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194
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Updated on
21/10/2025 [07:33:00]
Absstract of: WO2025215579A1
The disclosure relates to compounds of the disclosure and pharmaceutically acceptable salts thereof to their use in medicine; to compositions containing them; to processes for their preparation; and to intermediates used in such processes. The compounds of the disclosure may be useful in the treatment, prevention, suppression and amelioration of disease(s) such as atopic dermatitis, eosinophilic gastritis, atopic keratoconjunctivitis, allergy, alopecia, Alzheimer's disease, asthma, atherosclerosis, Bechet's disease, bullous pemphigoid, cancer, chronic obstructive pulmonary disease, chronic pruritis, chronic urticaria, Crohn's disease (CD), dermatitis, diabetic kidney disease, eosinophilic esophagitis, fungal keratitis, gout, idiopathic pulmonary fibrosis (IPF), keloids, non-alcoholic steatohepatitis (NASH), primary biliary cirrhosis, prurigo nodularis, psoriasis, psoriatic arthritis, rhinosinusitis, scleroderma, systemic lupus erythematosus (SLE), systemic sclerosis, ulcerative colitis (UC), vitiligo, or hidradenitis suppurativa. The compounds of the disclosure may be useful in the treatment, prevention, suppression and amelioration of a dermatological condition or a respiratory condition.
Absstract of: WO2025213771A1
A pharmaceutical crystal having low water solubility, a preparation method therefor, and the use thereof. A crystal form I of pamoate of a compound Z has characteristic diffraction peaks at the diffraction angles 2θ of 9.9±0.2°, 10.7±0.2°, 12.1±0.2°, 14.9±0.2°, 17.3±0.2°, 19.4±0.2°, 20.7±0.2° and 23.1±0.2°. Compared with amorphous pamoate of the compound Z, said crystal has good stability, is free of polymorphic transformation, does not involve obvious increases of related substances, and has a low solubility. A production process therefor is simple and only uses water as a solvent in the whole process without the need of using an organic solvent. Using the crystal form I of pamoate of the compound Z for preparing a long-acting sustained-releasing pharmaceutical composition for Parkinson's disease can achieve a high drug encapsulation ratio and low burst release, and enables drug release of the composition in vivo to last for over two weeks, so that the frequency of drug administration to patients can be reduced, and the medication compliance is improved.
Absstract of: WO2025213608A1
The present invention provides an miR 1978 inhibitor and use thereof in treating a senescence-related disease. The etiology of senescence-related diseases is proposed, and the overexpression of miR 1978 derived from the mitochondrial genome in cell line SHSY5Y is proven to give rise to decreased mRNA expression and protein levels of DHFR, KIF5C, and MSH3; as a result, SHSY5Y cell division is arrested in the G1 phase of the cell cycle, the number of cells in the G2/M phase is significantly reduced, and meanwhile, the number of apoptotic cells is increased. Compared with a control, cells containing miR1978 have different nuclear DNA methylation states. A plurality of Alzheimer's disease-related proteins, such as APP, Tau, p-Tau, and APOE, or Parkinson's disease-related proteins, such as α-synuclein, are increased in the SHSY5Y cell line containing miR 1978, etc.
Absstract of: US2025320503A1
Aspects of the disclosure relate to compositions and methods for reducing expression or activity of superoxide dismutase 1 (SOD1) in a cell or subject. In some embodiments, the compositions, such as nucleic acid and viral vectors, comprise artificial microRNAs (amiRNAs) having a SOD1-targeting sequence positioned within a microRNA scaffold. In some embodiments, the compositions further comprise a human SMN1 promoter. In some aspects, the methods comprise administering a composition of the disclosure to a subject, for example a subject having amyotrophic lateral sclerosis (ALS).
Absstract of: WO2025217259A1
Provided herein are postbiotic compositions prepared using fermentation and herbal substrate compositions for the treatment or prevention of the disruption of the gut-brain axis. Such postbiotic compositions can be used to treat or prevent neurological diseases or disorders, such as synucleinopathies, including but not limited to Parkinson's disease.
