Optimizing Development Of Viral Vector Gene Therapies And Companion Diagnostics In The US

By Travis Harrison, Ph.D.; Kennon Daniels, Ph.D.; and Deborah Phippard, Ph.D.

Gene therapy offers transformative potential for treating genetic diseases, but its development is complex and highly regulated. Success hinges on overcoming challenges such as vector immunogenicity, potency, and persistence, while ensuring safety through rigorous preclinical studies. Adeno-associated virus (AAV) vectors remain a leading platform, yet pre-existing immunity and rare integration events demand careful risk assessment. Sponsors must also navigate FDA guidance on trial design, accelerated approval pathways, and biomarker validation to support efficacy claims.

Companion diagnostics (CDx) play a critical role in patient selection and treatment monitoring, particularly for detecting neutralizing antibodies that impact therapeutic outcomes. Developing CDx in parallel with gene therapy is essential for regulatory alignment and co-approval. Additionally, immunogenicity assays require meticulous validation under IDE or IVDR frameworks, emphasizing early engagement with regulators.

With strategic planning—spanning regulatory roadmap development, biomarker integration, and CDx co-development—sponsors can mitigate risks and accelerate timelines. Explore best practices for optimizing gene therapy programs from bench to bedside and learn how to position your development for success in a rapidly evolving regulatory landscape.