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LastUpdate Última actualización 04/05/2025 [07:29:00]
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Solicitudes publicadas en los últimos 60 días / Last 60 days publications
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MUTATION-INDEPENDENT ALLELE-SPECIFIC CRISPR TARGETING STRATEGY FOR TREATING GENETIC DISEASES

NºPublicación:  WO2025056018A1 20/03/2025
Solicitante: 
THE HONG KONG UNIV OF SCIENCE AND TECHNOLOGY [CN]
HONG KONG CENTER FOR NEURODEGENERATIVE DISEASES LTD [CN]
THE HONG KONG UNIVERSITY OF SCIENCE AND TECHNOLOGY,
HONG KONG CENTER FOR NEURODEGENERATIVE DISEASES LIMITED
WO_2025056018_A1

Resumen de: WO2025056018A1

Provided is the new compositions and methods useful for the treatment, prevention, and potential cure of a genetic disease, such as familial Alzheimer's Disease, by disrupting the genomic sequence harboring one or more SNPs that are of high prevalence in a population but no relevance to the particular disease except for their genomic locations being in the same gene exon as a disease-relevant allele and upstream from such disease-relevant allele present in the genome of a treatment recipient.

BENZOTRIAZOLE COMPOUND

NºPublicación:  US2025092064A1 20/03/2025
Solicitante: 
DAIICHI SANKYO COMPANY LTD [JP]
KYOTO PHARMACEUTICAL IND LTD [JP]
DAIICHI SANKYO COMPANY, LIMITED,
KYOTO PHARMACEUTICAL INDUSTRIES, LTD
US_2025092064_PA

Resumen de: US2025092064A1

The present invention aims to provide a medicament capable of treating and/or preventing diseases associated with oxidative stress by inhibiting the protein-protein interaction between Keap1 and Nrf2 and activating Nrf2. The present invention relates to a compound represented by the following formula (1):wherein each symbol is as described in the DESCRIPTION, or a pharmaceutically acceptable salt thereof. In addition, the present invention also relates to a medicament containing the aforementioned compound, for the prophylaxis and/or treatment of diseases involving oxidative stress selected from the group consisting of chronic kidney disease, non-alcoholic steatohepatitis, chronic obstructive pulmonary disease, radiation skin disorder, radiation mucosal disorder, cardiac failure, pulmonary arterial hypertension, Parkinson's disease, Friedreich's ataxia, multiple sclerosis, age-related macular degeneration, retinitis pigmentosa and glaucoma.

Methods for Preventing Ectopic Brain Mineralization in Alzheimer's Disease and Dementias

NºPublicación:  US2025090505A1 20/03/2025
Solicitante: 
TIAN ERMING [US]
ZANGARI MAURIZIO [US]
TZERMA LLC [US]
Tian Erming,
Zangari Maurizio,
Tzerma LLC
US_2024390336_PA

Resumen de: US2025090505A1

Provided herein are methods for preventing biomineralization in brain tissue or neuronal tissue in vivo or in vitro by inhibiting the deposition of hydroxyapatite therein. Also provided are methods for delaying the progression or onset of Alzheimer's disease in a subject and for preventing the development of Alzheimer's disease in a post-traumatic brain injury subject. Contacting the brain tissue or neuronal tissue with or administering to a subject the drugs Levamisole and/or Foscarnet and/or a bisphosphonate inhibits or decreases levels of alkaline phosphatase and calcium and counteracts alpha-glycerophosphate therein to inhibit the activation of spontaneous biomineralization within a brain tissue to decrease or prevent ectopic biomineralization.

