Resumen de: WO2025228429A1
An RNAi agent targeting an amyloid precursor protein, such as a double-stranded small interfering RNA (siRNA) agent. A method for inhibiting the expression of the APP gene by using the RNAi agent and a method for preventing and treating APP-related diseases, such as cerebral amyloid angiopathy (CAA) or Alzheimer's disease (AD), including early-onset familial Alzheimer's disease (EOFAD). The siRNA significantly inhibits the expression level of the APP gene and has a long-lasting drug effect.
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The present invention relates to pharmaceutical compositions and diagnostic methods for neurodegenerative diseases, particularly Parkinson's disease. The invention includes compositions comprising anti-Clostridium tetani agents such as tetanus vaccines, anti-tetanus immunoglobulins, and antibiotics. Methods for diagnosis and monitoring Parkinson's disease comprising detecting C. tetani-derived nucleic acids, proteins, or analogues thereof, in biological specimens, are further provided.
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Provided are methods of phospho-tau aggregation-based biomarker discovery, and new utilities for discovered biomarkers in Alzheimer's disease (AD) diagnosis, differentiation, treatment, and identification of the presence of pretangles in a subject. Novel p-tau sites, p-tau198, p-tauS356, p-tau396, and p-tau422, identified through such methods showed comparable or superior characteristics with established p-tau biomarkers, and identified biomarkers were capable of differentiating AD or mild cognitive impairment (MCI) from cognitively normal controls.
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Provided herein is a method of treating teenager Huntington's disease in a subject in need thereof, the method comprising orally administering a pharmaceutical composition comprising pridopidine and/or an analog thereof or a pharmaceutically acceptable salt thereof.
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The present invention relates to combination of donepezil or a pharmaceutically acceptable salt thereof and cevimeline or a pharmaceutically acceptable salt thereof for use in the treatment of Alzheimer's disease. Furthermore, a pharmaceutical formulation containing donepezil or a pharmaceutically acceptable salt thereof, cevimeline or a pharmaceutically acceptable salt thereof, and at least one pharmaceutically acceptable excipient is provided.
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Provided herein are compounds, compositions, uses, and methods for increasing cell viability of dopaminergic neurons, or for preventing or treating dopaminergic neuronal death. In certain examples, methods are provided for reducing symptoms and/or for preventing or treating Parkinson's disease in an individual in need thereof, which can include the step of treatment with a GDP-binding form of Rab1a (Rab1aGDP), one or more manifestation nucleic acids encoding Rab1aGDP, or a combination thereof.
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Disclosed is a method for the treatment of a neurological or movement disorder, e.g., Parkinson's disease, in a patient in need thereof, by parenteral administration of levodopa and a dopa decarboxylase inhibitor (DDCI), such as carbidopa, benserazide or any combination thereof.
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Provided herein is an anti-CD2 antibody or antigen binding fragment thereof for treating and/or preventing ALS in a subject in need thereof.
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Disclosed herein are recombinant gene therapy vectors comprising a PTEN-induced kinase 1 (PINK1) encoding gene that is operatively linked to a promoter and methods of using the recombinant therapy vectors for inhibiting, reducing, or delaying degeneration or death of neurons of a subject.
Resumen de: MX2025012729A
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: MX2025002654A
The disclosure relates to a combination of active ingredients/adjuvants for the treatment of neurological disorders and diseases such as Alzheimer's disease and mild cognitive impairment (MCI) and memory and cognitive disorders and conditions. In particular, combinations of scyllo-inositol and treatments for Alzheimer's disease such as aducanumab and/or combinations with essential fatty acids such as mixtures of linolenic acid/linoleic acid or vitamin D or vitamin D compounds such as calcifediol are disclosed as useful. The combinations may be in the form of separate dosage forms of each active ingredient or may be an oral dosage form having multiple active ingredients in a single capsule or tablet or oral solution. The invention also relates to a method of treating patients having mild cognitive impairment with MMSE criteria of between 22 to 26 with a pharmaceutically effective amount of scyllo-inositol to treat the disease and to slow down progression to Alzheimer's disease.
Resumen de: MX2025012236A
Disclosed is a method for the treatment of a neurological or movement disorder, e.g., Parkinson's disease, in a patient in need thereof, by parenteral administration of levodopa and a dopa decarboxylase inhibitor (DDCI), such as carbidopa, benserazide or any combination thereof.
Resumen de: CN118924896A
The invention relates to a method for treating and preventing Alzheimer's disease. The present invention provides methods of reducing clinical decline in a subject suffering from early Alzheimer's disease; a method of converting an amyloid-positive subject suffering from early Alzheimer's disease to an amyloid-negative subject; methods of reducing brain amyloid levels in a subject; and methods of preventing Alzheimer's disease comprising administering a composition comprising a therapeutically effective amount of at least one anti-A beta profibril antibody. In some embodiments, the subject is ApoE4 positive. In some embodiments, the at least one anti-A beta protofibril antibody is BAN2401.
Resumen de: AU2024284125A1
Disclosed herein are methods for detecting the presence of at least one misfolded form of human Superoxide Dismutase 1 (SOD1) in a biological sample obtained from a human subject. In some aspects, the subject is suspected of having, or has, one or more neurodegenerative diseases, such as, for example, Amyotrophic Lateral Sclerosis, Parkinson's disease, or Alzheimer's disease.
