Technology ID
TAB-2953

Modified AAV5 Vectors for Enhanced Transduction and Reduced Antibody Neutralization

E-Numbers
E-097-2015-0
Lead Inventor
Chiorini, John (Jay) (NIDCR)
Co-Inventors
Afione-Wainer, Sandra (NHLBI)
Agbandje-Mckenna, Mavis (University of Florida)
Halder, Sujata (University of Florida)
Applications
Therapeutics
Therapeutic Areas
Oncology
Immunology
Geriatrics
Endocrinology
Development Stages
Pre-Clinical (in vitro)
Development Status
  • Early-stage
  • In vivo data available (animal)
Lead IC
NIDCR
ICs
NIDCR
NHLBI
Scientists at the NIH disclosed a mutated adeno-associated virus (AAV) serotype 5 by modifying sialic acid binding regions which mediate viral entry into host cells. Preliminary results from animal studies suggest that this modification can increase transduction by 3-4 folds in salivary glands and muscles, and can significantly decrease the potential of being neutralized by preexisting antibodies compared to the wild type AAV. Thus, the modified AAV5 vectors seem to be optimal for gene therapy. This invention overcomes two major issues in AAV-based gene therapy: the ability to efficiently transduce the target cells and the ability to evade the immune response to vectors.
Commercial Applications
  • Genetically engineered AAV5 vectors for gene therapy.
Competitive Advantages
  • Enhanced transduction activity.
  • Reduced the potential for being neutralized by preexisting antibodies.
Licensing Contact:
Knezevic, Vladimir
vlado.knezevic@nih.gov