Derek Jantz, PhD, is the co-founder and CSO of Precision BioSciences and a 15-year veteran of the genome editing industry. As a protein engineer, he was an early developer of zinc finger technology and has spent most of his career designing proteins for genome editing applications. He performed his graduate studies in biophysics at Johns Hopkins University School of Medicine. Dr. Jantz co-founded Precision BioSciences in 2006 after co-inventing a novel method to modify the DNA-recognition properties of meganucleases. Read his full bio.

Interview with Derek Jantz from Precision BioSciences

Q: What need is Precision BioSciences addressing?

A: Precision’s ARCUS genome editing technology is a proprietary, non-CRIPSR platform that has applications across cell therapy, gene therapy, and agriculture. We are able to edit everything from plants to primates, so prioritizing projects is an imporant part of what we do. In agriculture, we are focused on enhancing nutritional value and fortifying at-risk crops. In medicine, we are developing ex vivo gene edited cell therapies that fight cancer as well as in vivo gene editing therapies targeted to the liver and the eye.

Q: What are the products and/or services Precision BioSciences offers/develops to address this need? What makes Precision BioSciences unique?

A: Our lead product is an off-the-shelf CD19 CAR T to treat acute lymphoblastic leukemia (ALL) and non-Hodgkin lymphoma (NHL). This is made from the T cells of healthy donors, which we edit in two ways – first, we add a receptor that targets cancerous cells and second, we knock out a receptor that mediates graph-versus-host interactions to eliminate the need for donor-patient matching. Using this method, we can generate hundreds of vials of CAR T cells from a single manufacturing run to treat patients when and where they need it. Another even newer area of genetic medicine we are excited about is in vivo therapeutic genome editing. Because the ARCUS editing platform works effectively and reproducibly in non-human primates and other large animal models, we can look beyond basic mouse studies and begin using editing to therapeutically suppress or correct expression in models that inform the development of human therapeutics.

Q: What is your role at Precision BioSciences and what excites you about your work?

A: I am the CSO of Precision BioSciences and also a co-founder, along with Matt Kane (our CEO) and Jeff Smith (our CTO). We started the company back in 2006, soon after Jeff and I created the earliest version of ARCUS, but I’d already been working in genome editing for almost 10 years by then. I have studied and worked with zinc finger nucleases, TALENs, and CRISPRs, but have always preferred the homing endonuclease (aka meganuclease), which ARCUS is based on, for therapeutic applications. The time and effort our team at Precision has put into this platform has been nothing short of heroic, and now we are seeing the pay off as we progress toward clinic in both cell and gene therapy.

Q: When thinking about Precision BioSciences and the domain Precision BioSciences is working in, what are some of the recent breakthroughs that are propelling the field forward and how will they impact healthcare?

A: The field has made real headway into editing human cells in vitro, particularly for CAR T. Several new gene edited CAR T therapies are either in the clinic or expected to enter the clinic within the next year. In vivo therapeutic editing isn’t quite as far along but there are a couple of ZFN-based therapies in very early clinical studies. We recently published a study in collaboration with the Wilson lab at Penn demonstrating, I think for the first time, high-efficiency, long-term in vivo editing in primate liver. We knocked-out the PCSK9 gene in several animals, resulting in therapeutically-relevant reductions in LDL cholesterol that are stable now well into the second year of the study. We think the study provides a blueprint for developing additional liver-targeted in vivo editing therapies using the ARCUS platform.

Q: What are the short-term challenges that Precision BioSciences and its peers are facing?

A: For the field at large, translating our success from the bench to the bedside has been challenging. Gene editing has revolutionized biomedical research, but creating bona fide in vivo therapies is a big ask and the place everyone in this space would like to go. This means creating new medicines able to cure disease by editing patient DNA at the organ or tissue of interest, preferably with a single treatment. It’s the “holy grail” of genome editing and what I have been working toward my whole career. We’re close but we aren’t there yet. The transition to large animal models – getting beyond mouse work – is a crucial step toward bringing these therapies to the clinic. And as the first wave of ex vivo edited cell therapies enter clinical trials we will gain a greater understanding of what safety risks, if any, are posed by gene editing. This, in turn, should help to clarify the regulatory path for the subsequent wave of in vivo therapeutics.

