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George Aslanidi, PhD, associate professor and leader of the Molecular Bioengineering and Cancer Vaccine lab at the Hormel Institute has been awarded a grant from the US Food and Drug Administration (FDA) to develop gene therapy for Cockayne Syndrome. Aslanidi is a primary investigator of the study along with Christina Pacak, PhD, Assistant Professor in the Department of Neurology at the University of Minnesota.

The three-year, $1.8 million grant was awarded to the team by the Center for Biologics Evaluation and Research of the Food and Drug Administration. It is an FDA R01 award to develop efficient manufacturing and proof-of-concept for an Adeno-associated virus-based gene therapy for Cockayne syndrome.

Cockayne syndrome is an ultra-rare pediatrics disorder described as premature aging. There are no treatments available at this time. Symptoms include accelerated aging, short stature, microcephaly, photosensitivity, neurological dysfunction, liver and kidney damage, and digestive problems.

“For the next three years, we will work to optimize gene therapy, develop a prototype of cGMP production process, and show that our treatment works in a relevant model. We hope that gene therapy will provide a healthy copy of the affected gene, which will have therapeutic effects resulting in a more positive outcome for patients,” Aslanidi said.

Throughout the process, the research team will be communicating closely with the Center for Biologics Evaluation and Research of the FDA about the project. The team hopes that communication will help to better understand FDA requirements for new biological treatments and streamline the process of an investigational new drug submission at the end of the three-year grant.

“We hope to collect comprehensive data that support moving forward our developments into a clinical trial, and ultimately, to provide a therapeutic option for patients with Cockayne Syndrome,” Aslanidi said.

In addition to Drs. Aslanidi and Pacak, the team also includes collaborators Peter B. Kang, MD, Director of the Paul and Sheila Wellstone Muscular Dystrophy Center and Kah-Wey Peng, PhD, Director of Toxicology Lab at the Mayo Clinic.