An invitation-only workshop jointly organized by UC Santa Barbara (UCSB) Neuroscience Research Institute and the MIT Laboratory for Financial Engineering (LFE).
Meeting Co-Hosts: Professor Kenneth S. Kosik, UCSB; and Professor Andrew W. Lo, LFE
Date: February 1, 2018
Location: Mosher Alumni House, UC Santa Barbara, Santa Barbara, CA 93106
The aim of this workshop is to explore innovative approaches to developing new therapeutics and technologies for the treatment of Alzheimer’s disease, and new financing structures and business models for addressing the associated funding challenges in a scalable and profitable manner. Participants will have the opportunity to learn more about specific Alzheimer’s-related projects and R&D efforts, and biomedical experts will have the opportunity to learn more about funding opportunities and potential financing methods for attracting capital.
The Problem
Compared to many other diseases, Alzheimer’s is considerably more challenging because new therapies and technologies for neurodegenerative diseases are more expensive to develop, take longer, require a larger sample of patients, and are generally more complex. And in an environment in which funding for biomedical research is declining, Alzheimer’s-related R&D becomes even more difficult.
The Hypothesis
The so-called “Valley of Death” in translational medicine is the result of many causes, but a common thread among them is increasing financial risks in the biopharma industry and greater uncertainty surrounding the economic, political, and academic environments within the biomedical ecosystem. Increasing risk and uncertainty inevitably leads to an outflow of capital as investors and other stakeholders seek more attractive opportunities in other industries.
The Proposal
By spreading the risk of translational medicine across a much larger pool of investors and increasing the number of “shots on goal”, the risk to all investors is lowered and the chances of one or two successes is much higher. If several research organizations were able to pool their biomedical intellectual property, they might be able to attract much larger amounts of capital even for highly speculative but transformative early-stage R&D.
This meeting will bring together a multi-disciplinary group of stakeholders and key thought leaders from academic medical centers and university research laboratories, financial institutions, foundations and endowments, pharma and biotech, non-profits and patient advocacy organizations, and government to engage in active dialogue regarding specific funding needs, potential new business and legal structures, and new sources of capital to fund such enterprises. Topics will include:
- Innovative scientific directions that deserve more attention/funding or are not currently being pursued
- The current funding challenges in Alzheimer’s research and drug development
- Potential new business and financing structures to address these challenges
Objectives
To explore innovative approaches to Alzheimer’s research and drug development, and develop a comprehensive and actionable plan for implementing them.
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Christopher P. Austin
Director, National Center for Advancing Translational Sciences
Christopher Austin is Director of the National Center for Advancing Translational Sciences (NCATS) at the U.S. National Institutes of Health. NCATS’ mission is to catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions. Before becoming NCATS Director in September 2012, he was Director of the NCATS Division of Preclinical Innovation, which focuses on translating basic science discoveries into new treatments, particularly for rare and neglected diseases, and developing new technologies and paradigms to improve the efficiency of therapeutic and diagnostic development. In this role, he founded and directed numerous initiatives including the NIH Chemical Genomics Center (NCGC), the Therapeutics for Rare and Neglected Diseases (TRND) program, and the Toxicology in the 21st Century (Tox21) program. In 2016, Dr. Austin was elected chair of the International Rare Disease Research Consortium (IRDiRC). Before joining NIH in 2002, Dr. Austin directed research programs genomics-based target discovery, pharmacogenomics, and neuropsychiatric drug development at Merck, with a particular focus on schizophrenia. Austin earned an A.B. in biology from Princeton University and an M.D. from Harvard Medical School. He completed clinical training in internal medicine and neurology at Massachusetts General Hospital, and a research fellowship in genetics at Harvard.
David Baltimore
Robert Andrews Millikan Professor of Biology and President Emeritus, Caltech
After serving as President of the California Institute of Technology for nine years, David Baltimore was appointed President Emeritus and the Robert Andrews Millikan Professor of Biology in 2006. Awarded the Nobel Prize at the age of 37 for research in virology, Dr. Baltimore has profoundly influenced national science policy on such issues as recombinant DNA research and the AIDS epidemic. He is an accomplished researcher, educator, administrator and public advocate for science and engineering and is considered one of the world’s most influential biologists.
Born in New York City, Baltimore became interested in biology during high school when he spent a summer at the Jackson Memorial Laboratory and worked with research biologists on mammalian genetics. He received his B.A. in Chemistry from Swarthmore College in 1960 and a Ph.D. in 1964 from Rockefeller University, where he returned to serve as President from 1990-91 and faculty member until 1994.
For almost 30 years, Dr. Baltimore was a faculty member at Massachusetts Institute of Technology where his early investigations examined the molecular processes underlying the ability of poliovirus to infect cells. This led him to work on other RNA viruses and then to a consideration of how cancer-causing RNA viruses manage to infect and permanently alter a healthy cell. He identified the enzyme reverse transcriptase in the virus particles, thus providing strong evidence for a process of RNA to DNA conversion, the existence of which had been hypothesized some years earlier. Dr. Baltimore and Howard Temin (with Renato Dulbecco, for related research) shared the 1975 Nobel Prize in Physiology or Medicine for their discovery, which provided the key to understanding the life-cycle of retroviruses such as HIV. In the following years, he has contributed widely to the understanding of cancer, AIDS and the molecular basis of the immune response.
His present research focuses on control of inflammatory and immune responses, on the roles of microRNAs in the immune system and on the use of gene therapy methods to treat HIV and cancer in a program called “Engineering Immunity”. He has become Director of the Joint Center for Translational Medicine, an activity that joins Caltech and UCLA in a program to translate basic science discoveries into clinical realities and where an active clinical program is under way.
Dr. Baltimore has several outstanding administrative and public policy achievements to his credit. In the mid-1970s, he played an important role in creating a consensus on national science policy regarding recombinant DNA research. He served as founding director of the Whitehead Institute for Biomedical Research at MIT from 1982 until 1990. An early advocate of federal AIDS research, Dr. Baltimore co-chaired the 1986 National Academy of Sciences committee on a National Strategy for AIDS and was appointed in 1996 to head the National Institutes of Health AIDS Vaccine Research Committee. Dr. Baltimore served as a member of the Independent Citizen’s Oversight Committee to the California Institute for Regenerative Medicine until 2007 and on the Board of Directors for both MedImmune until 2007 and Cellerant until 2008.
He has played an important role in the development of American biotechnology since his involvement in the 1970’s in the formation of Collaborative Genetics. He helped found other companies such as Calimmune and Immune Design and he presently serves on the Board of Directors at several companies and non-profit institutions including the Broad Foundation and Broad Institute, and Amgen and Regulus Therapeutics. He is a member of numerous Scientific Advisory Boards, including the Broad Institute, Ragon Institute, Regulus Therapeutics and Immune Design. He is a Scientific Partner to the venture capital firm, The Column Group, and was a Director of the Swiss investment company BB Biotech through 2011.
Dr. Baltimore’s numerous honors include the 1970 Gustave Stern Award in Virology, 1971 Eli Lilly and Co. Award in Microbiology and Immunology, 1999 National Medal of Science, and 2000 Warren Alpert Foundation Prize. He was elected to the National Academy of Sciences in 1974, and is also a fellow of the American Academy of Arts and Sciences, and a foreign member of both the Royal Society of London and the French Academy of Sciences. He is past-President and Chair of the American Association of the Advancement of Science (2007-2009) and was mostly recently named a Fellow of the American Association for Cancer Research (AACR). He has published more than 700 peer-reviewed articles.
Ed Boyden
Professor of Biological Engineering and Brain and Cognitive Sciences, MIT
Ed Boyden is a professor of Biological Engineering and Brain and Cognitive Sciences at the MIT Media Lab and the MIT McGovern Institute. He leads the Synthetic Neurobiology Group, which develops tools for analyzing and repairing complex biological systems such as the brain, and applies them systematically to reveal ground truth principles of biological function as well as to repair these systems. These technologies include expansion microscopy, which enables complex biological systems to be imaged with nanoscale precision, and optogenetic tools, which enable the activation and silencing of neural activity with light, amongst many other innovations. He co-directs the MIT Center for Neurobiological Engineering, which aims to develop new tools to accelerate neuroscience progress.
