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165
resultados
Última actualización
27/09/2026 [07:37:00]
Resumen de: US20260287576A1
0000 The invention provides, in some embodiments, a potency assay matrix for assessing potency of dopaminergic neuronal progenitor cells (DANPCs) that are intended for use in treating a neurodegenerative disease such as Parkinson's Disease. The potency assay matrix can include a bioassay for L-DOPA decarboxylase (DDC) activity. In addition to the DDC activity assay matrix, the potency assay matrix may also include one or more additional mechanism of action-based assays such as bioinformatics-based assays for determining whether the DANPCs are dopaminergic neuronal progenitor cells, for determining whether the DANPCs are likely to engraft when implanted into a subject brain, and for determining whether neuronal cells that are derived from the DANPCs are likely to produce dopamine after implantation.
Resumen de: US20260284023A1
Methods of use of CFTR corrector compounds, particularly lumacaftor and tezacaftor, to treat reduced cerebral perfusion associated with heart failure, sudden sensoneurinal hearing loss, vascular dementia, arterial hypertension, ischemic stroke, hemorrhagic stroke, heart disease, diabetes and Alzheimer's disease by treatment with an amount sufficient to provide a proteostatic effect on cystic fibrosis transmembrane conductance regulator (CFTR) protein expression in cerebral artery smooth muscle cells and an increase in cerebrovascular perfusion in the patient.
Resumen de: DE102025111075A1
Die vorliegende Erfindung betrifft ein Verfahren zur Herstellung von dopaminergen neuronalen Organoiden (d-NOs) mit Nachweis der Dopaminfreisetzung in biologisch wirksamen Konzentrationen. Darüber hinaus umfassen d-NOs neben dopaminergen Neuronen auch funktionell miteinander verbundene GABAerge, glutamaterge und cholinerge Neuronen sowie Gliazellen, wodurch sie zu einem multizellulären Organoid mit einer für das Mittelhirn repräsentativen Zellzusammensetzung und Funktion werden. Daher zeigen die d-NOs der Erfindung eine komplexe Regulation dopaminerger Neuronen durch verwandte exzitatorische und inhibitorische Neuronen, was auf Kontrollmechanismen hindeutet, die beispielsweise eine Überproduktion und Freisetzung von Dopamin verhindern können. Die Erfindung bezieht sich auch auf die n-NOs als Implantat oder zur Verwendung bei der Behandlung der Parkinson-Krankheit. Darüber hinaus bezieht sich die Erfindung auf die Verwendung der d-NOs für Wirkstoffscreenings.
Resumen de: AU2025235730A1
The present disclosure is generally directed to tetracyclic analogs that modulate autophagy in a subject suffering from alpha-1 antitrypsin deficiency (ATD) and possibly other autophagy associated diseases or disorders, such as Alzheimer's disease, Parkinson's disease, Huntington's disease, and amyotrophic lateral sclerosis.
Resumen de: US20260284230A1
0000 Provided herein are compositions and methods of treating and/or preventing amyotrophic lateral sclerosis (ALS) in a subject in need thereof, the methods comprising administering an agent wherein the agent modifies neurons via changes in Kcnn1 protein expression or activity, which in turn modifies neuron firing and/or increases the clearance or protection from toxicity of an ALS-causing protein.
Resumen de: US20260284097A1
0000 The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the APP gene, as well as methods of inhibiting expression of an APP gene and methods of treating subjects having an APP-associated disease or disorder, such as Alzheimer's disease (e.g., early onset Alzheimer's disease), using such dsRNAi agents and compositions.
Resumen de: US20250152747A1
0000 Provided herein are expression cassettes for expressing a transgene in a cell, wherein the transgene encodes a GCase polypeptide. Also provided are methods to treat Gaucher Disease or GBA-PD. Further provided herein are vectors (e.g., rAAV vectors), viral particles, pharmaceutical compositions, and kits for expressing an GCase polypeptide in an individual in need thereof.
Resumen de: WO2025104230A1
The present disclosure relates to compounds that are IL-17A modulators. The compounds have the structural Formula I defined herein. The present disclosure also relates to processes for the preparation of these compounds, to pharmaceutical compositions comprising them, and to their use in the treatment of diseases or disorders associated with modulation of IL-17A activity.
Resumen de: EP4810493A1
The present invention relates to a heterocyclic carbonyl derivative modulator, a preparation method therefor, and the use thereof. Specifically, the present invention relates to a compound represented by general formula (II-B), a preparation method therefor, a pharmaceutical composition containing said compound, and a use thereof as a modulator in the treatment of Alzheimer's disease, schizophrenia, pain, addiction, and sleep disorders, wherein each substituent in general formula (II-B) is as defined in the description.
Resumen de: MX2026005980A
The invention provides compositions and methods for the treatment of diseases associated with amyloid deposits of Aβ in the brain of a patient, such as Alzheimer's Disease. Such methods entail administering a pharmaceutical composition comprising an immunogenic fragment of Aβ capable of inducing a beneficial immune response in the form of antibodies to Aβ. The immunogenic fragments comprise linear or multivalent peptides of Aβ. Pharmaceutical compositions comprise the immunogenic fragment chemically linked to a carrier molecule which may be administered with an adjuvant.
Resumen de: US20260275351A1
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a Superoxide Dismutase 1 (SOD1) gene. The SOD1 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an SOD1 gene. Pharmaceutical compositions that include one or more SOD1 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described SOD1 RNAi agents to central nervous system (CNS) tissue, in vivo, provides for inhibition of SOD1 gene expression and a reduction in SOD1 activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including amyotrophic lateral sclerosis (ALS).
Resumen de: US20260272976A1
Described herein are methods of monitoring and treating a subject with ALS based on biomarkers.
