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. At Dharmacon she has worked extensively on the mechanism, function, specificity, and delivery of RNA interference (RNAi) and its applications. Read her full bio.

Interview with Nicola McCarthy and Emily Anderson of Horizon Discovery

Q: What products and services do Horizon Discovery offer to address the needs of the precision medicine research community?

A: Data from genomic screens are enabling the right drug to be prescribed to the right person at the right time. This is the essence of personalized medicine and Horizon Discovery is at the forefront of driving forward this powerful new model.

Translational genomics is a crucial component in the development of precision therapies and Horizon Discovery’s capacity to generate precisely engineered cell lines and in vivo models is helping to validate hypotheses generated from genomic studies. By editing the human genome, the effects of genetic variation found in patients can be reproduced in a laboratory setting, enabling important biological questions to be addressed much earlier in the drug discovery process. Horizon Discovery also employs its state-of-the-art CRISPR–Cas9 screening platform to examine gene–gene and gene–drug interactions in vitro and in vivo, enabling the identification of genetic dependencies that can be exploited in drug development, and likely routes to resistance in the clinic, as well as mutations that might lead to an increase in sensitivity to a given drug.

Horizon Discovery offers products in the form of gene modulation reagents (siRNA, shRNA and sgRNAs) that can be bought as single agents or as a screening library to aid research on precision medicine approaches. We offer engineered cell lines and animal models that can be applied in understanding complex biological process or the identification and validation of a new druggable target. Horizon also offers a comprehensive set of services, that includes the generation of tailored engineered cell lines and animal models, high-throughput cell line panel drug screens, and bespoke CRISPR knockout, activation (CRISPRa) and inhibition (CRISPRi) screens.

Q: What makes Horizon Discovery unique?

A: Horizon Discovery understands in detail the products and services that it offers. Our scientists have developed and road tested these, and can give expert advice when providing cell-based solutions to the challenges that our clients and customers face. We can help our customers ask the right questions using an optimal cell-based platform and we can provide solutions for the tribulations that arise in all aspects of biological research. Indeed, the breadth of products and services offered by Horizon Discovery is one of the characteristics that makes the company unique: from understanding the fundamentals of a cellular system by generating an in vivo model to providing reference standards for CLIA labs carrying out diagnostic assays; from target identification in primary immune cells to combining new biologicals with standard of care drugs in oncology; and from generating a reporter cell line for a bespoke drug discovery assay to improving the production of biologicals in CHO cells. We achieve all of this through applying the latest gene engineering technologies.

Q: What is your role at Horizon Discovery and what excites you about your work?

Emily Anderson: I am a senior scientist in the product development group at Horizon’s Colorado site. I lead projects to develop applications of gene modulation and gene engineering in mammalian cells. While technologies like RNA interference and CRISPR-mediated gene knockout and gene editing have revolutionized many aspects of basic biology, getting these tools into the hands of all researchers and maximizing their efficiency, specificity, and ease-of-use has been my passion for the last fifteen years. I’m excited to work in a dynamic environment with others who are also engaged and committed to advancing science and improving human health. I like the discovery that comes along with experimentation and lab-based science, but also using the knowledge I’ve gained to solve real-world problems and connect into a larger picture of the work we’ve done.

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

A: Aside from the aspects already discussed re CRISPR–Cas, the capacity to more easily edit the human genome has brought the treatment of rare diseases to the fore. These have been largely overlooked in the past for many different reasons, but if nuclease-mediated gene editing proves to be a new form of surgery in which a rogue gene can be effectively removed from a cell outside of the patient and the repaired cells re-infused with few long terms side effects, then some of the monogenic rare diseases might be more effectively treated.

Q: What are the short-term challenges that Horizon Discovery and its peers are facing?

A: Most of the short-term challenges involve simply keeping up with the pace of change in terms of gene editing capabilities and the widespread use to which CRISPR–Cas and other nucleic-acid-interacting nucleases are being put. Understanding how and where these nucleases lead to off-target effects is also an important area of the field, as is detecting these changes efficiently in engineered cells.

Interview with Ken Bloom of Ambry Genetics

Q: Tell us more about your organization/company. What patient population are you serving and which services are you specializing in?

A: Ambry Genetics is a recognized leader in high quality complex genetic testing. We seek to find the genomic cause or contributors to rare diseases, abnormal phenotypes and hereditary disorders.

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Interview with Lee Pierce of Sirius Computer Solutions

Q: What is the state of big data and analytics in healthcare, and how to best use the reams of data available?

A: More than ever, Healthcare organizations are achieving measurable value through use of their data and analytics assets. There is more raw material available than ever to create value. This raw material is the data flowing from internal systems and applications and also from devices and systems external to healthcare organizations.

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Interview with Anita Nelsen of PAREXEL

Q: There are various new, emerging technologies that bring us closer towards a cure for life-threatening disorders such as cancer, HIV, or Huntington’s disease. Prominent examples include the popular gene editing tool CRISPR or new and improved cell and gene therapies. By when can we expect these new technologies being part of routine clinical care?

A: Today’s emerging technologies are making the promise of individualized treatment a reality.

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Interview with Ilan Kirsch of Adaptive Biotechnologies

Q: The Nobel Prize in Medicine was awarded recently to James Allison and Tasuku Honjo for their work on unleashing the body’s immune system to attack cancer, a breakthrough that has led to an entirely new class of drugs and brought lasting remissions to many patients who had run out of options. The Nobel committee hailed their accomplishments as establishing “an entirely new principle for cancer therapy.” What is your first-hand experience the impact that those new drugs had on patients?

