Nilufer Ertekin-Taner, M.D., Ph.D., is a neurogeneticist and behavioral neurologist at Mayo Clinic Hospital in Jacksonville, Florida. Her laboratory aims to discover and characterize genetic factors underlying the complex genetics of Alzheimer's disease (AD) and related neurodegenerative conditions.

Himakshi Jhala, M.P.H., M.B.A., FACHE, is an Administrator at Mayo Clinic and works on driving innovative strategic initiatives focusing on discovery research, impactful translation, and synergistic growth areas that meet the unmet needs of the patients across the Mayo Clinic enterprise. She is board certified in healthcare management and is recognized as a Fellow of the American College of Healthcare Executives. 

Julie Allickson, Ph.D., is the Chief Technology Officer at Mayo Clinic’s Center for Regenerative Biotherapeutics and also directs Biomanufacturing and Process Development. She's a consultant for Laboratory Medicine and Pathology and an associate professor of regenerative medicine. Dr. Allickson focuses on product development for the center in line with Mayo Clinic's 2030 vision. With decades of experience, she specializes in Cell and Gene Therapies, Tissue Engineering, and 3D Bioprinting, aiming to position Mayo Clinic as a leader in regenerative medicine. She leads biomanufacturing strategy, new therapy introductions, and external relationships. With over 25 years in cellular therapy and regenerative medicine, Dr. Allickson has expertise in business management, strategic planning, and project management. She has worked in both industry and academia and was an executive officer at a cell banking company.

Yan Asmann, Ph.D. has a broad background and expertise in biochemistry, molecular biology, computational biology, and data science. As a postdoctoral fellow at the Mayo Clinic, I developed a binary indexing sequence alignment algorithm for electronically profiling gene expressions in the EST database. In my role as a computational biology faculty member at Mayo, I have developed and published multiple tools and algorithms for the analysis of omics data, including: (1) a method for analyzing 3' tag digital gene sequencing data; (2) an algorithm for detecting fusion transcripts in RNA-Seq data; (3) an analytic pipeline for Exome-Seq data analysis; (4) an algorithm for identifying copy number variations in exome data; (5) a boosting model for prioritizing and calling variants in RNA-Seq data; (6) an integrated workflow and novel algorithms for cancer neoantigen discovery and prioritization in tumor sequencing data; (7) analytic methods to address disparities in omics data among individuals of different ancestries; (8) optimization of machine learning and artificial intelligence models, along with model interpretation for omics data; and (9) a deep neural network model to identify biomarkers for overall survival in cancer patients using transcriptome data. I have worked in cancer research and collaborated with both clinicians and basic scientists for over 15 years. I serve as the co-leader of the Mayo Clinic Neoantigen Vaccine Therapy Program and as the co-PI for multiple Investigational New Drug (IND) phase I and II trials involving immune checkpoint inhibitors and neoantigen combination therapies. Additionally, I am the MPI of an NCI U24 grant, co-leading the Cancer Adoptive Cell Therapy (Can-ACT) Network Coordinating Center. I am also a co-director of the Bioinformatics and Biostatistics Core of the Mayo Clinic Multiple Myeloma SPORE, and lead the Cancer Novel Biotherapeutics Data Science team at the Mayo Clinic Comprehensive Cancer Center.

Wenchun Qu, MD, PhD, is a Professor of Anesthesiology and Physical Medicine and Rehabilitation (PM&R) at Mayo Clinic Alix School of Medicine. He earned his MD from the Second Military Medical University and his MS and PhD from the University of Southern California. Dr. Qu serves as the Jorge and Leslie Bacardi Associate Director of the Mayo Clinic Center for Regenerative Biotherapeutics in Florida. He is also a consultant and Vice Chair of Research and Innovation in the Department of Pain Medicine. His discovery research focuses on RNA and extracellular vesicle engineering platforms for targeted delivery of biotherapeutics for inflammatory processes and cancer. His translational research includes clinical trials of cell- and RNA-based therapeutics for degenerative disc disease and spinal cord injury. Dr. Qu has published more than 80 high-impact manuscripts and holds leadership roles in national academic organizations.

