Focused Ultrasound for Gene and Cell Therapy Workshop Participants

Karen Aboody, MD

Dr. Aboody received her MD from Mount Sinai School of Medicine and completed her post-doctoral training in Molecular Neurogenetics at Massachusetts General Hospital, Harvard Medical School, where she discovered the tumor-tropic properties of neural stem cells (NSCs). She has been internationally renowned as a pioneer in this field since her seminal publication in the Proceeding of the National Academy of Sciences (PNAS) in 2000. She joined City of Hope in 2003 to head a translational stem cell-oncology research laboratory focused on using NSCs to selectively deliver therapeutic payloads to invasive and metastatic solid tumors. She is at the forefront of stem cell targeted cancer therapies, with 4 first-in-human phase I clinical trials for high-grade glioma patients. Therapeutic strategies include enzyme/prodrug gene therapies and oncolytic viro-immunotherapy. Her team is currently investigating potential applications of NSC targeted treatment for metastatic cancers. She has also recently been investigating the use low intensity focused ultrasound (LIFU) to target i.v. delivered stem cell exosomes to the brain, as an anti-inflammatory therapeutic for central nervous system degenerative disorders, including chemobrain.

Raag Airan MD, PhD is a clinical neuroradiologist at Stanford University and directs a laboratory developing and clinically translating technologies for focused ultrasound targeted drug and gene delivery, and ultrasonic glymphatic modulation.

Jose Amich is a graduate of Harvard University, and part of the MIT Sloan MBA deferred admission program. Prior to Z Imaging, Jose was a research fellow at Harvard Medical School studying breast cancer. He also co-founded Dotacle Technologies, where he led a clinical study with the Massachusetts General Hospital in Western Kenya to evaluate the efficacy of a novel low-cost visual aid system.

Dr Arvanitis is a joint Assistant Professor at the Woodruff School of Mechanical Engineering and the Coulter Department of Biomedical Engineering at Georgia Institute of Technology and Emory University. Dr Arvanitis research program is focused on ultrasound biophysics and bioengineering and its overarching goal is the discovery and translation to the clinics of novel therapeutic interventions against human disease. His lab is particularly active in the field of brain research, where they investigate ultrasound meditated mass transport and drug delivery in the brain and develop computational tools to support the more rational design of focused-ultrasound-based treatment of brain cancer.

Isabelle Aubert obtained her PhD from the Department of Neurology & Neurosurgery at McGill University. As a post-doctoral fellow at the Salk Institute for Biological Studies, she developed regenerative gene therapy approaches for the aging brain. Currently, she works as a Senior Scientist at Sunnybrook Research Institute and a Professor at the University of Toronto. Dr. Aubert holds a Tier 1 Canada Research Chair in Brain Repair and Regeneration and is an elected Fellow of the Canadian Academy of Health Sciences.

Through collaborative work, she leads the Brain Repair Group at Sunnybrook. The team uses ultrasound-mediated blood-brain barrier modulation, with and without exogenous therapeutics, to reduce pathology and improve brain plasticity, regeneration, and function in animal models of neurodegenerative disorders. For over a decade, non-invasive gene delivery to the brain using recombinant adeno-associated viruses and focused ultrasound has been central to Dr. Aubert’s research program. In building partnerships, Dr. Aubert aims to bring the potential of focused ultrasound-mediated gene therapy to patient populations.

Alec Batts is currently a PhD candidate under the guidance of Dr. Elisa Konofagou in the Department of Biomedical Engineering at Columbia University. One of Alec’s active research aims is centered around using novel platforms for focused ultrasound-facilitated blood-brain barrier opening for viral vector-mediated gene delivery in experimental models of Parkinson’s disease and Huntington’s disease.

