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Solicitudes publicadas en los últimos 60 días / Last 60 days publications
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COMBINATION TREATMENT OF ALZHEIMER'S DISEASE

NºPublicación:  WO2025087971A1 01/05/2025
Solicitante: 
TREEWAY TW001 B V [NL]
TREEWAY TW001 B.V
WO_2025087971_A1

Resumen de: WO2025087971A1

The invention relates to the treatment of Alzheimer's disease in a human patient, said treatment comprising administration of an anti-Aβ antibody component and co-administration of edaravone, the anti-Aβ antibody component being selected from anti-Aβ antibody, an Aβ- binding fragment of an Aβ antibody, a vectorised anti-Aβ antibody and a vectorised Aβ- binding fragment of an Aβ antibody.

HEXAHYDRO-2H-PYRIDO2,1-AISOQUINOLINE VMAT2 INHIBITORS AND METHODS OF USE

NºPublicación:  AU2023347329A1 01/05/2025
Solicitante: 
NEUROCRINE BIOSCIENCES INC
NEUROCRINE BIOSCIENCES, INC
AU_2023347329_A1

Resumen de: AU2023347329A1

This disclosure relates to, inter alia, certain compounds, compositions, and pharmaceutical compositions thereof, that modulate the activity of the transporter protein vesicular monoamine transporter-2 (VMAT2) and are directed to methods useful in the treatment of transporter protein vesicular monoamine transporter-2 mediated disorders, such as, neurological or psychiatric disease or disorders, including but not limited to, hyperkinetic movement disorders (e.g., tardive dyskinesia, Tourette's syndrome, Huntington's disease, tics, ataxia, chorea (such as, chorea associated with Huntington's disease), dystonia, hemifacial spasm, myoclonus, restless leg syndrome, and tremors). The disclosure further relates to synthetic methods and intermediates useful in the preparation of the compounds.

METHODS OF TREATMENT USING A TAU PET LEVEL

NºPublicación:  AU2023406056A1 01/05/2025
Solicitante: 
EISAI R&D MANAGEMENT CO LTD
EISAI R&D MANAGEMENT CO., LTD
AU_2023406056_A1

Resumen de: AU2023406056A1

Disclosed herein are methods of diagnosing, 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 using a tau PET level.

MEDICAL AGENT FOR TREATING OR SUPPRESSING PROGRESSION OF AMYOTROPHIC LATERAL SCLEROSIS

NºPublicación:  US2025134863A1 01/05/2025
Solicitante: 
MITSUBISHI TANABE PHARMA CORP [JP]
MITSUBISHI TANABE PHARMA CORPORATION
US_2025134863_A1

Resumen de: US2025134863A1

Disclosed is a medical agent for treating or suppressing progression of at least one symptom selected from a group consisting of amyotrophic lateral sclerosis and symptoms resulting from amyotrophic lateral sclerosis in a subject. The medical agent contains 3-methyl-1-phenyl-2-pyrazolin-5-one or a physiologically acceptable salt thereof, or a hydrate or solvate thereof. A blood uric acid level of the subject before administration of the medical agent is 4.2 mg/dL or higher.

CRISPR-MEDIATED MANIPULATION OF APP EXPRESSION AS A THERAPEUTIC APPROACH FOR AMYLOID PATHOLOGIES

NºPublicación:  US2025135034A1 01/05/2025
Solicitante: 
THE HONG KONG UNIV OF SCIENCE & TECHNOLOGY [CN]
HONG KONG CENTER FOR NEURODEGENERATIVE DISEASES LTD [CN]
The Hong Kong University of Science & Technology,
HONG KONG CENTER FOR NEURODEGENERATIVE DISEASES LIMITED
US_2025135034_PA

Resumen de: US2025135034A1

Compositions and methods are provided for the treatment or prophylaxis of amyloid pathologies such as Alzheimer's Disease.

TREATMENT FOR SOD1 ASSOCIATED DISEASE

NºPublicación:  US2025136993A1 01/05/2025
Solicitante: 
BLACK SWAN PHARMACEUTICALS INC [US]
Black Swan Pharmaceuticals, Inc
US_2025136993_PA

Resumen de: US2025136993A1

The present invention relates to antisense oligonucleotides that are complimentary to SOD1, leading to decreased expression of SOD1. Reduced expression of SOD1 is beneficial in medical disorders such as Amyotrophic Lateral Sclerosis.

