Element Genomics

Element Genomics Acquired by UCB

The Duke-based startup uses novel epigenomic editing techniques to improve understanding of genome structure and function to identify potential new drug targets 

Element Genomics, a biotech startup founded by Charles GersbachTim ReddyKris Wood and Gregory Crawford, was recently acquired by UCB, a global pharmaceuticals company with a focus on neurology and immunology. Gersbach, the Rooney Family Associate Professor of Biomedical Engineering at Duke University, and his collaborators founded the company in 2015 after developing novel technology to characterize the non-coding genome, opening up new classes of drug targets for common diseases.


The basis for the Element Genomics platform is the comprehensive mapping of gene function and regulation. This includes technology developed at Duke using the CRISPR/Cas9 platform, a genetic editing technique that allows researchers to make changes to targeted sequences of DNA. Typically, researchers will use the platform to make specific, permanent edits to a genome by cutting the DNA. But rather than permanently change the DNA sequence, Element Genomics’ technology alters how a portion of the DNA is regulated, allowing them to study how genes and pathways of interest interact by turning the targeted sections of the genome on or off.

“We were using these tools to characterize the non-coding genome, which includes the 98 percent of the human genome. We don’t really understand how most of it works, but it is clear that it plays a large role in drug response and disease susceptibility,” said Gersbach. “We were developing tools for perturbing that portion of the genome, and that opened up a whole new class of drug targets for common diseases. We quickly realized the technology was something that could go beyond our academic lab.”

In August of 2015, Gersbach and his colleagues approached Barry Myers in the Duke-Coulter Translational Partnership, which had supported Gersbach’s research at Duke, for help to form a start-up. The team received further support from John Oxaal, who was then working as the first Entrepreneur-in-Residence at Duke BME. Oxaal, an alumnus of Duke Engineering and a serial entrepreneur himself, worked with the team to finalize licenses and find space in an incubator in Durham, and currently serves as CEO of the company.



The BRiDGE Incubator space in the Chesterfield Building in downtown Durham.

Metastasizing brain tumor start-up Cereius lands $6M+

Cereius, Inc., a School of Medicine and School of Engineering start-up company developing novel approaches to treat solid tumor brain metastases, closed a $6.75m financing.

Cereius Logo

Cereius was founded by Dr. Michael Zalutsky, Professor of Radiology, Radiation Oncology and Biomedical Engineering at Duke University and Dr. Kimberly Blackwell, Vice President, Early Phase Development and Immuno-oncology at Lilly Oncology and Adjunct Professor of Medicine, Duke University based on technology developed by Dr. Ganesan Vaidyanathan and Dr. Zalutsky. Dr. Vaidyanathan is a professor in the Department of Radiology at Duke University and is a member of the Nuclear Medicine track of the Medical Physics Graduate Program. With over 20 years of c-level experience, Ed Field is the CEO of Cereius currently operating out of BioLabs North Carolina, a co-working space for life science startups in downtown Durham.

The BRiDGE Incubator space in the Chesterfield Building in downtown Durham.

The Duke spinout has acquired $6.75 million in equity financing, with $6.5 million Series A round led by BioInnovation Capital, to include several individual investors.  Cereius has received an additional $250,000 loan from the North Carolina Biotechnology Center.

Cereius is working on solutions to solid tumor brain metastasis developed from cancer cells growing in other parts of the body, such as breast cancer.  30% of advanced solid tumor patients developing brain metastasis represents between 100,000-170,000 patients/year in the US.

doctor holding a laptop in front of a DNA hologram, 3d illustration

Actus Therapeutics

MANAGEMENT: Sheila Mikhail
DUKE INVENTOR: Dwight Koeberl, Priya Kishnani

Actus Therapeutics, a privately held portfolio company of Asklepios BioPharmaceutical, Inc. (AskBio), was incorporated in 2017 to develop new therapies for rare diseases including Pompe disease and epilepsy. Founded by Sheila Mikhail and Phillipe Moullier, Actus will initially pursue gene therapies for Pompe disease based on technology licensed from Dwight Koeberl’s laboratory at Duke University.

Actus Logo

The Company’s goal is to use targeted, low-dose gene therapies to address this glycogen storage disorder. AskBio’s double strand adeno-associated virus vectors (AAV) have the potential to provide treatment at a lower effective dose and with more cell type specificity and less potential systemic effects than other AVV.

Pompe Disease

Pompe Disease is an inherited rare disorder (< 1:40,000 births) caused by the deficiency of acid-alpha-glucosidase (GAA) in muscle. This deficiency results in the accumulation of glycogen in organs and tissues, especially muscles, which can impair their ability to function normally. While enzyme replacement has shown promise in patients with infantile-onset Pompe disease, no curative therapy is available. More information on Pompe disease is available at: https://ghr.nlm.nih.gov/condition/pompe-disease.

