NEWARK, Del., July 22, 2025 — The National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) has awarded funding to Johns Hopkins University to assess the capabilities of long-read next-generation sequencing (NGS) methods for characterizing and controlling the quality of adeno-associated viral vectors (AAV vectors). The award stems from NIIMBL’s Viral Vector Program’s March 2025 Request for Applications.
The 9-month project is expected to begin in September.
With the emergence of gene therapies, the biopharmaceutical industry needs innovative analytical technologies to assess the quality of AAV vectors, which are the delivery vehicles used in gene therapy to carry healthy genes into cells. This project explores the application of long-read next-generation sequencing as an innovative solution for high-resolution characterization of AAV vectors. Long-read next-generation sequencing is a method that reads entire strands of DNA to detect errors more accurately. By enabling precise detection of mutations and residual DNA, this approach has the potential to overcome key limitations of current assays and set a new standard for quality control in advanced biomanufacturing.
Led by Principal Investigator Winston Timp, Johns Hopkins plans to use Oxford Nanopore Technologies (ONT) and Pacific Biosciences (PacBio) platforms to sequence entire AAV genomes as single reads, revealing crucial details about vector structure and composition that remain hidden to conventional methods.
This technology has the potential to transform AAV manufacturing quality control by:
- Detecting vector genome issues earlier in production
- Accelerating troubleshooting with detailed genetic data
- Improving safety and consistency across the industry
The project is sponsored by the NIIMBL Viral Vector Program, a collaboration between industry, academia, and government agencies to develop an open-access AAV vector manufacturing and analytics platform. The Program addresses cost, time, and skill barriers in the way of sustainable development of gene therapies for prevalent indications and ultra-rare diseases.
“This project will help broaden the use of DNA sequencing tools to better understand the quality of gene therapy products,” said Chris Williams, NIIMBL Viral Vector Program Lead. “It’s an exciting step forward toward designing a manufacturing process for improving the safety and efficacy of gene therapies.”
To learn more about NIIMBL, including how to be a member, visit NIIMBL.org.
About NIIMBL
The National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) is a public-private partnership whose mission is to accelerate biopharmaceutical innovation, support the development of standards that enable more efficient and rapid manufacturing capabilities, and educate and train a world-leading biopharmaceutical manufacturing workforce, fundamentally advancing U.S. competitiveness in this industry. NIIMBL is part of Manufacturing USA®, a network of federally sponsored manufacturing innovation institutes, and is funded through a cooperative agreement with the National Institute of Standards and Technology (NIST) in the U.S. Department of Commerce with additional support from its members.
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