Viral vector gene therapies, particularly AAV-based approaches are advancing rapidly, but development remains complex. Regulatory expectations, immunogenicity risks, small patient populations, and companion diagnostic considerations can influence trial success and approval timelines.

This white paper provides a practical, FDA-aligned guide to optimizing U.S. gene therapy development from preclinical planning through commercialization.

What this Whitepaper Covers

• FDA expectations for AAV-based gene therapy development, including preclinical, clinical, and accelerated approval considerations

• Key scientific and operational risks such as immunogenicity, potency, persistence, and genotoxicity — and how to address them early

• Use of biomarkers and viral antibody assays (NAb/TAb) to guide patient selection, dose optimization, and trial design

• U.S. regulatory strategy for companion diagnostics (CDx), including when IDE may be required and how to coordinate therapeutic–CDx approvals

• Practical guidance for rare-disease trial feasibility (recruitment, small-sample design, natural history) and proactive FDA engagement (OTP, INTERACT/pre-IND, Q-Sub) to de-risk decisions early

Why Download

You’ll gain clear, experience-driven guidance to:

• Reduce regulatory risk early
• Design scientifically sound and FDA-aligned trials
• Integrate CDx and immunogenicity strategies from the start
• Improve the likelihood of successful approval and commercialization
  

Download the white paper to strengthen your U.S. gene therapy development strategy.