Absstract of: US2025320493A1
Provided herein are methods of administering ISIS 814907 for ameliorating Alzheimer's disease, reducing Tau RNA, or reducing Tau protein in a human subject in need thereof. In certain embodiments, the Alzheimer's disease is mild Alzheimer's disease, Mild Cognitive Impairment (MCI) Due to Alzheimer's Disease, and/or Alzheimer's Disease Dementia (e.g., Mild Alzheimer's Disease Dementia). In certain instances, methods are useful for ameliorating at least one symptom or hallmark of a disease or disorder associated with Tau protein. In certain instances, the disease or disorder associated with Tau protein is a neurodegenerative disease or disorder. In certain instances, the disease or disorder associated with Tau protein is Alzheimer's disease or Fronto-temporal Dementia (FTD). In certain embodiments, the Alzheimer's disease is mild Alzheimer's disease, Mild Cognitive Impairment (MCI) Due to Alzheimer's Disease, and/or Alzheimer's Disease Dementia (e.g., Mild Alzheimer's Disease Dementia). In certain instances, the disease or disorder associated with Tau protein is a tauopathy. In certain instances, the disease or disorder associated with Tau protein is Frontotemporal Dementia with Parkinsonism-17 (FTDP-17), Progressive Supranuclear Palsy (PSP), Chronic Traumatic Encephalopathy (CTE), Corticobasal Ganglionic Degeneration (CBD), Pick Disease, Argyrophilic Grain Disease (AGD), Globular Glial Tauopathies, Epilepsy, and/or Dravet's Syndrome. Such symptoms or hallmarks include loss of
Absstract of: US2025320286A1
The invention provides antibodies or antigen binding fragments thereof that bind to 3pE Aβ and methods of making and using the antibodies or antigen binding fragments thereof, including use for formulations, administration and kits. The antibody and antigen binding fragments thereof and methods disclosed are useful for diagnosis, prognosis and treatment of Alzheimer's disease or other β-amyloid-related diseases.
Absstract of: US2025320213A1
The disclosure relates to compounds of the disclosure and pharmaceutically acceptable salts thereof to their use in medicine; to compositions containing them; to processes for their preparation; and to intermediates used in such processes. The compounds of the disclosure may be useful in the treatment, prevention, suppression and amelioration of disease(s) such as atopic dermatitis, eosinophilic gastritis, atopic keratoconjunctivitis, allergy, alopecia, Alzheimer's disease, asthma, atherosclerosis, Bechet's disease, bullous pemphigoid, cancer, chronic obstructive pulmonary disease, chronic pruritis, chronic urticaria, Crohn's disease (CD), dermatitis, diabetic kidney disease, eosinophilic esophagitis, fungal keratitis, gout, idiopathic pulmonary fibrosis (IPF), keloids, non-alcoholic steatohepatitis (NASH), primary biliary cirrhosis, prurigo nodularis, psoriasis, psoriatic arthritis, rhinosinusitis, scleroderma, systemic lupus erythematosus (SLE), systemic sclerosis, ulcerative colitis (UC), vitiligo, or hidradenitis suppurativa. The compounds of the disclosure may be useful in the treatment, prevention, suppression and amelioration of a dermatological condition or a respiratory condition.
Absstract of: US2025319105A1
The present disclosure relates to compositions, kits and methods of treating Alzheimer's disease comprising administering an NMD A receptor positive allosteric modulator or a CYP46A1 inhibitor.
Absstract of: AU2025238004A1
CARBIDOPA AND L-DOPA PRODRUGS AND THEIR USE TO TREAT PARKINSON'S DISEASE Abstract The present disclosure relates to (a) carbidopa prodrugs, (b) pharmaceutical combinations and compositions comprising a carbidopa prodrug and/or an L-dopa prodrug, and (c) methods of treating Parkinson's disease and associated conditions comprising administering a carbidopa prodrug and an L-dopa prodrug to a subject with Parkinson's disease. CARBIDOPA AND L-DOPA PRODRUGS AND THEIR USE TO TREAT PARKINSON'S DISEASE Abstract The present disclosure relates to (a) carbidopa prodrugs, (b) pharmaceutical combinations and compositions comprising a carbidopa prodrug and/or an L-dopa prodrug, and (c) methods of treating Parkinson's disease and associated conditions comprising administering a carbidopa prodrug and an L-dopa prodrug to a subject with Parkinson's disease. ep - ' e p b s t r a c t h e p r e s e n t d i s c l o s u r e r e l a t e s t o ( a ) c a r b i d o p a p r o d r u g s , ( b ) p h a r m a c e u t i c a l c o m b i n a t i o n s a n d c o m p o s i t i o n s c o m p r i s i n g a c a r b i d o p a p r o d r u g a n d o r a n - d o p a p r o d r u g , a n d ( c ) m e t h o d s o f t r e a t i n g a r k i n s o n ' s d i s e a s e a n d a s s o c i a t e d c o n d i t i o n s c o m p r i s i n g a d m i n i s t e r i n g a c a r b i d o p a p r o d r u g a n d a n - d o p a p r o d r u g t o a s u b j e c t w i t h a r k i n s o n ' s d i s e a s e
Absstract of: AU2024253757A1
