108 results about "Macula lutea degeneration" patented technology

This disclosure features chemical entities (e.g., a compound or a pharmaceutically acceptable salt thereof) that degrade and / or otherwise modulate (e.g., inhibit) NIMA Related Kinase 7 (NEK7). Said chemical entities are useful, e.g., for treating a subject (e.g., a human subject) having one or more disorders or diseases associated with NLRP3 inflammasome activation. Said disorders or diseases include but are not limited to, autoinflammatory and autoimmune disorders (e.g., gout, inflammatory bowel disease, rheumatoid arthritis, multiple sclerosis), neurodegenerative diseases (e.g., Alzheimer's disease, Parkinson's disease), cardiovascular and metabolic disorders (eg. pericarditis, atherosclerosis, Type 2 diabetes, obesity, and metabolic syndrome), fibrotic disorders (e.g. interstitial lung disease, chronic kidney disease), hematology (eg. anemia of inflammation) and eye disorders (eg. macular degeneration). In embodiments, and while not wishing to be bound by theory, it is believed that the chemical entities described herein directly target (e.g., directly bind to) NEK7, thereby altering (e.g., attenuating) the inflammatory response modulated by the NLRP3 inflammasome. This disclosure also features compositions containing the same as well as methods of using and making the same.

The invention belongs to the technical field of nano-drugs, and discloses a multifunctional nano-drug delivery system for treating neovascular eye diseases and a preparation method of the multifunctional nano-drug delivery system. The nano drug delivery system disclosed by the invention is a composite nano preparation which takes dendritic mesoporous silica nanoparticles as a carrier and co-loads cerium zirconium oxide nano enzyme and an anti-VEGF (vascular endothelial growth factor) drug, various active oxygen can be efficiently removed, oxygen is continuously released through catalytic decomposition of hydrogen peroxide, a retina hypoxia microenvironment is improved, and the retina hypoxia effect is improved. According to the present invention, the anti-VEGF drug delivery system can reduce the oxidative stress induced retinal pigment epithelial cell apoptosis rate, reduce the inflammatory reaction, and simultaneously achieve the slow release and the controlled release of the anti-VEGF drug, and the drug effect maintenance time can achieve more than 60 days, such that the multi-target synergistic treatment can be achieved through the synergistic regulation of the multiple pathological links such as oxygen deficit-oxidative stress-inflammation-angiogenesis, and the anti-VEGF drug delivery effect can be achieved. The choroidal neovascularization can be effectively inhibited by single intravitreal injection, and a new strategy of multi-target collaborative treatment is provided for neovascular macular degeneration and other eye diseases.

The present invention relates to a fusion protein comprising an anti-C3b antibody or an anti-C5 antibody and an angiogenesis inhibitor, and a composition for treating eye diseases, particularly macular degeneration, by using the same. The protein may not only inhibit complement-related pathways, but can also efficiently regulate angiogenesis. Therefore, the fusion protein dimer can be effectively used to treat and prevent complement-related diseases, particularly eye diseases such as macular degeneration, and thus has high industrial applicability.

The invention relates to an adeno-associated virus vector for treating yolk-like macular degeneration. Aspects of the present disclosure relate to methods and compositions useful for the treatment of yolk-like macular dystrophy, such as yolk-like macular degeneration.

Disclosed herein are compositions and methods for the modulation of 3-hydroxy-3-methylglutaryl-CoA reductase (HMGCR) expression, wherein the compositions include an siRNA duplex or a conjugated siRNA duplex. Also disclosed herein are methods, and compositions for use in methods, of treating age-related macular degeneration (AMD) or hyperlipidemia. The compositions disclosed herein comprise an siRNA duplex that comprises a sense sequence and an antisense sequence, wherein the sense sequence and the antisense sequence are at least partially complementary to each other.

