Tratamientos de Alzheimer, Parkinson, Huntington y Esclerosis lateral amiotrófica

HDAC6 INHIBITORS AND USES THEREOF

Resumen de: AU2025230659A1

Abstract Provided herein are compounds that inhibit HDAC6, a protein whose activity is associated with a variety of diseases (e.g., cancer, neurological disorders). Also provided are pharmaceutical compositions and kits comprising the compounds, and methods of treating HDAC6-related diseases and disorders (e.g., Alzheimer's disease, cancer) with the compounds in a subject, by administering the compounds and/or compositions described herein. Abstract Provided herein are compounds that inhibit HDAC6, a protein whose activity is associated with a variety of diseases (e.g., cancer, neurological disorders). Also provided are pharmaceutical compositions and kits comprising the compounds, and methods of treating HDAC6-related diseases and disorders (e.g., Alzheimer's disease, cancer) with the compounds in a subject, by administering the compounds and/or compositions described herein.

OPTIMISED DOSAGE OF DIAMINOPHENOTHIAZINES IN POPULATIONS

Resumen de: US2025302843A1

The invention provides novel dosing regimens for Leuco-Methylthioninium (LMT) compounds which maximise the proportion of subjects in which the MT concentration will exceed concentrations in which therapeutic efficacy in relation to treatment of neurodegenerative disorders such as Alzheimer's disease and rontotemporal dementias can be achieved, while maintaining a desirable clinical profile. Also provided are LMT-containing dosage units and other compositions.

METHODS TO DETECT AB PROTEOFORMS AND USE THEREOF

Resumen de: US2025306037A1

The present disclosure relates to methods useful to identify subjects having an increased risk for conversion to mild cognitive impairment (MCI) due to Alzheimer's disease (AD) and/or stage a subject prior to the onset of mild cognitive impairment (MCI) due to Alzheimer's disease (AD) and/or identify subjects with Aβ amyloidosis and/or to identify subjects who should or should not undergo further testing or treatment for Aβ amyloidosis, as well as methods for treating subjects diagnosed with Aβ amyloidosis by the methods disclosed herein.

METHODS OF TREATING NEUROCOGNITIVE DISORDERS, CHRONIC PAIN AND REDUCING INFLAMMATION

Resumen de: US2025302851A1

The disclosure provides methods for treating a subject in need thereof comprising administering to the subject a therapeutically-effective dose of psilocybin. The methods described herein may be used to treat a variety of diseases, disorders, and conditions. For example, the methods may be used to treat neurocognitive disorders (e.g., Alzheimer's disease, Parkinson's disease), ADHD, Epilepsy, Autism, Sleep-wake disorders, Chronic pain, Inflammatory Disorders, IBD, Stroke, ALS, and/or Multiple Sclerosis.

BIOMARKERS OF AMYOTROPHIC LATERAL SCLEROSIS AND USES THEREOF

Resumen de: AU2024238511A1

The present disclosure relates to biomarkers and uses thereof in methods for selecting a patient diagnosed with amyotrophic lateral sclerosis (ALS) for an ALS therapy. The present disclosure further relates to methods for identifying the severity of ALS in a patient, treating an ALS patient, and monitoring efficacy of an ALS treatment.

COMPOUNDS AND METHODS FOR MODULATING ALPHA-SYNUCLEIN EXPRESSION

Resumen de: AU2024220937A1

Provided herein are compounds, phannaceutical compositions, and methods of use for reducing the amount or activity of SNCA mRNA in a cell or subject, and in certain instances reducing the amount of alpha-synuclein protein in a cell or subject. Such compounds, pharmaceutical compositions, and methods of use are useful to ameliorate at least one symptom or hallmark of a synucleinopathy. Such synucleinopathies include Parkinson's disease, dementia with Lewy bodies (DLB), diffuse Lewy body disease, Parkinson's disease dementia (PDD), pure autonomic failure, multiple system atrophy (MSA), neuronopathic Gaucher's disease, and Alzheimer's disease.

