Unrecognizable scientist with a robot, mending or inventing.

Realtime Robotics Awarded Competitive Grant: $225,000

Small Business Innovation Research Program Provides Seed Funding for R&D

Realtime Robotics has been awarded a National Science Foundation (NSF) Small Business Innovation Research (SBIR) grant for $225,000 to conduct research and development (R&D) work on Risk-Aware Motion Planning for Autonomous Vehicles.

realtime robotics logo

This R&D work will change the way that autonomous vehicles plan their motions, particularly in challenging urban driving scenarios.  Realtime Robotics is working on a special-purpose computer processor that can plan thousands of times faster than existing solutions, and this speed will enable it to make many plans at a time, each of which considers a different possible set of behaviors from the other agents—cars, bicycles, pedestrians—in the environment.  This technology will enable risk-aware, normal speed driving, which is critical for widespread adoption by the general public.

“The National Science Foundation supports small businesses with the most innovative, cutting-edge ideas that have the potential to become great commercial successes and make huge societal impacts,” said Barry Johnson, Director of the NSF’s Division of Industrial Innovation and Partnerships. “We hope that this seed funding will spark solutions to some of the most important challenges of our time across all areas of science and technology.”

As explained by Peter Howard, CEO of Boston-based Realtime Robotics, “The new technology is a game-changer for autonomous vehicles, which currently look impressive on the freeway, but drive slowly and haltingly in urban environments.  Our technology will make autonomous cars drive like humans—in that they drive at normal speeds and plan for unpredictable behaviors—just much better.  Computer processors don’t get distracted by their phones or drowsy after long hours of driving.”

Once a small business is awarded a Phase I SBIR/STTR grant (up to $225,000), it becomes eligible to apply for a Phase II grant (up to $750,000). Small businesses with Phase II grants are eligible to receive up to $500,000 in additional matching funds with qualifying third-party investment or sales.

NSF accepts Phase I proposals from small businesses twice annually in June and December. Small businesses with innovative science and technology solutions, and commercial potential are encouraged to apply. All proposals submitted to the NSF SBIR/STTR program undergo a rigorous merit-based review process.

To learn more about America’s Seed Fund powered by NSF, visit: https://seedfund.nsf.gov/


About Realtime Robotics


DUKE INVENTORS: Daniel Sorin, George Konidaris

Faster than Human Motion in Unstructured Workspaces

Realtime Robotics has developed a specialized processor able to, within microseconds, generate motion plans that adapt to process variation and dynamic work environments. The new special-purpose processor allows robots and autonomous vehicles to react instantly to their surroundings, enabling them to function in unstructured, collaborative workspaces as they react to changes and obstacles within their environment.



Realtime Robotics Vemeo Video

Watch it in action

This specialized processor allows people and robots to work safely together in the same space.  This proprietary hardware generates new motion plans as fast as they are being perceived.  Watch the video and see it in action!


About the National Science Foundation’s Small Business Programs

America’s Seed Fund powered by NSF awards $200 million annually to startups and small businesses, transforming scientific discovery into products and services with commercial and societal impact. Startups working across almost all areas of science and technology can receive up to $1.5 million in non-dilutive funds to support research and development (R&D), helping de-risk technology for commercial success. America’s Seed Fund is congressionally mandated through the Small Business Innovation Research (SBIR) program. The NSF is an independent federal agency with a budget of about $7.8 billion that supports fundamental research and education across all fields of science and engineering.

Dr. Howie Levinson shows off his hernia mesh design to President Price. Deep Blue is addressing the unacceptably high rate of hernia occurrence and recurrence. Photo by Jared Lazarus/Duke Photography

Deep Blue Awarded $295K

Medical device startup Deep Blue Medical Advances has raised $295,000 in a debt round, adding to their $517,000 secured in January.

Deep Blue is a start-up company founded in 2014 by a Duke plastic surgeon, Howard Levinson, MD.

deep blue logo
Dr. Howie Levinson shows off his hernia mesh design to President Price. Deep Blue is addressing the unacceptably high rate of hernia occurrence and recurrence. Photo by Jared Lazarus/Duke Photography
Dr. Howie Levinson shows off his hernia mesh design to President Price. Deep Blue is addressing the unacceptably high rate of hernia occurrence and recurrence. Photo by Jared Lazarus/Duke Photography

The company has been advancing a hernia mesh with enhanced anchoring strength to resist wounds from gapping and bursting open, as well as a suture anchoring device to overcome large suture knots associated with wide sutures. In separate efforts, Dr. Levinson is working on additional translational projects including an anti-biofouling Foley catheter, a non-invasive light imaging technology to diagnose skin disorders, and tissue-engineered skin that resists contraction.

“I have worked closely with Duke OLV to protect intellectual property, navigate entrepreneurial activities, and to raise non-dilutive grant funds to advance ideas. Duke OLV’s support has been critical to our success and is a clear distinguishing factor between Duke University and its existing peer institutes.” –Howard Levinson, MD, Associate Professor of Surgery, School of Medicine

Young assistant analyzing substances in tubes

$34 Million in Series D Financing Awarded to PhaseBio

Proceeds will advance clinical development of PB2452, a potentially first-in-class reversal agent for patients on ticagrelor, and PB1046, a novel treatment for PAH


PhaseBio Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company focused on the development and commercialization of novel therapies for orphan diseases, with an initial focus on cardiopulmonary disorders, today announced the completion of a $34 million Series D financing, including the conversion of existing convertible promissory notes.

New investors in the financing include Cormorant Asset Management, Rock Springs Capital and Mountain Group Partners. Existing investors New Enterprise Associates, Hatteras Venture Partners, AstraZeneca (NYSE: AZN), Johnson & Johnson Innovation – JJDC, Inc., Syno Capital and Fletcher Spaght Ventures also participated in the financing.

“PhaseBio has the potential to bring life-saving therapies to patients with orphan cardiopulmonary diseases. PB2452 addresses a growing unmet medical need for patients on ticagrelor who are experiencing a major bleeding event or who need urgent surgery,” said Clay B. Thorp, Executive Chairman of PhaseBio and General Partner of Hatteras Venture Partners. “In addition, with PB1046 and future product candidates based on our elastin-like polypeptide (“ELP”) technology, PhaseBio has the potential to build a significant pipeline in the orphan disease space.”

PhaseBio plans to use the proceeds from the Series D financing to advance the clinical development of its lead product candidate PB2452. PB2452 is a potentially first-in-class reversal agent for the antiplatelet drug ticagrelor that is being developed for the treatment of patients on ticagrelor who are experiencing a major bleeding event or who require urgent surgery, and is currently in a Phase 1 proof of concept study. Additionally, the company plans to initiate a Phase 2 clinical study of its ELP-based therapy PB1046 in pulmonary arterial hypertension (“PAH”) in the third quarter of 2018 and to explore additional indications for its half-life extending ELP technology platform.

“We are encouraged by the enthusiasm of our new and existing investors for the potential of our clinical programs and our proprietary ELP technology platform,” said Jonathan P. Mow, Chief Executive Officer of PhaseBio. “We look forward to the readout of data from the Phase 1 study of PB2452, exploring partnership opportunities for our drug products outside of the U.S. and dosing patients in the Phase 2 study of our second product candidate, PB1046, in patients with PAH shortly. In addition, we plan to continue to leverage our ELP technology to expand our development pipeline.”

Just this past February, PhaseBio was awarded a $2.8 million Fast Track Small Business Innovation Research (SBIR) grant from the National Institutes of Health (NIH) to support the clinical development of PB1046, a first-in-class, sustained-release vasoactive intestinal peptide (VIP) analogue, in patients with pulmonary arterial hypertension (PAH).

