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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.

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.

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.

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.

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.

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.

(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.

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.

Compound having kdm5 inhibitory activity and pharmaceutical use thereof

NºPublicación:  NZ793960A 19/12/2025
Solicitante: 
ONO PHARMACEUTICAL CO LTD
ONO PHARMACEUTICAL CO. LTD
ES_3037418_T3

Resumen de: NZ793960A

The present invention provides KDM5 inhibitor. The compound disclosed herein represented by the general formula (I) : wherein all symbols have the same meanings as the definitions described in the specification; or a salt thereof is useful as a prophylactic and/or therapeutic agent for cancer, Huntington’s disease, Alzheimer’s disease and the like.

Nutritional composition for improving cell membranes

NºPublicación:  NZ795279A 19/12/2025
Solicitante: 
N V NUTRICIA
N.V. Nutricia
ES_2987545_T3

Resumen de: NZ795279A

An early in life nutritional intervention with a lipid component having both large lipid globules with phospholipids and an increased amount of palmitic acid in the sn-2 position in triglycerides were found to improve the fatty acid composition of cell membranes, in particular brain membranes. The nutritional composition comprising the lipid can therefore be used for improving the fatty acid composition of cell membranes, improving cognitive performance, improving cognitive development, improving behavioural performance, improving behavioural development, improving visual acuity, improving fine motor skills and preventing or treating other disorders associated with impaired brain membrane fatty acid composition, e.g., attention deficiency, ADHD, autism, dyslexia, depression, bipolar depression, anxiety, schizophrenia, obsessive compulsive disorder, bulimia, abuse of alcohol or drugs, borderline personality disorder, panic disorder, social phobia, cognitive impairment, dementia, Alzheimer’s disease or Parkinson’s disease.

Continuous administration of pharmaceutical composition for treatment of neurodegenerative disorders

NºPublicación:  NZ768098A 19/12/2025
Solicitante: 
LOBSOR PHARMACEUTICALS AB
LOBSOR PHARMACEUTICALS AKTIEBOLAG
US_2025213518_PA

Resumen de: NZ768098A

A method of treating patients by using a pharmaceutical composition which comprises (i) a dopamine replacement agent selected from levodopa, melevodopa, etilevodopa and combinations thereof or a pharmaceutically acceptable salt of levodopa, (ii) a dopamine decarboxylase inhibitor (DDI) selected from carbidopa, benserazide and combinations thereof, and (iii) a catechol-O-methyltransferase (COMT) inhibitor selected from entacapone, tolcapone, opicapone and combinations thereof for treating Parkinson’s Disease, where the composition is administered in two dosages, the second dosage being continuously administered.

Aav treatment of huntington’s disease

NºPublicación:  NZ751950A 19/12/2025
Solicitante: 
UNIV OF MASSACHUSETTS
UNIVERSITY OF MASSACHUSETTS
JP_2025063053_A

Resumen de: NZ751950A

Aspects of the disclosure relate to compositions and methods useful for treating Huntington’s disease. In particular, the disclosure provides interfering nucleic acids (e.g., artificial mature miRNAs flanked by a miR-155 or a miR-30 backbone sequence) targeting the huntingtin gene (HTT) and methods of treating Huntington’s disease using the same.

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.

TREATMENTS FOR AMYOTROPHIC LATERAL SCLEROSIS USING DAZUCORILANT

NºPublicación:  US2025381178A1 18/12/2025
Solicitante: 
CORCEPT THERAPEUTICS INCORPORATED [US]
Corcept Therapeutics Incorporated
US_2025381178_PA