5,6-DISUBSTITUTED ALKYL 1H AND 2H-INDAZOLES AS DUAL BUTYRYLCHOLINESTERASE AND p38α MITOGEN-ACTIVATED PROTEIN KINASE INHIBITORS FOR USE IN THE TREATMENT OF NEURODEGENERATIVE AND INFLAMMATORY DISEASES

NºPublicación:  WO2025056518A1 20/03/2025
Solicitante: 
UNIVERZA V LJUBLJANI [SI]
UNIVERZA V LJUBLJANI
WO_2025056518_A1

Resumen de: WO2025056518A1

The present invention relates to the field of medicinal chemistry and more specifically to 5,6- disubstituted 1H- and 2H- indazoles as pharmaceutically active compounds. The compounds of the present invention are dual inhibitors of butyrylcholinesterase and p38α mitogen-activated protein kinase, and thus are particularly useful for the treatment of various diseases which may be therapeutically modified by altering the activity of butyrylcholinesterase (BChE) and/or p38α MAP kinase such as dementias including Alzheimer's disease (AD) and other conditions involving chronic inflammation.

AGENT FOR SUPPRESSING AMYLOID β AGGREGATION/DEPOSITION AND AGENT FOR SUPPRESSING/IMPROVING COGNITIVE DECLINE OF SUBJECT

NºPublicación:  WO2025058089A1 20/03/2025
Solicitante: 
KANEKA CORP [JP]
\u682A\u5F0F\u4F1A\u793E\u30AB\u30CD\u30AB
WO_2025058089_A1

Resumen de: WO2025058089A1

The purpose of the present invention is to provide an agent for suppressing amyloid β aggregation/deposition and an agent for suppressing/improving cognitive decline of a subject both of which are expected to have the effect of treating and/or preventing Alzheimer's disease. Specifically, the present invention pertains to an agent for suppressing amyloid β aggregation/deposition or an agent for suppressing/improving cognitive decline of a subject, the agent comprising an extract of a plant belonging to Myrtaceae.

TECHNOLOGY FOR DEGENERATIVE BRAIN DISEASE-THERAPEUTIC PROTEIN DELIVERY SPECIFICALLY TO BRAIN BY USING EXOSOMES COMPRISING DEGENERATIVE BRAIN DISEASE-THERAPEUTIC PROTEIN

NºPublicación:  WO2025058473A1 20/03/2025
Solicitante: 
EXOSTEMTECH CO LTD [KR]
RESEARCH \uFF06 BUSINESS FOUNDATION SUNGKYUNKWAN UNIV [KR]
\uC8FC\uC2DD\uD68C\uC0AC \uC5D1\uC18C\uC2A4\uD15C\uD14D,
\uC131\uADE0\uAD00\uB300\uD559\uAD50\uC0B0\uD559\uD611\uB825\uB2E8
WO_2025058473_A1

Resumen de: WO2025058473A1

The present invention relates to: a pharmaceutical composition for selectively preventing or treating neurodegenerative diseases including Parkinson's disease, comprising as an active ingredient exosomes into which has been delivered a degenerative brain disease-therapeutic protein via a photocleavable protein; and a method for preparing same. Parkinson's disease therapeutic proteins, such as Parkin protein, effectively inhibit the death of damaged dopaminergic neurons, and have protective effects against neurotoxic substances, and thus have an effect of preventing damage to neurons.

CARD9 ATTENUATES AB PATHOLOGY AND MODIFIES MICROGLIAL RESPONSES IN AN ALZHEIMER’S DISEASE

NºPublicación:  US2025090571A1 20/03/2025
Solicitante: 
UNIV OF VIRGINIA PATENT FOUNDATION [US]
University of Virginia Patent Foundation
US_2025090571_PA

Resumen de: US2025090571A1

Provided herein are methods and compositions to enhance or activate protective microglial activities (such as phagocytosis of neurotoxic material) by activating CARD9.

Methods and compositions for the treatment of rare diseases

NºPublicación:  AU2025201530A1 20/03/2025
Solicitante: 
SANGAMO THERAPEUTICS INC
Sangamo Therapeutics, Inc
AU_2025201530_A1

Resumen de: AU2025201530A1

The present disclosure is in the field of modulation of genes involved in rare diseases including for diagnostics and therapeutics for rare diseases such as Angelman's Syndrome, 5 Facioscapulohumeral Muscular Dystrophy (FHMD), Amyotrophic Lateral Sclerosis (ALS), Frontotemporal dementia (FTD) and Spinal Muscular Atrophy (SMA).