Margot Cousin, Ph.D. is an Assistant Professor of Medical Genetics in the Mayo Clinic College of Medicine and Science in the departments of Neurology and Molecular Medicine. As Director of Mayo Clinic’s N of 1 Therapeutics Program in the Center for Individualized Medicine, she leads an end to end pathway that moves eligible rare disease patients from genetic diagnosis to targeted treatment. She is focusing on individualized antisense oligonucleotide (ASO) medicines that are custom therapies designed for a single patient and delivered through N-of-1 clinical trials, but expanding to other modalities. Her team couples patient derived models with automated pipelines that identify ASO amenable variants in a scalable translational infrastructure. Dr. Cousin serves on Mayo’s RNA Discovery & Therapeutics Implementation team, participates in the N=1 Collaborative global consortium, and leverages external partnerships to streamline and advance the care of individuals suffering from rare disease.

Dev Mukhopadhyay, Ph.D

Marion Curtis, Ph.D  is an Associate Professor at Mayo Clinic in Phoenix, Arizona, specializing in cancer immunology and ovarian cancer. Her translational research examines how the tumor microenvironment, immune metabolism, and DNA damage response shape anti-tumor immunity, while leveraging immunopeptidomics to discover new tumor antigens and therapeutic targets. Since joining Mayo Clinic in 2018, she has received numerous honors, including an NCI R37 MERIT Award, and published in leading scientific journals. Her research is increasingly focused on translating laboratory discoveries into new immunotherapies for ovarian cancer, including a Phase 1 clinical trial of autologous MUC1-activated T cells and the development of a first-in-human personalized vaccine targeting cryptic antigens derived from the cancer dark proteome.

Matthew T. Auton, Ph.D.

Wenqian Hu, Ph.D

Xue Wang, Ph.D., is a bioinformatician and an assistant professor in the Department of Quantitative Health Sciences at Mayo Clinic. She is originally from China and spent a few years in Atlanta, Georgia and San Diego, California before joining Mayo Clinic in Florida. Her passions include developing computational algorithms and applying existing bioinformatic tools for RNA and drug discovery. As a collaborative scientist, she is interested in working with bioinformaticians, biologists, and physicians to facilitate the translation from basic science to clinical potential.

Jane Zhu, M.D., Ph.D., is an Associate Professor of Biochemistry and Molecular Biology. She earned her MD in Pediatrics from Jilin University and her PhD in Developmental Biology from the National University of Singapore. Dr. Zhu's clinical and research interests center on developing more effective therapies for childhood solid tumors, particularly neuroblastoma and osteosarcoma. Her laboratory uses zebrafish models to investigate the molecular and cellular mechanisms underlying neuroblastoma pathogenesis, identify novel circulating prognostic biomarkers, and develop innovative therapeutic strategies, including personalized targeted therapies and monocyte/macrophage-mediated immunotherapies. She is also deeply committed to education and mentorship, with a particular interest in training and inspiring the next generation of researchers and physician-scientists in pediatric oncology.

Arthur (Art) Krieg, MD is an Adjunct Professor at the UMass Chan Medical School RNA Therapeutics Institute. He received his MD from Washington University in 1983 and earned a BS in Biology from Haverford College. Art currently serves on the scientific advisory boards of several companies developing oligonucleotide therapeutics. He founded Zola Therapeutics in 2023 and serves as CEO, with the goal of developing a new generation of TLR7/8/9 oligonucleotide agonists for immunotherapy of cancer and infectious diseases, as well as small molecule antagonists for the treatment of SLE and other autoimmune diseases. Previously, Art founded Checkmate Pharmaceuticals, which was acquired by Regeneron in 2022. Prior to that, he served as Chief Scientific Officer at Sarepta (2008-2014), co-founder and CEO of RaNA Therapeutics (2011-2013), Chief Scientific Officer of Pfizer’s Oligonucleotide Therapeutics Unit (2008-2011), and co-founder and CSO of Coley Pharmaceutical Group (1997-2008). He was also Professor of Medicine in the Division of Rheumatology at the University of Iowa College of Medicine (1991-2001). Art co-founded the journal Nucleic Acid Therapeutics, serving as editor for 16 years, and helped establish the Oligonucleotide Therapeutics Society, for which he recently served as President. He has authored more than 250 scientific publications and is an inventor on more than 50 issued U.S. patents covering oligonucleotide technologies.

