Session Abstract – PMWC 2022 Silicon Valley


Track Chair:
Jane Grogan, Graphite Bio

Sessions:

  • Building Future Precision Care in Qatar
    Session Chair: Khalid Fakhro, Sidra Medicine
    - Barbro Friden, Sidra Medicine
    - Ziyad Hijazi, Sidra Medicine
    - Edison Liu, Jackson Labs
  • Advances in Gene & Cell Therapies
    - Peter Nell, Mammoth
    - Laura Sepp-Lorenzino, Intellia Tx
    - Amy Simon, Beam Therapeutics
    - Eric Ostertag, Poseida Therapuetics
    - Jane Grogan, Graphite Bio
  • Translating CGT to the Clinic
    Session Chair: Nicole K. Paulk, UCSF
    - Matthew Porteus, Stanford
    - Eric Crombez, Ultragenyx Gene Therapy
    - Peter Francis, 4D Molecular Therapeutics
    - Erandi De Silva, Forge Biologics
    - Patrice Hugo, Q2 Solutions
  • Bioethics & Ethical Implications in Cell and Gene Therapies
    Session Chair: Ilana Yurkiewicz, Stanford University
  • Past, Present and Future of CRISPR Where will CRISPR Take Us and How is the Technology Evolving to Realize its Full Potential
    Session Chair: Janice Chen, Mammoth
    - Fyodor Urnov, UC Berkeley
    - Kiana Aran, Cardea Bio
    - Laura Sepp-Lorenzino, Intellia Tx

 Session Chair Profile

Ph.D., Chief Scientific Officer, Graphite Bio

Biography
Dr. Jane Grogan is chief scientific officer of Graphite Bio, a gene editing and cell therapy company, leading clinical research and development efforts. Prior to Graphite Bio, Dr. Grogan was chief scientific officer at ArsenalBio; and head of adaptive tumor immunity and principal scientist of cancer immunology at Genentech. Her work has identified key regulators and epigenetic modifiers of immune cells, including TIGIT, for autoimmune and oncology indications. Dr. Grogan has published over 60 papers in a wide array of journals and is the inventor on over 20 patents. She obtained her Ph.D. from Leiden University, her BSci (Hons) degree from the University of Melbourne, and her post-doctoral training as an Alexander von Humboldt Fellow at the German Rheumatism Research Centre Berlin and as a Howard Hughes Fellow at UCSF.


 Session Chair Profile

Ph.D., Assistant Professor, UCSF

Biography
Dr. Paulk’s lab is focused on next-generation AAV gene delivery and gene editing platforms. They are species/disease/organ/tissue/pathway/cell type/gene agnostic, working at the platform level to forge new paths engineering novel ways to make all AAV therapies cheaper, easier and faster. Dr. Paulk has developed gene therapies for numerous rare diseases and cancer, and has applied high-throughput comparative proteomic and epigenomic approaches to uncover fundamental AAV biology. Dr. Paulk sits on the Scientific Advisory Boards for Dyno Tx, CEVEC, Excision Bio, WhiteLab Genomics and Sarepta; Scientific Editorial Boards for the journals Human Gene Therapy and Gene Therapy; and currently sits on the American Society of Gene & Cell Therapy Translational Science & Product Development Committee. Paulk earned her BS in Medical Microbiology, her PhD in Viral Gene Therapy from Oregon Health & Science University, and completed her Postdoctoral Fellowship in Human Gene Therapy at Stanford University.


 Session Chair Profile

Ph.D., CTO and Co-Founder, Mammoth Biosciences

Biography
Janice Chen is co-founder/CTO of Mammoth Biosciences, a biotechnology company based in the San Francisco Area that harnesses the diversity of nature to power the next generation of CRISPR products across diagnostics and therapeutics. Through its discovery of novel CRISPR systems, the company enables the potential of its platform to read and write the code of life. Janice received her PhD from the lab of Nobel Laureate Jennifer Doudna at University of California, Berkeley. She investigated mechanisms of CRISPR proteins and developed technologies leading to multiple papers and patents, and co-invented the programmable CRISPR-based detection technology called DETECTR®. Janice was selected as a Forbes 30 Under 30 in Healthcare, Business Insider's 30 Under 40 in Healthcare, Endpoints Top 20 Women in Biopharma, MIT Technology Review 35 Innovators Under 35, EY Entrepreneur Of The Year, SF Business Times Most Influential Women, and delivered a TEDx talk on the potential for CRISPR to democratize diagnostics.


