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151
resultados
Última actualización
24/11/2025 [07:21:00]
Resumen de: WO2025240469A1
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: WO2025239588A1
The present invention relates to: a fusion protein comprising a mitochondrial outer membrane anchoring peptide and a cerebrovascular endothelial cell surface protein binding site; modified mitochondria to which the fusion protein is bound; and a pharmaceutical composition comprising the modified mitochondria as an active ingredient. The modified mitochondria comprising the cerebrovascular endothelial cell surface protein binding site according to the present invention can pass through the blood-brain barrier at the cellular and animal level. In addition, administering the modified mitochondria to a Parkinson's disease mouse model can alleviate movement disorders. Therefore, the modified mitochondria according to the present invention can be used as a therapeutic agent for brain diseases caused by mitochondrial dysfunction.
Resumen de: WO2025239904A1
The application relates to monoclonal antibody clone 1A11, which is highly specific for a C-terminal sequence of human alpha-synuclein (hαSyn) containing three nitrated tyrosines (3NYαSyn) and does not bind to the corresponding unmodified sequence. A CAR specific for 3NYαSyn has been produced, comprising the antigen-recognizing site of the mAb 1A11 fused to an intracellular region containing the signaling motifs of CD3zeta and CD28. Tregs expressing said anti-3NYαSyn CAR have been shown to attenuate neurodegeneration and neuroinflammation in a mouse model for Parkinson's disease. The T reg-response specific for 3YNαSyn exerts a therapeutic effect attenuating the loss of dopaminergic neurons in a preclinical model and represents a promising therapeutic strategy to treat Parkinson's disease patients..
Resumen de: US2025354188A1
The present invention provides a method of synthesizing celosianin II, a method of synthesizing a betaxanthin, an amyloid-β polymerization inhibitor or a therapeutic or preventive agent for Alzheimer's, an amyloid peptide aggregation inhibitor, and an HIV-1 protease activity inhibitor. A gene having a celosianin II synthesis ability has been isolated from quinoa, and a method of synthesizing celosianin II of the present invention has been constructed. Besides, it has been recognized that celosianin II or the like serves as an active ingredient of each of an amyloid-β polymerization inhibitor or a therapeutic or preventive agent for Alzheimer's, an amyloid peptide aggregation inhibitor, and an HIV-1 protease activity inhibitor.
Resumen de: US2025353904A1
The present invention addresses the problem of providing an agent for preventing or treating amyotrophic lateral sclerosis (ALS), Parkinson's disease (PD), Huntington's disease (HD), spinocerebellar ataxia (SCA), aging-related degenerative or neurological disease, brain aging, or diseases associated with brain aging, as well as a more stable antibody that exhibits an effect of preventing or treating these diseases, Alzheimer's disease (AD), or frontotemporal lobar degeneration (FTLD). A human monoclonal antibody that specifically binds to human HMGB1, wherein the human monoclonal antibody (anti-human HMGB1 antibody) comprises a heavy chain CDR1, heavy chain CDR2, and heavy chain CDR3 each consisting of a specific amino acid sequence and a light chain CDR1, light chain CDR2, and light chain CDR3 each consisting of a specific amino acid sequence, is used as an agent for preventing or treating ALS, PD, HD, SCA, aging-related degenerative or neurological disease, brain aging, or diseases associated with brain aging. An antibody in which the light chain complementarity determining region (CDR) 3 of the anti-human HMGB1 antibody has been modified is used.
Resumen de: US2025353841A1
The present disclosure provides a compound of Formula (I) or a pharmaceutically acceptable salt thereof and its use in, e.g. treating a condition, disease, or disorder in which lowering mutant huntingtin protein (“mHTT”) in a subject is of therapeutic benefit, specifically in treating Huntington disease (“HD”). This disclosure also features a composition containing the same as well as methods of using and making the same.
Resumen de: US2025352533A1
A method of treating a human patient afflicted with Huntington's disease, comprising periodically orally administering to the patient a pharmaceutical composition comprising pridopidine, its analog or a pharmaceutically acceptable salt thereof.
Resumen de: US2025352494A1
Disclosed herein are patches, methods, devices, and systems for delivering a non-aggregating peptide, such as alcadein and its fragments, into a subject. In some aspects, the patch includes a backing, a matrix comprising a non-aggregating peptides disposed within the matrix, and a release liner. In other aspects, the method includes opening at least one channel in the subject's skin, applying the patch described herein, thereby treating a disease or disorder associated with the brain, such as Alzheimer's disease.