Javier Blesa was graduated in Biochemistry and Biology at the University of Navarra, Spain. He obtained a PhD in Neurosciences from the Center for Applied Medical Research (CIMA) under the expert guidance of Dr. Jose A. Obeso. During his PhD he attained grants to join Dr. Thomas Wichmann lab (Emory University, Atlanta) and Dr. Oleh Hornykiewicz (Univ. Vienna) lab as well. After obtaining his PhD, he obtained the fellowship from the Spanish Minister of Education, and he moved to New York to the lab of Dr. Serge Przedborski at Columbia University. After 4 years at Columbia, he joined the lab of Dr. Paul Greengard (2000 Nobel Prize in Physiology or Medicine) at Rockefeller University in New York until he moved back to Spain to join the new Centre for Integrative Research in Madrid as a Research Scientist. He is member of the Editorial Board of Neurobiology and Disease and Frontiers of Neuroanatomy and ad hoc reviewer of top neuroscience journals. He is author or co-author of more than 55 publications in the field of Parkinson’s disease in top journals like Nature Reviews Neuroscience, Nature Neuroscience, Neuron, Science Advances, Molecular Therapy or Annals of Neurology (H factor=28; >6300 cites). He has served as expert evaluator for the European Commission, the French National Research Agency (FRA), the Dutch Research Council (NOW), the Luxembourg National Research Fund (FNR), and other national public and private entities. In the last years he has been honored to receive the “Marie Sklodowska-Curie Actions Seal of Excellence” and The Alpha&Omega Prize for young researchers, and different funding from public and private institutions.

Dr. Bollard received her medical degree at the University of Otago in Dunedin, New Zealand. She is board certified both in pediatrics and hematology. She worked in New Zealand and London, England, before moving to Houston, Texas, in 2000 where she was a Professor of Pediatrics, Medicine, and Immunology at Baylor College of Medicine (BCM). In August 2013, she was recruited to Washington, DC, to join Children’s National Hospital and George Washington University. She is the Bosworth Chair for Cancer Biology, Director of the Center for Cancer and Immunology Research, and Director of the Program for Cell Enhancement and Technologies for Immunotherapy (CETI) at Children’s National Hospital. She is a Professor of Pediatrics and of Microbiology, Immunology, and Tropical Medicine at George Washington University and the Associate Center Director for Translational Research and Innovation at the GW Cancer Center. Dr. Bollard is a member of the American Society for Clinical Investigation, is a past president of the International Society for Cellular Therapy, and the current President of the Foundation for the Accreditation of Cellular Therapy. She was an associate editor for the journal Blood from 2014-2021 and is currently Editor in Chief of Blood Advances. She has >200 peer reviewed publications and has been independently NIH funded for over a decade. Her bench and translational research focuses on improving outcomes for patients after transplant and on the development of novel cell therapies for cancer and virus-associated diseases.

Mayumi is a Senior Principal Engineer in the Pharma Technical Development, Device & Packaging Development at Genentech, A Member of the Roche Group. She has +25 years of experience and a broad range of expertise in R&D, cGMP, and commercialization for biologics, drugs, and combination products development including drug formulation, drug delivery system, process engineering, and sterilization development in the Biopharmaceutical and Pharmaceutical industries.

Philip J. (P.J.) Brooks is the acting director of NCATS’ Division of Rare Diseases Research Innovation. Brooks represents NCATS in the NIH-wide Gene Therapy Working Group, the Regenerative Medicine Innovation Project and the International Rare Diseases Research Consortium (IRDiRC). He also is the working group co-coordinator for the NIH Common Fund program on Somatic Cell Genome Editing, one of the leaders of the Platform Vector Gene Therapy (PaVe-GT) pilot project and the co-chair of the Bespoke Gene Therapy Consortium. In May 2022, Brooks was selected as the recipient of the 2022 Sonia Skarlatos Public Service Award by the American Society of Gene & Cell Therapy for consistently fostering and enhancing the field of gene and cell therapy. Brooks received his doctorate in neurobiology from The University of North Carolina at Chapel Hill. After completing a postdoctoral fellowship at The Rockefeller University, he became an investigator in the NIH intramural program, where he developed an internationally recognized research program focused on two distinct areas: the molecular basis of alcohol-related cancer, and rare neurologic diseases resulting from defective DNA repair, including xeroderma pigmentosum, Cockayne syndrome and Fanconi anemia.

Diane Carlisle PhD is an Associate Professor in the University of Pittsburgh Department of Neurological Surgery. Her research program focuses identifying new therapeutic targets and developing pharmacologic therapies in neurodegenerative diseases, including Huntington’s Disease, ALS, and age-related dementia. Her research program incorporates focus ultrasound to open the blood brain barrier to improve brain distribution of potential therapeutics.