The present disclosure relates to an orthogonal system comprising a first bi-specific polypeptide that comprises D-streptavidin or a variant thereof covalently linked to an antibody or antibody fragment and a second bi-specific polypeptide that comprises L-biotin covalently linked to a therapeutic or diagnostic agent. The disclosed systems can be useful in, for example, treating a disease or a condition (e.g., cancer, non-Hodgkin lymphoma, multiple sclerosis, Crohn's disease, rheumatoid arthritis, asthma, macular degeneration, psoriasis, Hodgkin lymphoma, paroxysmal nocturnal hemoglobinuria, X-linked hypophosphatemia). Also described are peptides and polypeptides useful in preparing the disclosed bi-specific polypeptides and methods of making same. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

Provided are an anti-VEGF and complement bifunctional fusion protein, and a use thereof. The fusion protein comprises a VEGF binding domain, a human IgG Fc domain, and a complement binding domain, and has high expression, high activity, and stable properties. The protein can simultaneously effectively bind VEGF factors and complement molecules, exhibits stronger anti-VEGF and anti-complement effects, and has a better treatment effect on age-related macular degeneration.

The present invention relates to compounds of formula 1: # imgabs0 # and their use as STING antagonists, for example, the composition is used for treating diseases selected from the group consisting of systemic lupus erythematosus (SLE), (single and double gene) interferon diseases (including STING-related vascular disease (SAVI), Aicardi-Goutires syndrome (AGS), COPA syndrome and familial chilblain-like lupus, DNASE2 or ATAD3A gene mutated type I interferon disease, age-related macular degeneration (AMD), retinopathy, glaucoma, lupus erythematosus, lupus erythematosus, lupus erythematosus, lupus erythematosus, lupus erythematosus, lupus erythematosus, lupus erythematosus, lupus erythematosus, lupus erythematosus, lupus erythematosus, and lupus erythematosus. Amyotrophic lateral sclerosis (ALS), diabetes, obesity, inflammatory bowel disease (IBD), chronic obstructive pulmonary disease (COPD), Bloom syndrome, sicca syndrome, Parkinson's disease, heart failure and cancer, systemic sclerosis (SSc), dermatomyositis, non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), chronic plus acute liver failure (ACLF), interstitial lung disease (ILD), idiopathic pulmonary fibrosis (IPF), and the like. , aging / muscle disease, sepsis, heart failure, rheumatoid arthritis, and osteoarthritis.

The invention discloses application of cnidium lactone in preparation of a medicine for treating AMD or corneal injury, and belongs to the field of biological medicine. It is found for the first time that the cnidium lactone can inhibit sodium iodate induced retinal pigment epithelial cell injury, and it is also found for the first time that the cnidium lactone can promote injured cornea repair; based on the discovery, the invention respectively provides application of cnidium lactone in preparation of medicines for treating age-related macular degeneration and preparation of medicines for treating corneal injury. Experimental results show that cnidium lactone can remarkably reverse the retinal pigment epithelial cell degradation trend caused by age-related macular degeneration and can also remarkably promote corneal notch healing. The invention provides a new optional medicine for treating age-related macular degeneration and corneal injury, expands the application range of cnidium lactone, and has outstanding clinical practical value and wide application prospect.

This disclosure features chemical entities (e.g., a compound or a pharmaceutically acceptable salt thereof) that degrade and / or otherwise modulate (e.g., inhibit) NIMA Related Kinase 7 (NEK7). Said chemical entities are useful, e.g., for treating a subject (e.g., a human subject) having one or more disorders or diseases associated with NLRP3 inflammasome activation. Said disorders or diseases include but are not limited to, autoinflammatory and autoimmune disorders (e.g., gout, inflammatory bowel disease, rheumatoid arthritis, multiple sclerosis), neurodegenerative diseases (e.g., Alzheimer's disease, Parkinson's disease), cardiovascular and metabolic disorders (eg. pericarditis, atherosclerosis, Type 2 diabetes, obesity, and metabolic syndrome), fibrotic disorders (e.g. interstitial lung disease, chronic kidney disease), hematology (eg, anemia of inflammation) and eye disorders (eg. macular degeneration). In embodiments, and while not wishing to be bound by theory, it is believed that the chemical entities described herein directly target (e.g., directly bind to) NEK7, thereby altering (e.g., attenuating) the inflammatory response modulated by the NLRP3 inflammasome. This disclosure also features compositions containing the same as well as methods of using and making the same.

This disclosure relates to RNAi agents capable of inhibiting complement factor B (CFB) gene expression. Pharmaceutical compositions containing CFB RNAi agents and methods of use thereof are also disclosed. The CFB RNAi agents disclosed herein may be conjugated to a targeted ligand containing an N-acetyl-galactosamine ligand to facilitate in vivo delivery to hepatocytes. RNAi agents can be used in methods of treating diseases, disorders, or conditions partially mediated by CFB gene expression, including IgA nephropathy (IgAN), C3 glomerulopathy (C3G), immune complex-mediated membrane proliferative glomerulonephritis (IC-MPGN), lupus nephritis (LN), anti-glomerular basement membrane antibody disease (anti-GBM), ischemia-reperfusion injury and T-cell-mediated rejection in kidney transplantation (TCMR), anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis, age-related macular degeneration (AMD) including early and / or intermediate-stage AMD, geographic atrophy (GA), glaucoma, Doyne honeycomb retinal dystrophy, paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), pre-eclampsia, rheumatoid arthritis (RA), and / or other complement-mediated disorders.

This disclosure features chemical entities (e.g., a compound or a pharmaceutically acceptable salt thereof) that degrade and / or otherwise modulate (e.g., inhibit) NIMA Related Kinase 7 (NEK7). Said chemical entities are useful, e.g., for treating a subject (e.g., a human subject) having one or more disorders or diseases associated with NLRP3 inflammasome activation. Said disorders or diseases include but are not limited to, autoinflammatory and autoimmune disorders (e.g., gout, inflammatory bowel disease, rheumatoid arthritis, multiple sclerosis), neurodegenerative diseases (e.g., Alzheimer's disease, Parkinson's disease), cardiovascular and metabolic disorders (eg. pericarditis, atherosclerosis, Type 2 diabetes, obesity, and metabolic syndrome), fibrotic disorders (e.g. interstitial lung disease, chronic kidney disease), hematology (eg. anemia of inflammation) and eye disorders (eg. macular degeneration). In embodiments, and while not wishing to be bound by theory, it is believed that the chemical entities described herein directly target (e.g., directly bind to) NEK7, thereby altering (e.g., attenuating) the inflammatory response modulated by the NLRP3 inflammasome. This disclosure also features compositions containing the same as well as methods of using and making the same.

Described herein are antibodies that bind to VEGF_A or ANG-2, or bispecific antibodies that bind to VEGF_A and ANG-2. The antibodies of the present disclosure are useful for treating eye diseases, including macular degeneration.

Compositions and methods are described for the delivery of a fully human post-translationally modified (HuPTM) monoclonal antibody (“mAb”) or the antigen-binding fragment of a mAb against human vascular endothelial growth factor (“hVEGF”)—such as, e.g., a fully human-glycosylated (HuGly) anti-hVEGF antigen-binding fragment—to the retina / vitreal humour in the eye(s) of human subjects diagnosed with ocular diseases caused by increased neovascularization, for example, neovascular age-related macular degeneration (“nAMD”), also known as “wet” age-related macular degeneration (“WAMD”), age-related macular degeneration (“AMD”), and diabetic retinopathy.

Novel anti-C3 and anti-C5 antibodies capable of modulating complement activity by specifically binding to human complement factor C3, C3a, C3b, and / or C5 are provided. Also described herein are recombinant AAV (rAAV) comprising a variant adeno-associated virus (AAV) capsid and a transgene encoding an anti-C3 and / or anti-C5 antibody are provided. Also provided are methods of delivering the transgene to the retina and methods of treating dry age-related macular degeneration and geographic atrophy secondary to age-related macular degeneration disorders by contacting retinal cells with the rAAV)

In certain aspects, provided herein are RNA complexes that inhibit Myeloid differentiation primary response gene 88 (MyD88) and / or Toll-like receptor 3 (TLR3) and are useful in the treatment of age-related macular degeneration (AMD). In certain aspects, provided herein are pharmaceutical compositions comprising such RNA complexes and methods of using such RNA complexes and pharmaceutical compositions.

The present invention addresses the problem of providing a protective agent for retinal pigment epithelial cells, which is useful for the prevention or treatment of age-related macular degeneration. A protective agent for retinal pigment epithelial cells according to the present invention, which is a solution for the problem, contains, as an active ingredient, a peptide in which a protein transduction domain is attached to the N-terminus of a peptide having an inhibitory effect on mitochondrial calpains. A specific example of the peptide having an inhibitory effect on mitochondrial calpains is a peptide comprising the amino acid sequence (PDALKSRTJR) represented by SEQ ID NO: 1 (wherein J represents L or I). A specific example of the protein transduction domain is a peptide comprising an amino acid sequence that is at least a part of the amino acid sequence for the transduction domain of HIV-1 Tat.

The present invention relates to a composition for preventing and alleviating or treating glaucoma and macular degeneration, containing a Centella asiatica extract. Particularly, the composition increases the glucose metabolism efficiency of cells so as to increase the survival rate of retinal neurons and pigment epithelial cells and protects cells from oxidative damage caused by A2E, thereby being effectively usable as a composition for eye health and for preventing and alleviating or treating glaucoma and macular degeneration.

The present invention provides methods and compositions for treating and / or preventing age related macular degeneration and other conditions involving macular degeneration, ocular neovascularization, or ocular inflammation. In an exemplary embodiment, a method is disclosed that involves administering an expression vector that delivers a secretable and cell penetrating CARD to a subject in need of treatment or prevention of age-related macular degeneration or another condition involving macular degeneration or ocular neovascularization.

The present invention relates to a nanoparticle comprising a nanomaterial and at least a first ligand and a second ligand tethered to the nanoparticle. The present invention also relates to a nanoparticle for use as a medicament or diagnostic agent. The present invention also relates to a nanoparticle for use in a method for preventing or treating a disease selected from the group consisting of diabetic nephropathy, glomerulonephritis, glomerular VEGF A dysregulation, endothelial VEGF A dysregulation, diabetic retinopathy, rheumatoid arthritis, age-related macular degeneration and cancer, such as breast cancer. Furthermore, the present invention relates to a method of preparing a nanoparticle.

The present invention relates to peptides that have preventive, ameliorative, or therapeutic effects against amyloidosis and / or macular degeneration, are safe for living organisms, and have few side effects including abnormal reactions, as well as pharmaceutical compositions and health functional foods containing the same.

A method for treating macular degeneration includes administering a composition comprising an effective amount of Aucuba japonica extract to a subject in need thereof. The composition may prevent or treating macular degeneration. The macular degeneration may be age-related macular degeneration. The Aucuba japonica extract may be extracted from a 5 leaf, a stem or a mixture thereof of Aucuba japonica.

The present disclosure relates to RNAi agents able to inhibit Complement Factor B (CFB) gene expression. Also disclosed are pharmaceutical compositions that include CFB RNAi agents and methods of use thereof. The CFB RNAi agents disclosed herein may be conjugated to targeting ligands, including ligands that comprise N-acetyl-galactosamine, to facilitate the in vivo delivery to hepatocyte cells. The RNAi agents can be used in methods of treatment of diseases, disorders, or symptoms mediated in part by CFB gene expression, including IgA nephropathy (IgAN), C3 glomerulopathy (C3G), immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN), lupus nephritis (LN), Anti-Glomerular Basement Membrane disease (anti-GBM), ischemia reperfusion injury and T-cell mediated rejection (TCMR) in kidney transplantation, anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis, age-related macular degeneration (AMD), including early and / or intermediate AMD, geographic atrophy (GA), glaucoma, Doyne honeycomb retinal dystrophy, paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), pre-eclampsia, rheumatoid arthritis (RA), and / or other complement-mediated diseases.

