Changing delivery.

Dr. Aravind Asokan is Professor and Director of the Danaher Beacon for Gene Therapy Innovation at Duke University in Durham, North Carolina, USA. He has appointments in the Departments of Surgery, Biomedical Engineering, Molecular Genetics and Microbiology. His research group is focused on studying and engineering viruses & RNA platforms to enable transformative, new medicines. Dr. Asokan has co-founded multiple start-ups including Stride Bio (acquired by Ginkgo), Warden Bio (acquired by Kriya Tx), Sphere Gene Therapies and Torque Bio, where he currently serves scientific advisor and board director. He is a director-at-large for the American Society of Gene and Cell Therapy (ASGCT) and scientific advisor to Ring Therapeutics, Atsena Therapeutics, Mammoth Bio, Coave Therapeutics and Ginkgo Bioworks.

Jacob Giehm Mikkelsen is a Professor at the Department of Biomedicine, Aarhus University.

Jacob is a trained retrovirologist with >25 years of experience in gene transfer and virus-based gene delivery technologies with focus on gene therapy, genetic engineering, and more recently genome editing using CRISPR. Over the last 10 years, Jacob’s lab has fronted the development of lentivirus-derived nanoparticles for delivery of genome-modifying proteins and ribonucleoprotein complexes. Part of his current research focuses on providing a better understanding of virus-based protein delivery and cell-vehicle interactions to develop enveloped CRISPR/Cas delivery technologies further and bring these new tools closer to the clinic.

J. Keith Joung is a leading innovator in the field of gene editing. He is currently a Lead Translator at Arena BioWorks and is Professor of Pathology (on leave 2023-2024) at Harvard Medical School. He was previously the Robert B. Colvin, M.D. Endowed Chair in Pathology and a Pathologist at Massachusetts General Hospital (MGH). Dr. Joung has been a pioneer in the development of important technologies for targeted gene editing and epigenetic editing of human cells. He has received numerous awards including the American Society of Gene and Cell Therapy Outstanding Achievement award in 2023, the 2022 Samsung Ho-Am Prize in Medicine, an NIH Director’s Pioneer Award, an NIH Director’s Transformative Research Project R01 Award, the MGH Research Scholar Award (2011 and 2016), an NIH R35 MIRA (Maximizing Investigators Research Award), election into the American Association of University Pathologists, and designation as a “Highly Cited Researcher” for the past eight consecutive years (2016-2023) by Thomson Reuters/Clarivate Analytics. He serves on the editorial boards of Cell Genomics, Genome Biology, Human Gene Therapy, and Trends in Biotechnology. He has co-founded multiple biotechnology companies including Beam Therapeutics (NASDAQ: BEAM), Chroma Medicine, Editas Medicine (NASDAQ: EDIT), Nvelop Therapeutics, Pairwise Plants, SeQure Dx, Inc., and Verve Therapeutics (NASDAQ: VERV). Dr. Joung holds a Ph.D. in genetics from Harvard University, an M.D. from Harvard Medical School and an A.B. magna cum laude in biochemical sciences from Harvard College.

Sadik Kassim, PhD, serves as Chief Technology Officer of Genomic Medicines for the Life Sciences companies at Danaher Corporation. He has extensive experience in the biotechnology industry with a specific focus on cell and gene therapy bioprocessing and translational research. In his prior roles, Dr. Kassim and his teams have contributed to the successful BLA and MAA applications for three of the commercially available CAR-T therapies: Kymriah®, Yescarta®, and Tecartus®. Prior to Danaher, Dr. Kassim was Chief Technology Officer at Vor Bio where he built the technical operations team responsible for process development, analytical development, supply chain and manufacturing support of a CRISPR gene-edited HSPC product and oversaw the company’s CAR-T efforts. Previously, Dr. Kassim served as Executive Director at Kite Pharma and led the development of manufacturing processes for autologous CAR-T and TCR-based cell therapies. As the Chief Scientific Officer at MustangBio, Dr. Kassim managed the foundational build-out of the company’s preclinical and manufacturing activities. Earlier in his career, he was Head of Early Analytical Development for Novartis’ Cell and Gene Therapies Unit and worked on research teams as post-doctoral research fellow at the National Cancer Institute with Dr. Steven Rosenberg, the University of Pennsylvania Gene Therapy Program with Dr. Jim Wilson, and Johnson and Johnson’s Immunology Discovery group. He received his PhD in microbiology and immunology from Louisiana State University and his BS in cellular and molecular biology from Tulane University.

David R. Liu is the Richard Merkin Professor and director of the Merkin Institute of Transformative Technologies in Healthcare, vice-chair of the faculty at the Broad Institute of MIT and Harvard, the Thomas Dudley Cabot Professor of the Natural Sciences at Harvard University, and a Howard Hughes Medical Institute (HHMI) investigator. Liu’s research integrates chemistry and evolution to illuminate biology and enable next-generation therapeutics. His major research interests include the engineering, evolution, and in vivo delivery of genome editing proteins such as base editors and prime editors to study and treat genetic diseases; the evolution of proteins with novel therapeutic potential using phage-assisted continuous evolution (PACE); and the discovery of bioactive synthetic small molecules and synthetic polymers using DNA-templated organic synthesis and DNA-encoded libraries.

Base editing—the first general method to perform precision gene editing without double-stranded breaks, and a Science 2017 Breakthrough of the Year finalist—as well as prime editing, PACE, and DNA-templated synthesis are four examples of technologies pioneered in his laboratory. These technologies are used by thousands of labs around the world and have enabled the study and treatment of genetic diseases. Five base editing clinical trials are already underway to treat leukemia, hypercholesterolemia, sickle-cell disease, and beta-thalassemia, and the first clinical benefit of a base edited therapeutic in a T-cell leukemia trial has been reported.

