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Solicitudes publicadas en los últimos 60 días / Last 60 days publications
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PREVENTIVE OR THERAPEUTIC AGENT FOR NEURODEGENERATIVE DISEASE

NºPublicación:  MX2025013089A 01/12/2025
Solicitante: 
MEDILABO RFP INC [JP]
UNIV PUBLIC CORPORATION OSAKA [JP]
MEDILABO RFP, INC,
UNIVERSITY PUBLIC CORPORATION OSAKA
WO_2024228392_PA

Resumen de: MX2025013089A

The purpose of the present invention is to provide a drug that exhibits the effect of inhibiting aggregation of a causative protein of an HRE-related neurodegenerative disease such as ALS. According to the present invention, rifampicin or a related substance selected from the group consisting of rifampicin, a derivative thereof, and salts thereof, and/or resveratrol or a related substance selected from the group consisting of resveratrol and a derivative thereof is an active ingredient of a preventive or therapeutic agent for a neurodegenerative disease caused by TDP-43 accumulation, or an active ingredient of a preventive or therapeutic agent for ALS.

TREATMENT OF PARKINSON'S DISEASE IN A PATIENT USING A GLUCOCEREBROSIDASE ACTIVATOR

NºPublicación:  MX2025010647A 01/12/2025
Solicitante: 
BIAL R&D INVEST S A [PT]
BIAL - R&D INVESTMENTS, S.A
AU_2024237252_A1

Resumen de: MX2025010647A

Methods for preventing, limiting or delaying clinical motor progression in a subject with Parkinson's disease with low GCase activity, such as a PD patient with a pathogenic variant in the glucocerebrosidase 1 (GBA1) gene (GBA-PD) is provided, said methods comprising administering a therapeutically effective amount of 5,7-dimethyl-N-((1R,4R)-4- (pentyloxy)cyclohexyl)pyrazolol1,5-apyrimidine-3-carboxamide (Compound A), or a pharmaceutically acceptable salt thereof, to said subject.

RNAI AGENTS FOR INHIBITING EXPRESSION OF ATAXIN-2 (ATXN2), COMPOSITIONS THEREOF, AND METHODS OF USE

NºPublicación:  MX2025010867A 01/12/2025
Solicitante: 
ARROWHEAD PHARMACEUTICALS INC [US]
ARROWHEAD PHARMACEUTICALS, INC
AU_2024240456_A1

Resumen de: MX2025010867A

Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a Ataxin-2 (ATXN2) gene. The ATXN2 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an ATXN2 gene. Pharmaceutical compositions that include one or more ATXN2 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described ATXN2 RNAi agents to central nervous system (CNS) tissue, in vivo, provides for inhibition of ATXN2 gene expression and a reduction in ATXN2 activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including spinocerebellar ataxia type 2 (SCA2) or amyotrophic lateral sclerosis (ALS.)

Aav treatment of huntington's disease

NºPublicación:  IL323940A 01/12/2025
Solicitante: 
UNIV OF MASSACHUSETTS [US]
UNIVERSITY OF MASSACHUSETTS
IL_323940_A

Resumen de: ZA201902226B

Aspects of the disclosure relate to compositions and methods useful for treating Huntington' s disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and methods of treating Huntington's disease using the same.

VARIANT RNAi

NºPublicación:  CA3255657A1 01/12/2025
Solicitante: 
GENZYME CORP [US]
Genzyme Corporation
AU_2025205501_A1

Resumen de: CA3255657A1

Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.

Treatment for parkinson’s disease

NºPublicación:  NZ748592A 28/11/2025
Solicitante: 
SUN PHARMA ADVANCED RES COMPANY LIMITED
SUN PHARMA ADVANCED RESEARCH COMPANY LIMITED
US_2024066014_PA

Resumen de: NZ748592A

The invention relates to a method of treating or preventing Parkinson’s disease in a subject comprising administering a compound of Formula I wherein, R1is -NHC(O) C3-6cycloalkyl and R2is hydrogen; or R1 and R2along with the carbon atoms to which they are attached form a six membered aromatic ring, wherein the ring is substituted with one or more groups selected from hydrogen, halogen and C1-6alkyl; R3and R4are independently selected from group comprising hydrogen, halogen, C1-3alkyl, OC1-alkyl, NO3, SC2alkyl, C1-3haloalkyl, OC1-3haloalkyl, and SC1-3haloalkyl; or a pharmaceutically acceptable salt thereof.