Bradford Casey, Ph.D., Senior Associate Director in the Fox Foundation’s Discovery and Translational Research division, leads the Foundation’s Genomics, Computational Biology, and Data Science research portfolios. He collaborates with academic, industry, and clinical researchers to manage and develop MJFF’s global research portfolio and ensure that MJFF research priorities and resources reflect and best serve the ultimate needs of patients. He routinely engages with key experts across disciplines to identify and develop new approaches to address gaps in the field, and to establish strategic partnerships. In addition to academic research, Bradford has diverse experience working with researchers, legislators, and government agencies on initiatives to advocate for and support neuroscience research.

Bradford is passionate about leveraging emerging technologies to improve biomedical research, and achieving the Foundation’s mission to understand and treat Parkinson’s disease.

 Dr. Kuldip Dave is Senior Vice President of Research and joined The ALS Association in May 2019. In this role, he leads the world’s largest philanthropic research funding program for ALS. He develops and implements the broader research vision by setting annual priorities and department goals including research funding strategy, research programs, and strategic initiatives and is also responsible for the operational, staffing, and budgetary oversight of the research program. Dr. Dave received his bachelor’s degree in biology from Rutgers University. He received his Ph.D. degree in Pharmacology and Physiology from Drexel University College of Medicine in Philadelphia. He went on to work for pharmaceutical industry for 5 years before joining The Michael J. Fox Foundation for Parkinson’s research. After nine years at MJFF, he joined the ALS Association as the head of the Research program.

Yves De Koninck, PhD, FCAHS, FRSC is Professor of Psychiatry & Neuroscience at Université Laval, Scientific Director of the CERVO Brain Research Centre and Director of Research of the Quebec Integrated University Health and Social Services Centre. Former President of the Canadian Association for Neuroscience, he founded the Quebec Pain Research Network. He is a fellow of the Canadian Academy of Health Sciences and of the Royal Society of Canada. He holds a Canada Research Chair in Chronic Pain and Related Brain Disorders. His main areas of research include chronic pain, drug dependence, cognitive ageing, and neurodegenerative disorders. He founded the Neurophotonics Centre (neurophotonics.ca), bringing together physicists, engineers, mathematicians, and biologists to stimulate technology development to probe the brain. He now leads Sentinel North a global initiative to harness the power of light for the benefit of health, environment, and sustainable development in the North. He currently leads the Canadian Brain Research Strategy. Accolades include the Distinguished Career Award from the Canadian Pain Society, the Emily Gray Award from the Biophysical Society and the Award for Education in Neuroscience from the Society for Neuroscience.

Anna Debski is a fourth year PhD student in the Price Lab at the University of Virginia. Her research focuses on developing non-viral gene delivery platforms for glioblastoma multiforme (GBM). She is also investigating effects of CRISPR-mediated P-glycoprotein knockdown for enhanced therapeutic delivery in GBM. Previously, she received a bachelor of science degree in Materials Science and Engineering at the Ohio State University.

Ben Deverman is the Senior Director of Vector Engineering and an Institute Scientist at the Broad Institute of MIT and Harvard. Ben’s laboratory in the Stanley Center for Psychiatric Research at the Broad Institute develops and applies novel high-throughput screens and data-driven protein and genome engineering techniques to create more efficient and targeted AAV vectors to advance gene therapy and support scientific discovery. In prior work at the California Institute of Technology, Ben and colleagues identified numerous capsids, including AAV-PHP.B and an enhanced variant, AAV-PHP.eB, that made efficient brain-wide gene delivery possible for the first time.

Brad Elmer, PhD is a Lab Head and Principal Scientist in the Genomic Medicine Unit at Sanofi. He trained as a neuroscientist studying mechanisms of synapse formation and brain development at the University of California, Davis and at Cold Spring Harbor Labs. He has spent the last 9 years at Sanofi, with a focus on developing gene therapies for the treatment of neurodegenerative diseases.

Esteban is Director of Neuroscience Platform Technology Research at Spark Therapeutics in Philadelphia. Esteban has over 40 publications in peer-reviewed journals and patent applications dealing with viral-based gene therapies. Prior to joining Spark Therapeutics, he was a Principal Investigator and Group Leader at the Princeton University Neuroscience Institute, developing neurotropic viral vectors for gene transfer, and to diseect mamalian brain connectivity and function. He was the co-Director of the NIH-funded Center for Neuroanatomy with Neurotropic Viruses (CNNV). Esteban has trained over 20 undergraduate students, graduate students and postdoctoral fellows and have obtained private, State, and Federal research grants.

