Gene Editing Institute Annual Report 2025

CELEBRATING 10 YEARS OF SCIENTIFIC BREAKTHROUGHS: Shaping the Next Era of Gene Editing

Annual Report Special Edition

Together, we continue to bring genomic medicine to the forefront of cancer care. ChristianaCare’s Gene Editing Institute accelerates innovation to improve patient outcomes in Delaware and beyond.

This is not science for the distant future — it’s science for today.

And the work being done right here at ChristianaCare is going to change lives, here in Delaware and around the world.

Jennifer Schwartz, Esq., Chief Strategy & Legal Officer at ChristianaCare

Translational Research Our initial focus was on lung cancer, which is the leading cause of cancer deaths among adults in Delaware. We found that disabling the NRF2 gene, which is responsible for treatment resistance in cancer cells, led to reduced tumor size and improved sensitivity to chemotherapy.

This discovery has encouraged our team to investigate the potential of NRF2 knockout in other solid tumors with a specific focus on head and neck cancers.

Ultimately, our goal is to develop treatments that enhance the effectiveness of chemotherapy by targeting the NRF2 gene, regardless of the type of cancer a patient has. While this remains a long-term objective, recent research by Kelly Banas, Ph.D. and Natalia Rivera-Torres, Ph.D., has shown significant progress, bringing us closer to achieving this vision every day.

Publications These publications show the expertise of our scientists as we advance cancer treatment through the use of gene editing.

Exon skipping induced by CRISPR-directed gene editing regulates the response to chemotherapy in non-small cell lung carcinoma cells

Mutation-Specific CRISPR Targeting with SaCas9 and AsCas12a Restores Therapeutic Sensitivity in Treatment-Resistant Melanoma

Kelly Banas, Shirin Modarai, Natalia Rivera-Torres,

Brett M. Sansbury, Sophia B. Masciarelli, Salma

Byung-Chun Yoo, Pawel A. Bialk, Connor Barrett,

Kaouser, Olivia M. Tharp, Kelly H. Banas,

Mona Batish & Eric B. Kmiec

& Eric B. Kmiec

Kinetics of Nuclear Uptake and Site-Specific DNA Cleavage during CRISPRDirected Gene Editing in Solid Tumor Cells Kelly Banas, Natalia Rivera-Torres, Pawel Bialk, Byung-Chun Yoo, & Eric B Kmiec

In October 2022, the Gene Editing Institute launched

Researchers have successfully demonstrated

CorriXR Therapeutics, poised to use gene editing

proof of concept in preclinical studies for non-

technology to create novel treatments in areas

small cell lung cancer, marking a major milestone

of unmet medical need. Now, in 2025, under the

in the development of our approach. Building

strategic direction of COO Jan Case, CorriXR has

on this momentum, the team has expanded

developed a broad-based gene editing platform

efforts to include head and neck, esophageal and

that utilizes a unique CRISPR/Cas biomolecular

glioblastoma cancers—laying the groundwork for

tool to disable genes in tumor cells responsible for

IND-enabling studies planned for 2026.

drug resistance.

#1

Startup in Deleware (2024)

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Companies to Watch in the Accelerating Gene Editing Race

Education & Outreach Since its inception in 2019, the education program at the Gene Editing Institute remains committed to demystifying gene editing for high school and college students. Lessons are built around the CRISPR in a Box™ toolkit, developed in-house by leading scientists in their field, and provides students with a chance to perform a full gene editing experiment and see a color change readout.

This year, we created our first introductory course designed for freshmen and sophomore high school students. After launching the pilot in the fall of 2024, we were amazed by the positive feedback from the students.

Students were given the chance to conduct a DNA extraction experiment using strawberries while learning proper lab techniques.

They also had the opportunity to load a gel using food dye, water and glycerol to understand how molecules separate based on size. The results showed a colorful spread of supercharged macromolecules.

