Modified AAV5 Vectors for Enhanced Transduction and Reduced Antibody Neutralization


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.

Potential Commercial Applications: Competitive Advantages:
  • Genetically engineered AAV5 vectors for gene therapy.
 
  • Enhanced transduction activity.
  • Reduced the potential for being neutralized by preexisting antibodies.


Development Stage:
  • Early-stage
  • In vivo data available (animal)


Related Invention(s):




E-329-2003-0
E-227-2004-0



E-142-2011-0
E-232-2011-1
E-736-2013-1
E-175-2015-0


Inventors:

John Chiorini (NHLBI)  ➽ more inventions...

Sandra Afione-Wainer (NIDCR)  ➽ more inventions...

Mavis Agbandje-Mckenna

Sujata Halder


Intellectual Property:
U.S. Pat: 10,081,659 issued 2018-09-25
US Application No. 15/092,482

Publications:
Afione S, et al. PMID 25410855
Chiorini J, et al. US Patent 6,468,524
Chiorini J, et al. US Patent 6,984,517
Chiorini J, et al. US Patent 7,479,554
Chiorini J, et al. US Patent 6,855,314

Collaboration Opportunity:

The National Institute of Dental and Craniofacial Research is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize modified AAV5 vector for gene therapy. For collaboration opportunities, please contact David Bradley, Ph.D. at bradleyda@nidcr.nih.gov.


Licensing Contact:
Vladimir Knezevic, M.D.
Email: vlado.knezevic@nih.gov
Phone: 301.443.5560

OTT Reference No: E-097-2015-0
Updated: May 1, 2019