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Tratamientos de Alzheimer, Parkinson, Huntington y Esclerosis lateral amiotrófica

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LastUpdate Última actualización 07/01/2026 [07:44:00]
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Solicitudes publicadas en los últimos 60 días / Last 60 days publications
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COMPOUNDS AS NLRP3 PET RADIOTRACERS AND COMPOSITIONS AND USES THEREOF

NºPublicación:  WO2026006503A1 02/01/2026
Solicitante: 
VIRGINIA COMMONWEALTH UNIV [US]
VIRGINIA COMMONWEALTH UNIVERSITY

Resumen de: WO2026006503A1

NLRP3 PET radiotracers are provided, as are methods of using the NLRP3 PET radiotracers to image and/or diagnose diseases and conditions associated with inflammation, such as multiple sclerosis (MS), Alzheimer's disease (AD), traumatic brain injury (TBI), Parkinson's disease (PD), acute myocardial infarction (AMI), heart failure, gout, rheumatoid arthritis, COVID- 19, diabetes, macular degeneration, and autoimmune/autoinflammatory diseases.

INHIBITOR OF RHO GTPASE CDC42 FOR USE IN THE PREVENTION AND/OR TREATMENT OF PARKINSON'S DISEASE OF THE HUMAN OR ANIMAL BODY

NºPublicación:  WO2026002845A1 02/01/2026
Solicitante: 
UNIV ULM KOERPERSCHAFT DES OEFFENTLICHEN RECHTS [DE]
UNIVERSIT\u00C4T ULM, K\u00D6RPERSCHAFT DES \u00D6FFENTLICHEN RECHTS
DE_102024205840_PA

Resumen de: WO2026002845A1

The invention relates to an inhibitor of Rho GTPase Cdc42 for use in the prevention and/or treatment of Parkinson's disease of the human or animal body. In a mouse model in which the mice were genetically modified to accumulate α-synuclein and develop Parkinson's disease, it has been shown that the administration of a Cdc42 inhibitor in the diseased mice reduces the accumulation of α-synuclein, improves the symptoms of Parkinson's disease, increases the average life expectancy, and prolongs the total life span. Only minimal to no side effects occurred in the diseased mice.

TAU-TARGETING RNA INTERFERENCE METHOD, NUCLEIC ACID AND APPLICATION THEREOF

NºPublicación:  WO2026002277A1 02/01/2026
Solicitante: 
EXORNA BIOSCIENCE NANJING CO LTD [CN]
EXORNA BIOSCIENCE SHANGHAI CO LTD [CN]
\u827E\u7801\u751F\u7269\u79D1\u6280\uFF08\u5357\u4EAC\uFF09\u6709\u9650\u516C\u53F8,
\u827E\u7801\u7533\u534E\u751F\u7269\u79D1\u6280\uFF08\u4E0A\u6D77\uFF09\u6709\u9650\u516C\u53F8

Resumen de: WO2026002277A1

The present invention provides a nucleic acid molecule used for regulating the level or amount of Tau mRNA. Specifically, the present invention provides the delivery of a primary microRNA to form a precursor after in vivo processing, and a microRNA used for the in vivo inhibition of the expression of Tau mRNA. The present invention also provides a delivery system for the nucleic acid molecule, comprising a vector, an exosome and a cell, and a pharmaceutical composition containing same. The present invention also provides an application of the nucleic acid molecule and the delivery system in neurodegenerative disease treatment and drug preparation, in particular a method and a drug targeting Alzheimer's disease.

OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF

NºPublicación:  WO2026006477A1 02/01/2026
Solicitante: 
WAVE LIFE SCIENCES LTD [SG]
LAKE STEPHEN LISTER [US]
HU XIAO SHELLEY [US]
GAO JIANXIN [US]
ZHANG YILIN [US]
ROSS PHILIP [US]
ADUDA VINCENT [US]
SHIMIZU MAMORU [US]
XU DANLIN [US]
BOWMAN KEITH ANDREW [US]
LAMORE SARAH DIANE [US]
PANZARA MICHAEL ANGELO [US]
ALAM MOHAMMED ROWSHON [US]
JANG HYUN GYUNG [US]
BANDARA ASELA KALYANAPRIYA [GB]
HAEGELE JOSEPH ANDREW [US]
HURTT MARK RICHARD [US]
GOEL VARUN [US]
NARAYANAN PADMAKUMAR [US]
LI KWAI CHEUNG ANNE MARIE [GB]
CARACTA CYNTHIA FAJARDO [US]
TROMBINO ANTHONY [US]
XIE LANYI [US]
WAVE LIFE SCIENCES LTD,
LAKE, Stephen Lister,
HU, Xiao Shelley,
GAO, Jianxin,
ZHANG, Yilin,
ROSS, Philip,
ADUDA, Vincent,
SHIMIZU, Mamoru,
XU, Danlin,
BOWMAN, Keith Andrew,
LAMORE, Sarah Diane,
PANZARA, Michael Angelo,
ALAM, Mohammed Rowshon,
JANG, Hyun Gyung,
BANDARA, Asela Kalyanapriya,
HAEGELE, Joseph Andrew,
HURTT, Mark Richard,
GOEL, Varun,
NARAYANAN, Padmakumar,
LI-KWAI-CHEUNG, Anne-Marie,
CARACTA, Cynthia Fajardo,
TROMBINO, Anthony,
XIE, Lanyi

Resumen de: WO2026006477A1

Among other tilings, the present disclosure provides various technologies including chirally controlled oligonucleotide compositions and technologies for manufacturing and using such oligonucleotide compositions. In some embodiments, the present disclosure provides technologies useful for allele-specific knockdown of mutant Huntingtin transcripts. In some embodiments, the present disclosure provides technologies usefill for reducing the expression, level, amount, and/or activity of mutant Huntingtin transcripts or products thereof. In some embodiments, the present disclosure provides methods for treating Huntington's disease.

SULFOPROPANOIC ACID DERIVATIVES FOR TREATING NEURODEGENERATIVE DISORDERS

NºPublicación:  EP4671757A2 31/12/2025
Solicitante: 
ALZHEON INC [US]
Alzheon, Inc
CN_121045036_PA

Resumen de: EP4671757A2

Provided herein is the use of a compound of Formula I:or a pharmaceutically acceptable salt thereof, for treating a disease characterized by amyloid and amyloid-like aggregates, e.g., Alzheimer's disease.

COMPOSITION COMPRISING HAPLN1 AS ACTIVE INGREDIENT OR PREVENTING OR TREATING SENILE DEGENERATIVE BRAIN DISEASES

NºPublicación:  WO2025263948A1 26/12/2025
Solicitante: 
CHUNG ANG UNIV INDUSTRY ACADEMIC COOPERATION FOUNDATION [KR]
\uC911\uC559\uB300\uD559\uAD50 \uC0B0\uD559\uD611\uB825\uB2E8

Resumen de: WO2025263948A1

The present invention relates to a composition comprising HAPLN1 as an active ingredient for preventing or treating senile degenerative brain diseases. Specifically, recombinant human HAPLN1 protein (rhHAPLN1) lowers the protein level of p16 in cultured human astrocytes to inhibit cellular senescence caused by the accumulation of beta amyloid peptides, and further inhibits phosphorylation (p-p38 MAPK) of p38 MAPK protein, thereby also having the possibility of inhibiting inflammatory responses associated with the onset of Alzheimer's disease and Parkinson's disease. In addition, the recombinant human HAPLN1 protein (rhHAPLN1) exhibits significant memory and learning improvement effects in in vivo experiments performed using a mouse acute Alzheimer's disease model, and thus can be expected to exhibit preventive and therapeutic effects against Alzheimer's disease that may occur with aging and the like. In addition, the inhibitory effect of the rhHAPLN1 protein on cellular senescence and inflammatory responses of astrocytes can provide a very important clue for establishing prevention and treatment strategies not only for aging itself but also for brain functions, motor behaviors, memory, seizures, dementia, brain tumors, and the like.

