Advancing Drug Development in ATTR Amyloidosis in an Evolving Treatment Landscape

Frederick L. Ruberg, MD is an Associate Professor of Medicine and Radiology at Boston University (BU) Chobanian & Avedisian School of Medicine and clinical cardiologist at Boston Medical Center (BMC), specializing in cardiac imaging and infiltrative heart disease. He attended the University of Pennsylvania Perelman School of Medicine, completed internal medicine training at Brigham and Women’s Hospital, cardiovascular disease fellowship at Boston Medical Center/Boston University School of Medicine, and a fellowship in cardiovascular magnetic resonance imaging at Beth Israel Deaconess Medical Center in Boston. He is Associate Chief of Cardiovascular Medicine for Academic Affairs, Associate Director of the Cardiovascular Medicine Fellowship program, and director of the cardiac MR program at BMC. Since 2017 he has directed the Integrated Pilot Grants Program of the Boston University Clinical and Translational Science Institute (CTSI), transitioning in 2023 to lead the BU CTSI Workforce Development programs. He serves as Senior Associate Editor of Circulation: Cardiovascular Imaging. Dr. Ruberg is a Fellow of the American Heart Association and a member of the Association of University Cardiologists. His NIH and industry funded research program applies non-invasive cardiac imaging techniques to improve the recognition and care of patients with cardiac amyloidosis, particularly that owing to the hereditary genetic TTR variant Val122Ile (or V122I). An active clinical cardiologist at the BU Amyloidosis Center, he has also co-directed numerous collaborative clinical guidelines initiatives. He is co-PI of the $7.2m NHLBI-funded Screening for Cardiac Amyloidosis with Nuclear Imaging (SCAN-MP) study. Dr. Ruberg is committed to the development of a diverse clinical and scientific workforce through direct mentorship of trainees as well as service on numerous committees charged with this mission. He has been recognized with mentorship (2017) and citizenship (2022) awards from the BU Department of Medicine.

Isabelle Lousada has been the driving force behind ARC, building successful collaborations and programs across the sectors to advance the science and understanding of the amyloidosis diseases.

Isabelle was diagnosed with AL amyloidosis and was one of the first patients to successfully undergo a stem cell transplant. For the past twenty years she has been committed to empowering other patients while serving on several boards and committees, speaking at leadership meetings and key events to encourage research, increase access, and support the critical and unmet needs of amyloidosis patients.

Dr. Maurer is a general internist and geriatric cardiologist with advanced training in heart failure and cardiac transplantation. He is the Arnold and Arlene Goldstein Professor of Cardiology at Columbia University Irving Medical Center, Vagelos College of Physicians & Surgeons, where directs the Clinical Cardiovascular Research Laboratory for the Elderly (CCRLE). Dr. Maurer is a member of the Advanced Cardiac Care Center at NewYork Presbyterian Hospital – Columbia Campus.

He has published over 350 articles including peer reviewed manuscripts, reviews and book chapters. He was Chair of the American College of Cardiology’s Geriatric Cardiology Member Section, which is the largest organization dedicated to advancing the care of older adults with cardiovascular disease. He was co-chair of the Steering Committee of the ATTR-ACT trial showing tafamidis was a safe and effective therapy for transthyretin amyloid cardiomyopathy. Throughout his career, he has promulgated an approach to older adults with cardiovascular disease that offers the best of both geriatric and cardiovascular medicine in which a comprehensive holistic approach to enhance functional capacity and quality of life is at the forefront of emerging techniques to address cardiovascular physiologic derangement that disproportionately afflict older adults.

Prem Soman is tenured Professor of Medicine, and Clinical & Translational Science at the University of Pittsburgh, Associate Chief of Cardiology (faculty development), Director of the Multidisciplinary Cardiac Amyloidosis Center, and Director of Nuclear Cardiology and the Cardiac Imaging Fellowship the at the University of Pittsburgh Medical Center (UPMC).

After initial medical training in India, he was a clinical research fellow at the Northwick Park Hospital and Institute of Medical Research at Harrow, London, UK, where he obtained a PhD. His Cardiology and Advanced Imaging Fellowships in the US were at Tufts Medical Center, Boston, after which he joined the faculty at UPMC in 2005.

Dr. Soman practices clinical cardiology (focusing on amyloid heart disease and coronary artery disease), nuclear cardiology and echocardiography at UPMC. His research interest is in the use of imaging in heart diseases. Dr. Soman has co-authored numerous original research papers, guidelines, reviews and textbook chapters. He is an Associate Editor of Circulation CV Imaging.