Susan Catalano, Ph.D., is a biopharmaceutical executive and board director with 25 years of experience discovering, developing, operationalizing, and funding novel small molecule and gene therapy modalities. She has scaled companies from concept to IPO, raised $200M+ in private investor, venture capital, foundation, and NIH funding over her career, and served as the lead scientist on pioneering clinical therapeutics to treat neurological disorders and cancer. Her career spans business strategy and drug development roles across startup (Cognition Therapeutics, which IPO’d in 2021, Acumen Pharmaceuticals and CODA Biotherapeutics), mid-sized (Rigel Pharmaceuticals and Capsida Biotherapeutics) and large (Roche) pharmaceutical companies. She is a trusted advisor to seed-round, VC-backed, and early clinical-stage companies. Susan has been recognized for entrepreneurship by a Big Four professional services firm, a world-renowned STEM education center, and a top regional venture capital financing entity. Additionally, she serves on five NIH advisory and editorial boards. Additionally, she serves on five NIH advisory and editorial boards. Susan holds a Ph.D. in Anatomy and Neurobiology from the University of California, Irvine, and a B.A. in Biology and English Literature from Barnard College, Columbia University. She completed postdoctoral work in molecular and cell biology at the California Institute of Technology and the University of California, Berkeley.

Vikram Agarwal, PhD, is Senior Distinguished Scientist and Head of mRNA Platform Design Data Science at Sanofi. He earned his PhD from the Massachusetts Institute of Technology (MIT) and his BS in Biology from The University of Texas at Austin. Dr. Agarwal completed his doctoral training in Dr. David Bartel's laboratory at MIT and his postdoctoral fellowship in Dr. Jay Shendure's laboratory at the University of Washington. He currently leads an international computational team at Sanofi's mRNA Center of Excellence, where he applies artificial intelligence and data science approaches to design and optimize next-generation mRNA therapeutics. His work focuses on leveraging computational biology and machine learning to enhance the development of innovative RNA-based medicines.

Eric Wickstrom, PhD, is Co-Founder and Chief Scientific Officer of Bound Therapeutics LLC. He earned a BS with Honor in Biology from the California Institute of Technology and a PhD in Chemistry from the University of California, Berkeley. Dr. Wickstrom's scientific career has spanned more than five decades and has focused on the molecular mechanisms of RNA and DNA and their applications in cancer therapeutics. As a student at Caltech, he investigated mitochondrial DNA topology, and during his doctoral studies at UC Berkeley, he examined AUG selection as the initiation codon in mRNA. His subsequent research at the University of Colorado and the University of Denver advanced the understanding of transfer RNA structure dynamics and the role of initiation factor 3 in mRNA translation. At Southern Biotech, Dr. Wickstrom pursued recombinant DNA approaches for interferon-γ expression and DNA synthesis technologies. During his tenure at the University of South Florida, his laboratory demonstrated that short synthetic DNA strands could suppress cancer gene mRNAs in cells and animal models. At Thomas Jefferson University, where he served from 1992 to 2023, his laboratory developed radiolabeled bispecific PNA-peptide analogs for imaging cancer gene mRNA activity in tumors and pioneered bispecific RNA-peptide analogs for cancer therapy. In 2023, he co-founded Bound Therapeutics LLC, where he currently serves as Chief Scientific Officer, leading the development of innovative RNA-based therapeutics for cancer treatment.

