Fall 2016 HRA Members’ Meeting (Bethesda)

Sept. 27-28, 2016

Additional Event Info:

Meeting Info

This meeting covered the following topics:

  • Understanding preprints, an emerging model for publication in biology (Tue)
  • Interactions with Industry (Tue)
  • Mainstream Media Distorting Science – and What as Funders Can We Do About It? (Tue)
  • FDA Priorities and Interactions with Non-Profits (Wed)
  • Communicating Impact of our Funding (Wed)
  • Interactions with National Center for Advancing Translational Sciences (NCATS) (Wed)
  • Scientific Session (Wed)

Meeting Agenda, Speaker Bios and Presentations (click on each day to expand)

September 27, Tuesday

10:00 - 10:15 AM

(15 min)

Welcome to the Members’ Meeting and Introductions

10:15am – 11:15am

(60 min)

FDA Priorities and Interactions with Non-Profits

The Acting Chief Scientist will update HRA members on FDA’s strategic priorities, and specifically opportunities for FDA and non-profit medical research funders to work together towards our common goal of advancing health through science.

Moderator

Amy Laster, PhD

Dr. Amy Laster, Director, Grants and Award Programs, oversees FFB’s $20 million Research Portfolio consisting of research awards in four funding programs supporting career development, laboratory-based science research, translational research and multi-investigator program projects. In this role, Dr. Laster has fostered partnerships with other biomedical research funders to expand the Foundation’s career development program as well as its translational research program. Following the expansion of these programs, Dr. Laster has assisted the Foundation’s Development staff to secure major donors to ensure their success and longevity. Additionally, Dr. Laster strategically designs the science programming for the Foundation’s VISIONS and Day of Science annual national conferences.Prior to joining FFB, Dr. Laster was a faculty member of the Department of Biology at Stevenson University. Dr. Laster completed a Postdoctoral fellowship at Johns Hopkins School of Medicine in the Solomon Synder Department of Neuroscience. Her area of research focus was the study of neurodegenerative diseases, specifically understanding the neurobiology of the autism spectrum disorder, Rett Syndrome. Dr. Laster holds a B.S. Natural Science from Spelman College, Atlanta, GA and a Ph.D. Biological Sciences from Purdue University, West Lafayette, IN.

Presenter

Luciana Borio, MD

Dr. Luciana Borio is FDA’s acting chief scientist. In this capacity, she is responsible for leading and coordinating FDA’s cross-cutting scientific and public health efforts.The Office of the Chief Scientist works closely with FDA’s product centers, providing strategic leadership and support for FDA’s regulatory science and innovation initiatives, including the Advancing Regulatory Science Initiative, the Critical Path Initiative, scientific professional development, scientific integrity, and the Medical Countermeasures Initiative (MCMi).Since 2011, Dr. Borio has served as the assistant commissioner for counterterrorism policy and director of the Office of Counterterrorism and Emerging Threats (OCET) in the Office of the Chief Scientist at FDA. In this capacity, Dr. Borio provided leadership, coordination, and oversight for FDA’s national and global health security, counterterrorism, and emerging threat portfolios and led the MCMi.Dr. Borio has been instrumental in coordinating FDA’s response to the 2009 H1N1 influenza pandemic and continues to oversee FDA’s preparedness and response activities for emerging threats, such as the avian influenza A (H7N9) virus and the West Africa Ebola epidemic.Before joining FDA as a medical reviewer in 2008, Dr. Borio served as a senior associate at the University of Pittsburgh Medical Center – Center for Biosecurity, assistant professor of medicine at the University of Pittsburgh, and advisor on biodefense programs for the U.S. Department of Health and Human Services. Dr. Borio received her M.D. from the George Washington University, and continues to practice medicine at Johns Hopkins Hospital.