Resumen de: US2025352509A1
Compositions and methods for treating amyotrophic lateral sclerosis. A method of treating amyotrophic lateral sclerosis comprising administering to a subject in need thereof an effective amount of quercetin, vitamin B3, vitamin C, zafirlukast and optionally folic acid. Also disclosed are methods of reducing, slowing or abating the progression of amyotrophic lateral sclerosis or a symptom thereof, comprising administering to a subject in need thereof an effective amount of quercetin, vitamin B3, vitamin C, zafirlukast and optionally folic acid.
Resumen de: WO2025240956A1
Described is small molecule 6-methoxy-7-(3-(pyrrolidin-1-yl)propoxy)-4-(tetrahydro-2H-pyran-4-yl)quinolin-2-amine (MS152) that inhibits methyltransferases G9a/EHMT2. This inhibitor can be used for the treatment of patients with G9a/EHMT2 related diseases such as Prader-Willi Syndrome and Alzheimer's Disease. Formula (I).
Resumen de: US2025352609A1
Disclosed herein are methods of treating or preventing Alzheimer's disease or dementia and concurrently treating hyperparathyrodism by administering a blood-brain barrier (BBB)-impermeable calcimimetic and/or a BBB-permeable calcilytic along with the administration of anti-amyloid-beta therapies. Also disclosed herein are methods of increasing calcium-sensing receptor (CaSR) homodimer formation, increasing expression or activity of the CaSR homodimer and reducing CaSR/GABA-B1 receptor heterodimer formation, reducing expression or activity of the CaSR/GABA-B1 receptor heterodimer in both peripheral tissues and central nervous system (CNS) in a subject.
Resumen de: US2024310389A1
This document provides methods and materials related to screening for and treating Alzheimer's disease (AD), including late-onset Alzheimer's disease (LOAD).
Resumen de: AU2025256123A1
Provided is a method of administering a vesicular monoamine transport 2 (VMAT2) inhibitor to a patient in need thereof, wherein the patient experiences one or more clinically significant parkinson-like signs or symptoms. Provided is a method of administering a vesicular monoamine transport 2 (VMAT2) inhibitor to a patient in need thereof, wherein the patient experiences one or more clinically significant parkinson-like signs or symptoms. ct c t
Resumen de: AU2025256148A1
The present disclosure relates to compositions of levodopa 4’-monophosphate and carbidopa 4’-monophosphate having a weight by weight ratio of about 20:1 and methods of treating Parkinson's disease and associated conditions by subcutaneous administration of such compositions. The present disclosure relates to compositions of levodopa 4'-monophosphate and carbidopa 4'-monophosphate having a weight by weight ratio of about 20:1 and methods of treating Parkinson's disease and associated conditions by subcutaneous administration of such compositions. ct c t
Resumen de: AU2024267789A1
Provided are heterocyclic compounds of Formula I or Formula II or their pharmaceutically pharmaceutically acceptable salts, solvates, hydrates, tautomers or stereoisomers for imaging tau aggregates. Compounds of Formula I or Formula II may be used for the detection of tau aggregates in the diagnosis or monitoring of the progression of a disease or disorder such as Alzheimer's disease, corticobasal degeneration and progressive supranuclear palsy.
Resumen de: AU2024269632A1
The invention provides methods of treating patients with Parkinson's disease (PD) associated with wild-type LRRK2. The invention recognizes that analysis of genetic modifiers of LRRK2 in such patients allows identification of those patients who will respond to LRRK2 inhibitors. Thus, the invention provides methods of identifying PD patients who will respond to LRRK2 inhibitors and methods of treating such patients.
Resumen de: AU2024271182A1
The present invention relates to the use of the HIP/PAP protein, or a derivative thereof, in the treatment and prevention, and in particular in the treatment, of a cognitive disorder associated with anxiety disorder(s) in an individual in need thereof, as well as for improving cognition in an individual affected by a neurological disorder associated with anxiety disorder(s) or for alleviating cognitive deficit in an individual affected by a disorder selected from the group consisting of Obsessive-compulsive disorder, Attention deficit disorder, Dementia with Lewy bodies disease, Early onset dementia, Epilepsy-related cognitive dysfunction, Fronto-temporal dementia, Posterior cortical atrophy, Huntington's disease (HD), Parkinson's disease, bipolar disorder, substance abuse, attention deficit disorders, psychotic disorders and a sars-cov-2 infection, and also in the prevention and/or treatment of diet induced cognitive and anxiety deficits in an individual in need thereof, and in particular high fat diet induced cognitive and anxiety deficits.
Resumen de: AU2024283314A1
Herein is reported a bispecific antibody specifically binding to human Abeta protein and human transferrin receptor (bispecific anti-Abeta/TfR antibody) as well as the use of such bispecific antibodies as a medicament in the treatment of Alzheimer's Disease, including where the bispecific antibody is administered intravenously at a dose of 0.2 mg/kg to 7.2 mg/kg once every four weeks.
