Gene Therapy Assays: Regulatory Compliance With Unified Immunogenicity

By Deborah Phippard, PhD and Maham Ansari

Assessing immunogenicity is a crucial aspect of developing gene therapies, as pre-existing immunity can significantly impact the therapeutic effectiveness of the product. The journey from creating an immunogenicity assay to obtaining companion diagnostic (CDx) approval for adeno-associated virus (AAV) gene therapy is intricate and lengthy, particularly when the assay is intended for use in international clinical trials. To ensure that the appropriate assay is available at the right time throughout each phase of a clinical program, it is vital for gene therapy developers to engage in meticulous planning.

Here we delve into the regulatory frameworks governing gene therapy assays in both the United States and the European Union. We also provide insights into best practices for achieving compliance with a global regulatory strategy to ensure that developers are well-prepared to navigate the complexities of the approval process.