Craig L. Duvall, PhD, is the Cornelius Vanderbilt Professor of Biomedical Engineering at Vanderbilt University. He earned his PhD in Biomedical Engineering from Georgia Tech and Emory University and completed postdoctoral training at the University of Washington. Dr. Duvall founded the Duvall Advanced Therapeutics Laboratory (ATL) at Vanderbilt in 2010 and has since established an internationally recognized research program focused on the molecular engineering of peptides, RNAs, and biomaterials for therapeutic applications. He was promoted to Associate Professor in 2016, Professor in 2019, and named Cornelius Vanderbilt Professor in 2019. His laboratory specializes in engineering peptides and RNA therapeutics for pharmacokinetic modulation and disease targeting, as well as developing antioxidant polymeric biomaterials for sustained drug delivery and tissue repair. His work on siRNA therapeutics focuses on conjugate structural optimization for applications including triple-negative breast cancer, osteoarthritis, and diseases of the central nervous system. Dr. Duvall is the recipient of numerous prestigious honors, including the Presidential Early Career Award for Scientists and Engineers (PECASE), the National Science Foundation CAREER Award, the Society for Biomaterials Young Investigator Award, the Controlled Release Society Gene Delivery and Gene Editing Focus Group Young Investigator Award, and the Cellular and Molecular Bioengineering Young Innovator Award. He is a Fellow of both the American Institute for Medical and Biological Engineering (AIMBE) and the Biomedical Engineering Society (BMES) and has served as a standing member of the National Institutes of Health Gene and Drug Delivery Study Section.

Deborah ("Deb") Day Barbara, MBA, is a distinguished life sciences executive with more than 30 years of experience spanning therapeutics, diagnostics, research tools, and enabling technologies. She earned an MBA in Management from Loyola College Maryland and a BA in Biochemistry and Molecular Biology from the University of Maryland. Deb currently serves as Chief Business Officer of Eclipse Bio, where she leads business development and strategic partnerships. She also serves as Chair of the Strategic Advisory Board of 4basebio, Strategic Advisor to Primrose Bio and UserCue, and a Board Member of Meridian Bioscience. In addition, she serves as Chair of the Foundation for the Alliance for mRNA Medicines. Previously, Deb was Vice President of Strategy & Business Development at Maravai LifeSciences and held leadership positions at Thermo Fisher Scientific, Strategic Diagnostics, GeneLogic, Johns Hopkins University, and Amersham Life Sciences. At Johns Hopkins, she led technology development and commercialization efforts, collaborating closely with faculty and research teams to advance discoveries toward industry partnerships and clinical translation. Her expertise includes technology commercialization, strategic partnerships, corporate development, licensing, and venture creation across the life sciences industry, with a track record of building collaborations that accelerate innovation and bring transformative technologies to market.

Timothy Miller, MD, PhD, is the David Clayson Professor of Neurology at Washington University School of Medicine in St. Louis. He earned his MD and PhD from Washington University School of Medicine and his BS in Chemistry and French from the University of Virginia. Dr. Miller directs the Miller Lab, co-directs the ALS Center, and serves as Vice Chair of Research for the Department of Neurology at Washington University School of Medicine. His research focuses on developing innovative therapies for neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and tauopathies. He is internationally recognized for pioneering work in antisense oligonucleotide (ASO) therapeutics, leading successful clinical trials of ASO therapies for SOD1-related ALS and advancing the development of ASO-based treatments for tauopathies. A highly accomplished physician-scientist, Dr. Miller has received numerous distinguished honors, including the Sheila Essey Award for ALS Research, the Healey Center Prize for Innovation in ALS, the Rainwater Prize for Outstanding Innovation in Neurodegenerative Disease Research, and the Potamkin Prize for Research in Pick’s, Alzheimer’s, and Related Diseases. In recognition of his contributions to medicine and neuroscience, he was elected to the National Academy of Medicine in 2023 and the Association of American Physicians in 2026.

Amy Walker, Ph.D.

Andrew Geall, Ph.D., is the co-founder and Chief Development Officer at Replicate Bioscience, a San Diego based clinical-stage company pioneering novel self-replicating RNA (srRNA) technology for applications across infectious disease, immunology, and other therapeutic areas.  He started his career in RNA vaccine in 2008, when he initiated and lead the self-amplifying RNA vaccines program at Novartis Vaccines.  His clinical interest are in infectious disease, Replicate Bio just completed its 1^st phase 1 clinical trail with a rabies vaccines and the data was published in Jan 2025 in Nature Communications.

