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Tratamientos de Alzheimer, Parkinson, Huntington y Esclerosis lateral amiotrófica

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LastUpdate Última actualización 02/05/2026 [08:47:00]
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HTT REPRESSORS AND USES THEREOF

NºPublicación:  US20260116932A1 30/04/2026
Solicitante: 
TAKEDA PHARMACEUTICAL COMPANY LTD [JP]
TAKEDA PHARMACEUTICAL COMPANY LIMITED
US_20260116932_A1

Resumen de: US20260116932A1

Disclosed herein are improved methods and compositions for diagnosing, preventing and/or treating Huntington's Disease. Among other things, provided herein is a gene therapy construct encoding a non-naturally occurring codon-optimized transcription factor (ZFP-TF) comprising a zinc-finger protein (ZFP) sequence and a sequence encoding a transcriptional repression domain, wherein the ZFP-TF expression is driven by a phosphoglycerate kinase 1 (PGK), ubiquitin C (UBC), an EFS, or an EF1alpha promoter.

GENE SEQUENCE CONSTRUCT USED FOR TREATMENT OF CENTRAL NERVOUS SYSTEM DISEASES

NºPublicación:  US20260117234A1 30/04/2026
Solicitante: 
KANGLIN BIOTECH HANGZHOU CO LTD [CN]
Kanglin Biotech (Hangzhou) Co., Ltd.
US_20260117234_A1

Resumen de: US20260117234A1

0000 A gene sequence construct used for the treatment of central nervous system diseases: by means of the construction of an auto-processing expression vector, tyrosine hydroxylase (TH), GTP-cyclohydrolase I (GCH1), aromatic amino acid dopa decarboxylase (AADC), and so on may be simultaneously expressed; proteins are connected by means of an auto-processing unit (APU); the use of a viral vector to introduce the construct into a target cell may ultimately result in the high-efficiency expression of tyrosine hydroxylase (TH), GTP-cyclohydrolase I (GCH1), aromatic amino acid dopa decarboxylase (AADC), and so on having independent functions, being used in the prevention or treatment of Parkinson's disease, Alzheimer's disease and other neurodegenerative diseases.

GENE CORRECTION INDUCED NEURAL STEM CELLS USING HUNTINGTIN GENE-SPECIFIC GUIDE RNA, AND CELL THERAPEUTIC AGENT USING THE SAME

NºPublicación:  US20260115318A1 30/04/2026
Solicitante: 
KOREA UNIV RESEARCH AND BUSINESS FOUNDATION [KR]
KOREA UNIVERSITY RESEARCH AND BUSINESS FOUNDATION
US_20260115318_A1

Resumen de: US20260115318A1

The present invention relates to: a gene editing composition comprising a guide RNA for editing mutated CAG repeat sequences of neural stem cells (ciNSCs) induced from somatic cells of a Huntington's disease patient; the edited ciNSCs; and a cell therapeutic agent for Huntington's disease, comprising same, wherein, in the edited ciNSCs, Huntington gene mutations are removed by the guide RNA of the present invention, and thus treatment for Huntington's disease and neuronal proliferation can be improved.

METHODS OF TREATING AMYLOID PATHOLOGY

NºPublicación:  WO2026090552A1 30/04/2026
Solicitante: 
THE REGENTS OF THE UNIV OF CALIFORNIA [US]
THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
WO_2026090552_A1

Resumen de: WO2026090552A1

Methods and compositions are provided for treating Alzheimer's disease by administering interleukin-33 (IL-33) to a subject. Methods of gene therapy are also provided for expressing IL-33 in vivo in the brain of a subject in effective amounts sufficient to treat amyloid plaque pathology and reduce numbers of plaques in the brain.

ELASTIN-LIKE POLYPEPTIDES FOR NON-COVALENT CAPTURE OF VESCALAGIN AND ENHANCED PERMEATION THROUGH NASAL MUCOSA

NºPublicación:  WO2026090369A1 30/04/2026
Solicitante: 
RENSSELAER POLYTECHNIC INST [US]
NELSON DEREK [US]
RENSSELAER POLYTECHNIC INSTITUTE
NELSON, Derek
WO_2026090369_A1

Resumen de: WO2026090369A1

A composition and method of using the composition to treat neurological disorders including Alzheimer's disease are disclosed wherein the composition includes an elastin-like polypeptide (ELP) and a drug. The ELP undergoes a transition from a solution phase to a gel phase as the temperature is raised above a transition temperature, and in doing so sequesters at least a portion of the drug into the gel phase. The composition is configured such that intranasal delivery of the composition results in gelation and sequestration of the drug by the ELP, thereby allowing the drug to traverse olfactory mucosa into brain tissue in order to treat neurological disorders, including Alzheimer's disease.