Dr. Mario Fabiilli is an Associate Professor of Radiology and Biomedical Engineering at the University of Michigan. His lab develops ultrasound-based therapies with a focus on biomaterials for drug delivery and tissue regeneration, including strategies for blood vessel and bone growth.

Dr. Ferrara is Professor of Radiology and Division Chief of the Molecular Imaging Program at Stanford University. She previously served as the founding chair of the Department of Biomedical Engineering at UC Davis. Dr. Ferrara is a member of the National Academy of Engineering and a fellow of the American Association for the Advancement of Science, Institute of Electrical and Electronic Engineers, Biomedical Engineering Society, Acoustical Society of America, World Molecular Imaging Society and American Institute of Medical and Biological Engineering. Her publications include 300 manuscripts in the area of image-guided drug and gene delivery.

Delaney Fisher is an American Heart Association Pre-Doctoral Fellow pursuing her Ph.D. in the Department of Biomedical Engineering at the University of Virginia. Her research in the Price Lab centers on leveraging the non-invasive, therapeutic technology of focused ultrasound to overcome challenges in treating disorders of the brain. Specifically, her dissertation work bridges the fields of biomedical engineering, neuroscience, and radiology to develop MRI-guided focused ultrasound platforms for the treatment of cerebral cavernous malformation, a disease that causes hemorrhagic, vascular growths in the brain. Previously, Delaney received her B.S. in Chemical and Biomolecular Engineering from the University of Tennessee.

Jessica Foley, PhD is the Chief Scientific Officer and a Managing Director for the Focused Ultrasound Foundation, whose mission is to accelerate the development and adoption of focused ultrasound, a breakthrough noninvasive therapeutic medical technology. Dr. Foley guides strategy, development, and implementation of scientific and research programs for the Foundation, and aligns these with business operations, communications, and development activities. She leads alliance-building efforts with external stakeholders including governmental policymakers, regulatory agencies, and disease-specific foundations whose interests are complementary to those of the Foundation. She is a spokesperson and advocate for the Foundation and the focused ultrasound community among a wide variety of stakeholders. Prior to the Foundation she spent several years in the medical device industry (Insightec, Medtronic) and was a AAAS Science and Technology Policy Fellow at the National Science Foundation. She holds a BSE in Biomedical Engineering from Duke University and a PhD in Bioengineering from the University of Washington.

Dr. Robert M. Friedlander is Chair of the Department of Neurological Surgery and Walter E. Dandy Distinguished Professor at the University of Pittsburgh School of Medicine. Clinically, Dr. Friedlander specializes on the management complex cerebrovascular lesion, brain tumors and Chiari Malformations. Scientifically, he was first to slow Huntington’s disease and ALS in mice and has focused his career on delineating and manipulating caspase and mitochondrial cell death pathways. Dr. Friedlander is an elected member of the National Academy of Medicine, ASCI and AAP and prior member of the NINDS Advisory Council. He graduated with a BA and MA from Brandeis University, MD from Harvard Medical School and completed Neurosurgery residency at MGH. Prior to being recruited to Pittsburgh, Dr. Friedlander was Vice Chair of Neurosurgery at the Brigham and Women’s Hospital and Professor at Harvard Medical School.

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Senior research scientist at the University of Virginia studying targeted gene delivery to the endothelium with focused ultrasound.

Dr. Yutong Guo is a postdoctoral scholar at Stanford University. Her research is focused on ultrasound-actuated microbubble dynamics and the induced immuno-mechano-biological effects, with the overarching goal of developing effective targeted cell and gene therapy against brain diseases. Her research approach involves the integration of computational modeling that supports the rational design of focused ultrasound-based treatment and quantitative imaging.

Justin Hanes is the Lewis J Ort Professor at the Johns Hopkins School of Medicine, where he directs the Center for Nanomedicine. He is a member of the National Academy of Medicine and the National Academy of Inventors. His research is focused on the development of new methods for improved drug and gene delivery to various locations in the body. He has collaborated with Prof. Rich Price of UVA for nearly 20 years on projects related primarily to the use of MRgFUS / microbubbles to enhance tissue-specific delivery of unique non-viral gene delivery systems, especially to the brain.