He was the President of the American Society of Nuclear Cardiology in 2018, and previously, Chair of the Imaging Council of the ACC, and President of the CV Council of the Society of Nuclear Medicine and Molecular Imaging. He is a recipient of the Hermann Blumgart award for distinguished service to nuclear cardiology from the CV Council of the Society of Nuclear Medicine and Molecular Imaging in 2016 and the Outstanding Educator award in 2022.

Dr. Preston Dunnmon received his medical degree from the Duke University School of Medicine, following which he completed his Internal Medicine residency and Cardiology fellowship at UT Southwestern Medical Center in Dallas. He was the recipient of an American Heart Association / Bugher Foundation Molecular Cardiology Fellowship, during which his research focus was G-protein mediated transmembrane signal transduction in the heart, and he reviewed manuscripts regarding G-proteins for Circulation. He is a Fellow of the American College of Physicians and a Fellow of the American College of Cardiology. After serving in the US Air Force as Division Chief of Cardiology at Andrews Air Force Base Medical Center and cardiac catheterization laboratory staff at both Walter Reed Army Medical Center and Bethesda National Naval Medical Center, Preston transitioned to private industry where he was Section Head of late phase clinical development for cardiovascular, GI, and GU development programs globally at Procter & Gamble Pharmaceuticals. In 2010, Preston returned to government service, most recently as clinical team leader and cross-disciplinary team leader for the Division of Cardiology and Nephrology at the US Food and Drug Administration’s Center for Drug Evaluation and Research. After departing FDA in 2021, Preston transitioned to Janssen Research and Development where he is currently Vice President, Data Science, Cardiovascular/Metabolism & Pulmonary Hypertension. He holds current board certifications in both Internal Medicine and Cardiovascular Diseases.

Rosalyn Adigun, MD, PharmD, MSc received her MD and PharmD degrees from University of Texas Medical Branch and University of Houston College of Pharmacy respectively. She completed training in Adult Cardiology, Advanced Echocardiology, and Advanced Heart Failure and Transplant Cardiology at Mayo Clinic Rochester. She also holds a M.Sc. in Clinical and Translational Sciences from Mayo Clinic School of Biomedical Sciences with an area of interest in Cardiac Amyloidosis. Dr. Adigun is a Medical Officer within the Division of Cardiovascular and Renal Products, Office of New Drugs in CDER/FDA.

Adrian Hernandez, MD, MHS, is Executive Director, Duke Clinical Research Institute, and Vice Dean of the Duke University School of Medicine. Dr. Hernandez has research interests in improving cardiovascular health and accelerating clinical evidence through outcomes research, clinical trials, comparative effectiveness and health policy. Dr. Hernandez is a member of the American Society for Clinical Investigation and American Association of Physicians. He had led multiple large-scale patient-centered research programs, registries, research alliances and clinical trials aimed at improving health across multiple conditions such as NIH’s Health System Collaboratory and the PCORI-funded PCORnet®, the National Patient-Centered Clinical Research Network. He has authored over 700 peer-reviewed publications.

Dr. Masri’s specializes in caring for patients with conditions that result in abnormally thickened hearts, such as hypertrophic cardiomyopathy, amyloidosis and Fabry’s disease. Dr. Masri trained in Internal Medicine at the Cleveland Clinic, where he developed his passion for improving the lives of patients with heart disease. Subsequently, he moved to the University of Pittsburgh, where he completed training in cardiovascular diseases. Dr. Masri is also an expert in cardiac imaging. He spent two years at the University of Pittsburgh supported by a grant from the National Institute of Health, focusing on cardiac magnetic resonance imaging, nuclear cardiology, and echocardiography. These modalities constitute the cornerstone of diagnosing and following up patients with thickened hearts. From diagnostic approaches to therapeutics, Dr. Masri led numerous research projects that resulted in publications in leading academic journals. 

Dr. Alanna A. Morris is an Associate Professor of Medicine in the Division of Cardiology at Emory University School of Medicine and the Emory Cardiovascular Clinical Research Institute. Currently she serves as the Director for Heart Failure Research. Dr. Morris received her undergraduate degree in Biology from Xavier University of Louisiana, and her medical degree from the Harvard Medical School. She completed her residency at Brigham and Women’s Hospital in Boston, Massachusetts. Subsequently, she came to Emory University to complete fellowships in general cardiology, and advanced heart failure and transplant. Her clinical time is spent as faculty in Emory’s Center for Heart Failure Therapy and Transplantation. Her research program focuses on studying mechanisms that underlie race- and gender disparities in heart failure risk and progression, including the role of biologic pathways as well as the social determinants of health.

