Adeno-associated virus (AAV) gene therapies have transformed the treatment landscape for rare and genetic diseases, delivering potentially life-changing outcomes for patients and validating the promise of genetic medicine. As the field continues to mature, however, the challenges facing developers are evolving. The question is no longer simply whether an AAV therapy can demonstrate clinical efficacy. Increasingly, success depends on whether organizations can reliably develop, manufacture, scale, and deliver these therapies in a commercially sustainable way.
Today, gene therapy developers must navigate a complex environment characterized by rising demand, manufacturing capacity constraints, global supply chain considerations, and increasing expectations for commercial readiness. Traditional approaches that worked during early clinical development may not be sufficient as programs advance toward registration, launch, and long-term commercial supply. As a result, the industry is placing greater emphasis on platform technologies, integrated development strategies, resilient supply chains, and manufacturing innovations that can accelerate timelines while improving scalability and consistency.
This eBook brings together perspectives from across the AAV development and manufacturing continuum. It explores how early commercial planning can help avoid costly delays, how standardized platform approaches and off-the-shelf plasmids can streamline development, and how next-generation innovations are helping overcome long-standing manufacturing bottlenecks. It also examines the growing importance of supply chain orchestration and case management in ensuring therapies reach patients safely, efficiently, and on time.
As the number of AAV-based therapies advances through clinical development and toward commercialization, organizations that successfully integrate manufacturing strategy, supply chain planning, and technological innovation will be best positioned to deliver therapies at scale. The lessons and insights presented in these articles highlight practical approaches for addressing today’s challenges while preparing for the future of gene therapy. Together, they provide a roadmap for developers seeking to accelerate development, optimize manufacturing performance, strengthen supply resilience, and ultimately improve patient access to transformative AAV-based medicines.
