Summit on Access and Affordability in Cell and Gene Therapies
Join the ISSCR in Los Angeles, California in collaboration with the California Institute for Regenerative Medicine (CIRM) and UCLA Broad Stem Cell Research Center.
Registration is now closed.
Program Description: As cell and gene therapies move from experimental treatments to approved interventions, the healthcare community faces a critical challenge: ensuring these groundbreaking therapies reach patients who need them most.
This summit brings together senior leaders in research, clinical practice, market access, policy, and patient advocacy to address the urgent need for sustainable pricing models, scalable manufacturing approaches, regulatory strategies, and innovative financing mechanisms. Through collaborative dialogue, we aim to balance continued innovation with the fundamental principle that medical breakthroughs should benefit all patients, regardless of economic circumstances or geographic location.
Summary of Sessions
Strategies for Reducing Risks and Addressing Commercial Insurer Participation
Emerging Policy for New Cell and Gene Therapy Affordability Models
Manufacturing and Regulatory Innovations
Registration is by invitation. Invited attendees will receive an email communication outlining the registration process. Use this form to indicate your interest.
This program is in collaboration with:
Champion Sponsors:
Program
9:00 AM – 10:00 AM Welcome and Keynote Session
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Dana P. Goldman is University Professor of Public Policy, Pharmacy, and Economics at the University of Southern California and the Founding Director of the USC Schaeffer Institute for Public Policy & Government Service. The Institute houses the Schaeffer Center for Health Policy & Economics, a program now ranked 3rd globally in health economics. He is a special advisor to the Provost and previously served as Dean of the USC Price School of Public Policy. Dr. Goldman’s research spans economics, medicine, and public policy. He is the author of more than 400 articles with 25,000+ citations. He pioneered a “Netflix model” for patient treatment and has been awarded more than $120 million in research support. He is an elected member of the National Academy of Medicine, the National Academy of Social Insurance, and the National Academy of Public Administration. He is a co-founder of EntityRisk and Precision Health Economics and a scientific advisor to several companies. He is a research associate at the National Bureau for Economic Research. He graduated from Cornell University summa cum laude and earned a Ph.D. in economics from Stanford University.
10:30 AM – 12:00 PM Panel 1: Strategies for Reducing Risks and Addressing Commercial Insurer Participation
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Dave works as an advisor and consultant in the pharmacy, health care delivery and health insurance industries. He has over 50 years of experience in the health care field and has served as the CEO of several health care service and technology companies. In 2018 he co-founded Emerging Therapy Solutions (ETS) to provide the payer industry with services to manage patients in need of complex and costly treatments such as bone marrow transplants, solid organ transplants, cell therapies, and gene therapies. As CEO, Dave was responsible for all strategic planning and development, operations, and financial performance until his retirement at the end of 2023.
Prior to founding ETS, Dave served as CEO at Medication Management Systems, NovoLogix, RxHub, and United Resource Networks.
Earlier in his career he worked in executive positions in the development and management of HMOs and PBMs with American MedCenters, Partners National Health Plans and Clinical Pharmaceuticals. He started his career as a hospital pharmacist and holds a PhD in Pharmacy Administration from the University of Minnesota, and a Bachelor of Science in Pharmacy from Ohio State University.
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Panos Kefalas is the Director of the Access Strategy Group at the Cell and Gene Therapy Catapult. The group provides strategic advice to address regulatory and reimbursement evidence generation needs for novel cell and gene therapies and maximize their adoption potential; it also drives various initiatives to optimize the environment for successful therapy adoption; it includes non-clinical, clinical, regulatory, health economics and market access expertise. Previously in his career, Panos gained extensive experience in pharmaceutical pricing, reimbursement and health economics from senior roles with global consultancy firms (IOVIA, ICON, Evidera), the industry and from managing guidance development for England’s Health Technology Assessment body NICE. He has a PhD in Molecular Medicine from King’s College London and an MBA from Cranfield University (UK).
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Harlan Levine, M.D., is president of health innovation and policy for City of Hope. He also serves as the chairman of the board of AccessHope, a spinout company from City of Hope that is focused on serving the employer market and making leading-edge cancer care available to all regardless of geographical location.
Prior to City of Hope, Dr. Levine held executive roles at UnitedHealth Group, Towers Watson and Anthem Inc. He currently serves as a board member for BioScienceLA, Reimagine Health, Active Life Scientific and Vida Health, and in an advisory capacity for Quantum Health and Laguna Health. In addition, he is a member of the Accessibility and Affordability Working Group for California Institute for Regenerative Medicine and a senior fellow for Health Evolution.
He is certified by the American Board of Internal Medicine and licensed by the Medical Board of California. He earned his undergraduate degree from Harvard College and his medical degree from the University of California, San Francisco. He interned at Mount Zion Hospital and Medical Center in San Francisco and completed his residency in internal medicine at Cedars-Sinai Health System in Los Angeles.
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Anup Malani is the Lee and Brena Freeman Professor at the University of Chicago Law School, currently on leave to serve as the Chief Economist for the Centers for Medicare and Medicaid Services in Washington, D.C. His research spans health economics and policy, law and economics, and development economics. He has taught courses in health law and policy, health economics, microeconomics, law and economics, food and drug law, securities law, bankruptcy, corporate, contract, and insurance law. Malani also holds a courtesy appointment at the Pritzker School of Medicine and is a Research Associate at the National Bureau of Economic Research. He is the founding Faculty Director of the International Innovation Corps at the Harris School of Public Policy. An elected member of the American Academy of Arts and Sciences and a member of the COVID Crisis Group, Malani is a two-time recipient of the Emergent Ventures Prize. He clerked for Justice Sandra Day O’Connor on the U.S. Supreme Court and Judge Stephen Williams on the D.C. Circuit. Malani holds a J.D. and Ph.D. in economics from the University of Chicago.
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Dr. LaShawn McIver is a proven public health leader with more than 20 years of experience advancing health equity, improving health outcomes, and promoting access to quality care. In July 2023, she joined AHIP as Senior Vice President and Chief Health Equity Officer, where she leads AHIP’s industrywide Health Equity Strategic Roadmap and provides strategic direction for member-driven equity initiatives. Previously, Dr. McIver served as Director of the Centers for Medicare & Medicaid Services (CMS) Office of Minority Health and a member of the Federal Senior Executive Service, guiding CMS’s health equity strategy, co-leading the National Rural Health and Quality Strategies, and shaping the HHS Social Determinants of Health framework. She also served on the White House Interagency Policy Councils on Maternal Health and Social Determinants of Health. Earlier, as Senior Vice President for Government Affairs and Advocacy at the American Diabetes Association, she led national efforts to expand diabetes research, prevention, and equitable access to care. Dr. McIver holds an MD in International Health and Medicine and an MPH from Johns Hopkins University.
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Dr. Bob Nordyke is Vice President and Senior Policy Advisor at BlueRidge Life Sciences, where he advises biopharmaceutical organizations on commercialization, health economics, and market access strategy. With more than 25 years of experience in the health sciences and pharmaceutical industry, he brings deep expertise in health policy research, health technology assessment, value assessment, and evidence-based communication. His work focuses on identifying opportunities to increase value within healthcare systems and translating complex evidence for diverse stakeholders, including payers, policymakers, and industry leaders.
Prior to joining BlueRidge, Dr. Nordyke held leadership and advisory roles across manufacturers, policy associations, and strategy consultancies, where he built and led health economics and access strategy groups. He has authored numerous peer-reviewed publications on health economics and value assessment and has taught at the USC Sol Price School of Public Policy and the UCLA Fielding School of Public Health.
1:00 PM – 2:30 PM Panel 2: Emerging Policy for New Cell and Gene Therapy Affordability Models
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Darius Lakdawalla is Chief Scientific Officer at the USC Schaeffer Center for Health Policy & Economics and Quintiles Chair of Pharmaceutical Development and Regulatory Innovation at the USC Mann School. He holds faculty appointments at the USC School of Pharmacy and USC Price School of Public Policy, is a research associate at the National Bureau of Economic Research, and is a member of US National Academy of Medicine. Lakdawalla was recognized for transforming the study of value in healthcare, using economic insights to reorient health care value assessment around what matters to patients and their families. He developed Generalized Risk Adjusted Cost-Effectiveness (GRACE), demonstrating that health improvements are worth more to those in poorer health. His research focuses on the economics of health risks, medical innovation, health insurance markets, and healthcare industrial organization. He has testified before congressional committees on pharmaceutical innovation and drug pricing. Darius received his Ph.D. in Economics from the University of Chicago and B.A. in Mathematics and Philosophy from Amherst College. He has received multiple awards including the PhRMA Foundation Value Assessment Challenge Award and ISPOR Excellence in Research Methodology Award.