Amy Feinberg (Boulder, CO) is in pursuit of life by being an active advocate and representative of the Amyloidosis community for the purpose of scientific advancement and to keep her kids from ever having to suffer the journey down the disease’s pathway. Amy is also a theatre director, producer, and developer and coaches acting whenever she can. Prior to diagnosis, she was the producing artistic director of The Hypothetical Theatre Company in NYC, Associate Professor of Theatre at The University of the Arts in Philadelphia, and the creator of Ellis Island Stories which ran for 8 years at the Immigration Museum with the National Park Service. She’s the proud mom of Josh and Sydney, 21 and 17.

Andrew Slugg is the Senior Vice President and Global Head of Regulatory Affairs at Alnylam Pharmaceuticals, Inc where he’s worked with the development teams that have helped bring forth two approved products for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis. Prior to joining Alnylam, Andrew served as Vice President of Regulatory Affairs at ARIAD Pharmaceuticals and served in roles in regulatory affairs, quality assurance, and clinical operations in biopharmaceutical companies and contract research organizations.

Angela Dispenzieri, M.D., is a consultant in the Division of Hematology, Department of Internal Medicine at Mayo Clinic. She has joint appointments in the Division of Clinical Biochemistry and Immunology, Department of Laboratory Medicine and Pathology, and the Department of Molecular Medicine. Dr. Dispenzieri holds the academic rank of Professor of Medicine and of Laboratory Medicine and Pathology, Mayo Clinic College of Medicine, and she is recognized with the distinction of a named professorship, the Serene M. and Frances C. Durling Professorship. She joined the staff of Mayo Clinic in 1998 and served as Chair of Hematology Research from 2014 to 2022. She served as the President of the International Society of Amyloidosis from 2016 to 2018, and currently chairs the Mayo Clinic Amyloid Interest Group.

Cynthia Welsh, M.D., is a medical officer on the Rare Diseases Team in the Division of Rare Diseases and Medical Genetics at the Food and Drug Administration’s (FDA’s) Center for Drug Evaluation and Research (CDER) where her duties include rare disease educational programming for reviewers and serves as a member of a multidisciplinary resource team for rare disease product development. Dr. Welsh joined the FDA in 2006 as a reviewer of diagnostic and therapeutic radiopharmaceuticals and radiation medical countermeasures prior to moving to the Rare Diseases Team. She received her M.D. and completed her residency in radiation oncology at Georgetown University.

Prof. Dalia Dawoud is Associate Director (Research) at the National Institute for Health and Care Excellence (NICE).  She holds a PhD in pharmaceutical policy and economics from King’s College London. Her current work is focused on advancing the methods of HTA and clinical guideline development where she is leading NICE input into European Commission funded projects, such as IMI EHDEN and HORIZON 2020 HTx, and its newly established HTA Innovation Laboratory (HTA Lab). She serves on a number of research projects’ advisory boards and as Associate Editor of ISPOR journal Value in Health. She also holds Professor position at the Faculty of Pharmacy, Cairo University, Egypt and is Director-elect (2023-2026) on ISPOR Board of Directors.

Dr. Dhruv Kazi is a cardiologist and health economist who serves as the Associate Director and Section Head of Health Economics at the Richard A. and Susan F. Smith Center for Outcomes Research in Cardiology in Boston, Massachusetts.  He is an Associate Professor at Harvard Medical School, the Director of Cardiac Critical Care at Beth Israel Deaconess Medical Center, and an affiliate faculty member of the Center for Vulnerable Populations at the University of California San Francisco. Dr. Kazi’s research examines the real-world cost effectiveness of clinical and policy interventions for cardiovascular prevention in vulnerable populations. A particular focus of his work is examining the long-term health and economic outcomes of novel prevention and treatment strategies when deployed at scale – across a health system, region, or nationwide. His research has helped improve access to novel cardiovascular therapies and continues to influence national clinical guidelines. Through his scientific leadership role in the American Heart Association, Dr. Kazi advocates for universal access to affordable cardiovascular prevention and care, including in resource-scarce settings in the US and overseas.

After completing medical school at the University of Mumbai, Dr. Kazi completed his clinical training at Baylor College of Medicine and the University of California San Diego, and his research training as at the London School of Economics and Stanford University. Dr. Kazi’s research has been funded by the National Institutes of Health, the Agency for Healthcare Research and Quality, the Centers for Disease Control and Prevention, the American Heart Association, the World Heart Federation, and the Hellman Family Foundation and has been published in numerous high-impact journals. Relevant to this talk, Dr. Kazi led the economic evaluation of tafamidis that appeared in Circulation in 2020.

