Alerta

TREATMENT OF HUNTINGTON'S DISEASE

Resumen de: WO2025264449A1

The present invention describes methods of treating Huntington's disease in a subject in need thereof. The method comprising administrating to the subject in need thereof a therapeutically effective amount of 2-3-(2,2,6,6-tetramethylpiperidin-4-yl)-3H-1,2,3triazolo4,5-cpyridazin-6-yl-5-(2H-1,2,3-triazol-2-yl)phenol or a pharmaceutically acceptable salt thereof.

COMPOSITIONS OF BI-FUNCTIONAL ALPHA HELICAL PEPTIDES AND METHODS THEREOF FOR TREATING TDP-43 PROTEINOPATHIES

Resumen de: WO2025264861A1

Compositions and methods for disrupting pathological aggregation and/or mis-localization of TDP-43 in the brain/CNS have been developed. Compositions including engineered helical polypeptides that bind TDP-43's amyloidogenic core but resist β-sheet conversion are provided. In some forms, the engineered polypeptides include peptide degradation motifs (PDM) to enhance proteolytic degradation of aggregates, and/or targeting motifs to direct the peptides to the brain/CNS. Recombinant constructs including nucleic acids expressing or encoding the polypeptides are also provided. Methods of using the engineered peptides to treat or prevent one or more diseases or disorders associated with pathological aggregation and/or mis-localization of TDP-43 in the brain/CNS are also provided. In some forms, the methods treat or prevent ALS or FTD in a subject in need thereof.

USE OF ANAVEX3-71 FOR MEDICAL TREATMENTS

Resumen de: WO2025264845A1

The present disclosure relates to the novel use of ANAVEX3-71 and related compounds in medical treatments, such as Parkinson's Disease, Frontotemporal Dementia, Schizophrenia, and Alzheimer's Disease.

TREATMENT OF DISEASE

Resumen de: WO2025264823A1

This disclosure relates to the use of Purine Nucleoside Phosphorylase (PNP) inhibitors such as ulodesine and its salts, in the treatment and/or prevention of diseases associated with nicotinamide adenine dinucleotide (NAD+) depletion, including diseases of mitochondrial dysfunction (including neurodegeneration and peripheral neuropathies), the preservation of cognitive function and in muscular disorders such as sarcopenia; and in metabolic syndrome and associated conditions. In particular the disclosure provides the use of PNP inhibitors such as ulodesine in the treatment of neurodegenerative conditions such as Parkinson's disease and amyotrophic lateral sclerosis.

AGENTS AND/OR COMPOSITIONS USEFUL FOR MODULATING CIS-REGULATORY ELEMENTS IN SYNUCLEINOPATHIES AND METHODS FOR IDENTIFYING AGENTS AND COMPOSITIONS THEREOF

Resumen de: WO2025264967A1

The present disclosure relates to methods of preventing, or delaying the progression of, death of neurons and/or microgliosis and/or astrogliosis that contributes to the death of neurons. The present disclosure also relates to methods of treating, preventing, or delaying the progression of, a synucleinopathy (e.g., Parkinson's disease). Also disclosed are related in vitro, ex vivo, and in vivo methods of identifying agents and/or compositions useful for preventing, or delaying the progression of, death of neurons and/or microgliosis and/or astrogliosis that contributes to the death of neurons and agents and/or compositions useful for treating, preventing, or delaying the progression of, a synucleinopathy. The agents and/or compositions of the present disclosure decrease the level and/or activity of a cis-regulatory element that propagates the misfolding and aggregation of proteins encoded by synucleinopathy-associated genes in neurons and/or glial cells.

COMPOSITION COMPRISING HAPLN1 AS ACTIVE INGREDIENT OR PREVENTING OR TREATING SENILE DEGENERATIVE BRAIN DISEASES

Resumen de: WO2025263948A1

The present invention relates to a composition comprising HAPLN1 as an active ingredient for preventing or treating senile degenerative brain diseases. Specifically, recombinant human HAPLN1 protein (rhHAPLN1) lowers the protein level of p16 in cultured human astrocytes to inhibit cellular senescence caused by the accumulation of beta amyloid peptides, and further inhibits phosphorylation (p-p38 MAPK) of p38 MAPK protein, thereby also having the possibility of inhibiting inflammatory responses associated with the onset of Alzheimer's disease and Parkinson's disease. In addition, the recombinant human HAPLN1 protein (rhHAPLN1) exhibits significant memory and learning improvement effects in in vivo experiments performed using a mouse acute Alzheimer's disease model, and thus can be expected to exhibit preventive and therapeutic effects against Alzheimer's disease that may occur with aging and the like. In addition, the inhibitory effect of the rhHAPLN1 protein on cellular senescence and inflammatory responses of astrocytes can provide a very important clue for establishing prevention and treatment strategies not only for aging itself but also for brain functions, motor behaviors, memory, seizures, dementia, brain tumors, and the like.