This course gives early high school students the chance to learn about concepts usually not seen until college, giving them a leg up on curriculum and easing the transition into higher education. We want students to build their skills prior to coming back to participate in the CRISPR in a Box™ experiment.

We were thrilled to see CRISPR in a Box™ chosen as a Top 10 innovation by The Scientist, a leading life sciences magazine.

Even more exciting, it was the first educational kit to ever be featured on this prestigious list of technological marvels.

This recognition is a powerful validation of our scientists, educators and partners who made this achievement possible. Receiving this recognition not only elevates the CRISPR toolkit itself, but also shines a spotlight on our team’s ongoing efforts to expand gene editing education across the state and beyond the Eastern Seaboard.

Philanthropic Support

Philanthropy in 2024-2025

Students come to us with some proficiency in lab principles and biology, and end up leaving with this whole new view of CRISPR in health care and other scientific fields With key donations, we’ve continued to that they’re already really interested in. It’s building on foundational skills to create inspire the next generation of scientists. The a more equipped student scientist, ready and prepared for their future studies and generosity of our supporters brings more career path. I am so amazed and humbled to continue building out our mission to students to our classroom, helps us develop bring new perspectives into the lab space.” new program offerings and enables us to

- Sarah LaTorre, education program coordinator stand among giants in the field with a small, dedicated crew. Thank you!

We’ve engaged over 2,000 students so far, but where do they come from?

We’re proud to have reached all three counties in Delaware, demystifying gene editing and building a pipeline of future scientists with fresh perspectives on the advancement of CRISPR.

This year, we saw change at the leadership level.

Kelly Banas, Ph.D., Pawel Bialk, Kathy Cao and Natalia Rivera-Torres, Ph.D. comprise our new directorial team, working with our scientists to manage our research, technology development, finance and operations. We prompted our scientific team to explore what their new roles mean and where they see the institute going in the years to come.

Kelly Banas, Ph.D. How has our work on gene editing affected the field around us?

Our research has made and continues to make great strides in the gene editing field. Over the years, we have worked to undercover how CRISPR works in human cells and the impact CRISPR can have therapeutically, leading to major publications in journals like Gene Therapy, Molecular Therapy Oncolytics and the CRISPR Journal. What makes us unique is that we follow where the data and science leads us, regardless if the outcome is favorable or not, because we want CRISPR-directed gene editing to be safe and successful for patients.

What are you most excited to be working on right now?

Our research team is working on many exciting projects in all areas of gene editing, whether it’s understanding the mechanism and genetic diversity of CRISPR-directed gene editing to developing a new method for regulation of CRISPR in cells to identifying new targets in cancers. Each project is just as exciting as the next one with spirited and fruitful scientific discussions.

What makes your research team really stand out?

Each member of our research team comes from a different discipline of science whether it’s molecular biology, biomedical engineering, evolutionary biology or neuroscience. This brings new perspectives and unique thought processes to the table, fostering innovation and deeper collaboration. Each scientist is highly motivated by the science they do every day and the potential impact the research has on our community and the world. Their passion and drive for high-quality science helps maintain the scientific excellence and integrity of GEI.

Pawel Bialk Why are patents so important to the institute’s mission?

Patenting is essential for protecting our discoveries made during the research process and facilitates commercialization through licensing to drive economic impact, attract investment and ensure the invention has the potential to benefit patients. This is particularly important in the translational research space where the goal is to transform our discoveries into practical applications that improve human health.

What are some key patents that the Gene Editing Institute has been awarded?

Our most important patents relate to our discoveries surrounding NRF2, which have been licensed by CorriXR Therapeutics, and are continuing to be developed into a therapy for use against squamous cell carcinoma of the head and neck.

What new intellectual properties are being developed at the Gene Editing Institute that you are most excited about?

In addition to NRF2, we are also developing novel technology platforms. One of these technologies utilizes CRISPR in a way that greatly increases editing efficiency in tumor-specific targeting, and another uses CRISPR as a self-limiting therapeutic that deactivates its own activity upon successful editing of its target gene. Both technologies have the potential for clinical impact on a broad range of genetic diseases.