 Session Chair Profile

M.D., Faculty Physician, Stanford University

Biography
Dr. Yurkiewicz is a faculty physician at Stanford University. Trained as an oncologist and internist, she practices primary care for cancer survivors and those with genetic diagnoses conferring elevated risk. When not practicing medicine, she writes about it as a medical journalist. Her journalism has appeared in The Atlantic, Scientific American, Undark Magazine, Aeon Magazine, and STAT News and has been featured in The Best American Science and Nature Writing. She has done research in genomics and bioethics and is interested in real world consequences of cellular and gene therapies. She holds an MD from Harvard Medical School and a BS from Yale University.


 Speaker Profile

Ph.D., Chief Business Officer, Head of Therapeutics, Mammoth Biosciences

Biography
Peter builds on more than 20 years of pharma and biotech expertise to drive corporate strategy and partnering efforts in his current role. Mammoth is a private company, co-founded by Nobel Laureate Jennifer Doudna, and focuses on next generation CRISPR systems translating into therapeutic and diagnostic applications. Peter joined the company from Casebia Therapeutics which he co-founded as a Joint Venture between Bayer and CRISPR Therapeutics, and which is now fully owned by CRISPR Tx. Prior to Casebia, Peter has been at Bayer Healthcare in various positions in the US and Germany covering commercial, business development and licensing, project management, operations, and R&D within various indication fields. Peter completed postdoctoral studies at Stanford University and holds a Ph.D. in Organic Chemistry from the University of Marburg. He is a co-inventor of more than 45 patents including for the approved drug Letermovir.


 Speaker Profile

M.D., Chief Medical Officer, Beam Therapeutics

Biography
Amy Simon, M.D. is Chief Medical Officer of Beam Pharmaceuticals. Dr. Simon brings has over 20 years of clinical experience, serving in roles as a physician-scientist in academia and the biotechnology industry. Dr. Simon joined Beam in March 2021 from Alnylam Pharmaceuticals, where she spent over a decade in various roles with increasing responsibility for the clinical development of RNAi-based medicines, most recently serving as vice president, clinical development. During her tenure at Alnylam, she led the successful execution of clinical programs from natural history studies to Phase 1 through Phase 4 studies, regulatory interactions with both U.S. and ex-U.S. authorities, and drug approvals in the U.S. and EU. Dr. Simon was the lead clinician developing GIVLAARI® (givosiran) for patients with acute hepatic porphyria, which was approved by the Food and Drug Administration in 2019. Prior to entering the biotech industry, Dr. Simon worked in academia at Tufts University, serving as a professor and a director of the Asthma Center in the Pulmonary and Critical Care Division at Tufts University School of Medicine and as a professor at Tufts Graduate School of Biomedical Science where her laboratory conducted basic science research on asthma. She began her career in clinical practice, training as a resident in internal medicine and as a fellow in pulmonary and critical care medicine at Tufts Medical Center.  Dr. Simon holds a B.A. in history and science from Harvard University, and an M.D. from Tufts University School of Medicine.


 Speaker Profile

M.D., Ph.D., CEO, Poseida Therapeutics, Inc.

Biography
Eric Ostertag, M.D., Ph.D., is founder and CEO of Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure. A pioneer in cell and gene therapies, Dr. Ostertag was the first graduate of the Gene Therapy Program at the University of Pennsylvania School of Medicine. He received both his Ph.D. in molecular biology and his M.D. from the University of Pennsylvania School of Medicine and his B.S. in genetics from the University of Wisconsin-Madison. From there he went on to found and lead multiple biotechnology companies, including Poseida Therapeutics in 2015 and Transposagen Biopharmaceuticals, Inc., Poseida’s parent company and an early leader in the development of gene-engineering technologies. Dr. Ostertag is an inventor on more than 190 patents and patent applications, the author on numerous peer-reviewed publications, and the recipient of many scientific and clinical awards.