11:15 AM -12:45PM

(90 min)

Understanding preprints, an emerging model for publication in biology

How are funding organizations using their own grant data and other data sources to inform their program planning and evaluation projects? More than ever, we have access to huge amounts of data. In addition to the award data in our grants management systems, we have award data from other funders (HRA RePorter, UberResearch Dimensions); publication data from PubMed and private data sources; patents from USPTO (Patents); and Altmetrics. How do we find the answers to our questions in all of this data? How do we show value for the cost and effort required to assemble and analyze it? How do we use this data to inform program decisions, patient advocates, donors, or our Board? The presenters offer a wide range of experience across funding organization and will provide examples from successful projects. The objective of these case studies is to provide some ideas you can use today and to encourage discussion about the opportunities, and challenges facing funders in analyzing big data

Moderator

Michael Stebbins, PhD

Vice President of Science and Technology | Laura and John Arnold Foundation

Michael previously served as a science advisor to the Obama Campaign and on the Obama Presidential Transition Team. He is the former director of biology policy for the Federation of American Scientists and the former president of Scientists and Engineers for America Action Fund. He also co-founded and served on the board of directors for Scientists and Engineers for America, and is a former adjunct professor of bioethics at the University of Pennsylvania. In addition, Michael worked as a legislative fellow for U.S. Senator Harry Reid and a public policy fellow for the National Human Genome Research Institute. Before coming to Washington, he was a senior editor at Nature Genetics. He received his B.S. in biology at SUNY Stony Brook and his Ph.D. in genetics while working at Cold Spring Harbor Laboratory.

Presenters

John Inglis, PhD

Publisher and Executive Director | Cold Spring Harbor Laboratory Press Co-founder | bioRXiv

Dr John Inglis is the co-founder of bioRxiv, Cold Spring Harbor Laboratory’s preprint service for the life sciences. He is also the founding Executive Director and Publisher of Cold Spring Harbor Laboratory Press in New York, a not-for-profit publisher of journals, books, and online media in molecular and cellular biology. He graduated from the University of Edinburgh Medical School with a Ph.D. in immunology and was previously Assistant Editor of The Lancet, founding editor of Trends in Immunology, and managing editor of other Trends journals.

Presentation

Jessica Polka, PhD

Postdoctoral Research Fellow | Harvard Medical School President of the Board of Directors, Future of Research Director | ASAPbio

Jessica Polka, PhD is the Director of ASAPbio, a researcher-driven initiative to promote discussions about the effective use of preprints in biology. She is also a visiting postdoc in the Department of Systems Biology at Harvard Medical School and President of the Board of Directors of Future of Research, a non-profit organization that represents voices of junior scientists in advocating for systemic changes to the research enterprise.

Presentation

Neil Thakur, PhD

Special Assistant to the Deputy Director | Office of Extramural Research | NIH

Neil Thakur has served as Special Assistant to the National Institutes of Health (NIH) Deputy Director for Extramural Research since 2005. He also serves as program manager for the NIH public access policy, and has spent a year on detail to the US Senate Special Committee on Aging. Prior to his time at NIH, he was Assistant Director of Health Services Research and Development at the Department of Veterans Affairs. Dr. Thakur holds a Ph.D. in Health Policy from Yale University School of Public Health and completed a NIMH postdoctoral fellowship in mental health services research at the Cecil G. Sheps Center for Health Services Research at the University of North Carolina at Chapel Hill.

Presentation

12:45 - 2:00 PM

(75 min)

Small Group Breakouts over Lunch

2:00 - 2:30 PM

(30 min)

Introduction To Our Meeting Sponsors

Presenters

Stephen Rose, PhD

Chief Research Officer | Foundation Fighting Blindness

Dr. Rose joined the Foundation Fighting Blindness as Chief Research Officer in December 2004. He manages and oversees the day-to-day operations of the Science Department, works closely with the Foundation’s Scientific Advisory Board and Research Oversight Committee, and provides overall leadership to its funding program. In addition, Dr. Rose and the Chief Drug Development Officer of the Foundation Fighting Blindness Clinical Research Institute (FFB CRI), the clinical arm of the Foundation, work in concert to establish a seamless pipeline of science and clinical studies to move preventions and treatments into clinical trials while partnering with pharma and biotech to maximize potential commercialization.Prior to joining the Foundation, Dr. Rose served Director, Division of Clinical Recombinant DNA Research, Office of Biotechnology Activities (OBA), NIH Office of Science Policy. In this position, he served as the Executive Secretary for the Recombinant DNA Advisory Committee and provided oversight and coordination for the recombinant DNA program to address issues regarding recombinant DNA, including human gene transfer clinical protocols.Dr. Rose received his B.S. in Biology with Honors from American University and his Ph.D. from the University of Virginia. Dr. Rose currently sits on the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee and is a Health Research Alliance Board member.