Dr. Guosong Hong received his Ph.D. in chemistry from Stanford University and completed postdoctoral training at Harvard University. Dr Hong joined Stanford Materials Science and Engineering and Wu Tsai Neurosciences Institute as an assistant professor in September 2018. His research at Stanford aims to develop novel optical and electronic materials for minimally invasive brain interfacing. He is a recipient of the NIH Pathway to Independence Award, the MIT Technology Review ‘35 Innovators Under 35’ Award, the Science PINS Prize for Neuromodulation, the NSF CAREER Award, the Walter J. Gores Award for Excellence in Teaching, and the Rita Allen Foundation Scholars Award.

I am a postdoc from Dr. Szablowski’s lab at Rice University. More recently, we have developed a novel therapeutic paradigm called Regionally Activated Interstitial Drugs (RAID), enabling noninvasive and non-genetic neuromodulation and therapy of specific brain regions (Huang, et al. bioRxiv 2023.10.12.562069). My long-term research goal is to develop technologies for the noninvasive control and monitoring of endogenous signaling pathways in intact brain cells and tissues, with the aim of advancing brain treatment and neuroscience discovery.

Victor Jeannot was born in France in 1983. He holds a Pharm.D and a Ph.D. in biotechnology (University Grenoble Alpes – France) and is specialized in the development of theranostic solutions. He joined Bracco Suisse SA in 2016 (Geneva, Switzerland) to manage the therapeutic ultrasound Lab. He led projects using gas microbubble for therapeutic application including gene delivery. In 2023 he became R&D Program Manager of Bracco Suisse managing a portfolio of innovation projects (Imaging – Therapy and Software development).

Robin Ji is an associate research scientist at Columbia University working on focused ultrasound mediated blood brain barrier opening for the delivery of biologics, including the delivery of AAVs for gene editing applications.

Yun Jing received his B.S. degree in acoustics from Nanjing University, China, in 2006 and his Ph.D. degree from Rensselaer Polytechnic Institute in 2009 with a focus on architectural acoustics. He is currently a professor in the Graduate Program in Acoustics at Penn State University. Prior to joining Penn State University in 2020, he was a research fellow at Brigham and Women’s Hospital during 2009-2011, and an assistant professor and associate professor at North Carolina State University during 2011-2019. He is broadly interested in acoustic functional materials, biomedical ultrasound, nonlinear acoustics, and noise control. He has published over 100 peer-reviewed scientific manuscripts. He is a fellow of the Acoustical Society of America and a senior member of IEEE. He has received numerous awards such as the 2018 R. Bruce Lindsay Award from the Acoustical Society of America, the 2018 IEEE Ultrasonics Early Career Investigator award, 2018 MIT Technology Review Innovator under 35 China award, and the IEEE UFFC Star Ambassador Lectureship Award

Rikke H Kofoed is post doc at Aarhus University and Aarhus University Hospital, Denmark. She has a biochemical background in neurodegenerative diseases and for the past 5 years (while mainly being at Sunnybrook Research Institute, Toronto, Canada) her research has focused on establishing and fine-tuning gene delivery strategies to the brain using focused ultrasound and microbubbles.

Elisa E. Konofagou is the Robert and Margaret Hariri Professor of
Biomedical Engineering and Professor of Radiology as well as Director of
the Ultrasound and Elasticity Imaging Laboratory at Columbia University in
New York City. Her main interests are in the development of novel elasticity
imaging techniques and therapeutic ultrasound methods and more
notably focused ultrasound in the brain for drug delivery and modulation,
breast tumor ablation monitoring with harmonic motion imaging,
myocardial elastography, electromechanical wave imaging, pulse wave
imaging with several clinical collaborations in the Columbia Presbyterian
Medical Center and beyond.

Jeffrey Kordower is the founding director of the ASU-Banner Neurodegenerative Disease Research Center and endowed chair as The Charlene and J. Orin Edson Distinguished Director at the Biodesign Institute at ASU. He has been a pioneer in the field of neural transplantation techniques and his pathbreaking investigations into the underpinnings of neurodegenerative disease have made him a leader in the field. Kordower’s interests include the study of gene and stem cell therapies, disease pathogenesis including the morphological and molecular changes during the course of neurodegeneration, learning and memory, and aging. He has also been a pioneer in the field of neural transplantation techniques. He comes to ASU from the Rush University Medical Center in Chicago, where he was faculty member for more than 30 years.