Q: Is there anything else you would like to share with the PMWC audience?

A: How soon and how completely genome editing will reshape the future of medicine are still big questions, but I am more and more confident this will happen sooner and with a bigger impact than many appreciate. As a scientist and as a person, I could not be happier to have made this my life’s work.

Interview with Daniel Chen from IGM Biosciences

Q: Checkpoint inhibitors, particularly with PD-L1/PD-1 targeting agents, have benefited a broad range of patients with cancer. How will we improve on this?

A: It’s true that PD-L1/PD-1 inhibitors have led to durable responses in a subset of patients, and survival benefit in many of the patients treated- either as monotherapy or combination.

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Call from PMWC 2019 Silicon Valley Program Committee – We Must Accelerate and Deliver on the Promise of Precision Medicine

Precision medicine advancements are real as demonstrated by the high volume of molecular, “precise” drugs on the market, which are based on extensive molecular and translational understanding of the specific drug targets.

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#AI Play in Patient Diagnosis? How Can We Prepare the Next Generation to Make Sense of Enormous Amounts of Health-related Data?

What role should artificial intelligence play in patient diagnosis? How can we best prepare the next generation to make sense of enormous amounts of health-related data? These were just a few of the questions explored at the 15th Precision Medicine World Conference held at Duke University September 24-25, 2018.

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Interview with Daniella Beller, Manager, Maccabi Research Institute Biobank

Q: What makes the Maccabi Research Institute biobank unique?

A: To explain the uniqueness of the Maccabi Biobank (named “Tipa” in Hebrew which means “drop” or “just a little”), first you must know a little about Maccabi.

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Why We Need Public-Private Partnerships (PPP) To Foster Drug Discovery

Investments in pharma R&D has substantially increased over the last decades. Yet there appears to be no clear correlation to the number of newly approved drugs. This fact is accompanied by ever-increasing healthcare costs, fueled by an aging population and the parallel rise in the chronic disease burden.

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Precision Medicine: A Decade of Improving the Standard of Care

In January, PMWC will host its 2019 Silicon Valley event, the largest Precision Medicine conference in the world with over 2,500 attendees gathering at the Santa Clara Convention Center. We are humbled and honored to have reached this stage of growth and are looking forward to continuing our work with key stakeholders and decision makers across the industry to ever strengthen this forum for exchange of critical and timely topics, to move the field of precision medicine forward and to improve the Standard of Care.

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Interview with David Hong from Karius

Q: What need is Karius addressing?

A: Physicians often have difficulty pinpointing the exact pathogen that is causing disease. Conventional diagnostics like blood cultures or PCR can have poor sensitivity due to pretreatment with antibiotics, the breadth of potential pathogens present, and the requirement for invasive procedures to access deep-seated infections.

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Interview with Charles Jaffe, MD, PhD from HL7

Q: What need is HL7 addressing?

A: For more than three decades, HL7 has provided the platform to enable global health data interoperability. This is more important than ever, as the cost of healthcare has increased exponentially, and the complexity of clinical evidence has grown to an almost unmanageable state.

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Interview with Eden Haverfield from Invitae

Q: What need is Invitae addressing?

A: Our mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. Invitae is uniquely positioned to answer some of life’s most serious and complex questions with the highest quality genetics and at an affordable price.

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Call by Ralph Snyderman (Duke U.) for Big Changes to Get to the Next Level of Precision Medicine

Beyond next-generation gene sequencing and developing diagnostic tools and targeted therapies, theoverall approach to clinical care has to be re-envisioned to fulfill the promise of precision medicine. Care must move from sporadic treatment of episodic disease (a reactive mode) to predicting disease and then acting to prevent and mitigate it (a proactive mode).

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Interview with William Hearl From Immunomic Therapeutics, Inc.

Q: What need is Immunomic Therapeutics, Inc. addressing?

A: Immunomic Therapeutics’ nucleic acid vaccines have the potential to utilize the body’s natural biochemistry to develop a broad immune response, including antibody production, cytokine release and critical immunological memory.

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Interview with Ralph Snyderman from Duke University

Q: What are some of the critical contributions that academic institutions and medical centers are making to implement and accelerate precision medicine?

