Preclinical Toxicology Considerations For Cell And Gene Therapy

Cell and gene therapeutics have inherently complex biological behaviors and an unpredictable pattern of interaction with human host systems. Consequently, the clinical development model for small and large molecule drugs cannot adequately assess the safety of these complex products. As the development of these complex products is a risk-based development, clients need a partner with scientific and regulatory experience as experts working as an extension of their team supporting an integrated development program from start to finish.

Maintaining a state of compliance is one of the more important elements of your toxicology program. However, with each cell therapy and gene therapy program being unique, nonclinical programs are established on a case-by-case basis. Therefore, it is critical to make each program product specific to meet regulatory expectations, and the route of administration should mimic the intended clinical route as closely as possible. Preclinical assessment of the safety of an investigational cell and gene therapy product contributes to the definition of an acceptable risk-benefit ratio for a proposed clinical trial. Finding an experienced partner is crucial for demonstrating a novel therapy product's safety and efficacy to establish the optimal dosing range.