Amongst other recognitions, he has received the Breakthrough Prize in Life Sciences (2016), the BBVA Foundation Frontiers of Knowledge Award (2015), the Carnegie Prize in Mind and Brain Sciences (2015), the Jacob Heskel Gabbay Award (2013), the Grete Lundbeck Brain Prize (2013), the NIH Director’s Pioneer Award (2013), the NIH Director’s Transformative Research Award (twice, 2012 and 2013), and the Perl/UNC Neuroscience Prize (2011). He was also named to the World Economic Forum Young Scientist list (2013), the Technology Review World’s “Top 35 Innovators under Age 35” list (2006), and is an elected member of the American Academy of Arts and Sciences (2017).
His group has hosted hundreds of visitors to learn how to use new biotechnologies, and he also regularly teaches at summer courses and workshops in neuroscience, and delivers lectures to the broader public (e.g., TED (2011); TED Summit (2016); World Economic Forum (2012, 2013, 2016)). He received his Ph.D. in neurosciences from Stanford University as a Hertz Fellow, where he discovered that the molecular mechanisms used to store a memory are determined by the content to be learned. Before that, he received three degrees in electrical engineering, computer science, and physics from MIT. He has contributed to over 300 peer-reviewed papers, current or pending patents, and articles, and has given over 300 invited talks on his group’s work.
Katja Brose
Science Program Officer, Chan Zuckerberg Initiative
Katja Brose, Ph.D., is a Science Program Officer at the Chan Zuckerberg Initiative. The goals of Chan Zuckerberg Science Initiative are to support basic science and technology that will make it possible to cure, prevent, or manage all diseases by the end of the century. Before joining CZI, she was part of the editorial team at Cell Press for 17 years, where from 2004-20017 she was Editor-in-Chief of Neuron and a Publishing Director at Cell Press-Elsevier. During her tenure as Editor, Neuron undertook a major expansion of its scope building on its historical strengths in molecular and cellular neuroscience to cover all areas of neuroscience from molecular/cellular mechanisms to systems and cognitive neuroscience, genetics, neurological and psychiatric disease, theoretical neuroscience and emerging technologies. As Publishing Director, she was responsible for Cell Press strategy for review content, including oversight of the Trends family of review journals. She also led Cell Press’ efforts around rigor and reproducibility. She has been an active committee member at the Society for Neuroscience, as a member of the Professional Development and Neuroscience Training Committees. She speaks frequently on topics related to scientific publishing and communication, including publication ethics and rigor and reproducibility in science. She earned her undergraduate degree in 1990 from Brown University, with a double concentration in Biology and European History. She received her PhD in Biochemistry from the University of California-San Francisco (1994-2000). For her graduate work, she worked in the laboratory of Dr. Marc Tessier Lavigne focusing on axon guidance mechanisms in the developing spinal cord.
Miyoung Chun
Former Executive Vice President of Science Programs, Kavli Foundation
Miyoung Chun’s career spans a wide range of experience in academia and industry. Her academic career began as an Assistant Professor of Biochemistry and a member of Whitaker Cardiovascular Institute at Boston University School of Medicine in 1995. There she taught in the areas of cell biology and molecular medicine, and conducted research in signal transduction of G-protein coupled receptors.
From 1999 to 2004 she worked for Millennium Pharmaceuticals Inc. as a scientist and project leader, where her research focused on genomics/functional genomics and on molecular imaging in drug discovery and development. She discovered and characterized novel genes that are important to inflammatory and cardiovascular diseases, and has over 30 U.S. and International issued/published patents.
In 2004 she moved back to academia as Assistant Dean of Science and Engineering at the University of California, Santa Barbara (UCSB), in particular serving the California Nanosystems Institute. She was also appointed Director of International Research Advancement at UCSB. In this role she was active in building partnerships among academia, government and industry around the globe.
Dr. Chun was Vice President of Science Programs at The Kavli Foundation since 2007 and was Executive Vice President of Science Programs from 2013 to 2017. She obtained her Ph.D. degree in Molecular Genetics from The Ohio State University in 1990 and was a Postdoctoral Fellow at MIT’s Whitehead Institute studying the cell and molecular biology of receptors.
Jean-Jacques Degroof
Entrepreneur and Investor
Jean-Jacques Degroof started his career in the financial services industry and, since 1999, has been an active venture investor in the Boston area and in Europe, including in the biomedical sector. During part of this period, Dr. Degroof also worked as a researcher at the MIT Industrial Performance Center. Earlier, he was a Sloan Fellow at the MIT Sloan School of Management and a BCG Fellow at the Mossavar-Rahmani Center for Business and Government at Harvard University. He obtained an M.S. and Ph.D. in Management from the MIT’s Sloan School of Management. Philanthropically, he has supported several initiatives at MIT, including the Laboratory for Financial Engineering’s application of financial tools to drug discovery and development, specifically related to Alzheimer’s disease.
Carole Ho
Chief Medical Officer and Head of Development, Denali Therapeutics
Carole Ho is the Chief Medical Officer and Head of Development at Denali Therapeutics in South San Francisco. Denali Therapeutics is dedicated to therapeutic discovery and development of treatments for neurodegenerative diseases. Prior to Denali, Dr. Ho most recently served as Vice President of Genentech Early Clinical Development. In that role, she led a team of Medical Directors responsible for delivery of pivotal trial-ready therapeutic candidates in Neurology, Ophthalmology, Immunology, and Infectious Disease and served as the Chair of the Development Review Committee. During her 8 year tenure at Genentech, Dr. Ho and her team led the successful development of multiple therapeutics that are currently in Ph3 studies or have been approved, including ocrelizumab for Multiple Sclerosis, lampalizumab for dry AMD, rituxan for Wegener’s granulomatosis and microscopic polyangiitis and crenezumab for Alzheimer’s Disease. Dr. Ho and her team also lead the Alzheimer’s Prevention Initiative with Banner Health to spearhead a landmark Alzheimer prevention study in cognitively healthy individuals who are destined to develop Alzheimer’s disease because of their genetic history.
She received her undergraduate degree in Biochemical Sciences from Harvard College and her M.D. from Cornell University. She completed her Medical Internship and Neurology residency at Harvard and served as Chief Resident of Neurology at the Massachusetts General Hospital / Brigham and Women’s Hospital. Prior to Genentech, Dr. Ho was on Faculty in the Department of Neurology at Stanford University and subsequently a Medical Director at Johnson and Johnson.
David Hung
CEO, Axovant
David Hung, M.D., founded Medivation (NASDAQ: MDVN) in October 2003, taking the company public through a reverse merger into a SPAC (Orion Acquisition Corporation) in December 2004, thereby completely bypassing the traditional venture capital funding route. Medivation was founded to develop technologies that represented true “medical innovation” in particularly challenging disease indications of high unmet need, such as Alzheimer’s disease and prostate cancer. Medivation ultimately developed and commercialized Xtandi for the treatment of metastatic and non-metastatic prostate cancer, driving the program from invention to FDA approval in 7 years, one of the fastest development times in biopharmaceutical history. By 2016, Xtandi had become the world’s leading prostate cancer therapy, with nearly $2.5 billion in annual revenue. Medivation was uniquely capital efficient in its development as Dr. Hung raised a total of $440MM in public offerings over the life of the company to ultimately reach a market capitalization of $14.3B, delivering for his investors a total return on investment of 21,000% in 13 years. Dr. Hung established partnerships with world-class pharmaceutical companies (Pfizer and Astellas) worth ~$1.5B in upfront payments and milestones, excluding profit splits and royalties, with both partners paying the majority of development costs. In October 2016, when Medivation was sold to Pfizer for $14.3B in an all-cash deal, it was the largest sale of a biopharma company ever by an actively-seated founding CEO.