ANTI-SOD1 NANOBODIES

Resumen de: US2025388697A1

Composition and methods of diagnosing, monitoring, and treating subjects with a motor neuron pathology, such as motor neuron disorders (including but not limited to amyotrophic lateral sclerosis (ALS)) and neuropathies.

(AZA)SPIROHEPTANE DERIVATIVES FOR THE TREATMENT OF NEURODEGENERATIVE DISORDERS

Resumen de: US2025388592A1

The application relates to compounds of formula (B1A), (B1B), (B1C) or a salt, a solvate, a hydrate, a polymorph, a tautomer, a racemate or a stereoisomer thereof, to pharmaceutical compositions comprising such a compound, and to the compounds for use as a medicine, in particular for use in the treatment of neurodegenerative disorders such as Alzheimer's disease.

COMPOSITIONS AND METHODS FOR THE TREATMENT OF PARKINSON S AND GAUCHER DISEASE

Resumen de: EP4667461A2

Compounds and pharmaceutical compositions are useful for the treatment of Gaucher disease and Parkinson's disease. Methods of treating Gaucher disease or Parkinson's disease include administration of one or more compounds or pharmaceutically acceptable salts or prodrugs thereof to a subject diagnosed with, or at risk of developing Gaucher disease or Parkinson's disease.

Nutritional composition for improving cell membranes

Resumen de: NZ795279A

An early in life nutritional intervention with a lipid component having both large lipid globules with phospholipids and an increased amount of palmitic acid in the sn-2 position in triglycerides were found to improve the fatty acid composition of cell membranes, in particular brain membranes. The nutritional composition comprising the lipid can therefore be used for improving the fatty acid composition of cell membranes, improving cognitive performance, improving cognitive development, improving behavioural performance, improving behavioural development, improving visual acuity, improving fine motor skills and preventing or treating other disorders associated with impaired brain membrane fatty acid composition, e.g., attention deficiency, ADHD, autism, dyslexia, depression, bipolar depression, anxiety, schizophrenia, obsessive compulsive disorder, bulimia, abuse of alcohol or drugs, borderline personality disorder, panic disorder, social phobia, cognitive impairment, dementia, Alzheimer’s disease or Parkinson’s disease.

Compound having kdm5 inhibitory activity and pharmaceutical use thereof

Resumen de: NZ793960A

The present invention provides KDM5 inhibitor. The compound disclosed herein represented by the general formula (I) : wherein all symbols have the same meanings as the definitions described in the specification; or a salt thereof is useful as a prophylactic and/or therapeutic agent for cancer, Huntington’s disease, Alzheimer’s disease and the like.

Continuous administration of pharmaceutical composition for treatment of neurodegenerative disorders

Resumen de: NZ768098A

A method of treating patients by using a pharmaceutical composition which comprises (i) a dopamine replacement agent selected from levodopa, melevodopa, etilevodopa and combinations thereof or a pharmaceutically acceptable salt of levodopa, (ii) a dopamine decarboxylase inhibitor (DDI) selected from carbidopa, benserazide and combinations thereof, and (iii) a catechol-O-methyltransferase (COMT) inhibitor selected from entacapone, tolcapone, opicapone and combinations thereof for treating Parkinson’s Disease, where the composition is administered in two dosages, the second dosage being continuously administered.

Aav treatment of huntington’s disease

Resumen de: NZ751950A

Aspects of the disclosure relate to compositions and methods useful for treating Huntington’s disease. In particular, the disclosure provides interfering nucleic acids (e.g., artificial mature miRNAs flanked by a miR-155 or a miR-30 backbone sequence) targeting the huntingtin gene (HTT) and methods of treating Huntington’s disease using the same.

METHODS AND COMPOSITIONS FOR TREATMENT OF NEURODEGENERATIVE DISORDERS AND REDUCING TAU PROTEIN AGGREGATES

Resumen de: US2025381230A1

Disclosed are compositions and methods for treating Alzheimer's disease or for use in treating Alzheimer's disease. Also disclosed are compositions and methods for treating or for use in treating a neurodegenerative disorder characterized by the presence of tau protein aggregates. Furthermore, disclosed are compositions and methods for reducing tau protein aggregates or for use in reducing tau protein aggregates.

