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136
results.
Updated on
06/05/2025 [07:37:00]
Results 50 to 75 of 136
Absstract of: US2025115911A1
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a SNCA gene, particularly in a CNS tissue, as well as methods of inhibiting expression of a SNCA gene and methods of treating subjects having a SNCA-associated neurodegenerative disease or disorder, e.g., Parkinson's Disease (PD), multiple system atrophy (MSA), Lewy body dementia (LBD), among other synucleinopathies, using such dsRNAi agents and compositions.
Absstract of: US2025115604A1
Compounds of formula (I)wherein A, R, W, Q, n and m have the meaning according to the claims can be employed, inter alia, for the treatment of tauopathies and Alzheimer's disease.
Absstract of: WO2025076222A1
Human tau protein phosphorylated at the amino acid, serine 413 (pS413 tau), can serve as a biomarker for tauopathies such as Alzheimer's disease. Detection and quantitation of pS413 tau in a biological sample such as cerebrospinal fluid can be useful in developing therapeutics for certain tauopathies. However, pS413 tau is present in biological samples at very low levels. Thus, the invention provides a highly sensitive assay for the detection and quantitation of pS413 tau in a biological sample comprising a series of steps as described herein.
Absstract of: WO2025076156A1
This invention provides a method for determining whether a human subject is afflicted with Alzheimer's disease ("AD") or non-Alzheimer's dementia ("non-ADD") when the subject is suspected of being afflicted with AD or non-ADD, comprising the steps of (a) synchronizing a population of suitable cells derived from the subject, wherein the suitable cells are cultured skin cell fibroblasts or cultured B lymphocytes; and (b) in the resulting synchronized cell population, measuring the expression levels of two or more genes selected from the group consisting of FAM149B, NHLH1, SHISA5, URB2, and WASF2, whereby (i) the subject is afflicted with AD if the expression levels measured in step (b) are consistent with those genes' expression levels in corresponding synchronized cells derived from AD patients, and (ii) the subject is afflicted with non-ADD if the expression levels measured in step (b) are consistent with those genes' expression levels in corresponding synchronized cells derived from non-ADD patients. This invention also provides related diagnostic and therapeutic methods, including diagnostic methods based on NDS subject gene expression levels.
Absstract of: WO2025076181A1
The present disclosure relates to transcription modulator molecules having a first terminus, a second terminus, and an oligomeric backbone and methods for treating Huntington's disease (HD).
Absstract of: WO2025075789A1
Human tau protein phosphorylated at the amino acid, serine 413 (pS413 tau), can serve as a biomarker for tauopathies such as Alzheimer's disease. Detection and quantitation of pS413 tau in a biological sample such as cerebrospinal fluid can be useful in developing therapeutics for certain tauopathies. However, pS413 tau is present in biological samples at very low levels. Thus, the invention provides a highly sensitive assay for the detection and quantitation of pS413 tau in a biological sample comprising a series of steps as described herein.
Absstract of: AU2023276707A1
Provided herein are methods and kits for treating neurodegenerative diseases such as Amyotrophic Lateral Sclerosis, Alzheimer's Disease Parkinson's Disease, Myasthenia Gravis, Multifocal Motor Neuropathy, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Kennedy's Disease, and Spinocerebellar Ataxia. Also provided are methods of predicting or measuring a response to a treatment by measuring biomarker levels in a sample, and methods of modulating biomarker levels.
Absstract of: KR20250047115A
본 발명은 파킨슨병에서 우울/불안과 같은 비운동 증상과 신경병리 사이의 상호작용에 신경 활동이 중요한 역할을 한다는 점을 규명하고, 특히, c-Fos 억제제인 T-5224가 비운동 증상과 신경병리 모두를 표적으로 하여 이를 효과적으로 개선시킬 수 있음을 입증한 것으로, 파킨슨병 초기 환자에 c-Fos 억제제를 치료제로 이용하면 비운동 증상과 신경병리 모두를 표적으로 하여 우수한 치료 효과를 발휘할 수 있는바, 파킨슨병 환자의 질병 진행을 늦추고 파킨슨병 환자의 삶의 질을 향상시킬 수 있는 장점이 있다.
Absstract of: US2025108016A1
Provided herein are cannabinoid-containing complex mixtures suitable for use as active pharmaceutical ingredients. The complex mixtures comprise at least a first major cannabinoid, at least a first minor cannabinoid, and optionally at least a first selected terpene. Also provided are methods of making the complex mixtures; pharmaceutical compositions comprising the complex mixture, and methods of using the pharmaceutical compositions for the treatment of neurodegenerative diseases, including Alzheimer's disease, Parkinson's disease, Lewy Body Dementia, or Huntington's disease.
Absstract of: AU2022481088A1
The present invention relates to the use of borate derivatives in the treatment of amyotrophic lateral sclerosis (ALS), which is a neurodegenerative disease. More particularly, it relates to the development and administration of pharmaceutical formulations comprising at least one borate derivative disclosed in the said invention.
Absstract of: AU2023341167A1
The present disclosure relates to methods of treating Alzheimer's disease, diseases and/or conditions associated with Covid-19 infection, including long COVID, a post-acute infection syndrome, or symptoms of orthostatic intolerance comprising administration of a therapeutically-effective combination of a COX-2 inhibitor and an antiviral compound.
Absstract of: US2025109398A1
Aspects of the disclosure relate to compositions and methods useful for treating neurological diseases and disorders. In some embodiments, the disclosure provides a method for treating a neurological disease or disorder comprising administration of both a viral vector comprising interfering nucleic acids (e.g., artificial miRNAs) and a viral vector comprising a CYP46A1 protein. In some embodiments, the disclosure provides a method for treating Huntington's disease comprising administration of both a viral vector comprising interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and a viral vector comprising a CYP46A1 protein. In some embodiments, the viral vector comprises a modified viral capsid, such as for preferentially targeting cells in the CNS or PNS.
Absstract of: US2025109131A1
Provided herein is a synthetic process for preparing a compound of Formula (1).The disclosure also provides useful intermediates and salts, amorphous and polymorph forms of the compound of Formula (1). These compounds are useful for various disease including cancer, abnormal cellular proliferation, angiogenesis, Alzheimer's disease, and osteoarthritis as well as Wnt-related diseases.
Absstract of: WO2025071299A1
The present invention found that neural activity plays an important role in the interaction between non-motor symptoms such as depression/anxiety and neuropathies in Parkinson's disease, and particularly, demonstrates that T-5224, which is a c-FOS inhibitor, targets both non-motor symptoms and neuropathies, thereby effectively alleviating both of them. When used as a therapeutic agent in patients with early Parkinson's disease, a c-FOS inhibitor exhibits excellent therapeutic effects by targeting both non-motor symptoms and neuropathy, and thus has an advantage in that the progression of diseases in Parkinson's disease patients can be delayed and the quality of life of Parkinson's disease patients can be improved.
Absstract of: WO2025072637A1
Disclosed herein is a class of small-molecules having oxygenated heterocyclic ring structure. Compounds disclosed herein are lysine demthylase-1 (LSD-1) inhibitors, and accordingly, also disclosed herein is the use the compounds as therapeutics for the treatment of hematological disorders (e.g., sickle cell disease (SCD), β-thalassemia), cancer (e.g., acute myeloid leukemia (AML), multiple myeloma, biliary tract cancer, non-small cell lung cancer (NSCLC), chronic lymphocytic leukemia, advanced solid tumor, advanced malignancies), and/or a neurological disorder (e.g., Alzheimer's disease (AD), Amyotrophic Lateral Sclerosis (ALS), Parkinson disease (PD), Schizophrenia, Huntington disease (HD)), a metabolic disorder (e.g., type-2 diabetes (T2D), obesity) and other conditions related to LSD-1 activity (e.g., mild to moderate Alzheimer's disease, myocardial fibrosis, autism, complex neurodevelopmental diseases).
Absstract of: WO2025068990A1
Provided are compounds of Formula (I) and related compositions and methods, including methods of therapy for treating neurological diseases and disorders, including multiple sclerosis (MS) and amyotrophic lateral sclerosis (ALS), and methods for in vivo imaging of AMPA receptor, or surrogate thereof, in the brain.
Absstract of: WO2025065506A1
Disclosed are a benzylamine compound having a structure as shown in formula (I), or a pharmaceutically acceptable salt or stereoisomer thereof, a synthesis method therefor, and a use thereof. A biscarbamoyl ester derivative containing a benzylamine structure provided by the present invention has a significant inhibitory effect on butyrylcholinesterase, can be used as a butyrylcholinesterase inhibitor, and can be used to prepare a medicament for treating or preventing diseases related to butyrylcholinesterase, such as hyperlipidemia, Alzheimer's disease, Parkinson's disease, etc.
Absstract of: WO2025067354A1
Provided are an siRNA inhibiting the expression of an amyloid precursor protein (APP) gene in a human cell, a polypeptide-oligonucleotide drug, and a use. The siRNA has good APP expression inhibitory activity, and a suitable modification is made to the siRNA to improve the silencing capability against a target and reduce off-target activity. The siRNA and a conjugate thereof are expected to be applied for clinically preventing and treating an APP target-related disease such as cerebral amyloid angiopathy (CAA), early-onset familial Alzheimer's disease (EOFAD), or Alzheimer's disease (AD).