COMPOUNDS

NºPublicación:  WO2026088070A1 30/04/2026
Solicitante: 
PFIZER INC [US]
PFIZER INC.
WO_2026088070_A1

Resumen de: WO2026088070A1

The present invention relates to novel compounds and pharmaceutical compositions. More particularly, the present invention provides novel compounds which are useful for the treatment of and prevention of neurodegenerative disorders including, but not limited to, Alzheimer's Disease (AD), Parkinson's Disease (PD), Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD), and Nasu-Hakola Disease (NHD).

Methods for Providing Rapid Relief of Motor Fluctuations in a Parkinson's Disease Patient

NºPublicación:  US20260115160A1 30/04/2026
Solicitante: 
MERZ PHARMACEUTICALS LLC [US]
Merz Pharmaceuticals, LLC
US_20260115160_A1

Resumen de: US20260115160A1

The present invention provides methods of providing rapid relief of motor fluctuations in a Parkinson's disease patient. The methods of the invention comprise pulmonary administration of levodopa by inhalation at therapeutically effective concentrations such that the patient's plasma levodopa concentration increases by at least about 200 ng/ml within 10 minutes or less post inhalation as compared to the concentration of levodopa in the patient's plasma prior to inhalation of the levodopa and wherein the patient's plasma concentration remains increased by at least about 200 ng/ml for a time period of at least 15 minutes after inhalation. The methods of the invention are particularly useful for treatment of motor fluctuations which arise as a result of poorly controlled levodopa plasma levels in a patient.

INDAZOLE INHIBITORS OF THE WNT SIGNAL PATHWAY AND THERAPEUTIC USES THEREOF

NºPublicación:  US20260116878A1 30/04/2026
Solicitante: 
BIOSPLICE THERAPEUTICS INC [US]
BioSplice Therapeutics, Inc.
US_20260116878_A1

Resumen de: US20260116878A1

Indazole compounds for treating various diseases and pathologies are disclosed. More particularly, the present invention concerns the use of an indazole compound or analogs thereof, in the treatment of disorders characterized by the activation of Wnt pathway signaling (e.g., cancer, abnormal cellular proliferation, angiogenesis, Alzheimer's disease, lung disease and osteoarthritis), the modulation of cellular events mediated by Wnt pathway signaling, as well as genetic diseases and neurological conditions/disorders/diseases due to mutations or dysregulation of the Wnt pathway and/or of one or more of Wnt signaling components. Also provided are methods for treating Wnt-related disease states.

METHODS OF TREATING A NEURODEGENERATIVE DISEASE

NºPublicación:  WO2026088136A1 30/04/2026
Solicitante: 
ARIBIO CO LTD [KR]
ARIBIO CO., LTD.
WO_2026088136_A1

Resumen de: WO2026088136A1

The present disclosure provides a method of treating a neurodegenerative disease such as Alzheimer's disease in a subject. The method includes administering to the subject about 30 mg of AR1001 orally once daily, or a pharmaceutically acceptable salt thereof, and monitoring plasma phosphorylated tau 181 (pTau181) or phosphorylated tau 217 (pTau217) levels in the subject during treatment. The method may further include continuing AR1001 treatment if the subject's plasma pTau181 or pTau217 levels decrease during treatment. The subject may have mild cognitive impairment due to Alzheimer's disease or mild Alzheimer's disease dementia. The method may also include assessing the subject's cognitive function using one or more standardized tests during treatment. AR1001 may be administered in a pharmaceutical composition in the form of a tablet or capsule.

2-(4-PIPERIDYL)-6-(IMIDAZO1,2-BPYRIDAZIN-6-YL)THIENO3,2-BPYRIDINE DERIVATIVES THAT REDUCE THE PROTEIN LEVEL OF HUNTINGTIN (HTT) FOR THE TREATMENT OF HUNTINGTON'S DISEASE

NºPublicación:  WO2026087399A1 30/04/2026
Solicitante: 
F HOFFMANN LA ROCHE AG [CH]
HOFFMANN LA ROCHE INC [US]
F. HOFFMANN-LA ROCHE AG
HOFFMANN-LA ROCHE INC.
WO_2026087399_A1

Resumen de: WO2026087399A1

The invention relates to a compound of formula (I) that reduce the protein level of huntingtin (HTT) and which are useful in the treatment of Huntington's disease.

PROTEIN FOR INHIBITING CONSERVED HELIX OF TDP-43 AND USE THEREOF

NºPublicación:  WO2026086833A1 30/04/2026
Solicitante: 
SHANGHAITECH UNIV [CN]
SHANGHAI JIAO TONG UNIV [CN]
\u4E0A\u6D77\u79D1\u6280\u5927\u5B66
\u4E0A\u6D77\u4EA4\u901A\u5927\u5B66
WO_2026086833_A1

Resumen de: WO2026086833A1

Disclosed in the present invention are a protein for inhibiting the conserved helix of TDP-43 and the use thereof. The protein contains an amino acid sequence as shown in SEQ ID NO: 1 or SEQ ID NO: 2. Further disclosed in the present invention are the use of the protein in the preparation of a drug for diagnosing, preventing and/or treating neurodegenerative diseases, and the use in the preparation of an inhibitor of a target protein containing the conserved helix of TDP-43 LCD. In the present invention, by means of using artificial intelligence-assisted protein design techniques, a high-affinity non-natural protein that precisely binds to the conserved helix of a TDP-43 protein is designed, which is used for inhibiting the participation of the conserved helix region in the formation of a β-sheet aggregation core, is mainly used in the treatment of diseases targeting the TDP-43 protein, such as amyotrophic lateral sclerosis, and is used as a research tool for TDP-43 protein phase separation experiments.

HETEROARYL-AMINE COMPOUNDS AND USE THEREOF AS HDAC6 INHIBITORS

NºPublicación:  EP4731216A1 29/04/2026
Solicitante: 
AUGUSTINE THERAPEUTICS [BE]
Augustine Therapeutics
WO_2024261329_A1

Resumen de: WO2024261329A1

The present invention relates to a compound of formula (I) or a pharmaceutically acceptable salt and/or solvate thereof, wherein Y1 is a 9- or 10-membered bicyclic heteroaryl, Y2 is a 5- or 6-membered heteroaryl or 6-membered aryl, L is a linker, Z1' is selected from -NR23' R24' and -(C3-C7) heterocyclo alkyl comprising at least one nitrogen atom, and R23' and R24' may be various groups. The present invention further relates to a compound of formula (I) as HDAC6 inhibitor, typically for use in the treatment and/or the prevention of an HDAC6-associated disease, such as cancers, neurodegenerative diseases, neuropathies, cardiovascular diseases, or metabolic or hormonal disorders.

ANTI-TDP-43 ANTIBODIES AND USES THEREOF

NºPublicación:  AU2024342582A1 23/04/2026
Solicitante: 
PROTHENA BIOSCIENCES LTD
AU_2024342582_PA

Resumen de: AU2024342582A1

The present disclosure provides antibodies that specifically bind to human TDP-43 and methods of using these antibodies to treat patients with TDP-43-related diseases, including Amyotrophic Lateral Sclerosis (ALS).

THERAPEUTIC AGENT FOR NON-MOTOR SYMPTOMS ASSOCIATED WITH PARKINSON'S DISEASE

NºPublicación:  US20260108494A1 23/04/2026
Solicitante: 
NEUROINNOVATECH APS [DK]
US_20260108494_A1

Resumen de: US20260108494A1

0000 The purpose of the present invention is to provide: a therapeutic agent or a recurrence preventive agent for serotonergic system- or dopaminergic system-related diseases, in particular, mental dysfunction symptoms and other non-motor symptoms of Parkinson's disease; and a method for treating the aforesaid symptoms or preventing the recurrence of the same. A compound represented by formula (1) wherein each symbol is as defined in the description or a pharmaceutically acceptable salt thereof can exhibit an effect of treating serotonergic system- or dopaminergic system-related diseases, in particular, mental dysfunction symptoms and other non-motor symptoms of Parkinson's disease and/or an effect of preventing the recurrence of the same. 0000

TREATMENT OF NEUROPSYCHIATRIC DISORDERS WITH TILIVAPRAM

NºPublicación:  AU2024357688A1 23/04/2026
Solicitante: 
ALTO NEUROSCIENCE INC
AU_2024357688_PA

Resumen de: AU2024357688A1

This invention relates to the treatment of a neuropsychiatric disorder, such as schizophrenia or Parkinson's disease, by administration (for example, transdermally) of tilivapram, zatolmilast, roflumilast, or a pharmaceutically acceptable salt thereof.

BIFIDOBACTERIUM LONGUM SUBSP. INFANTIS FOR ALLEVIATING PARKINSON'S DISEASE AND USE THEREOF

NºPublicación:  US20260108568A1 23/04/2026
Solicitante: 
WECARE PROBIOTICS CO LTD [CN]
US_20260108568_A1

Resumen de: US20260108568A1

0000 Provided are a Bifidobacterium longum subsp. infantis for alleviating Parkinson's disease and a use thereof, and the Bifidobacterium longum subsp. infantis for alleviating Parkinson's disease is named as Bifidobacterium longum subsp. infantis BI03 strain, with a deposit number of CGMCC No. 24473 and deposit date of Mar. 7, 2022. The strain can significantly alleviate the symptoms of Parkinson's disease, specifically manifested in: alleviating Parkinson's disease-related dyskinesia and corticosterone elevation; weakening the neuroinflammation associated with Parkinson's disease; promoting glutathione and weakening brain oxidative stress damage.

ALZHEIMER-TYPE DEMENTIA SUPPRESSANT, AND METHOD FOR PRODUCING SAME

NºPublicación:  US20260108572A1 23/04/2026
Solicitante: 
SAKAMOTO YAKUSOUEN LLC [JP]
US_20260108572_A1

Resumen de: US20260108572A1

0000 The objective of the present invention is to provide an Alzheimer-type dementia suppressant that is safe and thus can be administered daily and utilized as a health food and by which Alzheimer-type dementia can be suppressed, and a method for producing the Alzheimer-type dementia suppressant. In addition, the objective of the present invention is also to provide a use of an aboveground part of Sesamum indicum or an extract thereof for suppressing Alzheimer-type dementia, and a method for suppressing Alzheimer-type dementia. The Alzheimer-type dementia suppressant according to the present invention is characterized in comprising an aboveground part of Sesamum indicum or an extract thereof as an active ingredient.

VACCINES AND ANTIBODIES FOR THE TREATMENT AND PREVENTION OF NEURODEGENERATIVE DISORDERS AND INFLAMMATION RELATED HEALTH CONDITIONS

NºPublicación:  WO2026085054A1 23/04/2026
Solicitante: 
LONGHORN VACCINES AND DIAGNOSTICS LLC [US]
WO_2026085054_A1

Resumen de: WO2026085054A1

The invention is directed to immunological compositions of one or more peptides containing epitopes of PGN, LTA and LPS molecules that induce an immunological response in a mammal, and to multiple antibodies that bind to these epitopes. Immunological compositions and antibodies disclosed herein can be used in the treatment and/or prevention of human health disorders such as bacterial sepsis, inflammation, cancers, tumors, inflammatory diseases and disorders, and neurodegenerative disorders such as, but not limited to Alzheimer's disease, frontotemporal dementia, chronic traumatic encephalopathy (CTE), Lewy body dementia and/or limbic predominant age-related TDP-43 encephalopathy (LATE).

ANTI-BETA AMYLOID ANTIBODIES AND USES THEREOF

NºPublicación:  WO2026082154A1 23/04/2026
Solicitante: 
LEPU BIOPHARMA CO LTD [CN]
WO_2026082154_A1

Resumen de: WO2026082154A1

Deposition of N-terminally truncated and pyroglutamate-modified Abeta peptides (AβpE3) play an important role in the aggregation of the amyloid-β (Aβ) protein in the brain. The N-terminally truncated and pyroglutamate-modified Abeta peptides, therefore, can be a suitable target for the prevention and treatment of Alzheimer's Disease (AD). Disclosed herein are antibodies and fragments having excellent specificity to N-terminally truncated and pyroglutamate-modified Abeta peptides, as well as uses of these antibodies and fragments for the prevention and treatment of AD.

COMPOSITION CONTAINING BRAIN PROTEIN HYDROLYSATE, PREPARATION METHOD, AND USE

NºPublicación:  WO2026081146A1 23/04/2026
Solicitante: 
LIU SHUANG [CN]
\u5218\u723D
WO_2026081146_A1

Resumen de: WO2026081146A1

A composition containing a brain protein hydrolysate, a preparation method, and a use. The composition comprises the following raw materials: porcine brain protein hydrolysate, deer brain protein hydrolysate, bovine collagen peptide powder, Gastrodia elata powder, sea buckthorn powder, hawthorn powder, chicken gizzard membrane powder, vitamin C, vitamin B1, vitamin B3, vitamin B5, vitamin B6, and vitamin B12. The composition has anti-inflammatory and antioxidant effects, and can also be used for preparing a medicament for preventing Alzheimer's disease.

