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WO/2024/097900A1 |
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-2325 and compositions, methods, and uses thereof.
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WO/2024/095005A1 |
The present invention provides compounds of formula (I) and pharmaceutically acceptable salts, N-oxides, solvates and prodrugs thereof: wherein V, W, X, Y, Z, A1, A2, A3, A4, A5, A6, A7, A8, A9 and R3 are as defined in the specification,...
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WO/2024/095026A1 |
In the present invention azidation is used to render a molecule photoactivable and to influence its binding to its cognate specific binding partner in the eye. Thus, the invention relates to a compound for use in a method of treating a s...
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WO/2024/094759A1 |
There is provided herein a composition comprising liposomes, which comprise a lipid component and a phospholipid component, which compositions are useful in the treatment of ocular surface disorders.
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WO/2024/096629A1 |
The present invention provides an eye drop composition for treating, preventing, or ameliorating dry eye syndrome or a dry eye-related disease, the eye drop composition comprising a compound represented by chemical formula 1 at a concent...
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WO/2024/069230A3 |
Embodiments of the disclosure provide ophthalmic compositions and methods of use thereof. The embodiments disclose ophthalmic compositions including devices such as a punctal plug. Some embodiments are directed to a method of preparing a...
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WO/2024/044745A3 |
The present disclosure provides pharmaceutical compositions that comprise a microparticle or nanoparticle suspension of a collagen IV-derived peptide or pharmaceutically-acceptable salt thereof, as well as uses of these suspensions for t...
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WO/2024/097439A1 |
A method of treating X-linked juvenile retinoschisis (XLRS) in a human subject includes subretinally delivering to the human subject a therapeutically effective amount of an rAAV vector. The rAAV vector includes a nucleic acid sequence c...
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WO/2024/095268A1 |
Methods for treating or reducing inflammation, in is in a site or an organ excluding the central nervous system of a subject in need thereof, by administering a pharmaceutical composition comprising the translocator protein ligand 2-C1-M...
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WO/2024/097861A1 |
Provided herein are methods of ameliorating a disease or disorder associated with dysregulation of the alternative complement pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.
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WO/2024/093907A1 |
Provided are a compound of formula (I) or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof. The compound of formula (I) is contained inside a nucleotide, or at a 5' end and/or a 3' end, and is used for improving the ...
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WO/2024/095003A1 |
The present invention provides compounds of formula (I) and pharmaceutically acceptable salts, N-oxides, solvates and prodrugs thereof: Formula (I) wherein Z, X, R1, R2, R4, R5, R6 and R7 are as defined in the specification, processes fo...
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WO/2024/090941A1 |
The present invention relates to a composition for the prevention, amelioration or treatment of an anterior segment eye disease, the composition comprising a plant extract of the genus Aloe as an active ingredient. When the Aloe vera ext...
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WO/2024/089691A1 |
The present disclosure relates to small molecule compound (SMC) modulators, compositions comprising the SMC modulators, uses thereof and methods for their use in modulating activity of CCR1 in a Muller cell and treating retinal disease.
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WO/2024/089296A1 |
The present invention is directed to novel modulators of the Ca2+-signaling cascade, acting as potentiators of the P2RY2 purinergic receptor and of the inositol triphosphate receptors, ITPRs. The compounds are useful in the treatment of ...
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WO/2024/091952A1 |
Disclosed herein are compositions comprising antioxidants and methods of use thereof.
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WO/2024/090606A1 |
The present invention provides a method for treating posterior scleritis, comprising a first step of administering a steroid to a posterior scleritis patient through an intravenous injection, and a second step of orally administering a s...
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WO/2024/091523A1 |
The present disclosure relates in some aspects to certain phenethylamine analogs, such as benzocyclobutene and benzocyclopentene analogs, including those of Formula (1). Also provided are methods of synthesizing the compounds, compositio...
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WO/2024/088175A1 |
Disclosed in the present disclosure are a gRNA targeting VEGFA, a gene editing system, and the use thereof. The gRNA in the present disclosure comprises a guide sequence which has at least 80% sequence identity to any one of SEQ ID NOs: ...
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WO/2024/090512A1 |
The present invention relates to novel crystals of KUS121 that are useful as a pharmaceutical, and a method for producing the same.
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WO/2024/088110A1 |
Provided are a heterocyclic macrocyclic compound containing an indazole structure and used as a protein kinase inhibitor, and a preparation method therefor. The compound has the structure represented by general formula (I). Additionally ...
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WO/2024/088364A1 |
The present disclosure relates to a pharmaceutical composition containing an isoquinolinone compound, and to a preparation method therefor. Specifically, the present disclosure provides a pharmaceutical composition containing 9,10-dimeth...
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WO/2024/088156A1 |
The present invention relates to a novel use of recombinant human ocriplasmin in the preparation of a drug for treating and/or preventing a choroidal vascular disease.
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WO/2024/091653A1 |
The present invention relates to methods of orally administering water soluble extracts of spearmint to subjects in a therapeutically effective amount to attenuate retinal cell damage and other neural cell damage outside the central nerv...
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WO/2024/091789A1 |
Myopia progression is safely and effectively reduced by topical administration of a storage-stable low-dose formulation in which atropine is present at a concentration of 0.01%. Unexpectedly, such formulation was proven more effective to...
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WO/2024/091659A1 |
The present invention provides for the treatment, prevention, and/or reduction of a risk of an ocular disease, disorder, or condition in which aldehyde toxicity is implicated in the pathogenesis, by the use of a disclosed formulations.