Absstract of: US2025109165A1
Provided herein are compounds of Formula (I-A), (I-B), or (I-C), and pharmaceutically acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, isotopically enriched forms, prodrugs, or mixtures thereof, and compositions thereof. Also provided are methods and kits involving the inventive compounds or compositions for treating and/or preventing diseases and/or conditions (e.g., neurological disease (e.g., Alzheimer's disease, multiple sclerosis, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis), metabolic disorder (e.g., obesity, diabetes, X-linked adrenoleukodystrophy (X-ALD)), proliferative disease (e.g., cancers), hepatic disease (e.g., liver cirrhosis), conditions associated with autophagy (e.g., neurodegenerative disease, infection, cancer, conditions associated with aging, heart disease), conditions associated with aging, conditions associated with modulating the mPTP, cardiovascular conditions (e.g., ischemia-reperfusion injury), stroke, heart attack, conditions associated with oxidative stress, mitochondrial diseases, or other diseases associated with cyclophilins) in a subject, as well as for reducing oxidative stress. Provided are methods of inhibiting a cyclophilin in a subject, cell, tissue, and/or biological sample. Provided are methods of selectively inhibiting a cyclophilin (e.g., CypD, CypE) in a subject, cell, tissue, and/or biological sample.
Absstract of: WO2025068459A2
The present invention provides an isolated protein exhibiting an antiaggregating activity and/or disaggregating activity toward a target protein comprising an extended polyQ stretch. The protein comprises a Zn2+-binding region, wherein the conserved motif is HxxEHx75-80E and x is any amino acid. The nucleic acid construct encoding said protein as well as the corresponding mRNA sequence are also provided. The protein, the nucleic acid construct or mRNA sequence are for use in a method for prevention or treatment of a neurodegenerative disease that is caused by aggregates comprising at least one target protein and/or by the mRNA encoding for said target protein, wherein the target protein causes e.g. Huntington's disease or Machado-Joseph disease.
Absstract of: WO2025063407A1
The present application relates to a novel therapeutic agent for Parkinson's disease and a method for treating Parkinson's disease by using same.
Absstract of: WO2023228204A1
The present disclosure provides methods for treating Alzheimer's disease (AD) comprising administering clomipramine or a pharmaceutically acceptable salt thereof. The administration of clomipramine increases the levels of TAp73 and decreases the levels of proliferating cell nuclear antigen (PCNA) and cleaved caspase-3 in the AD patients. The methods of the present disclosure reduce the neurodegeneration and improve the cognitive and functional decline in AD patients.
Absstract of: KR20250044169A
본 발명은 알파-시누클레인 폴리몰피즘 조절시스템 및 파킨슨질환 치료에 대한 유효성 예측용 바이오마커에 관한 것으로, 보다 상세하게는 GCN5를 포함하는 알파-시누클레인 폴리몰피즘 제어 시스템 또는 유효성 예측용 바이오마커 조성물에 관한 것이다. 본 발명에서는 알파-시누클레인 과발현 및 A53T 돌연변이에 의해 알파-시누클레인 폴리몰피즘 형성이 증가하는 것을 확인하였으며, GCN5 발현 GCN5 발현이 증가하면 알파-시누클레인 과발현이 억제되는 것을 확인하였으므로, 본 발명의 GCN5 바이오마커는 알파-시누클레인 폴리몰피즘 제어를 위한 조절 시스템에 적용할 수 있다. 또한, 알파-시누클레인 과발현 모델에서 GCN5 동시 과발현 및/또는 약리학적 활성화는 알파-시누클레인에 의한 독성을 억제하는 것을 확인하였으므로, GCN5의 치료적 활성화는 알파-시누클레인병증 또는 파킨슨병의 치료에 도움을 줄 수 있다.
Absstract of: WO2025064824A1
A method for treatment of a human subject for Alzheimer's disease (AD) comprises sequentially administering two or more doses of a recombinant, fully human, anti-amyloid beta monoclonal antibody to the human subject. In preferred embodiments, the antibody is administered subcutaneously in tissue near or at an abdomen of a human subject in increasing amounts over a period of time.
Nº publicación: WO2025061918A1 27/03/2025
Applicant:
JULIUS MAXIMILIANS UNIV WUERZBURG [DE]
JULIUS-MAXIMILIANS-UNIVERSIT\u00C4T W\u00DCRZBURG
Absstract of: WO2025061918A1
The present invention relates to therapeutical uses of non-classical human major histocompatibility complex (MHC) molecules (also named MHC class Ib molecules) in combination with peptide antigens for the treatment of Parkinson's disease. The invention more specifically relates to recombinant polypeptides comprising peptide antigens and one or more domains of a non-classical MHC class Ib molecule. The invention also relates to methods of producing such recombinant polypeptides, pharmaceutical compositions comprising the same, as well as their uses for treating Parkinson's disease.
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