AAV-Aquaporin-1 Gene Therapy for Sjogren's Syndrome

Sjögren's syndrome is a chronic inflammatory disease affecting over 2 million Americans, whereby moisture-producing glands are attacked by the body's immune system. The disease is marked by disabling dryness of the mouth and eyes as well as fatigue and pain. Researchers at the National Institute of Dental and Craniofacial Research have developed a therapy that alleviates xerostomia in an animal model of Sjögren's syndrome. This technology consists of local delivery of adeno-associated virus (AAV) mediated aquaporin-1 (AQP1) fusion protein to salivary glands. Using a murine model that mimics Sjögren's dry mouth symptoms, it was discovered that treatment restored salivary fluid movement upon expression of AQP1. Targeted delivery of the AAV-AQP1 system makes this invention a novel and potential long-term therapeutic for restoration of exocrine gland function and prevention of xerostomia-associated pain associated with Sjögren's syndrome.

Potential Commercial Applications: Competitive Advantages:
  • Prevention of dry mouth (xerostomia) associated with salivary gland dysfunction in patients with Sjögren's syndrome.
  • AAV gene transfer to salivary glands is highly efficient.
  • AAV-AQP1 promotes de novo salivary flow.

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

Related Invention(s):


John Chiorini (NHLBI)  ➽ more inventions...

Intellectual Property:
US Pat: 10,166,299 issued 2019-01-01
PCT Application No. PCT/US13/57632 filed on 2013-08-30
US Application No. 14/423,774 filed on 2015-02-25

Collaboration Opportunity:

The National Institute of Dental and Craniofacial Research, AAV Biology Section, is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize AAV-Aquaporin-1 Gene Therapy for Sjögren's. 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-139-2011-1
Updated: May 1, 2019