Resumen de: US20260275333A1
Disclosed herein are oligonucleotides with one or more spacers or without a spacer. In various embodiments, STMN2, KCNQ2, UNC13A, or SMN2 oligonucleotides with spacer(s) reduce STMN2 transcripts with cryptic exons or reduce mis-spliced KCNQ2, UNC13A, or SMN2 transcripts and increase full length STMN2, KCNQ2, UNC13A, SMN transcripts, thereby imparting therapeutic efficacy against neurological diseases such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Alzheimer's disease (AD) or spinal muscular atrophy (SMA).
Resumen de: AU2026226429A1
An effective amount of quercetin, vitamin B3, vitamin C, zafirlukast and optionally folic acid for use in treating amyotrophic lateral sclerosis in a subject. Also disclosed are said combination for use in reducing, slowing or abating the progression of amyotrophic lateral sclerosis or a symptom thereof. ep e p
Resumen de: AU2025225323A1
The present invention relates to a solid composition, and its use in the treatment of Parkinson's disease, comprising at least two compartments, a first active pharmaceutical ingredient (API) and a second API, preferably for increasing uptake of the second API thereby optimizing its use in the treatment of Parkinson's disease, in particular improving its clinical activity and reducing its side effects associated with the treatment of Parkinson's disease.
Resumen de: AU2025215085A1
Disclosed herein is a compound of Formulas (I) and (II) as an FTO inhibitor with novel structures. Also disclosed herein is a pharmaceutical composition comprising the same, and a method of inhibiting weight gain, promoting weight loss, reducing serum LDL, cholesterol, LDL-c, or triglycerides, or treating obesity or an obesity-related disease (esp. obesity-related diabetes, hyperglycemia, diabetic nephropathy, hyperlipemia, coronary heart disease, atherosclerosis, hypertension, cardiovascular or cerebrovascular disease) or Alzheimer's disease by inhibiting FTO by using the compound disclosed herein.
Resumen de: US20260272856A1
This disclosure relates to administration of a combination of: 1) about 100-110 mg, about 104-106 mg, or about 105 mg or less of bupropion hydrochloride, or a molar equivalent amount of the free base form or another salt form of bupropion; and 2) about 40-50 mg, about 44-46 mg, or about 45 mg or less of dextromethorphan hydrobromide, or a molar equivalent amount of the free base form or another salt form of dextromethorphan in human patients for treating neurological and psychiatric conditions, such as agitation associated Alzheimer's disease and/or reducing relapse of agitation in Alzheimer's disease.
Resumen de: US20260274931A1
Disclosed herein are methods of conducting clinical trials in Alzheimer's disease (AD). Methods of selecting a medicament and treating an AD subject are disclosed. Methods, systems, kits, and devices for selecting a subject in AD clinical trials and treatment protocols are disclosed herein.
Resumen de: US20260275352A1
The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.
Resumen de: US20260275314A1
The disclosure relates to compositions and methods for, inter alia, altering, e.g., enhancing, the level of GBA1 protein via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful, inter alia, in the treatment of subjects who have, have been diagnosed with, or are at risk of having a GBA1-related disorder, e.g., Parkinson's Disease (PD), Gaucher Disease (GD), Parkinson's Disease Dementia (PDD), Dementia with Lewy Bodies (DLB), or Lewy Body Dementia (LBD).
Resumen de: WO2026189377A1
Provided is use of an RIPK1 inhibitor in the treatment of Alzheimer's disease. Specifically, provided is a potential treatment method for Alzheimer's disease. The method uses the RIPK1 inhibitor to ameliorate and inhibit brain inflammation and the development of Alzheimer's disease caused by changes in the activity or expression level of myeloid INPP5D or RIPK1 activation, thereby ameliorating symptoms of late-onset Alzheimer's disease, especially early AD progression, and reducing brain inflammation associated with Alzheimer's disease.
Resumen de: WO2026192853A1
The present disclosure provides pharmaceutical compositions and methods for the treatment, delaying progression and prevention of degenerative cognitive disorders such as Alzheimer's disease (AD) and its variations. There are also provided methods of treating AD in human subject by reducing amyloid pathology and improving synaptic integrity.
Resumen de: WO2026193437A1
The present disclosure provides methods and kits related to treatment of Parkinson's disease in a subject by reducing the expression of a CaV1.3 protein in a target cell by administering to the subject a therapeutically effective amount of an engineered genetic system.
Resumen de: WO2026190230A1
The present invention relates to alpha-synuclein antibodies which are useful in diagnostic assays for detecting aggregates, oligomers and fibrillar forms of alpha- synuclein in patient samples. These antibodies enable diagnosis and efficient treatment of alpha-synucleinopathies such as Parkinson's Disease (PD) or Multiple System Atrophy (MSA) patients by assessing and monitoring the pathological alpha-synuclein levels in patients.
Nº publicación: WO2026193281A1 17/09/2026
Solicitante:
TRANSCRIPTA BIO INC [US]
MOXHAM CHRISTOPHER M [US]
LEONE HADITSCH URSULA [US]
MELLINA CLAYTON E [US]
TRANSCRIPTA BIO, INC.
MOXHAM, Christopher M.
LEONE HADITSCH, Ursula
MELLINA, Clayton E.
Resumen de: WO2026193281A1
Provided herein are compositions and methods for reducing expression of RBM39 and for treating diseases and/or conditions characterized by increased expression of RBM39 (e.g., a cancer) in subjects in need thereof, by administering compositions comprising compounds described herein. Also provided herein are compositions and methods for reducing expression of MSH3 and for treating diseases and/or conditions characterized by increased expression of MSH3 (e.g., Huntington's Disease) in subjects in need thereof, by administering compositions comprising compounds described herein.
BOPI
Sede Electrónica
