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Home » Research at NEI » Unit on Ocular Gene Therapy » Peter Colosi, Ph.D.

Peter Colosi, Ph.D.
Unit on Ocular Gene Therapy

Building 6, Room 331B
6 Center Drive
Bethesda, Maryland 20892-0610
Phone: (301) 594-5376
Fax: (301) 480-9917

Mission Statement

The ocular gene therapy laboratory was established to develop gene-based therapeutics for ocular diseases and to evaluate these novel drugs clinically. The laboratory also conducts research projects employing gene transfer technology to elucidate novel and exploitable mechanisms of pathology that cause ocular diseases. The purpose of this research is to identify new drug targets for the development of human therapeutics.

Research Overview

Prior to coming to the NEI, I directed research and development groups for 11 years at Avigen Inc., a small gene therapy company. During that time, groups under my direction developed several adeno-associated virus (AAV) vector-based gene therapeutics and invented a method to produce clinical-grade AAV gene therapy vectors. This work culminated in three Phase 1/2 clinical trials evaluating AAV vectors for the treatment of hemophilia B (clotting Factor IX deficiency) and Parkinsonís disease. I came to the NEI because I believe that ocular applications are among the most promising for AAV gene therapeutics and have the highest probability of clinical success. The laboratory has three major areas of interest: clinical development of AAV vector-based gene therapeutics, vector refinement including the reduction of preexisting immunity and small-molecule regulation of transgene expression, and basic research into the mechanisms of pathology of age-related macular degeneration and retinitis pigmentosa.

Our current areas of focus are:

Selected Publications

Department of Health and Human Services NIH, the National Institutes of Health