James is the CEO and co-founder of Precision NanoSystems, Inc. (PNI), a commercial stage biotechnology company at the convergence of nanotechnology, genomics, and precision medicine. PNI empowers researchers to advance nanomedicines for the treatment and diagnosis of disease. James earned a B.A.Sc. in engineering physics from UBC and a Ph.D. in genetics from the Institute for Systems Biology in Seattle, WA. James worked at the Seattle based Venture Capital firm, the Accelerator Corporation concurrent with his Ph.D. and has extensive experience in the science and commercialization of microfluidics, nanotechnology, and systems biology. Read his full bio.

Interview with James Taylor of Precision NanoSystems

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: Patients are already receiving treatment using novel gene and cell therapies. The field is advancing rapidly and this year we have seen CAR-T therapies, RNAi therapies, and gene therapies become commercially available in the US. Additionally, there’s a bolus of clinical studies, in particular mRNA therapies and mRNA vaccines, that would yield yet more new genetic medicines. As more therapies for a range of indications become approved in the US and globally, the clinical use of these types of treatments will grow. The first clinical trials using CRISPR have been announced and patients are likely to be dosed shortly. As these platform technologies begin to prove themselves out, we will see an acceleration of new therapies becoming available.

Q: What other emerging technologies will have a significant impact on patient care in the near and far future?

A: Nanoparticle-based drug delivery is a key enabling technology for a wide range of genetic medicines, including for small RNA, mRNA, CRISPR, and many others. Nanoparticle technologies address a common challenge facing genetic medicines, namely the delivery of these macromolecules to the site of disease and into cells. As such, there is significant industry and academic innovation occurring in delivery technologies, and the platforms are rapidly becoming clinical realities. For example, the recent FDA approval of Patisiran, a lipid nanoparticle based RNAi therapy and the first approved RNAi therapy, is a validation that lipid nanoparticles are safe and effective. Already, there are clinical trials ongoing for lipid nanoparticle-based mRNA therapies and mRNA vaccines and there are companies using LNPs to deliver CRISPR components in late stages of preclinical research. So in both the near and far future, I see nanoparticle technologies playing an important role in enabling the clinical success of precision medicine and hence impacting patient care. Further in the future I envision personalized medicines being manufactured at the point of care where a disease is diagnosed, the molecular origin of the disease identified through sequencing, and a molecularly tailored medicine is designed and made for individual patients and their specific needs. Nanoparticle technologies will enable such individualized medicines as they are manufactured synthetically on demand, which is very different than cell based manufacture of proteins or manufacturing of viral delivered gene therapy.

Q: In order to maximize the potential of the aforementioned technologies in the clinic, what strategies need to be adopted?

 

A: The three pillars of clinical success are always quality, safety and efficacy. Successful strategies have to be built at all stages of development. Nanomedicines are a sophisticated drug product, whereby the excipient (nanoparticle delivery systems) can have as large or greater impact on quality, safety and efficacy, as the active pharmaceutical ingredient (i.e. mRNA, cas9, etc.). So to be successful, a plan needs to be built in place around all aspects of the drug product. Further, matching the indication with the technical advantages of the technology can have a significant impact on the success of a new therapeutic modality. For instance, it is important to consider how the disease manifests itself genetically and if it is a Mendelian or a complex disease, what tissues the disease occurs in, how often it will need to be dosed, etc. Often the therapeutic modalities are being tested for the first time (i.e. CRISPR gene editing), and it is important to minimize compounding technical risk.

 

Q: What are some of the major challenges that need to be overcome before we can see widespread applications across the clinic?

 

A: As we see the new genetic medicine modalities become proven in the clinic, we anticipate significant continued investment into these areas. We are already seeing huge capital investment by pharma and the venture community into these modalities, as they hold the promise to treat disease at its root genetic cause. Additionally, there is a diverse set of genetic medicine technologies, from siRNA and ASOs for gene silencing, mRNA for protein expression, DNA constructs for persistent expression, genomic editing for permanent change, and a variety of powerful cell therapy approaches requiring ex vivo cell manipulation. The pace of innovation is unlike anything we have seen in the past, and deficits faced by one method are an advantage for another.

One major challenge however, is turning these early concepts into clinical products. It is important that as these drug products are translated from bench scale development to the clinic, they are consistent and maintain the performance seen as what you’ve built your data and evidence of performance around. The field has seen many failures of translating programs that demonstrate great performance at the bench scale, but could not be translated into clinical setting. At PNI, we have focused on this concept specifically, to create manufacturing technologies that allow seamless scale up of formulations from discovery to the clinic, and to provide the technical expertise to do this well. This ensures reliable results and high quality formulations throughout the development process.

Q: How can and should the community work together to get those various technologies safely into the clinic?

A: The community should continue to promote and share knowledge on how to successfully develop these exciting new classes of drugs. The potential of these drugs is immense, but they often comprise the combination of multiple disparate technologies to be successful. It is also important that the academic and industry community maintains a fluid collaborative environment, where multiple technologies and areas of expertise can be shared and combined.

Nanotechnologies present new opportunities for advancing medical science and disease treatment in human health care. At PNI, we strive to create and drive knowledge into the community through our Nanomedicine Innovation Network (NIN). The purpose of the Nanomedicine Innovation Network is to provide an overview of the latest research and developments and highlight the remarkable work happeninging to foster learning and inspiration. We are committed to an exchange of ideas and cultivation collaborations with the recent establishment of five global NIN Network sites located in the University of Strathclyde, Evonik’s Darmstadt Site, Harvard Medical School ncRNA Core Facility, PNI’s Vancouver Headquarters and in our San Francisco office all that will conduct workshops and onsite training.

