Cell And Gene Therapy Team Guides Troubled Rare Disease Programs To Success

A clinical-stage biotechnology start-up asked Veristat to run a new European trial of their complex gene therapy. After a successful bid defense, the sponsor asked us to rescue three ongoing studies because of quality concerns. Veristat experts complemented the client’s small team taking on their first clinical stage program. Our global cell and gene therapy team brought knowledge in genetic material handling, relationships with key global regulatory agencies, and expertise in accelerated pathways to approval.

Study Demographics

Rare Pediatric Global Trial:
Patients from all over the world with central sites and local primary care centers

Primary Services Provided:

• Project management
• Clinical monitoring
• Medical monitoring
• Medical affairs
• Data management