Erica Barnes is the Executive Director of the MN Rare Disease Advisory Council, a Minnesota state agency which was created in 2019 following grassroots efforts by a cross-sector coalition of advocates. She has over a decade of advocacy and non-profit leadership experience, having co-founded with her husband Chloe’s Fight Rare Disease Foundation in honor of her late daughter. Ms. Barnes has a number of committee roles related to rare diseases. She chairs the NIH funded Global Leukodystrophy Initiative Clinical Trials Network patient advocacy consortium, is a member of the Sickle Cell Data Collection (SCDC) Program and is a coalition leader of CureMLD.

Hao Yan, Ph.D.  studied chemistry and earned his Bachelor’s degree at Shandong University, China. He obtained his PhD in Chemistry under Professor N. C. Seeman, New York University in 2001, working on design and construction of sequence dependent DNA nanomechanical devices. He then moved to the Computer Science Department at Duke University, where he continued to explore his interests in DNA based molecular computing and programming. Following a three year period as an Assistant Research Professor at Duke University, he joined Arizona State University as Assistant Professor in Department of Chemistry and Biochemistry in 2004. In 2008, he was promoted with early tenure directly to Full Professor and he is currently the Regents Professor & Milton D. Glick Distinguished Professor in Chemistry and Biochemistry and Director of the Center for Molecular Design and Biomimetics in the Biodesign Institute at Arizona State University. He is leading an interdisciplinary team to design bio-inspired molecular building blocks and program their higher order assembly into systems that will perform complex functions. Dr. Yan has published more than 250 papers, including more than 55 papers published in journals such as Nature, Science, Cell and their sub-journals. He has received honors including the Foresight Institute Feynman Prize in Nanotechnology, the Rozenberg Tulip Award in DNA Computing, Humboldt Research Award, Fast Company 100 Most Creative People, Alfred P. Sloan Research Fellowship, NSF Career Award, AFOSR Young Investigator Award, etc. He has served as president of the International Society for Nanoscale Science, Computation and Engineering. He has served as General Chair of the Annual FNANO conference and played an important role in shaping the global biomolecular nanoscience community. He is an Elected Fellow of the National Academy of Inventors (NAI), the American Institute for Medical and Biological Engineering (AIMBE), the American Association for Advancement of Science (AAAS), the European Academy of Sciences (EurASc), and the European Academy of Sciences and Arts (EASA). He currently serves as Associate Editor for Science Advances and ACS Applied Bio Materials.

Heather Gray-Edwards, DVM, Ph.D. is an Associate Professor whose research focuses on the development and translation of adeno-associated virus (AAV) gene therapies for rare and ultra-rare genetic diseases. Her work includes evaluating novel therapeutic approaches in naturally occurring and CRISPR genome-edited large and small animal models of human genetic diseases, including Tay-Sachs disease, Sandhoff disease, GM1 gangliosidosis, spastic paraplegia 4, Huntington’s disease, sialidosis, GM3 synthase deficiency, cerebellar ataxia, nemaline myopathy, neurofibromatosis, HNRNPU-related disorders, mucolipidosis II, and Maple Syrup Urine Disease. Dr. Gray-Edwards has contributed to the translation of AAV gene therapies from preclinical research to human clinical trials for GM1 gangliosidosis, Tay-Sachs disease, and Sandhoff disease. In addition to her research, she supports academic groups in navigating the regulatory pathway for novel therapies, providing expertise throughout the pre-IND and IND submission processes to help advance promising treatments from the laboratory to the clinic.

Jicong Cao, Ph.D. is the co-founder and Chief Executive Officer of Raina Biosciences. He earned his Ph.D. in Chemical Engineering from Rensselaer Polytechnic Institute and completed his postdoctoral training at Massachusetts Institute of Technology (MIT). During his doctoral and postdoctoral research, Dr. Cao developed synthetic biology tools for mRNA engineering and AI-powered high-throughput screening platforms for DNA and RNA regulatory elements. Following his time at MIT, he co-founded Bota Biosciences, where he served as Vice President of Engineering. Bota Biosciences focused on developing automated, high-throughput platforms for enzyme discovery, screening, and engineering. In 2023, Dr. Cao co-founded Raina Biosciences, where he leads the development of GEMORNA, integrating generative AI with high-throughput experimental screening for therapeutic mRNA design and optimization.