COMPOSITIONS FOR PREVENTING AND TREATING NEURODEGENERATIVE DISEASES

NºPublicación:  US2025134894A1 01/05/2025
Solicitante: 
ARIBIO CO LTD [KR]
ARIBIO CO., LTD
US_2025134894_A1

Resumen de: US2025134894A1

The present invention provides a composition for preventing or treating a neurodegenerative disease containing a phosphodiesterase 5 inhibitor (PDE5 inhibitor) and an N-methyl-D-aspartate-receptor (NMDA-receptor) antagonist and a method using thereof, wherein the PDE5 inhibitor is mirodenafil, sildenafil, vardenafil, tadalafil, udenafil, dasantafil, avanafil, pharmaceutically acceptable salts, solvates, hydrates, or a mixture thereof; and the NMDA-receptor antagonist is selected from among memantine, amantadine, ketamine, traxoprodil, lanicemine, rislenemdaz, pethidine, levorphanol, methadone, dextropropoxyphene, tramadol, ketobemidone, dextromethorphan (DXM), phencyclidine (PCP), and methoxetamine (MXE), pharmaceutically acceptable salts, solvates, hydrates and a mixture thereof; and the neurodegenerative disease is dementia, Parkinson's disease (PD), Dementia with Lewy body (DLB), Alzheimer's disease (AD), Huntington's disease (HD), Multiple sclerosis (MS), Vascular Dementia (VaD), Amyotrophic Lateral Sclerosis (ALS), frontotemporal dementia, or a mixed etiologies thereof.

ANTI-TAU THERAPEUTIC ANTIBODY FOR AD AND TAUOPATHIES

NºPublicación:  WO2025085971A1 01/05/2025
Solicitante: 
THE UNIV OF QUEENSLAND [AU]
THE UNIVERSITY OF QUEENSLAND
WO_2025085971_A1

Resumen de: WO2025085971A1

The present disclosure provides human Tau protein (Tau)-binding proteins comprising an antigen binding domain of an antibody, wherein the antigen binding domain binds specifically to an epitope of Tau that results in inhibition of Tau aggregate seeding, and could be used to inhibit spreading of Tau aggregates in vivo. Also disclosed herein are nucleic acids, expression vectors, and recombinant viruses encoding such Tau-binding proteins, as well as compositions comprising such Tau-binding proteins. Also disclosed are methods for use of such compositions in the diagnosis, prophylaxis, treatment, and prognosis of Tauopathies such as Alzheimer's Disease, frontotemporal dementia, Pick's disease, and progressive supranuclear palsy. Genetically modified cells for expression for the disclosed Tau-binding proteins, and their use for production of the Tau-binding proteins are also disclosed.

TARGETING ALS-FUS AGGREGATION WITH PROTEASOME INHIBITORS

NºPublicación:  WO2025088613A1 01/05/2025
Solicitante: 
TECHNION RES & DEVELOPMENT FOUNDATION LIMITED [IL]
TECHNION RESEARCH & DEVELOPMENT FOUNDATION LIMITED
WO_2025088613_A1

Resumen de: WO2025088613A1

Provided are methods of treating a subject having a disease characterized by accumulation of FUS -associated aggregates by administering to the subject a therapeutically effective amount of a proteasome inhibitor, an agent which upregulates SAT1, or an agent which upregulates spermine, thereby treating the subject. Also provided are proteasome inhibitors, an agent which upregulates SAT1, or an agent which upregulates spermine for use in treating a subject having a disease characterized by accumulation of FUS-associated aggregates, and methods of reducing accumulation of FUS-associated aggregates in cells of a subject, the method comprising contacting the cells of the subject with a proteasome inhibitor an agent which upregulates SAT1, or an agent which upregulates spermine, thereby reducing the accumulation of FUS-associated aggregates in the cells of the subject.

METHOD OF TREATING PRECLINICIAL ALZHEIMER'S DISEASE

NºPublicación:  WO2025090815A1 01/05/2025
Solicitante: 
JANSSEN PHARMACEUTICALS INC [US]
AC IMMUNE SA [CH]
JANSSEN PHARMACEUTICALS, INC,
AC IMMUNE SA
WO_2025090815_PA

Resumen de: WO2025090815A1

The application describes a phosphorylated tau targeted active Immunotherapy to treat preclinical Alzheimer's Disease.

