Editor’s note: Doug Edgeton, CEO of the North Carolina Biotechnology Center, gave a thumb’s up overview about the state’s life science sector in a briefing to the boards of the Biotech Center, the CED and NCBio earlier this week. Here’s a recap.

​RALEIGH, N.C. – There’s no sure thing but change in North Carolina’s life science future, but there are likely to be some interesting blips on the near-term radar.

That’s the thumbnail view given by North Carolina Biotechnology Center President and CEO Doug Edgeton during a luncheon briefing for the boards of the state’s three leading life science booster organizations.

The gathering preceded Tuesday’s opening of the two-day 2016 CED Life Science Conference at the Raleigh Convention Center, presented each year by CED in partnership with NCBiotech and NCBio.

Edgeton was asked to provide the board attendees with his view on what’s likely to make headlines statewide in the coming decade. He told the board members this is what’ll likely be in the spotlight from our state’s labs, fields and entrepreneurial businesses:

  • Gene Editing Technologies

Currently the buzz is over a version called CRISPR-Cas9. It’s being touted as the “next big thing” in biotech because of its unprecedented precision in editing DNA. But that’s also why it’s under heavy scientific and ethical scrutiny. It’s important and elegant, but it raises big questions like, “Where are the boundaries on editing the human race — and all other organisms for that matter?”

CRISPR, the acronym for “clustered, regularly interspaced short palindromic repeats,” uses RNA to guide the molecular scissors, the Cas9 enzyme, to a specific DNA sequence where it makes a specific cut in the DNA and triggers the cell’s DNA repair machinery to address the genetic defect. In other words, it can precisely cut out a mutated section of DNA and replace it with one that functions properly. What “cut-and-paste” does for word processing, CRISPR does for gene editing.

Cas9 is the enzyme most studied and used so far, but researchers are also exploring CRISPR systems that use other enzymes. It’s already drawing some early investment. For example, an Atlanta-area company, Cocrystal Pharma, recently licensed certain patents and know-how from Duke and Emory Universities related to CRISPR-Cas9 technologies, to help it develop cures for hepatitis B and human papilloma virus (HPV) infections.

Editas Medicine became the first publicly traded company using CRISPR to develop drugs. Editas has licensed technology from Duke that the university’s researchers are testing in mice to treat Duchenne muscular dystrophy. UNC is also using CRISPR-Cas9 in lab animals.

CRISPR also holds a lot of promise for ag biotech and food science. Researchers at NC State have been working on it for about a decade and continue to publish discoveries that help explain how this technology can be applied. Interestingly, CRISPR could lead to new kinds of crops and animal proteins that could, technically, avoid the controversial category of genetically modified organisms.

  • The Microbiome

This is another area that shows a lot of promise for human and animal therapeutics as well as in plant-based agricultural applications.

Novozymes, for example, is digging into this field — quite literally. That’s because we have come to learn that a spoonful of soil contains about 50 Billion microbes, including bacteria and fungi, representing up to 10,000 different species. A standard Tupperware lunch container of soil contains more organisms than the number of people who have ever lived on Earth. And microbiologists have come to understand that many of them have very useful characteristics for promoting plant health and vigor. Adding them to seeds will boost crop production. Right now researchers are testing more than 2,000 different microbial seed coatings on half a million test plots in the U.S. alone.

Similar opportunities abound in the bodies of humans and other animals. The latest published estimates are that in an average-sized human, the large intestine, where most of our microbiome lives, contains 39 Trillion bacterial cells. And another trillion inhabit other parts of our bodies. That means we’re about equal part human cells and microbial “passengers.” We’re just beginning to understand how to tap into that vast diversity to turn it into health and well-being. So that’s another fast-growing opportunity for the special mix of life science researchers and entrepreneurs who drive North Carolina’s life science leadership.

  • Precision (personalized) medicine

This may well be where all of the above come together. And more. North Carolina is a recognized leader in both life sciences and big data. You’ll see them working together more often in coming months and years.

Delivering precise treatments customized for each person’s unique genetic makeup is the powerful promise of personalized medicine, and North Carolina is already a leader in this new era of genomics-based health care.

In fact, a study by the Battelle Technology Partnership Practice identified the sector as one of the most likely to flourish in North Carolina’s near-term life science future, based on its analysis of innovation, research and industry activity among the state’s universities and companies.

Companies and university researchers throughout the state are using genomics, biomarkers, sequencing and other cell and tissue technologies to deliver custom molecular therapeutics and diagnostics tailored to individual patient profiles. Here are some prime examples:

  • Argos Therapeutics is focused on the development and commercialization of personalized immunotherapies, based on research at Duke, to treat cancer and infectious diseases. It’s building a $57 million, 100,000-square-foot manufacturing facility in Durham with plans to add nearly 250 employees. The company raised $45 million in a February 2014 initial public offering.
  • Metabolon of Durham is a pioneer in metabolomics, the systematic study of metabolites, the unique chemical fingerprints left behind by the body’s vital cellular processes, or metabolism. The company’s technology and informatics data systems are yielding new insight into how the presence and quantity of various metabolites in body fluids and tissues can indicate states of health and disease. Since its founding in 2000, Metabolon has completed more than 4,000 research projects for nearly 700 clients from both industry and academia and has developed its own products, two diagnostic blood tests for pre-diabetes. The company closed on $15 million in a Series E financing in 2014 and employs about 160 people.
  • Genova Diagnostics of Asheville is a clinical laboratory that provides systems-based testing to help physicians provide targeted diagnosis, treatment and prevention of chronic disease.  The company serves 8,000 health care providers with more than 125 specialized diagnostic assessments covering digestion, immunology, metabolic function, endocrinology and nutrition. Genova employs more than 200 people.
  • Lucerno Dynamics of Morrisville is developing sensor technology that can detect and measure the uptake of radio-labeled biomarkers used in health care. The noninvasive technology can improve the effectiveness of PET and CT scans and provide oncologists an analysis of a tumor’s response to therapy early in a cancer patient’s treatment.
  • ClinGen is an NIH-funded clinical genomics resource launched in May 2015 by a consortium including researchers from the UNC School of Medicine and the UNC Lineberger Comprehensive Cancer Center. Its purpose is to determine the clinical relevance of genetic variants for use in precision medicine and research. ClinGen is developing standard procedures to assess whether there is a true relationship between a given gene and disease, whether a variant in that gene causes or contributes to disease, and if so, whether there is a specific therapeutic or monitoring strategy that could help a patient with that variant.

Health Informatics/Big Data

This is another area of promise for North Carolina, as identified by Battelle.

North Carolina has substantial university and company assets devoted to health informatics, a quickly evolving field that uses databases, electronic medical records, bioinformatics and other tools to support clinical decisions that improve health care efficiency, delivery and outcomes. Some examples:

  • Cloud Pharmaceuticals in RTP uses cloud technology, supercomputing and quantum chemistry for faster and more information-driven drug discovery, design and development. The company’s technology, developed at Duke, aims to predict accurately which molecules are likely to be the best drug candidates, reducing some of the expensive trial-and-error guesswork of traditional drug development.  Once promising molecules are identified, Cloud uses extensive virtual partnering to develop and test them.  The company and its partners have several drugs in development for a wide range of therapeutic areas including cancer, inflammation, CNS disorders and rare diseases.
  • Allscripts, a publicly traded Chicago company with more than 1,200 employees in Raleigh, provides healthcare IT solutions that advance clinical, financial and operational results. The company’s open IT architecture platform connects people, places and data to deliver a single view of the patient record, enabling caregivers to make better decisions and deliver better care. The company offers dozens of software products and services to make doctors’ offices, hospitals, health systems and extended care facilities more efficient.
  • The Carolina Health Informatics Program (CHIP) at UNC is dedicated to improving human health through health informatics research, data sharing, development, and education. CHIP conducts basic and translational research and offers graduate training in health informatics for scientists and for clinicians in medicine, nursing, public health, dentistry, and pharmacy. Program participants and graduates are applying their knowledge in hospitals and clinics, public health agencies, medical research centers and health-related companies.
  • The Duke Center for Health Informatics is an interdisciplinary center focused on producing a new generation of physicians, nurses and health care administrators with expertise in using informatics to improve healthcare. Students at Duke can study a full informatics spectrum including bioinformatics (genomic, proteomic, metabolomics, and imaging), clinical research informatics, applied clinical informatics and community and global health informatics.
  • The Bioinformatics Research Center at NC State conducts research and trains students in the application of quantitative methods to massive biological datasets using computer science and statistics. Faculty and students study gene interactions, pharmacogenetics, protein sequence data interpretation, microarray data analysis and other topics with implications for personalized medicine and better healthcare.

Regenerative medicine, data storage on DNA

“One of the most fascinating mergers of big data and life science applications involves the use of DNA as a data storage medium,” says Edgeton. “We’re running out of effective ways to store and share all the information we’re generating around the world. Some researchers are toying with the very real possibility of storing digitized information within the highly compact binary structure of DNA. The possibilities are amazing.”

Edgeton concluded that our state’s existing leadership in contract research and contract manufacturing will also continue to expand North Carolina’s life science contributions, as will exciting specialties in advanced wound healing and regenerative medicine.

(C) N.C. Biotechnology Center