 Speaker Profile

M.D., Ph.D., Professor of Pediatrics, Stanford University School of Medicine

Biography
Matthew Porteus is the Sutardja Clark Professor of Definitive and Curative Medicine in the Department of Pediatrics and Institute of Stem Cell Biology and Regenerative Medicine at Stanford. He is the co-Director for the Stanford Center for Definitive and Curative Medicine (CDCM). His primary research focus is on developing genome editing as an approach to cure disease, particularly those of the blood and immune system (including sickle cell disease) and other organ systems. His goal is to combine his research and clinical interests to develop innovative curative therapies. His dream is to one day develop gene editing so that patients are cured of their disease before they or their parents even knew they had it. He served on the 2017 National Academy Study Committee of Human Genome Editing and currently serves on the Scientific Advisory Board for WADA on Cell and Gene Doping and the NIH NExTRAC advisory committee evaluating the emergence of new technologies.

Talk
Genome Editing of Stem Cells
Genome editing provides unprecedented precision in developing personalized medicines for patients. By engineering cells ex vivo with high precision, it may be possible to generate cures for both rare and common diseases. I will discuss our progress towards this ambitious goal.


 Speaker Profile

M.D., Chief Medical Officer, Ultragenyx Gene Therapy and Inborn Errors of Metabolism

Biography
Dr. Crombez joined Ultragenyx following the acquisition of Dimension Therapeutics, a liver directed AAV gene therapy company. During his tenure at both companies, Dr. Crombez led the clinical development efforts for four clinical stage programs including gene therapy programs for hemophilia B, hemophilia A (in partnership with Bayer), ornithine transcarbamylase (OTC) deficiency and glycogen storage disorder type Ia (GSDIa). Dr. Crombez is also serving as an Industry Representative on the FDA Cellular, Tissue, and Gene Therapies Advisory Committee. Prior to this work in gene therapy, Dr. Crombez worked at Shire in their Human Genetics Therapy business unit for nearly eight years. Prior to joining industry, Dr. Crombez was Assistant Professor, Department of Pediatrics, Division of Medical Genetics at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA).


 Speaker Profile

M.D., Ph.D., Chief Scientific Officer, 4D Molecular Therapeutics

Biography
Peter Francis, M.D., Ph.D., served as our Chief Medical Officer January 2019 through February 2020 before becoming our Chief Scientific Officer. Dr. Francis previously served as our Senior Vice President, Clinical Translational R&D, and Retina Therapeutic Area Head from August 2018 to January 2019. Dr. Francis previously served as Chief Medical Officer at RetroSense Therapeutics from February 2012 until August 2016 when it was purchased by Allergan Inc. Dr. Francis practices as a physician at Orion Eye Center. Dr. Francis received his B.Sc. in Molecular Cell Biology from the University of Southampton, England in 1991. He earned his M.D. from the University of Southampton, England in 1992. He earned his Ph.D. in ophthalmic genetics from University College, London in 2000.


 Speaker Profile

Ph.D., Co-Founder and Chief Scientific Officer, Cardea Bio

Biography
Dr. Kiana Aran is the Co-Founder and Chief Scientific Officer at Cardea Bio, an Associate Professor of Medical Diagnostics and Therapeutics at Keck Graduate Institute, a member of the Claremont Colleges, and a visiting Assistant Professor at the University of California, Berkeley. Dr. Aran also serves as a Consultant of Drug Delivery and Medical Diagnostics for the Bill & Melinda Gates Foundation. She received her undergraduate degree in electrical engineering from the City University of New York in 2007 and her Ph.D. in biomedical engineering from Rutgers University in 2012. She then continued her postdoctoral studies in bioengineering at the University of California Berkeley and was a recipient of the National Institutes of Health (NIH) postdoctoral training fellowship at the Buck Institute for Aging Research in 2015. Her research efforts focus on designing novel biosensing platforms, using 2D nanomaterials, for early disease diagnosis as well as utilizing biology as tech elements for a variety of biosensing applications. In addition to biosensing, she combines various engineering modules to develop tools to better understand the process of aging. Dr. Aran’s scientific vision is to explore the utility of nano-electronic systems to develop transformative and customizable platforms for multiomics applications and commercialization of these platform. Her efforts have been recognized within the scientific community by the Clinical OMICs 10 under 40 Award, Athena Pinnacle Award, and Nature Research Awards for Inspiring Women in Science: Scientific Achievement Category’s Overall Winner. Dr. Aran was also the recipient of the NSF Career Award to develop the next generation of electronic sensors.


