From Platform Promise To Commercial Reality: Accelerating Gene Therapy Commercialization Through Structured Data

Despite significant advances in vector design and transgene biology, downstream manufacturing remains a major constraint in AAV gene therapy development. Operational challenges such as slow and resource-intensive tech transfers, fragmented and siloed data, lengthy sponsor onboarding, and rising cost of goods continue to limit efficiency and scalability. These issues are particularly acute for rare disease programs, where small patient populations and compressed timelines leave little margin for manufacturing or CMC missteps, potentially threatening overall program viability.

In response, many organizations have embraced the concept of platforming to improve speed and consistency across programs. However, in practice, these efforts often focus narrowly on shared equipment, host cell lines, or high-level process similarities, delivering only incremental benefits. True platform leverage requires a more comprehensive approach—one that enables systematic capture, reuse, and continuous evolution of process knowledge across development stages and manufacturing campaigns.

A standardized, data-driven CMC backbone addresses this gap by integrating process execution, analytics, and documentation into a cohesive framework. By enabling consistent data capture and real-time insight across batches, such platforms reduce variability, accelerate tech transfer, and support more efficient regulatory interactions. Over time, this approach lowers operational risk, shortens onboarding timelines, and creates a scalable foundation for commercial success as AAV programs advance from early development to late-stage and commercial manufacturing.