlin-Brandenburg School for Regenerative Therapies (BSRT). He is leading a multi-disciplinary team of medical and scientific professionals who are actively involved in all aspects of planning and conducting clinical trials in patients suffering from rare and de­bilitating diseases. Additionally, he has more than eight years of experience in the development and clinical translation of cell and gene therapies and a strong interest in public and global health and the equitable access to health care. He was awarded the prestigious Eisenhower Fellowship and elected as a member of the Arab-German Young Academy of Sciences and Humanities. He also acts as the regional secretary of the International Society for Cellular Therapy and is an active member of many international expert networks.

David Schwicker has biopharmaceutical consulting expertise spanning more than 25 years. He is the founder of ORPHA Strategy Consulting, based in Basle, Switzerland, and focussed on the timely patient access to orphan and advanced therapy medicinal products. This dedication to client consulting has enabled David to develop a capacity for rapid complex problem solving allowing him to provide creative and analytically rigorous advice on strategic challenges and to devise effective solutions to drive his clients’ programmes forward. David has longstanding scientific experience and expert contacts in rare diseases, has authored and coauthored peer-reviewed publications and is a speaker at inter­national meetings. He is an accomplished trainer in rare diseases, orphan, gene and cell therapy medicinal products, and leads Masterclasses as well as cus­tomised in-house training workshops. As VP with PAREXEL International in the US, David was responsible for registries, outcomes research, market access and expanded access programs. Since returning to Europe, David has designed and implemented real-world studies including more than 200,000 patients. From 2010, David’s capabilities have expanded to rare diseases, including acute my­elogenous leukaemia (AML), multiple myeloma (MM), neuroendocrine tumors, pulmonary arterial hypertension (PAH), Niemann Pick disease, acromegaly, graft versus host disease (GvHD), primary sclerosing cholangitis (PSC), Mu­copolysaccharidosis (MPS), Myasthenia Gravis (MG), Graves Orbitopathy (GO), Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), Antibody-Mediat­ed Organ Rejection (AMR), Narcolepsy and Friedrich’s ataxia. David is currently working in rare autoantibody-mediated diseases, oncology/hematology, meta­bolic, CNS and GI indications. David has developed a unique understanding of accelerating marketing authorisation and market access in the hyper-dynamic European environment. Based on this, David supports clients in the attain­ment of “fast to market” objectives by integrating rare disease development, regulatory and market access strategies. This involves indication prioritisation, evidence generation planning (RCTs and RWE), patient engagement and PROs, orphan designation, compassionate use programs, product differentiation and early value demonstration with fewer data. A crucial component of early access strategy is the application of innovative regulatory, health technology assess­ment, pricing and reimbursement pathways that leverage the use of real-world evidence (RWE).

Associate Professor Matthias Klugmann works as Lead Vectorology Gene Ther­apy at Takeda in Vienna. He is also Head of the CNS Gene Therapy Research Group in the Translational Neuroscience Facility at UNSW Sydney. Matthias re­ceived his Ph.D. in Neuroscience from Heidelberg University. He has published 90 peer- reviewed research articles attracting over 7000 citations and has 20 years of work experience in the space of in vivo gene therapy with the AAV gene delivery platform both in academia and biopharma.

Dr. Lutz Müller is a project leader in toxicology with F. Hoffmann-La Roche in Basel, Switzerland. The focus of his work is on projects in R&D in therapeutic areas such as nervous system, cardiovascular, inflammation, immunology, oncology, and rare diseases. These span small molecules, peptides, antisense molecules, and biologicals. In terms of scientific expertise, genotoxicity, impurities, carcinogenicity, immunotoxicity, in vitro systems, risk assessment, regulatory toxicology are his primary reference areas.

Dr. Müller has been involved in the ICH guideline process since its inception in the early nineties of the last century. As disease therapeutic area liaison for NORD (Neuroscience, Rare Diseases, Ophthalmology) in pharma research and early development (Roche pRED), Dr. Lutz Müller supports the overall strategy of the company in developing new medicines in these areas. Scientific publication record: More than 100 original publications in peer reviewed journals and various book chapters. Organiser/lecturer of/on multiple international meetings and workshops.

In his current position, Dr. Olaf Ritzeler seeks and executes external R&D op­portunities to address the Sanofi Group´s strategic priorities. He helps to define the external innovation strategy and perform global external searches and as­sessments in the therapeutic area of rare diseases. He is working in a global transversal team to which he brings his strong expertise on drug discovery and development he gained over 20 years working in pharmaceutical industries and his deep insights in the European scientific ecosystem. In addition, he is a lecturer on the subject of target and compound profiling and has led Sanofi’s multi-unit collaboration with Charité, Germany. The research teams led by him delivered one phase IIb-, two phase I-, five preclinical- and six lead candidates. His contribution to Sanofi’s drug pipeline was awarded by the “Meilenstein Pre­is” and “Drug Hunter Award” for outstanding efforts and key contribution to drug innovation and approval.