RNAi Agents for Inhibiting Expression of Microtubule Associated Protein Tau (MAPT), Compositions Thereof, and Methods of Use

Resumen de: US2025302980A1

Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a microtubule associated protein tau (MAPT) gene. The MAPT RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of a MAPT gene. The MAPT RNAi agents are conjugated to an antigen binding protein that may enable subcutaneous delivery of the RNAi agents by facilitating crossing of the blood brain barrier (BBB). Pharmaceutical compositions that include one or more MAPT RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described MAPT RNAi agents to central nervous system (CNS) tissue, in vivo, provides for inhibition of MAPT gene expression and a reduction in MAPT activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including Alzheimer's disease, Frontotemporal lobar degeneration dementia (FTLD), Progressive supranuclear palsy, and other tauopathies.

METHOD AND MOLECULES FOR REDUCING AXONAL TAU PROTEIN ACCUMULATION THROUGH BLOCKING OF HNRNP R-MEDIATED MAPT MRNA TRANSPORT FOR TREATMENT OF ALZHEIMER'S DISEASE

Resumen de: US2025304958A1

This application includes methods for reducing axonal tau protein. Those methods include inhibiting the binding between MAPT mRNA and hnRNP R.

ANTIBODIES TO AMYLOID BETA

Resumen de: US2025304670A1

Antibody for human amyloid beta. Antibody selectively binds human amyloid beta 42 peptide over human amyloid beta 40 peptide. Antibodies specific for amyloid beta 42 as therapeutic agents for binding amyloid beta 42 peptide and treating conditions associated with amyloidosis, such as Alzheimer's disease.

MULTISPECIFIC BINDING MOLECULES AND METHODS OF USE THEREOF

Resumen de: US2025304709A1

Multispecific binding molecules having a first binding domain targeting a blood brain barrier target and a second binding domain targeting a neuron target, astrocyte target and/or glial cell target, and uses thereof in aiding the treatment of central nervous system diseases including neurodegenerative diseases such as Alzheimer's disease, Huntington's disease, Parkinson's diseases, Progressive Supranuclear Palsy (PSP), Amyotrophic Lateral Sclerosis (ALS), Frontal Temporal Dementia (FTD), autism, catalepsy, encephalitis, migraine, and Tourette's.

CRYSTALS OF ANTI-HUMAN TAU MONOCLONAL ANTIBODY

Resumen de: WO2025207501A1

Crystals of an antibody that specifically binds to human tau protein phosphorylated at serine 413 are provided, as well as methods of producing such antibody crystals, compositions comprising the crystals, and uses of such compositions for treating a disease, e.g., Alzheimer's disease. Crystals suitable for X-ray diffraction are also provided, and the inventors herein have used such crystals to solve the three-dimensional structure of the antibody to 2.76 Å resolution.

COMPOSITIONS OF BIOACTIVE AGENTS

Resumen de: WO2025201405A1

A pharmaceutical composition comprising at least three components selected from the group consisting of Compound A, Compound B, Compound C, and Compound D, and their use in treating a neurodegenerative disease (e.g., Alzheimer's disease) and improving cognition or memory.

METHODS FOR TREATING ALS WITH A RHO KINASE INHIBITOR BASED ON USE OF NEUROFILAMENT LIGHT CHAIN BIOMARKER

Resumen de: WO2025207476A1

A method of treating amyotrophic lateral sclerosis (ALS), includes: determining for a patient diagnosed with ALS, a pre-treatment amount of neurofilament light chain (NfL) in the patient's serum or plasma; orally administering to the patient a first rho kinase inhibitor in a predetermined amount for a predetermined period of time; determining a post-treatment amount of NfL in the patient's serum or plasma; when the post-treatment of amount of NfL is determined to be lower than the pre-treatment amount of NfL, orally administering to the patient a second rho kinase inhibitor in a therapeutically effective amount for treating ALS; and when the post-treatment of amount of NfL is determined to be not lower than the pre-treatment amount of NfL, withholding administration of the second rho kinase inhibitor to the patient.