This disclosure relates to compounds of Formula (I-1) or (I-2): The compounds of the present disclosure are capable of inhibiting the activity of tyrosine kinase 2 (TYK2) and are useful for the treatment of diseases or disorders, such as e.g. inflammation, autoimmune disease, neuroinflammation, arthritis, rheumatoid arthritis, spondyloarthropathies, systemic lupus erythematous, lupus nephritis, arthritis, osteoarthritis, gouty arthritis, pain, fever, pulmonary sarcoisosis, silicosis, cardiovascular disease, atherosclerosis, myocardial infarction, thrombosis, congestive heart failure and cardiac reperfusion injury, cardiomyopathy, stroke, ischaemia, reperfusion injury, brain edema, brain trauma, neurodegeneration, liver disease, inflammatory bowel disease, Crohn's disease, ulcerative colitis, nephritis, retinitis, retinopathy, macular degeneration, glaucoma, diabetes (type 1 and type 2), diabetic neuropathy, viral and bacterial infection, myalgia, endotoxic shock, toxic shock syndrome, autoimmune disease, osteoporosis, multiple sclerosis, endometriosis, menstrual cramps, vaginitis, candidiasis, cancer, fibrosis, obesity, muscular dystrophy, polymyositis, dermatomyositis, autoimmune hepatitis, primary biliary cirrhosis, primary sclerosing cholangitis, vitiligo, alopecia, Alzheimer's disease, skin flushing, eczema, psoriasis, atopic dermatitis and sunburn. The disclosure further provides methods of preparing the compounds.
Absstract of: US2025319073A1
The present disclosure provides compounds, compositions, and/or methods for treating, preventing, inhibiting, amelio-rating, or delaying the onset of Huntington's disease and/or a HTT proteinopathy in a subject. The methods can comprise administering to the subject an effective amount of a peptidomimetic compound, such as (R)-2-amino-N—((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5- guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and/or solvate thereof, alone or in combination with one or more other therapeutic agents.
Absstract of: EP4631499A1
A delayed timed release pharmaceutical composition, a preparation method therefor, and use thereof. The pharmaceutical composition comprises a tablet core. The tablet core comprises a drug-containing layer and a boosting layer stacked on the drug-containing layer. The drug-containing layer comprises a drug active ingredient. The drug active ingredient is levodopa or a derivative thereof, or a mixture of levodopa or a derivative thereof and a DOPA decarboxylase inhibitor, and accounts for 5-72.5 wt% of the content of the drug-containing layer. The pharmaceutical composition is a capsule-shaped tablet, can realize the effects of 1-3 h delayed release of the drug active ingredient and reaching a peak concentration at 6-10 h, can be used for reducing morning stiffness in patients with Parkinson's disease, and has good application prospects.
Absstract of: WO2024121173A1
The present invention is in the field of amyotrophic lateral sclerosis (ALS) and relates to human interleukin-2 (IL-2) for use in the treatment of amyotrophic lateral sclerosis in a human subject, wherein each dose of human IL-2 administered to said subject is between 0.1 x106 to 3x106 international units (IU) and the subject has a low to medium concentration of p-NFH in cerebrospinal fluid (CSF p-NFH) or a low to medium concentration of NFL or NFM in cerebrospinal fluid, blood, serum or plasma before human IL-2 administration. The invention also relates to medical uses where the CSF p-NFH or CSF, blood, serum or plasma NFL or NFM concentration is used to select the best administration scheme or as a biomarker for stratified randomization of a cohort of ALS patients in the context of a clinical trial assessing the therapeutic efficiency of a candidate ALS treatment.
Absstract of: AU2024230525A1
The present invention provides for methods of treating obsessive-compulsive disorder (OCD) and OCD-related disorders (body dysmorphic disorder, hoarding disorder, trichotillomania (hair-pulling disorder), excoriation (skin-picking) disorder, substance/medication-induced obsessive-compulsive and related disorder, obsessive- compulsive and related disorder due to another medical condition, and other specified and unspecified obsessive-compulsive and related disorders), Tic disorders including Tourette syndrome, autism spectrum disorder (ASD) and glutamate excitotoxicity related disorders including amyotrophic lateral sclerosis (ALS), Parkinson's disease, traumatic brain injury, multiple sclerosis, Huntington's disease, and schizophrenia, comprising the step of administering an effective amount of a histamine type 1 receptor agonist and/or histamine type 3 receptor antagonist, such as betahistine or its pharmaceutically acceptable salts, analogs, metabolites, prodrugs, derivatives, metabolites, co-crystals, modifications, solvates, hydrates, isotopes, tautomers, esters, polymorphs or stereoisomers.