METHODS AND COMPOSITIONS FOR REDUCING SYMPTOMS OF PARKINSON'S DISEASE

NºPublicación:  AU2024250257A1 27/11/2025
Solicitante: 
NEURODERM LTD
NEURODERM, LTD
AU_2024250257_A1

Resumen de: AU2024250257A1

Disclosed is a method for the treatment of a neurological or movement disorder, e.g., Parkinson's disease, in a patient in need thereof, by parenteral administration of levodopa and a dopa decarboxylase inhibitor (DDCI), such as carbidopa, benserazide or any combination thereof.

APOMORPHINE PRODRUGS AND USES THEREOF

NºPublicación:  US2025361252A1 27/11/2025
Solicitante: 
EVER NEURO PHARMA GMBH [AT]
EVER Neuro Pharma GmbH
US_2025361252_A1

Resumen de: US2025361252A1

The present invention relates to a compound of formula (I), which is a phosphate ester of apomorphine, or a pharmaceutically acceptable salt thereof. The apomorphine phosphate ester according to the invention exhibits remarkably advantageous properties as a therapeutic, including a favorable tolerability, an improved side effect profile, particularly a reduced occurrence of skin nodule formation and panniculitis when administered subcutaneously, as well as pharmacokinetic and metabolic properties rendering it particularly well-suited as an apomorphine prodrug. The invention further relates to the compound of formula (I) for use as a medicament, particularly for use in the treatment of Parkinson's disease.

INJECTION PREPARATION

NºPublicación:  WO2025243901A1 27/11/2025
Solicitante: 
SENTAN PHARMA INC [JP]
\u682A\u5F0F\u4F1A\u793E\u3000\uFF33\uFF25\uFF2E\uFF34\uFF21\uFF2E\u3000\uFF30\uFF48\uFF41\uFF52\uFF4D\uFF41
WO_2025243901_PA

Resumen de: WO2025243901A1

Provided is an injection preparation for the treatment of Parkinson's disease or restless legs syndrome, the injection preparation comprising microparticles each containing a free dopamine receptor agonist and a biodegradable polymer. The volume average particle diameter D50 of the microparticles is 50 μm or less, and the concentration of the dopamine receptor agonist in plasma of a subject to which the injection preparation has been administered is retained for 1 month or longer.

2-(4-CHLORO-3-FLUOROPHENOXY)-N-TRANS-4-5-3-(TRIFLUOROMETHOXY)-1-AZETIDINYL-1,3,4-OXADIAZOL-2-YLCYCLOHEXYL-ACETAMIDE FOR USE IN THE TREATMENT OF VANISHING WHITE MATTER, HUNTINGTON'S DISEASE, CHARCOT MARIE TOOTH SYNDROME, AMYOTROPHIC LATERAL SCLEROSIS OR FOR INCREASING THE GUANINE NUCLEOTIDE EXCHANGE FACTOR ACTIVIT

NºPublicación:  WO2025245500A1 27/11/2025
Solicitante: 
REVIR THERAPEUTICS INC [US]
SHENZHEN ZHONGGE BIOLOGICAL TECH CO LTD [CN]
REVIR THERAPEUTICS, INC,
SHENZHEN ZHONGGE BIOLOGICAL TECHNOLOGY CO., LTD
WO_2025245500_PA

Resumen de: WO2025245500A1

The invention described herein provides eIF2B agonist (e.g., COMPOUND 1 ) and uses thereof for treatment of diseases, especially neurodegenerative diseases, including vanishing white matter (VWM), Huntington's disease (HD), Charcot Marie Tooth syndrome (CMT), and Amyotropic Lateral Sclerosis (ALS).