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WO/2024/088921A1 |
The invention is concerned with a method of predicting response to an IL-6 angatonist such as anti-IL-6 antibody by determing the concentration of IL-6 in human aqueous humor. The invention is also concerned with an IL-6 angatonist for u...
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WO/2024/091014A1 |
The present invention relates to a pharmaceutical composition for preventing or treating hearing loss, comprising ammonium lactate as an active ingredient. Since it was confirmed that the number of normal sensory hair cells increased whe...
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WO/2024/092086A1 |
The invention relates to regenerating corneal endothelium to treat vision loss in a subject in need thereof. The invention includes a lipid nanoparticle (LNP) capable of regenerating corneal endothelium in a subject in need thereof. The ...
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WO/2024/092210A1 |
Provided herein are methods to treat fibrosis and/or inhibit angiogenesis.
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WO/2024/085100A1 |
The present disclosure relates to a solid particle dispersed oily preparation. The present disclosure also provides a solid particle dispersed oily preparation suited to eyedrops or intraocular administration. The solid particle disperse...
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WO/2024/086555A1 |
The present disclosure generally relates to methods of treating geographic atrophy (GA), reducing the growth rate of total GA lesion area, slowing the loss of visual acuity, and preventing or slowing development and/or progression of cho...
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WO/2024/083956A1 |
The present invention relates to a formulation of josamycin or a related compound in a crosslinked poly- or oligosaccharide, and the use of this formulation in ophthalmic treatment of infection and fibrosis.
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WO/2024/085081A1 |
Provided is an antibody that specifically binds to the extracellular domain of human LPA1 but does not specifically bind to the extracellular domain of human LPA2 or the extracellular domain of human LPA3. The antibody preferably has act...
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WO/2024/083013A1 |
Disclosed are a human mitochondrial dynamin like GTPase and the use thereof. The gene sequence encoding four variants of the recombinant human type II mitochondrial dynamin like GTPase is optimized, and the experiments on the sequence st...
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WO/2024/086304A1 |
Provided herein are compositions for ophthalmic administration and methods of use.
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WO/2024/083176A1 |
Disclosed are the uses of amniotic fluid in the preparation of a drug for treating and/or preventing macrophage-mediated diseases, a drug for treating and/or preventing type M1 macrophage-mediated diseases, a drug for treating and/or pre...
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WO/2024/086182A1 |
The present disclosure relates to the use of stimulators of soluble guanylate cyclase (sGC), pharmaceutically acceptable salts thereof and pharmaceutical formulations or dosage forms comprising them, alone or in combination with one or m...
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WO/2024/079755A1 |
The present disclosure provides a method for obtaining RPE (retinal pigment epithelium) cells from iPSCs (induced pluripotent stem cells) comprising: (a) generating embryoid bodies from a culture of iPSCs, wherein the embryoid bodies are...
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WO/2024/080952A1 |
The present invention relates to usage of hydrogen peroxide compounds as active substance in elimination of diabetic retinopathy diseases and the symptoms resulting from these diseases, and pharmaceutical compositions comprising hydrogen...
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WO/2024/081163A1 |
Provided herein are methods of treating leukoencephalopathy with brainstem and spinal cord involvement and lactate elevation (LBSL) in a subject in need thereof, the method comprising: administering a therapeutic agent to the subject, wh...
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WO/2024/081442A1 |
Soluble proteins and extracellular vesicles (EVs) produced by optic nerve lamina region neural progenitor cells (ONLR-NPCs) are provided. Such proteins and EVs include growth factors and survival factors that can be used in the treatment...
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WO/2024/077793A1 |
The present invention relates to a compound based on oxidative stress and an anti-amyloid aggregation mechanism, a preparation method therefor, and a use thereof. The compound of the present invention has a structure represented by formu...
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WO/2024/079326A1 |
A composition of oral administration for use in the prevention and/or and treatment degenerative diseases of the eye that it comprises lysates of probiotic micro-organisms in the form of a dry powder of the genus Bacillus, Lactobacillus,...
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WO/2024/078515A1 |
The present invention relates to an ophthalmic pharmaceutical composition containing levamisole, a preparation method therefor, and a use thereof, and in particular relates to an ophthalmic preparation. The ophthalmic preparation compris...
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WO/2024/078124A1 |
Provided in the present invention is the use of a TDGF1 gene in the preparation of a drug for treating aging-related diseases or reversing cell aging. Compared with the prior art, the present invention has the following advantages: the u...
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WO/2024/079449A1 |
The invention relates to a construct and AAV particle comprising a wildtype NDP nucleic acid sequence or a codon-optimised NDP nucleic acid sequence. The invention further relates to products and methods for the treatment of one or more ...
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WO/2024/081410A1 |
In certain embodiments, the invention is directed to a compound of formula (I): wherein; X1 is selected from N or N+Y1; X2 is selected from NH or NY2; X3 is selected from NH or NY3; Y1 is selected from -CH2OCO(OCH2CH2)n1OM1; or -CH2OCO(C...
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WO/2024/074670A1 |
The invention relates to the fields of medicine and immunology. In particular, it relates to novel antisense oligonucleotides that may be used in the treatment, prevention and/or delay of an USH2A-related disease or condition.
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WO/2024/076577A1 |
An aqueous formulation includes a PEDF-derived short peptide (PDSP); a blend of PVP/PVA in different ratios around 8/2 – 2/8; and a tonicity agent, which is preferably non-ionic. The PDSP is preferably 14-39 amino acids long and has th...
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