A Novel Adeno-Associated Virus for Gene Therapy


Scientists at the NIH disclosed a novel adeno-associated virus (AAV) termed "44-9." AAV44-9 based vectors have high gene transfer activity in a number of cell types, including salivary gland cells, liver cells, and different types of neurons (e.g., cells of the cortex, olfactory bulb, and brain stem, and Purkinje cells of the cerebellum). These vectors can increase the transduction efficiency and decrease the potential of being neutralized by preexisting antibodies compared to the wild type AAV. Preliminary results from animal studies suggest that AAV44-9 vectors can efficiently deliver genes of interest, and the protein products of the delivered genes can be detected in bloodstream and at the local tissues. Therefore, these vectors are suitable for gene therapy for cells/tissues that are not efficiently targeted by other vectors.

Potential Commercial Applications: Competitive Advantages:
  • AAV44-9 can be used as a delivery vector in gene therapy.
 
  • High gene transfer activity in a number of cell types including salivary gland cells, liver cells, and different types of neurons (e.g., cells of the cortex, olfactory bulb, and brain stem, and Purkinje cells of the cerebellum).
  • As a gene transfer vector for cells that are not efficiently targeted by other vector.


Development Stage:
  • In vitro data available
  • In vivo data available (animal)


Related Invention(s):


E-308-2001-0
E-105-2003-0
E-329-2003-0
E-227-2004-0
E-179-2005-0
E-194-2010-0
E-087-2011-0
E-142-2011-0
E-232-2011-1
E-736-2013-1
E-097-2015-0


Inventors:

John Chiorini (NHLBI)  ➽ more inventions...

Giovanni Di Pasquale (NIDCR)  ➽ more inventions...


Intellectual Property:
US Application No. 62/160,552

Publications:
Schmidt M, et al. PMID 16641301

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 AAV44-9 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-175-2015-0
Updated: May 1, 2019