Richard Li is a senior PhD candidate in Biology at Caltech supervised by Professor Mikhail Shapiro. HIs research centers upon noninvasive surveillance and perturbation of neural circuits to study brain functions and to treat disorders in central nervous system. Li is currently focusing on engineering viral vectors for acoustically targeted gene transfer to achieve noninvasive, site-specific, cell type-specific, ultrasound-mediated genetic material delivery to the brain in various animal models. His long-term research goal is to develop primary/translational technologies for nonintrusive, real-time in vivo gene circuit monitoring and interfering in the brain through integration of biomolecular ultrasound, synthetic biology, tissue engineering, and vector engineering.

Shibi Likhite, MD is a Research Scientist at the Nationwide Children’s Hospital in Columbus, Ohio with over 14 years of experience in the fields of gene and cell therapy, as well as in vitro disease modeling. His work is focused on the development of novel tools for the translation of gene and cell therapy to human clinical trials, for treatment of both pediatric and adult-onset fatal diseases in the areas of neuromuscular, neurodegenerative, neurological, metabolic disorders and cancer. So far, Dr. LIkhite has been able to translate 5 gene therapy programs to the clinic and one program (Zolgensma® – onasemnogene abeparvovec) to the market with multiple other gene and cell therapy treatments in the pipeline. I have extensive experience in viral vector development and delivery with comprehensive knowledge of vector biology, rational design, vector manufacturing, and analytics required for efficacy and safety studies that are required for translation to the clinic. He has worked with multiple collaborators and industry partners on the key aspects of gene therapy such as vector development, manufacturing and vector delivery, notably AveXis-Novartis, Alcyone Therapeutics etc. Dr. Likhite also has expertise in utilizing CRISPR/Cas gene editing technology for development of off-the-shelf Chimeric Antigen Receptor expressing Natural Killer or T cell therapy (CAR-NK/CAR-T). Besides the gene and cell therapy work, he also has extensive experience in the cell-based model systems for studying the disease mechanisms and high-throughput screening of potential therapeutics.

Dr. Lockhart is an accomplished biotech executive with more than 25 years of experience across all phases of drug discovery, drug development and technology development. Prior to becoming president and CSO of ReCode Therapeutics in January 2022, he served as CEO and president of the company since March 2020. Prior to serving as CEO and president of ReCode, he was CEO and president of its predecessor, TranscripTx from 2014 – 2020. Prior to TranscripTx, he was the Chief Scientific Officer at Amicus Therapeutics from 2006 through 2013. He led a team at Amicus that brought multiple rare disease programs into clinical trials. Previously, he co-founded Ambit Biosciences, serving as Chief Scientific Officer and president for 5 years, during which the company developed a novel kinase profiling technology and new kinase inhibitors for cancer and other diseases. Prior to Ambit, Dr. Lockhart served as Director of Genomics at the Genomics Institute of the Novartis Research Foundation (GNF). Dr. Lockhart began his biotech career at Affymetrix, where he was the primary inventor and developer of gene expression profiling with DNA microarrays. Dr. Lockhart received his Ph.D. in Chemistry from Stanford University and was a postdoctoral fellow at the Whitehead Institute in the Biology Department at MIT. He has published more than 80 peer-reviewed scientific articles on pharmacological chaperones, mutation-specific selection of patients for clinical trials, clinical studies with pharmacological chaperones, genomics, kinase technology, kinase inhibitor discovery, and the use of genomic tools to address important biological and biomedical problems. He is also the inventor on more than 40 issued U.S. patents.

Dr. Lung holds MSc and PhD degrees in Biomechanics from the University of Wisconsin-Madison and National Yang-Ming University, respectively. He has over 25 years of medical device product design, development, marketing, and selling experience, which has translated into 24 product launches during his career at United Orthopedic and Medtronic. He also has extensive entrepreneurship experience in the healthcare industry.