Diane Bovenkamp, PhD

Vice President of Scientific Affairs | BrightFocus Foundation

Diane Bovenkamp, PhD, Vice President of Scientific Affairs, oversees all of BrightFocus Foundation’s research programs, serves as the scientific liaison for the organization in local, national, and international forums, and identifies and develops new research initiatives, partnerships, and funding policies consistent with the mission of BrightFocus. Dr. Bovenkamp obtained her Ph.D. in Biochemistry from Queen’s University in Kingston, Ontario, Canada, completed a Postdoctoral Fellowship in the Vascular Biology Program at Boston Children’s Hospital/Harvard Medical School, and conducted further research at the Johns Hopkins University Bayview Proteomics Center in the Division of Cardiology at Johns Hopkins School of Medicine in Baltimore, Maryland.

Presentation

2:30 - 3:45 PM

(75 min)

Interactions with Industry

This session focuses on the various ways in which non-profit, non-government funders of research collaborate with industry. Christopher Penland will discuss CFF’s venture philanthropy model, including what CFF brings to a successful relationship with companies. Chris will also talk about Kalydeco and Orkambi as business case studies. Are these unique situations or are there broad lessons for other non-profit funders? Three HRA member organizations will then share their strategies and ideas on how best to form and then capitalize on interactions with industry in pursuit of their mission.

Moderator

Shannon Gallagher-Colombo, PhD

Program Administrator | AACR Foundation

Shannon Gallagher-Colombo received her PhD in Molecular Cell Biology from Thomas Jefferson University in 2009, followed by a postdoctoral fellowship in the Department of Radiation Oncology at the University of Pennsylvania, where she was involved in basic, preclinical, and clinical research investigating the use of photodynamic therapy as a treatment option for solid malignancies. Her postdoctoral work specifically focused on determining the influence of cell signaling on photodynamic therapy efficacy, and the use of molecular targeting agents in combination with photodynamic therapy to improve therapeutic outcomes. In her current role as a program administrator at the AACR, Gallagher-Colombo applies her scientific background to grants administration. She provides scientific support to the AACR’s non-SU2C grants portfolio, including scientific review of grantee progress, interacting with grantees and funders to address areas of need, and providing additional scientific expertise as needed. She is serves as a member of the Program Committee and as a co-chair of the Open Science Task Force for the HRA.

Presenters

Christopher Penland, PhD

Vice President of BioPharma Programs | Cystic Fibrosis Foundation

Chris Penland, Ph.D. earned a B.Sc. at Pfeiffer College (University) in Sports Medicine and Management, a M.Sc. from Appalachian State University in Exercise Science and completed his doctoral studies at East Carolina University in the Department of Physiology where he examined ion transport of bronchiolar tissues. A postdoctoral fellowship at Stanford University followed in the laboratory of Jeffrey Wine, Ph.D. where Dr. Penland was part of a larger team seeking to identify a naturally occurring primate model of cystic fibrosis. Although no such animal was identified Dr. Penland was given a chance to further his efforts in CF, this time behind a desk rather than standing at a bench, when he joined the US Cystic Fibrosis Foundation as Director of Research in 1999. For the next 15 years Dr. Penland oversaw the basic science investments the Foundation made and served on the advisory committees of numerous drug discovery and development projects. In 2015 Dr. Penland transitioned to a new role in the Foundation where he now facilitates relationships between it and biopharmaceutical companies engaged in CF-related drug discovery and development.