A: Over the past decade, the field of precision medicine has created technologies enabling far more personalized and effective health care delivery. Many of the most dramatic advances have come in the field of oncology but targeting care to the needs of the individual is rapidly achieving broader applications.

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Interview with Catherine Reinis Lucey from UCSF

Q: What research are you or your lab focusing on and why, and what problem(s) are you trying to solve?

A: My work involves designing, implementing and studying innovations in medical education that allow our medical schools to fulfill our social contract to improve the health of our communities and reduce the suffering of our patients.

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Interview with Gunnar Carlsson from Ayasdi

Q: What need is Ayasdi addressing?

A: Ayasdi is pioneering the application of artificial intelligence to value-based care by targeting two of the most complex problems in healthcare: population risk stratification and clinical variation management.

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Interview with Nikole Kimes from Siolta Therapeutics

Q: What need is Siolta Therapeutics addressing?

A: Chronic diseases, including inflammatory diseases such as asthma, now represent the leading cause of mortality and morbidity worldwide.

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Johns Hopkins
University Of Michigan

The Precision Medicine World Conference (PMWC), in its 16th installment, will take place in the Santa Clara Convention Center (Silicon Valley) on January 20-23, 2019. The program will traverse innovative technologies, thriving initiatives, and clinical case studies that enable the translation of precision medicine into direct improvements in health care. Conference attendees will have an opportunity to learn first-hand about the latest developments and advancements in precision medicine and cutting-edge new strategies and solutions that are changing how patients are treated.

Agenda highlights:

  • Five tracks will showcase sessions on the latest advancements in precision medicine which include, but are not limited to:
    • AI & Data Science Showcase
    • Clinical & Research Tools Showcase
    • Clinical Dx Showcase
    • Creating Clinical Value with Liquid Biopsy ctDNA, etc.
    • Digital Health/Health and Wellness
    • Digital Phenotyping
    • Diversity in Precision Medicine
    • Drug Development (PPPs)
    • Early Days of Life Sequencing
    • Emerging Technologies in PM
    • Emerging Therapeutic Showcase
    • FDA Efforts to Accelerate PM
    • Gene Editing
    • Genomic Profiling Showcase
    • Immunotherapy Sessions & Showcase
    • Implementation into Health Care Delivery
    • Large Scale Bio-data Resources to Support Drug Development (PPPs)
    • Microbial Profiling Showcase
    • Microbiome
    • Neoantigens
    • Next-Gen. Workforce of PM
    • Non-Clinical Services Showcase
    • Pharmacogenomics
    • Point-of Care Dx Platform
    • Precision Public Health
    • Rare Disease Diagnosis
    • Resilience
    • Robust Clinical Decision Support Tools
    • Wellness and Aging Showcase

Agenda highlights:

    • Five tracks will showcase sessions on the latest advancements in precision medicine which include, but are not limited to:
      • AI & Data Science Showcase
      • Clinical & Research Tools Showcase
      • Clinical Dx Showcase
      • Creating Clinical Value with Liquid Biopsy ctDNA, etc.
      • Digital Health/Health and Wellness
      • Digital Phenotyping
      • Diversity in Precision Medicine
      • Drug Development (PPPs)
      • Early Days of Life Sequencing
      • Emerging Technologies in PM
      • Emerging Therapeutic Showcase
      • FDA Efforts to Accelerate PM
      • Gene Editing / CRISPR
      • Genomic Profiling Showcase
      • Immunotherapy Sessions & Showcase
      • Implementation into Health Care Delivery
      • Large Scale Bio-data Resources to Support Drug Development (PPPs)
      • Microbial Profiling Showcase
      • Microbiome
      • Neoantigens
      • Next-Gen. Workforce of PM
      • Non-Clinical Services Showcase
      • Pharmacogenomics
      • Point-of Care Dx Platform
      • Precision Public Health
      • Rare Disease Diagnosis
      • Resilience
      • Robust Clinical Decision Support Tools
      • Wellness and Aging Showcase
  • Luminary and Pioneer Awards, honoring individuals who contributed, and continue to contribute, to the field of Precision Medicine
  • 2000+ multidisciplinary attendees, from across the entire spectrum of healthcare, representing different types of companies, technologies, and medical centers with leadership roles in precision medicine
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