 Speaker Profile

Ph.D., Co-Founder and Vice President, Forge Biologics

Biography
Dr. De Silva is an experienced scientist and entrepreneur, with more than a decade of experience in discovery research, therapeutic development, and partnering across oncology, infectious disease, and gene therapy. Prior to founding Forge, she held R&D and leadership roles in both academic and industry settings, including at Genentech, Ohio State University and Myonexus Therapeutics.


 Speaker Profile

Ph.D., CSO, EVP, Intellia Therapeutics

Biography
Laura Sepp-Lorenzino, Ph.D. leads Research and Early Development at Intellia Therapeutics. Before joining Intellia, Laura was at Vertex, Alnylam, Merck and Memorial Sloan-Kettering. She received her professional degree in Biochemistry from the University of Buenos Aires, Argentina and both her M.S. and Ph.D. in Biochemistry from New York University. Laura is a member of the BOD of Taysha Gene Therapies, the Alliance for Regenerative Medicine and the Oligonucleotide Therapeutics Society, and sits on the SABs of Thermo Fisher Scientific, Zymergen, and the U.K. Nucleic Acid Therapy Accelerator.

Talk
Advances in Therapeutic CRISPR/Cas9 Genome Editing
Intellia Therapeutics is a leading clinical-stage genome editing company, developing novel, potentially curative therapeutics using CRISPR/Cas9 technology. Highlights from our clinical programs in vivo and ex vivo will be presented, including the first demonstration of CRISPR knockout in humans.


 Speaker Profile

Ph.D., Chief Scientific Officer, Q2 Solutions

Biography
Dr. Patrice Hugo is responsible for the medical affairs and scientific activities for the Central Laboratories, and Specialty Testing Centers of Excellence worldwide including the Expression Analysis Genomics, Vaccine, Biomarkers Translational Science and Innovation Laboratory and BioAnalytical/ADME laboratories. Prior to joining Q² Solutions, Dr. Hugo was Associate Vice President and Chief Clinical Trial Scientist at LabCorp/Covance Central Lab Division and Chief Scientific Officer at Clearstone Central Lab acquired by LabCorp. Previously, he worked at Caprion (now CellCarta) as Executive Vice President R&D, and Principal Investigator at the Montreal Clinical Research Institute with academic affiliation at McGill University and University of Montreal. Dr. Hugo obtained his Ph.D. in Experimental Medicine & Immunology at McGill University and held post-doctoral positions at the Walter and Eliza Hall Institute, Australia, and the Howard Hughes Medical Institute, USA. He is author or co-author of over 75 scientific manuscripts and has over 25 years of biomarker experience.


 Speaker Profile

Professor, MCB Department, UC Berkeley

Biography
TBA


 Speaker Profile

M.D., President and CEO, The Jackson Labs

Biography
Edison T. Liu, M.D. is a global leader in translational genomic medicine who has been at the helm of research institutions in US academia, the US National Cancer Institute, and in Asia. He propelled Singapore’s genomics efforts onto the international stage, helping that country become a regional powerhouse in biomedical sciences. In the last ten years, Dr. Liu guided The Jackson Laboratory to a doubling of its size and revenues, and expanded its footprint from North America into China and Japan. As an active physician scientist, his research is centered on the complex genetics of human cancers with a special focus on the genomic biology of breast cancer. Dr. Liu has published over 320 papers, reviews, and books, and is a recipient of numerous national and international awards. He is an advisor to many institutions across the world seeking to establish a competitive position in the global biomedical sciences.