Dr Emmanouil Zouridakis is Leading Senior Medical Assessor at the Healthcare, Quality and Access Group of the UK Medicines & Healthcare Products Regulatory Agency (MHRA), working in the Assessment Unit that is responsible for cardiovascular, diabetes and renal products.

Dr Zouridakis is a cardiologist and worked at different teaching hospitals before joining the MHRA in 2004. During his time at MHRA he has been the lead clinical assessor in a wide range of regulatory procedures involving licensing of new cardiovascular and diabetes medicines, drug/device combinations, class-wide benefit-risk reviews and scientific advices to companies. He is a principal assessor at the Cardiovascular Expert Advisory Group of the UK Commission on Human Medicines.

Dr. Franca Angeli is a Global Clinical Lead at Pfizer. In her current role, Franca is responsible for leading the Tafamidis program. She is also the global product development representative for internal pipeline assets in early phase development and for technical due diligence on business development opportunities in Rare Cardiology.

Prior to Pfizer, Franca has served in senior clinical research and development roles for several biotech companies and has been an academic clinical researcher at Texas Heart Institute, University of California, San Francisco and University of Pennsylvania Medical School.

Franca holds a PhD in Clinical Research from the Pontificia Universidade Catolica (PUC-RS), Brazil, and a MD from the Universidade de Caxias do Sul, Brazil. She completed her post-doctoral fellowship at the University of Texas, Texas Heart Institute in Houston and underwent a Cardiology Fellowship at the PUC-RS, Brazil.

Francesca (Fran) Cunningham, Pharm.D. is Associate Chief Consultant, Pharmacy Benefits Management Services (PBM) and the Director of the VA Center for Medication Safety (VAMedSAFE). Dr. Cunningham’s work focuses on assessing new agents where safety data is lacking, and older medications exhibit emerging safety signals requiring evaluation. She designed the VAMedSAFE Drug Safety Quality Improvement programs which aids in tracking multiple ADEs and drug safety initiatives at the national level. Under her direction, the Center became VA’s comprehensive pharmacovigilance program contributing to VA national decisions and provider awareness of drug and vaccine safety issues. Dr. Cunningham serves as the VA’s federal Interagency representative for medication and vaccine safety. Her work has led to national awards for her contributions in pharmacovigilance and medication safety including the Mark A. Wolcott National Award for Leadership in Healthcare: Medication Safety and the Arthur S Fleming Award, given for her work in medication safety.

George “Hoss” Christensen is a 68 year old retired hightech business executive and a fourth generation native California farmer. Born and raised on the central coast of California, he lives on the family farm with his wife where they grow premium table vegetables.

During his 25+ years working in Silicon Valley he held senior executive positions at notable companies like Cisco Systems, JDS Uniphase and Avaya. Hoss is well known for his international customer management and problem solving skills. During that same period, he also helped start and manage new companies in the field of solar energy, intelligent E911 consoles and other telecommunications related opportunities.

When not managing farm issues, Hoss enjoys outdoor activities, live sports events and anything to do with his four grandsons.

Dr. Jie (Jenni) Li is Associate Director for Real World Evidence (RWE) at the Office of Surveillance and Epidemiology in CDER FDA, leading office activities relating to RWE matters. She oversees OSE’s scientific input into guidance and policy development for RWE. Jenni has more than 15 years research experiences in pharmacoepidemiology and clinical studies, designing and conducting observational studies using large electronic healthcare databases, national surveys and patient registries. Jenni joined FDA as an epidemiology reviewer in 2012, then worked as Team Leader, covering various therapeutic areas over the years.

Jonathan C Fox, MD, PhD, FACC, is currently President and Chief Medical Officer for the cardiovascular and renal diseases division at BridgeBio Pharma (NASDAQ: BBIO). Prior to joining BridgeBio, Dr. Fox was the founding Chief Medical Officer of MyoKardia, Inc. He has previously held executive and leadership positions at SmithKline Beecham, Merck Research Laboratories and AstraZeneca.

Dr Fox received his AB, PhD, and MD from the University of Chicago. He completed postgraduate training in Internal Medicine and Cardiology at Duke University and is a Fellow of the American College of Cardiology.