NOVEL COMPOUNDS FOR THE DIAGNOSIS OF TDP-43 PROTEINOPATHIES

NºPublicación:  AU2023348297A1 20/03/2025
Solicitante: 
AC IMMUNE SA
AC IMMUNE SA
AU_2023348297_PA

Resumen de: AU2023348297A1

The present invention relates to compounds which are suitable for imaging TDP-43 (Transactive response (TAR) DNA binding protein 43 kDa) aggregates. The compounds can be used, for example, for diagnosing a disease, disorder or abnormality associated with TDP-43 aggregates or a TDP-43 proteinopathy, such as amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), Frontotemporal dementia (FTD) and limbic-predominant age-related TDP-43 encephalopathy (LATE).

Methods for treating Alzheimer's disease

NºPublicación:  AU2025201237A1 20/03/2025
Solicitante: 
BIOGEN INT NEUROSCIENCE GMBH
Biogen International Neuroscience GmbH
AU_2025201237_A1

Resumen de: AU2025201237A1

METHODS FOR TREATING ALZHEIMER'S DISEASE A method for treatment of a human patient for Alzheimer's disease (AD) comprises sequentially administering multiple doses of a recombinant, fully human, anti-amyloid beta monoclonal antibody to the patient. In preferred embodiments, the antibody is administered in increasing amounts over a period of time. In preferred embodiments, the susceptibility of the patient to amyloid related imaging abnormalities (ARIA) is thereby reduced.

Methods for Treating Neurodegenerative Disorders

NºPublicación:  US2025090541A1 20/03/2025
Solicitante: 
PRAETEGO INC [US]
Praetego Inc
WO_2023212510_A1

Resumen de: US2025090541A1

Disclosed are methods for treating or inhibiting development of a disorder selected from the group consisting of a frontotemporal dementia (FTD), Pick's disease, progressive supranuclear palsy, Huntington's disease, Parkinson's disease, corticobasal degeneration, amyotrophic lateral sclerosis (ALS), Lewy body disease, and hippocampal sclerosis (HS), by administering a compound of formula I.

AMINOPYRIMIDINYL DERIVATIVES FOR THE TREAMENT OF PARKINSON'S DISEASE

NºPublicación:  EP4522603A1 19/03/2025
Solicitante: 
UNIV DUNDEE [GB]
University Of Dundee
CN_119325470_A

Resumen de: WO2023218187A1

Disclosed are heterobifunctional compounds that can induce the degradation of leucine-rich repeat kinase 2 (LRRK2) and PDE6D. These compounds can engage LRRK2 on one end and bind to an ubiquitin E3 ligase (e.g. cereblon, Von Hippel-Lindau, or Cellular Inhibitor of Apoptosis (cIAP)) on the other end and therefore bring LRRK2 in close proximity to the E3 ligase and induce the ubiquitination and degradation of the LRRK2 protein. Also disclosed are pharmaceutical composition comprising the heterobifunctional compounds and methods of using the compounds to treat LRRK2 and PDE6D- related diseases and disorders.

THERAPEUTIC METHODS FOR TREATING ALS

NºPublicación:  EP4522147A1 19/03/2025
Solicitante: 
BIOJIVA LLC [US]
Biojiva LLC
AU_2023269025_PA

Resumen de: AU2023269025A1

Disclosed are therapeutic methods for reducing the rate of functionality loss in patients suffering from amyotrophic lateral sclerosis (ALS) during the accelerated stages of this 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:  EP4522217A1 19/03/2025
Solicitante: 
UNIV WUERZBURG J MAXIMILIANS [DE]
Julius-Maximilians-Universit\u00E4t W\u00FCrzburg
AU_2023267136_PA

Resumen de: AU2023267136A1

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

Composition for preventing ameliorating or treating Parkinson's disease comprising trolox as effective component

