Q: What are the benefits of the Precision BioSciences’ ARCUS genome editing platform and what makes it unique?

A: ARCUS is a next-generation genome editing platform derived from a class of naturally occurring enzymes called homing endonucleases. In fact, you can think of homing endonucleases as nature’s genome editing system. The key advantages of ARCUS include exquisite control over off-target modifications, ease of delivery to the intended tissue or cell type, and greater efficiency for gene repair or insertions. Put simply – safety and efficacy. This novel nuclease architecture required over nine years and $20 million to develop but today gives us what, I believe, is the first therapeutic-grade genome editing platform and the only that has both clear freedom to operate and the ability to fully control its use.

Q: How Precision BioSciences applies the ARCUS genome editing technology to cancer immunotherapy?

A: To develop cancer immunotherapies, our team uses ARCUS to re-program human immune cells to become highly targeted, cancer-eliminating, cellular machines. This is part of a class of therapies broadly known as Chimeric Antigen Receptor T cells (CAR Ts). Precision’s first CAR T program is, in many ways, a next generation version of the recently approved CD19 targeting CAR Ts, Kymriah and Yescarta, now being used to treat certain types of acute lymphoblastic leukemia (ALL) and non-Hodgkin lymphoma (NHL). While representing significant steps towards an effective solution for ALL and NHL, these first generation treatments are limited by the scope of patients they can address and the product’s ability to efficiently scale in a manner that will reach the patients in greatest need. Precision’s 2nd generation approach uses ARCUS genome editing to enhance and arm qualified, healthy donor immune cells – rather than cells taken from cancer patients – to create a highly scalable, off-the-shelf product that could greatly improve and expand upon the effectiveness and safety of the first generation class of products. We anticipate seeking approval for our first human clinical trial to test this method in the second half of 2018.

Q: What are some of the clinical applications that your approach for gene therapy has the potential to address the unmet needs of patients and families dealing with devastating and, as yet, untreatable diseases?

A: Today, our gene therapy pipeline focuses on diseases that may be mediated by editing in either the liver or the eye. This includes potential cures for diseases such as Hepatitis B, Hemophilia A, Familial Hypercholesterolemia (think extremely high levels of bad cholesterol), and a form of Retinitis Pigmentosa, a disorder that leads to blindness. I am incredibly excited about the promise of genome editing for patients and families dealing with devastating genetic diseases; this is something I can say that every member of the team at Precision cares deeply about. However, we also understand the challenges that in vivo genome editing presents beyond that of ex vivo cell therapies. We can’t simply treat cells in the lab and then analyze and sort them before administering to the patient. We have to get it right the first time, every time. But, as a company dedicated to improving life, these are the challenges that we both embrace and are uniquely qualified to succeed in.

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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Note from Dr. Patrick Conway, President & CEO, Blue Cross and Blue Shield NC to PMWC

VC activity is booming in healthcare, with second quarter drawing in $5.1 billion in capital. That is 22% of the total $23 billion raised by all VC-based companies in the U.S., according to latest Pricewaterhouse Coopers quarterly report.

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Interview with Atul Sharan, Co-founder and CEO, CellMax Life

Q: CellMax has developed non-invasive blood tests based on Circulating tumor cells CTC. How do you see these tests being adopted clinically?

A: Survival rates are greater than 90% for cancers that are detected at an early stage.

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Interview with Wendell Jones from Q² Solutions | EA Genomics

Q: What need is Q² Solutions | EA Genomics addressing?

A: As a leading provider of genomic services in clinical trials and discovery, Q² Solutions | EA Genomics advances science by harnessing technological expertise to drive understanding of the human genome and disease biology to detect the effects of therapies.

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University of Michigan

The Precision Medicine World Conference (PMWC), held annually in Silicon Valley, is coming back to North Carolina for its 2nd Conference back at Duke University on September 24-25, 2018.

PMWC 2018 Duke, the 15th installment of the conference, will spotlight the explosion of biomedical technologies, driving initiatives that enable the translation of precision medicine into direct improvements in health care.

 

  • Track 1 will showcase sessions on the latest advancements in precision medicine which include, but are not limited to:
    • Digital Phenotyping
    • Precision Public Health
    • Pharmacogenomics
    • The Microbiome
    • Rare Disease Diagnosis
    • Digital Health/Health and Wellness
    • Early Days of Life Sequencing
    • Diversity in Precision Medicine
    • Resilience
    • AI and Machine Learning
    • Gene Editing
    • Large Scale Bio-data Resources to Support Drug Development (PPPs)
    • Point-of Care Dx Platform
    • FDA Efforts to Accelerate PM
    • Implementation into Health Care Delivery
    • Next Gen. Workforce of PM
    • Immunotherapy
    • Robust Clinical Decision Support Tools
    • Creating Clinical Value with Liquid Biopsy ctDNA, etc.
    • Neoantigens
    • Emerging Technologies In PM

The Precision Medicine World Conference (PMWC), held annually in Silicon Valley, is coming back to North Carolina for its 2nd Conference back at Duke University on September 24-25, 2018.

PMWC 2018 Duke, the 15th installment of the conference, will spotlight the explosion of biomedical technologies, driving initiatives that enable the translation of precision medicine into direct improvements in health care.

 

    • Track 1 will showcase sessions on the latest advancements in precision medicine which include, but are not limited to:
      • Digital Phenotyping
      • Precision Public Health
      • Pharmacogenomics
      • The Microbiome
      • Rare Disease Diagnosis
      • Digital Health/Health and Wellness
      • Early Days of Life Sequencing
      • Diversity in Precision Medicine
      • Resilience
      • AI and Machine Learning
      • Gene Editing
      • Large Scale Bio-data Resources
        to Support Drug Development (PPPs)
      • Point-of Care Dx Platform
      • FDA Efforts to Accelerate PM
      • Implementation into Health Care Delivery
      • Next Gen. Workforce of PM
      • Immunotherapy
      • Robust Clinical Decision Support Tools
      • Creating Clinical Value with Liquid Biopsy
        ctDNA, etc.
      • Neoantigens
      • Emerging Technologies In PM
  • Track 2- PMWC 2018 Duke Showcase, will enable 15-minute company presentations on latest novel technologies. Apply to present here.
big data diagnostics session

Clinical Dx Showcase

crispr showcase

Emerging Therapeutics Showcase

NGS Showcase

Genomic Profiling Showcase

AI Showcase Session Image

AI and Data Sciences Showcase

  • Luminary and Pioneer Awards, honoring individuals who contributed, and continue to contribute, to the field of Precision Medicine
  • 500+ 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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