Gene therapy programs continue to push the boundaries of modern medicine — but they also push the boundaries of traditional regulatory frameworks. Unlike standard BLA or MAA submissions, gene therapy applications often contain data that do not align cleanly with conventional categories, templates, or expectations. As shown on page 2 of our PDF, even basic CTD sections frequently require reinterpretation or adaptation to fit the science.
For medical writers, this creates a unique challenge: how to translate complex, unconventional datasets into clear, defensible narratives that regulators can easily evaluate. This requires more than strong writing. It requires strategic judgment, deep familiarity with gene therapy modalities, and an ability to contextualize emerging science within evolving regulatory structures.
Our new resource, 5 Medical Writing Best Practices for Gene Therapy BLA/MAA Submissions, outlines essential principles medical writing teams must apply to ensure accuracy, clarity, and regulatory alignment. These best practices help teams avoid common pitfalls, position nontraditional data effectively, and build submissions that withstand scrutiny from global agencies.
If your organization is preparing a gene therapy submission — or refining your approach — this guide is an invaluable tool.
Download the full PDF to access the best practices:
