AAV Gene Therapy: Charting The Course Through Clinical Challenges

A significant number of clinical holds in adeno-associated virus (AAV) gene therapy programs are attributed to Chemistry, Manufacturing, and Controls (CMC) and quality-related issues. These include incomplete or underdeveloped potency assays, the presence of empty or truncated capsids, lapses in comparability protocols, and broader noncompliance with current Good Manufacturing Practices (cGMP). These challenges can delay development timelines, increase regulatory scrutiny, and ultimately impact patient access to life-changing therapies.

Discover how MilliporeSigma's BioReliance® Testing Services are designed to help gene therapy developers overcome these hurdles.