Viral Vector Manufacturing and Analytics Program

The NIIMBL Viral Vector program will develop and adopt technologies related to vector manufacturing.

Industry Need

One of the most important vector platforms in use in the biopharmaceutical industry today is adeno-associated virus (AAV) vectors. Our Program focuses on advancing the manufacturing process and analytical capabilities for AAV vectors.

NIIMBL will bring together the right resources to accelerate development of core manufacturing process platforms and essential analytical capabilities for viral vector product and process characterization, which can

Ensure manufacturing platforms are flexible and robust to accelerate business objectives
Improve therapy yield of known manufacturing platforms
Reduce individual investment and resources needed to help manufacture life-improving and life-saving gene therapies

Solution

Establishment of industrially relevant NIIMBL platform processes for AAV production could significantly enhance access to and utility of viral vectors for gene-based therapeutic research and development. Such processes will be “open” to the entire community and made available as a standard process for academic and small companies.

See the Updates tab for a breakdown of specific workloads by workstream by stage.

View our Program page for a program timeline and additional information.

Impacts

Develop a “NIIMBL platform” that can serve as an open, common platform for all manufacturers, thereby reducing individual investment and resources

Develop a shared understanding in discussions with health authorities and streamline regulatory reviews through more standardized and integrated evidence of product quality, safety, and efficacy

Spur advances that are critical for broad and rapid expansion of the emerging industry of gene-based therapeutics and ultimately the many patients and families who can benefit from access to these novel therapies

Achieve the Program’s vision of a gene-based therapeutics industry capable of serving patients across the full spectrum of unmet needs—from prevalent indications to ultra-rare diseases—that has access to high-quality viral vectors without cost or speed limitations

Publications

Lee, K. H., & Gao, G. (2023). NIIMBL’s viral vector program: A cross-gene therapy community collaboration to transform viral vector development and manufacturing. Molecular Therapy : The Journal of the American Society of Gene Therapy, 31(12), 3359–3360. https://doi.org/10.1016/j.ymthe.2023.11.009