Prior to Medivation, Dr. Hung was President & Chief Executive Officer of Pro•Duct Health, Inc., a venture-backed startup medical device company founded in 1998 which developed, manufactured, and commercialized a breast microcatheter — which he himself invented — for a minimally invasive procedure called ductal lavage for early breast cancer detection and risk assessment. After raising a total of $22MM in venture capital and completing a successful 507 patient high-risk breast cancer pivotal trial, Dr. Hung obtained FDA clearance for the ductal lavage catheter and sold Pro•Duct Health in 2001 for $168MM to Cytyc Corporation, the world’s leading maker of Pap smears for the cervix, delivering a 10X ROI for his Series A investors in 3 years.
Dr. Hung previously spent several years in pre-clinical research and development roles of increasing responsibility at Chiron Corporation, including as VP of New Projects and VP of Lead Discovery and Development. Earlier in his career, he was a practicing physician at the hospitals and clinics of the University of California, San Francisco, School of Medicine, holding positions of Division Chief, Transfusion Medicine Division, Attending Physician, Hematology-Oncology/Transfusion Medicine and Assistant Professor in Residence, Department of Laboratory Medicine. He also completed basic science molecular biology research fellowships at the Brain Tumor Research Center (BTRC) and the Cardiovascular Research Institute (CVRI), as well as three clinical fellowships in Hematology, Oncology and Transfusion Medicine. He has been published in dozens of top peer-reviewed basic science and clinical journals including Science, Cell, Nature, The New England Journal of Medicine and The Lancet.
Dr. Hung is the recipient of numerous distinctions including the Ernst and Young Entrepreneur of the Year Award in the Life Sciences Category as well as Overall US Winner of the Ernst and Young Entrepreneur of the Year Award in 2014 (the world’s most prestigious award in entrepreneurialism), the Pfizer Scholar’s Award (awarded yearly to the top 5 junior academic faculty in a biomedical discipline), the Hulga Irene Duggan Scholar’s Award from the Arthritis Foundation, the Arlo Guthrie Award from the Huntington’s Disease Society of America and the Outstanding Teaching Award for three years in a row from the University California, San Francisco, School of Medicine.
He received his A.B., summa cum laude, in Biology from Harvard College, and his M.D., Alpha Omega Alpha, from the University of California, San Francisco, School of Medicine.
Dr. Hung is an avid violinist (formerly the Concertmaster of the Waltham Symphony Orchestra) and pianist, tennis player, former 7-handicap golfer, and former head chef at Marco Polo, a pop-up Italian-Asian fusion restaurant he started as a summer vacation project.
Currently, Dr. Hung is the CEO of Axovant Sciences (NASDAQ: AXON) in New York City, a position he assumed in April 2017. Axovant is a late clinical stage neurology-focused biotech company with 3 different small molecule therapeutic candidates (intepirdine, nelotanserin and RVT-104) all targeting Dementia with Lewy Bodies (DLB), which accounts for up to 20% of all dementias worldwide, as well as potentially other dementias such as Alzheimer’s disease and Parkinson’s Disease. Axovant recently (September 26, 2017) announced the failure of the phase 3 MINDSET trial of intepirdine in Alzheimer’s disease, a program that was initiated nearly 2 years prior to his arrival at the company. However, Axovant continues to advance the intepirdine and nelotanserin programs in other potentially pivotal dementia studies, both with readouts in the next 6 months. Additionally, Axovant remains focused on further building out its pipeline of potential therapeutic candidates.
Terrence J. Sejnowski
Investigator, Howard Hughes Medical Institute
Francis Crick Chair, Salk Institute for Biological Studies
Distinguished Professor of Biology, University of California San Diego
Terrence J. Sejnowski is a pioneer in computational neuroscience and his goal is to understand the principles that link brain to behavior. His laboratory uses both experimental and modeling techniques to study the biophysical properties of synapses and neurons and the population dynamics of large networks of neurons. New computational models and new analytical tools have been developed to understand how the brain represents the world and how new representations are formed through learning algorithms for changing the synaptic strengths of connections between neurons. He has published over 500 scientific papers and 12 books, including The Computational Brain, with Patricia Churchland. He is a member of the National Academy of Medicine, National Academy of Sciences and the National Academy of Engineering, one of only 10 living persons to be a member of all 3 national academies.
He received his PhD in physics from Princeton University and was a postdoctoral fellow in the Department of Neurobiology at Harvard Medical School. He was on the faculty in the Department of biophysics at the Johns Hopkins University before moving to La Jolla where he is now an Investigator with the Howard Hughes Medical Institute and holds the Francis Crick Chair at The Salk Institute for Biological Studies. He is also a Distinguished Professor of Biology at the University of California, San Diego, where he is co-director of the Institute for Neural Computation and co-director of the NSF Temporal Dynamics of Learning Center.
Dr. Sejnowski is the President of the Neural Information Processing Systems (NIPS) Foundation, which organizes an annual conference attended by over 4500 researchers in machine learning and neural computation and is the founding editor-in-chief of Neural Computation published by the MIT Press. He was elected to the American Academy of Arts and Sciences and is also a Fellow of the American Association for the Advancement of Science, a Life Fellow of the Institute of Electrical and Electronics Engineers and a Fellow of the Cognitive Science Society. He has received many honors, including the NSF Young Investigators Award, the Wright Prize for interdisciplinary research from the Harvey Mudd College, the Neural Network Pioneer Award from the Institute of Electrical and Electronics Engin eers, the Hebb Prize from the International Neural Network Society and the Rosenblatt Award from the Institute of Electrical and Electronics Engineers. He re ceived an honorary doctorate from the University of Zurich.
Dr. Sejnowski was instrumental in shaping the BRAIN Initiative that was announced by the White House in 2013 and served on the Advisory Committee to the Director of NIH for the BRAIN Initiative.
Li-Huei Tsai
Director, Picower Institute for Learning and Memory at MIT
Picower Professor of Neuroscience, MIT
Li-Huei Tsai combines molecular, genetic and circuit-based techniques for a “whole-systems” approach to understanding neurological disorders that affect cognition as we age. Landmark discoveries include pinpointing major genetic risk for Alzheimer’s disease to immune genes, identifying chromatin-modifiers and kinases that regulate brain flexibility and can be targeted to improve cognition in Alzheimer’s disease, and discovering that genomic integrity is critical for neuronal protection during both aging and neurodegenerative disease.
Her discoveries in Alzheimer’s disease research, including therapeutic reversal strategies for cognitive defects, have been highlighted in Nature, Cell, and Neuron and featured on National Public Radio’s RADIOLAB, and in The Atlantic, The Smithsonian, and BBC News. She is currently spearheading MIT’s new Aging Brain Initiative – a far-reaching, collaborative effort to integrate life sciences, physical sciences, information technology, engineering and disciplines beyond to meet the global challenges of dementia and Alzheimer’s disease.
Professor Tsai obtained her Ph.D. from the University of Texas Southwestern Medical Center and postdoctoral training at Cold Spring Harbor Laboratories and Massachusetts General Hospital. Tsai became Assistant Professor of Pathology at Harvard Medical School and was promoted to tenure Professor at Harvard in 2002. She was recruited to MIT in 2006. She was an Investigator of the Howard Hughes Medical Institute from 1997 to 2013.
Dr. Tsai is a recipient of the Glenn Award for Research in Biological Mechanisms of Aging, the Javits Neuroscience Investigator Award, and the Mika Salpeter Lifetime Achievement Award for her research on brain development, neurological disorders, and Alzheimer’s disease. She is a Fellow of the American Association for the Advancement of Science, a member of the National Academy of Medicine, and an Academician of the Academia Sinica in Taiwan.