MICROTUBULE ASSOCIATED PROTEIN TAU (MAPT) iRNA FORMULATIONS AND METHODS OF USE THEREOF

NºPublicación:  WO2026085233A1 23/04/2026
Solicitante: 
ALNYLAM PHARMACEUTICALS INC [US]
WO_2026085233_A1

Resumen de: WO2026085233A1

The disclosure relates to formulations of double stranded ribonucleic acid (dsRNAi) agents targeting a MAPT gene, as well as methods of inhibiting expression of a MAPT gene and methods of treating subjects having a MAPT-associated neurodegenerative disease or disorder, e.g., Alzheimer's disease, FTD, PSP, or other tauopathy, using such dsRNAi agent formulations.

TREATMENT OF PATHOLOGIES ASSOCIATED WITH DOWN SYNDROME WITH ANTI-FSH ANTIBODIES

NºPublicación:  WO2026085535A2 23/04/2026
Solicitante: 
ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI [US]
WO_2026085535_A2

Resumen de: WO2026085535A2

Disclosed is a method for treating for treating pathologies associated with Down syndrome (e.g., symptoms of Alzheimer's disease and metabolic abnormalities), preventing the onset of pathologies associated with Down syndrome in patients with Down syndrome, reducing cognitive or functional decline, or reducing symptom load in a subject having Down Syndrome, in need or at risk thereof, comprising administering to said subject a therapeutically effective amount of an anti-Follicle Stimulating Hormone (FSH) antibody, or an antigen-binding portion thereof.

FUSED HETEROARYL DERIVATIVE, PREPARATION METHOD THEREFOR, AND APPLICATION THEREOF

NºPublicación:  WO2026082004A1 23/04/2026
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
WO_2026082004_A1

Resumen de: WO2026082004A1

The present invention relates to a fused heteroaryl derivative, a preparation method therefor, and an application thereof. The fused heteroaryl derivative can be used in a sodium channel inhibitor, and/or in a GABA receptor agonist, or in treating neuropsychiatric disorders such as epilepsy, Parkinson's, schizophrenia, or depression.

KRÄUTERLÖSUNG, DIE RUTA GRAVEOLENS UND SAXIFRAGA ENTHÄLT, UND VERFAHREN ZU IHRER HERSTELLUNG

NºPublicación:  DE102024130715A1 23/04/2026
Solicitante: 
SCHILLING JOHANN GEORG [DE]
DE_102024130715_A1

Resumen de: DE102024130715A1

Die vorliegende Offenbarung bezieht sich auf eine pflanzliche Mundspüllösung, die Ruta graveolens und Saxifraga enthält und speziell zur Vorbeugung von neurologischen Erkrankungen wie Parkinson, Alzheimer, Lähmungen und Nervenschäden entwickelt wurde. Verfahren zur Herstellung der pflanzlichen Mundspüllösung durch Einweichen einer fein gemahlenen Mischung aus Ruta graveolens und Saxifraga in Alkohol. Die resultierende Lösung wird als Mundspülung verabreicht und zum Spülen und Gurgeln verwendet. Die Erfindung bietet eine wirksame, natürliche therapeutische Alternative zur Behandlung neurodegenerativer Erkrankungen, zur Förderung des Neuroschutzes und zur möglichen Unterstützung der Regeneration geschädigter Nervenzellen.

USE OF NLRP3 INHIBITOR FOR TREATING PARKINSON'S DISEASE OR PARKINSONISM

Nº publicación: WO2026082108A1 23/04/2026

Solicitante:

QINGDAO BORSON TAI TECH CO LTD [CN]
\u9752\u5C9B\u535A\u68EE\u6CF0\u79D1\u6280\u6709\u9650\u516C\u53F8

WO_2026082108_A1

Resumen de: WO2026082108A1

The present invention relates to use of an NLRP3 inhibitor for treating Parkinson's disease or Parkinsonism. Specifically, the present invention relates to use of a compound represented by formula (I) or a pharmaceutically acceptable salt thereof in the preparation of a drug for treating Parkinson's disease or Parkinsonism, wherein each substituent is as defined in the specification.

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