Regimen for Treating Amyotrophic Lateral Sclerosis Having Onset 24 Months Prior to Treatment

Resumen de: US2025381197A1

The present invention relates to the treatment of an ALS patient having disease onset of at least 24 months prior to initiation of treatment with fasudil. Fasudil is administered at a dose of 60-240 mg/day according to specific treatment regimens. This results in an anticipated 25-50% reduction in the average decline over at least three months as measured using the revised ALS Functional Rating Scale.

METHOD FOR TREATING A PARKINSON'S DISEASE

Resumen de: US2025381305A1

Provided is a method for treating a Parkinson Disease, comprising a TRIM72 protein modulator. Further provided is a composition comprising the TRIM72 protein modulator and use thereof.

TREATMENTS FOR AMYOTROPHIC LATERAL SCLEROSIS USING DAZUCORILANT

Resumen de: US2025381178A1

Applicant discloses methods and compositions for treating a patient suffering from amyotrophic lateral sclerosis (ALS) comprising administration of a heteroaryl ketone fused azadecalin compound. In embodiments, the heteroaryl ketone fused azadecalin compound is dazucorilant: (R)-(1-(4-fluorophenyl)-6-((4-(trifluoromethyl)phenyl)sulfonyl)-4,4a,5,6,7,8-hexahydro-1-H-pyrazolo3,4-gisoquinolin-4a-yl)(pyridin-2-yl)methanone, having the chemical structure illustrated asSuitable doses include daily administration of 150 milligrams and 300 milligrams of dazucorilant. Suitable doses include daily administration of dazucorilant with food, or with water, or with food and water. Daily administration of dazucorilant is effective to increase dazucorilant exposure up to about 2-fold when continued for seven days or more. Administration of such a heteroaryl ketone fused azadecalin compound may comprise oral administration, enteral administration, or other administration. Pharmaceutical compositions comprising dazucorilant are useful in the treatment of patients suffering from ALS. Suitable pharmaceutical compositions comprising dazucorilant include, e.g., pharmaceutical compositions for oral administration and pharmaceutical compositions for enteral administration.

MAGNESIUM COMPOSITIONS AND USES THEREOF FOR NEUROLOGICAL DISORDERS

Resumen de: US2025381158A1

A composition for administration to a subject, such as oral administration to a subject, for example, has been provided. Such a composition may comprise at least one magnesium-counter ion compound. A magnesium-counter ion composition described herein may be useful for any of a variety of applications provided herein, such as maintaining, enhancing, and/or improving health, nutrition, and/or another condition of a subject, and/or cognitive, learning, and/or memory function. A magnesium-counter ion composition provided herein may be useful for administration to a subject presenting magnesium deficiency, mild cognitive impairment, Alzheimer's disease, attention deficit hyperactivity disorder, ALS, Parkinson's disease, diabetes, migraine, anxiety disorder, mood disorder, and/or hypertension. A kit, method, and other associated technology are also provided.

Gene constructs for silencing alpha-synuclein and uses thereof

Resumen de: US2025382611A1

The present invention relates to a nucleic acid, to a composition, to the medical use of said composition in the treatment and/or prevention of Parkinson's Disease (PD), Multiple System Atrophy (MSA) and/or other α-synucleopathies.

COMPOSITIONS AND METHODS FOR THE TREATMENT OF PARKINSON'S AND GAUCHER DISEASE

Resumen de: US2025382274A1

Compounds and pharmaceutical compositions are useful for the treatment of Gaucher disease and Parkinson's disease. Methods of treating Gaucher disease or Parkinson's disease include administration of one or more compounds or pharmaceutically acceptable salts or prodrugs thereof to a subject diagnosed with, or at risk of developing Gaucher disease or Parkinson's disease.

SMALL MOLECULE INHIBITORS OF DYRK/CLK AND USES THEREOF

Resumen de: US2025382301A1

This invention is in the field of medicinal chemistry. In particular, the invention relates to a new class of small-molecule compounds having a 6,6-heterocyclic structure (e.g., compounds having a naphthyridine, pyrido-pyridazine, pyrido-pyrazine, quinoline, pyrazino-pyridazine, pyrimido-pyrimidine, quinazoline, quinoxaline or cinnoline ring system) which function as inhibitors of DYRK1A, DYRK1B, DYRK2, DYRK3, CLK1, CLK2, CLK3, CLK4, CDK7, CDK8/19, PI3K, PDGFrA/B, mTOR, WNT, homeodomain-interacting kinases (HIPKs), and/or CMGC kinases leading to inhibition of WNT signaling, and their use as therapeutics for the treatment of Alzheimer's disease, down syndrome, Parkinson's disease, Huntington's disease, diabetes, autoimmune diseases, inflammatory disorders (e.g., airway inflammation, osteoarthritis (e.g., knee related osteoarthritis)), cancer (e.g., glioblastoma, prostate cancer, metastatic breast cancer, metastatic lung cancer, multiple myeloma, secondary metastatic tumors of the brain, colorectal cancer and metastatic colorectal cancer (e.g., metastatic colorectal cancer in the liver)), and other diseases.

