Session Abstract – PMWC 2022 Silicon Valley


Track Chair:
Vanessa Soros, Graphite Bio

Sessions:

  • Gene & Cell Therapies Overview
    - Janice Chen, Mammoth
  • Cutting Edge Technologies for Gene Engineering and Cell Therapy (PANEL)
    Session Chair: Vanessa Soros, Graphite Bio
    - Mark McKee, 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
    - Peter Francis, 4D Molecular Therapeutics
    - Erandi De Silva, Forge Biologics
    - Patrice Hugo, Q2 Solutions
    - Susanne Fors, Ultragenyx
  • PMWC Showcase
    - Yuri Fesko, Labcorp
    - Yan Zhang, Mission Bio
    - Anil Narasimha, Mekonos Inc.
    - Emily Anderson, Horizon, a PerkinElmer Company
    - Jesse Salk, Twinstrand
    - William Shrader, AcureX Therapeutics
  • Emerging Gene Therapies for Epidermolysis Bullosa
    - Peter Marinkovich, Stanford
  • 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
    - Kiana Aran, Cardea Bio
    - Mark McKee, Intellia Tx
    - Josh Lehrer, Graphite Bio

 Session Chair Profile

Ph.D., Assistant Professor, UCSF

Biography
Dr. Paulk is a pioneer in the development of next-generation AAV platforms for gene repair, gene transfer and gene editing for numerous rare diseases and cancer, directed evolution to evolve novel viral capsids, and comparative multiomic approaches to interrogate translational AAV biology. Dr. Paulk is a global KOL in gene therapy and consults extensively for big pharma, and sits on the SABs for Sarepta, Astellas, Dyno, CEVEC, Excision Bio, Whitelab Genomics, and the John's Hopkins Gene Therapy Initiative. She is regularly quoted in STAT, Endpoints, Phacilitate, GEN, Biopharma Dive, Wall Street Journal, and is a frequent guest on Biotech Clubhouse. Dr. Paulk sits on the Scientific Editorial Boards of the journals Nature Gene Therapy and Human Gene Therapy, and is extensively involved in numerous committees within the American Society of Gene & Cell Therapy.


 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

Ph.D., VP, Research and Early Development, Graphite Bio

Biography
Dr. Vanessa Soros is VP of Research and Early Development of Graphite Bio, a gene editing and cell therapy company, leading discovery research and development efforts. Prior to Graphite Bio, Dr. Soros was Sr. Director of Antibody Development and Engineering at Tizona Therapeutics, where her work led to several first-in-class antibody therapeutics to novel immune-oncology targets. Prior to Tizona, Vanessa was a lead scientist in the development of discovery platforms and screening systems for novel biologics at both Catalyst Biosciences and Sea Lane Biotechnologies. Vanessa obtained her Ph.D. from the University of Toronto in Molecular Biology and Medical Genetics and pursued her postdoctoral studies at the Gladstone Institute of Virology and Immunology, at the University of California, San Francisco.


 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., Executive Chairman, Poseida Therapeutics

Biography
Eric Ostertag, M.D., Ph.D., is founder and Executive Chairman of Poseida Therapeutics, Inc., 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 served as Poseida’s CEO for seven years before transitioning to the role of Executive Chairman in February 2022.


 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.


 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

PHD, Co-Founder and SVP, 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 RD and leadership roles in both academic and industry settings, including at Genentech, Ohio State University and Myonexus Therapeutics.


 Speaker Profile

M.D., Ph.D., VP, Clinical Development, Intellia Therapeutics

Biography
Mark McKee, M.D leads the medical sciences group within Clinical Development at Intellia therapeutics. In addition, he has served as the head of program teams for several of Intellia’s lead assets during his tenure at the company. Prior to joining Intellia, Mark was in the Oncology Clinical Development group at AbbVie and was a physician and researcher at the University of Chicago. He received his Bachelor of Science and Medical Degrees from Northwestern University and completed his clinical training at Harvard Beth Israel Deaconess medical Center and the National Institutes of Health.


 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

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

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.

Talk
Single-cell Biology to Accelerate Discovery, Development, Delivery of Prec. Med


 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.

Talk
Ex-vivo Silicon-based Delivery of Macromolecules into Cells
At Mekonos, we are developing a platform for delivery of payloads into cells in a precise, efficient, and automated manner. We take advantage of scalable silicon technology to create a method that is advantageous for complex gene editing, targeted delivery, and dose control at the single cell level.


