How To Minimize The Impact Of Stability Testing On Gene Therapy Batch Yield

It is widely recognized that gene therapy manufacturing processes result in low yields, particularly in early product development stages. Gene therapy products, however, are largely subject to many of the same regulatory requirements and expectations as other large molecule biopharmaceuticals that are produced at significantly larger scales. Often, if gene therapy manufacturers adhered to common paradigms for stability studies, the outcome would be little, if any, remaining product for patients or studies to support investigational new drug applications.

This article outlines strategies for reducing the volumes required for stability studies, with the goal of conserving product for patients, while remaining compliant and delivering data on critical quality attributes across the shelf life of gene therapy products. The recommendations include considerations for both drug substance (DS) and drug product (DP).

Best Practices • Testing