Dr. Fox is currently adjunct Professor of Medicine at the Stanford University Cardiovascular Institute and was previously on the faculty of the University of Pennsylvania School of Medicine. He is a longstanding Trustee of the Lankenau Institute for Medical Research in Wynnewood, PA, and serves on the Board of Directors of both MLBio Solutions (a majority held BridgeBio affiliate) and Edgewise Therapeutics.

Professor Julian Gillmore undertook MD and PhD degrees in the field of amyloidosis.  His research interests include pathogenesis, diagnosis and treatment of amyloidosis.  He was appointed Centre Head at UCL’s Centre for Amyloidosis in 2019 and is research lead at the UK National Amyloidosis Centre.  He has published more than 300 peer-reviewed articles, has supervised a number of students through post-doctoral research degrees, is CI/PI on a range of amyloid-related clinical trials, and has received extensive grant funding.

Dr. Ferdinand is Professor of Medicine at Tulane University School of Medicine and Gerald S. Berenson Endowed Chair in Preventative Cardiology. He received his medical degree from the Howard University College of Medicine in Washington, DC. He is board-certified in internal medicine and cardiovascular disease, and certified in nuclear cardiology. Dr. Ferdinand is a past Chair of the National Forum for Heart Disease and Stroke Prevention and prior Chief Science Officer and chair of the Association of Black Cardiologists (ABC).

Ken SAKUSHIMA is a medical reviewer of Pharmaceuticals and Medical Devices Agency (PMDA).

He is a neurologist and engaged in reviews of new drugs for neurological diseases.

He is also involved in orphan drugs and patient centiricity activities in PMDA.

Dr. Keyur Shah joined the Pauley Heart Center at Virginia Commonwealth University in 2009 and is the Chief of the Section of Heart Failure. He specializes in Advanced Heart Failure and Heart Transplantation, and has a special interest for caring for patients with amyloid cardiomyopathy. At VCU, he leads a multidisciplinary amyloidosis clinical program that cares for patients from throughout the state.

Kristen has over ten years of clinical operations experience within the pharmaceutical industry. She has a wealth of experience in managing complex global clinical trials for novel treatments of rare diseases. Kristen is building and overseeing the ARC’s clinical research program and manages a prioritized portfolio of vital research.

Kristen has over 20 years of experience designing and implementing a broad range of health research studies. In her current role at QualityMetric (QM), she directs a quantitative research team dedicated to patient-centered research and outcome measurement. While at QM, much of her research has focused on better understanding the impacts of both light chain (AL) and transthyretin (ATTR) amyloidosis on patients and caregivers, culminating in more than 10 peer-reviewed manuscripts, 40 de novo conference presentations, and the development of a disease-specific PRO for use in ATTR amyloidosis.

Dr. Jawidzik is currently the acting deputy director in the Division of Neurology 1 in the Office of Neuroscience. Dr. Jawidzik earned her undergraduate degree in molecular biology from Princeton University, and her M.D. from the Georgetown University School of Medicine. She is board certified in neurology, epilepsy, and clinical neurophysiology. Prior to joining FDA, Dr. Jawidzik was in academic practice at Rush University Medical Center in Chicago. She has been with the FDA since 2015 initially as a clinical reviewer on the migraine team. Since that time, she has served in various positions in DN1 including clinical reviewer, team leader, associate director, and deputy director.

L.J. Wei is a professor of Biostatistics at Harvard University. Before joining Harvard, he was a professor at University of Wisconsin, University of Michigan, and George Washington University. His main research interest is in the clinical trial methodology, especially in design, monitoring and analysis of studies. He has developed numerous novel statistical methods which are utilized in practice. He received the prestigious Wald Medal in 2009 from the American Statistical Association for his contribution to clinical trial methodology. He is a fellow of American Statistical Associating and Institute of Mathematical Statistics. In 2014, to honor his mentorship, Harvard School of Public Health established a Wei-family scholarship to support students studying biostatistics. His recent research area is concentrated on translational statistics, the personalize medicine under the risk-benefit paradigm via biomarkers and revitalizing clinical trial methodology. He has more than 250 publications and served on numerous editorial and scientific advisory boards including data monitoring for governments and industry. L. J. Wei has extensive working experience in regulatory science for developing and evaluating new drugs/devices.

Dr. Lynnette Henshaw, PharmD, BCOP is a clinical pharmacy specialist in hematology/oncology at Boston Medical Center as well as the residency program coordinator for the PGY-2 oncology pharmacy residency program. She received her doctorate of pharmacy degree from St. John’s University. Her first post graduate training year was completed at NewYork-Presbyterian Brooklyn Methodist Hospital, subsequently she completed a PGY-2 hematology/oncology pharmacy residency program at Boston Medical Center where she currently works. In her role she has an active focus on specialty pharmacy/target therapies and is passionate about patient-centered care, quality improvement/research and teaching.

