The Sponsor's Clinical Development Handbook For Gene Therapy Clinical Trials

By Raul P. Lima, Executive Vice President, Clinical Operations, inSeption Group

Gene therapy has existed for decades, but the field is not as mature as it may seem. Its unique pace, structure, and funding dynamics present distinct challenges. Raul P. Lima, Executive Vice President of Clinical Operations at inSeption Group, addresses these challenges in a series of articles.

In "Natural History Studies in Gene Therapy Trials: Benefits, Timing, and Execution," Lima discusses the importance of natural history studies (NHS) in understanding the clinical course of rare diseases and developing accurate endpoints for gene therapy.

The second article, "Rare Disease Clinical Endpoints: Ingenuity Meets Practicality," emphasizes the need for practical and meaningful biomarker endpoints to gauge therapy efficacy and specificity.

Finally, in "High-Velocity Development: Gene Therapy vs. Small Molecule," Lima explores the rapid and overlapping clinical tasks in gene therapy development, highlighting the need for a carefully plotted approach to minimize risks and maximize success.