Henrik Zetterberg

Biography

Niren Murthy

Biography
Dr. Niren Murthy is a professor in the Department of Bioengineering at the University of California at Berkeley and a member of the Innovative Genomics Institute. Dr. Murthy’s scientific career has focused on the molecular design and synthesis of new materials for drug delivery and molecular imaging. The Murthy laboratory is currently focused on developing next generation lipid nanoparticles that can deliver mRNA and gene editing enzymes in vivo. Several start-up companies and licenses have been generated from the laboratory, in particular, the start-up companies GenEdit, BioAmp Diagnostics, Microbial Medical and Opus Biosciences were formed based upon research conducted in the Murthy lab.

Jay Bradner

Biography
Dr. James "Jay" Bradner is executive vice president, Research and Development. He is responsible for advancing Amgen's pipeline, which includes potential first-in-class or best-in-class medicines in all stages of development across the company's four therapeutic areas of focus: oncology, inflammation, general medicine and rare disease, in addition to biosimilars. He is also responsible for Amgen's worldwide research efforts. Prior to joining Amgen, Dr. Bradner served as President of the Novartis Institutes for BioMedical Research, where he was a member of the Executive Committee of Novartis. His research and leadership have contributed to numerous development programs, multiple investigational new drug applications and positive proof-of-concept studies in clinical investigation. He also has been a clinician at the Dana-Farber Cancer Institute and an associate professor at Harvard Medical School. As an entrepreneur, he co-founded and built several biotechnology startups. Additionally, he has co-authored dozens of scientific publications and is a named inventor on numerous U.S. patent applications. Dr. Bradner is a graduate of Harvard College and the University of Chicago Medical School. He completed his residency in Medicine at Brigham & Women's Hospital, his fellowship in Medical Oncology and Hematology at the Dana-Farber Harvard Cancer Center, and his postdoctoral training in Chemistry and Chemical Biology at Harvard University.

Adrian Krainer

Biography
Prof. Adrian Krainer completed his undergraduate education at Columbia University in 1981, with a B.A. in Biochemistry. He then completed his Ph.D. in Biochemistry at Harvard University in 1986, in the lab of Prof. Tom Maniatis. After graduation, he became the first Cold Spring Harbor Fellow, and in 1989 he joined the faculty at Cold Spring Harbor Laboratory. He is currently the St. Giles Foundation Professor at CSHL, and the Deputy Director of Research of the CSHL Cancer Center. He is also a co-founder and Director of Stoke Therapeutics. Adrian's lab at CSHL uses multidisciplinary approaches to elucidate pre-mRNA splicing mechanisms and alternative splicing regulation. An important goal is to apply this knowledge to uncover the roles of defective splicing in genetic diseases and cancer. The lab also uses antisense technology to develop novel therapeutics that target pre-mRNA or mRNA to modulate gene expression post-transcriptionally. One notable application—in collaboration with Ionis Pharmaceuticals and Biogen—was the development of the antisense oligonucleotide nusinersen (Spinraza), which became the first approved drug for spinal muscular atrophy, a severe genetic disease that causes motor-neuron degeneration. Research from the Krainer lab has also implicated splicing alterations in cancer, and his lab is currently pursuing antisense-therapeutic approaches in the context of various cancers. Adrian is a recipient of the Albany Medical Center Prize in Medicine and Biomedical Research, the Wolf Prize in Medicine, the Life Sciences Breakthrough Prize, the RNA Society’s Lifetime Achievement Award, the Reemtsma Foundation International Prize in Translational Neuroscience, the Speiser Award in Pharmaceutical Sciences, the Ross Prize in Molecular Medicine, the Gabbay Award in Biotechnology and Medicine, the Takeda Pharmaceuticals Innovators in Science Senior Scientist Award in Rare Diseases, and the Watanabe Prize in Translational Research. He served as President of the RNA Society, and is a member of the National Academy of Sciences, the National Academy of Medicine, the National Academy of Inventors, and the American Academy of Arts & Sciences.

Alexis C. Komor

Biography
Alexis C. Komor is a pioneering genome‑editing researcher who co‑developed the first CRISPR base editors with David Liu at the Broad Institute. Her work established base editing as a new class of genome engineering tools that enable precise single‑nucleotide changes in DNA without double‑strand breaks, laying the foundation for correcting disease‑causing point mutations with high precision. She continues to lead efforts to translate these innovations into direct in vivo therapeutics, including strategies to deliver base editors via mRNA and other nucleic‑acid platforms to repair genes within patients’ cells. As her work established the scientific foundation of Beam Therapeutics, Komor has helped drive the clinical development of base editing, underscoring the broad impact of her work on programmable gene editing and precision medicine. Her groundbreaking contributions are influencing a new generation of in vivo RNA and gene‑based therapies (spanning mRNA, siRNA, and antisense oligonucleotide modalities) and inspiring novel small‑molecule approaches to treat genetic diseases.

Gene Yeo

Biography
Gene Yeo is a Professor of Cellular and Molecular Medicine at UC San Diego. He earned degrees in Chemical Engineering and Economics from the University of Illinois at Urbana-Champaign, a PhD in Computational Neuroscience from MIT, and an MBA from the UCSD Rady School of Management. His research focuses on RNA processing in development and disease, leveraging iPSC and murine models. His lab pioneered in vivo RNA targeting with CRISPR/Cas systems and developed enhanced CLIP technologies for large-scale protein–RNA interaction mapping. Dr. Yeo serves on the editorial boards of Cell Reports and Cell Research, has received multiple prestigious awards, and is a co-founder of several RNA-focused biotechnology companies.

Ben Deverman

Biography
Ben Deverman is the senior director of the vector engineering research group at the Stanley Center for Psychiatric Research at the Broad Institute of MIT and Harvard, where he is also an institute scientist. The vector engineering team develops innovative gene delivery solutions for studying the central nervous system (CNS), with the aim of uncovering new avenues for treating psychiatric disorders. His group applies a variety of approaches including protein engineering, high-throughput in vivo selection and screening methods, and machine learning to develop novel AAV vectors that overcome pressing gene delivery challenges. In recent work, the Deverman team and collaborators found that the AAV-PHP.B family of capsids, which efficiently deliver genes throughout the mouse CNS, cross the blood-brain barrier by engaging a novel receptor on the vasculature, providing mechanistic insights that can be leveraged to engineer the next generation of AAV capsids for human CNS gene therapy. In response to the COVID-19 pandemic, the Deverman lab built and maintains COVID-19 CG, an interactive, open access browser to help vaccine, therapeutics, and diagnostics developers and public health officials track SARS-CoV-2 mutations and lineages by location and time. Deverman joined the Broad in March 2018. Before this, he was the director of the CLARITY, Optogenetics and Vector Engineering Research (CLOVER) Center within the Beckman Institute at the California Institute of Technology. At Caltech, Deverman and colleagues identified numerous capsids, including AAV-PHP.B and an enhanced variant, AAV-PHP.eB, that made efficient brain-wide gene delivery in the adult possible for the first time. AAV-PHP.B and AAV-PHP.eB are now in use in laboratories around the world and are enabling a wide range of translational and basic neuroscience experiments. Deverman led the generation of the panel of AAV-PHP capsids by developing a novel AAV selection method, called Cre Recombinase-based Targeted Evolution (CREATE), that uses Cre transgenics to select for AAVs that transduce defined target cell types. Deverman has nearly 14 years of experience as a molecular biologist studying neuroscience and AAV engineering. As a postdoctoral fellow and research scientist at the California Institute of Technology, Deverman’s research focused on viral vector development and the roles of cytokines during neurodevelopment and in the context of demyelinating disease. He received a Ph.D. in molecular and cellular biology from Washington University School of Medicine and a B.S. in biochemistry from the University of Detroit Mercy. In 2019 he received the Broad Institute Excellence Award in Mentorship, Teaching, and Training and is a recipient of a Merkin Institute Fellowship at the Broad.

Timothy Yu

Biography
Timothy Yu, MD, PhD, is recognized as a leader in the field of gene therapy, particularly in the development of personalized gene therapies for individuals with rare genetic disorders. A graduate of Harvard College, he completed MD-PhD training at UCSF, neurology residency at Massachusetts General Hospital and Brigham and Women’s Hospital, and a fellowship in neurogenetics at Boston Children’s Hospital. He joined the faculty of Boston Children’s Hospital in the Division of Genetics and Genomics in 2013. He is also an Associate Member at the Broad Institute Dr. Yu’s research group works at the intersection of genomics, informatics, and neurobiology with a focus on understanding and treating disorders of brain development. He was an early pioneer in methodology for genome sequencing and interpretation, which he applied to discover genes responsible for brain development and autism. He is also recognized for having developed the world’s first individualized genomic medicine: milasen, an antisense oligonucleotide targeting a unique genetic mutation causing a child’s fatal neurodegenerative condition – in under one year. Milasen was the first in a pilot series of individualized therapeutics for severe neurogenetic conditions that his team has since created. It also inspired efforts to advance individualized therapeutics for dozens of neglected genetic conditions worldwide, including the N-of-1 Collaborative, an international coalition founded by Dr. Yu to advance scientific, regulatory, and ethical best practices for this budding field. Dr. Yu has authored more than 80 scientific publications and has been recognized with numerous awards and honors, including the NORD Rare Impact Award, the Global Genes Rare Champion award, and the ANA Denny-Brown Young Neurologic Scholar award. He is dedicated to advancing precision medicine and improving patient outcomes through his research and clinical practice.

Randy Schekman

Biography
Randy W. Schekman is a Nobel laureate and Professor of Molecular and Cell Biology at UC Berkeley. He shared the 2013 Nobel Prize in Physiology or Medicine for discovering the machinery that regulates vesicle transport—how cells organize, package, and deliver cargo such as proteins and hormones. Cellular Transport: He mapped the fundamental genetic and biochemical pathways of yeast cell transport. These discoveries apply directly to human cells and are used widely in the biotech industry (e.g., producing recombinant insulin).

Shicheng Guo

Biography
Shicheng Guo isSenior Director of Translational Genetics & Data Science at Arrowhead Pharmaceuticals, where I lead human-genetics-driven target discovery and biomarker strategy for RNA-based therapeutics. Dr. Guo's work sits where human data, genomics, and AI meet drug development — using population-scale biology, biobanks, and real-world evidence to power an end-to-end, AI-native discovery engine, increasingly orchestrated by agentic AI systems that reason, plan, and act across the target-to-medicine pipeline.