Xiling Shen, Ph.D., is a Professor of GI Medical Oncology at MD Anderson Cancer Center and a CPRIT Scholar. He earned his BS, MS, and PhD from Stanford University. Dr. Shen serves as Co-Director of the CRC Moonshot at MD Anderson Cancer Center and as Acting Director of the Terasaki Institute for Biomedical Innovation. He previously received the prestigious National Science Foundation (NSF) Faculty CAREER Award while at Cornell University. His leadership roles have included serving on the editorial board of the NIH Director's New Innovator Award (DP2) program, Chair of the Steering Committee for the NCI Patient-Derived Models of Cancer Consortium, and Chair of the Tissue Engineering Collaborative. He has also served as Cancer Track Chair for the Biomedical Engineering Society (BMES) Annual Meeting and as Director of the Woo Center for Big Data and Precision Health at Duke University. Dr. Shen has founded multiple biotechnology startups that have translated discoveries from his laboratory into ongoing clinical trials. His research focuses on cancer biology, stem cells, precision therapeutics, and the gut-brain axis, with an emphasis on advancing innovative approaches to cancer diagnosis and treatment.

John Cooke, Ph.D. is Professor and Chair of the Department of Cardiovascular Sciences and Director of the Center for RNA Therapeutics at Houston Methodist Research Institute. He trained in internal medicine and cardiovascular medicine at Mayo Clinic and subsequently held faculty appointments at Harvard Medical School and Stanford University before joining Houston Methodist in 2013. Dr. Cooke’s research focuses on vascular and RNA biology, with support from federal agencies, nonprofit organizations, and industry. He has authored more than 500 research papers, reviews, and patents, and his recent research has explored the non-canonical functions of telomerase and their role in reversing cellular senescence. His contributions to biomedical innovation have been recognized through several honors, including Stanford University’s Outstanding Inventor award, election to the National Academy of Inventors, and the Mayo Clinic Distinguished Alumni Award. As Director of the Center for RNA Therapeutics, Dr. Cooke oversees efforts to advance RNA-based technologies and therapeutics. The Center’s RNACore has generated more than 650 constructs for over 150 clients and, in 2025, led an academic effort to develop, synthesize, encapsulate, validate, and administer a personalized mRNA cancer vaccine.

Morrie Ruffin has more than 30 years of experience in the regenerative medicine, biotechnology, and healthcare industries. He is the founder of Adjuvant Partners and previously served as Executive Vice President of Capital Formation and Business Development at the Biotechnology Innovation Organization (BIO), where he helped develop the organization’s global business development and investor outreach programs. Throughout his career, Mr. Ruffin has played a significant role in establishing and leading several prominent life sciences organizations. He co-founded the Alliance for Regenerative Medicine (ARM), serving as its Managing Director from 2009 to 2017, and later helped establish the Alliance for Artificial Intelligence in Healthcare (AAIH). In 2023, he helped found the Alliance for mRNA Medicines (AMM), where he currently serves as Senior Advisor, supporting efforts to advance mRNA and next-generation RNA therapeutics and vaccines.Earlier in his career, Mr. Ruffin served as a senior legislative assistant to U.S. Senator Arlen Specter. He holds an MA in International Studies and Economics from the Johns Hopkins School of Advanced International Studies (SAIS) and a BA from the University of Virginia.