 Speaker Profile

Ph.D., Principal Scientist, Horizon, PerkinElmer

Biography
Emily has managed projects involving the development and application of gene engineering and gene modulation technologies including Dharmacon™ ON-TARGETplus™ siRNA and Accell™ siRNA, as well as the introduction of Dharmacon™ Edit-R™, the first rationally-designed, genomewide library of synthetic CRISPRs for gene disruption. She received a Ph.D. in Biochemistry from the University of Colorado at Boulder on the structure and function of protein:single-stranded DNA interactions at telomeres. She has worked extensively on the mechanism, function, specificity, and delivery of RNAi as well as its applications. She is an inventor on many issued patents and has authored peer-reviewed publications in the fields of both RNAi and CRISPR.

Talk
Pin-point™ – Modular Base Editing for Clinical Applications
Pin-point™ uses aptameric guide RNA for deaminase recruitment. We demonstrate in CAR-T cells multitargeting of several therapeutically relevant loci, retaining proliferative and cytotoxic ability, including advantages over Cas9. Aptameric recruitment enables simultaneous knock-in and knockout and allows targeting polygenic disorders.


 Speaker Profile

M.D., Associate Professor, Stanford University

Biography
Dr. Marinkovich received his dermatology training at Oregon Health Sciences University, and his research training in the laboratory of Dr. Robert Burgeson, who discovered type VII collagen. Dr. Marinkovich's early work led to the characterization of several key basement membrane components involved in epidermolysis bullosa, including laminin-332 and laminin-311. He later joined the faculty at Stanford University and served as an attending in the National EB Registry at Stanford University. He currently directs the Stanford Blistering Disease Clinic. Dr. Marinkovich’s laboratory has had a longstanding focus on the development of molecular therapy for various subtypes of epidermolysis bullosa.

Talk
Emerging Gene Therapies for Epidermolysis Bullosa
Dystrophic epidermolysis bullosa is a skin fragility disorder characterized by blistering in response to minor trauma. Advances in our understanding of epidermolysis bullosa pathophysiology and gene therapy vectors have led to successive advancements in dystrophic epidermolysis bullosa molecular correction, demonstrating that gene therapy for epidermolysis bullosa is safe and effective.


 Speaker Profile

M.D., Ph.D., CEO, Twinstrand Biosciences

Biography
Dr. Jesse Salk is a medical oncologist and a Co-Founder of TwinStrand Biosciences. His research interests lie in studying somatic evolution and the genomic processes that occur on the continuum between “normal”, preneoplastic and cancer, and applying this to knowledge to clinical medicine. He developed the ultra-high accuracy Duplex Sequencing technology with colleagues at the University of Washington, where he completed his medical and graduate degrees as well as clinical training. Since 2017, Dr. Salk has led TwinStrand, applying Duplex Sequencing to new areas of high sensitivity early and residual cancer detection, evolution of drug resistance and genetic toxicology, among other fields. He holds a clinical faculty appointment at the University of Washington and continues to care for cancer patients part time at the VA Puget Sound in Seattle.


Clinical Dx showcase:
Twinstrand Biosciences

TwinStrand develops ultrasensitive Duplex Sequencing-based assays for research and clinical applications in cancer, cancer therapeutics and other areas of basic science and clinical medicine.

Duplex Sequencing for Gene Editing and Cell Therapy Applications
Duplex Sequencing enables an unprecedented level of NGS accuracy, with error rates below one-in-ten-million. The technology is ideally suited to detection of exceptionally low frequency DNA variants. Here we present Duplex Sequencing for cutting-edge gene editing and cellular therapy applications.

 Speaker Profile

Ph.D., CEO/CSO, AcureX Therapeutics

Biography
Prior to AcureX, Bill ran research and development at Edison Pharmaceuticals where he and his team advanced three drugs into the clinic for ALS, Parkinson’s disease and orphan neurodegenerative diseases. Of note, EPI-589 is partnered with Sunovion/Sumitomo and is in phase 2b clinical trials for ALS and Parkinson’s disease. And Vatiquinone™ was acquired by PTC Therapeutics and is in pivotal approval trials for pediatric seizure and fatal pediatric neurodegenerative diseases. Earlier at Celera Genomics, Dr. Shrader invented, advanced and partnered with AbbVie, the tissue factor/factor VIIa inhibitor (PCI-27483) for pancreatic cancer. Dr Shrader holds a Ph.D. in organic chemistry from the University of California, Berkeley and was on faculty at the California Institute of Technology as a NIH postdoctoral fellow. Dr. Shrader has authored 30 scientific publications and an inventor on 21 issued US patents.


Emerging Therapeutics Showcase:
AcureX Therapeutics

AcureX Therapeutics mission is to halt major neurodegenerative diseases driven by impaired mitophagy, starting with Parkinson’s disease and FTD-Tau.

Leveraging Miro1 to Halt Neurodegeneration
AcureX Therapeutics mission is to halt major neurodegenerative diseases driven by impaired mitophagy.