Presentation

Marie Candelore, MA

Associate Director, Research Business Development | JDRF

Maria Luisa Candelore, M.A., is Associate Director for Research Business Development at JDRF, where she supports efforts to identify, analyze and implement strategic alliances and partnership collaborations with biotechnology, device and large pharmaceutical companies to further the JDRF mission. Ms. Candelore supports JDRF’s research programs in Beta Cell Replacement (devices & stem cell sources) and Prevention (vaccines, microbiome).Prior to joining JDRF in 2012, Ms. Candelore worked at Merck where she was laboratory head in the Diabetes Department before moving the licensing group (External Scientific Affairs) in which Ms. Candelore supported biology (oncology, immunology, diabetes, and infectious diseases) and technology (biomarkers, proteomics, genomics, imaging, informatics, in vivo models, drug delivery and formulation) deals from early-stage clinical development through to commercialization (US and emerging markets.) Ms. Candelore obtained a B.S. in biology from Vassar College and a M.A. in biology from New York University.

Presentation

Maneesh Kumar, MD, PhD

Scientific Program Manager | Breast Cancer Research Foundation

Dr. Maneesh Kumar joined BCRF in March 2016. He works closely with Drs. Hurlbert and Flowers to manage and develop the translational research and special programs including a $15-million Drug Research Collaborative with investigators and industry sponsors, a $31-million Founder’s Fund to study metastatic breast cancer, and additional partnerships with professional cancer societies. Maneesh received his PhD in Free Radical and Radiation Biology and an MD from the University of Kansas. After a 2-year postdoctoral fellowship in neuroscience, he left the lab-bench for a career in non-profit. Prior to BCRF, Maneesh worked closely with graduate student and postdoctoral associations to improve opportunities and funding for young scientists.

Presentation

Mary DeRome

Translational Research Manager | Multiple Myeloma Research Foundation

Mary DeRome joined MMRF as Translational Research Manager in February 2014. In this role she is responsible for managing the $1.3 million yearly MMRF research grant portfolio. She also helps manage the MMRF CoMMpass trial, an 8 year study of 1000 Multiple Myeloma patients, and a number of other MMRF translational research and precision medicine initiatives. Prior to her current position, Mary spent twelve years as a Senior Investigator in oncology drug discovery at Bayer HealthCare in West Haven CT and seven years as Senior Scientist in nanotechnology vaccine development at Artificial Cell Technologies in New Haven CT. Mary received her BS in chemistry, did graduate work in Pharmacology, and received her MS in Cell and Molecular Biology from the University of Connecticut, Storrs CT/University of Connecticut Health Center, Farmington CT.

Presentation

4:15 - 5:30 PM

(75 min)

Mainstream Media Distorting Science – and What as Funders Can We Do About It?

The misinterpretation of scientific data by mainstream media poses a problem for scientists, advocacy organizations, funders, and sometimes public health researchers. Funding organizations are often embroiled in the subsequent controversies as experts in the field, funders of research, or advocates for appropriate health care. This session will focus on how organizations, broadly, manage their reputational risk prepare and respond to controversial inquiries. A communications expert will share how she and her colleagues work within and across organizations to prepare organizations and grantees for topics of interest to the media. Members will also hear from a journalist about how the media community engages with funders, the lens through which they look at the broader landscape of perspectives on these types of controversies, and how funder input is viewed. At the end of the session. HRA member organizations will share how their organizations responded to, or prepared for, controversies in the face of mainstream media reporting.

Moderator

Heather Snyder, PhD

Senior Director of Medical and Scientific Operations | Alzheimer’s Association

Heather M. Snyder, Ph.D., is Senior Director of Medical and Scientific Operations at the Alzheimer’s Association. She manages the Association’s International Research Grant Program, through which the Association funds research around the world. Snyder oversees the Association’s relationship with the leading disease journal in clinical neurology, Alzheimer’s & Dementia: The Journal of the Alzheimer’s Association, and its two companion open access journals. She is responsible for implementing the Alzheimer’s Association Women’s Alzheimer’s Research Initiative and leads the Association’s efforts to understand the role of vascular factors in Alzheimer’s and dementia. Dr. Snyder received her doctorate in Molecular Biology from Loyola University Chicago Stritch School of Medicine, her bachelor’s degree in Biology and Religious Studies from The University of Virginia, and completed her postdoctoral fellowship at Children’s Memorial Research Center, affiliated with Northwestern University, in Chicago. In addition, she serves on the board of directors for the Health Research Alliance and on the programmatic review committee US Army Medical Research and Materiel Command Peer Reviewed Alzheimer’s Research Program.