AGENTS AND/OR COMPOSITIONS USEFUL FOR MODULATING CIS-REGULATORY ELEMENTS IN SYNUCLEINOPATHIES AND METHODS FOR IDENTIFYING AGENTS AND COMPOSITIONS THEREOF

NºPublicación:  WO2025264967A1 26/12/2025
Solicitante: 
THE JOHNS HOPKINS UNIV [US]
THE JOHNS HOPKINS UNIVERSITY

Resumen de: WO2025264967A1

The present disclosure relates to methods of preventing, or delaying the progression of, death of neurons and/or microgliosis and/or astrogliosis that contributes to the death of neurons. The present disclosure also relates to methods of treating, preventing, or delaying the progression of, a synucleinopathy (e.g., Parkinson's disease). Also disclosed are related in vitro, ex vivo, and in vivo methods of identifying agents and/or compositions useful for preventing, or delaying the progression of, death of neurons and/or microgliosis and/or astrogliosis that contributes to the death of neurons and agents and/or compositions useful for treating, preventing, or delaying the progression of, a synucleinopathy. The agents and/or compositions of the present disclosure decrease the level and/or activity of a cis-regulatory element that propagates the misfolding and aggregation of proteins encoded by synucleinopathy-associated genes in neurons and/or glial cells.

CRYSTALLINE FORMS OF 3-(5-(4-((3,3-DIMETHYL-4-((1-(6-(5-(1-METHYLCYCLOPROPOXY)-1HINDAZOL-3-YL)PYRIMIDIN-4-YL)PIPERIDIN-4-YL)METHYL)PIPERAZIN-1-YL)METHYL)PIPERIDIN-1-YL)-4-FLUORO-1-OXOISOINDOLIN-2-YL)PIPERIDINE-2,6-DIONE

NºPublicación:  WO2025265035A1 26/12/2025
Solicitante: 
ARVINAS OPERATIONS INC [US]
ARVINAS OPERATIONS, INC

Resumen de: WO2025265035A1

Provided are crystalline forms of 3-(5-(4-((3,3-dimethyl-4-((1-(6-(5-(1- methylcyclopropoxy)-1H-indazol-3-yl)pyrimidin-4-yl)piperidin-4-yl)methyl)piperazin-1- yl)methyl)piperidin-1-yl)-4-fluoro-1-oxoisoindolin-2-yl)piperidine-2, 6-dione, pharmaceutical compositions comprising the crystalline form, and their use in treating conditions associated with LRRK2, such as Parkinson's disease, dementia, Progressive Supranuclear Palsy, Huntington's disease, or neuroinflammation.

APP-TARGETING RNA INTERFERENCE METHOD, NUCLEIC ACID, AND USE THEREOF

NºPublicación:  WO2025261396A1 26/12/2025
Solicitante: 
EXORNA BIOSCIENCE NANJING CO LTD [CN]
EXORNA BIOSCIENCE SHANGHAI CO LTD [CN]
\u827E\u7801\u751F\u7269\u79D1\u6280\uFF08\u5357\u4EAC\uFF09\u6709\u9650\u516C\u53F8,
\u827E\u7801\u7533\u534E\u751F\u7269\u79D1\u6280\uFF08\u4E0A\u6D77\uFF09\u6709\u9650\u516C\u53F8

Resumen de: WO2025261396A1

Provided is a nucleic acid molecule used for regulating the level or amount of APP mRNA. Specifically, provided is delivery of primary microRNA for formation of pre- and microRNA following in-vivo processing and for use in the in-vivo inhibition of APP mRNA expression. Provided is a delivery system for the nucleic acid molecule, the delivery system comprising a carrier, an exosome, and a cell, and a pharmaceutical composition containing same. Provided is the use of the nucleic acid molecule and the delivery system in amyloidosis treatment and drug preparation, in particular a method and a drug for Alzheimer's disease.

COMPOSITIONS OF BI-FUNCTIONAL ALPHA HELICAL PEPTIDES AND METHODS THEREOF FOR TREATING TDP-43 PROTEINOPATHIES

NºPublicación:  WO2025264861A1 26/12/2025
Solicitante: 
YALE UNIV [US]
YALE UNIVERSITY

Resumen de: WO2025264861A1

Compositions and methods for disrupting pathological aggregation and/or mis-localization of TDP-43 in the brain/CNS have been developed. Compositions including engineered helical polypeptides that bind TDP-43's amyloidogenic core but resist β-sheet conversion are provided. In some forms, the engineered polypeptides include peptide degradation motifs (PDM) to enhance proteolytic degradation of aggregates, and/or targeting motifs to direct the peptides to the brain/CNS. Recombinant constructs including nucleic acids expressing or encoding the polypeptides are also provided. Methods of using the engineered peptides to treat or prevent one or more diseases or disorders associated with pathological aggregation and/or mis-localization of TDP-43 in the brain/CNS are also provided. In some forms, the methods treat or prevent ALS or FTD in a subject in need thereof.

