Eric Kmiec, Ph.D

Executive Director and Chief Scientific Officer, The Gene Editing Institute ChristianaCare

  • Wilmington DE

Dr. Kmiec, a pioneer in the field, has achieved several ground-breaking firsts in CRISPR gene editing.

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Spotlight

3 min

CorriXR Launches Bold Collaboration to Create First Inhaled CRISPR Therapy for Lung Cancer

CorriXR Therapeutics, ChristianaCare’s first commercial biotherapeutics spinout, has launched a major collaboration with InhaTarget Therapeutics and Merxin Ltd to develop an inhaled genetic therapy for lung cancer. The goal is to deliver a CRISPR-based treatment straight to tumors in the lungs to improve effectiveness and cut harmful side effects. A New Way to Treat Lung Cancer Lung cancer remains one of the deadliest cancers worldwide. Squamous cell lung carcinoma, an aggressive form of non-small cell lung cancer, accounts for up to 30% of cases. More than 380,000 people are diagnosed each year, yet the five-year survival rate stays under 15%. Standard chemotherapy and immunotherapy often become less effective, and many patients develop resistance that leaves them with few options and rising toxicity. CorriXR is taking aim at this problem. Its CRISPR gene editing system targets NRF2, a key driver of treatment resistance. By switching off NRF2, the therapy has the potential to make tumors sensitive to chemotherapy again and give patients a chance at better outcomes. As reported in a recent paper in Molecular Therapy Oncology, researchers at ChristianaCare’s Gene Editing Institute showed in preclinical lung cancer models that disabling NRF2 can resensitize tumors to chemotherapy with minimal off-target effects. “This partnership is about more than science. It’s about hope for patients,” said Eric Kmiec, Ph.D., founder and CEO of CorriXR Therapeutics and chief scientific officer at ChristianaCare’s Gene Editing Institute. “Lung cancer patients deserve therapies that work and improve quality of life. By combining our CRISPR-based technology with inhaled delivery, we can target tumors directly and reduce systemic toxicity. Our goal is to make treatment simpler, more effective and less invasive.” How the Inhaled Delivery System Works The treatment will be given through inhalation using InhaTarget’s lipid nanoparticle formulation delivered by Merxin Ltd’s advanced inhaler platform. The goal is a non-invasive therapy that patients could use at home. “Combining our pulmonary drug delivery LNP platform with CorriXR’s groundbreaking science and Merxin Ltd’s device technology has the potential to reshape the landscape of lung cancer treatment. We are eager to advance work on this novel combination,” said Frédéric De Coninck, Ph.D., co-founder and CEO of InhaTarget Therapeutics. Merxin Ltd’s technology is central to the approach. Its inhalers are built to deliver precise, consistent doses straight to the lungs. For this collaboration, Merxin Ltd is adapting its device to handle lipid nanoparticle formulations for the first time in a cancer treatment. “Our advanced inhaler technology is designed to ensure non-invasive, precise, consistent delivery of novel therapeutics,” said Philippe Rogueda, Ph.D., co-founder and chief business officer of Merxin Ltd. “We are excited to contribute to this vital effort and help bring innovative solutions to patients with lung cancer.” Why This Matters Patients with squamous cell lung carcinoma often face a fast-moving disease and few treatment choices. A therapy that can reach tumors directly, reduce toxicity and avoid resistance would mark a major shift. “This collaboration underscores the power of combining innovative science with practical delivery solutions,” said Kmiec. “Our CRISPR-based approach is designed to overcome one of the toughest challenges in oncology: treatment resistance. By partnering with experts in inhalation technology, we are moving closer to a therapy that is not only effective but accessible.” Studies will begin soon, with a substantial set of results on effectiveness and impact expected by spring 2026.

Eric Kmiec, Ph.D

3 min

ChristianaCare Gene Editing Institute Achieves CRISPR Breakthrough That Reverses Chemotherapy Resistance in Lung Cancer