Matt has over 22 years of drug development experience across numerous therapeutic areas, building and leading teams in different functions, including Clinical Science, Regulatory, Safety Science, and Medical Affairs.

He graduated from the Johns Hopkins University School of Medicine and completed his internal medicine residency at UCSF. After practicing and teaching at the Greater Baltimore Medical Center internal medicine residency program, Matt joined the biotech/pharmaceutical industry where he held positions of increasing responsibility in clinical development and medical affairs, developing small molecules/biologics in oncology, cardiology, anesthesiology, ID, rheumatology/immunology, and ophthalmology

Matt joined Attralus as Senior VP of Clinical Development in April 2023.

After working as a clinical academic cardiologist in the UK NHS for more than 20 years (with a particular interest in heart failure, and innovation and implementation) I was delighted to take up the role of Clinical Vice President in Late Stage (Cardiovascular, Renal & Metabolic) R & D at AstraZeneca, based out of Cambridge UK, as from August 2022. From April 2023 I became responsible for the cardiovascular (including heart failure) portfolio.

Previously I was Chair of the Digital Health Committee of the European Society of Cardiology (2019-2022) and from 2016-2020 was a Non-Executive Director of the National Institute for Health and Care Excellence (NICE). I was a Consultant Cardiologist at the Royal Brompton Hospital (part of Guy’s & St Thomas’ NHS Foundation Trust) and led the multi-professional heart failure service there from 2001 until 2022, looking after thousands of patients with complex cardiovascular problems.

Dr. Megan Azzarone, PharmD is a clinical pharmacist currently practicing specialty pharmacy at Shields Health Solutions with a focus in CLL/SLL and cardiology. As part of the Shields clinical pharmacist team, she helps provide outstanding patient care where individual needs and differences are cared for and valued. She received her Doctor of Pharmacy degree from Duquesne University.

Michael Maitland, MD, PhD, is Sr. Director, Clinical Development at Intellia Therapeutics. Intellia is a clinical stage biotechnology company in Cambridge, MA that develops therapeutics based on gene-editing tools. For 2.5 years Dr. Maitland has served as the Medical Lead for the NTLA-2001 program, an investigational CRISPR-Cas9-based gene-editing therapy for transthyretin amyloidosis.

He is also Professor of Medicine at the University of Virginia and a member of the University of Virginia Comprehensive Cancer Center. Before joining Intellia Dr. Maitland led a research program with > 20 years of continuous NIH funding to work at the Academic/Industry interface to develop genetic, proteomic, and imaging biomarkers to improve safety and efficacy of prescription medications. He recently completed service as a Director of the American Board of Clinical Pharmacology, is a former member of the National Cancer Institute Investigational Drug Steering Committee, and currently serves on the American Society of Clinical Oncology Cancer Research Committee.

Dr. Roberts is co-founder and CEO of Corino Therapeutics. He is a pharmaceutical and biotech consultant and owner of MAC B Consulting. Prior to starting Corino, Dr. Roberts was VP of Business Development and Corporate Officer of Chelsea Therapeutics, Inc., Director of Business Development for their Nektar Therapeutics’ Molecule Engineering technology, and Manager of Biopharmaceutical Research at Shearwater Corporation. Dr. Roberts has been an executive in the pharmaceutical research, development and corporate development space for more than 25 years.

Michele F. Mercuri, MD, PhD, FAHA, FESC, is a physician scientist professionally devoted to foster the discovery,
research, translation and development of innovative diagnostic, prophylactic, and therapeutic medical tools. Currently, Dr.
Mercuri, lead the Metabolic and other Diseases Therapeutic Area [MeTA+] at Alexion/AstraZeneca Rare Disease
He is a member of the Scientific Advisory Board for Perosphere Tech. Dr. Mercuri graduated in medicine and surgery from the University of Perugia (Italy), and trained in Internal and Geriatric Medicine at the Universities of Perugia, Parma (Italy) and Modena (Italy). A fellow in Cerebrovascular Disease and Vascular Ultrasound at the Bowman Gray School of Medicine at Wake Forest University (Winston-Salem, NC, USA), Dr. Mercuri served on the Medical School Faculty and as the Co-Director of the Division of Vascular Ultrasound. Dr. Mercuri joined the Pharmaceutical Industry with Merck Research Laboratory (Rahway, NJ, USA) in 1996 and worked on a wide range of cardiometabolic drugs in all phases of research and development. In 2003, Dr. Mercuri moved to Novartis Pharmaceuticals’ Clinical Development & Medical Affairs (East Hanover, NJ, USA) to lead Cardiometabolic Disease and Atherosclerosis drug development prior to assuming leading positions within the Methodology and Innovation Global Development group, and in the Division of Hematology and Oncology. Prior to joining Alexion Pharmaceuticals in 2018, Dr. Mercuri worked at Daiichi Sankyo where he led the development of edoxaban—an oral, direct factor Xa inhibitor. Dr. Mercuri had various appointment serving as the Global Head for cardio, vascular, metabolic and renal disease Therapeutic Area for translational sciences and clinical development, Chief Medical Advisor to the Head of Development, and Head of Clinical Development for the Americas. Dr. Mercuri published over 100 articles in peer reviewed medical and scientific journals in collaboration with leading scientists from around the world.