USE OF ANAVEX3-71 FOR MEDICAL TREATMENTS

NºPublicación:  WO2025264845A1 26/12/2025
Solicitante: 
ANAVEX LIFE SCIENCES CORP [US]
MISSLING CHRISTOPHER U [US]
SELVEY ALANI [US]
ANAVEX LIFE SCIENCES CORP,
MISSLING, Christopher U,
SELVEY, Alani

Resumen de: WO2025264845A1

The present disclosure relates to the novel use of ANAVEX3-71 and related compounds in medical treatments, such as Parkinson's Disease, Frontotemporal Dementia, Schizophrenia, and Alzheimer's Disease.

TREATMENT OF DISEASE

NºPublicación:  WO2025264823A1 26/12/2025
Solicitante: 
METASHAPE PHARMA AG [CH]
MEHRLING THOMAS [CH]
BANTIA SHANTA [CH]
METASHAPE PHARMA AG,
MEHRLING, Thomas,
BANTIA, Shanta

Resumen de: WO2025264823A1

This disclosure relates to the use of Purine Nucleoside Phosphorylase (PNP) inhibitors such as ulodesine and its salts, in the treatment and/or prevention of diseases associated with nicotinamide adenine dinucleotide (NAD+) depletion, including diseases of mitochondrial dysfunction (including neurodegeneration and peripheral neuropathies), the preservation of cognitive function and in muscular disorders such as sarcopenia; and in metabolic syndrome and associated conditions. In particular the disclosure provides the use of PNP inhibitors such as ulodesine in the treatment of neurodegenerative conditions such as Parkinson's disease and amyotrophic lateral sclerosis.

TREATMENT OF HUNTINGTON'S DISEASE

NºPublicación:  WO2025264449A1 26/12/2025
Solicitante: 
PTC THERAPEUTICS INC [US]
BEERS BRIAN MICHAEL [US]
BHATTACHARYYA ANURADHA [US]
PTC THERAPEUTICS, INC,
BEERS, Brian Michael,
BHATTACHARYYA, Anuradha

Resumen de: WO2025264449A1

The present invention describes methods of treating Huntington's disease in a subject in need thereof. The method comprising administrating to the subject in need thereof a therapeutically effective amount of 2-3-(2,2,6,6-tetramethylpiperidin-4-yl)-3H-1,2,3triazolo4,5-cpyridazin-6-yl-5-(2H-1,2,3-triazol-2-yl)phenol or a pharmaceutically acceptable salt thereof.

(AZA)SPIROHEPTANE DERIVATIVES FOR THE TREATMENT OF NEURODEGENERATIVE DISORDERS

NºPublicación:  US2025388592A1 25/12/2025
Solicitante: 
REMYND N V [BE]
REMYND N.V
JP_2025527459_PA

Resumen de: US2025388592A1

The application relates to compounds of formula (B1A), (B1B), (B1C) or a salt, a solvate, a hydrate, a polymorph, a tautomer, a racemate or a stereoisomer thereof, to pharmaceutical compositions comprising such a compound, and to the compounds for use as a medicine, in particular for use in the treatment of neurodegenerative disorders such as Alzheimer's disease.

ANTI-SOD1 NANOBODIES

NºPublicación:  US2025388697A1 25/12/2025
Solicitante: 
UNIV OF MASSACHUSETTS [US]
University of Massachusetts
WO_2023250016_A2

Resumen de: US2025388697A1

Composition and methods of diagnosing, monitoring, and treating subjects with a motor neuron pathology, such as motor neuron disorders (including but not limited to amyotrophic lateral sclerosis (ALS)) and neuropathies.

