NC Creates Foundational Precision Health Initiative, Sara Imhof, Senior Director, Precision Health

North Carolina has long been a leading state for precision medicine innovation and leadership. The North Carolina Biotechnology Center (NCBiotech) has played a key role in cultivating precision health advancement for years, using partnership building, research grants and smallbusiness loans among other investments. For example, from 1993 to 2017, we invested $1.2 million in state dollars in Jude Samulski’s gene therapy research, which were met with $1.25 billion in follow on funding. More importantly, these investments have propelled the field of gene therapy, launched several technologies, and seeded six companies.

By providing grants and loans to support the best precision health research and commercialization initiatives, NCBiotech continues to foster growth of our strong and growing precision health industrial ecosystem. The breadth and depth of precision health companies in our state spans diagnostics, therapeutics, clinical research, data, informatics and digital health, education and advocacy, consulting, research tools and others. The diversity of the industrial base, in turn, drives the growth of precision health in the state.

And most recently, NCBiotech created its own focused initiative around precision health, primarily in the form of the North Carolina Precision Health Collaborative (NCPHC). It’s an interdisciplinary, public-private consortium of precision health experts strategically aligning statewide resources and strengths to elevate precision health.

The vision of the NCPHC is to advance transformative precision health through innovation and partnership. The mission is to accelerate collaborative initiatives that foster research, enable providers, engage industry and empower citizens to improve health outcomes and optimize resource utilization in North Carolina.1 We plan to accomplish our goals through the leadership and engagement of our steering committee – a diverse mix of experts representing university and not-for-profit research, health care (academic and community), payers, investors, and industry.2 

NCPHC defines precision health as a combination of data about an individual’s genes, environment, and lifestyle with innovation and diverse partnership to more precisely predict and diagnose disease, target therapies, and personalize health and wellness plans. To enable a common vision across stakeholders and framework for our efforts, NCPHC created an infographic that describes critical elements, such as innovation and diverse partnerships, requisite for fully achieving the broad possibilities of precision health.3

Some of the core interest areas for NCPHC include population screening, pharmacogenomics, education and policy opportunities, and data and bioinformatics.

NCPHC also hosts external events and is proud to be partnering with the Precision Medicine Leaders’ Summit to host the Summit at NCBiotech in Research Triangle Park on April 30, 2020.4 

This Summit includes multi-stakeholder discussions on advancing the science and implementation of evidence-based precision medicine.  Thought leaders will provide guidance and roadmaps for addressing policy and other challenges holding back the field.  Finally, we are incorporating interdisciplinary interactive breakout discussions facilitated by local experts for the strategic advancement of precision health in N.C.

Please plan to join us.

Jude Samulski, Ph.D., president and chief scientific officer at Asklepios BioPharmaceutical, Inc. (AskBio) and former director of the University of North Carolina School of Medicine’s Gene Therapy Center, is well-known as a pioneer of the use of the live, but harmless, recombinant adeno-associated virus (AAV) as a delivery vector for gene therapy.  Samulski was recruited to North Carolina in 1993 with a $430,000 grant and other support to UNC from the North Carolina Biotechnology Center. NCBiotech continued to support his academic research and commercial technologies with nearly $1.2 million over the next 20 years. Three grants provided for instrumentation and company collaborations and three loans helped bootstrap startup companies.

These awards of state dollars supporting his innovative research and products were met with $1.25 billion in follow-on funding (a leveraging ratio of more than 1,000:1). Samulski and his team also started six companies:

•  Asklepios BioPharmaceutical (AskBio)

•  Chatham Therapeutics (Acquired by Baxter)

•  Molecular Orthopaedics (Closed, assets sold to Calimmune)

•  NanoCor Therapeutics

•  Bamboo Therapeutics (Acquired by Pfizer)

•  Brain Neurotherapy Bio

Further, Samulski’s AAV technologies underlie both gene therapies so far approved by the FDA:  Luxturna (Spark Therapeutics, a member of the Roche Group) for a rare inherited vision loss; and Zolgensma (AveXis) for spinal muscular atrophy, an inherited and fatal neuromuscular disease.