First-in-Human Gene Therapy Clinical Trial

Explore how TFS HealthScience delivered operational excellence in a rare neuromuscular disease study—overcoming the unique challenges of a gene therapy clinical trial.

Full-Service CRO for Early-Phase Gene Therapy Trials

Gene therapy clinical trials—especially those targeting rare neuromuscular diseases—require extreme precision, cross-functional coordination, and regulatory expertise. In this exclusive case study, learn how TFS applied a tailored project management framework to drive success in a highly complex, first-in-human Phase I/IIa trial involving critically ill pediatric patients.

Inside the case study:

• Strategies for site and country selection in rare disease trials
• Approaches to navigating ATMP and biosafety regulations across global regions
• Training models to support protocol compliance in high-risk patient populations
• Techniques for managing sample logistics and specialized vendors with precision

This case study is a must-read for sponsors developing gene therapy products or launching early-phase trials in rare indications. If you’re planning or managing a gene therapy clinical trial—especially one involving rare diseases or advanced therapy medicinal products (ATMPs)—this case study offers practical insight into how TFS helped ensure regulatory alignment, operational readiness, and patient safety from start to finish.

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