Resumen de: US2025332164A1
Use of particular substituted heterocycle fused gamma-carboline compounds as pharmaceuticals and pharmaceutical compositions comprising them for the treatment of one or more disorders involving the 5-HT2A, SERT and/or dopamine D2 pathways are disclosed. In addition, the compounds may be combined with other therapeutic agents for the treatment of one or more sleep disorders, depression, psychosis, dyskinesias, and/or Parkinson's disease or any combinations.
Resumen de: AU2023437235A1
A 1,4-polyisoprene dispersion system, a pharmaceutical active ingredient and the use thereof. The dispersion system is stable in the gastric acid environment of mammals without precipitation of 1,4-polyisoprene clots, has no oral toxicity to mammals, and does not contain allergens. The 1,4-polyisoprene dispersion system comprises a 1,4-polyisoprene solution dispersion system and a 1,4-polyisoprene emulsion dispersion system. The 1,4-polyisoprene dispersion system can serve as a pharmaceutical active ingredient to be used for preparing drugs for treating diseases including atherosclerotic cardiovascular and cerebrovascular diseases, type II diabetes, hypercholesterolemia, hypertriglyceridemia, fatty liver, colitis, obesity, polycystic ovarian syndrome and Alzheimer's disease.
Resumen de: AU2024260221A1
Provided herein are methods of treating diseases and disorders related to TDP-43 aggregation (e.g., ALS) with an antibody that specifically binds to OxPC or a polynucleotide encoding an antibody that specifically binds to OxPC.
Resumen de: US2025334594A1
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: WO2025223573A1
Provided in the present invention is the use of an Achyranthes bidentata polypeptide combined with a human umbilical cord mesenchymal stem cell-derived exosome in the preparation of a drug for preventing and/or treating Parkinson's disease. A Parkinson's disease (PD) model mouse is established using 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine (MPTP), and then an Achyranthes bidentata polypeptide is used in combination with a human umbilical cord mesenchymal stem cell-derived exosome to treat the mouse. It is found that the treatment can significantly relieve PD-related symptoms, for example, restoring the number of dopaminergic neurons in the substantia nigra, improving the expression of tyrosine hydroxylase in the midbrain, relieving motor and olfactory dysfunction of the PD model mouse, reducing the activation of microglia and astrocytes in the olfactory bulb and midbrain, and improving the activity of neurons in the olfactory bulb.
Resumen de: WO2025224308A1
The current invention relates to QPCTL inhibitors represented by formula (I), and corresponding compositions and uses. Preferably, the inhibitors and compositions are for use in the treatment of cancer, neurodegenerative diseases such as Alzheimer's disease, synucleinopathies, Huntington's disease, bacterial infections such as periodontitis and related disorders, and inflammatory diseases.
Resumen de: WO2025224502A1
The invention is directed to peptide fragments of FKBP52 that inhibit Tau protein aggregation and ameliorate tauopathies like Alzheimer's Disease (AD). It also involves modifications to these peptides to improve their pharmacokinetic and pharmacodynamic properties.
Resumen de: WO2025224501A1
The invention is directed to peptide fragments of FKBP52 that inhibit Tau protein aggregation and ameliorate tauopathies like Alzheimer's Disease (AD). It also involves modifications to these peptides to improve their pharmacokinetic and pharmacodynamic properties.
Resumen de: WO2024155781A1
A method of treating and preventing Alzheimer's disease and related dementias is disclosed. The method includes administering to a patient an effective amount of a drug that is operable to inhibit an interaction between 14-3-3G protein and hexokinase-1 protein in the patient. The drug may be ezetimibe, conivaptan, lumacaftor, ebastine, digitoxin, or astemizole.
Resumen de: AU2024240456A1
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a Ataxin-2 (ATXN2) gene. The ATXN2 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an ATXN2 gene. Pharmaceutical compositions that include one or more ATXN2 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described ATXN2 RNAi agents to central nervous system (CNS) tissue, in vivo, provides for inhibition of ATXN2 gene expression and a reduction in ATXN2 activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including spinocerebellar ataxia type 2 (SCA2) or amyotrophic lateral sclerosis (ALS.)
Nº publicación: US2025327081A1 23/10/2025
Solicitante:
FONDAZIONE ST ITALIANO DI TECNOLOGIA [IT]
FUND CENTRE DE REGULACIO GEN\u00D2MICA [ES]
INST CATALANA DE RECERCA I ESTUDIS AVANCATS [ES]
KINGS COLLEGE LONDON [GB]
THE UNIV COURT OF THE UNIV OF EDINBURGH [GB]
FONDAZIONE ISTITUTO ITALIANO DI TECNOLOGIA,
FUNDACI\u00D3 CENTRE DE REGULACI\u00D3 GEN\u00D2MICA,
INSTITUCI\u00D3 CATALANA DE RECERCA I ESTUDIS AVAN\u00C7ATS,
KING'S COLLEGE LONDON,
THE UNIVERSITY COURT OF THE UNIVERSITY OF EDINBURGH
Resumen de: US2025327081A1
The invention relates to a short single-stranded DNA or RNA aptamer that is capable of binding the TDP-43 protein and of detecting all of the different TDP-43 structures individually, from the soluble monomer to the TDP-43 larger aggregates. The aptamer of the invention is also capable of inhibiting aggregation of TDP-43. Because of these properties, the RNA aptamer of the invention is suitable for use in both the diagnosis and therapeutic treatment and prevention of TDP-43-related proteinopathies, such as ALS and FTD.