George Vradenburg
Chairman and Co-Founder, UsAgainstAlzheimer’s
George Vradenburg is the Chairman and Co-Founder of UsAgainstAlzheimer’s (UsA2), a disruptive and catalytic force committed to stopping Alzheimer’s by 2020. The UsA2 platform seeks to escalate the fight against Alzheimer’s through a broad range of powerful voices from various walks of life.
UsA2 serves as the convener of the only industry coalition dedicated to stopping Alzheimer’s—the Global CEO Initiative (CEOi) on Alzheimer’s—as well as the co-convener of a 60+ member coalition of the Alzheimer’s-serving community, Leaders Engaged on Alzheimer’s Disease. As a result of his UsA2 work, Mr. Vradenburg was appointed to the World Dementia Council by the UK Secretary of State for Health in March, 2014.
In 2011, the United States Secretary of the Department of Health and Human Services named Mr. Vradenburg to serve on the National Alzheimer’s Advisory Council on Research, Care and Services for the first-of-its-kind National Alzheimer’s Strategic Plan. In 2013 Mr. Vradenburg was appointed by Congress to the Long Term Care Commission charged with devising a comprehensive long term support and services plan for the United States.
Among other efforts, Mr. Vradenburg has testified twice before the U.S. Congress regarding the Global Alzheimer’s pandemic; conceived and supported the Alzheimer’s Study Group; and, through the Vradenburg Foundation, has supported the Alzheimer’s Disease International World Alzheimer’s Reports and the National Institute of Health’s Global Alzheimer’s Research Summit. Before his retirement, Mr. Vradenburg served in senior executive positions at AOL/Time Warner, Fox and CBS.
Mr. Vradenburg is a member of the Council on Foreign Relations and the Economic Club of Washington. He received his B.A. from Oberlin College, magna cum laude, where he was elected to Phi Beta Kappa, and his J.D. from Harvard Law School, cum laude.
Thursday, February 1, 2018
8:00am—6:30pm
Alumni Hall, 2nd Floor
Mosher Alumni House
University of California Santa Barbara
Santa Barbara, CA 93106
(map)
Agenda
8:00am–8:30am | Continental Breakfast and Registration Check-In |
8:30am–9:15am | Welcome, Introductions, and Background Kenneth S. Kosik, UC Santa Barbara Andrew W. Lo, MIT A. History, motivating factors and anticipated outcomes for meeting (Kosik) B. Funding challenges and new business models (Lo) |
9:15am–10:30am | Science I: Scientific Basis for Expanding Shots on Goal Facilitator: Katja Brose, Chan Zuckerberg Initiative Initial Comments: Randall Bateman, Washington University at St. Louis; Scott Berry, Berry Consultants; Anirvan Ghosh, Biogen; John Hardy, University College London |
10:30am–10:45am | Break |
10:45am–12:00pm | Science II: Implementing an Expanded Shots-on-Goal Approach Facilitator: Carole Ho, Denali Therapeutics Initial Comments: Richard Hodes, National Institute on Aging; Zach Malchano, Cognito Therapeutics; George Vradenburg, UsAgainstAlzheimer’s |
12:00pm–1:30pm | Lunch Keynote Address: David Baltimore, Caltech Introduction by Terrence Sejnowski, Salk Institute |
1:30pm–1:45pm | Break |
1:45pm–3:00pm | Business I: Stakeholder Perspectives Facilitator: Miyoung Chun Initial Comments: Daniel Arbess, Xerion Investments; James Momtazee, KKR; Teresa Valko, Alzheimer’s Association California Central Chapter; Steve Wood, Amgen |
3:00pm–3:15pm | Break |
3:15pm–4:30pm | Business II: Potential Business and Financing Structures Facilitator: Christopher Austin, NIH/NCATS Initial Comments: Matthew Gline, Roivant Sciences; Jennifer Goldstein, Silicon Valley Bank; Neil Littman, California Institute for Regenerative Medicine (CIRM); Michele Park, Clarus Ventures |
4:30pm–5:00pm | Wrap-Up and Next Steps Ken Kosik and Andrew Lo |
5:30pm–6:30pm | Cocktail Reception and Conversation Location: Beachside Bar Cafe, 5905 Sandspit Road, Goleta, CA |
6:30pm | Dinner Location: Beachside Bar Cafe |
Daniel J. Arbess
CEO, Xerion Investments
Daniel J. Arbess is an investor and policy analyst recognized for his prescient calls on some of the major developments of the past 30 years, including the economic transition toward markets from communism in Eastern Europe & China; the U.S. housing/financial crisis and monetary policy-led market recovery; and the transformative impact of information technology on the political and economic frameworks of the post-WWII era.
Mr. Arbess was the first American lawyer to relocate to Eastern Europe, after having been in the Kremlin when Mikhail Gorbachev unveiled the policies of Glasnost and Perestroika in February 1987, and making the call that those policies would herald the demise of Communist economics. He moved to Prague in early 1990 to advise the new Czechoslovak government on its economic transition, privatization policy and transactions. He became (at 31 in ‘92) the youngest partner in the history of White & Case, and Head of its Global Privatization Group.
Mr. Arbess continued his involvement in the economic transition process as a principal investor in newly privatized European companies, first as CEO of Michael Dingman’s Stratton Investments, later as co-founder with Morton Meyerson of Taiga Capital Group. As his focus followed developments from Europe to Asia, starting with the impact of China’s industrialization and other reforms on US manufacturers, Dan co-founded and managed distressed corporate credit investments for Triton Partners, a $1.6 Billion New York-based structured credit asset management firm. He then launched the Xerion Hedge Funds in 2003 with the backing of S. Donald Sussman and his Paloma Partners, and merged Xerion with Perella Weinberg Partners in 2007. Xerion’s assets peaked around $3.25 Billion in 2011.
Xerion generated 12-year net returns of +240% (12.5% annualized) from 2003 through November 2014, including +112% (21% annualized) without a single down year through the four years of financial crisis and early recovery (’07-’10). Mr. Arbess returned Xerion investor capital in late 2014 and sold his stake in Perella Weinberg Partners in 2015 to spend more time on economic and geopolitical policy, and pursue “virtualization”-themed investment opportunities through his private firm, Xerion Investments.
Mr. Arbess was born in Montreal, Canada, and is a United States citizen. He received an LLB from Osgoode Hall Law School in Toronto, and an LLM from the Harvard Law School. He was an affiliate at the John F. Kennedy School of Government and is a lifetime member of the Council on Foreign Relation and a co-Founder of No Labels, which promotes bipartisan Congressional collaboration.
Christopher P. Austin
Director, National Center for Advancing Translational Sciences
Christopher Austin is Director of the National Center for Advancing Translational Sciences (NCATS) at the U.S. National Institutes of Health. NCATS’ mission is to catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions. Before becoming NCATS Director in September 2012, he was Director of the NCATS Division of Preclinical Innovation, which focuses on translating basic science discoveries into new treatments, particularly for rare and neglected diseases, and developing new technologies and paradigms to improve the efficiency of therapeutic and diagnostic development. In this role, he founded and directed numerous initiatives including the NIH Chemical Genomics Center (NCGC), the Therapeutics for Rare and Neglected Diseases (TRND) program, and the Toxicology in the 21st Century (Tox21) program. In 2016, Dr. Austin was elected chair of the International Rare Disease Research Consortium (IRDiRC). Before joining NIH in 2002, Dr. Austin directed research programs genomics-based target discovery, pharmacogenomics, and neuropsychiatric drug development at Merck, with a particular focus on schizophrenia. Austin earned an A.B. in biology from Princeton University and an M.D. from Harvard Medical School. He completed clinical training in internal medicine and neurology at Massachusetts General Hospital, and a research fellowship in genetics at Harvard.
David Baltimore
Robert Andrews Millikan Professor of Biology and President Emeritus, Caltech
After serving as President of the California Institute of Technology for nine years, David Baltimore was appointed President Emeritus and the Robert Andrews Millikan Professor of Biology in 2006. Awarded the Nobel Prize at the age of 37 for research in virology, Dr. Baltimore has profoundly influenced national science policy on such issues as recombinant DNA research and the AIDS epidemic. He is an accomplished researcher, educator, administrator and public advocate for science and engineering and is considered one of the world’s most influential biologists.