Dr. Michelle Campbell is the Associate Director for Stakeholder Engagement and Clinical Outcomes in the Office of Neuroscience, Office of New Drugs (OND) in FDA’s Center for Drug Evaluation and Research. Previously, Dr. Campbell was a reviewer on the Clinical Outcome Assessments (COA) Staff and Scientific Coordinator of the COA Qualification Program in OND. Dr. Campbell’s focus is in patient-focused drug development and the use of patient experience data in the regulatory setting.

Michelle Kittleson is Professor of Medicine at Cedars-Sinai and Director of Education in Heart Failure and Transplantation at the Smidt Heart Institute.  She graduated from Harvard College and received her medical degree from Yale University.  She completed residency training at Brigham and Women’s Hospital and cardiology fellowship at Johns Hopkins, where she also received a PhD in Clinical Investigation.

Dr. Kittleson is Deputy Editor of the Journal of Heart and Lung Transplantation, on writing committees for the 2022 ACC/AHA Hypertrophic Cardiomyopathy Guidelines and the 2022 ACC/AHA/HFSA HF Guidelines, Co Editor-in-Chief for the ACC Heart Failure Self-Assessment Program, and on the Board of Directors for the Heart Failure Society of America.  Her essays have appeared in the New England Journal of Medicine, Annals of Internal Medicine, and JAMA Cardiology and poems in JAMA and Annals of Internal Medicine. Her book, Mastering the Art of Patient Care, is available from Springer publishing.

Mona Fiuzat, PharmD, FACC, FHFA, FHFSA is an Associate Professor of Medicine at Duke University, Senior Scientific Advisor for FDA, Division of Cardiology and Nephrology (CDER, DCN), former Executive Editor and current editorial consultant for JACC: Heart Failure, and former Senior Scientific Advisor to the FDA Commissioner. Her clinical research experience has been in clinical trials with a focus on pharmacogenetics in heart failure, and she helped file an NDA for the first proposed pharmacogenetically targeted heart failure drug. She worked in the Heart Failure research program at Duke on a number of key clinical trials as an investigator and steering committee member, and has authored or co-authored over 100 papers in the field of heart failure. Most recently, she helped develop and facilitate the “Heart Failure Collaboratory”, an effort of government agencies, academicians, patients, societies and payers to improve heart failure research and therapeutic development. She is a former Program Co-Chair for the Heart Failure Society of America’s (HFSA) Scientific Sessions, former Membership Co-Chair, Research Network Taskforce, HFSA Secretary, and now serves as HFSA Treasurer. She serves on the HFSA Board of Directors and Executive Committee, ACC Education and Outcomes Committee, and AHA Nominations Committee. She was the recipient of the 2019 ACC Distinguished Associate Award.

Motiur Rahman is a Senior Epidemiologist at Real-World Evidence (RWE) Analytics in the Office of Medical Policy, Center for Drug Evaluation and Research (CDER), Food and Drug Administration (FDA). His responsibilities include developing guidance, improving internal Agency processes, stakeholder engagement, collaborating on Agency-funded demonstration projects, and providing consultancy on RWE study submissions. He joined FDA in April 2022 after a 10+ years of working experience in academia and industry in conducting RWE studies across a wide range of therapeutic areas. Dr. Rahman received his Pharmacy degree from University of Dhaka, Bangladesh, and his PhD in Pharmacoepidemiology and Master’s in Statistics from Auburn University, AL.

Dr García-Pavia is the director of the Inherited Cardiac Diseases and Heart Failure Unit at the Department of Cardiology of Hospital Universitario Puerta de Hierro in Madrid, Spain. The Unit is designed as national reference unit (CSUR) for inherited cardiovascular diseases by the Spanish Ministry of Health and also acts as a European reference center for rare cardiovascular diseases (ERN).

