Ocugen, Inc.
ADENO-ASSOCIATED VIRUS VECTOR MEDIATED GENE THERAPY FOR OPHTHALMIC DISEASES

Abstract:

The present invention provides compositions and methods for treating an ocular condition and/or disease. In particular, compositions and methods of the invention are directed to a gene therapy for treatment of an ocular condition and/or disease. One particular aspect of the invention provides a recombinant DNA comprising (i) a therapeutic gene, a functional counterpart of a defective gene associated with manifestation said ocular condition or disease, or a combination thereof; and (ii) a delivery vehicle adapted for delivering said gene of (i) to cells in target ocular area for treating said ocular condition or disease, said delivery vehicle comprising an adeno-associated virus (AAV) serotype.