Presenters

Paula Waters

Executive Vice President | Edelman Public Relations

Paula Waters has 25 years of international business and communications experience in corporate, non-profit and consulting environments in both the United States and Europe. She has worked on many of the key issues that have transformed healthcare. As an executive vice president in the Chicago office, Paula leads the team’s corporate health assignments and provides strategic counsel to other health business. Her experience ranges from executive positioning to public health campaigns, labor relations, product launches and crisis communications. She has worked with clients in many industries, including non-governmental agencies, education, travel, banking and every sector of healthcare, including hospital systems, managed care firms, pharmaceutical and nutrition companies, device and diagnostic makers, and professional and patient advocacy associations. Paula worked on the merger of Pharmacia and Upjohn and later on that company’s subsequent joining with Monsanto. Additionally she supported the Astra and Zeneca and Fujisawa and Yamanouchi mergers. She also helped launch the Global Alliance for Vaccines and Immunization, funded by the Bill and Melinda Gates Foundation, at the 2000 World Economic Forum in Davos, Switzerland.Paula’s clients have included Abbott, The Blue Cross and Blue Shield Association, Bayer, Hoechst AG, Merck, Johnson & Johnson, Astellas, Pfizer, AstraZeneca, Danaher, Dignity Health, Serono, Glaxo SmithKline, University of Illinois Medical Center, Advocate Healthcare, the Joint Commission on Accreditation of Healthcare Organizations, the Rehabilitation Institute of Chicago, Humana, the Blue Cross and Blue Shield Association, United Health Care, Cigna, Pfizer, Takeda, Astellas, The Alzheimers Association, HFMA, and the European Vaccine Manufacturers Association.Paula joined Edelman after five years at Fleishman-Hillard where she headed the healthcare practice. Prior to that position she was employed for 15 years in WPP Group companies, where she lead health care units for Burson Marstellar, Hill & Knowlton and Ogilvy.Paula’s communications career began in the corporate sector as director of public relations at G. D. Searle. The company was engaged in an extensive program of refocusing, which included major restructuring and multiple divestitures. She headed their investor relations, corporate identity, and media relations functions and managed their corporate philanthropy program. Before beginning her career in communications, she spent 10 years in pharmacological research in university, government, and corporate laboratories.

Presentation

Jessica Firger

Staff Writer | Newsweek

Jessica Firger is a staff writer at Newsweek, covering health, medicine and science. She’s held health reporter posts at CBS News and Everyday Health. More broadly, her writing has been featured in numerous national media outlets including the Wall Street Journal, Al Jazeera America, New York Times, Marie Claire, Elle and Salon, among others. Additionally, she has extensive experience reporting on metro, crime and breaking news. She holds a Master’s in health and medical reporting from the CUNY Graduate School of Journalism, and lives in New York City.

Presentation

David McKeon

Chief of Staff | New York Stem Cell Foundation

David McKeon is the Chief of Staff at The New York Stem Cell Foundation (NYSCF) where he works with the Chief Executive Officer and the executive team on strategy and organizational development. He also directs NYSCF’s policy and government affairs and leads scientific communications for the organization. David has been at NYSCF for 8 years, during which time NYSCF has invested approximately $150M in stem cell research.He also represents NYSCF at national and international conferences, and has given talks at federal and local government meetings. In addition, he has served on several working groups including the New York State Stem Cell Science (NYSTEM) Education Workgroup as well as the NYSTEM Advocacy Subcommittee.Prior to his work at NYSCF, David conducted research utilizing stem cells to regrow new tissues in the body at Tufts University. David graduated from Tufts University with a degree in Engineering Science and Biomedical Engineering and captained the Men’s Soccer team.