NºPublicación:  KR20250038283A 19/03/2025
Solicitante: 
경상국립대학교산학협력단

Resumen de: KR20250038283A

본 발명은 트롤록스를 유효성분으로 포함하는 파킨슨병의 예방, 개선 또는 치료용 조성물에 관한 것으로, 상기 트롤록스는 PD 마우스를 이용한 행동분석에서 운동기능 장애를 개선하는 효과 있으며, PD 마우스의 선조체 및 SNpc에서 α-시누클레인의 발현량을 감소시키고, 도파민 합성 및 수송을 담당하는 도파민성 신경 세포 마커(TH, VMAT2 및 DAT)의 발현량을 증가시키며, 산화스트레스 관련 단백질 Nrf2 및 HO-1의 발현량을 증가시킬뿐만 아니라, ROS 및 LPO 함량을 감소시키는 효과가 있고, 성상세포 및 신경아교세포의 활성 마커인 GFAP 및 Iba1의 발현량을 감소시키는 효과가 있으며, 신경염증을 유발하는 전염증성 사이토카인(NF-κB, TNF-α 및 IL-1β)을 감소시키는 효과가 있으므로, 파킨슨병의 예방, 개선 또는 치료를 위한 의약품, 건강기능 식품 또는 사료 첨가제로 유용하게 사용할 수 있다.

Vanillic Acid Derivative Compositions for Alzheimer's Treatment and Neuroinflammation Therapy

NºPublicación:  KR20250037364A 17/03/2025
Solicitante: 
주식회사테라노큐어

Resumen de: KR20250037364A

본 발명은 바닐릭산 유도체 화합물에 관한 것으로서, 바닐릭산과 카테콜아민 간의 탈수축합반응에 의해 형성될 수 있다. 본 발명에 따른 바닐릭산 유도체 화합물은 알츠하이머병 및 신경염증 치료제제로 사용할 수 있다는 점에 특징이 있다. 또한 본 발명은 상기 화합물을 포함하는 알츠하이머 치료제 조성물 및 신경염증 치료제 조성물에 관한 것이다.

HYDROGEN-GAS-CONTAINING DRUG FOR CAUSAL TREATMENT OF ALZHEIMER'S DISEASE (DISEASE-MODIFYING DRUG)

NºPublicación:  AU2023316187A1 13/03/2025
Solicitante: 
H2 GLOBAL GROUP S R O
H2 GLOBAL GROUP S.R.O
AU_2023316187_PA

Resumen de: AU2023316187A1

Provided is a drug for treating Alzheimer's disease, the drug enabling retention of cognitive function amelioration and nerve quality improvement for a specific time even after treatment ends. This drug for causal treatment of Alzheimer's disease (disease-modifying drug) contains hydrogen gas as an active ingredient.

M4 ACTIVATORS/MODULATORS AND USES THEREOF

NºPublicación:  AU2023341169A1 13/03/2025
Solicitante: 
CEREVEL THERAPEUTICS LLC
CEREVEL THERAPEUTICS, LLC
AU_2023341169_A1

Resumen de: AU2023341169A1

The present disclosure provides compounds of Formula I: (I), or an N- oxide thereof, or a pharmaceutically acceptable salt of the compound or the N-oxide, wherein: A, Y, m, n, p, R1, R2, R3, R3a, R4, R5, R6, R7, 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.

METHODS OF REDUCING NEURODEGENERATION ASSOCIATED WITH NEURODEGENERATIVE DISEASES

NºPublicación:  AU2023347307A1 13/03/2025
Solicitante: 
EISAI R&D MANAGEMENT CO LTD
WASHINGTON UNIV
EISAI R&D MANAGEMENT CO., LTD,
WASHINGTON UNIVERSITY
AU_2023347307_A1

Resumen de: AU2023347307A1

The disclosure relates to lemborexant, a dual orexin receptor antagonist, and compositions and methods for use in treatment of Alzheimer's disease (AD), e.g., in a subject who has AD or who is at risk for developing AD.

ESCHERICHIA COLI ENGINEERING STRAIN FOR FUSION EXPRESSION OF TAU-4R PROTEIN

NºPublicación:  US2025084135A1 13/03/2025
Solicitante: 
YUANBEN ZHUHAI HENGQIN BIOTECHNOLOGY CO LTD [CN]
YUANBEN (ZHUHAI HENGQIN) BIOTECHNOLOGY CO., LTD
JP_2025503145_A

Resumen de: US2025084135A1

Fusion protein Tau-4R can be recombined into Escherichia coli to achieve the fusion expression. It is confirmed by means of experiments that the tau protein prepared by the strain may be used in the treatment research of Alzheimer's disease.

TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)

NºPublicación:  US2025084435A1 13/03/2025
Solicitante: 
VOYAGER THERAPEUTICS INC [US]
THE CURATORS OF THE UNIV OF MISSOURI [US]
Voyager Therapeutics, Inc,
THE CURATORS OF THE UNIVERSITY OF MISSOURI
CN_119242711_A

Resumen de: US2025084435A1

The present invention relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and/or canine degenerative myelopathy (DM).

Melafenoxate, 2-(1-adamantylamino)ethyl 2-(4-chlorophenoxy)acetate and derivatives thereof for treating circadian rhythm sleep disorders with neurodegenerative diseases such as Alzheimer's disease

NºPublicación:  US2025082598A1 13/03/2025
Solicitante: 
NLS PHARMACEUTICS AG [CH]
NLS Pharmaceutics AG
JP_2024525988_PA

Resumen de: US2025082598A1

The invention concerns a compound of formula (I) R1=H or acyl group, preferably R1=H; R2=halogen atom selected in the group consisting of: F, Cl, Br, I, preferably R2=Cl or a pharmaceutically acceptable isomer, salt and/or solvate thereof, for use in preventing and/or treating circadian rhythm sleep disorders and/or circadian rhythm sleep neurological diseases and/or any medical condition associated due to sleep deprivation.

AAV-BASED MODULATION OF GBA1 AND RELATED COMPOSITIONS AND USES THEREOF

NºPublicación:  US2025082686A1 13/03/2025
Solicitante: 
ASPEN NEUROSCIENCE INC [US]
Aspen Neuroscience, Inc
WO_2023004370_PA

Resumen de: US2025082686A1

The present disclosure provides adeno-associated vector (AAV)-based methods of increasing gene expression of a GBA1 in floor plate midbrain progenitor cells, determined dopamine (DA) neuron progenitor cells, and/or DA neurons, or glial cells, including those differentiated from pluripotent stem cells, and methods of lineage specific differentiation of the same. Also provided are compositions and uses thereof, such as for treating neurodegenerative diseases and conditions, including Parkinson's disease.

SPECIFIC DOSAGE OF PHENOTHIAZINE COMPOUNDS FOR USE IN TREATMENT OR PREVENTION OF ALZHEIMER'S DEMENTIA

NºPublicación:  US2025082647A1 13/03/2025
Solicitante: 
IMMUNGENETICS AG [DE]
IMMUNGENETICS AG
JP_2025501408_A

Resumen de: US2025082647A1

The present invention relates in a first aspect to a compound of formula (I) for use in the treatment and/or in prevention of Alzheimer's dementia, wherein the compound of formula (I) is administered in a dosage<20 mg. In a second aspect, the invention relates to a pharmaceutical composition comprising the compound according to the first aspect.

NOVEL BIFUNCTIONAL HETEROCYCLIC COMPOUND HAVING BTK DEGRADATION FUNCTION VIA UBIQUITIN PROTEASOME PATHWAY, AND USE THEREOF

Nº publicación: US2025082762A1 13/03/2025

Solicitante:

KOREA RESEARCH INSTITUTE OF CHEMICAL TECH [KR]
UBIX THERAPEUTICS INC [KR]
KOREA RESEARCH INSTITUTE OF CHEMICAL TECHNOLOGY,
UBIX THERAPEUTICS, INC

US_2025073341_A1

Resumen de: US2025082762A1

The present invention relates to a novel heterocyclic compound and a composition, for preventing or treating a cancer, an autoimmune disease, and an inflammatory disease, comprising same. The novel heterocyclic compound of the present invention is a bifunctional compound having a Bruton's tyrosine kinase (BTK) degradation function via a ubiquitin proteasome pathway, and may be utilized as a composition for preventing or treating a cancer, an autoimmune disease, and Parkinson's disease.

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