Natalia Rivera-Torres, Ph.D. How has science evolved over the last 10 years?

CRISPR gene editing technology has revolutionized molecular biology over the last 10 years. It has become a standard molecular biology tool to accelerate research development, a Nobel Prize winner and has obtained FDA approval. We have worked extensively over the last 10 years on developing a potential cancer treatment utilizing CRISPR gene editing.

We have tested our approach in cell culture and animal models, where we have shown we can re-sensitize tumor cells to chemotherapy. Although we initially started the work in non-small cell lung cancer, we have now shown it can be applied to other solid tumors, with special focus on head and neck cancer. As advances in the delivery vehicles have been made, we are now using a lipid nanoparticle as our carrier, similar to the COVID vaccine. With the optimal combination of target genes, CRISPR/Cas9 and LNP delivery, we are marking significant milestones in cancer therapy. Through CRISPR gene editing, we’re working to overcome treatment resistance in cancer.

Where do you see the future of gene editing going?

Gene editing has been breaking boundaries and accelerating the pace of science at an unprecedented speed. Although many efforts still need to be made to fully understand the mechanism and outcomes of these technologies, I am sure that we will keep seeing a rise in therapeutic applications.

Top 10 Achievements in 10 Years We’re so delighted to be celebrating 10 years of innovation, discovery, breakthroughs and community at the Gene Editing Institute. Though there were many challenges along our journey, we also saw exceptional triumphs that drove us forward and gave us purpose.

Here are ten of those achievements.

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The Gene Editing Institute launches in 2015 out of the Helen F. Graham Cancer Center and Research Institute’s Center for Translational Cancer Research, under the watchful eye of Eric Kmiec, Ph.D., and Medical Director Nicholas Petrelli, M.D. Dedicating the institute to education, technology development and scientific research of the human genome, Kmiec and team spearhead a novel idea in the genomic science space: work hand-in-hand with clinicians to develop a patient-first research methodology to tackle health care needs.

In 2017, the Gene Editing Institute partners with Delaware Technical Community College to bring revolutionary gene

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editing tools to classrooms through a National Science Foundation grant. We co-develop a gene editing curriculum for community college students that teaches leading-edge biomedical skills. Eventually, this grant will lead to the creation of CRISPR in a Box™, the gene editing educational toolkit that guides students through a full gene editing experiment with variable outcomes, a color change reaction and a full spectrographic analysis.

The Binational Industrial Research and Development (BIRD) Foundation awards the Gene Editing Institute a grant in partnership with the biotechnology company Novellus Therapeutics (now Fore Biotherapeutics). This grant gives the two entities a pathway to develop and license a new approach to gene editing technology to improve the identification of patient specific cancer treatment.

These studies led to an in-vitro gene editing platform where researchers could work outside of the cell to study the mechanism of CRISPR gene editing. The work done through this partnership presents new opportunities for clinicians: the chance to study not just a model, but an exact replica of a patient’s genetic profile.

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In 2018, our scientists published their first findings on knocking out the NRF2 gene to enhance the effectiveness of first-line chemotherapies in the journal Molecular Therapy Oncolytics.

The study demonstrated that by disabling the NRF2 gene using CRISPR-Cas9, tumor cells exhibited reduced growth and increased sensitivity to chemotherapy. These discoveries laid the foundation for the institute’s ongoing research into the applications of CRISPR in cancer treatment.

DECODR (which stands for DEconvolution of COmplex DNA Repair) is developed to quantify edits present in CRISPRedited Sanger sequencing data in collaboration with Rohan Kanchana, a high school student from Charter School of Wilmington. Its broad analysis window and more precise analysis of indels makes it stand out among its competitors.

This starts the institute’s foray into product development for nonprofit and academic use to further collaborate in gene editing.

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Moving day! In 2020, we relocated to a new lab space on the University of Delaware STAR Campus in Newark, DE. Our move to the STAR Campus provides the expanded lab space we needed to advance our scientific mission, along with office space to support our growing team.

The United States Patent and Trademark Office issues us our first patent for the use of NRF2 to reduce cancer cell proliferation. This marks a major milestone in our intellectual property portfolio, advancing our scientific mission and opening the door to new opportunities in patent development and technology commercialization. Through this licensable material, we enhance our clinical partnerships, giving us more opportunities to collaborate with companies and institutes when licensing these technologies.

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Founded in 2019, the education program at the Gene Editing Institute remains committed to demystifying gene editing for high school and college students. That’s seen clearly in our development of CRISPR in a Box™, a teaching toolkit. This kit is comprehensive enough for college-level instruction yet approachable for high school classrooms, helping us bridge the gap between our lab and our community. The kit is licensed by Carolina Biological Supply Company for national sale. Our box has now been used in over 15 states across the country, including Hawaii and Texas. Even territories like Guam have learned from our cutting-edge technology!

In 2022, the Gene Editing Institute takes a bold step into a new frontier. We become a wholly owned subsidiary of ChristianaCare — an evolution that expanded our operations and positioned us alongside major players in the midAtlantic bioscience ecosystem. With a growing scientific footprint and increasing visibility in the field, this transition enables us to accelerate our development pipeline and advance research toward CRISPR-based therapies with greater speed and impact.

ChristianaCare then launches its first biotechnology private start-up company: CorriXR Therapeutics. CorriXR’s close relationship with the Gene Editing Institute allows it to use CRISPR gene editing technology to develop new, clinically relevant oncologic therapeutics in areas of unmet medical need.

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In 2023, we launched the Learning Lab, an innovative space for students to experience a gene editing experiment in close proximity to our lab space and alongside our expert researchers. This experience prompts them to be science ambassadors and bring this knowledge back to their respective schools and communities, highlighting our mission to bring more perspectives and experiences into the exciting field of gene editing. In the first two years of the program, we see over 1,500 students, engage over 30 schools, and host nearly 100 workshops for students from Maryland, Pennsylvania and all three counties in Delaware.

We’re honored you joined us on this journey through a decade of impact. Thank you for celebrating these milestones with us. We’re proud to reflect on the triumphs that have defined our work—but even more grateful for the people who made them possible: our dedicated team, our generous partners and our visionary leader, Dr. Eric Kmiec.

Here’s to the next 10 years of bold innovation, meaningful discovery and patient-centered science!

We’ve come a long way – and there’s a long journey ahead. But we’re still walking our own path.

When I first came into the field, I was seen as a sort of pioneer rebel in the world of genomic science. We were working with all sorts of tools – TALENS, zinc-finger nucleases, base editing – and getting variable results. Eventually, we came to CRISPR as our gene editing tool of choice, and the results were staggering. Suddenly, work that was taking weeks was being done in hours, and with an efficiency that promised fewer indels and better outcomes. My team powered forward with a clear mission: use this new technology to make better outcomes for patients.

I’m proud to say that we’ve achieved our goal, and then some.

Our first project in using NRF2 to restore effectiveness of first-line chemotherapies exceeded our expectations, and we anticipate that our foundational studies will lead to IND studies on its effectiveness in head and neck cancer in summer of 2026. Our Learning Lab has seen thousands of students, and we’ve affected hundreds more through CRISPR in a Box™, now produced by the nation’s leading educational supply company. We’ve put together an exceptional group of scientists, educators and leaders that push our mission forward every day.

The journey ahead will be riddled with new challenges, and in a landscape of constantly evolving science, we’ll need to work hard to keep up. But we’ve kept up this far. I can only see us improving as we advance and learning as we struggle. With continued, uncompromising dedication to excellence and talented new leadership emerging, there is no doubt that the Gene Editing Institute will continue to be a mainstay in the field of genetic medicine.

Best, Eric Kmiec, Ph.D.