Supporting Multiple Global Gene Therapy Studies For Rare Diseases

A long-term client called upon the Veristat team for help conducting two investigational Phase I/II studies. These global studies focused on proving the safety and efficacy of gene therapy for two rare genetic disorders. Working side-by-side with the client, our cell and gene therapy experts addressed many complicated challenges – including site start-up issues, stringent regulatory requirements, and the logistics of handling highly sensitive genetic material.

Study Demographics

Rare Disease Global Trials - Multiple Phase I/II studies across the US, Canada, Australia and Israel

Primary Services Provided:

• Regulatory Consulting
• Adaptive Design Planning
• Project Management
• Monitoring
• Biometrics
• Data Management
• Medical Monitoring
• Safety Oversight
• Medical Writing
• Submissions