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Laurie McWright recently retired from the Centers for Medicare and Medicaid Services as the Deputy Director of the Seamless Care Models Group in the Center for Medicare and Medicaid Innovation at the Centers for Medicare & Medicaid Services. She oversaw early development of concepts for Medicare Parts B and D as well as for states. She led successful negotiations with two pharmaceutical manufacturers of recently FDA-approved gene therapies for sickle cell disease paving the way for their participation in the Center’s novel Cell and Gene Therapy Access Model. This model tests a federal-private partnership that has outcomes-based agreements with two manufacturers for deploying with states. She is now running her leadership coaching practice and consulting on improving access to high quality, affordable health care and drug therapies.
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Dr. S. Monica Soni (she/her) is the Chief Medical Officer at Covered California, leading the organization’s Health Equity and Quality Transformation division. She is working to improve the health of the nearly 2 million Covered California enrollees and the quality and affordability of care for all Californians. She is a board-certified internal medicine physician with more than a decade of experience working in both inpatient and outpatient settings and continues to see patients. She is an Associate Professor within the UCLA Department of Medicine and the Charles R. Drew University Department of Internal Medicine. Prior to joining Covered California, Dr. Soni served as Associate Chief Medical Officer within Evolent Health focused on the delivery of high-quality, cost-effective specialty care for the over 16 million supported Medicaid lives across the United States. Dr. Soni also served as the Director of Specialty Care for the Los Angeles County Department of Health Services, the second-largest municipal health system in the United States. She graduated from Harvard College and Harvard Medical School and completed residency at the University of California, San Francisco.
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Mark C. Walters, MD, is the Jordan Family Director of the Blood and Marrow Transplantation Program at UCSF Benioff Children’s Hospital, Oakland and professor and Chief, Hematology division in the Department of Pediatrics at University of California, San Francisco School of Medicine. He is Program Director of the California Institute of Regenerative Medicine alpha clinic at UCSF. He has been active in cooperative clinical transplantation trials and has participated in NIH-supported investigations of hematopoietic cell transplantation for sickle cell disease and thalassemia and in industry-sponsored gene therapy clinical trials for hemoglobin disorders. Currently, research interests are focused on genomic editing and gene addition therapies as a strategy to extend curative therapy in all patients who inherit a clinically significant hemoglobinopathy such as sickle cell disease.
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Manar Zaghlula is the Director of Public Impact at the Innovative Genomics Institute (IGI), where he leads a team of specialized program managers and researchers working to align the diverse IGI research portfolio with societal needs and values. Prior to this role, Manar served as the IGI Health Policy Manager, where he worked with key stakeholders and experts to develop innovative pathways for improving cell and gene therapy affordability and access, primarily through policy and regulation.
Before joining IGI, Manar was a CCST Science & Technology Policy Fellow in the California State Legislature. There, he served both as a committee consultant in the Assembly Environmental Safety and Toxic Materials Committee and as a legislative staffer to Assemblymember Dr. Bill Quirk. Manar analyzed numerous environmental policy proposals, guided a diverse bill portfolio through the legislative process, and covered policy areas including health and education policy. Manar holds a B.S. in Biology and Chemistry from the George Washington University and a Ph.D. in Translational Biology and Molecular Medicine from Baylor College of Medicine, where he investigated novel therapeutic entry points for a rare neurogenetic disorder.
3:00 PM – 4:30 PM Panel 3: Manufacturing and Regulatory Innovations
Sponsored by: Mytos Bio
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Amander Clark, PhD, is Professor of Molecular, Cell and Developmental Biology at UCLA and the Founding Director of the UCLA Center for Reproductive Science, Health and Education. Professor Clark is an award-winning scientist and internationally recognized expert in stem cell biology, developmental biology, and reproductive science. Results from the Clark Lab provide the foundation for engineering reproductive cells and tissues from stem cells to develop solutions for infertility and reproductive dysfunction at all stages of life. Her current interests include in vitro gametogenesis, understanding the formation of the ovarian reserve, and preserving ovarian health as a strategy to promote the health span of women and girls. Dr. Clark has authored more than 100 scientific articles with over 20,000 citations. She is regularly invited as a subject matter expert for The New York Times, The Economist, The New Yorker, and Public Radio. From 2023–2024, she served as President of the International Society for Stem Cell Research (ISSCR) and currently serves on the National Academies of Sciences, Engineering, and Medicine Health Sciences Policy Board, advancing equitable, inclusive, and publicly informed science.
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Dr. Smith is Executive Director Global Scientific Portfolio Management, Cell and Gene Therapy, CDMO at Charles River Laboratories. He has more than 40 years of experience in the cell and gene therapy field, with expertise spanning a variety of cell types and viral vector platforms. His background includes work in research, process development, GMP manufacturing, quality control and assurance, analytical and assay development, facility design and operations, and supply chain management. Most recently, he served as Chief Technology Officer at Ambys Medicines, Inc., and previously held senior leadership roles at Akouos, Inc., Bellicum Pharmaceuticals, and LifeNet Health. Earlier in his career, he was President and CEO of Cognate BioServices and held executive and R&D leadership positions at Osiris Therapeutics, Aastrom Biosciences, Geneic Sciences, Baxter Healthcare, and HyClone Laboratories. Dr. Smith has contributed to more than 30 successful regulatory applications in cell and gene therapy and has held adjunct faculty appointments at several universities. He holds a B.S. in Chemistry and a Ph.D. in Biochemistry.
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Debra Aub Webster, PhD, is a senior regulatory strategist with more than 30 years of experience advancing biotechnology and advanced therapy programs from early development through marketing authorization. Her background spans FDA review, executive leadership within emerging biotech companies, and board-level advisory roles, providing a comprehensive perspective on regulatory risk, development strategy, and long-term value creation.
Dr. Webster serves as Senior Vice President, Global Regulatory Strategy at SSI Strategy, where she partners with companies to align regulatory pathways with corporate milestones and investor objectives. She advises across therapeutic modalities and global jurisdictions, evaluates evolving regulatory landscapes, and prepares leadership teams for critical health authority interactions.
Previously, she held senior regulatory leadership positions at BlueRock Therapeutics and Cardinal Health Regulatory Sciences. She began her career as a reviewer at the U.S. Food and Drug Administration.
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Ignacio Willats is the cofounder of Mytos, a company dedicated to reducing costs and bottlenecks in regenerative medicine manufacturing to scale life-changing therapies to millions who need them. He brings enterprise sales and product experience from multiple startups, fostering a deep customer-focused culture within his team while leading sales efforts. Ignacio is a Y Combinator alumnus and a Forbes 30 Under 30 honoree.
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Krishna is Director and Head of New Modalities Business Development at Lonza, where he leads and manages strategic customer and business opportunities for the Specialized Modalities Platform that encompasses cell and gene therapy, exosomes, mRNA, and microbial protein modalities. Prior to his commercial role, Krishna managed the development of phase-appropriate processes and analytics for customers manufacturing clinically-efficacious therapies for clinical trials to commercial products at Lonza. Before joining Lonza, he was a bioprocess development engineer at the Centre for Commercialization of Regenerative Medicine in Toronto, Canada. Krishna earned his PhD in Chemical Engineering from the University of Calgary, Canada.
4:45 PM – 5:30 PM Closing Keynote
Jeannette Hacker-Stratton, City of Hope and Breakthrough T1D Participant Advisory Council, USA
5:30 PM – 7:00 PM Networking and Reception
Organizers
Amander Clark, PhD
University of California, Los Angeles, USA
Darius Lakdawalla, PhD
University of Southern California, USA
Deepak Srivastava, MD
Gladstone Institutes, USA
Maria Bonneville
California Institute for Regenerative Medicine (CIRM), USA
David J. McLean, PhD
Emerging Therapy Solutions, USA
Thomas A. Rando, MD, PhD
University of California, Los Angeles, USA
Travel & Accommodations
Hotel
The ISSCR has secured a limited number of rooms at the Luskin Hotel for the night of 19 March. Guest rooms are conveniently located above the meeting space at the UCLA Meyer & Renee Luskin Conference Center.
Luskin Hotel conveniently located above the meeting space at the UCLA Meyer & Renee Luskin Conference Center
Guest rooms based on available at the rate of $333.00 USD plus 16% occupancy tax (tax percentage is subject to change).
Contact the Luskin Hotel Reservation Team: +1-855-522-8252 and provide group code: LG2603ISS2.
Venue
UCLA Meyer & Renee Luskin Conference Center
425 Westwood Plaza, Los Angeles, CA 90095, USA
Transportation
Ground Transportation
The Luskin Convention Center is located approximately 11 miles from the Los Angeles International (LAX) Airport and an estimated 40-minute car ride.
Taxis, shuttle services, and public transit are easily available. Details and additional information can be found here.
Parking
There are several parking structures available throughout the UCLA campus. It is recommended that you park in Lot #8 as it is the closest to Summit’s venue. Click here to see additional information regarding UCLA Campus Transportation.
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Sponsors
Supporting Sponsors
Official Meeting Journal
Stem Cell Reports is an open access forum communicating basic discoveries in stem cell research, in addition to translational and clinical studies. Stem Cell Reports focuses on manuscripts that report original research with conceptual or practical advances that are of broad interest to stem cell biologists and clinicians.