Presentation

Krishna Knabe

Director of Communications | Alzheimer’s Drug Discovery Foundation

Krishna Knabe leads the communications team at the Alzheimer’s Drug Discovery Foundation. She has more than 15 years of strategic communications experience, with a focus on developing and implementing successful brand and content strategies for nonprofit organizations. Before joining the Alzheimer’s Drug Discovery Foundation, she was Director of Communications for the International Center of Photography (ICP). During her tenure, she created and led digital campaigns that garnered coverage in the Wall Street Journal and other publications and resulted in substantial growth of ICP’s online audiences. From 2007 to 2013, Ms. Knabe managed communications at the William T. Grant Foundation, a leading funder of research to improve the lives of American youth. Her experience also includes marketing and editorial roles at major educational and arts institutions as well as national and regional publications. Ms. Knabe earned a master’s of science in strategic communications from Columbia University and a bachelor of arts from the University of Chicago.

Presentation

Alycia Halladay, PhD

Chief Science Officer | Autism Science Foundation

Alycia Halladay, Ph.D. has been involved in autism research for the past 22 years, starting her career working with one of the first NIEHS and EPA funded Children’s Centers for Environmental Health (CCEH). After completing a PhD in psychology at Rutgers she became a post-doctoral fellow in the Department of Pharmacology and Toxicology at Rutgers, working in collaboration with scientists developing the first animal models of autism spectrum disorders, capturing multiple symptoms of the disorder. In 2005 she became an Associate Director of Research at the National Alliance for Autism Research, managing the growing grants portfolio, directing the Autism Tissue Program (now Autism BrainNet) and developing collaborative research programs in high risk siblings, gene/environment interactions, environmental epidemiology and early intervention. After the merger of NAAR with Autism Speaks, she expanded early detection, intervention, environmental sciences and epidemiology portfolios. In 2014 she became the first CSO at the Autism Science Foundation, where she co-leads the outreach and communication of findings of Autism BrainNet, and coordinates the Autism Sisters Project, and manages the early career awards of the ASF. She has authored over two dozen scientific articles in autism research and writes a weekly autism science podcast for the autism community. She also has a adjunct faculty position at Rutgers University.

Presentation

5:30 - 6:45 PM

Group Reception

September 28, Wednesday

8:30AM – 9:30AM

(60 min)

Small Group Discussions over breakfast

9:30 - 10:30 AM

(60 min)

HRA News and Updates

10:30 - 12:00 PM

(60 min)

Communicating Impact of our Funding

One of the major challenges of biomedical funding organizations is to accurately and effectively communicate the impact that our funding has on advancing science, accelerating new treatment development, supporting the next generation of researchers, and ultimately helping patients. This session will feature a marketing professional who has assisted various non-profit and for-profit organizations of various sizes and communications bandwidths, including The Milken Institute, Brunswick Group, APA, etc., who will present her experiences, metrics, success stories, and even lessons learned from not-so-successful efforts to accurately and effectively communicate the impact of an organization’s funding on achieving their goals. After her presentation, the results of the HRA survey asking how members track and communicate outcomes will be shared.

Moderator

Diane Bovenkamp, PhD

Vice President of Scientific Affairs | BrightFocus Foundation

Prior to joining FFB, Dr. Laster was a faculty member of the Department of Biology at Stevenson University. Dr. Laster completed a Postdoctoral fellowship at Johns Hopkins School of Medicine in the Solomon Synder Department of Neuroscience. Her area of research focus was the study of neurodegenerative diseases. Dr. Laster holds a B.S. Natural Science from Spelman College and a Ph.D. Biological Sciences from Purdue University.