Liu graduated first in his class at Harvard College in 1994. During his doctoral research at UC Berkeley, Liu initiated the first general effort to expand the genetic code in living cells. He earned his PhD in 1999 and became assistant professor of chemistry and chemical biology at Harvard University in the same year.

He was promoted to associate professor in 2003 and to full professor in 2005. Liu became a Howard Hughes Medical Institute investigator in 2005 and joined the JASONs, academic science advisors to the U.S. government, in 2009. In 2016 he became a Core Institute Member and Vice-Chair of the Faculty at the Broad Institute of MIT and Harvard, and Director of the Chemical Biology and Therapeutics Science Program.

Liu has published more than 250 papers and is the inventor on more than 110 issued U.S. patents. Liu has been elected to the U.S. National Academy of Sciences, the U.S. National Academy of Medicine, and the American Association for the Advancement of Science. He is the 2022 King Faisal Prize Laureate in Medicine. He has earned several University-wide distinctions for teaching at Harvard, including the Joseph R. Levenson Memorial Teaching Prize, the Roslyn Abramson Award, and a Harvard College Professorship. His research accomplishments have earned distinctions including the Ronald Breslow Award for Biomimetic Chemistry, the American Chemical Society David Perlman Award, ACS Chemical Biology Award, the American Chemical Society Pure Chemistry Award, the Arthur Cope Young Scholar Award, the NIH Marshall Nirenberg Lecturer, and awards from the Sloan Foundation, Beckman Foundation, NSF CAREER Program, and Searle Scholars Program.

IIn 2016, 2019, 2020, and 2021 he was named one of the Top 20 Translational Researchers in the world by Nature Biotechnology, and was named one of Nature’s 10 researchers in world. Liu was named as a leading global thinker by the Foreign Policy Leading Global Thinkers in 2017, to the STATUS List of 46 leaders in health, medicine, and science by STAT News, and as one of the “Most Influential People in Biopharma in 2023” by Fierce Biotech. Liu is the founder or co-founder of several public and private biotechnology and therapeutics companies, including Beam Therapeutics, Prime Medicine, Editas Medicine, Pairwise Plants, Chroma Medicine, Exo Therapeutics, Resonance Medicine, and Nvelop Therapeutics.

Outside of the lab, Liu pursues a variety of hobbies at the intersection of science and art, including photography around the world, raising large bonsai trees indoors under LED light canopies he designed, creating art from wood, metal, stone, and/or electronic components, and painting.

Luigi Naldini, M.D., Ph.D., is Professor of Cell and Tissue Biology and of Gene and Cell Therapy at the San Raffaele University School of Medicine and Scientific Director of the San Raffaele Telethon Institute for Gene Therapy (Milan, Italy). He has received his medical degree from the University of Turin (Italy) and his PhD from the University “La Sapienza” of Rome (Italy). For the past 30 years he has pioneered the development and applications of lentiviral vectors for gene therapy, which have become one of the most widely used tools in biomedical research and are providing a long-sought hope of cures for several otherwise deadly human diseases. Throughout this time, he has continued to investigate strategies to overcome the major hurdles to safe and effective gene therapy, bringing about innovative solutions that are not only being translated into new therapeutic strategies for genetic diseases and cancer, but have also allowed novel insights into hematopoietic stem cell function and tumor angiogenesis. He also contributed to pioneer and advance the use of artificial nucleases for targeted genome editing in cell and gene therapy. He has published >300 scientific papers. SCOPUS Author h-index: 108.

Member of the European Molecular Biology Organization (EMBO), has been President of the European Society of Gene and Cell Therapy (ESGCT), appointed as expert on the “Human Gene Editing Study” of the US National Academies of Sciences and of Medicine, and on the Italian National Committee for Biosafety, Biotechnology and Life Sciences. He was awarded the Outstanding Achievement Award from the American Society of Gene and Cell Therapy (ASGCT) in 2014 and from ESGCT in 2015, an Honorary doctorate from the Vrije University, Brussel, in 2015, the Jimenez Diaz Prize in 2016, the Beutler Prize from the American Society of Hematology (ASH) in 2017 and the 2019 Jeantet-Collen Prize for Translational Medicine. He was nominated “Grande Ufficiale dell’Ordine Al Merito della Repubblica Italiana”, one of the highest-ranking honors in Italy, and elected member of “Accademia dei Lincei”, the oldest and most prestigious national academic society. He is also co-founder of three innovative biotech start-up companies: Genenta (recently listed on Nasdaq), Epsilen Bio (now acquired by Chroma Medicine) and Genespire.

Gabor has 25+ years of experience in the development of cell and gene therapy products focusing mostly on genetic diseases. Currently the SVP of Translational Research at Mammoth Biosciences, he previously served as CSO of Vedere Bio II, Head of Gene Therapy at BioMarin Pharmaceuticals, Vice President of Translational Research at bluebird bio, Sangamo, etc. He initiated and coordinated multiple research programs using different gene delivery platforms to correct genetic and acquired diseases and cancer. He led several programs from discovery, pre-clinical research and through late-stage development, including approved gene therapy treatment for CALD (SKYSONA), β-thalassemia (ZYNTEGLO), sickle cell disease (LYFGENIA), multiple myeloma (ABECMA), and hemophilia A (ROCTAVIAN).