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 Peter Marks of FDA

Q: The CBER’s Regenerative Medicine Advanced Therapy Designation program has been very successful, with about 100 requests for designation in the two years of its existence. Can you please tell us about the program and how it was put together?

A: The Regenerative Medicine Advanced Therapy (RMAT) Designation program came into being as part of the 21st Century Cures Act that was signed into law on December 13, 2016.

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Interview with Calum MacRae of Harvard Medical School

Q: What patient data do we need to better understand the underlying cause of disease and how to prevent it?

A: Medicine at present is highly underdetermined and data poor. To be precise, one must be comprehensive, so medicine (with our consent) will use not only what we currently conceive of as biomedical information, but also data from across our lives.

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Headlines from PMWC 2019 Silicon Valley

A big ‘Thank You’ to all of our presenters and attendees for celebrating 10 years of precision medicine progress with us! PMWC 2019 Silicon Valley was attended by 2000 participants from 35 countries, which included over 400 speakers in 5 parallel tracks!

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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.

Read More

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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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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