COMPOSITIONS AND METHODS FOR THE TREATMENT OF PARKINSON S AND GAUCHER DISEASE

NºPublicación:  EP4667461A2 24/12/2025
Solicitante: 
CHILDRENS HOSPITAL MED CT [US]
Children's Hospital Medical Center
EP_4667461_A2

Resumen de: EP4667461A2

Compounds and pharmaceutical compositions are useful for the treatment of Gaucher disease and Parkinson's disease. Methods of treating Gaucher disease or Parkinson's disease include administration of one or more compounds or pharmaceutically acceptable salts or prodrugs thereof to a subject diagnosed with, or at risk of developing Gaucher disease or Parkinson's disease.

SMALL MOLECULE INHIBITORS OF DYRK/CLK AND USES THEREOF

NºPublicación:  US2025382301A1 18/12/2025
Solicitante: 
ARIZONA BOARD OF REGENTS ON BEHALF OF THE UNIV OF ARIZONA [US]
UNIV OF DUNDEE [GB]
Arizona Board of Regents on Behalf of the University of Arizona,
University of Dundee
US_2025382301_A1

Resumen de: US2025382301A1

This invention is in the field of medicinal chemistry. In particular, the invention relates to a new class of small-molecule compounds having a 6,6-heterocyclic structure (e.g., compounds having a naphthyridine, pyrido-pyridazine, pyrido-pyrazine, quinoline, pyrazino-pyridazine, pyrimido-pyrimidine, quinazoline, quinoxaline or cinnoline ring system) which function as inhibitors of DYRK1A, DYRK1B, DYRK2, DYRK3, CLK1, CLK2, CLK3, CLK4, CDK7, CDK8/19, PI3K, PDGFrA/B, mTOR, WNT, homeodomain-interacting kinases (HIPKs), and/or CMGC kinases leading to inhibition of WNT signaling, and their use as therapeutics for the treatment of Alzheimer's disease, down syndrome, Parkinson's disease, Huntington's disease, diabetes, autoimmune diseases, inflammatory disorders (e.g., airway inflammation, osteoarthritis (e.g., knee related osteoarthritis)), cancer (e.g., glioblastoma, prostate cancer, metastatic breast cancer, metastatic lung cancer, multiple myeloma, secondary metastatic tumors of the brain, colorectal cancer and metastatic colorectal cancer (e.g., metastatic colorectal cancer in the liver)), and other diseases.

THERAPY OF CNS DISORDERS

NºPublicación:  WO2025259709A1 18/12/2025
Solicitante: 
THE REGENTS OF THE UNIV OF CALIFORNIA [US]
THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
WO_2025259709_A1

Resumen de: WO2025259709A1

The present disclosure provides methods of screening for, identifying and using a Gi-GPCR agonist for a CNS disorder. The CNS disorder can be any disorder in which astrocyte morphology and/or astrocyte tissue support are altered or compromised (e.g., OCD, Alzheimer's disease, or Huntington's disease). Provided herein are methods of screening for and identifying Gi-GPCR agonist ex vivo based on assessment of astrocyte morphology and/or Gi-GPCR activation (e.g., wherein the Gi-GPCR is GPR3711, S1PR1, EDNRB, GRM3, or AD0RA2A). Also provided herein are methods of identifying a therapeutic agent for the treatment of a CNS disorder in vivo at least in part based on its effect on astrocyte morphology and/or Gi-GPCR activation. Also provided herein are methods for the treatment or prevention of a CNS disorder comprising administering to a subject a Gi-GPCR (e.g., GPR3711, S1PR1, EDNRB, GRM3, or AD0RA2A) agonist.

COMPOSITIONS AND METHODS FOR THE TREATMENT OF PARKINSON'S AND GAUCHER DISEASE

NºPublicación:  US2025382274A1 18/12/2025
Solicitante: 
CHILDRENS HOSPITAL MEDICAL CENTER [US]
CHILDREN'S HOSPITAL MEDICAL CENTER
US_2025382274_A1

Resumen de: US2025382274A1

Compounds and pharmaceutical compositions are useful for the treatment of Gaucher disease and Parkinson's disease. Methods of treating Gaucher disease or Parkinson's disease include administration of one or more compounds or pharmaceutically acceptable salts or prodrugs thereof to a subject diagnosed with, or at risk of developing Gaucher disease or Parkinson's disease.