Born in New York City, Dr. Baltimore became interested in biology during high school when he spent a summer at the Jackson Memorial Laboratory and worked with research biologists on mammalian genetics. He received his B.A. in Chemistry from Swarthmore College in 1960 and a Ph.D. in 1964 from Rockefeller University, where he returned to serve as President from 1990-91 and faculty member until 1994.
For almost 30 years, Dr. Baltimore was a faculty member at Massachusetts Institute of Technology where his early investigations examined the molecular processes underlying the ability of poliovirus to infect cells. This led him to work on other RNA viruses and then to a consideration of how cancer-causing RNA viruses manage to infect and permanently alter a healthy cell. He identified the enzyme reverse transcriptase in the virus particles, thus providing strong evidence for a process of RNA to DNA conversion, the existence of which had been hypothesized some years earlier. Dr. Baltimore and Howard Temin (with Renato Dulbecco, for related research) shared the 1975 Nobel Prize in Physiology or Medicine for their discovery, which provided the key to understanding the life-cycle of retroviruses such as HIV. In the following years, he has contributed widely to the understanding of cancer, AIDS and the molecular basis of the immune response.
His present research focuses on control of inflammatory and immune responses, on the roles of microRNAs in the immune system and on the use of gene therapy methods to treat HIV and cancer in a program called “Engineering Immunity”. He has become Director of the Joint Center for Translational Medicine, an activity that joins Caltech and UCLA in a program to translate basic science discoveries into clinical realities and where an active clinical program is under way.
Dr. Baltimore has several outstanding administrative and public policy achievements to his credit. In the mid-1970s, he played an important role in creating a consensus on national science policy regarding recombinant DNA research. He served as founding director of the Whitehead Institute for Biomedical Research at MIT from 1982 until 1990. An early advocate of federal AIDS research, Dr. Baltimore co-chaired the 1986 National Academy of Sciences committee on a National Strategy for AIDS and was appointed in 1996 to head the National Institutes of Health AIDS Vaccine Research Committee. Dr. Baltimore served as a member of the Independent Citizen’s Oversight Committee to the California Institute for Regenerative Medicine until 2007 and on the Board of Directors for both MedImmune until 2007 and Cellerant until 2008.
He has played an important role in the development of American biotechnology since his involvement in the 1970’s in the formation of Collaborative Genetics. He helped found other companies such as Calimmune and Immune Design and he presently serves on the Board of Directors at several companies and non-profit institutions including the Broad Foundation and Broad Institute, and Amgen and Regulus Therapeutics. He is a member of numerous Scientific Advisory Boards, including the Broad Institute, Ragon Institute, Regulus Therapeutics and Immune Design. He is a Scientific Partner to the venture capital firm, The Column Group, and was a Director of the Swiss investment company BB Biotech through 2011.
Dr. Baltimore’s numerous honors include the 1970 Gustave Stern Award in Virology, 1971 Eli Lilly and Co. Award in Microbiology and Immunology, 1999 National Medal of Science, and 2000 Warren Alpert Foundation Prize. He was elected to the National Academy of Sciences in 1974, and is also a fellow of the American Academy of Arts and Sciences, and a foreign member of both the Royal Society of London and the French Academy of Sciences. He is past-President and Chair of the American Association of the Advancement of Science (2007-2009) and was mostly recently named a Fellow of the American Association for Cancer Research (AACR). He has published more than 700 peer-reviewed articles.
Randall Bateman
Charles F. and Joanne Knight Distinguished Professor of Neurology, Washington University at St. Louis
Director, Dominantly Inherited Alzheimer Network (DIAN)
Director, DIAN Trials Unit
Dr. Bateman is the Charles F. and Joanne Knight Distinguished Professor of Neurology, Director of the Dominantly Inherited Alzheimer Network (DIAN, http://dian.wustl.edu), and Director of the DIAN Trials Unit (DIAN-TU). Dr. Bateman’s research focuses on the pathophysiology and development of improved diagnostics and treatments of Alzheimer’s disease. Dr. Bateman’s lab accomplishments include pioneering the central nervous system Stable Isotope Labeling Kinetics (SILK) measurements in humans, furthering insights of human circadian patterns of amyloid-beta and soluble APP, and human in vivo control of the alpha-secretase, beta-secretase, and gamma-secretase processing of amyloid-beta. His lab has developed methods to quantify the pharmacodynamic action of drugs targeting amyloid-beta, amyloid precursor protein, and Apolipoprotein E. His lab recently reported on an accurate blood test for Alzheimer’s disease plaques. Dr. Bateman’s research in DIAN has provided evidence for a cascade of events beginning decades before symptom onset that leads to AD dementia, supporting development of Alzheimer’s disease prevention trials.
Dr. Bateman directs the DIAN-TU, which launched the first prevention trial in families with early onset Alzheimer’s disease in 2012. The DIAN-TU trial is an advanced world-wide adaptive trial platform that tests the most advanced therapeutics targeting early onset dominantly inherited Alzheimer’s disease. The DIAN-TU has now launched its third drug arm, an oral medication that blocks amyloid-beta formation with the goal to slow, stop or reverse Alzheimer’s disease.
Dr. Bateman worked with dominantly inherited AD families to start the first world-wide meeting for family members (http://dian.wustl.edu/for-families/family-conferences/) which have grown into annual meetings with more than 150 family members attending from most continents. The DIAN Expanded Registry is the portal for families with dominantly inherited Alzheimer’s disease to become connected to the DIAN studies and other families around the world.
Dr. Bateman has received a number of awards including the Beeson Award for Aging Research, Alzheimer’s Association (Zenith Award), Scientific American, Chancellor’s Award for Innovation and Entrepreneurship, the Glenn Award for Aging Research, and the MetLife Foundation Award for Medical Research. Dr. Bateman has been the primary mentor for junior faculty, clinical fellows, post-doctoral researchers, and graduate and medical students, who have been successful in their desired scientific careers.
Scott Berry
President and Senior Statistical Scientist, Berry Consultants
Scott Berry is President and a Senior Statistical Scientist at Berry Consultants, LLC. He earned his PhD in statistics from Carnegie Mellon University and was an Assistant Professor at Texas A&M University before co-founding Berry Consultants in 2000. He is adjunct faculty in the Department of Biostatistics at the University of Kansas Medical Center. Dr. Berry was elected as a Fellow of the American Statistical Association in 2013.
Since 2000, he has been involved in the design of hundreds of Bayesian adaptive clinical trials of pharmaceuticals and medical devices and has become an opinion leader in the field of Bayesian adaptive clinical trials. Some of these trials have been groundbreaking trial designs, setting new standards for innovation and flexibility in trial design. These include the trials supporting the first fully Bayesian approval by CDER of the United States FDA (Pravastatin-Aspirin combination) and the statistical design for Time Magazine’s #2 Medical Breakthrough of 2007 (Veridex’s GeneSearch BLN Assay), and an adaptive phase II/III seamless trial for Dulaglutide leading to FDA approval in September 2014. Recently he has been involved in the design of the IMI Funded platform trial for prevention of Alzheimer’s disease (EPAD), the DIAN-TU platform trial in dominantly inherited Alzheimer’s, and the EU funded platform trial for the treatment of severe community acquired pneumonia (AD-SCAP) in a preparedness setting (PREPARE).