Resumen de: US2025381178A1

Applicant discloses methods and compositions for treating a patient suffering from amyotrophic lateral sclerosis (ALS) comprising administration of a heteroaryl ketone fused azadecalin compound. In embodiments, the heteroaryl ketone fused azadecalin compound is dazucorilant: (R)-(1-(4-fluorophenyl)-6-((4-(trifluoromethyl)phenyl)sulfonyl)-4,4a,5,6,7,8-hexahydro-1-H-pyrazolo3,4-gisoquinolin-4a-yl)(pyridin-2-yl)methanone, having the chemical structure illustrated asSuitable doses include daily administration of 150 milligrams and 300 milligrams of dazucorilant. Suitable doses include daily administration of dazucorilant with food, or with water, or with food and water. Daily administration of dazucorilant is effective to increase dazucorilant exposure up to about 2-fold when continued for seven days or more. Administration of such a heteroaryl ketone fused azadecalin compound may comprise oral administration, enteral administration, or other administration. Pharmaceutical compositions comprising dazucorilant are useful in the treatment of patients suffering from ALS. Suitable pharmaceutical compositions comprising dazucorilant include, e.g., pharmaceutical compositions for oral administration and pharmaceutical compositions for enteral administration.

MAGNESIUM COMPOSITIONS AND USES THEREOF FOR NEUROLOGICAL DISORDERS

NºPublicación:  US2025381158A1 18/12/2025
Solicitante: 
NEUROCENTRIA INC [US]
Neurocentria, Inc
US_2025381158_A1

Resumen de: US2025381158A1

A composition for administration to a subject, such as oral administration to a subject, for example, has been provided. Such a composition may comprise at least one magnesium-counter ion compound. A magnesium-counter ion composition described herein may be useful for any of a variety of applications provided herein, such as maintaining, enhancing, and/or improving health, nutrition, and/or another condition of a subject, and/or cognitive, learning, and/or memory function. A magnesium-counter ion composition provided herein may be useful for administration to a subject presenting magnesium deficiency, mild cognitive impairment, Alzheimer's disease, attention deficit hyperactivity disorder, ALS, Parkinson's disease, diabetes, migraine, anxiety disorder, mood disorder, and/or hypertension. A kit, method, and other associated technology are also provided.

Gene constructs for silencing alpha-synuclein and uses thereof

NºPublicación:  US2025382611A1 18/12/2025
Solicitante: 
UNIQURE BIOPHARMA B V [NL]
uniQure biopharma B.V
JP_2024523445_A

Resumen de: US2025382611A1

The present invention relates to a nucleic acid, to a composition, to the medical use of said composition in the treatment and/or prevention of Parkinson's Disease (PD), Multiple System Atrophy (MSA) and/or other α-synucleopathies.

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.

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.

Fasting-mimicking diet (FMD) as an intervention for Alzheimer's disease (AD)

NºPublicación:  AU2025271406A1 18/12/2025
Solicitante: 
UNIV OF SOUTHERN CALIFORNIA
University of Southern California
AU_2025271406_A1

Resumen de: AU2025271406A1

A method of treating Alzheimer’s associated pathology includes a step of identifying subject having a pathology that is associated with Alzheimer’s disease. A fasting mimicking diet (FMD) is then administered to the subject for a first time period. Methods for treating amyloid plaque formation and/or elevated levels of tau protein as well as neuroinflammation are also provided. ov o v

smp30 Use Of Smp30 For Therapeutic Target For Alzheimer's Disease Or Elongation Of Lifespan And Health Lifespan

NºPublicación:  KR20250175781A 18/12/2025
Solicitante: 
고려대학교산학협력단

Resumen de: KR20250175781A

본 발명은 smp30의 알츠하이머병 치료 또는 수명 및 건강수명의 연장 용도에 관한 것이다. 본 발명에 따른 smp30은 알츠하이머병 질환 내 인산화된 타우 단백질의 감소 뿐만 아니라, 운동 능력 증가, 산화 스트레스에 대한 저항성 증가, 항산화 효과 증가, 굶주림 스트레스 저항성 증가 및 노화 감소에 대한 효과를 가져, 알츠하이머병 치료, 알츠하이머병 치료제 후보물질 발굴, 및 수명 및 건강수명 연장 또는 개선에 활용이 가능하다.

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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