METHODS AND COMPOSITIONS FOR TREATMENT OF NEURODEGENERATIVE DISORDERS AND REDUCING TAU PROTEIN AGGREGATES

NºPublicación:  US2025381230A1 18/12/2025
Solicitante: 
SANBIO CO LTD [JP]
KEIO UNIV [JP]
SanBio Co. Ltd,
Keio University
US_2025381230_PA

Resumen de: US2025381230A1

Disclosed are compositions and methods for treating Alzheimer's disease or for use in treating Alzheimer's disease. Also disclosed are compositions and methods for treating or for use in treating a neurodegenerative disorder characterized by the presence of tau protein aggregates. Furthermore, disclosed are compositions and methods for reducing tau protein aggregates or for use in reducing tau protein aggregates.

Regimen for Treating Amyotrophic Lateral Sclerosis Having Onset 24 Months Prior to Treatment

NºPublicación:  US2025381197A1 18/12/2025
Solicitante: 
WOOLSEY PHARMACEUTICALS INC [US]
Woolsey Pharmaceuticals, Inc
US_2025381197_A1

Resumen de: US2025381197A1

The present invention relates to the treatment of an ALS patient having disease onset of at least 24 months prior to initiation of treatment with fasudil. Fasudil is administered at a dose of 60-240 mg/day according to specific treatment regimens. This results in an anticipated 25-50% reduction in the average decline over at least three months as measured using the revised ALS Functional Rating Scale.

METHOD FOR TREATING A PARKINSON'S DISEASE

NºPublicación:  US2025381305A1 18/12/2025
Solicitante: 
SINEUGENE THERAPEUTICS CO LTD [CN]
SINEUGENE THERAPEUTICS CO., LTD
US_2025381305_PA

Resumen de: US2025381305A1

Provided is a method for treating a Parkinson Disease, comprising a TRIM72 protein modulator. Further provided is a composition comprising the TRIM72 protein modulator and use thereof.

METHODS OF TREATMENT USING AN ANTI-ABETA PROTOFIBRIL ANTIBODY

NºPublicación:  AU2024286486A1 18/12/2025
Solicitante: 
EISAI R&D MANAGEMENT CO LTD
EISAI R&D MANAGEMENT CO., LTD
AU_2024286486_A1

Resumen de: AU2024286486A1

Disclosed herein are methods of selecting, monitoring, and treating subjects with Alzheimer's disease (AD) or suspected of having AD or another disorder associated with amyloid accumulation in the brain based on the risk of an ARIA event or brain hemorrhage. Also disclosed herein are methods of treating subjects having or suspected of having AD comprising subcutaneously administering an anti-Aβ protofibril antibody.

COMPOSITION COMPRISING GV1001 FOR PREVENTING OR TREATING PERIODONTAL DISEASES AND DISORDERS CAUSED BY PERIODONTAL DISEASES

NºPublicación:  AU2024285799A1 18/12/2025
Solicitante: 
GEMVAX & KAEL CO LTD
GEMVAX & KAEL CO., LTD
AU_2024285799_PA

Resumen de: AU2024285799A1

The present invention relates to a pharmaceutical composition for the prevention or treatment of periodontal disease, or atherosclerosis or Alzheimer's disease caused by periodontal disease. More specifically, the present invention relates to a pharmaceutical composition for the prevention or treatment of periodontal disease, or atherosclerosis or Alzheimer's disease caused by periodontal disease, the composition comprising a peptide with the amino acid sequence of SEQ ID NO: 1, which is effective in inhibiting osteoclast formation, suppressing the formation of Porphyromonas gingivalis colonies, and inhibiting gingipain expression, while being safe for the body and having fewer side effects, including adverse reactions.

METHODS AND COMPOSITIONS FOR TREATMENT OF NEURODEGENERATIVE DISORDERS AND REDUCING TAU PROTEIN AGGREGATES

Nº publicación: WO2025258415A1 18/12/2025

Solicitante:

SANBIO CO LTD [JP]
KEIO UNIV [JP]
SANBIO CO. LTD,
KEIO UNIVERSITY

WO_2025258415_A1

Resumen de: WO2025258415A1

Disclosed are compositions and methods for treating Alzheimer's disease or for use in treating Alzheimer's disease. Also disclosed are compositions and methods for treating or for use in treating a neurodegenerative disorder characterized by the presence of tau protein aggregates. Furthermore, disclosed are compositions and methods for reducing tau protein aggregates or for use in reducing tau protein aggregates.

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