Absstract of: US2025312408A1
The present invention provides methods for to sex-specific treatment and dose titration of diseases associated with an aberrant functionality of activity-dependent neuroprotective protein (ADNP) and/or cytoskeleton such as progressive supranuclear palsy (PSP), schizophrenia, amnestic mild cognitive impairment (aMCI), Alzheimer's disease, and autism. The treatment in these diseases, e.g., the dose and/or the regimen, differs between sexes and has to be adapted to obtain the desired effect. Specifically, use of davunetide in treatment of women suffering from PSP or of men suffering from schizophrenia or aMCI are provided.
Absstract of: US2025312375A1
Disclosed herein are human blood fractions depleted of disease-causing extracellular vesicles, prepared by plasma exchange, that may find use in the treatment of a condition selected from the group consisting of neurodegenerative disease, autoimmune disease, cardiovascular disease, renal disease, and liver disease. In particular, the depleted blood fractions may find use in treating neurodegenerative diseases such as Parkinson's Disease or Alzheimer's Disease.
Absstract of: US2025312385A1
Lachnospiraceae spp and Ruminococcus lactaris new strains of bacteria for use solely or in combination, in the treatment and prevention of memory decline in an individual, in particular declines of aging or Alzheimer's disease-related origin. Compositions, in particular, an oral composition, including the Lachnospiraceae spp and Ruminococcus lactaris strains and uses thereof.
Absstract of: US2025313842A1
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of LRRK2 RNA in a cell or animal, and in certain instances reducing the amount of LRRK2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include Parkinson's disease.
Absstract of: US2025313574A1
Compound of Formula (I)-(V), compositions comprising at least one compound chosen from compounds of Formula (I)-(V), and methods of using the same, including in treatment of Alzheimer's disease.
Absstract of: US2025313539A1
Provided in the present invention are an α,β-unsaturated amide compound, and a preparation method therefor, and a pharmaceutical composition and the use thereof. Specifically, provided in the present invention is a compound as represented by formula I, wherein the definition of each group is as described in the description. The compound can be used as a compound for improving cerebral blood flow and is used for preparing a pharmaceutical composition for treating neurodegenerative diseases such as Alzheimer's disease and vascular dementia and strokes.
Absstract of: US2025312323A1
This invention relates to methods and materials for treating mammals having, or at risk of developing, one or more movement disorders (e.g., essential tremor, epilepsy, and/or Parkinson's disease). For example, compositions including one or more T-type calcium channel antagonists (e.g., one or more Cav3 antagonists such as CX-8998) are provided, as well as methods for administering such compositions to a mammal having, or at risk of developing, one or more movement disorders (e.g., essential tremor, epilepsy, and/or Parkinson's disease) to treat the mammal.
Absstract of: US2025313570A1
The present invention relates to a use of a novel compound, for preventing, improving or treating amyotrophic lateral sclerosis (ALS), wherein the present inventors have found that SOD1 aggregation is one of the important causes of ALS, and have proposed the possibility that WT-SOD1 aggregation, caused by suppressing the regulation of intracellular stress or TDP-43, may be a cause of sALS. In addition, the present inventors have discovered the novel compound PRG-A-01(SLC-B036) as a SOD1 aggregation and misfolding inhibitor.
Absstract of: WO2025211909A1
The present invention provides a novel thiophene derivative, an optical isomer thereof, or a pharmaceutically acceptable salt thereof. A novel thiophene derivative according to the present invention has excellent inhibitory activity against KDM4, and thus is useful as a therapeutic agent for brain diseases such as Alzheimer's disease.
Nº publicación: WO2025212933A1 09/10/2025
Applicant:
ENCODED THERAPEUTICS INC [US]
ENCODED THERAPEUTICS, INC
Absstract of: WO2025212933A1
The present disclosure provides methods and compositions for the treatment of Tauopathies, including Alzheimer's Disease (AD) and Frontotemporal Dementia and Parkinsonism Linked to Chromosome 17 (FTDP-17), by inhibiting expression of the MAPT gene. Aspects of the disclosure provide engineered inhibitory RNA agents, e.g., siRNA, miRNA, and antisense oligonucleotides, that knock down endogenous mutated or wild-type MAPT transcripts in neuronal cells in a subject. Expression vectors encoding the engineered inhibitory RNA agents, e.g., in an AAV vector, are also provided.
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