Presenter

Cecilia Arradaza

Head of Marketing, US | Brunswick Group

Cecilia Arradaza is U.S. Head of Marketing for the Brunswick Group, a global advisory firm specializing in critical issues and corporate relations. Prior to Brunswick, Cecilia was the executive director of communications for the Milken Institute where she led its global communications and marketing strategy – amplifying the impact of its programs, research, and policies and engaging with a broad range of stakeholders. Previously she led communications and policy efforts for FasterCures, a center of the Milken Institute, for six years. Before joining the institute, Cecilia brought corporate communications strategies, marketing campaigns, and public affairs initiatives to bear for clients of public relations firms Chandler Chicco Agency, Hyde Park Communications, and Powell Tate. As well, she’s worked on public relations and media campaigns for the American Psychiatric Association and the National Osteoporosis Foundation. She is on the founding board of the Women Against Alzheimer’s network and is active in a number of local and international nonprofits.

Presentation

Diana Shineman, PhD

Senior Director, Scientific Affairs | Alzheimer’s Drug Discovery Foundation

Diana Shineman, PhD is the Senior Director for Scientific Affairs at the Alzheimer’s Drug Discovery Foundation, where she develops and manages the Foundation’s drug discovery and development grant programs and strategic initiatives. Combining scientific and business expertise, the ADDF manages its research funding portfolio to balance risk, stage of development, and drug target mechanism of action, ensuring that grants meet key milestones before securing follow-on funding. As a measure of success, projects funded by the ADDF have gone on to garner nearly $2 billion in follow-on funding. The ADDF also works strategically with foundations, government and industry partners to tackle unmet needs in the field. As an example of such an initiative, Dr. Shineman led an interdisciplinary effort to standardize animal model study design to improve research efficiency and translatability.Diana joined the ADDF in 2008. She earned a Ph.D. in Cell and Molecular Biology from the University of Pennsylvania working in the Center for Neurodegenerative Disease Research led by Drs. Virginia Lee and John Trojanowski. She also worked as an Editorial Intern for the Journal of Clinical Investigation and was an active member of the Penn Biotechnology Group. Diana received a B.A. in Biology with a Nutrition concentration from Cornell University, where she was named a Howard Hughes Undergraduate Research Scholar. In addition to maintaining various professional memberships, Diana has also authored numerous articles and peer-reviewed publications.

Presentation

1:00 - 2:00 PM

(60 min)

Interactions with National Center for Advancing Translational Sciences (NCATS)

The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) was established in FY 2012 to transform the translational science process so that new treatments and cures for disease can be delivered to patients faster. NCATS leads innovative and collaborative approaches in translational science across all scientific disciplines and sectors, including with investigators from NIH, universities and medical centers, other federal agencies, small businesses and industry, and patient groups and advocacy organizations. Dr. Austin will talk about NCATS, its approach to accelerating translational science, and how the center works with foundations and patient advocacy organizations.

Moderator

Mary DeRome

Translational Research Manager | Multiple Myeloma Research Foundation

Mary DeRome joined MMRF as Translational Research Manager in February 2014. In this role she is responsible for managing the $1.3 million yearly MMRF research grant portfolio. She also helps manage the MMRF CoMMpass trial, an 8 year study of 1000 Multiple Myeloma patients, and a number of other MMRF translational research and precision medicine initiatives. Prior to her current position, Mary spent twelve years as a Senior Investigator in oncology drug discovery at Bayer HealthCare in West Haven CT and seven years as Senior Scientist in nanotechnology vaccine development at Artificial Cell Technologies in New Haven CT. Mary received her BS in chemistry, did graduate work in Pharmacology, and received her MS in Cell and Molecular Biology from the University of Connecticut, Storrs CT/University of Connecticut Health Center, Farmington CT.

Presenter

Christopher P. Austin, MD

Director | National Center for Advancing Translational Sciences (NCATS), NIH

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. 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.

Presentation

2:00 - 3:00 PM

(60 min)

Scientific Session

Advancing a potential therapy out of the laboratory and into a clinical trial is expensive, challenging, and risky, but essential to getting life-changing therapies out to the people who need them. The process, known as translational research, can cost hundreds of millions of dollars, yet the chance of success is only about 10 percent. How do promising therapies make it through the formidable translational process to become commercialized drugs, treatments, and devices? In this session, we will discuss the translational process from a funding perspective, and how a funder can leverage investments in lab studies that have commercial potential. We will hear from an industry expert about her organization’s development of a successful gene therapy program that has advanced through a Phase III clinical trial, and learn how funder dollars can help de-risk and potentially accelerate translational science.