About PhaseBio

PhaseBio Pharmaceuticals, Inc., is a clinical-stage biopharmaceutical company developing therapies for the treatment of orphan diseases. PhaseBio is leveraging its proprietary elastin-like polypeptide (ELP) biopolymer technology platform to develop therapies with the potential for less-frequent dosing and better patient compliance. PhaseBio’s lead development candidate, PB1046, is a first-in-class weekly vasoactive intestinal peptide (VIP) receptor agonist for the treatment of pulmonary arterial hypertension. The company is also developing PB2452, a reversal agent for the antiplatelet therapy ticagrelor. PhaseBio is privately owned, with headquarters and research laboratories in Malvern, PA.

blood vessels

Humacyte lands $150M from Fresenius Medical Care

This past June, Humacyte, an emerging biotechnology company focusing on technology to grow stable, “off-the-shelf” human tissue replacements exclusively licensed their human acellular vessel, Humacyl, to Fresenius Medical Care.  The investment gives Fresenius 19% fully diluted ownership stake in Humacyte.

humacyte logo

This investment is added to the $75,000 in Series C preferred stock financing secured in March.  Prior to Fresenius, the company had received more than $405 million in total funding to date, with more than $365 million in private investments and awards of up to $40 million through federal and state grants and contracts.

Humacyte was founded by former Duke professor Laura Niklason and includes Duke alums Shannon Dahl and Juliana Blum. A target goal of the company has been to create lifesaving and long-lasting vascular access or replacement for patients with End Stage Renal Disease.

HUMACYL is an investigational product that has not yet been approved by the FDA. However, clinical trials to date suggest that HUMACYL has the potential to become a part of the body’s living tissue. These trials also suggest that HUMACYL may have the potential to last longer with fewer complications than existing synthetic alternatives used for hemodialysis access, possibly lowering healthcare costs by decreasing the number of surgical interventions.

About Humacyte

Humacyte, Inc., a privately held company founded by Dr. Laura E. Niklason, M.D., Ph.D., in 2004, is a medical research, discovery and development company with clinical and pre-clinical stage investigational products. Humacyte is primarily focused on developing and commercializing a proprietary novel technology based on human tissue-based products for key applications in regenerative medicine and vascular surgery. The company uses its innovative, proprietary platform technology to engineer human, extracellular matrix-based tissues that can be shaped into tubes, sheets, or particulate conformations, with properties similar to native tissues. These are being developed for potential use in many specific applications, with the goal to significantly improve treatment outcomes for many patients, including those with vascular disease and those requiring hemodialysis. The company’s proprietary technologies are designed to create off-the-shelf products that, once approved, can be utilized in any patient.

Experimental lab test-tubes with test substance

Grid Therapeutics Signs Exclusive License Agreement

In August 2017, Grid Therapeutics, LLC, an oncology-focused biotechnology company announced the execution of an exclusive license agreement with Duke University to develop the first human-derived antibody as a targeted immunotherapy for cancer. Under the agreement, Grid has acquired the exclusive rights to all intellectual property, including relevant patents, related to Complement Factor H (CFH) antibodies as a cancer therapy, and diagnostics around CFH.

Grid Therapeutics logo
Edward Patz

Grid is based on the innovative science developed by Edward F. Patz, Jr., MD and his team at Duke University. Grid is currently developing its lead candidate for the treatment of solid tumors and plans to begin clinical trials in cancer patients in early 2019.

“We are pleased to sign this exclusive license agreement with Duke to advance this truly innovative science into clinical trials and begin evaluating the therapeutic treatment options for multiple forms of cancer,” said Dr. Paolo Paoletti, on Business Wire.  Dr. Paoletti is a Grid Board member and CEO of GammaDelta Therapeutics. “This agreement allows Grid to rapidly develop a new generation of cancer immunotherapies based on a human-derived antibody that inhibits CFH, a protein that serves to protect cancer cells.” As part of the license agreement, Duke University has become a shareholder in Grid.

This past April, Grid closed on Series B Financing.  They used this money to accelerate and expand the development of their lead therapeutic candidate, GT103, for the treatment of solid tumors, and to prepare for clinical trials starting early next year.

About Grid Therapeutics

Grid Therapeutics is a biotech company based on the innovative platform for discovering and isolating human derived antibodies. Dr Edward F. Patz, Jr, is the co-founder and CEO of Grid, located in Durham, North Carolina. The lead antibody was discovered in exceptional outcome early stage lung cancer patients who did not progress to develop metastasis. The antibody was isolated from patient’s B-cells using state of the art molecular genomic techniques. GT103, the company’s lead asset, will begin a Phase 1 clinical trial in cancer patients with advanced-stage solid tumors.

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.

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.