Session Abstract – PMWC 2022 Silicon Valley


Track Chair:
Jane Grogan, Graphite Bio

Sessions:

  • Advances in Gene & Cell Therapies
    Session Chair: Jane Grogan, Graphite Bio
    - Peter Nell, Mammoth
    - Laura Sepp-Lorenzino, Intellia Tx
    - Amy Simon, Beam Therapeutics
    - Eric Ostertag, Poseida Therapuetics
  • Translating CGT to the Clinic (PANEL)
    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
    - Fyodor Urnov, UC Berkeley
    - Sandy Macrae, Sangamo Therapeutics
  • PMWC Showcase
    - Yuri Fesko, Labcorp
    - Yan Zhang, Mission Bio
    - Anil Narasimha, Mekonos Inc.
  • Past, Present and Future of CRISPR Where will CRISPR Take Us and How is the Technology Evolving to Realize its Full Potential (PANEL)
    Session Chair: Janice Chen, Mammoth
    - Fyodor Urnov, UC Berkeley
    - Kiana Aran, Cardea Bio
    - Laura Sepp-Lorenzino, Intellia Tx
    - Josh Lehrer, Graphite Bio

 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 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

Ph.D., Professor, Molecular and Cell Biology Department, UC Berkeley

Biography
In his work at Sangamo Therapeutics (2000-16), Fyodor co-developed and co-named human genome editing at native loci with engineered nucleases, and co-led efforts to develop its fundamental toolbox (gene correction, knockout, and integration). Fyodor then led collaborative teams to establish at-scale applications of genome editing for human somatic cell genetics and model animal and crop reverse genetics. Fyodor was a key member of the team that developed the first-in-human application of genome editing (2009), and then led a cross-functional team through basic discovery and to IND of the first-in-human clinical trials for the hemoglobinopathies, beta-thalassemia and sickle cell disease targeting the erythroid enhancer of BCL11A (as of Nov 2021 with 4 subjects free of disease symptoms post-dosing, and 22 subjects major-symptom-free on the follow-up clinical trial for the same target using Cas9). Fyodor co-led efforts to develop the fundamental toolbox of human epigenome editing as a disease therapeutic (2000-2003), and then co-led a team that developed in vivo epigenome editors for Hungington's disease and Tau dementia (2010-2016). At the IGI, Fyodor’s focus is on establishing turnkey editability of the human genome and epigenome for clinical use, and building and leading collaborative teams to first-in-human applications of experimental CRISPR-based editing therapeutics for sickle cell disease and genetic disorders of the immune system, as well as epigenome editing therapeutics for radiation injury, neuroinflammation, and neurodegeneration. Fyodor is an author on 98 scientific publications and a named inventor on 75 issued US patents related to genome editing and targeted gene regulation technology.


 Speaker Profile

M.D., Executive Medical Director, Quest Diagnostics

Biography
Yuri Fesko, M.D., is Cheif Medical Office of Oncology at Quest Diagnostics. As a leading oncologist, Dr Fesko plays a key role in helping Quest advance the field of precision medicine and give new hope to people with cancer. Prior to joining Quest in 2017, Dr Fesko was Director of Medical Oncology at the Duke Cancer Institute in Raleigh, North Carolina, where he was responsible for the oncology service line and development of inpatient oncology, multiple network sites and infusion centers. He also was responsible for strategies including the determination of appropriate clinical trials for population and resource utilization. Dr Fesko received his bachelor’s degree and medical degree from Case Western Reserve University School of Medicine. He is certified by the American Board of Internal Medicine in hematology and medical oncology.


Emerging Therapeutics Showcase:
Quest Diagnostics

Quest Diagnostics is the Largest Reference laboratory with products and services that benefit patients, health care providers, Pharmaceutical companies , Life insurance companies and employers. Quest Diagnostics empowers people to take action to improve health outcomes.

 Speaker Profile

M.D., M.Phil., CEO, Graphite Bio

Biography
Dr. Josh Lehrer is CEO of Graphite Bio, where he bridges his clinical background as a practicing physician with decades of experience at global biopharmaceutical companies leading businesses and high-functioning teams across all stages of drug development. Before joining Graphite Bio, he was CMO of Global Blood Therapeutics, served in leadership roles at Genentech in clinical development and business development, and held attending physician roles at Stanford University Medical Center and the Palo Alto Veteran’s Affairs Health System. Dr. Lehrer earned his Doctor of Medicine at the UCSF School of Medicine and completed his residency at UCSF in internal medicine. He holds an A.B. in Biochemical Sciences from Harvard University and a Master of Philosophy in Biological Sciences from the University of Cambridge. Dr. Lehrer served as a clinical and postdoctoral fellow in cardiovascular medicine at Stanford University and attended the Institute for Entrepreneurship at the Stanford Graduate School of Business.


 Speaker Profile

M.D., Ph.D., CEO, Sangamo Therapeutics

Biography
Sandy Macrae, M.B., Ch.B., Ph.D., has served as Sangamo’s President and Chief Executive Officer and as a member of the Board of Directors since June 2016. He has twenty years of experience in the pharmaceutical industry including Global Medical Officer of Takeda Pharmaceuticals, from 2012 to March 2016, where he established and led the Global Medical Office, which encompasses medical affairs, regulatory affairs, pharmacovigilance, outcomes research and epidemiology, quantitative sciences and knowledge and informatics. From 2001 to 2012, Dr. Macrae held roles of increasing responsibility at GlaxoSmithKline, including Senior Vice President, Emerging Markets Research and Development (R&D), from 2009 to 2012. From 2007 to 2008, he was Vice President, Business Development. Earlier in his career, he worked for SmithKline Beecham, where he was responsible for clinical development in the therapeutic areas of neurology and gastroenterology.


 Speaker Profile

Ph.D., CEO, Mission Bio

Biography
As a veteran of the genomics industry, Yan is passionate about empowering teams to deliver highly differentiated innovative solutions realizing the mission of accelerating their application to improve human health. Before Mission Bio, Yan served executive roles at Thermo Fisher Scientific, where she led commercial teams in China for rapid market success and general management roles for Reproductive Health and Microarray businesses. She also led product management and commercialization efforts for genetic analysis solutions at Affymetrix, NuGEN Technologies, and Molecular Devices.


 Speaker Profile

Ph.D., CEO, Mekonos

Biography
Dr. Anil Narasimha is an accomplished scientist and founder in the field of personalized medicine. He co-founded Mekonos in 2017 to come up with a platform to automate delivery of payloads into fragile cell types while remaining precise and scalable. Prior to Mekonos, Anil completed his PhD at UC San Diego before moving to Stanford University to become a postdoctoral fellow in Dr. Michael Snyder's laboratory. There, he was introduced to cell and gene therapy and identified some of the pitfalls associated with it, including the lack of automation and precision when it came to delivering payloads into primary cells. His goal is to utilize his platform to make cell and gene therapies more accessible.