Improved therapy for AMD Age-related macular degeneration (AMD) is a leading cause of visual loss (www.nei.nih.gov/eyedata/pbd4.asp) affecting more than 1.75 million people in the USA. Vascular endothelial growth factor (VEGF) is a major driver of choroidal neovascularisation, a central process in the pathogenesis of wet AMD. FDA approval of inhibitors of VEGF has revolutionized therapy for this serious disease. However, such therapies require repeated, monthly injections and are associated with significant morbidity. We have developed an AAV-based inducible gene transfer system for local delivery of therapeutic antibodies (Fang, 2006, 2007). Preliminary work demonstrates tightly regulated, long-term (>1 yr.), high level expression (>1 mg/ml) of an antibody suitable for therapy of AMD. The overall goal of the present application is to utilize our rapamycin-regulated AAV system to deliver an anti-VEGF antibody into the eye. This novel therapeutic approach is expected to eliminate monthly injections, reduce neovascularization and curtail side effects (e.g. endophthalmitis) of present therapies.

Public Health Relevance

Improved therapy for AMD Age-related macular degeneration (AMD) is a leading cause of visual loss (www.nei.nih.gov/eyedata/pbd4.asp) affecting more than 1.75 million people in the USA. FDA approval of inhibitors of vascular endothelial growth factor (VEGF) has revolutionized therapy for this serious disease. However, such therapies require repeated, monthly injections that can have serious side effects. We have developed an improved therapy that will require only one treatment.

Agency
National Institute of Health (NIH)
Institute
National Eye Institute (NEI)
Type
Small Business Innovation Research Grants (SBIR) - Phase I (R43)
Project #
1R43EY019794-01A1
Application #
7805653
Study Section
Special Emphasis Panel (ZRG1-ETTN-E (12))
Program Officer
Wujek, Jerome R
Project Start
2010-03-01
Project End
2012-02-28
Budget Start
2010-03-01
Budget End
2012-02-28
Support Year
1
Fiscal Year
2010
Total Cost
$200,786
Indirect Cost
Name
Panorama Research, Inc.
Department
Type
DUNS #
556962439
City
Sunnyvale
State
CA
Country
United States
Zip Code
94089