Claes Wahlestedt, M.D., Ph.D., is Leonard M. Miller Professor and Director of the Center for Therapeutic Innovation at the University of Miami Miller School of Medicine where he also serves as Associate Dean for Therapeutic Innovation. A native of Sweden, Dr. Wahlestedt obtained his MD and PhD degrees from Lund University. Prior to joining the U. Miami, Dr. Wahlestedt was a founding Professor and a Director at The Scripps Research Institute’s Florida campus. Between 1997 and 2005 he directed the Genome Center at the Karolinska Institute (a joint venture with Pharmacia & Upjohn) where he was also an endowed Professor of Pharmacogenomics and Department Chair. He has also been a faculty member at Cornell University Medical College and at McGill University. At different stages in his career, he has directed large R&D organizations in the pharmaceutical industry for Astra-Zeneca or Pharmacia/Pfizer. In addition, he has founded several biotechnology companies including CuRNA (now with Camp4) and TheRNA. Dr. Wahlestedt has published over 300 peer-reviewed scientific publications (h-factor 110) and a number of patents. In the context of RNA-targeted therapeutics research, he has made several original contributions: 1) His team was the first to deliver antisense oligonucleotides to the living brain (Science 1993; Nature 1993 etc.); (2) he introduced the use of locked nucleic acids (LNA) in functional antisense and siRNA molecules (PNAS 2000; NAR 2005 etc.); and 3) he has discovered and targeted a wide variety of non-coding RNAs with oligonucleotides including the invention of AntagoNATs for gene up-regulation (Science 2005: Nature Rev Drug Disc 2013, 2023 etc.).

Samir El-Andaloussi, Ph.D. is Professor of Biomolecular Medicine and Advanced Therapies at the Department of Laboratory Medicine, Karolinska Institutet. He is Principal Investigator for the Biomolecular Medicine lab and Head of Division for Biomolecular and Cellular Medicine within the department. He is also the Co-director of Research at the Karolinska ATMP Center. Prof Andaloussi is also a visiting Professor at University of Oxford. Additionally, he is also co-founder of, and scientific adviser for, Evox Therapeutics that develops engineered extracellular vesicles (EVs) for treatment of genetic diseases. Samir has worked on various aspects related to non-viral extrahepatic delivery of RNA and protein therapeutics, now with emphasis on the use engineered EVs and synthetic LNPs. He has recently established EV engineering methods that enable efficient targeted delivery of macromolecular therapeutics, such as gene editors and mRNAs in pre-clinical models with focus CNS and cancer. Samir has co-authored 220+ publications with more than 34k citations in total.

Ken Yamada, Ph.D., is currently an Assistant Professor in RNA Therapeutics Institute at UMass Chan Medical School (2021-current) and is investigating transformative novel chemistry platforms for opening difficult extra-hepatic tissues to oligonucleotide-therapeutics intervention. His exploration with Nucleic Acid Chemistry started when he was a PhD student in Prof. Mitsuo Sekine’s lab, where he cultivated his discipline as a nucleic acid and oligonucleotide chemist. After his career path led to a postdoctoral position in Prof. Masad Damha’s lab at McGill University (2012-2014) and an Assistant Professor position in Prof. Fumi Nagatsugi’s lab at Tohoku University (2014-2017), he joined Prof. Anastasia Khvorova’s lab to explore his potential as a chemist to contribute to the siRNA therapeutics field. In a dynamic collaborative scientific culture in RTI, he established several innovative chemistry platforms that surpass current state-of-art siRNA technology. His inventions generated multiple patents, part of those licensed out to top-leading biotech companies.

Simon Frost, Ph.D. and his wife, Nina, are the parents of Annabel, who lives with AHC, and her older sister, Clara. Following Annabel’s diagnosis, Simon and Nina co-founded RARE Hope and have worked to accelerate rare-disease research and therapeutic development. Simon also co-founded the Genomic Answers for Children’s Health Alliance, whose work helped advance the bipartisan Genomic Answers for Children’s Health Act (H.R. 7118), introduced in 2026 to establish Medicaid coverage of whole-genome and whole-exome sequencing as first-tier diagnostic tools for children with suspected rare or genetic disorders. His board and advisory service has included Global Genes, RARE-X, the NIH Cures Acceleration Network Review Board, IRDiRC, Uncommon Cures, and Cure AHC. In business, Simon co-founded and led real-estate platforms that acquired more than 19,000 homes and deployed over $5 billion in capital.

Casey McPherson