Large molecule (biologic) drugs continue to be a steadily growing component of the pharmaceutical industry. The advent of large molecule therapeutics requires a different perspective on the assays needed to support development through preclinical and clinical testing, and post market monitoring. Included in this growth are cell therapy and gene therapy products. These new therapeutic approaches have the advantage of targeted specific delivery of a drug. Unmet medical needs are benefiting from advances with new therapeutic modalities. These advances in technology are not met with seamless incorporation of current regulatory expectations for traditional large protein therapeutics. Strategies for assessing immunogenicity and collecting data to assess PK and PD profiles are more complicated for cell therapy and gene therapy products. Positives controls and appropriate matrices can be difficult to obtain. The timeline must be established with sufficient time to prepare good quality positive controls, even for products with an expectation of escalated approval. Targeted delivery of a cell or gene therapy product may not have elevated and quantifiable concentrations that compartmentalize in the blood. Biodistribution studies may therefore be more relevant than serum or plasma PK analyses. However, there is still an expectation that samples for serum PK will be analyzed. The introduction of surrogate matrices is also an important step when developing the methods. The complex nature of these new drug modalities requires additional consideration for development of robust, transferable, specific, selective, and sensitive validated methods. This webinar is designed to offer an overview of developing and validating assay methodologies for cell and gene therapy. Key aspects for development and validation will be addressed with comparisons of the methodologies. The format of the webinar offers an examination of hurdles and offers suggestions of strategies useful to overcome issues when establishing assays for immunogenicity, Pk, and PD for cell therapy and gene therapy products. In addition, current best practices will be addressed. Scientists who attend this webinar will gain knowledge that will be beneficial in helping to achieve well-controlled validated methods for cell therapy and gene therapy products.

Areas Covered

Course Level - Intermediate

Who Should Attend

Why Should You Attend

Large molecule therapeutics require complex assays in preclinical and clinical trials. The complexity of compartmentalization, immunogenicity, and sample matrices can make developing and validating robust assays appear daunting. With growth in cell therapy and gene therapy products in drug pipelines, there is an increasing and evolving need to approach assay development and validation strategically. Regulatory guidelines have provided a general overview but have not provided specific clarity on the requirements for assays supporting the development of cell therapy and gene therapy products. With expected escalated approval of cell therapy and gene therapy products with oncology targets, the need to quickly develop and validate assays is of paramount importance. These assays must meet all parameters of a successful validation. In particular, selectivity for PK assays can be an issue when matrix for the subjects with disease has limited availability. Immunogenicity assays can also be more complex than for other large molecule therapeutics. Prior exposure to the parent virus used as the vector for the therapeutic agent can confound interpretation of immunogenicity data. If the assay cut point is not set statistically more conservatively than with typical large molecule therapeutics, subjects may be excluded from the trials or treatment due to potential safety issues. It is therefore essential that good quality assays are developed and validated. This webinar is designed to address the challenges scientists face with assays supporting drug development for cell therapy and gene therapy products.