As science has enabled care for previously untreatable, ultra-rare conditions, health systems and governments are struggling with ways of financing research and development of these therapies. What are the solutions when the science has progressed, but the economics haven't?
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; statistical methods for incorporating patient preferences into the drug approval process; predicting clinical trial outcomes via machine learning techniques; and novel funding and reimbursement models for creating a robust gene therapy ecosystem. He is a co-founder and director of BridgeBio Pharma, a director of AbCellera, Atomwise, and Vesalius, a co-founder and chairman of QLS Advisors, and a member of the advisory board to the American Cancer Society’s BrightEdge Impact Fund. Dr. Lo received his B.A. in economics from Yale University and his A.M. and Ph.D. in economics from Harvard University.
Philip J. (P.J.) Brooks is the acting director of NCATS’ Division of Rare Diseases Research Innovation. He is also the co-coordinator for the NIH Common Fund program on Somatic Cell Genome Editing, one of the leaders of the Platform Vector Gene Therapy (PaVe-GT) pilot project PaVe-GT (nih.gov) and the co-chair of the Bespoke Gene Therapy Consortium AMP® Bespoke Gene Therapy Consortium (BGTC) | FNIH. He also represents NCATS in the International Rare Diseases Research Consortium (IRDiRC) IRDiRC - International Rare Diseases Research Consortium.
In May 2022, Brooks was selected as the recipient of the 2022 Sonia Skarlatos Public Service Award(link is external) by the American Society of Gene & Cell Therapy for consistently fostering and enhancing the field of gene and cell therapy.
Sarah received her Ph.D. in Molecular Genetics at Ohio State University where she trained in rare inherited cancer syndromes. She has over 20 years of experience in clinical development and research across academia, pharmaceutical companies, and CROs. Sarah brings significant strengths and experience as an accomplished research geneticist, rare disease drug developer, and clinical trialist. She is acclaimed for forging key strategic partnerships across rare disease sectors and has driven efficiencies to decrease patient/ caregiver burden in clinical research. Most notably, Sarah combines the professional expertise and training with the perspective of a parent. This allows Sarah to not only personally understand the challenges faced by our patients and their families, but also to translate this understanding into n-Lorem’s paradigm-shifting platform solution for the ultra-rare community.
Nessan is an Operating Partner at Khosla Ventures focused on life science companies with an emphasis on nucleic acid editing, novel delivery systems, gene and cell therapy, novel target identification and data analytics for drug discovery and development.
Having founded multiple companies in the life sciences realm, Nessan is a serial biotech entrepreneur and investor. Most notable among his companies are Intellia Therapeutics, a top-10 biotech startup in 2014, Triplet Therapeutics, and Korro Bio. At Intellia, Nessan served as president and CEO from inception through 2017, leading the company from concept through partner deals, multiple financing rounds and an IPO prior to its second anniversary.
Nessan also held investment roles at Atlas Venture and Omega Fund Management, where he successfully invested in and managed multiple investments across the United States and Europe. He is currently the chairman of F-star and executive chair at Everyone Medicines, Korro Bio and Stylus Biomedicine. He also currently sits on the board of CellFE, Deep Genomics, Liberate Bio and Ochre Bio and is a board observer with CHARM Therapeutics.
Previously he was a member of the equity research team at UBS, an independent advisory board member of the California Institute of Regenerative Medicine (CIRM) and MerckSerono KGaA. He received his Ph.D. in Molecular Biology from Imperial College London, and was a Howard Hughes Associate Fellow at Baylor College of Medicine.
Jared Whitlock is the features editor at Endpoints. Previously a freelance journalist and MIT knight science journalism fellow in fall 2021, his work has appeared in publications like WIRED, STAT and The New York Times. Prior to this, he was a biotech, healthcare and special projects reporter at The San Diego Business Journal. When not fussing over words, he can be found surfing at various San Diego beaches.