Christiana Care’s Gene Editing Institute takes the next step toward its first investigational drug application for FDA approval to treat lung cancer using CRISPR

Gene Editing Institute’s study will be a novel investigation of the use of CRISPR technology to treat K-ras-positive non-small cell lung cancer

Wilmington, Del, April 30, 2019 – In a historic next step in the development of a clinical trial for lung cancer patients using CRISPR gene editing technology, Christiana Care Health System’s Gene Editing Institute is preparing its preliminary investigational new drug application (IND) with the U.S. Food and Drug Administration. This marks the first time Christiana Care’s Gene Editing Institute is developing its own investigator-initiated clinical trial for FDA approval.

For this inaugural clinical trial protocol, the Gene Editing Institute will use the CRISPR genome editing platform to improve the effectiveness of chemotherapy for treating K-ras-positive non-small-cell lung cancer. Patients with this disease have such a strong resistance to chemotherapy that even patients not yet treated for their cancer are resistant to it. Other patients become resistant to chemotherapy after they have been treated.

Researchers from the Gene Editing Institute published a proof of concept paper for this CRISPR approach in Molecular Therapy – Oncolytics in December 2018. In the experiment, they used CRISPR-Cas9 to disable the Nuclear Factor Erythroid 2-Related Factor (NRF2) gene in lung cancer cells by disrupting the NRF2 nuclear export signal. In tissue culture, cells lacking this gene were found to proliferate at a slower rate and be more sensitive to chemotherapeutic agents, such as cisplatin and carboplatin.

“While we all hope for cures, the pioneering work of our gene editing team is making it a priority to improve the lives of today’s patients who are suffering with cancer and other diseases,” said Janice E. Nevin, M.D., MPH, president and CEO of Christiana Care Health System. “Our work in this promising frontier of medicine is guided strongly by our values of love and excellence.”

“This exciting study represents a trio of firsts,” said Nicholas J. Petrelli, M.D., FACS, Bank of America endowed medical director of the Helen F. Graham Cancer Center & Research Institute at Christiana Care. “It will be the first lung cancer clinical trial carried out by a gene editing program embedded in a community cancer center. While our Gene Editing Institute has a great deal of translational research experience, this will be its first study to move scientific discovery toward an IND application. And, it will also be the first time that we will be investigating technology that was grown organically at the Gene Editing Institute.”

“We are confident that the results of our animal studies now enable us to take the next step in the FDA approval process for a cancer clinical trial using CRISPR,” said Eric Kmiec, Ph.D., director of Christiana Care’s Gene Editing Institute. “It is an exciting journey. Our effort positions the Gene Editing Institute as a leader in bringing this heralded technology to people who need it most. Lots of translational scientists are providing advice on this genetic target. Once we get to a clinical trial, our results could have a major impact on our patients, and patients around the country and world.”

The Gene Editing Institute will work closely with the highly regarded Certara®’s regulatory science division, Synchrogenix, in its IND application with the FDA.

“Certara brings expertise for this process and we are delighted to have them on board,” said Dr. Kmiec. The company has authored more than 20 investigational new drug applications the past two years, for both small molecules and biologics across a broad range of therapeutic areas and indications.

“We are proud to align with Christiana Care’s Gene Editing Institute on this landmark research,” said Certara’s CEO Edmundo Muniz, M.D., Ph.D. “This therapeutic innovation holds great promise for correcting mutations at the precise location of the genome to treat genetic diseases and for disabling genes responsible for resistance to standard therapy.”

About the Gene Editing Institute
The Gene Editing Institute is the only research institute of its kind in the nation based within a community health care system. Led by gene editing pioneer, Eric Kmiec, Ph.D., and based at Christiana Care Health System’s Helen F. Graham Cancer Center & Research Institute, the Gene Editing Institute is a worldwide leader in gene editing biomedical research in cancer and other inherited diseases, and the only one working in the same space with oncologists, genetic counselors and patients, bringing translational research from basic science to patient treatment to a new level. You can find the Gene Editing Institute on Twitter and Facebook.

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