Woman with brain tumor


DUKE INVENTOR: Mike Zalutsky (Radiology) and Ganesan Vaidyanathan (Radiology)

Cereius, Inc., a biotechnology company developing novel approaches to treat solid tumor brain metastases.

Cereius Logo

Cereius was founded by Dr. Michael Zalutsky and Dr. Kimberly Blackwell. Dr. Zalutsky is Professor of Radiology, Radiation Oncology and Biomedical Engineering at Duke University.

Dr. Blackwell is Vice President, Early Phase Development and Immuno-oncology at Lilly Oncology and adjunct Professor of Medicine, Duke University. Ed Field is CEO of Cereius which operates out of BioLabs North Carolina, a co-working space for life science startups in downtown Durham.

Exclusive License

Cereius’ intellectual property includes next-generation radiolabeling chemistries developed by Dr. Zalutsky and exclusively licensed from Duke University. These techniques increase the uptake of radionuclides within the tumor site by up to 5-fold compared to existing methods while simultaneously reducing the off-target levels in healthy tissues.

Moreover, the chemistries can employ a variety of medically advantageous radionuclides to either enhance the detection sensitivity of diagnostic imaging or to improve the therapeutic index of targeted therapies. By combining these technologies with advancements in the field of high-affinity, tumor-targeting agents that can cross the blood brain barrier, Cereius has positioned itself to deliver new targeted classes of both radiologic diagnostics and radiotherapeutics for the treatment of cancers in the brain.

Closeup of a calculator and a stethoscope healthcare and expenses concept

SV Analytics

MANAGEMENT: Ryan D. Schulteis
DUKE INVENTOR: Ryan D. Schulteis

SV Analytics specializes in the analysis of medical billing and healthcare claims data. We do more than your claims clearinghouse, more than your claims “scrubber”, and more than your denials management software. They use modern predictive analytics to forecast payer behavior allowing you to proactively anticipate changes in the healthcare financial landscape.

SV Analytics logo

The Problem :

Hospitals and Healthcare Systems lose over $100B every year due to claims
denials by insurance companies

The Solution:

Invariant reduces variation by forecasting changes in payer behavior, allowing hospitals to be proactive. It eliminates standalone excel analysts, providing a standard interface for healthcare revenue analysts.

Programmers working on computer program

“Best Machine Learning Company” Award for Infinia ML

Infinia ML, a machine learning company founded by Lawrence Carin, vice provost for research and professor of electrical and computer engineering for Pratt, bumped elbows with the likes of Qualcomm and Google as the winner for the “Best Machine Learning” award from AI Breakthrough, an independent organization that recognizes the top companies, technologies and products in the global Artificial Intelligence (AI) market today.

infinia logo

“We’re honored by this award, which affirms our focus on making real business impact with machine learning,” Robbie Allen, CEO at Infinia ML said to PRNewswire. “We’re especially grateful to our clients, whose data and domain expertise make our work possible. Their trust empowers us to reduce costs, increase efficiency, and achieve new breakthroughs.”

AI Breakthrough Awards was started in order to recognize the excellence in innovation for top companies and technologies within the AI industry. This year, over 2,500 nominations came in from 15+ different countries worldwide.

Machine learning is the science of teaching a computer to think as humans do while improving over time with the addition of more data and information. Machine learning algorithms have been around for a long time.  In fact, Arthur Samuel coined the phase in 1959.  However, the big leap forward is being able to quickly apply difficult mathematical calculations to big data.

AI Breakthrough Award for Best Machine Learning Company

About Infinia ML

Infinia ML is a team of advanced machine learning experts focused on making business impact. The company helps enterprise clients reduce costs, increase efficiency, and achieve breakthroughs with data science. Infinia ML serves industries from manufacturing and healthcare to marketing and human resources. The company’s capabilities include natural language processing, recommendation engines, object detection, 3D image modeling, and anomaly detection.

The Durham, North Carolina company is led by CEO Robbie Allen, an experienced AI entrepreneur, Chief Scientist Lawrence Carin, Ph.D., one of the world’s most published machine learning experts and Duke University’s Vice Provost for Research, and Executive Chairman and Carrick Capital Partners Managing Director Mike Salvino. Together, the Infinia ML team has produced 31 patents, 11 books, 7 Ph.D.s, and more than 575 published papers.

About AI Breakthrough

Part of the Tech Breakthrough Awards organization, the AI Breakthrough Awards program is devoted to honoring excellence in Artificial Intelligence technologies, services, companies and products. The AI Breakthrough Awards provide public recognition for the achievements of AI companies and products in categories including AI Platforms, Robotics, Business Intelligence, AI Hardware, NLP, Vision, Biometrics, industry vertical AI applications and more. For more information visit www.AIBreakthroughAwards.com

Scientist working with chemicals in laboratory, female researcher analyzing liquid reagent in lab glassware

Precision BioSciences Raises $110M

[Originally posted by Precision BioSciences — June, 2018]

DURHAM, North Carolina, USA, June 26 2018 –Precision BioSciences today announced the closing of an oversubscribed $110M Series B financing to further product development efforts based on its ARCUS® genome editing platform.


The financing was led by ArrowMark Partners and was joined by new investors Franklin Templeton Investments, Cowen Healthcare Investments, Brace Pharma Capital, Pontifax AgTech, OCV Partners, Adage Capital Management, Cormorant Asset Management, Gilead Sciences, Vivo Capital, Alexandria Venture Investments, Ridgeback Capital, Agent Capital, and entities affiliated with Leerink Partners. Existing investors venBio, F-Prime, RA Capital Management, Amgen Ventures, Osage University Partners, DUMAC, and the Longevity Fund also participated in the financing.

Precision intends to expand applications of its ARCUS genome editing platform in the areas of immuno-oncology, genetic disease, and food on its way to building a fully integrated biotechnology company. ARCUS is Precision’s proprietary, homing endonuclease-derived genome editing platform that leverages the small size and high specificity of a natural genome editing system. During 2018, Precision plans to utilize the proceeds from this financing to accelerate and expand its product portfolio. Specifically, Precision targets taking its lead, off-the-shelf CAR-T product into the clinic while advancing both its lead in vivo gene therapy program into IND-enabling studies and its flagship food program into field trials.

“We are thrilled to have such strong support from these leading healthcare investors who share in our vision for the future of human health through innovations in food and medicine,” said Matt Kane, CEO of Precision. “This financing provides us with a strong foundation from which we can advance our translational genome editing programs in multiple sectors.”

In conjunction with the Series B financing, Tony Yao of ArrowMark Partners has joined the Precision Board of Directors alongside existing directors Matt Kane, Derek Jantz, CSO of Precision, and Robert Adelman of venBio.

“We believe that Precision’s core gene editing technology represents a new way to alter the genome, and we are utilizing this technology to address several areas of great unmet need,” said Tony Yao, M.D., Ph.D. “I look forward to working with the team as we advance our programs.”

About Precision BioSciences

At Precision, we utilize a proprietary genome editing method we call ARCUS combined with a team made up of some of the leading minds and pioneers in genome editing in an effort to overcome cancers, cure genetic diseases, and enable the development of safer, more productive food sources.

Genome editing technologies allow us to rethink our approach to a broad array of serious challenges faced by the world today. We now have the ability to precisely edit the DNA of a living organism, opening up the possibility of correcting genetic problems at their source.

scientist hands with dropper or pipette, examining samples and liquid

Clinical Trials for Pompe Disease Gene Therapy Begins This Fall

[Originally posted on Duke Health News]

Published March 28, 2018

By Samiha Khanna

DURHAM, N.C. — After decades pioneering treatments for Pompe disease, Duke Health researchers have developed a gene therapy they hope could enhance or even replace the only FDA-approved treatment currently available to people with the rare, muscle-crippling disorder.

The experimental therapy uses a modified virus to deliver a gene to the liver, where it produces GAA, an enzyme missing in people with Pompe disease. The method was tested in mice, as described in a study published in 2017 in the journal Molecular Therapy – Methods & Clinical Development.

Researchers are currently screening adults with late-onset Pompe disease as they prepare for a phase 1 clinical trial to test the safety of the treatment in 20 people with the condition. The trial will be funded bythe National Institute of Arthritis and Musculoskeletal and Skin Diseases, with support from the National Center for Advancing Translational Sciences. Both agencies are part of the National institutes of Health. The team also received support from the Duke Clinical & Translational Science Institute.


Pompe disease is an inherited condition that affects approximately 1 in 20,000 babies and can also appear in adulthood. Without the enzyme GAA, bodies can’t metabolize the sugar, glycogen. As a result, glycogen builds up in the muscles, leading to degradation of the tissue. If undiagnosed and untreated, it can lead to respiratory problems, heart failure and death.

“The outlook for Pompe disease is much improved since enzyme replacement has become available — it can reverse involvement of the heart and prolong survival,” said Dwight Koeberl, M.D., Ph.D., professor of pediatrics and a medical genetics specialist at Duke who developed the new therapy.

