Improved Large Scale Production of Adeno-Associated Viruses

Technology #15803

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Researchers
Nathalie Clement
Barry John Byrne
Laura A. Adamson
Managed By
April Kilburn
Assistant Director 352-392-8929

Enables a More Efficient and More Potent Production of AAV Vectors

This production of highly concentrated and potent Adeno-Associated Virus (AAV) vectors has significant advantage over available protocol and allows for large-scale manufacturing of the virus. More than 6 percent of all gene therapy clinical trials worldwide use AAV vectors due to their ability to transfer genetic material to non-dividing cells and apparent lack of a human immune response. Unfortunately, AAV vectors are expensive because they only can be manufactured in small batches. University of Florida researchers have used a suspension format to produce high quality AAV vectors that are significantly more potent than existing vectors and allow for large-scale manufacturing.

Application

Large-scale production of highly potent AAV vectors

Advantages

  • Allows for large-scale manufacturing, promoting the design of comprehensive and exhaustive pre-clinical and clinical studies
  • Promotes large-scale clinical research, potentially impacting many genetic diseases in need of efficient therapeutic protocol
  • Takes less time and work to produce, significantly reducing cost per unit of drug and making it more accessible to the patient population

Technology

AAV vectors are typically produced using transfection or infection on an adherent flat platform, which limits scale-up manufacturing. By changing this protocol, University of Florida researchers were able to produce a higher yield of a more potent vector particle in a format suitable to scale up, both for research and commercial goals.