AAV Mediated CTLA-4 Gene Transfer to Treat Sjogren's Syndrome

Sjögren’s syndrome is an autoimmune disease that affects over 2 million Americans, primarily over the age of 40. One of the major outcomes of Sjögren's syndrome is xerostomia (dry mouth) that is caused by immune system attack on moisture producing salivary glands. Researchers at the National Institute of Dental and Craniofacial Research have developed a therapy that alleviates xerostomia in a murine model of Sjögren's syndrome. This technology consists of a local delivery of adeno-associated virus (AAV) mediated cytotoxic T-lymphocyte antigen 4 Immunoglobulin-G (CTLA4IgG) fusion protein to salivary glands. The system effectively blocks CTLA4 ligand interactions with T cell surface receptors, resulting in immune suppression and reversal of autoimmune-related xerostomia. Targeted delivery of the AAV-CTLA4-IgG system makes this invention a novel therapeutic for the prevention of xerostomia-associated pain and discomfort caused by Sjögren's syndrome.

Potential Commercial Applications: Competitive Advantages:
Prevention of salivary gland destruction and xerostomia development in patients with Sjögren's syndrome. 
  • Current treatments temporarily reduce the discomfort of xerostomia but do not prevent the deleterious effects of this disorder.
  • AAV gene transfer to salivary glands is highly efficient.
  • AAV therapy is safe and noninflammatory.

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


Hongen Yin (NIDCR)  ➽ more inventions...

John Chiorini (NIDCR)  ➽ more inventions...

Intellectual Property:
US Application No. 61/476,168
PCT Application No. PCT/US12/33556
US Application No. 14/111,604

Zheng C, et al. PMID 21625005
Kanaya K, et al. PMID 12589145
Genovese MC, et al. PMID 16162882

Collaboration Opportunity:

The NIDCR is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize this technology. For collaboration opportunities, please contact David Bradley at bradleyda@nidcr.nih.gov.

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

OTT Reference No: E-087-2011-0
Updated: Nov 18, 2011