In a major step forward for cancer care, researchers at ChristianaCare’s Gene Editing Institute have shown that disabling the NRF2 gene with CRISPR technology can reverse chemotherapy resistance in lung cancer. The approach restores drug sensitivity and slows tumor growth. The findings were published Nov. 13, 2025 in the online edition of Molecular Therapy Oncology. This breakthrough stems from more than a decade of research by the Gene Editing Institute into the NRF2 gene, a known driver of treatment resistance. The results were consistent across multiple in vitro studies using human lung cancer cell lines and in vivo animal models. “We’ve seen compelling evidence at every stage of research,” said Kelly Banas, Ph.D., lead author of the study and associate director of research at the Gene Editing Institute. “It’s a strong foundation for taking the next step toward clinical trials.” Potential Beyond Lung Cancer The study focused on lung squamous cell carcinoma, an aggressive and common form of non-small cell lung cancer (NSCLC) that accounts for 20% to 30% of all lung cancer cases, according to the American Cancer Society. It’s estimated that over 190,000 people in the U.S. will be diagnosed in 2025. While the research centered on this cancer type, the implications are broader. Overactive NRF2 contributes to chemotherapy resistance in several solid tumors, including liver, esophageal and head and neck cancers. The results suggest a CRISPR-based strategy targeting NRF2 could help resensitize a wide range of treatment-resistant tumors to standard chemotherapy. “This is a significant step toward overcoming one of the biggest challenges in cancer therapy — drug resistance,” Banas said. “By targeting a key transcription factor that drives resistance, we’ve shown that gene editing can re-sensitize tumors to standard treatment. We’re hopeful that in clinical trials and beyond, this is what will allow chemotherapy to improve outcomes for patients and could enable them to remain healthier during the entirety of their treatment regimen.” Targeting a Master Switch for Resistance The research zeroed in on a tumor-specific mutation, R34G, in the NRF2 gene, which acts as a master regulator of cellular stress responses. When overactive, NRF2 helps cancer cells withstand chemotherapy. Using CRISPR/Cas9, the team engineered lung cancer cells with the R34G mutation and successfully knocked out NRF2. This restored sensitivity to chemotherapy drugs such as carboplatin and paclitaxel. In animal models, tumors directly treated with CRISPR to knockout NRF2 grew more slowly and responded better to treatment. “This work brings transformational change to how we think about treating resistant cancers,” said Eric Kmiec, Ph.D., senior author of the study and executive director of the Gene Editing Institute. “Instead of developing entirely new drugs, we are using gene editing to make existing ones effective again.” Editing Reaches Threshold Levels One of the most promising discoveries was that disrupting NRF2 in just 20% to 40% of tumor cells, was enough to improve the response to chemotherapy and shrink tumors. This insight is particularly relevant for clinical use, where editing every cancer cell may not be feasible. To test therapy in mice, the researchers used lipid nanoparticles (LNPs), a non-viral method with high efficiency and low risk of unintended, off-target effects. Sequencing confirmed that the edits were highly specific to the mutated NRF2 gene, with minimal unintended changes elsewhere in the genome. “The power of this CRISPR therapy lies in its precision. It’s like an arrow that hits only the bullseye,” said Banas. “This level of specificity with minimal unanticipated genomic side effects offers real hope for the cancer patients who could one day receive this treatment.”

Eric Kmiec, Ph.D

2 min

Kelly Banas, Ph.D., To Present Her Latest Discovery at CRISPR Medicine’s First International Conference

Global audience in Copenhagen, Denmark, will learn of Gene Editing Institute research targeting the NRF2 gene in cancer cells Kelly Banas, Ph.D., principal investigator at ChristianaCare’s Gene Editing Institute, will present her latest research discovery related to targeting the NRF2 gene in cancer cells at the first CRISPR Medicine Conference held in Copenhagen, Denmark, April 22 to 25. The Gene Editing Institute’s research has focused on the NRF2 gene and the strong immune response it causes within cancer cells, allowing them to grow resistant to chemotherapy and leading cancer treatments to fail. By disrupting the NRF2 gene in cancer cells while allowing healthy cells to continue producing it, chemotherapy treatment becomes more effective. Gene Editing Institute principal investigators Kelly Banas, Ph.D., and Natalia Rivera-Torres, Ph.D., in the lab. Banas’ latest research delves into the mechanism of DNA repair following the removal of NRF2, ensuring that surrounding DNA in healthy cells is not affected and that the repair does not produce an unexpected outcome. “I’m extremely honored to be invited to this conference to highlight the work that all of our researchers at the Gene Editing Institute have put into this study,” Banas said. “The work we have done to characterize the impact of CRISPR on the NRF2 gene has changed how we approach new cancer targets. “This has influenced how we design experiments and analyze our data,” she said, “so it’s got a big impact on not just our work, but the work of anyone we collaborate with in the future. This community is full of phenomenal voices, and we’re committed to sharing our work in contexts like this to continue building a foundation of CRISPR research that will uplift treatment for some of the deadliest and most resilient cancers and diseases.” Read about Banas’ earlier research here.