Katja Brose
Science Program Officer, Chan Zuckerberg Initiative
Katja Brose, Ph.D., is a Science Program Officer at the Chan Zuckerberg Initiative. The goals of Chan Zuckerberg Science Initiative are to support basic science and technology that will make it possible to cure, prevent, or manage all diseases by the end of the century. Before joining CZI, she was part of the editorial team at Cell Press for 17 years, where from 2004-20017 she was Editor-in-Chief of Neuron and a Publishing Director at Cell Press-Elsevier. During her tenure as Editor, Neuron undertook a major expansion of its scope building on its historical strengths in molecular and cellular neuroscience to cover all areas of neuroscience from molecular/cellular mechanisms to systems and cognitive neuroscience, genetics, neurological and psychiatric disease, theoretical neuroscience and emerging technologies. As Publishing Director, she was responsible for Cell Press strategy for review content, including oversight of the Trends family of review journals. She also led Cell Press’ efforts around rigor and reproducibility. She has been an active committee member at the Society for Neuroscience, as a member of the Professional Development and Neuroscience Training Committees. She speaks frequently on topics related to scientific publishing and communication, including publication ethics and rigor and reproducibility in science. She earned her undergraduate degree in 1990 from Brown University, with a double concentration in Biology and European History. She received her PhD in Biochemistry from the University of California-San Francisco (1994-2000). For her graduate work, she worked in the laboratory of Dr. Marc Tessier Lavigne focusing on axon guidance mechanisms in the developing spinal cord.
Miyoung Chun
Former Executive Vice President of Science Programs, Kavli Foundation
Miyoung Chun’s career spans a wide range of experience in academia and industry. Her academic career began as an Assistant Professor of Biochemistry and a member of Whitaker Cardiovascular Institute at Boston University School of Medicine in 1995. There she taught in the areas of cell biology and molecular medicine, and conducted research in signal transduction of G-protein coupled receptors.
From 1999 to 2004 she worked for Millennium Pharmaceuticals Inc. as a scientist and project leader, where her research focused on genomics/functional genomics and on molecular imaging in drug discovery and development. She discovered and characterized novel genes that are important to inflammatory and cardiovascular diseases, and has over 30 U.S. and International issued/published patents.
In 2004 she moved back to academia as Assistant Dean of Science and Engineering at the University of California, Santa Barbara (UCSB), in particular serving the California Nanosystems Institute. She was also appointed Director of International Research Advancement at UCSB. In this role she was active in building partnerships among academia, government and industry around the globe.
Dr. Chun was Vice President of Science Programs at The Kavli Foundation since 2007 and was Executive Vice President of Science Programs from 2013 to 2017. She obtained her Ph.D. degree in Molecular Genetics from The Ohio State University in 1990 and was a Postdoctoral Fellow at MIT’s Whitehead Institute studying the cell and molecular biology of receptors.
Anirvan Ghosh
Senior Vice President, Head of Research and Early Development, Biogen
Anirvan Ghosh serves as Senior Vice President, Head of Research and Early Development at Biogen. In this capacity he leads a team of 370 scientists and clinicians responsible for research and early clinical development across disease areas including Alzheimer’s Disease, Parkinson’s Disease and Movement Disorders, Neuromuscular Disorders, Multiple Sclerosis, Acute Neurology, Ophthalmology, Pain, Neuropsychiatry, and Immunology.
Prior to joining Biogen, Dr. Ghosh served as the founding Chief Scientific Officer at E-Scape Bio, a biotech company developing therapeutics for neurodegenerative disorders, and as VP/Global Head of Neuroscience Discovery and Biomarkers at Roche, where he led the Neuroscience research and drug discovery programs in Neurodegenerative Diseases, Neurodevelopmental Disorders, and Psychiatric Disorders. He also headed the Biomarkers and Clinical Imaging group. During his time at Roche he delivered 11 clinical candidate molecules to early clinical development in a number of areas including Alzheimer’s Disease, Spinal Muscular Atrophy, Schizophrenia, and Autism.
Dr. Ghosh has also held academic appointments at The Johns Hopkins School of Medicine and UC San Diego, where his research has been focused on understanding how connections in the brain are established during development and how they may be affected in neurodevelopmental disorders. Major contributions from his lab have included the identification of activity-dependent transcription factors that regulate neuronal development and plasticity, and the discovery of transmembrane Leucine Rich Repeat (LRR) proteins as key regulators of synapse formation and function.
Dr. Ghosh received his BS in Physics from Caltech, his Ph.D. in Neuroscience from Stanford University and did his postdoctoral work at Harvard Medical School. He is recipient of several awards including the Pew Scholar Award, Presidential Early Career Award for Scientists and Engineers, the Society for Neuroscience Young Investigator Award, and the UCSD Revelle College Outstanding Faculty Award.
Matthew Gline
Chief Financial Officer, Roivant Sciences
Matthew Gline has served as Chief Financial Officer of Roivant Sciences, Inc. since September 2017. Mr. Gline joined Roivant in March 2016 and previously served as Senior Vice President, Finance and Business Operations. From April 2014 to March 2016, he was a Vice President at Goldman Sachs, Fixed Income Digital Structuring, where he focused on technology and data strategy. Prior to Goldman Sachs, Mr. Gline was a co-founder of Fourthree, Inc., a risk analytics technology and consulting company. From 2008 to 2012, he served as Vice President at Barclays, Enterprise Risk Management Advisory, where he provided analysis for corporate clients related to capital markets access for financing and risk management. Mr. Gline earned his A.B. in Physics from Harvard University.
Jennifer Friel Goldstein
Managing Director, Head of Western Region & Accelerator, Life Sciences and Healthcare, Silicon Valley Bank
Jennifer Friel Goldstein, BSE/MB/MBA, is a managing director of Silicon Valley Bank’s Life Science and Healthcare practice.
Based in San Francisco, Ms. Goldstein is responsible for Healthcare across all sectors on the West Coast, runs the National Accelerator for Healthcare, and serves as the Head of our Biotechnology and Diagnostics Practice. She is also focused on Corporate Venture initiatives across the bank within the Healthcare vertical. Jennifer and her teams focus on banking, capital, and relationship solutions for our Healthcare clients of all life stages.
Ms. Goldstein joined SVB after a successful tenure at Pfizer, where she served as a Director on Pfizer’s Venture Capital team. While a consultant at Bain & Company in London, she focused on private equity deals. She has also had diverse operational and research experience at companies such as Chiron, Genelabs and Genencor.
Ms. Goldstein brings over 15 years of investing, banking, business development, portfolio management, and consulting experience as well as a biotechnology background to the team. She graduated magna cum laude with a BSE in Bioengineering and a Master’s of Biotechnology from the University of Pennsylvania. Ms. Goldstein was also named a Joseph Wharton Fellow while completing her MBA at the Wharton School.
In her free time, Ms. Goldstein enjoys remaining involved with SpringBoard Enterprises, serving on the board of the Leukemia and Lymphoma Society in Silicon Valley, and spending time with her husband and their twin children who keep them on their toes!
John Hardy
Chair of Department of Molecular Neuroscience and Reta Lila Weston Laboratories, UCL Institute of Neurology UK
John Hardy received his degree in Biochemistry from Leeds in 1976 and his PhD from Imperial College in Neuropharmacology in 1979. He did postdocs at the MRC Neuropathogenesis Unit and the Swedish Brain Bank, in Umea, where he started to work on Alzheimer’s disease. In 1985 he took the job of Lecturer in Biochemistry and Molecular Genetics at St Mary’s Hospital, Imperial College, where he began working on the genetics of Alzheimer’s disease. In 1991 he led the group which found the first mutation in the amyloid gene which caused Alzheimer’s disease. This finding led him and others to formulate the amyloid hypothesis for the disease. In 1992 he moved to the United States, to the University of South Florida. In 1996 he moved to the Mayo Clinic where he became Chair of the Department of Neuroscience in 2000. In 1998 he was part of the consortium which identified mutations in the tau gene in Pick’s disease. In 2001 he moved to the NIH to become the Chief of the Laboratory of Neurogenetics, where he was part of the group which found triplications in the synuclein gene caused Parkinson’s disease. He returned to the Department of Molecular Neuroscience at the Institute of Neurology in 2007.
