Physician Priority Link – For urgent issues or to speak with the specialist on-call
1-888-987-7997
Applied Gene and Cell Therapy Center to Increase Clinical Trials
Key Takeaways
The new Applied Gene and Cell Therapy Center at Cincinnati Children’s, opening later this year, will expand clinical trials of advanced treatments like gene and cell therapies for cancer, blood diseases and genetic disorders.
The center will create new positions and enhance research efforts to translate lab discoveries into real-world applications.
The center will build upon existing research at Cincinnati Children’s and integrate processes to accelerate access to new treatments.
Cincinnati Children’s is working on an Applied Gene and Cell Therapy Center that will enable the health system to increase clinical trials of innovative drugs and biological therapeutics for patients with cancer, blood diseases or genetic disorders. Set to open later this year, the center will focus on preparing gene, cell and immunotherapy products for clinical trials.
The Applied Gene and Cell Therapy Center will continue the success of the Cincinnati Children’s Translational Core Laboratory, which has been a leader in developing, testing and manufacturing drugs and therapeutics for early-phase clinical trials for more than 15 years.
“We are deeply invested in pediatric research that focuses on gene and cell therapy, which benefits children and families in our region and around the world,” says Steve Davis, MD, MMM, president and CEO of Cincinnati Children’s. “The Applied Gene and Cell Therapy Center will expand on the research efforts that have already earned our health system recognition by Fortune as one of America’s Most Innovative Companies, and which also contributed to U.S. News & World Report ranking Cincinnati Children’s as No. 1 in the nation for pediatric cancer care.”
A Research Powerhouse
Children and families grappling with cancer or genetic disorders often look to us for innovative treatments, and this new commitment by Cincinnati Children’s holds great potential for improved outcomes.
Stella Davies, MBBS, PhD
Cincinnati Children’s remains dedicated to research, with about one-third of its 19,000 employees engaged in efforts to find medical treatments or cures. Families from all 50 states and dozens of countries travel to Cincinnati Children’s each year to receive care for complex or rare disorders.
“There are many potential treatments in development or clinical trials,” Davies says. “This program expansion will allow us to increase the number of cell, gene and immunotherapy clinical trials for our patients. Our center’s experts will conduct both translational and clinical research on cell and gene therapy.”
Cincinnati Children’s is a research powerhouse, and this commitment to advancing child health outcomes through the Applied Gene and Cell Therapy Center demonstrates how excellence in research remains a central pillar of our mission.
Tina Cheng, MD
Tina Cheng, MD, director of the Cincinnati Children’s Research Foundation, chair of the Department of Pediatrics and chief medical officer of the health system, says research conducted at the new Applied Gene and Cell Therapy Center will help elevate the outstanding clinical care provided to children and families.
“Cincinnati Children’s is a research powerhouse, and this commitment to advancing child health outcomes through the Applied Gene and Cell Therapy Center demonstrates how excellence in research remains a central pillar of our mission,” Cheng says.
In line with this mission, the Applied Gene and Cell Therapy Center will create about two dozen new positions, half of which will be scientists, including biologists and quality assurance specialists for medicinal products using Current Good Manufacturing Practice (cGMP).
A One-Stop-Shop for Accelerating Therapies
The new center will build upon current innovative cell, gene and immunotherapy clinical trials. For example, in collaboration with Elixirgen, Cincinnati Children's researchers are testing a gene therapy for bone marrow failure syndrome caused by telomere biology disorders. This therapy, available only at Cincinnati Children’s, recently received orphan drug status from the Food and Drug Administration.
Researchers are also advancing treatments for hereditary pulmonary alveolar proteinosis (hPAP), a rare disease caused by mutations in the GM-CSF receptor genes that disrupt alveolar macrophage function and lead to lung surfactant buildup. Led by principal investigator, Bruce Trapnell, MD, our researchers are using gene therapy to restore macrophages and effectively clear surfactant and other debris in the lungs.
Scott Witting, PhD, director of Cincinnati Children’s Analytical and Quality Control Laboratory and interim director of the Cell Manipulations Laboratory, says Cincinnati Children’s is a one-stop-shop for accelerating gene and cell therapy clinical trials.
“From viral vector production, cell manipulation, quality design and testing, our process is efficient because we’re all in one center,” Witting says. “Most importantly, we support researchers through the translation phase so we can bring trials out of the research lab and into the clinic faster.”