Q: What solution is your organization providing to address what need in precision medicine?

A: As mentioned before, PNI provides a technology platform, expertise and services that address formulation and manufacturing challenges at all stages of drug development. Most of the top organizations in CRISPR, RNA, and gene therapies are using our technology and services to develop their products for targeted or personalized medicine. Essentially, we have the technology and the expertise needed to develop our own therapeutics in this space but we choose to leverage this to help numerous organizations and their personalized medicine programs be successful, and in so doing, we believe we have a greater impact on the field.

PNI’s mission is to accelerate the creation of transformative medicine that significantly impacts human well being. We are proud of the impact we have made in the field and look forward to working with our users to bring these important medicines to patients.

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

A: It is a tremendously exciting time in the life sciences. Similar to the digitization of genomic information through sequencing, we are seeing a digitization of therapeutic design through genetic medicines. The impact this will have on the human condition will be larger than anything we have seen in medicine, and provides a near infinite potential for us as scientists, industry leaders, and citizens.

Interview with Shannon J. McCall of Duke University

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: After several years of the promise of precision medicine and abundant clinical trial work, the recent FDA approval of solid-tumor-agnostic therapies dependent on molecular biomarkers has catapulted genomic/precision medicine into the standard-of-care for late stage cancer.

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Interview with Tao Chen of Paragon Genomics, Inc.

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: For whole genome sequencing to be a reliable clinical tool, it will largely depend on the cost of sequencing the genome and our ability to interpret the data.

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Call for Action: The Time is Now for Patient Data Interoperability

The use of new technologies can provide breakthrough benefits for both patients and providers. However, with increased sharing comes increased risks to the security and privacy of patient data. Currently data is being accumulated across many organizations and initiatives but is often either siloed or simply not accessible. Researchers suggest that patient education tactics can help quell security concerns during patient data sharing.

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Interview with Andrew Magis of Arivale

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. How soon, do you think, will we see what kind of accelerated growth?

A: I think the acceleration has already begun. Large sequencing projects such as NHLBI Trans-omics for Precision Medicine (TOPMed) and NIH All of Us are sequencing 150,000 and 1 million individuals, respectively.

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Interview with Emily Leproust of Twist Bioscience

Q: NGS is enhancing patient care through improved diagnostic sensitivity and more precise therapeutic targeting. Prominent examples include cystic fibrosis and cancer. What other clinical areas NGS will most likely to change the standard-of-care in the near future?

A: Preventative medicine – using genetic data to identify traits that have the potential to cause harm in the future.

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Interview with Michael Phelps of UCLA

Q: You invented the PET scanner that changed the lives of millions of patients with cancer, brain and heart diseases. What are the potential benefits to patients of combining PET with radio-ablation technologies?

A: PET provides imaging assays of the biology of disease in many diseases today.

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Interview with Daniela Ushizima of Lawrence Berkeley National Lab

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 really hope that human physicians will not be replaced by machines in the foreseeable future.

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Interview with Amy Compton-Phillips of Providence St. Joseph Health

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? What technological advancements are driving this change?

A: Genomic medicine is poised to move quickly from the research realm into integration with healthcare delivery, but there is always a time lapse between technology advances and what we do with those advances.

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Interview with James Taylor of Precision NanoSystems

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: Patients are already receiving treatment using novel gene and cell therapies.

Read More

Interview with Julie Eggington of Center for Genomic Interpretation

Q: Together with Robert Burton you founded the Center for Genomic Interpretation (CGI), a non-profit organization. Can you tell us more about CGI and the mission behind it?

A: CGI’s mission is to drive quality in clinical genetics and genomics. CGI works primarily with laboratories, health insurance payers, clinicians, and patients/consumers.

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Interview with Deven McGraw of Ciitizen

Q: Patient healthcare data aggregation and analysis is seen as both the panacea for tremendous breakthroughs in precision medicine and as one of its biggest challenges. Are both true and how so?

A:Yes, both are true. Achieving breakthroughs in precision medicine will require a lot of data – and yet it is often difficult for researchers to amass all of the data needed to advance precision medicine discoveries.

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Breaking News: CMS Takes Actions to Lower Prescription Drug and Other Healthcare Costs – Seema Verma Speaking @PMWC19

The cost of healthcare has been rising at an annual rate of 7% be it company-sponsored health insurance, public insurance such as Medicare and Medicaid, or private insurance. As such, healthcare was top of mind for many individuals this 2018. In the November midterm election many items related to healthcare such as Medicaid expansion, provider pay and indirect effects on the Affordable Care Act could be found on the ballot.

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Did You Catch All 6 of These Big Genomic Medicine Headlines in Recent Weeks?

Genomic sequencing, the driver of modern genomic medicine has come a long way in a short time, and its potential to continue driving innovations in precision medicine is enormous. PMWC 2019 Silicon Valley Jan. 20-23 in the Santa Clara Convention Center will focus on topics that are in the headlines and on everyone’s minds, in NGS and in precision medicine.

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Interview with Christopher Hopkins of Nemametrix

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: We should all be working towards integrating these technologies into routine patient care as quickly as possible, because genomic medicine has the capacity to make profound impacts now.

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Interview with Kristine Ashcraft of YouScript

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: It’s certainly hard to predict, but our goal is to see precision medicine tools in the hands of most providers in the next five years.

Read More
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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