Kieran Rooney has over 30 years of experience in the biopharma industry, with significant expertise in building biopharma businesses, corporate strategy and business development. Before joining Amryt, he founded Halo BioConsulting, a global healthcare advisory services firm focussed on business alliances and strategy consulting. He consulted to over 30 biopharma and professional servic­es companies including many of the top 10 global pharma companies, multiple biotech companies and he was a strategy and transactions advisor to PwC. Prior to this, he held business development roles across a range of life science, phar­ma and biotech companies and worked as a consultant for the UK government.

Kieran holds a PhD in neuropharmacology from the University of Wales, Cardiff, and a BSc in pharmacy from University of Sunderland.

Markus Peters, PhD MSc, is an experienced global biopharmaceutical execu­tive with a 23-year track record of developing and commercialising innovative, high-value orphan and specialty pharmaceuticals for patients with high unmet medical need. At present, Markus is an executive-level strategic consultant for biopharmaceutical companies. Before that, Markus was chief commercial of­ficer with Agilis Biotherapeutics, a biopharmaceutical company advancing gene therapies for patients with rare diseases of the central nervous system. In this role, he had responsibility for market access, medical affairs, marketing, diag­nostic field services, sales and business development, including the preparation for the launch of AGIL-AADC, the first gene therapy for ultra-rare AADC defi­ciency. Agilis was acquired by PTC Therapeutics in August 2018. Before that, he was VP of global marketing with Synageva BioPharma, and senior director of global marketing with Alexion Pharmaceuticals, with responsibility for the launches of two ultra-orphan franchises (Kanuma for LAL deficiency and Soliris for atypical HUS). Prior to that, Markus led the global launches of the ENBREL franchises for Wyeth/Pfizer (>$5 billion sales). He also worked five years in Ja­pan with Boehringer Ingelheim in commercial roles of increasing responsibility. Markus is a leader in sizing and building of global biotech organisations with comprehensive operational responsibility (Agilis, Synageva, Alexion), hiring and leading key organisational functions located across the globe. He has extensive operational experience on three continents: North America (US), Europe (Ger­many and UK) and Asia (five years in Japan; China). As commercial lead, he supported corporate deals, including the $8.4-billion acquisition of Synageva by Alexion, at the time the fourth-largest deal in the industry, and the acquisition of Agilis by PTC Therapeutics, a deal valued at about $1 billion ($200 million upfront with about $800 million future milestone payments). Markus has expe­rience across a broad range of therapeutic areas, leading pipeline commerciali­sation for AAV gene therapy in neurology (Agilis); therapies for inborn errors of metabolism, hepatology (Synageva); transplant, nephrology and immunology (Alexion); endocrinology (Merck); and oncology (Wyeth/Pfizer). Markus holds a PhD in biochemistry and a MSc in chemistry (Dipl.-Chem.) from Heinrich-Heine University Düsseldorf, Germany.

Dr. Micah Rose’s primary role at scientific advice is the validation and verifica­tion of economic models. His ancillary roles include the critical appraisal of clinical and economic evidence, and providing advice on evidence generation and synthesis for health technology assessment (HTA) processes. Previous to joining scientific advice, Micah was a health economic researcher in two evi­dence review groups (ERGs) for NICE technology appraisal (TA) programmes, including: single technology appraisals (STA), multiple technology appraisals (MTA), diagnostics assessment programme (DAP) and highly specialised tech­nologies (HST) across a variety of disease areas. Micah earned his MSc in health economics and health policy from the University of Birmingham, earning a degree with distinction.

Leung Ming Yu is a health economist and pharmacist (MSc) and has been working at the Norwegian Medicines Agency (NOMA) for over 10 years. His responsibilities in NOMA have been pricing and reimbursement, generic substitution, and single technology assessments (STA). Because of the recent reorganization of NOMA, Leung Ming Yu is now also involved in scientific advice. His primary role is the critical appraisal of clinical and economic evidence of STAs. He also worked on the Spinraza case in Norway, the first drug considered under the new “super-orphan” criteria for reimbursement i.e. extremely severe condition (> 30 QALYs lost), very small population (1/100 000 worldwide), significant expected benefit (> 2 QALYs gained). Leung Ming Yu will be discussing these criteria in his talk in Berlin.

Dr. Leone Atkinson is a neurologist with a doctorate in medical genetics, special­ising in clinical development for rare disease. She has over 16 years of experi­ence, working in pharmaceutical, biotechnology and CRO environments. She has provided scientific and medical oversight for rare disease indications across therapeutic areas, including Duchenne muscular dystrophy, fragile X syndrome, mucopolysaccharidosis IV, Pompe disease, progressive supranuclear palsy, Fab­ry disease, acute intermittent porphyria and others. Before joining Covance, Leone served as a medical consultant for biopharmaceutical companies and the National Institutes of Health. From 2007-2010, she served as executive direc­tor of clinical development at PTC Therapeutics, where she was responsible for the global clinical development program for ataluren in Duchenne muscular dystrophy and other rare genetic disorders. Prior to that, she served as a clini­cal research director at Sanofi-Aventis, and served as associate medical director of neuroscience at Novartis Pharmaceuticals. Leone received her MD and PhD in medical genetics from the University of British Columbia in Vancouver, BC, Canada. She completed a residency in neurology and a post-doctoral fellowship at Mount Sinai Medical Center in New York, NY.

