Scott Witting, PhD

My areas of interest include cell and gene therapy manufacturing techniques and quality control of cell and gene therapy products. With my work, I hope to generate assays to characterize the safety and efficacy of cell and gene therapy products.

I became interested in this field during my postdoctoral fellowship studies which focused on using adenoviral vectors expressing short hairpin RNA (shRNA) to silence gene expression in mouse liver cells. Although the goal of this work was to develop a tool to study liver gene function, it sparked my interest in gene therapy. Subsequently, I created a novel lentivirus vector pseudotype based on the Nipah virus. I also developed a new method for large-scale clinical-grade lentivirus production using flow electroporation to overcome manufacturing roadblocks.

Upon encountering a challenging funding climate in the early to mid-2010s, I accepted an opportunity to become the associate director of the Indiana University Vector Production Facility. Due to my previous work experience and successful collaborations, I successfully transitioned from an academic investigator to managing a gene therapy vector manufacturing and testing facility.

Now, at Cincinnati Children’s Hospital Medical Center, I direct a translational testing laboratory specializing in developing and performing assays for cell and gene therapy clinical trial monitoring.

Our laboratory meets all Clinical Laboratory Improvement Amendments (CLIA) standards and is accredited by the College of American Pathologists (CAP). We also meet all Good Manufacturing Practice (GMP) requirements for early phase clinical trial work.

I have served on two National Institutes of Health steering committees, including one for the National Heart, Lung, and Blood Institute, that provided support to academic investigators with early phase cell and gene therapy clinical trials. I am a member of the Gene Therapy Resource Program, and I serve as the translational technology committee chair for the Growing Gene and Cell Therapy Cooperative.

I look forward to further contributions to the growth of cell and gene therapy at Cincinnati Children’s. These therapies are often the best, if not the only, treatment available for an ever-increasing list of cancers and monogenetic diseases.