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Adeno-Associated Virus Gene Therapy for Diabetes and Obesity

Posted Nov 15 2012 7:00pm

Description of Invention:
This invention is directed to adeno-associated virus (AAV) vector delivery of exendin-4 (Ex-4) to salivary glands as treatment for diabetes and obesity. Ex-4 is a potent and long-acting agonist of the receptor for glucagon-like peptide 1 (GLP-1). Scientists at NIDCR have shown that AAV-mediated delivery of Ex-4 resulted in improved glucose homeostasis and weight profile in two rat models of obesity and type 2 diabetes. Further, AAV-mediated delivery of Ex-4 to rat salivary glands resulted in localized and sustained expression of Ex-4 that was biologically active and well tolerated.

AAV-mediated delivery of Ex-4 is superior to administering GLP-1 analogs in that AAV-Ex-4 expression is more stable and longer acting. Like GLP-1 analogs, Ex-4 expression also potentially provides beneficial effects like reduced hypoglycemia, appetite suppression, and potential weight loss.

Therapy for diabetes or obesity.

  • Potential for potent glucose homeostasis therapy with longer duration than current drugs.
  • More convenient than daily or weekly injections.

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

John A Chiorini (NIDCR)
Giovanni Di Pasquale (NIDCR)
Edward Mannucci (Careggi Teaching Hospital)

Patent Status:
HHS, Reference No. E-142-2011/0
PCT, Application No. PCT/US12/34268 filed 19 Apr 2012

Relevant Publication:
  1. Di Pasquale G, et al. [ PMID 22808093 ]

Collaborative Research Opportunity:
The NIDCR is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize treatment of diabetes by expression NGF-extendin 4 protein. For collaboration opportunities, please contact David Bradley, Ph.D. at 301-402-9242 or .

For Licensing Information Please Contact:
Lauren Nguyen-Antczak Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Phone: 301-496-7057
Fax: 301-402-0220

Ref No: 2499

Updated: 11/2012

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