The enzyme replacement therapy (ERT), developed using foundational research from Duke, was heralded as a breakthrough for an orphan disease in 2006, and was dramatized in the major motion picture “Extraordinary Measures.”

“But not everyone responds to this treatment,” Koeberl said. “Many patients make some antibodies, and this can really interfere with treatment. Some infants still die from Pompe disease. Others have to add immune suppression to their treatment, which can lead to other complications. Gene therapy could help these patients.”

In the research published in 2017, the Duke-led research team found that a single small dose of gene therapy was as effective as ERT in clearing the buildup of glycogen from the muscles in mice. A larger dose offered superior results to ERT.

A single treatment spurred the liver to continuously produce GAA without additional treatment, the study authors said. Enzyme therapy requires infusions two to four times a month to lower glycogen levels in the muscles.

Unlike ERT, the gene therapy doesn’t trigger an immune response, a reaction that can limit successful treatment in about half of babies with Pompe. In fact, the gene therapy appeared to reverse immune responses in mice that had previously developed antibodies in response to enzyme replacement, Koeberl said.

The gene therapy uses an inactivated form of adeno-associated virus, which does not cause illness and has been used as a delivery system for hemophilia B and muscular dystrophy treatments, among others, Koeberl said.

“Before enzyme replacement became available in 2006 for Pompe disease, we would continue to give parents bad news — take your beautiful baby home; he or she will die within their first year of life. I knew we had to do something about this.”

–Priya Kishnani, M.D.

Dr. Priya KishnaniShawn Rocco/Duke Health

The emerging gene therapy is just the latest development for a team of scientists at Duke that has been working for three decades to study the causes and potential treatments for glycogen-storage diseases and specifically Pompe.

“When we enter our careers in the field of genetics, we are faced with the many unmet needs for patients with rare diseases,” said Priya Kishnani, M.D., chief of the medical genetics division at the Duke University School of Medicine. “Before enzyme replacement became available for Pompe disease, we would continue to give parents bad news — take your beautiful baby home; he or she will die within their first year of life. I knew we had to do something about this.”

Kishnani has researched Pompe for 25 years, beginning under prominent geneticist Y.T. Chen, the previous chief of the medical genetics division at Duke. In the 1990s and 2000s, Chen and Kishnani worked with biotech firm Genzyme to develop ERT and lead clinical trials.

Since then, Kishnani and dozens of other Duke physicians have been national leaders in Pompe disease, continuing to research the condition, improve the delivery of ERT, manage immune responses to the drug and improve genetic counseling for families.

The Duke team has also helped develop a blood test to diagnose Pompe and a biomarker to monitor severity of the disease and patients’ response to treatment. For nearly 10 years, Kishnani has led a group of physicians to advocate nationally for universal newborn screening, which became a formal recommendation of the U.S. Department of Health and Human Services in 2015. The team’s next step is to develop small molecule or oral drugs that could suppress the buildup of glycogen in the muscles.

“There’s a rich heritage of expertise on glycogen storage disease at Duke, spanning about 40 years,” Kishnani said. “We have continued to grow from the era of cloning genes to developing animal models, to collaborating with pharma to conduct clinical trials here at Duke. I think we have come full circle, from bench to bedside and back to the bench in every aspect of the disease.”

Each year, the research team led by Dr Kishnani hosts patient reunions for both adult and pediatric patients, who travel from around the world for the event.

Pediatric participants include some children who have done so well on treatment, they are entering college.

Haley Hayes, 12, is one of those children, having grown up traveling to Duke for regular treatments and checkups. She has served as an outspoken advocate for research and newborn screening for Pompe disease, and the subject of a video profile by Duke Health videographer Shawn Rocco.

Disclosures: Koeberl developed the technology that is being used in the clinical trial of gene therapy. If the technology is commercially successful in the future, the developers and Duke University may benefit financially. Koeberl has received research and grant support from Sanofi Genzyme Corporation in the past, and the rhGAA (enzyme replacement agent) used in the development of the experimental therapy was supplied by Sanofi Genzyme. Koeberl and Kishnani hold equity in a company that will manufacture the gene therapy.

MIR Kevin Bowles and Rob Hallford, Director of Duke New Ventures chat at I&E’s Bullpen – the hub for all innovation and entrepreneurship activity at Duke University.

Successful 1st Year for New Ventures’ MIR Program

OLV’s Mentors-in-Residence (MIR) program didn’t reinvent the start-up/entrepreneurial wheel — rather, just enabled it to turn faster and more efficiently, heading toward the path to greater success.