A: For decades cancer was viewed as solely a cell-autonomous condition.

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BMS buys Celgene | Lilly buys Loxo Oncology – Does this Signal a Return to Strong Deal-Making Activities in 2019?

Bristol-Myers Squibb’s blockbuster $74B deal to buy Celgene creates an oncology powerhouse amid industrywide excitement about the rapidly evolving science and explosive growth of the sector. The agreement could signal a return to deal-making for the pharmaceutical industry in the $133B global oncology therapeutics market.

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Interview with Gini Deshpande of NuMedii

Q: What need is NuMedii addressing?

A: NuMedii, has been pioneering the use of Big Data, artificial intelligence (AI) and systems biology since 2010 to accelerate the discovery of precision therapies to address high unmet medical needs. Artificial Intelligence approaches are a natural fit to harness Big Data as they provide a framework to ‘train’ computers to recognize patterns and sift through vast amounts of new and existing genomic

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Interview with Minnie Sarwal of UCSF

Q: Genomic medicine is entering more hospitals and bringing with it non-invasive technology that can be used to better target and treat diseases. What are some key milestones that contributed to this trend?

A: Completion of complete sequence data from the human genome project, and the advances in proteomic, microRNA and epigenetic assays added a layer of pathway biology to the understanding of human diseases.

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Interview with Shidong Jia of Predicine

Q: Once sequencing has been validated as a clinical solution via trusted workflows, and coinciding with the technological developments driving costs lower, we can expect accelerated human genome profiling for clinical Dx. How soon, do you think, will we see accelerated growth and what can we expect?

A: We will see accelerated human genome profiling for clinical Dx in 2019 and the coming years as more biomarker-based cancer drugs are gaining approval.

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Interview with Iya Khalil of GNS Healthcare

Q: Artificial intelligence (AI) techniques have sent vast waves across healthcare, even fueling an active discussion of whether AI doctors will eventually replace human physicians in the future. Do you believe that human physicians will be replaced by machines in the foreseeable future? What are your thoughts?

A: I think that there’s a lot of speculation and uncertainty around AI, but I don’t foresee a time when we won’t need physicians.

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Interview with Ilya Michael Rachman of Immix Biopharma Inc.

Q: The Nobel Price in Medicine was awarded recently to James Allison and Tasuku for their work on unleashing the body’s immune system to attack cancer, a breakthrough that has led to an entirely new class of drugs and brought lasting remissions to many patients who had run out of options. The Nobel committee hailed their accomplishments as establishing “an entirely new principle for cancer therapy.” Besides CAR T-cell therapy what do you think next generation immunotherapies will look like to successfully combat cancer?

A: The next generation of immunotherapies will build on the insights discovered by immunologists like James Allison and Tasuku Honjo and extend them to modify the body’s response to tumors.

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Join me to Kick off PMWC Silicon Valley in the Santa Clara Convention Center, Focusing on Every Element of Precision Medicine

My team worked in collaboration with Bill Dalton, Kim Blackwell, Atul Butte / India Hook Barnard, Nancy Davidson and Sharon Terry to create a program that touches every component of precision medicine while bringing together all of its key stakeholders. Leading participating institutions including Stanford Health Care, UCSF, Duke Health, Duke University, John Hopkins University, University of Michigan and more will share their learnings and experiences and their successes and challenges, as they make precision medicine the new standard of care for all.

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Interview with Dominic Eisinger of Myriad RBM

Q: The Nobel Price in Medicine was awarded recently to James Allison and Tasuku for their work on unleashing the body’s immune system to attack cancer, a breakthrough that has led to an entirely new class of drugs and brought lasting remissions to many patients who had run out of options. The Nobel committee hailed their accomplishments as establishing “an entirely new principle for cancer therapy.” Besides CAR T-cell therapy what do you think next generation immunotherapies will look like to successfully combat cancer?

A: Next generation immunotherapies include CAR-Ts, TCRs, cancer vaccines, ADCs, bi-specific antibodies, and checkpoint inhibitors.

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2018 Year in Review Milestones: Stakeholder Partnerships Carving Precision Medicine’s Future

2018 has clearly been a year when significant opportunities intersected with strong partnerships to yield advancements. In particular, the clinical advancements that were realized are a testimony to stakeholders working together to deliver on promises affecting major aspects of precision medicine. 2018 has clearly been a year when significant opportunities intersected with strong partnerships to yield advancements.

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Interview with Lisa Alderson of Genome Medical

Q: Tell us a little bit about Genome Medical. What market need is Genome Medical addressing and how?

A: Genome Medical is a telegenomics company that is bridging the gap between available, genetic expertise and the clinical application of genomics.

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Interview with Nasir Bhanpuri of Virta Health

Q: What need is Virta Health addressing?

A: Virta Health delivers an evidence-based treatment to safely and sustainably reverse type 2 diabetes without the use of medications or surgery. In the U.S. alone, type 2 diabetes and prediabetes affects 115 million people, and the economic burden is well-over $300 billion and growing.

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Johns Hopkins
University Of Michigan

The Precision Medicine World Conference (PMWC), in its 17th installment, will take place in the Santa Clara Convention Center (Silicon Valley) on January 21-24, 2020. 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.

See 2019 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

See 2019 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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View the top 3 talks from PMWC here (password: top-videos).