SMALL MOLECULE INHIBITORS OF DYRK/CLK AND USES THEREOF

Resumen de: US2025382277A1

This invention is in the field of medicinal chemistry. In particular, the invention relates to a new class of small-molecule compounds having a 6,6-heterocyclic structure (e.g., compounds having a naphthyridine, pyrido-pyridazine, pyrido-pyrazine, quinoline, pyrazino-pyridazine, pyrimido-pyrimidine, quinazoline, quinoxaline or cinnoline ring system) which function as inhibitors of DYRK1A, DYRK1B, DYRK2, DYRK3, CLK1, CLK2, CLK3, CLK4, CDK7, CDK8/19, PI3K, PDGFrA/B, mTOR, WNT, homeodomain-interacting kinases (HIPKs), and/or CMGC kinases leading to inhibition of WNT signaling, and their use as therapeutics for the treatment of Alzheimer's disease, down syndrome, Parkinson's disease, Huntington's disease, diabetes, autoimmune diseases, inflammatory disorders (e.g., airway inflammation, osteoarthritis (e.g., knee related osteoarthritis)), cancer (e.g., glioblastoma, prostate cancer, metastatic breast cancer, metastatic lung cancer, multiple myeloma, secondary metastatic tumors of the brain, colorectal cancer and metastatic colorectal cancer (e.g., metastatic colorectal cancer in the liver)), and other diseases.

COMPOSITION FOR IMPROVING COGNITIVE FUNCTION USING SINGLE OR COMPLEX EXTRACT OF ACANTHOPANAX KOREANUM, ASTRAGALUS MEMBRANACEUS, AND/OR MOMORDICA CHARANTIA

Resumen de: WO2025258725A1

The present invention discloses a composition for improving cognitive function using a single or complex extract of Acanthopanax koreanum, Astragalus membranaceus, and/or Momordica charantia. The composition increases, in an in-vitro efficacy test, the cell viability of SH-SY5Y cells, which are human-derived neuroblastomas treated with scopolamine that is an oxidative stress-inducing drug, increases the expression of an apoptosis inhibition-related biomarker (Bcl-2), an autophagy-enhancing biomarker (Beclin-1), and an acetylcholine biosynthesis-related enzyme (ChAT), which are neurotransmitters, also increases the expression of the antioxidant-related biomarker HO-1, and increases the inactivation of GSK-3β, which is a therapeutic target for Alzheimer's disease, and furthermore, even in an in-vivo efficacy test, increases the expression of Bcl-2, Beclin-1, ChAT, and HO-1 in the hippocampus and cerebrum of an animal model with reduced cognitive function and memory induced by administration of scopolamine, and inhibits the expression of iNOS, which is an enzyme involved in NO biosynthesis that induces tissue damage.

THERAPY OF CNS DISORDERS

Resumen de: WO2025259709A1

The present disclosure provides methods of screening for, identifying and using a Gi-GPCR agonist for a CNS disorder. The CNS disorder can be any disorder in which astrocyte morphology and/or astrocyte tissue support are altered or compromised (e.g., OCD, Alzheimer's disease, or Huntington's disease). Provided herein are methods of screening for and identifying Gi-GPCR agonist ex vivo based on assessment of astrocyte morphology and/or Gi-GPCR activation (e.g., wherein the Gi-GPCR is GPR3711, S1PR1, EDNRB, GRM3, or AD0RA2A). Also provided herein are methods of identifying a therapeutic agent for the treatment of a CNS disorder in vivo at least in part based on its effect on astrocyte morphology and/or Gi-GPCR activation. Also provided herein are methods for the treatment or prevention of a CNS disorder comprising administering to a subject a Gi-GPCR (e.g., GPR3711, S1PR1, EDNRB, GRM3, or AD0RA2A) agonist.

METHODS OF TREATMENT USING AN ANTI-ABETA PROTOFIBRIL ANTIBODY

Nº publicación: AU2024286486A1 18/12/2025

Solicitante:

EISAI R&D MANAGEMENT CO LTD
EISAI R&D MANAGEMENT CO., LTD

Resumen de: AU2024286486A1

Disclosed herein are methods of selecting, monitoring, and treating subjects with Alzheimer's disease (AD) or suspected of having AD or another disorder associated with amyloid accumulation in the brain based on the risk of an ARIA event or brain hemorrhage. Also disclosed herein are methods of treating subjects having or suspected of having AD comprising subcutaneously administering an anti-Aβ protofibril antibody.