New Ventures logo

And since its creation just a short year ago, the MIRs have helped Duke New Ventures advance many new start-ups into the marketplace.  New Ventures is the starting point for turning a university-developed idea into a start-up company.  The center helps inventors think through opportunities, craft business plans and investment pitches, find funding and establish industry contacts.

MIR Fred Kornahrens chats with Joe Knight, CEO of InnAVasc Medical, Inc. a medical device company founded by Duke University surgeons and scientists, which designs and develops products for vascular access for hemodialysis.
MIR Fred Kornahrens chats with Joe Knight, CEO of InnAVasc Medical, Inc. a medical device company founded by Duke University surgeons and scientists, which designs and develops products for vascular access for hemodialysis.

Under Rob Hallford, Duke New Ventures developed several new strategies to vet faculty and staff inventions and support their development into spinoff companies.  One of those new strategies was hiring five MIRs—seasoned entrepreneurs who have successfully raised capital for, built, sold, or invested in start-up companies.

“What we’ve recruited for in our MIRs is a diverse set of experiences and expertise to help our faculty across multiple technology and industry sectors.  But more than that, we’ve been able to find entrepreneurs with an energy for engaging with and building out Duke’s entrepreneurial community,Hallford said.

The main focus of Duke New Ventures is on finding commercial channels and good management for these Duke innovations. However, much of the work is about building relationships.

MIRs have spent their careers building deep industry relationships. As such, they are matched with innovators within the same industry to help them move their ideas to the market.  An MIR not only helps the faculty or staff member develop their company plan such that introductions can be made to potential investors, management and partners, but they also help cultivate those connections.

“As a clinician and scientist, I’m adept at thinking about how products we develop may fit in the lives of patients and their caregivers.  But an important gap in my knowledge and comfort is understanding product/market fit from a business development perspective,” said Arif Kamal, Associate Professor of Medicine and co-founder of the mobile health platform Prepped Health.

MIR Kevin Bowles and Rob Hallford, Director of Duke New Ventures chat at I&E’s Bullpen – the hub for all innovation and entrepreneurship activity at Duke University.
MIR Kevin Bowles and Rob Hallford, Director of Duke New Ventures chat at I&E’s Bullpen – the hub for all innovation and entrepreneurship activity at Duke University.

Kamal was paired with MIR Kevin Bowles, who has over 25 years of entrepreneurial experience. “Kevin has been an invaluable resource in pressure testing our ideas before we head to market.  He is supportive and yet pushes back when needed, so that ultimately, we come to market with the best plan.  He is equal parts cheerleader and coach.”

Tech-transfer opportunities are increasingly important to faculty and staff. By tapping into these experienced entrepreneurs’ business and networking expertise, New Ventures aims to not only promote technology translation but also enable Duke inventors to have a better understanding of the business value proposition.

MIR Fred Kornahrens, who has supported over a dozen pioneering researchers this past year, saw this within the projects he mentored, “I witnessed significant enlightenment on the part of the innovators as to the true meaning of a value proposition for their technology.  Moving from a lack of understanding and the need for it to fully developing and embracing it.”

This last year saw the New Ventures team make 89 introductions and nurture numerous startup ventures based on Duke faculty/staff technologies, with 1 company acquired for $33 million.  15 of 16 start-ups formed in FY18 have stayed in the Durham/Triangle area, providing jobs and contributing to the thriving local economy.

In conjunction with OLV and through their own efforts, Duke start-ups have raised over $525 million this year.

Patient being scanned in tunnel

Polarean Imaging Enrolled 1st Patient in Clinical Trials

Polarean Imaging, a medical imaging technology company enrolled their first patient in its Phase III FDA clinical trial.  Polarean designs and manufactures equipment for production of hyperpolarized xenon or helium gas. They have developed a system that can better spot the early signs of chronic obstructive pulmonary disease (COPD).

Polarean logo

The goal of the clinical trial is to perform a non-inferiority study of its drug-device combination using hyperpolarised 129-Xenon (129Xe) gas MRI, against 133-Xenon (133Xe) scintigraphy. The theory is that this method is more accurate and less harmful than current methods. They will be evaluating a total of 80 patients with lung lobe resection and lung transplant procedures. The Phase III trial will be conducted at Duke University and the University of Virginia.

When used in conjunction with MRI, these gases offer a fundamentally new and non-invasive functional imaging platform. Additionally, xenon gas exhibits solubility and signal properties that enable it to be imaged within other tissues and organs.

Polarean, spun out of Dr. Bastiann Driehuys and Gary Cofer’s technology from the School of Medicine, launched their start-up in 2014.