LOW DOSE siRNA TREATMENTS OF HUNTINGTON'S DISEASE

NºPublicación:  WO2025090567A1 01/05/2025
Solicitante: 
OPHIDION INC [US]
OPHIDION INC
WO_2025090567_A1

Resumen de: WO2025090567A1

A composition for treating Huntington's disease (HD) by reducing endogenous Huntingtin (HTT) levels in a target includes a siRNA including a siRNA sequence selected from SEQ ID NOs:47 to 149, and a carrier peptide comprising a peptide sequence of SEQ ID NO:1. The carrier peptide may target the nicotinic acetylcholine receptor (nAChR) of neuronal cells in order to deliver the siRNA across the blood-brain barrier (BBB). In some embodiments, the siRNA may be conjugated with the carrier peptide. A method of reducing endogenous HTT levels to treat HD in the target may include administering a composition including a siRNA sequence selected from SEQ ID NOs: 47 to 149 to the target.

COMPOSITIONS AND METHODS FOR NEUROPROTECTION

NºPublicación:  WO2025090442A1 01/05/2025
Solicitante: 
RES DEVELOPMENT FOUNDATION [US]
RESEARCH DEVELOPMENT FOUNDATION
WO_2025090442_A1

Resumen de: WO2025090442A1

Methods and compositions for promoting neuroprotection and/or reducing neuronal death are provided. In some aspects, gene therapies to overexpress yin-yang 1 (YY1) are provided and can be used to decrease death of dopaminergic neurons during a neurodegenerative disease, such as Parkinson's disease, or after trauma.

SUSTAINED-RELEASE MICROPARTICLES CONTAINING DRUG AND PAMOIC ACID

NºPublicación:  EP4545071A1 30/04/2025
Solicitante: 
G2GBIO INC [KR]
G2GBIO, Inc
EP_4545071_PA

Resumen de: EP4545071A1

A sustained-release microsphere containing a drug, pamoic acid, and a biocompatible polymer, a pharmaceutical composition containing the sustained-release microsphere for prevention, improvement, or treatment of hepatitis B, prostate cancer, breast cancer, endometriosis, uterine fibroid, precocious puberty, acromegaly, gastro·entero·pancreatic endocrine tumor, Alzheimer's disease, or cognitive disorder, and a preparing method of the sustained-release microsphere are provided.

HMGB1 INHIBITORS FOR TREATMENT OF APOE4-RELATED TAUOPATHIES INCLUDING ALZHEIMER'S DISEASE

NºPublicación:  EP4543459A1 30/04/2025
Solicitante: 
THE J DAVID GLADSTONE INST A TESTAMENTARY TRUST ESTABLISHED UNDER THE WILL OF J DAVID GLADSTONE [US]
THE REGENTS OF UNIV OF CALIFORNIA [US]
The J. David Gladstone Institutes, A Testamentary Trust Established under The Will of J. David Gladstone,
The Regents of University of California
CN_119630405_A

Resumen de: WO2023250249A1

As described herein, inhibitors of High mobility group box protein 1 (HMGB1) can significantly reduce HMGB1 nucleo-cytoplasmic translocation, gliosis, neurodegeneration, Tau pathologies, and myelin deficits, especially in subjects having an AP0E4 allele. Methods are therefore described herein that include administering one or more inhibitors of High mobility group box protein 1 (HMGB1) to a subject having at least one genomic AP0E4 allele.

METHOD OF TREATING PRECLINICIAL ALZHEIMER'S DISEASE

NºPublicación:  US2025127867A1 24/04/2025
Solicitante: 
JANSSEN PHARMACEUTICALS INC [US]
AC IMMUNE SA [CH]
JANSSEN PHARMACEUTICALS, INC,
AC IMMUNE SA

Resumen de: US2025127867A1

The application describes a phosphorylated tau targeted active immunotherapy to treat preclinical Alzheimer's Disease.

COMPOSITIONS AND METHODS USING REELIN IN ALZHEIMER'S DISEASE

NºPublicación:  AU2023358527A1 24/04/2025
Solicitante: 
THE GENERAL HOSPITAL CORP
MASSACHUSETTS EYE AND EAR INFIRMARY
BANNER HEALTH
THE GENERAL HOSPITAL CORPORATION,
MASSACHUSETTS EYE AND EAR INFIRMARY,
BANNER HEALTH
AU_2023358527_A1

Resumen de: AU2023358527A1

Described herein are methods and compositions for treating Alzheimer's Disease (AD), as well as compositions comprising a reelin-derived peptide and methods of use thereof.

