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Where does Delaware fit with the regional precision medicine hub

June 5, 2024 |By Roger Morris

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Delaware was again recognized as a leader in the growing field of precision medicine after an announcement last October featured multiple Delaware institutions which would become active partners in the Greater Philadelphia Region Precision Medicine Tech Hub. The hub is a federally sponsored venture to leverage the area’s life sciences assets, research and development expertise and further the newer practice of precision medicine. As the next step, its members submitted funding proposals to the federal government on schedule this spring, according to Chief Scientific Officer of Ben Franklin Technology Partners Anthony Greene who is heading the hub efforts. “All proposals are currently in due diligence at the Economic Development Association,” Green told the Delaware Business Times. “But we don’t expect to hear anything until the end of June.” At the time of the hub’s fall announcement, Sen. Tom Carper noted, “Our region’s first-class workforce, combined with our history of medical innovation and robust supply chains, makes this a great place to invest in the tech economy.” Sen. Chris Coon further added, “I am proud to see our tri-state area designated as a Regional Technology and Innovation Hub and I look forward to our region continuing to lead the country and the world in biotechnology and precision medicine.” Beyond the importance of the regional hub announcement, Delaware already has a robust record in the development of precision medicine at all levels – from research and development to manufacturing protocols to clinical education to the discipline’s application in practice at medical facilities in Delaware. What then is precision medicine? It’s based on using multiple sources from genomics, biological data and transcriptomics crucial for prediction in order to be more precise and accurate in diagnosis, definitions and treatments of disease subtypes, according to the National Library of Medicine.

Precision medicine in practice

“The way to think about precision medicine is to think of it as personalized medicine,” said Dr. Nicholas J. Petrelli, medical director at ChristianaCare’s Helen F. Graham Cancer Center and Research Institute where some of the most-advanced examples of precision medicine are being practiced and researched. “As an example, at one time a patient with lung cancer was treated the same way as all patients with lung cancer,” he said. “But because of differences in different patients in the genetic mutation of lung cancers, different medicines today will target those individual mutations. The cancers may look the same under a microscope, but they are different. Now, we look at the individual patient, then the tumor, then its genetic profile and then decide on how to treat it.” According to Petrelli, the concept of precision medicine began in earnest with the treatment of breast cancer according to the types of receptors different cancers might have, which then dictated their treatment. But, he said precision medicine as a practice started becoming more frequently employed six or seven years ago. “It has skyrocketed in the past few years to where it is now the standard of care,” he said. “The overwhelming amount of cancer treatment is now precision medicine.” While cancer treatment is the current focus of much of this targeted practice, Petrelli sees cardiovascular precision medicine as being the next area of concentration along with pediatrics “and OBGYN and women’s medicine in general.” In addition to advancements in the general field of genomics – accelerated by the first published sequencing of the human genome in 2001 – the science of gene editing also offers possibilities for the advancement of precision medicine, especially as an adjunct to primary therapy. Here, too, ChristianaCare has had a major role with its Gene Editing Institute and its spin-off company, CorriXR Therapeutics which started in 2022. Researcher Eric Kmiec has had a hand in both ventures, currently as executive director and chief scientific officer at the Institute and founder and chief scientific officer, and former CEO, of CorriXR. The main tool in both is a gene-editing technique whose scientific shorthand name is “CRISPR,” a tool that allows scientists to perform microsurgery on DNA. Kmiec and others want to turn this tool into a therapeutic agent, something that appears close at hand, but not yet realized. In an earlier interview with DBT, Kmiec explained that most cancer patients seldom complete more than 10% of their course of chemotherapy treatment, primarily because they either can’t tolerate the side effects or they die from the disease. “We believe we can raise this to 70 to 80% which would be transformative,” he said. This could be accomplished, he explained, by using a patented CRIPR therapeutic to knock out a gene that prevents existing cancer therapies from working as well as they might while also having fewer side effects. In addition to cancer applications, gene editing may soon be of help in precision-medicine treatments of two other diseases caused by single-gene, single-nucleotide mutations – sickle cell and beta thalassemia.

The Public-Private Sector

While large pharmaceutical companies, some with a significant presence in Delaware, have been at the forefront of bringing precision medicine therapeutics to market, particularly for cancer treatments, the state’s reliance on research platforms that combine government, educational and private resources has helped foster the launch of smaller companies such as CorriXR. One such platform is The Innovation Space, a partnership with the State of Delaware, DuPont and the University of Delaware. Located on DuPont’s Experimental Station campus, the Space offers small and start-up companies more than 130,000 square feet of state-of-the-art, multi-use lab space. “We have at least three companies that are currently involved in precision medicine,” the Space’s President and CEO William Provine said. One of those is Cellergy Pharma whose developmental product is being geared to target two allergic diseases – uncontrollable severe allergic asthma and severe food allergy – using “CART T” cells produced by the patient that would target and permanently eliminate the cells that cause allergic diseases. Cellergy’s product has the experimental name of “CP-010.” The company’s President Ronald Dudek said he expects the drug to be in Phase I clinical trials in 2026. “CAR T cells are regarded as an effective therapy for treating hematologic cancers,” Dudek said. “There are four products approved by the FDA for treating B-cell leukemia and B-cell lymphoma and two products approved by the FDA for treating multiple myeloma. However, there is growing recognition that CAR T cells can be effective in treating diseases caused by the immune system itself.” Another area of broad-platform cooperation is in learning how to manufacture these new targeted medicines safely, effectively and economically which is within the province of the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), a public-private partnership focused on advancing biopharmaceutical manufacturing located on the University of Delaware’s STAR campus. “The field of precision medicine is maturing,” said Kelvin Lee, director of NIIMBL and professor of chemical and biomolecular engineering at the UD. “We’ve only had emphasis on cell therapy or gene therapy for the last seven to eight years, so no one really has a complete understanding of the best way to manufacture these medicines. They are very expensive to produce, and they involve a lot of extra work. Research is being done to address this both inside NIIMBL and outside NIIMBL.” About half of the member stakeholders represented in NIIMBL are from the biomedical industry, a third from academia and the remainder from nonprofit organizations and government. “NIIMBL can bring together all of these stakeholders,” Lee said, adding that one of NIIMBL’s goals is to create “agnostic platforms” for production of medicines. That is, manufacturing processes that have a commonality of approach that can be efficiently shared across industry for better, more-efficient production of multiple medicines. Another of NIIMBL’s goals is to “educate and train a world-leading biopharmaceutical manufacturing workforce, fundamentally advancing U.S. competitiveness in this industry.” “There is already a shortage of workers in all aspects of manufacturing, and the manufacture of precision medicine offers additional challenges,” Lee said. “It’s more labor-intensive, needing more-skilled workers. As we’re still in the early days, technology will evolve over time, so we are educating today's technicians and workers to be agile enough to adapt to these changes.” To further address these educational needs, the Delaware Biotechnology Institute and the Delaware BioScience Association recruited Katie Lakofsky in late 2022, a biologist and academic leader, to head a health care workforce development initiative. The program targets students from under-served populations, with entry requirements being a high-school or GED diploma. Additionally, people who are looking to switch fields early in their careers will be welcome into the program, Lakofsky said. In cooperation with the Delaware Center for Life Sciences Education and Training, Lakofsky reported, “During the first quarter of 2025, we will graduate the first cohort of 20-25 students trained in biomedical manufacturing space. The program involves about 150 hours of training.”

Into the Future

Artificial intelligence (AI) is adding to the growing precision medicine conversation, according to some professionals like Cathy Wu, the director of the Center for Bioinformatics & Computational Biology at the University of Delaware. She said AI has been at work in the field of precision medicine for some time, even though its public persona has only recently been developing. The goal, she added, is to advance it more quickly and more effectively. “AI has already had a dramatic impact in working with predictive models [in patient treatment],” she said. “In addition to genomics, we also have to consider social determinants of healthcare, including patient lifestyle as well as environmental considerations.” One such program, funded by the National Science Foundation (NSF), is working with VA hospitals to “mine associations between social determinants and health outcomes to promote health equity.” A more-ambitious proposal involves a current grant request to the NSF for $20 million to fund an AI institute for human-AI cooperation, an institution that would involve UD, Delaware State University and the universities of Pennsylvania, Florida and Princeton as partners. One of the proposed objectives strikes at the heart of Wu’s goal of closer interaction between AI mechanisms and patients for greater dynamic feedback and patient input, especially as it applies to the concept of health equity. “Patients are already showing a greater awareness of AI and how it can be directed toward their own personal well-being,” Wu said. It’s a goal that fits well within those of the new Philadelphia regional tech hub which is seeking up to $75 million of the $541 million of available federal funding to focus on the further development and implementation of precision medicine. U.S. Rep. Lisa Blunt Rochester noted on the announcement of the hub in the fall, “The capacity of Delaware and its regional partners to pave the way for the future of research and development, manufacturing and innovation was just expanded to the largest scale imaginable.”