METHODS FOR TREATING ALZHEIMER’S DISEASE

Resumen de: US2025302864A1

Methods for the prevention or treatment of Alzheimer's disease in a human patient are disclosed comprising administering a hydroxypropyl-beta-cyclodextrin.

INDAZOLE DERIVATIVE AND PREPARATION METHOD THEREFOR AND USE THEREOF

Resumen de: WO2025201511A1

The present invention relates to an indazole derivative and a preparation method therefor and a use thereof. The indazole derivative can be used for LRRK2 kinase inhibition or for treating Parkinson's disease (PD).

Methods for Reducing Ataxin-2 Expression

Resumen de: US2025304970A1

Provided herein are methods for decreasing Ataxin-2 mRNA expression. Such methods are useful to ameliorate symptoms of Ataxin-2 associated diseases. Such Ataxin-2 associated diseases include amyotrophic lateral sclerosis (ALS). Such symptoms include loss of motor function, reduced CMAP amplitude, denervation, and loss of motor neurons.

1-(CYCLOBUTYLIDENEMETHYL)-2,4,5-TRIMETHOXYBENZENE COMPOUND, PREPARATION METHOD THEREFOR AND USE THEREOF

Resumen de: EP4624448A1

A 1-(cyclobutylidenemethyl)-2, 4, 5-trimethoxybenzene compound and a preparation method and use thereof are provided, belonging to the field of drug development technology. The prepared 1-(cyclobutylidenemethyl)-2, 4, 5-trimethoxybenzene compound may be used for the preparation of an antiepileptic drug and drugs for treatment and/or prevention of traumatic craniocerebral injury disorders, ischemic stroke, hemorrhagic stroke, and Parkinson's disease.

L- Composition for preventing improving or treating Parkinson's disease comprising combination of lactic acid bacteria and L-tyrosine

Resumen de: KR20250141094A

본 발명의 일 예는 락티플란티바실러스 플란타룸(Lactiplantibacillus plantarum) KACC 11451 균주, 락티플란티바실러스 파라플란타룸(Lactiplantibacillus paraplantarum) KACC 12373 균주 또는 락티플란티바실러스 펜토수스(Lactiplantibacillus pentosus) KACC 12428 균주에서 선택되는 1종 이상의 균주 및 L-티로신을 포함하는 파킨슨병 예방, 개선 또는 치료용 조성물을 제공한다. 본 발명의 일 예에 따른 특정 유산균은 L-티로신의 레보도파로의 전환 활성이 매우 높다. 또한, 본 발명의 일 예에 따른 특정 유산균 및 L-티로신을 포함하는 조성물은 생체내에서 레보도파를 생산할 수 있도록 제형화될 수 있고, 파킨슨병의 예방, 개선 또는 치료를 위한 건강기능식품 또는 의약품으로 사용될 수 있다.

CLASS OF DEXBORNEOL OR FENCHYL ALCOHOL ESTER DERIVATIVES OF 2-HYDROXYNICOTINIC ACID AND PHARMACEUTICAL USES THEREOF

Resumen de: WO2025194870A1

The present invention belongs to the technical field of biomedicine, and relates to a class of dexborneol or fenchyl alcohol ester derivatives of 2-hydroxynicotinic acid, and pharmaceutical uses thereof. The structural general formula of the derivatives is (1), R1 being selected from -H, -Cl, a hydrocarbon group having 1-8 carbon atoms, -CF3, -CF2H, -CN, -SCH3, -OH, -CHO, -CH2OH, -CH2NH2, -COOH, halogenated phenyl, or -NR4R5; R4, R5 are independently selected from -H or -CH3; R2 is selected from -H, an alkyl group having 1-6 carbon atoms, an acyl group having 2-7 carbon atoms, or a carbamoyl group; R 3 is selected from formula (a) or formula (b); X is selected from -CH= or -N=. The derivatives have good inflammation-inhibiting effects, and at high concentrations, they exhibit low cytotoxicity and are safer. The derivatives can be used for preparing a drug for treating inflammation and inflammation-related diseases such as stroke, neuropathic pain, depression and Alzheimer's disease.

ANTI-TAU ANTIBODIES

Resumen de: WO2025198916A1

The present disclosure provides anti-tau antibodies, including compositions and methods of using such antibodies for treating Alzheimer's disease.

FORMULATIONS AND METHODS FOR TREATING DEMENTIA AND ALZHEIMER'S DISEASE

Resumen de: WO2025199248A1

Applicants have discovered differential expression and activity of various miRNAs in neurodegenerative diseases. These miRNAs can serve as therapeutic targets for prevention and treatment of neurodegenerative diseases. Accordingly, embodiments of the invention include formulations and methods for treating neurodegenerative diseases such as dementia and Alzheimer's disease. The formulations and methods can include five microRNAs and two small molecule therapeutics. In aspects, the miRNAs are MIR-145-3p, let-7c-3p, MIR-383-5p, MIR-548aj-3p and MIR-548x-3p. In aspects, the small molecule therapeutics are GATC-49, fucosterol and fucoidan. In aspects, the compounds work synergistically with one another.

PHARMACEUTICAL PRESERVATION OF CREE ACTIVATION WITH NITARSONE FOR USE IN THE TREATMENT OF NEURODEGENERATIVE DISEASES

Resumen de: US2025295627A1

A compound nitarsone, or salt thereof is provided for use in the treatment of a neurodegenerative disease, such as Alzheimer's disease (AD), dementia, Parkinson's disease (RD) or amyotrophic lateral sclerosis (ALS). A pharmaceutical composition is also provided that includes compound, or salt thereof for use in the treatment of a neurodegenerative disease.

BENZOTHIOPHENE COMPOUND

Resumen de: US2025296939A1

The present invention aims to provide a medicament capable of treating and/or preventing diseases associated with oxidative stress by inhibiting the protein-protein interaction between Keap1 and Nrf2 and activating Nrf2. The present invention relates to a compound represented by the following formula (1):wherein each symbol is as described in the DESCRIPTION, or a pharmaceutically acceptable salt thereof. In addition, the present invention also relates to a medicament containing the compound, for the prophylaxis and/or treatment of diseases involving oxidative stress selected from the group consisting of chronic kidney disease, non-alcoholic steatohepatitis, chronic obstructive pulmonary disease, radiation skin disorder, radiation mucosal disorder, cardiac failure, pulmonary arterial hypertension, Parkinson's disease, Friedreich's ataxia, multiple sclerosis, age-related macular degeneration, retinitis pigmentosa and glaucoma.

MMP-14 POTENCY ASSAY FOR MESENCHYMAL STEM CELLS

Resumen de: WO2025199451A2

Compositions and methods are disclosed herein for the treatment of Alzheimer's disease with allogeneic mesenchymal stem cells (MSCs). The methods of treatment involve an administration of a composition of allogeneic mesenchymal stem cells to a subject in need thereof, wherein the effectiveness of the treatment methods can be determined through the measurement of specific biomarkers and improved cognitive or quality-of-life function.

COMPOSITIONS AND METHODS FOR TREATMENT AND PREVENTION OF ALZHEIMER'S DISEASE

Nº publicación: WO2025199495A1 25/09/2025

Solicitante:

UNIV OF MARYLAND BALTIMORE [US]
THE GENERAL HOSPITAL CORP [US]
UNIVERSITY OF MARYLAND, BALTIMORE,
THE GENERAL HOSPITAL CORPORATION