APOE GENE THERAPY

NºPublicación:  AU2023360721A1 24/04/2025
Solicitante: 
SPARK THERAPEUTICS INC
SPARK THERAPEUTICS, INC
AU_2023360721_PA

Resumen de: AU2023360721A1

The present relates to polynucleotide constructs encoding an ApoE3 related protein optionally containing one or more intron. Potential uses of the different constructs include gene therapy targeting one or more disease or disorder, for example, diseases or disorders related to cholesterol levels, atherosclerosis, coronary heart disease, dementia, cerebral amyloid angiopathy, or Alzheimer's disease.

USE OF FACTOR H FOR THE TREATMENT OF DEMENTIA

NºPublicación:  WO2025083211A1 24/04/2025
Solicitante: 
INSTITUT NATIONAL DE LA SANTE ET DE LA RECH MEDICALE [FR]
UNIV DE BORDEAUX [FR]
INSTITUT NATIONAL DE LA SANT\u00C9 ET DE LA RECHERCHE M\u00C9DICALE,
UNIVERSIT\u00C9 DE BORDEAUX
WO_2025083211_A1

Resumen de: WO2025083211A1

The inventors hypothesized that inhibition of complement activation could reduce the inflammatory period observed even before clinical signs of Alzheimer's disease and thus slow down the onset and progression of AD. In order to validate the hypothesis, the inventors injected Factor H (FH: the main inhibitor of complement activation) into the brain of APP/PS1 AD- mice model at early or late stage of this pathology. The results showed effects of FH brain injection on the AD-onset as well as progression by reducing pro-inflammatory IL6, TNF-α, Il1β, MAC and Aβ levels associated with an increase of VGLUT1 and Psd95 neurotransmitters levels in hippocampal region leading to improvement of cognitive functions even at late stage of the pathology. The results thus prompt the inventors to consider that FH would be suitable for the treatment of dementia, and more particularly for the curative treatment of dementia.

METHOD OF INDUCING DENDRITIC AND SYNAPTIC GENESIS IN NEURODEGENERATIVE CHRONIC DISEASES

NºPublicación:  US2025127775A1 24/04/2025
Solicitante: 
PETCAVICH ROBERT JOHN [US]
Petcavich Robert John
CA_3148256_PA

Resumen de: US2025127775A1

The present invention discloses a method to recover and restore dendritic and synaptic neuron connections that have been degraded or destroyed by neurodegenerative diseases. In the present invention tryptamines are used to induce neuro plasticity and restore both dendritic density and synaptic connections of neurons in the brain. In the preferred embodiment LSD given in micro doses can induce dendritic and synaptic genesis in neuronal networks and improve the quality of life of people with neurodegenerative diseases such as Alzheimer's, Huntington's, Multiple Sclerosis, Parkinson's and Frontotemporal dementia.

COMPOSITION FOR DELAYING OR TREATING HUNTINGTON'S DISEASE COMPRISING LIN28A INHIBITOR AS ACTIVE INGREDIENT

NºPublicación:  WO2025084727A1 24/04/2025
Solicitante: 
KOREA UNIV RESEARCH AND BUSINESS FOUNDATION [KR]
\uACE0\uB824\uB300\uD559\uAD50 \uC0B0\uD559\uD611\uB825\uB2E8
WO_2025084727_PA

Resumen de: WO2025084727A1

The present invention relates to a composition for delaying or treating Huntington's disease, comprising a Lin28A inhibitor as an active ingredient. The present invention relates to various inhibitors capable of having the effect of preventing or treating Huntington's disease by inhibiting the expression of Lin28A, which is highly secreted specifically in Huntington's disease, and a screening method therefor.

BRAIN-CELL SPECIFIC PARTIAL CELLULAR REPROGRAMMING TREATMENT AND PREVENTION METHODS FOR ALZHEIMER'S DISEASE AND PROGERIA, COMPOSITIONS THEREFORE, AND USES THEREOF

NºPublicación:  WO2025085704A1 24/04/2025
Solicitante: 
YOUTHBIO THERAPEUTICS INC [US]
YOUTHBIO THERAPEUTICS INC
WO_2025085704_PA

Resumen de: WO2025085704A1

This disclosure relates to vectors, compositions, pharmaceutical compositions, and kits that provide for brain cell-specific expression of reprogramming genes such as the Yamanaka factors Oct4, Sox2, Klf4 and c-Myc (OSKM). Also provided are methods and uses comprising the same for treating Alzheimer' s disease and progeria through brain cell-specific expression of reprogramming genes such as OSKM.