Eric Kmiec, Ph.DNatalia Rivera-Torres, Ph.D
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Biography

Eric B. Kmiec, Ph.D., is the founder and executive director of the Gene Editing Institute at ChristianaCare. He is also co-founder and chief scientific officer of CorriXR Therapeutics. Widely recognized for his pioneering work in the fields of molecular medicine and gene editing, Dr. Kmiec has developed CRISPR based genetic therapies for Sickle Cell Disease and Non-Small Cell Lung Cancer. Dr. Kmiec holds faculty appointments at the University of Delaware and The Wistar Institute and has been an NIH and National Science Foundation supported principal investigator for 35 years. His laboratory has made major discoveries in the CRISPR/gene editing field, and he serves on numerous editorial boards, authoring 169 peer-reviewed publications and books. He has served as primary mentor for 18 doctoral students and numerous postdoctoral scholars.

He is Editor-in-Chief of the journal, Gene and Genome Editing.

Dr. Kmiec is an accomplished speaker in human gene editing and its social and ethical impacts. He has received numerous service medals and awards, including the Eminent Scholar at Marshall University, Proudford Award in Sickle Cell Disease, Bio-Science Innovation Awards and the Philadelphia Life Science Innovator Award.

Areas of Expertise

Gene Editing
Biomedical Research
Molecular Medicine

Education

University of Florida

Ph.D.

Molecular Biology / Biochemistry

1984

Southern Illinois University Edwardsville

M.S.

Cell Biology / Biochemistry

1980

Rutgers University

B.A.

Microbiology

1978

Media Appearances

Affordable CRISPR app reveals unintended mutations at site of CRISPR gene repair

EurekAlert! | The American Association for the Advancement of Science  online

2021-02-11

Wilmington, DE, Feb. 11, 2020 -Scientists have developed an affordable, downloadable app that scans for potential unintended mistakes when CRISPR is used to repair mutations that cause disease. The app reveals potentially risky DNA alterations that could impede efforts to safely use CRISPR to correct mutations in conditions like sickle cell disease and cystic fibrosis.

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New App Can Reveal Unintentional Mutations of a CRISPR Edit

CRISPR Medicine News  online

2021-02-15

A team of scientists from ChristianaCare's Gene Editing Institute in Delaware, US have developed a new customisable app that they say will make it much easier to find out what happened during a CRISPR experiment.

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Affordable CRISPR App Reveals Unintended Mutations At Site Of CRISPR Gene Repair

SCIENMAG  online

2021-02-11

Study in The CRISPR Journal reports the app advances CRISPR gene therapy R&D; 18-year-old software savant helped lead the app’s development

Wilmington, DE, Feb. 11, 2020 -Scientists have developed an affordable, downloadable app that scans for potential unintended mistakes when CRISPR is used to repair mutations that cause disease. The app reveals potentially risky DNA alterations that could impede efforts to safely use CRISPR to correct mutations in conditions like sickle cell disease and cystic fibrosis.

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Selected Papers and Publications

Potential Inequities in New Medical Technologies

Scientific American

Eric B. Kmiec, Jonathan Marron

2020-03-28

As headline-catching new technologies emerge—like tools to “edit” our DNA—researchers, doctors, patients and the general public are excited about the future of medicine and the research that informs its practice. For some, there are obvious and critical conversations taking place about the ethics of this research, including how we do it (think “CRISPR babies” in China) and the potential for edits (intentional or otherwise) that could be passed on to future generations.

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Understanding the diversity of genetic outcomes from CRISPR-Cas generated homology-directed repair

Nature

Brett M. Sansbury, Amanda M. Hewes & Eric B. Kmiec

2019-12-06

As CRISPR-Cas systems advance toward clinical application, it is essential to identify all the outcomes of gene-editing activity in human cells. Reports highlighting the remarkable success of homology-directed repair (HDR) in the treatment of inherited diseases may inadvertently underreport the collateral activity of this remarkable technology.

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Deconvolution of Complex DNA Repair (DECODR): Establishing a Novel Deconvolution Algorithm for Comprehensive Analysis of CRISPR-Edited Sanger Sequencing Data

Mary Ann Liebert, Inc.

Kevin Bloh, Rohan Kanchana, Pawel Bialk, Kelly Banas, Zugui Zhang, Byung-Chun Yoo, and Eric B. Kmiec

2021-02-19

During CRISPR-directed gene editing, multiple gene repair mechanisms interact to produce a wide and largely unpredictable variety of sequence changes across an edited population of cells. Shortcomings inherent to previously available proposal-based insertion and deletion (indel) analysis software necessitated the development of a more comprehensive tool that could detect a larger range and variety of indels while maintaining the ease of use of tools currently available.

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