Pigment Epithelium-derived Factor as a Therapeutic Agent for Vascular Leakage, and a Peptide or Small Molecule Surrogate as Therapeutic Agent for Vascular Leakage and Neovascularization

Increased vascular leakage is a common feature of a number of disease states, including but not limited to, diabetic retinopathy, diabetic nephropathy, nephrotic syndrome, ascites, and rheumatoid arthritis. Pigment Epithelium-derived Factor is a known protein found in the eye and in the bloodstream, capable of stimulating nerve growth and preventing new blood vessel formation. The routes of administration of PEDF, a 418 amino acid protein, is limited. For example, oral administration of PEDF will lead to its rapid degradation in the digestive tract. JHU researchers have discovered a 44 amino acid portion (about 10% of the 418 amino acid full-length PEDF protein) is capable of preventing vascular leakage and neovascularization. In addition to the above findings, four amino acid residues within PEDF have been discovered as important for both of these biological activities; these four amino acid residues represent the active site of this therapeutically important protein. Description (Set) �PEDF effectively prevents pathologic vascular leakage in diabetic retinopathy. �Discovery of the specific peptide with both anti-angiogenic and anti-vasopermeability bioactivities gives greater latitude to the possible routes of administration. �The identified active site is critical for the rational drug design of small molecules to mimic the active site. Proposed Use (Set) PEDF is a potential therapeutic agent for the treatment of diabetic retinopathy, diabetic nephropathy, nephrotic syndrome, ascites, and rheumatoid arthritis. JHU researchers used the eye as a model system, and showed that PEDF effectively prevents pathologic vascular leakage in diabetic retinopathy. The discovery of the speicifc peptide possessing both anti-angiogenic and anti-vasopermeability bioactivities give greater latitude to the possible routes of administration. In addition, the identification of the anti-angiogenic and anti-vasopermeability active site is critical for the rational drug design of small molecules to mimic the active site Patent (Set) WO 2005/041887

Inventor(s): Tong, Patrick

Type of Offer: Licensing



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