The described invention provides method for treating a complement disorder such as age-related macular degeneration (AMD) in a subject, comprising administering a pharmaceutical composition into an affected eye of a subject by ocular injection, wherein the composition comprises a nucleic acid encoding a soluble CD59 (sCD59) protein operably linked to a promoter, wherein the nucleic acid encoding sCD59 is packaged into a delivery vector and wherein the administering results in expression and secretion of the sCD59 protein by cells of the affected eye and the expression results in treatment of affected cells in the affected eye.

Described herein are retinoid compositions comprising (a) a retinoid preferably a C20-deuterated vitamin A that inhibits dimerisation to N-retinylidene-N-retinylethanolamine (A2E), (b) a lipid excipient preferably an omega-3 fatty acid comprising eicosapentaenoic acid (EPA) or docosahexaenoic acid (DHA), and (c) a surfactant, wherein the compositions increase the bioavailability of the retinoid in a subject, and methods of using such retinoid compositions for treating eye conditions including macular degeneration.

This invention relates to a kit and method for detecting pathogenic genes of hereditary macular degeneration (AMD). The kit includes a hybridization mixture containing a probe set for detecting AMD pathogenic genes. The probe set includes capture probes capable of simultaneously and specifically capturing pathogenic genes ABCA4, BEST1, PRPH2, ELOVL4, PROM1, IMPG1, IMPG2, EFEMP1, RP1L1, and TIMP3, as well as the pathogenic regions of MCDR3 and MCDR1. The capture probes capturing the pathogenic regions of MCDR3 and MCDR1 include probes with sequences shown in SEQ ID NO. 1-174 and SEQ ID NO. 175-235, respectively. This invention proposes a targeted amplification strategy for the non-coding pathogenic region of NCMD, successfully detecting two novel point mutations and one genomic structural variation, achieving the detection of point mutations and structural variations in non-coding regions.

An implantable device (100) comprising a substrate capable of capturing an intraocular target molecule and a substrate for use in the treatment of dry macular degeneration.

The present disclosure relates to a compound of Chemical Formula I or II, a solvate, a stereoisomer or a pharmaceutically acceptable salt thereof. Further, the present disclosure relates to a pharmaceutical composition for preventing or treating various ophthalmic diseases such as diabetic retinopathy, glaucoma, uveitis, wet and dry macular degeneration, and age-related macular degeneration, including the compound of Chemical Formula I or II, the solvate, the stereoisomer, or the pharmaceutically acceptable salt thereof as an active ingredient. A novel low molecular compound of the present disclosure exhibits an excellent effect in preventing and improving various ophthalmic diseases caused by retinal degeneration by inhibiting BET proteins to improve inflammation caused by retinal damage caused by epigenetic changes.

Provided are modified u-PA polypeptides and fusion proteins containing the modified u-PA polypeptides. The u-PA polypeptides are modified to have altered activity and / or specificity so that they cleave a complement protein, such as complement protein C3, to thereby inhibit complement activation. The modified u-PA polypeptides and fusion proteins that inhibit complement activation can be used for treatment of diseases and conditions that are mediated by complement activation, or in which complement activation plays a role. These disorders include ischemic and reperfusion disorders, including myocardial infarction and stroke, sepsis, autoimmune diseases, diabetic retinopathies, age-related macular degeneration, transplanted organ rejection, inflammatory diseases and diseases with an inflammatory component.

The invention discloses an application of CRX in inhibition of epithelial-mesenchymal transition of retinal pigment epithelial cells. The invention discloses an application of CRX in preparation of a medicine as shown in (1) or (2). The medicine comprises a carrier for over-expressing CRX; (1) a drug for inhibiting epithelial-mesenchymal transition of retinal pigment epithelial cells; and (2) a medicine for preventing and / or treating age-related macular degeneration subretinal fibrosis. The invention finds that the expression of E-cadherin and Occludin can be promoted by overexpressing CRX in RPE cells, so that the epithelial-mesenchymal transition of the RPE cells is inhibited, and the CRX can be used for preventing or treating age-related macular degeneration subretinal fibrosis and also can be used for preventing or treating highly myopia retinal degeneration.