Dr. McDannold is a Physicist in Radiology Department at Brigham and Women’s Hospital and the Director of the Focused Ultrasound Laboratory. His work has been primarily concerned with the development and implementation of targeted drug delivery, ablation, and other focused ultrasound therapies, and methods to guide and monitor these therapies. He has also worked on clinical implementation of focused ultrasound.

Kathrin C. Meyer, PhD, studied Cellular and Biomedical Sciences at the Institute of Cell Biology in Berne, Switzerland. Her post-doctoral research was performed in Brian Kaspars laboratory at the Center for Gene Therapy in Columbus Ohio. During that time, Dr. Meyer established a new and fast reprogramming method for in vitro modeling of neurodegenerative diseases using patient skin cells. Moreover, Dr. Meyer developed intrathecal gene therapy programs for several neurodegenerative diseases including Spinal Muscular Atrophy and Batten Disease. Multiple clinical trials that are based on this work are currently ongoing at Nationwide Children’s Hospital. In 2017, Dr. Meyer became a Principal Investigator at Nationwide Children’s Hospital. She is also an Assistant Professor in the Department of Pediatrics at The Ohio State University, Columbus, Ohio.

Following his studies in biophysics at Wageningen University, Chrit Moonen did a postdoctoral period at the University of Oxford (Sir Georg Radda). He joined UC Davis as a Visiting Research Scientist before becoming head of the NIH In Vivo NMR Research Center from 1987-1996. He was director of the laboratory “Molecular and Functional Imaging: from Physiology to Therapy” in Bordeaux until 2011. He was professor at the Division of Imaging at the University Medical Center in Utrecht, the Netherlands from 2011-2022. He recently joined the FUS Foundation as Scientific Programs Director.

Bernie obtained her PhD in Neuroscience from the UCL Institute of Prion Diseases at University College London. During her PhD she developed mouse models of a rare inherited form of prion disease to investigate the molecular mechanisms underlying the pathogenesis of this disease. She then went to do a post-doc at the UCL Institute for Women’s Health. There, she was involved in developing and testing novel gene therapies using different genetic models of paediatric epilepsy. She is currently a postdoctoral research fellow in the Focused Ultrasound Lab at Brigham and Women’s hospital. Her current work is centred on using focused ultrasound and microbubbles for AAV delivery in preclinical models of Huntington’s disease.

Frederic Padilla, PhD, is the Director of the Gene and Cell Therapy Program at the FUS Foundation. He joined the FUS Foundation in 2017 as a Merkin Fellow. Simultaneously, Frederic oversees the Foundation’s initiatives in applied physics and preclinical research, with a specific focus on advancing the potential of focused ultrasound to enhance immunotherapies for breast and pancreatic cancers. Before joining the Foundation, Frederic served as an Associate Professor at the French CNRS. He holds a Doctorate in Applied Physics from the University of Paris and an engineering degree from Centrale-Supelec. Frederic’s academic background includes a Fulbright Fellowship at Boston University and a Visiting Professorship at the University of Michigan.

Dr. Marie-Eve Paquet has developed and is Head of the CERVO Brain Research Centre Vectorology platform within the Canadian Optogenetics and Vectorology Foundry. She is an adjunct professor in the department of biochemistry, microbiology and bioinformatics at the faculty of science and engineering at Université Laval in Quebec City Canada. In this role she oversees the strategic direction and operation of the largest viral vector facility in Canada. She also runs a research lab with interests in micro biome, molecular tools, optogenetics and optimisation of viral vector based gene transfer approaches. She has secured millions in external funding for research and research Infrastructure. Her group has strong collaborative ties with various tools developers and industries and distributes more than 1000 vectors yearly to over 250 research groups across the world.

