Technology Bundle ID: TAB-2339

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

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Primary Inventors: 
Hongen Yin (NIDCR)
John Chiorini (NIDCR)
Therapeutic Area: 
Development Status: 
  • In vitro data available
  • In vivo data available (animal)
Institute or Center: 

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.


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.


PCT Application PCT/US12/33556
Filed on 2012-04-13
US Application 14/111,604
Filed on 2014-01-13
US Application 61/476,168
Filed on 2011-04-15


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


Nov 18, 2011

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