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Breaking New Ground in Cancer Immunotherapy

Breaking New Ground in Cancer Immunotherapy 1024 683 Pam Georgiana

Nationwide Children’s researchers develop a scalable, targeted approach to overcoming cancer’s toughest challenges.

Despite significant advances in treatments, cancer remains one of the leading causes of death globally. Traditional therapies, while improving patient outcomes, still have limitations — such as the need for frequent dosing, complex and costly manufacturing and severe side effects. These challenges are driving the search for more effective and sustainable solutions.

One of the most promising cancer treatment innovations is gene therapy, which offers a targeted approach to treating cancer cells while minimizing damage to healthy tissue. At the forefront of this innovation is Nationwide Children’s Hospital, where researchers have developed a technology that produces an off-the-shelf, single-dose treatment designed to address the shortcomings of current immunotherapies.

“Our approach merges immunology and gene therapy in a way that has not been done before,” says Timothy P. Cripe, MD, PhD, chief of the Division of Hematology, Oncology and Blood and Marrow Transplant at Nationwide Children’s Hospital and a professor of Pediatrics at The Ohio State University. “Gene therapy is most often used to correct genetic disorders, but here we’re taking a different path to deliver safer, more efficient and patient-friendly cancer care.”

Dr. Cripe co-founded Vironexis Biotherapeutics, Inc., which has developed the TransJoin™ technology — a scalable platform that produces targeted T cell cancer treatments. Unlike traditional CAR-T therapies, which are more complex, or bispecific therapies that require prolonged infusions, this one-time treatment provides long-term, consistent cancer cell killing, with fewer adverse side effects, even in metastatic cancers.

“Even though many traditional cancer treatments are highly personalized to the patient, over time, cancer cells can develop resistance to them, leaving patients constantly on edge, fearing relapse,” Dr. Cripe says.

The TransJoin™ technology uses an AAV vector infusion that instructs the liver to produce a bispecific protein, guiding T cells to consistently target and destroy cancer cells. This approach significantly reduces toxicity and provides an effective solution with the potential for sustained remission.

“This single infusion turns the liver into a drug factory that continuously produces and secretes a protein that turns T cells into cancer-killing cells,” Dr. Cripe explains. “Essentially, we’re creating a sort of sentry to look out for cancer cells and prevent them from spreading or coming back.”

The TransJoin™ technology is applicable across many disease indications, including blood-based cancers, solid tumors and cancer prevention. It requires only a targeted gene of interest. No patient customization is necessary.

“We’ve created a plug-and-play platform that can take any cancer cell target and slot it in to produce the appropriate protein to kill it,” Dr. Cripe says. “It’s a whole new paradigm in cancer treatment that avoids both the high cost and many adverse side effects of gene therapy.”

The first trial using the TransJoin™ technology is currently recruiting patients with CD19+ acute lymphoblastic leukemia at several sites across the country. Another trial will be ready for patients with metastatic HER2+ breast and gastric cancers in 2025. Vironexis has built a pipeline of more than ten therapies for a variety of cancers and cancer vaccines.

About the author

Pam Georgiana is a brand marketing professional and writer located in Bexley, Ohio. She believes that words bind us together as humans and that the best stories remind us of our humanity. She specialized in telling engaging stories for healthcare, B2B services, and nonprofits using classic storytelling techniques. Pam has earned an MBA in Marketing from Capital University in Columbus, Ohio.

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