He has won the Allied Signal, Potamkin, MetLife and Kaul Prizes, for his work on Alzheimer’s disease and the Anna Marie Opprecht Prize for his work on Parkinson’s disease. More recently he was awarded the 2011 Khalid Iqbal Lifetime Achievement Award in Alzheimer’s Disease Research and the IFRAD 2011 European Grand Prize for Alzheimer’s Research. In 2014 he was awarded The Dan David Prize endowed by the Dan David Foundation, headquartered at Tel Aviv University; the Thudichum Medal from the Biochemical Society and is the recipient of the 3rd Lord Brain Memorial Medal. Then he was awarded the Robert A. Pritzker Prize by the Michael J Fox Foundation. He has been elected a member of the Academy of Medical Sciences and has been awarded an honorary MD by the University of Umea, Sweden. He was made an FRS by the Royal Society in 2009 and in 2010 was awarded an honorary Doctor of Science degree by the University of Newcastle. During 2015 he was awarded the Piepenbrock-DZNE Award, and the Breakthrough Prize in Life Sciences followed in 2016 by the Helis Prize.
He has three adult children and three grandchildren who live in the US.
Carole Ho
Chief Medical Officer and Head of Development, Denali Therapeutics
Carole Ho is the Chief Medical Officer and Head of Development at Denali Therapeutics in South San Francisco. Denali Therapeutics is dedicated to therapeutic discovery and development of treatments for neurodegenerative diseases. Prior to Denali, Dr. Ho most recently served as Vice President of Genentech Early Clinical Development. In that role, she led a team of Medical Directors responsible for delivery of pivotal trial-ready therapeutic candidates in Neurology, Ophthalmology, Immunology, and Infectious Disease and served as the Chair of the Development Review Committee. During her 8 year tenure at Genentech, Dr. Ho and her team led the successful development of multiple therapeutics that are currently in Ph3 studies or have been approved, including ocrelizumab for Multiple Sclerosis, lampalizumab for dry AMD, rituxan for Wegener’s granulomatosis and microscopic polyangiitis and crenezumab for Alzheimer’s Disease. Dr. Ho and her team also lead the Alzheimer’s Prevention Initiative with Banner Health to spearhead a landmark Alzheimer prevention study in cognitively healthy individuals who are destined to develop Alzheimer’s disease because of their genetic history.
She received her undergraduate degree in Biochemical Sciences from Harvard College and her M.D. from Cornell University. She completed her Medical Internship and Neurology residency at Harvard and served as Chief Resident of Neurology at the Massachusetts General Hospital / Brigham and Women’s Hospital. Prior to Genentech, Dr. Ho was on Faculty in the Department of Neurology at Stanford University and subsequently a Medical Director at Johnson and Johnson.
Richard J. Hodes
Director, National Institute on Aging, NIH
Richard J. Hodes, M.D., is the Director of the National Institute on Aging (NIA) at the National Institutes of Health (NIH). The NIA leads the Federal effort supporting and conducting research on the biological, behavioral, and clinical aspects of aging.
Dr. Hodes has devoted his tenure to the development of a strong, diverse, and balanced research program. This has led to new and innovative ways to conduct research, share data and translate findings into practical interventions and public information. In biology, studies examine genetic and other factors influencing aging, how they affect longevity and how they intersect with the development of age related diseases. Research in geriatrics is uncovering new ways to combat frailty and other age-related conditions. Behavioral and social research is deepening understanding of the individual behaviors and societal decisions that affect well-being.
Dr. Hodes also directs the Federal effort to find effective ways to treat or prevent Alzheimer’s disease, as the NIA is the lead NIH institute for this mission. Cutting edge research conducted and supported by the NIA has revolutionized the way we think about Alzheimer’s disease. Studies in genetics, basic mechanisms, imaging and biomarkers have spurred the development of potential therapies aimed at a variety of targets and testing interventions at the earliest signs of disease.
An influential scientist in the field of immunology, Dr. Hodes is a graduate of Yale University and received his M.D. from Harvard Medical School. He is a Diplomate of the American Board of Internal Medicine, a member of The Dana Alliance for Brain Initiatives, a Fellow of the American Association for the Advancement of Science, and a member of the Institute of Medicine of the National Academy of Sciences.
Neil Littman
Director of Business Development and Therapeutics Team Operations, California Institute for Regenerative Medicine (CIRM)
Neil Littman is the Director of Business Development and Therapeutics Team Operations at CIRM. Neil is responsible for the development of platforms and best practices that increase industry and investor engagement and that further promote the visibility of CIRM’s funding opportunities and of its development and therapeutics portfolio.
Mr. Littman is responsible for establishing and maintaining relationships with pharma and biotech companies, and investors. In the past, Mr. Littman was responsible for managing CIRM’s strategic infrastructure programs, including the Alpha Clinics, Accelerating Center, Translating Center, iPSC Bank, Stem Cell Genomics Center of Excellence, and CIRM’s new $150 million Accelerating Therapies Public-Private Partnership.
Prior to joining CIRM in 2012, Mr. Littman was a Senior Associate at Burrill & Company, a life sciences venture capital firm and investment bank with over $1 billion of assets under management. Prior to joining Burrill & Company in 2009, he worked in the Healthcare Investment Banking groups at Thomas Weisel Partners and Deutsche Bank where he focused on strategic advisory and public and private financings, including: M&A, in-licensing, out-licensing, IPOs, follow-ons, registered directs, and private placements.
Mr. Littman received a Master of Science in Biotechnology with a concentration in Biotechnology Enterprise from The Johns Hopkins University, and a Bachelor of Arts in Molecular, Cellular and Development Biology from the University of Colorado, Boulder.
Zach Malchano
President, Cognito Therapeutics
Zach Malchano is the President of Cognito Therapeutics. He previously served as the General Manager and Vice President of Product Development for Cognito Therapeutics. Prior to this, Mr. Malchano was a founder and early-employee at a number of different medical device startups where he was responsible for company development, program leadership, and product development, which led to first in human clinical studies and regulatory clearance. Mr. Malchano was an Entrepreneur in Residence at Rock Health, where he analyzed opportunities in digital healthcare, performed diligence, and advised portfolio companies.
He received his undergraduate degree in Electrical Engineering from MIT and his Masters degree in Biomedical Engineering from the Harvard-MIT Division of Health Sciences and Technology. Mr. Malchano completed the Biodesign Innovation Fellowship at Stanford University. He also serves as an affiliate faculty member at the Center for Primary Care at Harvard Medical School, where he is on a working group establishing a medical device and healthcare innovation fellowship program and its accompanying courses.
James C. Momtazee
Head of Americas Health Care Industry Team, KKR
Jim Momtazee joined KKR in 1996 and is Head of the Americas Health Care industry team. Jim is a member of the Investment Committee for Americas Private Equity and is the Chairman of the Investment Committee for the KKR Health Care Strategic Growth Fund. He currently serves as Chairman of Air Medical Group Holdings and is on the board of directors of Ajax Health, BridgeBio, PRA Health Sciences, Entellus and Pharmerica. He was also previously involved in the investments in HCA and Jazz Pharmaceuticals, among others. Prior to joining KKR, Mr. Momtazee was with Donaldson, Lufkin & Jenrette where he worked on a broad range of corporate finance, mergers and acquisitions, and merchant banking transactions. He holds an A.B. and M.B.A. from Stanford University.
Michele Park
Partner, Clarus Ventures
Michele Park, PhD, serves as a Partner for the firm and leads the Clarus Cancer Fund, a novel investment model aimed at generating financial returns and amplifying scientific and medical impacts by donating a portion of the Cancer Fund’s returns to advance basic science research in cancer.
Dr. Park joined Clarus in 2006 with six years of sell-side equity research experience covering the biotechnology sector. From 2002-2005, Dr. Park was a research analyst at Credit Suisse First Boston, where she covered biotechnology stocks as a member of the firm’s US biotechnology team. Before joining CSFB, Dr. Park was a biotechnology research analyst at US Bancorp Piper Jaffray.