Dr. Lincoln Tsang, formerly a senior regulator, concentrates his practice on regulatory, compliance, enforcement and public policy matters affecting the life sciences industry. He has extensive experience in advising clients in develop­ing strategies for research and development, product life-cycle management, product acquisition and risk and crisis management, as well as internal investi­gations and litigation. He regularly represents clients before various regulatory bodies as well as legislative bodies including those in the EU and the US.

Raghuram “Ram” Selvaraju is currently a senior healthcare equity research ana­lyst and managing director at H.C. Wainwright & Co., LLC, a leading health­care-focussed investment bank headquartered in New York City, where he has worked since mid-2015. He was previously a senior healthcare equity research analyst and managing director at MLV & Co., LLC, which is now part of B. Riley/ FBR. Prior to MLV, Raghuram was head of healthcare equity research at Aegis Capital Corp. for roughly three years. Before joining Aegis, he held positions as senior biotechnology analyst at Morgan Joseph TriArtisan LLC and Noble Financial Group, as well as an appointment as senior VP and head of healthcare equity research at Hapoalim Securities U.S.A., Inc., the New York-based bro­ker/dealer subsidiary of Bank Hapoalim B.M., Israel’s largest financial services group. He possesses a total of nearly two decades’ worth of experience in the biotech and pharmaceutical sectors. Raghuram started his career in 2000 at the Serono Pharmaceutical Research Institute in Geneva, Switzerland, the applied research arm of Serono, Europe’s largest biopharmaceutical firm at the time. During his time in drug development, he was a highly decorated young scien­tist, winning both corporate awards and broader recognition within the aca­demic community. He began his equity research analyst career on Wall Street in 2005 at Rodman & Renshaw. Raghuram has been quoted in such publications as The Wall Street Journal, MSN Money, Barron’s, Bloomberg Businessweek, Reuters, Streetwise Reports, BioWorld Today, The Pink Sheet, PharmaVoice, The Wall Street Transcript and BioCentury, and has also made multiple appear­ances on Bloomberg TV, Bloomberg Radio and CNBC, discussing biotech and pharmaceuticals stocks and the outlook for the healthcare industry. He has been ranked by StarMine based on earnings estimate accuracy and his team at Rodman & Renshaw was ranked No. 1 in The Wall Street Journal’s annual “Best On The Street” survey among biotechnology analysts based on overall portfolio return. He has published highly-regarded sector reports on Alzheimer’s disease, multiple sclerosis, stroke, orphan neurological disorders and the most compre­hensive Wall Street research produced to date on U.S. healthcare reform policy. Among his academic qualifications, he holds a bachelor’s in biological sciences and technical writing from Carnegie Mellon University, a doctorate in cell biology and neuroscience and a MSc in molecular biology from the University of Ge­neva in Switzerland and an MBA from Cornell University’s accelerated one-year program for scientists and engineers. He has also published articles in leading peer-reviewed journals, presented research at various scientific conferences, and is a co-inventor on several drug development patents.

Juliet Hulse was promoted to the role of director of Research Nursing in 2016, having worked as a Research Nurse team manager and, prior to that, in a site coordinator/senior Research Nurse capacity. She has worked for the company since 2011 and has over 15 years’ experience within clinical research, com­mencing her career in cardiology research covering multiple phase I-IV trials within the NHS. Juliet more recently specialised in neurology research, in par­ticular the field of multiple sclerosis, working on both commercial and non-com­mercial phase I-IV trials at the University of Nottingham, UK. In Europe, Juliet has worked on trials for large pharmaceutical as well as biotechnology compa­nies. She has wide experience in the management of trial projects from initia­tion to close-out as well as working with site investigators to improve patient recruitment and patient retention to the study protocols. Juliet’s nursing skills are broadened to include ECG analysis, venepuncture, cannulation, advanced neurological assessment and the rapid response to complex adverse events. Her own clinical skills have supported expanding and developing the Research Nurse project management team widely. For the last five years her main focus has been the strategic development of the Research Nurse Services globally, being a primary point of contact with client companies and external organisa­tions. Juliet’s skill in finding solutions to complex project difficulties means the wider nursing team feels supported and confident in the delivery of research studies. She believes developing a strategy that places the patient in the centre of the trial is vital for a successful study. The coordination and management of the off-site research nursing team for numerous studies has been rewarding and successful for both Illingworth and the clients involved.