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153
resultados
Última actualización
03/05/2025 [07:44:00]
Resultados 50 a 75 de 153
Resumen de: US2025114358A1
The present disclosure provides methods for treating ALS using pentostatin and cyclophosphamide treatment followed by TREG and/or TREG/Th2 hybrid cells from de-differentiated T cells. The present disclosure further provides methods for producing TREG and TREG/Th2 hybrid cells from de-differentiated T cells, said TREG and TREG/Th2 hybrid cells, populations thereof and compositions thereof. Methods for producing de-differentiated T cells, said de-differentiated T cells, populations thereof and compositions thereof are also provided.
Resumen de: US2025114319A1
A levodopa derivative including a compound or pharmaceutically acceptable salt, hydrate, and/or solvate thereof, wherein the compound includes substituents which, in aggregate, contain at least 6 carbon atoms which are only bonded to either other carbon atoms or to hydrogen atoms. The levodopa derivative may be formulated as a composition including one or more pharmaceutically acceptable carriers or excipients. The levodopa derivative may be part of a pharmaceutical composition including micro or nano particles in which the levodopa derivative is encapsulated in the pharmaceutically acceptable polymer. The levodopa derivative can be used to treat Parkinson's disease by administering to a mammal an amount sufficient to treat Parkinson's disease.
Resumen de: 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.
Resumen de: AU2023354010A1
The present disclosure provides single- or double-stranded interfering RNA molecules (e.g., siRNA) that target a TAR DNA binding protein (TARDBP) gene. The interfering RNA molecules may contain specific patterns of nucleoside modifications and internucleoside linkage modifications, as pharmaceutical compositions including the same. The siRNA molecules may be branched siRNA molecules, such as di-branched, tri-branched, ortetra-branched siRNA molecules. The disclosed siRNA molecules may further feature a 5' phosphorus stabilizing moiety and/or a hydrophobic moiety. Additionally, the disclosure provides methods for delivering the siRNA molecule of the disclosure to the central nervous system of a subject, such as a subject identified as having a neurodegenerative disease (e.g., amyotrophic lateral sclerosis or frontotemporal dementia).
Resumen de: AU2023353995A1
The present disclosure provides single- or double-stranded interfering RNA molecules (e.g., siRNA) that target a membrane-spanning 4-domains AGA (MS4A6A) gene. The interfering RNA molecules may contain specific patterns of nucleoside modifications and internucleoside linkage modifications, as pharmaceutical compositions including the same. The siRNA molecules may be branched siRNA molecules, such as di-branched, tri-branched, or tetra-branched siRNA molecules. The disclosed siRNA molecules may further feature a 5' phosphorus stabilizing moiety and/or a hydrophobic moiety. Additionally, the disclosure provides methods for delivering the siRNA molecule of the disclosure to the central nervous system of a subject, such as a subject identified as having a neuroinflammatory disease (e.g., Alzheimer's disease).
Resumen de: AU2023330511A1
The present disclosure relates to an sgRNA and its application in the preparation of a product for the treatment of Huntington's disease. The present disclosure was designed and screened to obtain an sgRNA targeting exon 1 of the human HTT gene as shown in SEQ ID NO: 1 or SEQ ID NO: 2. The CRISPR/Cas9 system mediated HTT gene knockout strategy based on this sgRNA and its high homologue sgRNA can efficiently knock out the human Huntingtin gene and achieve gene therapy for Huntington's disease.
Resumen de: 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.
Resumen de: 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.
Resumen de: 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.
Resumen de: 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.
Resumen de: 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).
Resumen de: 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.
Resumen de: 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.
Resumen de: 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).
Resumen de: 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.
Resumen de: 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.
Resumen de: 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.
Resumen de: 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.
Resumen de: 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.
Resumen de: 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.
Resumen de: 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.
Resumen de: 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.
Resumen de: 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.
Resumen de: 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.
Nº publicación: US2025099434A1 27/03/2025
Solicitante:
T3D THERAPEUTICS INC [US]
T3D Therapeutics, Inc
Resumen de: US2025099434A1
Novel indane acetic acid compounds alone or in combination with anti-amyloid beta antibodies, for the treatment of Alzheimer's disease, for the reduction of Amyloid-related imaging abnormalities (ARIA), and for the treatment of Cerebral Amyloid Angiopathy and vasogenic edema (VE).
BOPI
Sede Electrónica
