Novel AAV-Based Vector Production Biomanufacturing Platform

 

Overview

Adeno-associated virus (AAV) is an unenveloped virus widely recognized for its exceptional ability to effectively and securely transport genetic material into cells. AAV has emerged as a well-established and highly promising vector for gene therapy, however, its widespread adoption has been severely limited by significant hurdles in its manufacturing.

A primary challenge lies in the astronomical production costs driven by the inherent complexities of current manufacturing methods. These methods rely on intricate mammalian cell culture systems, multi-plasmid transient transfection, and labor-intensive downstream purification processes. Such factors contribute to per-dose costs that are prohibitively high, creating a major barrier to patient access.

The inherent complexity of AAV production processes introduces significant challenges in consistently achieving the required quality, purity, and potency of the final vector product. The limited scalability of current production methods further exacerbates costs, as they struggle to meet the growing demand for vectors required for large-scale clinical trials and commercialization.

AAV vector diagram. Created on BioRender.

To address these critical obstacles, we aims to develop a novel cost-effective and highly scalable AAV vector production platform. Our goal is to engineer a solution that can revolutionize the manufacturing of this crucial gene therapy vector, making it more accessible and affordable for broader therapeutic development.

More information to come.


Key Researchers: