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Tratamientos de Alzheimer, Parkinson, Huntington y Esclerosis lateral amiotrófica

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LastUpdate Última actualización 10/10/2025 [06:46:00]
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Solicitudes publicadas en los últimos 60 días / Last 60 days publications
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METHODS FOR TREATING OBSESSIVE COMPULSIVE RELATED DISORDERS, TIC DISORDERS AND GLUTAMATE EXCITOTOXICITY RELATED DISORDERS

NºPublicación:  AU2024230525A1 09/10/2025
Solicitante: 
BIOHAVEN THERAPEUTICS LTD
BIOHAVEN THERAPEUTICS LTD
AU_2024230525_PA

Resumen de: AU2024230525A1

The present invention provides for methods of treating obsessive-compulsive disorder (OCD) and OCD-related disorders (body dysmorphic disorder, hoarding disorder, trichotillomania (hair-pulling disorder), excoriation (skin-picking) disorder, substance/medication-induced obsessive-compulsive and related disorder, obsessive- compulsive and related disorder due to another medical condition, and other specified and unspecified obsessive-compulsive and related disorders), Tic disorders including Tourette syndrome, autism spectrum disorder (ASD) and glutamate excitotoxicity related disorders including amyotrophic lateral sclerosis (ALS), Parkinson's disease, traumatic brain injury, multiple sclerosis, Huntington's disease, and schizophrenia, comprising the step of administering an effective amount of a histamine type 1 receptor agonist and/or histamine type 3 receptor antagonist, such as betahistine or its pharmaceutically acceptable salts, analogs, metabolites, prodrugs, derivatives, metabolites, co-crystals, modifications, solvates, hydrates, isotopes, tautomers, esters, polymorphs or stereoisomers.

TREHALOSE FOR TREATING HUNTINGTON'S DISEASE

NºPublicación:  EP4626442A1 08/10/2025
Solicitante: 
SEELOS THERAPEUTICS INC [US]
Seelos Therapeutics, Inc
AU_2023403437_PA

Resumen de: MX2025006287A

The present disclosure relates to compositions comprising trehalose and methods of using same for the treatment of Huntington's disease.

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

NºPublicación:  WO2025207476A1 02/10/2025
Solicitante: 
WOOLSEY PHARMACEUTICALS INC [US]
WOOLSEY PHARMACEUTICALS, INC

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.

COMPOSITIONS OF BIOACTIVE AGENTS

NºPublicación:  WO2025201405A1 02/10/2025
Solicitante: 
RENUMEMO THERAPEUTICS LLC [US]
JIANG TAO [CN]
RENUMEMO THERAPEUTICS LLC,
JIANG, Tao

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.

CRYSTALS OF ANTI-HUMAN TAU MONOCLONAL ANTIBODY

NºPublicación:  WO2025207501A1 02/10/2025
Solicitante: 
MERCK SHARP & DOHME LLC [US]
MERCK SHARP & DOHME LLC

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.

INDAZOLE DERIVATIVE AND PREPARATION METHOD THEREFOR AND USE THEREOF

NºPublicación:  WO2025201511A1 02/10/2025
Solicitante: 
SHANGHAI JINGXIN BIOMEDICAL CO LTD [CN]
ZHEJIANG JINGXIN PHARMACEUTICAL CO LTD [CN]
\u4E0A\u6D77\u4EAC\u65B0\u751F\u7269\u533B\u836F\u6709\u9650\u516C\u53F8,
\u6D59\u6C5F\u4EAC\u65B0\u836F\u4E1A\u80A1\u4EFD\u6709\u9650\u516C\u53F8

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).

COMPOUNDS AND METHODS FOR MODULATING ALPHA-SYNUCLEIN EXPRESSION

NºPublicación:  AU2024220937A1 02/10/2025
Solicitante: 
IONIS PHARMACEUTICALS INC
IONIS PHARMACEUTICALS, INC
AU_2024220937_A1

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.

MULTISPECIFIC BINDING MOLECULES AND METHODS OF USE THEREOF

NºPublicación:  US2025304709A1 02/10/2025
Solicitante: 
ELI LILLY AND COMPANY [US]
Eli Lilly and Company
US_2025304709_A1

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.

ANTIBODIES TO AMYLOID BETA

NºPublicación:  US2025304670A1 02/10/2025
Solicitante: 
MEDIMMUNE LTD [GB]
MedImmune Limited
US_2025304670_A1

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.

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

NºPublicación:  US2025304958A1 02/10/2025
Solicitante: 
JULIUS MAXIMILIANS UNIV WUERZBURG [DE]
JULIUS-MAXIMILIANS-UNIVERSIT\u00C4T W\u00DCRZBURG
US_2025304958_A1

Resumen de: US2025304958A1

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

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

NºPublicación:  US2025302980A1 02/10/2025
Solicitante: 
ARROWHEAD PHARMACEUTICALS INC [US]
Arrowhead Pharmaceuticals, Inc
US_2025302980_A1

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.

METHODS OF TREATING NEUROCOGNITIVE DISORDERS, CHRONIC PAIN AND REDUCING INFLAMMATION

NºPublicación:  US2025302851A1 02/10/2025
Solicitante: 
COMPASS PATHFINDER LTD [GB]
Compass Pathfinder Limited
US_2025302851_A1

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.

METHODS TO DETECT AB PROTEOFORMS AND USE THEREOF

NºPublicación:  US2025306037A1 02/10/2025
Solicitante: 
WASHINGTON UNIV [US]
Washington University
WO_2023220276_PA

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 for Reducing Ataxin-2 Expression

NºPublicación:  US2025304970A1 02/10/2025
Solicitante: 
IONIS PHARMACEUTICALS INC [US]
Ionis Pharmaceuticals, Inc
US_2022162615_A1

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.

HDAC6 INHIBITORS AND USES THEREOF

NºPublicación:  AU2025230659A1 02/10/2025
Solicitante: 
EIKONIZO THERAPAPEUTICS INC
Eikonizo Therapapeutics, Inc
JP_2022543106_PA

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.

BIOMARKERS OF AMYOTROPHIC LATERAL SCLEROSIS AND USES THEREOF

NºPublicación:  AU2024238511A1 02/10/2025
Solicitante: 
THE METHODIST HOSPITAL
COYA THERAPEUTICS INC
THE METHODIST HOSPITAL,
COYA THERAPEUTICS, INC
AU_2024238511_PA

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.

METHODS FOR TREATING ALZHEIMER’S DISEASE

NºPublicación:  US2025302864A1 02/10/2025
Solicitante: 
CYCLO THERAPEUTICS INC [US]
CYCLO THERAPEUTICS, INC
US_2025302864_A1

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.

OPTIMISED DOSAGE OF DIAMINOPHENOTHIAZINES IN POPULATIONS

NºPublicación:  US2025302843A1 02/10/2025
Solicitante: 
WISTA LABORATORIES LTD [SG]
WisTa Laboratories Ltd
US_2025302843_A1

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.

SPIRO DERIVATIVES AS M4 ACTIVATORS/MODULATORS AND USES THEREOF

NºPublicación:  MX2025007982A 01/10/2025
Solicitante: 
CEREVEL THERAPEUTICS LLC [US]
CEREVEL THERAPEUTICS, LLC
KR_20250133755_A

Resumen de: MX2025007982A

The present disclosure provides compounds of Formula I: (l) (I), or an N- oxide thereof, or a pharmaceutically acceptable salt of the compound or the N-oxide, wherein: A, Y, m, n,p, R<sup>1</sup>, R<sup>2</sup>, R<sup>3</sup>, R<sup>3a</sup>, R<sup>4</sup>, R<sup>5</sup>, R<sup>6</sup>, R<sup>7</sup>, and Z are as described herein; processes for the preparation of; intermediates used in the preparation of; and compositions containing such compounds, N-oxides, or salts, and their uses for treating M4-mediated (or M4-associated) disorders including, e.g., Alzheimer's Disease, Parkinson's Disease, schizophrenia (e.g., its cognitive and negative symptoms), pain, addiction, and a sleep disorder.

HUMANISED ANTIBODY AGAINST AMYLOID BETA 42

NºPublicación:  MX2025009326A 01/10/2025
Solicitante: 
ALZINOVA AB [SE]
ALZINOVA AB
AU_2024238598_A1

Resumen de: MX2025009326A

The present invention provides a humanised antibody comprising an antigen-binding domain capable of binding specifically to Aβ42 prefibrillar oligomers with β structure, said antigen-binding domain comprising: (iii) a heavy chain variable region (VH) comprising the sequence of SEQ ID NO.1; or (iv) a light chain variable region (VL) comprising the sequence of SEQ ID NO.2; or a combination thereof. Also provided is the use of the antibody in the treatment of an amyloid disease, and particularly Alzheimer's disease, conjugates and pharmaceutical compositions comprising the antibody, and nucleic acid molecules encoding the antibody or a heavy or light chain polypeptide thereof, as well as vectors and host cells comprising such a molecule.

DUAL INHIBITORS FOR THE TREATMENT OF ALZHEIMER'S DISEASE

NºPublicación:  MX2025010369A 01/10/2025
Solicitante: 
UNIV DE BARCELONA [ES]
QPS CLINICAL SERVICES GMBH [AT]
UNIVERSITAT DE BARCELONA,
QPS CLINICAL SERVICES GMBH
WO_2024184305_A1

Resumen de: MX2025010369A

Compounds (I) are provided, where R<sup>1</sup> and R<sup>2</sup> are H or (C1-C3)-alkyl; X is a linear methylene chain of formula -CH<sub>2</sub><sub>n</sub>- with n = 0, 1 or 2, or a biradical from a branched saturated (C2-C4)-alkylene chain; and A is either a C-radical from a non-aromatic polycyclic 6- to 15-membered carbocyclic ring system, or a C-radical from a polycyclic 6- to 15-membered heterocyclic ring system having one or two O, S or N; wherein the C-radicals are unsubstituted or substituted. Compounds (I) are simultaneously inhibitors of soluble epoxide hydrolase and inhibitors of glutaminyl cyclase. Besides, they reduce the levels of pro-inflammatory cytokines in LPS stimulated BV2 cells, display low cytotoxicity, and have good BBB permeability. Thus, they are useful as multitarget compounds for the prevention or treatment of Alzheimer's disease.

ALPHA-SYNUCLEIN BINDERS AND METHODS OF USE

NºPublicación:  MX2025010406A 01/10/2025
Solicitante: 
MERCK SHARP & DOHME LLC [US]
MERCK SHARP & DOHME LLC
WO_2024186584_PA

Resumen de: MX2025010406A

The invention is directed to compounds of Formula I (I) or their pharmaceutically acceptable salts, which may be suitable for imaging alpha-synuclein pathology and hence are useful in binding and imaging alpha-synuclein aggregates in patients with Parkinson's Disease. More specifically, this invention relates to a method of using the compounds of this invention as tracers in positron emission tomography (PET) imaging to study alpha-synuclein in brain <i>in vivo</i> to allow diagnosis of Parkinson's Disease and other neurodegenerative diseases characterized by alpha-synuclein pathology. The invention further relates to a method of measuring clinical efficacy of therapeutic agents for Parkinson's Disease and other neurodegenerative diseases characterized by alpha-synuclein pathology.

PYRIDAZINE COMPOUNDS, THEIR PREPARATION, AND THEIR THERAPEUTIC USES

NºPublicación:  MX2025010782A 01/10/2025
Solicitante: 
SANOFI [FR]
SANOFI
TW_202502746_A

Resumen de: MX2025010782A

The present invention relates to a compound of formula (I) wherein n is 1 or 2, R1 is halogen, (C<sub>1</sub>-C<sub>4</sub>)alkyl, halo(C<sub>1</sub>-C<sub>4</sub>)alkyl, (C<sub>1</sub>-C<sub>4</sub>)alkoxy, halo(C<sub>1</sub>-C<sub>4</sub>)alkoxy, ethynyl, propargyl, or (C<sub>3</sub>-C<sub>6</sub>)cycloalkyl, or two R1 form a cyclopentane ring fused to the phenol; R2 and R3 represent H, cyano, ethynyl, propargyl, (C<sub>1</sub>-C<sub>4</sub>)alkyl, hydroxy(C<sub>1</sub>-C<sub>4</sub>)alkyl or a halo(C<sub>1</sub>-C<sub>4</sub>)alkyl, or R2 and R3 form together with the atoms connecting them a (C<sub>5</sub>- C<sub>6</sub>)carbocyclic ring fused to the pyridazine ring; R4 and R5 form together with N to which they are attached an optionally substituted 3-7 membered monocyclic heterocycloalkyl ring, 8-11 membered bicyclic heterocycloalkyl ring or 7-12 membered bicyclic heterocyclic spiro ring. The present invention also relates to a medicament and a pharmaceutical composition comprising said compound of formula (I), as well as their therapeutic uses, in particular as inhibitor of NOD-like receptor protein 3 inflammasome for treating for example Parkinson's disease or frontotemporal Dementia.

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

NºPublicación:  EP4624448A1 01/10/2025
Solicitante: 
CHENGDU XINRUI TAIKANG TECH CO LTD [CN]
CHENGDU XINRUI TAIKANG TECHNOLOGY CO., LTD
EP_4624448_PA

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.

GLUCOCEREBROSIDASE (GBA) POLYMER CONJUGATE, PREPARATION METHOD AND USE FOR NANOTECHNOLOGICAL BASED ENZYME REPLACEMENT THERAPY

Nº publicación: MX2025006236A 01/10/2025

Solicitante:

FUNDACIO HOSPITAL UNIV VALL HEBRON INSTITUT DE RECERCA VHIR [ES]
UNIV AUT\u00D2NOMA DE BARCELONA [ES]
CENTRO DE INVESTIG PRINCIPE FELIPE [ES]
DANIEL RUIZ MOLINA [ES]
FUNDACI\u00D3 HOSPITAL UNIVERSITARI VALL HEBRON - INSTITUT DE RECERCA (VHIR),
UNIVERSITAT AUT\u00D2NOMA DE BARCELONA,
CENTRO DE INVESTIGACI\u00D3N PR\u00CDNCIPE FELIPE,
Daniel RUIZ MOLINA

CN_120731086_A

Resumen de: MX2025006236A

The present invention relates to the medical field, in particular, to a nanotechnological based Enzyme Replacement Therapy, preferably for Parkinson's disease, based on the restoration of lysosomal glucocerebrosidase activity through enzyme-polymer nanoconjugation of GBA, the GBA polymer conjugate for such use, and its manufacturing method.

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