Innovative Ways to Support Nano-rare | 2024 Nano-rare Patient Colloquium скачать в хорошем качестве

Innovative Ways to Support Nano-rare | 2024 Nano-rare Patient Colloquium

As n-Lorem scales to meet an ever-growing demand, new challenges arise. Many of these challenges will take innovative solutions and support from leaders and experts in a variety of different fields. Together as a community, we are creating a path forward for nano-rare patients and changing the way that genetic diseases are treated. This panel occurred during the n-Lorem Foundation's 2024 Nano-rare Patient Colloquium, hosted by Biogen, on October 31 in Cambridge, MA. https://www.nlorem.org/nano-rare-pati... About: Natacha Gassenbach joined Biogen in October 2018 and is the Senior Vice President, Corporate Affairs, and Chief Communications Officer. She leads the global corporate affairs function, including external and internal communications, branding and reputation, corporate responsibility and patient advocacy. She also has responsibility for the Biogen Foundation and philanthropy. Prior to joining Biogen, she was head of corporate affairs at Baxalta (acquired by Shire), Novartis Vaccines and Diagnostics, and Boston Scientific. Paul Compton is Chairman of Investment Banking for Barclays, based in New York. Previously, Paul served as the Global Head of Corporate & Investment Bank (comprising of Investment Banking, Markets and Corporate Banking), and President of Barclays Bank PLC (BBPLC), leading the provision of funding, financing, strategic advice and risk management for financial institutions, money managers, governments, supranationals and corporate clients. Andrew W. Lo is the Charles E. and Susan T. Harris Professor at the MIT Sloan School of Management, director of MIT’s Laboratory for Financial Engineering, and principal investigator at MIT’s Computer Science and Artificial Intelligence Laboratory. His healthcare-related research interests include: new financial engineering tools and business models for drug and device development and healthcare delivery, especially for rare and ultra-rare diseases; statistical methods for incorporating patient preferences into the drug approval process; predicting clinical trial outcomes via machine learning techniques; and novel reimbursement models for creating a robust gene and cell therapy ecosystem. He is a co-founder of BridgeBio Pharma, QLS Advisors, Quantile Health, and Uncommon Cures; a director of AbCellera, Atomwise, BridgeBio, Uncommon Cures, and Vesalius; and a member of the advisory board to the American Cancer Society’s BrightEdge Impact Fund. Lo received his B.A. in economics from Yale University and his A.M. and Ph.D. in economics from Harvard University. John Maraganore is the CEO and Principal of JMM Innovations, LLC, committed to the advancement of biomedical innovation to patients. Maraganore served as the founding CEO and a Director of Alnylam from 2002 to 2021, where he built and led the company from early platform research on RNA interference through global approval and commercialization of the first five RNAi therapeutics, creating a whole new class of medicines. At Alnylam, he built one of the biotech industry’s most successful companies with over $25B in market capitalization during his tenure. David Pearce is the chair of the consortium assembly for the International Rare Diseases Research Consortium (IRDiRC) that represents funding agencies, companies, and patient advocate groups that from approximately 80 countries. 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 Rochester, U.S., and Oxford University, UK. Dr. Pearce is an internationally recognized leader in a number of areas. He has published over 100 research papers on Batten disease. He founded an international registry for rare diseases known as the Coordination of Rare Diseases at Sanford (CoRDS). He has served on numerous NIH review committees, rare disease advisory boards and has organized rare disease workshops for the National Institute for Neurological Disorders and Stroke (NINDS) arm of the National Institutes of Health (NIH). He has presented at numerous international meetings on rare disease, precision medicine, and use of electronic medical records for biomedical research.