His areas of research include heart failure, amyloidosis and cardiomyopathies. He is former member of ESC Council on Basic Research, former member of the Nucleus of ESC working group on myocardial and pericardial diseases, and former president of Spanish Society of Cardiology’s working group on inherited cardiac diseases. He has published >200 articles in peer-reviewed journals including European Heart Journal, J Am Coll Cardiol, Circulation, Nature Communications, Nature Medicine and The Lancet.

Dr. Renee Bullock-Palmer is the Director of Non-Invasive Cardiac Imaging and Director of the Women’s Heart Center at Deborah Heart and Lung Center in NJ. She’s a Clinical Associate Professor, Department of Medicine, Division of Cardiology at Thomas Jefferson University, Philadelphia, PA.

She is a multimodality imaging expert. Her other areas of clinical interest include women’s heart health, cardiac amyloidosis and adult congenital heart disease.

Dr. Bullock-Palmer is Governor elect for the NJ chapter of the American College of Cardiology (ACC) and is President of the board of the Southern NJ American Heart Association (AHA). She is also on the American Society of Nuclear Cardiology (ASNC) board of directors. She is on the editorial board of the Circulation Imaging journal of the AHA. Dr. Bullock-Palmer has worked as a site Principal Investigator for the NIH sponsored PROMISE and the PROTECTION VI trial. She has several publications in peer reviewed journals.

Robyn Bent is the director of the Patient-Focused Drug Development (PFDD) Program in the Center for Drug Evaluation and Research (CDER). PFDD is an effort to systematically obtain patient input and facilitate the incorporation of meaningful patient input into drug development and regulatory decision making.

Prior to joining FDA, Robyn held several positions at the National Institutes of Health. Robyn has a background in pediatric oncology nursing and extensive experience in clinical trial design, conduct, and oversight. She continues to practice as a registered nurse. Robyn earned her Bachelor of Science in Nursing from The Catholic University of America and her Master of Science degree from the George Washington University.

Dr. Sylvia Kuehn is a clinical assessor at the Federal Institute for Drugs and Medical Devices (BfArM, Germany), unit of Neurology and Psychiatry. She has been involved in neurological procedures at the European Medicines Agency (EMA) and in scientific advice procedures for the Scientific Advice Working Party of the EMA.

Dr. Kuehn received her medical degree from the University of Mainz, where she earned her doctorate (Neurochemical Laboratory, Department of Psychiatry). Before joining the BfArM, she worked as intern and resident in Neurology at the University of Mainz and the Westerwaldklinik Waldbreitbach.

Dr. Sanjurjo currently serves as the Director of Clinical Development at Ionis Pharmaceuticals, providing strategic and clinical leadership while overseeing multiple Phase IV studies. As one of the original members of the CardioTTransform study, she prioritized ensuring equity within the trial. With a successful track record in pharmaceutical companies such as Lundbeck, Braeburn, Allergan, and Novartis, with significant contributions to medical affairs, clinical development, and medical operations. Throughout her 20 plus year career, Dr. Sanjurjo has received numerous accolades, but she takes particular pride in her recognition for advancing health equity initiatives. Her unwavering dedication involves advocating for healthcare access, addressing disparities, and fostering inclusivity in medical, clinical, and marketing strategies. All in pursuit of equal health opportunities for people of color.

Wiley A. Chambers, MD, is the Director of the Division of Ophthalmology in the Office of New Drugs, Center for Drug Evaluation and Research at the Food and Drug Administration (FDA). After receiving an undergraduate degree from Colgate University, Dr. Chambers completed medical school and a residency in Ophthalmology at The George Washington University School of Medicine and Health Sciences in Washington, DC. He is currently a Clinical Professor of Ophthalmology and Adjunct Assistant Professor of Computer Medicine at The George Washington University. He joined the FDA in 1987, as a primary reviewer for ophthalmic drug products and in 1990 became a Supervisory Medical Officer for Ophthalmologic Drug Products. In this capacity, Dr. Chambers has supervisory responsibility for the clinical review of ophthalmologic drug products and ophthalmic therapeutic biologic products submitted to the Center for Drug Evaluation and Research. Additionally, Dr. Chambers is the recepient of numerous Public Health Service, FDA and Center for Drug Evaluation and Research awards for his work with the FDA and he has served as the American Academy of Ophthalmology’s Delegate to past United States Pharmacopeia Conventions.