INHIBITORS OF RAB-RILPL INTERACTIONS AND USES THEREOF

NºPublicación:  WO2025085624A1 24/04/2025
Solicitante: 
UNIV OF GEORGIA RESEARCH FOUNDATION INC [US]
UNIVERSITY OF GEORGIA RESEARCH FOUNDATION, INC
WO_2025085624_PA

Resumen de: WO2025085624A1

The present disclosure provides synthetic polypeptides that mimic the Switch 2 domain of a Rab protein. The synthetic polypeptides described are capable of inhibiting, modulating, or decreasing the interaction between a Rab protein and an RILPL protein. Methods of treating neurological diseases, disorders, or conditions, such as Parkinson's disease, are also provided.

COMPOUND BINDING TO CRBN PROTEIN AND DEGRADATION AGENT OF PROTEIN

NºPublicación:  WO2025082069A1 24/04/2025
Solicitante: 
OCEAN UNIV OF CHINA [CN]
\u4E2D\u56FD\u6D77\u6D0B\u5927\u5B66
WO_2025082069_A1

Resumen de: WO2025082069A1

The present invention belongs to the technical field of medicinal chemistry, specifically relates to a compound capable of binding to a CRBN protein and a composition thereof, and also relates to a protein degradation agent based on a CRBN protein and the use thereof. Specifically disclosed is a compound capable of binding to a CRBN protein, wherein the compound is composed of two sub-structure units A and B, the two sub-structures A and B are connected by means of a chemical single bond between Y of the sub-structure A and one of C2 (carbon at position 2), C4 (carbon at position 4), C5 (carbon at position 5), C6 (carbon at position 6) and C7 (carbon at position 7) of the sub-structure B, and the compound is as shown in formula I. The compound can be used as a structural unit of an E3 ubiquitin ligase ligand in PROTAC technology, thereby providing more therapeutic means for cancers or diseases such as prostate cancer, breast cancer, non-small cell lung cancer, Alzheimer's disease, neuroblastoma, hepatocellular carcinoma, colorectal cancer, pancreatic cancer, malignant rhabdomyosarcoma, and oral squamous cell carcinoma.

APPLICATION OF BMSC-EXOS IN TREATING PD

NºPublicación:  US2025127817A1 24/04/2025
Solicitante: 
NANTONG UNIV [CN]
NANTONG UNIVERSITY
JP_2024514740_PA

Resumen de: US2025127817A1

An application of Bone Marrow Mesenchyml Stem Cell Exosomes (BMSC-Exos) in treating Parkinson's disease (PD) is provided, wherein the BMSC-Exos are generated by stimulating BMSCs with a culture solution and extracted from the culture solution after passage; and the culture solution of the BMSCs is an α-MEM culture solution containing FBS and PS. The BMSC-Exos can greatly improve a motor function of a model mouse with PD, protect dopaminergic neurons of the model mouse with PD, improve an olfactory function of the model mouse with PD, and also inhibit the activation of olfactory astrocytes of the model mouse with PD.

CAR-TREG-BASED THERAPIES FOR TREATING NEURODEGENERATIVE DISEASES

Nº publicación: US2025129158A1 24/04/2025

Solicitante:

AZTHERAPIES INC [US]
AZTherapies, Inc

JP_2024086870_A

Resumen de: US2025129158A1

The invention provides compositions and methods for suppressing autoimmune components of neurodegenerative diseases and thereby providing therapeutic effects to patients suffering from such diseases. Compositions and methods include immunosuppressive moieties such as regulatory T cells (Tregs) and proteins expressed by Tregs coupled to a chimeric antigen receptor or protein that specifically binds one or more glial cell markers. Therapeutically effective doses of said compounds for treating neurodegenerative diseases including progressive supranuclear palsy (PSP), Parkinson's disease (PD), Alzheimer's, Huntington's disease, amyotrophic lateral sclerosis (ALS), chronic traumatic encephalopathy (CTE), and prion diseases are disclosed.

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