# US 2020/0399361 A1 · Therapy for glaucoma and optic neuropathy by targeting colony stimulating factors > Agents: this data is available as JSON from the patentgrep API and MCP server — see https://patentgrep.com/llms.txt - Number: US 2020/0399361 A1 - Document ID: US20200399361A1 - Status: Published application - Kind: A1 - Application number: 16/977,783 - Filed: 2019-03-05 - Published: 2020-12-24 - Earliest claimed: 2018-03-05 - Assignees: The Schepens Eye Research Institute, Inc. - Applicants: The Schepens Eye Research Institute, Inc. - Inventors: Ji Min; Kin-Sang Cho; Dong Feng Chen - Web page: https://patentgrep.com/patent/US20200399361A1 ## CPC - A61P 27/06: Antiglaucoma agents or miotics - C07K 16/243: {Colony Stimulating Factors} - A61K 9/0048: {Eye, e.g. artificial tears} - A61K 38/193: {Colony stimulating factors [CSF]} - A61K 39/3955: {against proteinaceous materials, e.g. enzymes, hormones, lymphokines} - A61K 45/00: Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00 - A61P 27/02: Ophthalmic agents - C07K 16/2866: {against receptors for cytokines, lymphokines, interferons} - A61K 9/0019: {Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner (non-active ingredients are additionally classified in A61K47/00)} - A61K 39/395: Antibodies (agglutinins A61K38/36 {; as drug carriers A61K47/50}); Immunoglobulins; Immune serum, e.g. antilymphocytic serum - A61K 45/06: Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca - A61K 2039/505: {comprising antibodies} - A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS - C07K: PEPTIDES (peptides in foodstuffs A23; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs A23J; preparations for medicinal purposes A61K; peptides containing beta-lactam rings C07D; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, C07D; ergot alkaloids of the cyclic peptide type C07D519/02; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids C08G69/00; macromolecular products derived from proteins C08H1/00; preparation of glue or gelatine C09H; single cell proteins, enzymes C12N; genetic engineering processes for obtaining peptides C12N15/00; compositions for measuring or testing processes involving enzymes C12Q; investigation or analysis of biological material G01N33/00) - A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D) ## Abstract The invention provides compositions and methods for treating optic neuropathic disorders. ## Claims ### Claim 1 A method for treating an optic neuropathic disorder in a subject comprising locally administering to the eye an inhibitor of colony stimulating factor-1 (CSF1) or a receptor thereof or by locally administering to the eye a colony stimulating factor-2 (CSF2) protein or polypeptide. ### Claim 2 (depends on 1) The method of claim 1, wherein said subject is diagnosed with glaucoma. ### Claim 3 (depends on 1) The method of claim 1, wherein said inhibitor or CSF2 polypeptide is administered intravitreally. ### Claim 4 (depends on 1) The method of claim 1, wherein said inhibitor comprises an antibody specific for CSF1 or a CSF1 receptor (CSF1R). ### Claim 5 (depends on 4) The method of claim 4, wherein a CSF1R inhibitor comprises PLX3397, GW-2580, BLZ-945, RG-7155, FPA-008, M279 or combinations thereof. ### Claim 6 (canceled) ### Claim 7 A method of preventing or treating an optic neuropathic disorder or a method of suppressing or inhibiting microglial in a subject comprising locally administering to the eye, a pharmaceutical composition comprising a therapeutically effective amount of an inhibitor of colony stimulating factor-1 (CSF1) or a receptor thereof and a colony stimulating factor-2 (CSF2) polypeptide, thereby preventing or treating the optic neuropathic disorder. ### Claim 8 (depends on 6) The method of claim 6, wherein the inhibitor of CSF1 or a receptor thereof and a CSF2 protein or polypeptide recombinant protein suppress microglial activation. ### Claim 9 (depends on 6) The method of claim 6, wherein the inhibitor of CSF1 or a receptor thereof and a CSF2 protein or polypeptide recombinant protein protect loss of retinal ganglion cells (RGCs) and vision function. ### Claim 10 (depends on 6) The method of claim 6, wherein the inhibitor of CSF1 or a receptor thereof, comprises antibodies, antibody fragments, aptamers, small molecules, antisense oligonucleotides, siRNA reagents, Fab, Fab′, F(ab′)2 fragments, Fv fragments, single chain antibodies, antibody mimetics, peptoids, cytokines, cellular factors, enzymes or combinations thereof. ### Claim 11 (depends on 6) The method of claim 6, wherein the pharmaceutical composition comprises an anti-CSF1 antibody and/or CSF1 receptor (CSF1R) inhibitor and a CSF2 recombinant peptide. ### Claim 12 (depends on 6) The method of claim 6, wherein a CSF1R inhibitor comprises PLX3397, GW-2580, BLZ-945, RG-7155, FPA-008, M279 or combinations thereof. ### Claim 13 A pharmaceutical composition comprising a therapeutically effective amount of an inhibitor of colony stimulating factor-1 (CSF1) or a receptor thereof and a colony stimulating factor-2 (CSF2) protein or polypeptide. ### Claim 14 (depends on 13) The pharmaceutical composition of claim 13, wherein the inhibitor of CSF1 or a receptor thereof and a CSF2 protein or polypeptide recombinant protein suppress microglial activation. ### Claim 15 (depends on 13) The pharmaceutical composition of claim 13, wherein the inhibitor of CSF1 or a receptor thereof and a CSF2 protein or polypeptide recombinant protein protect loss of retinal ganglion cells (RGCs) and vision function. ### Claim 16 (depends on 13) The pharmaceutical composition of claim 13, wherein the inhibitor of CSF1 or a receptor thereof, comprises antibodies, antibody fragments, aptamers, small molecules, antisense oligonucleotides, siRNA reagents, Fab, Fab′, F(ab′)2 fragments, Fv fragments, single chain antibodies, antibody mimetics, peptoids, cytokines, cellular factors, enzymes or combinations thereof. ### Claim 17 (depends on 13) The pharmaceutical composition of claim 13, wherein a CSF1R inhibitor comprises PLX3397, GW-2580, BLZ-945, RG-7155, FPA-008, M279 or combinations thereof. ### Claim 18 (canceled) ### Claim 19 (canceled) ### Claim 20 (canceled) ### Claim 21 (canceled) ### Claim 22 (canceled) ### Claim 23 (canceled) ### Claim 24 (canceled) ### Claim 25 (canceled) ### Claim 26 (canceled) ### Claim 27 (depends on 13) The composition of claim 13, wherein the formulation comprises: a solution, suspension, gel, mesh, implant or emulsion.