Dr. Ramasamy Paulmurugan is a Professor in Radiology at Stanford University School of Medicine. He received his Masters in Biomedical Genetics and PhD in Molecular Virology from the University of Madras, India. After serving as a scientist for four years, he joined the School of Medicine at University of California, Los Angeles (UCLA) as a visiting scientist. In 2003, he moved to Stanford as a Senior Research Scientist. Since 2009, he has been a faculty in the Department of Radiology at Stanford University under the Molecular Imaging Program (MIPS) and Canary Center for Cancer Early Detection. He is a pioneer in developing split-reporter protein complementation systems for different reporter genes (luciferases, fluorescent proteins, and thymidine kinase), and has been using them for imaging cellular protein-protein interactions in living animals. Currently, his lab (Cellular Pathway Imaging Laboratory (CPIL)) is working on the application of different imaging strategies to evaluate the therapeutic role of ER, another important estrogen receptor, which is recently identified as one of the major players in estrogen biology. Other applications where his lab currently applying these assays include studying protein-protein interactions involved in estrogen receptor signaling, Nrf2-mediated antioxidant signaling in chemoresistance, p53-sumoylation mediated chemotherapy responses in cancer, NFkB mediated cytokine signaling in cancer, and signaling mechanisms associated with APP and Tau protein sumoylations in Alzheimer’s disease. In cancer therapy, his lab established microRNA-based reprogramming approaches to sensitizing drug-resistant cancers. In synthetic biology, his lab recently invented the application of a high-pressure microfluidic system in the reconstruction of biomolecules derived from cells (proteins and lipids) along with synthetic sources (phospholipids, polymers, and surfactants) to develop self-assembled nano- and micro-structures that mimic biological membranes for drug and microRNA delivery applications. As part of this process, his group developed biomimetic microbubbles (biMBs) and nanobubbles (biNBs) using tumor cell derived exosomes (TDEs) for cancer immunotherapy and imaging.

José Pineda is a dedicated researcher with a diverse background in engineering and biomedical sciences. He holds a degree in Telecommunications and Robotics Engineering and earned his Ph.D. in Biomedical Engineering from the Universidad Politécnica de Madrid. José’s research career has taken him through a remarkable journey across various institutions, including renowned organizations like the Fraunhofer Institute, universities, and more recently, hospital institutions. Currently, as a full-time researcher at hmCINAC, José Pineda is actively involved in two prominent research avenues. The first focuses on the development of image-based biomarkers for the early diagnosis of Parkinson’s disease, a critical area with significant implications for patient care and treatment. His work in this field seeks to provide innovative solutions for the early detection and management of this condition. The second major area of José’s research revolves around the development of therapeutic strategies for Parkinson’s disease patients using Focused Ultrasound. These strategies encompass a range of approaches, including thermoablation, blood-brain barrier disruption and drug-delivery, and neuromodulation.

Richard J. Price, PhD, is the Nancy and Neal Wade Endowed Chair Professor of Biomedical Engineering and Professor of Radiology & Medical Imaging at the University of Virginia. He is also Co-Director of the UVa Focused Ultrasound Cancer Immunotherapy Center. He has been studying ultrasound-targeted drug and gene delivery for ~20 years. He was amongst the first investigators to observe that interactions between contrast agent microbubbles and ultrasound can be used to open endothelial barriers for targeted nanoparticle delivery, with emphasis on delivery across the blood-brain barrier. Recent studies entail using this approach to treat infiltrative gliomas and brain metastases, as well as for reversing neurodegeneration in Parkinson’s Disease. Dr. Price is also studying how focused ultrasound can synergize with various immunotherapeutic approaches, with his research in this area supporting clinical trials at UVA combining focused ultrasound with chemo- and immunotherapy for multiple cancer indications (NCT04796220, NCT03237572 and NCT04116320). Dr. Price has received the Andrew J. Lockhart Memorial Award for Focused Ultrasound Cancer Research, the Jorge Heller Award for Outstanding Original Research Paper in the Journal of Controlled Release, and the Gerritsen Award for most highly cited review article from the Microcirculatory Society. He is a Fellow of the American Institute of Medical and Biological Engineering and the Controlled Release Society. He has served on the Executive Council of the Microcirculatory Society and the ISTU Board of Directors.

Dr. Ramsburg is an immunologist with many years experience in the development of vaccines and genetic medicines. Dr. Ramsburg began her academic career at Duke University where she was a member of the Duke Human Vaccine Institute and the Duke Virology Center. While at Duke Liz led a group aiming to develop vaccines and other countermeasures for emerging infections and HIV. In 2013 Liz joined Janssen Pharmaceuticals in the Netherlands, where she supported efforts to develop vaccines and gene therapies for neurodegenerative diseases. Liz joined Spark Therapeutics in 2021 as Head of Neuroscience Research where she manages all preclinical research within the Neuroscience therapeutic area.