Dr. Park received a PhD in Molecular Biology from Cornell University’s Graduate School of Biomedical Sciences, completing her PhD dissertation at Memorial Sloan-Kettering Cancer Center, and a BA in Molecular Biology from Princeton University.
Dr. Park is a member of Curesearch’s Catapult Advisory Council and represents Clarus on the Board of Directors of Lumos and as a Board observer of SFJ. Previous Board seats have included Sientra (NASDAQ:SIEN) and Board observer roles at Achillion and Comentis. Dr. Park is also a member of the Private Equity Women Investor Network (PEWIN), a member of the Council of Korean Americans (CKA), and a Board Trustee for the American Friends of the Royal Philharmonic Orchestra (AFRPO).
Teresa Valko
Board Chair, Alzheimer’s Association California Central Chapter
Teresa is Board Chair for the California Central Chapter of the Alzheimer’s Association. She has been a member of the chapter Board of Directors for three years and Chair for one year. She also serves as Ambassador to Rep. Julia Brownley as part of the Alzheimer’s Association’s advocacy program. Teresa sits on the Alzheimer’s Women’s Initiative committee for Ventura County, the Alzheimer’s Association’s Medical Advisory Board for Ventura County, and the Dementia Friendly Ventura County committee. She is an avid volunteer for the Alzheimer’s Association, teaching and speaking about Alzheimer’s across the country. She holds a Masters of Science from the University of Alabama at Birmingham and was an embryologist and Director of Laboratory Services in Ann Arbor, MI prior to her affiliation with the Alzheimer’s Association.
George Vradenburg
Chairman and Co-Founder, UsAgainstAlzheimer’s
George Vradenburg is the Chairman and Co-Founder of UsAgainstAlzheimer’s (UsA2), a disruptive and catalytic force committed to stopping Alzheimer’s by 2020. The UsA2 platform seeks to escalate the fight against Alzheimer’s through a broad range of powerful voices from various walks of life.
UsA2 serves as the convener of the only industry coalition dedicated to stopping Alzheimer’s—the Global CEO Initiative (CEOi) on Alzheimer’s—as well as the co-convener of a 60+ member coalition of the Alzheimer’s-serving community, Leaders Engaged on Alzheimer’s Disease. As a result of his UsA2 work, George was appointed to the World Dementia Council by the UK Secretary of State for Health in March, 2014.
In 2011, the United States Secretary of the Department of Health and Human Services named Mr. Vradenburg to serve on the National Alzheimer’s Advisory Council on Research, Care and Services for the first-of-its-kind National Alzheimer’s Strategic Plan. In 2013 Mr. Vradenburg was appointed by Congress to the Long Term Care Commission charged with devising a comprehensive long term support and services plan for the United States.
Among other efforts, Mr. Vradenburg has testified twice before the U.S. Congress regarding the Global Alzheimer’s pandemic; conceived and supported the Alzheimer’s Study Group; and, through the Vradenburg Foundation, has supported the Alzheimer’s Disease International World Alzheimer’s Reports and the National Institute of Health’s Global Alzheimer’s Research Summit. Before his retirement, Mr. Vradenburg served in senior executive positions at AOL/Time Warner, Fox and CBS.
Mr. Vradenburg is a member of the Council on Foreign Relations and the Economic Club of Washington. He received his B.A. from Oberlin College, magna cum laude, where he was elected to Phi Beta Kappa, and his J.D. from Harvard Law School, cum laude.
Stephen Wood
Scientific Director, Neuroscience Discovery Research, Amgen
Steve is a Scientific Director in the Neuroscience Department at Amgen where he leads a team of scientists focused on discovering and developing therapeutics for neurodegenerative diseases. Over the last 25 years, his primary scientific focus has been Alzheimer’s Disease (AD). Prior to joining Amgen, he worked at SmithKline Beecham where he and colleagues helped elucidate the mechanisms of abnormal protein aggregation, a common pathology across numerous neurodegenerative disorders. He has led drug development programs at all stages, from basic discovery to early clinical development and is currently the head of Amgen’s Alzheimer’s Discovery Research group.
The goal of this workshop is to explore new approaches for addressing the issues in developing novel therapeutics and technologies for the treatment of Alzheimer’s disease. To that end, we want to avoid re-hashing the current state of the field and would like everyone to assume that all attendees are already familiar with the background, science, and issues. We’ve put together the following selection of readings to help ensure that everyone is on the same page and strongly encourage you to familiarize yourself with the material, as needed, in advance.
- A Path Toward Understanding Neurodegeneration. Kenneth S. Kosik, Terrence J. Sejnowski, Marcus E. Raichle, Aaron Ciechanover, and David Baltimore, 2016, Science 353(6302), pp. 872–873.
- Alzheimer’s Disease. Colin L. Masters, Randall Bateman, Kaj Blennow, Christopher C. Rowe, Reisa A. Sperling, and Jeffrey L. Cummings, 2015, Nature Reviews Disease Primers 1, Article No. 150156.
- Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders: Workshop Summary. Forum on Neuroscience and Nervous System Disorders; Forum on Drug Discovery, Development, and Translation; Board on Health Sciences Policy; Institute of Medicine. Washington (DC): National Academies Press (US); 2015 Oct 22. doi: 10.17226/21732
- Alzheimer’s Disease Drug-Development Pipeline: Few Candidates, Frequent Failures. Jeffrey L. Cummings, Travis Morstorf, and Kate Zhong, 2014, Alzheimer’s Research & Therapy 6(4): 37.
- Parallel Discovery of Alzheimer’s Therapeutics. Andrew W. Lo, Carole Ho, Jayna Cummings, and Kenneth S. Kosik, 2014, Science Translational Medicine 6 (241), pp. 241cm5.
- Multicompany Trials Adapt to Disciplines Beyond Cancer. Asher Mullard, 2014, Nature Medicine 20 (3), doi:10.1038/nm0114-3.
Selected articles from Neuron Special Issue: Bridging the Gap between Basic Research Discoveries and Clinical Applications, 2014, Volume 84, Issue 3.
- NINDS Translational Programs: Priming the Pump of Neurotherapeutics Discovery and Development by Rajesh Ranganathan, pp. 515–520.
- Collaboration in Translational Neuroscience: A Call to Arms by Adrian J. Ivinson, pp. 521–525.
- Transforming Our Approach to Translational Neuroscience: The Role and Impact of Charitable Nonprofits in Research by Cynthia Joyce, pp. 526–532.
- Public-Private Partnership: A New Engine for Translational Research in Neurosciences by Declan G. M. Murphy, Michel Goldman, Eva Loth, and Will Spooren, pp. 533–536.
- Improving and Accelerating Drug Development for Nervous System Disorders by Diana E. Pankevish, Bruce M. Altevogt, John Dunlop, Fred H. Gage, and Steve E. Hyman, pp. 546–553.
- Medicines for the Mind: Policy-Based “Pull” Incentives for Creating Breakthrough CNS Drugs by Dennis W. Choi, Robert Armitage, Linda S. Brady, Timothy Coetzee, William Fisher, Steve Hyman, Atul Pande, Steven Paul, William Potter, Benjamin Roin, Todd Sherer, pp. 554–563.
- Rigor or Mortis: Best Practices for Preclinical Research in Neuroscience by Oswald Steward and Rita Balice-Gordon, pp. 572–581.
- How Clinical Development Can, and Should, Inform Translational Science by Melissa Barker-Haliski, Daniel Friedman, H. Steve White, and Jacqueline A. French, pp. 582–583.
- Unbiased Approaches to Biomarker Discovery in Neurodegenerative Diseases by Alice S. Chen-Plotkin, pp. 594–607.
- The Evolution of Preclinical Alzheimer’s Disease: Implications for Prevention Trials by Reisa Sperling, Elizabeth Mormino, and Keith Johnson, pp. 608–622.
Material presented at the workshop is available to participants only. Please CLICK HERE to access the password-protected area.