Coordinating Global Capabilities To Support A Gene Therapy Targeting A Rare Neurodegenerative Disorder

An emerging biotech company sought an experienced partner to navigate the complex early-phase development of a gene therapy targeting a rare neurodegenerative disease. They needed comprehensive support to ensure a successful IND submission, including GLP toxicology and biodistribution studies as well as Chemistry, Manufacturing, and Controls (CMC) services tailored to the unique challenges of their program.

By leveraging the expertise of multiple specialized teams, the collaboration enabled seamless integration of preclinical and regulatory efforts, ensuring robust data generation and compliance. Discover how this partnership paved the way for the biotech's program to advance toward clinical development.