Resumen de: US2025345312A1
The invention discloses formulation for use in the treatment of diseases such as Alzheimer's Disease, Stroke, and Diabetic Retinopathy as well as a method for treating said diseases by administering said formulation. The formulation comprises a therapeutically effective amount of (1-H indazole-4yl-)methanol, ethanol, and Miglyol812N at a ratio of 1 drug, to 10 Ethanol to 90 Miglyol. The formulation helps in improving retention, bioavailability, and blood-brain barrier penetration of (1-H indazole-4yl-)methanol and was effective in suppressing inflammation by blocking hGMF-β phosphorylation and activity.
Resumen de: US2025346584A1
The invention provides novel ligands of Kappa (κ) opioid receptors, such as can be used to modulate a Kappa opioid receptor. Methods of synthesis and methods of use are also provided. Compounds of the invention can be used therapeutically in the treatment of dissociative disorders or pain, or to provide neuroprotection, or to induce diuresis, or to modulate the immune system, or for treatment of one or more of an affective disorders comprising depression or stress/anxiety; an addictive disorder; alcoholism, epilepsy; a cognition deficiency; schizophrenia; Alzheimer's disease; or pain.
Resumen de: US2025345365A1
Methods and compositions for treating certain neurodegenerative diseases are provided. The method uses in vivo conversion of gilal cells to neurons by PTB and optionally nPTB knock down via CRISPR/Cas delivered by viral vectors (e.g., AAV vector). Examples of the neurodegenerative diseases include RGC loss-related degenerative disease and Parkinson's Disease,
Resumen de: WO2025231617A1
Provided is a polypeptide which contains a polypeptide having at least 80%, 85%, 90%, 95%, or 99% identity to amino acid sequence MTYRPGYNPFG (SEQ ID NO: 41) or amino acid sequence AKGYYRPYGVPV (SEQ ID NO: 22), or a polypeptide having one or more amino acid substitutions, deletions and/or additions relative to the amino acid sequence as shown in SEQ ID NO: 41 or 22. The provided polypeptide can interfere with the binding of a Brag2 synaptic protein to the C terminus of an AMPA receptor, thereby inhibiting NMDA-mediated excessive endocytosis of an AMPA receptor and neuronal apoptosis, and playing a role in protecting neuronal cell activity. Therefore, the polypeptide has application prospects and potential development values as a drug for treating nervous system diseases such as strokes, depression, Alzheimer's disease and drug addiction.
Resumen de: EP4647123A2
The present invention is in the field of amyotrophic lateral sclerosis (ALS) and relates to human interleukin-2 (IL-2) for use in the treatment of amyotrophic lateral sclerosis in a human subject, wherein each dose of human IL-2 administered to said subject is between 0.1 x10<sup>6</sup> to 3x10<sup>6</sup> international units (IU). Human IL-2 is preferably administered in cycles of 3 to 7 days of once-daily sub-cutaneous injection of 0.1 x10<sup>6</sup> to 3x10<sup>6</sup> IU human IL-2. The treatment does not comprise the administration of regulatory T cells to the subject, who is preferably also under riluzole treatment. The administered human IL-2 is preferably not complexed with anti-hIL-2 antibodies and the treatment also preferably does not comprise the administration of rapamycin or any other suppressive agent of effector T cells (Teffs) to the subject. The treatment permits to decrease plasma CCL2 concentration and to change the polarization of blood macrophages from an M1 inflammatory phenotype to an anti-inflammatory M2 phenotype involved in tissue repair.
Resumen de: NL4000209A
The present invention discloses a Polygonum multiflorum exosome and its extraction method and application. The specific extraction method of the Polygonum multifiorum exosome comprises the following steps: removing the rootlets of fresh Polygonum multifiorum fruits, cutting them into pieces, juicing, collecting the filtrate, subjecting the filtrate to continuous centrifugation and differential centrifugation to obtain the supernatant, continuing to perform ultrahigh-speed centrifugation on the supernatant to collect the precipitate, and resuspending the precipitate, which is the Polygonum multifiorum exosome extract. The Polygonum multiflorum exosome obtained by the present invention is applied in the preparation of drugs for the prevention and treatment of Alzheimer’s disease. By using 3XTgAD transgenic homozygous mice, the extracted Polygonum multiflorum exosome is injected into AD mice via tail vein injection, and small animal behavioral methods, such as open field test, novel object recognition, and water maze, are employed to observe changes in the cognitive behavior of the mice, thereby verifying the improvement of cognitive impairment in AD mice by the Polygonum multiflorum exosome.
Nº publicación: AU2024260719A1 06/11/2025
Solicitante:
ALNYLAM PHARMACEUTICALS INC
ALNYLAM PHARMACEUTICALS, INC
Resumen de: AU2024260719A1
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.
BOPI
Sede Electrónica