Moderator

James Richardson, PhD

Deputy Chief Preclinical Translational Research Program Officer | Foundation Fighting Blindness

Dr. James Richardson joined the Foundation Fighting Blindness (FFB) in 2016 as Deputy Chief, Preclinical Translational Research Programs. In this role, Dr. Richardson is guiding translational research projects, helping FFB-funded investigators move potential therapies toward clinical studies. Dr. Richardson has more than 20 years of leadership experience in biomedical research and development. He is accomplished in bio-pharmaceutical product development, virology, biochemistry and vaccine development.Prior to joining FFB, Dr. Richardson was employed by Advanced BioScience Laboratories (ABL) for more than ten years, with a focus on advancing development of therapeutic and preventive products into human studies. Prior to ABL, Jim was a scientist overseeing Virus/VLP Production and Quality Control for Integral Molecular and Genovo/Targeted Genetics in Philadelphia.Dr. Richardson holds a Doctor of Philosophy in Biomedical Sciences from Mt. Sinai School of Medicine, a Master of Science in Microbiology from Colorado State University and a Bachelor of Science in Microbiology from Cornell University. He has authored/co-authored numerous publications throughout his career.

Presentation

Presenter

Sue Washer, MBA

President and CEO | AGTC

Sue Washer, President and CEO of Applied Genetic Technologies Corporation (AGTC), has developed an impressive career over the past 32 years, with diverse experience in the pharmaceutical/biologics industry, including extensive corporate start-up experience, research and development expertise as well as significant community and industry association leadership.As the founding CEO, Sue has led AGTC for 14 years transforming it from a small academic spin-out to a leader in the field with five products in active development. AGTC uses its novel gene therapy platform to develop products designed to transform the lives of patients with severe orphan genetic diseases with a particular focus in ophthalmology. Sue secured investments of over $91M for AGTC from nationally recognized VCs and granting agencies, negotiated and closed on a major collaboration with a top five Biotech company, negotiated numerous licensing deals with academic, governmental and commercial entities, led the company in efficiently completing critical milestones including five INDs and three early stage clinical trials, and recruited an experienced and highly effective management team. Sue led the Company’s successful IPO and secondary financing and raised over $90M from public market investors and most recently led the negotiations with Biogen resulting in a wide ranging partnership deal with a $124M upfront payment.Sue is Chairman of the Board of SEBIO, a member of the Advisory Board Member for the Florida High Tech Corridor Council and a member of the Executive Committee and past-Chair of the Board of BioFlorida. Under her two-year term as Chair, BioFlorida returned to sustainable operations after several years of deficit spending.Sue gained pharmaceutical management and research experience during 9 years at Abbott Labs and Eli Lilly. As a group leader in technical product development at Abbott’s Diagnostic Division, she was responsible for bringing new products and technologies from the lab bench into the market place. During this period, she won the Presidential Award for leadership in bringing a novel automated technology to the market. As a sales representative at Eli Lilly, she gained experience in marketing, sales, logistics and communications. Sue also has 8 years of senior management experience with two other entrepreneurial firms in Florida. Previously she served on the Boards of the Gainesville Area Innovation Network (GAIN), the University of Florida Center for Entrepreneurship where she was a member of the executive committee for a number of years, and the Gainesville Area Chamber of Commerce, where she co-chaired a year-long strategic re-visioning process leading to tremendous gains in cooperation between community leaders, business leaders and the University of Florida.Sue earned her undergraduate degree in biochemistry from Michigan State University and an MBA from the University of Florida where she was one of the first graduates from the Warrington College of Business Entrepreneurship Program.

Presentation

3:00 – 3:15PM

(15 min)

Wrap-Up and Adjourn