1,4-DIHYDROBENZODPYRAZOLO3,4-F1,3DIAZEPINE DERIVATIVES AND RELATED COMPOUNDS AS LRRK2, NUAK1 AND/OR TYK2 KINASE MODULATORS FOR THE TREATMENT OF E.G. AUTOIMMUNE DISEASE

NºPublicación:  US2025361237A1 27/11/2025
Solicitante: 
ORIGENIS GMBH [DE]
Origenis GMBH
US_2025361237_PA

Resumen de: US2025361237A1

The present invention relates to compounds of formula (I) that are capable of modulating, e.g., inhibiting or activating, one or more kinases, especially LRRK2 and/or NUAK1 and/or TYK2 or mutants thereof. The compounds are useful for treating diseases, such as autoimmune diseases, inflammatory diseases, bone diseases, metabolic diseases, neurological and neurodegenerative diseases, cancer, cardiovascular diseases, allergies, asthma, Alzheimer's disease, Parkinson's disease, skin disorders, eye diseases, infectious diseases and hormone-related diseases. The present description discloses the synthesis and characterisation of exemplary compounds as well as pharmacological data thereof (e.g. pages 40 to 146; examples 1 to 63; compounds 1 to 248; tables 1 to 3). Preferred compounds are e.g. 1,4-dihydrobenzodpyrazolo3,4-f1,3diazepine derivatives and related compounds. An exemplary compound is e.g. 5-(2,6-difluorophenyl)-8-methoxy-1,4-dihydrobenzodpyrazolo3,4-f1,3diazepine (example 49). (Formula (II):

THE PROCESS OF PRODUCING EXOSOMES CONTAINING MIR-299-5P FOR THE TREATMENT OF ALZHEIMER'S DISEASE

NºPublicación:  WO2025243064A1 27/11/2025
Solicitante: 
JAFARLOU MAHDI [IR]
JAFARLOU, Mahdi
WO_2025243064_A1

Resumen de: WO2025243064A1

The main problem with treating Alzheimer' s is that there is no certain medicine for its treatment, or that there is medical resistance to it in the patient's body. By using the WJ-MSCs exosomes from the steam cells grade, the exosomes are extracted with the ultracentrifuge method and confirmed in the size of SEM and TEM exosomes in the range of less than 100nm. then the mir-299-5p is transfected to the cell WJ- MSCs, with the help of a vector, and the exosomes are then extracted, gathered, and formulated. Since the vesicles are under 100 nanometers (exo som es) and could enter the cell by passing the brain blood barrier, what is inside them is easily transferred into the neuronic cell and treats them. Also, it decreases the drug resistance in the body, and the existence of the mir-299-5p is targeted, has pharmaceutical effects, and recovers the neuronic cells that were injured.

PRIDOPIDINE FOR TREATING HUNTINGTON'S DISEASE

NºPublicación:  US2025360121A1 27/11/2025
Solicitante: 
PRILENIA NEUROTHERAPEUTICS LTD [IL]
Prilenia Neurotherapeutics Ltd
US_2025360121_PA

Resumen de: US2025360121A1

A method of treating a human patient afflicted with Huntington's disease, comprising periodically orally administering to the patient a pharmaceutical composition comprising pridopidine, its analog or a pharmaceutically acceptable salt thereof.

TREATMENT REGIMENS FOR EARLY IDIOPATHIC PARKINSON'S DISEASE

NºPublicación:  AU2023449890A1 27/11/2025
Solicitante: 
BIAL PORTELA & CA S A
BIAL-PORTELA & CA., S.A
AU_2023449890_A1

Resumen de: AU2023449890A1

Opicapone for use as adjunctive therapy to preparations of levodopa and a DOPA decarboxylase inhibitor (DDCI) in the treatment of Parkinson's disease; characterised in that a patient with Parkinson's disease is treatable with preparations of levodopa and a DDCI without clinically diagnosed motor complications.

METHODS OF DELAYING OR PREVENTING THE ONSET OF ALZHEIMER'S DISEASE USING CRENEZUMAB

NºPublicación:  US2025361292A1 27/11/2025
Solicitante: 
GENENTECH INC [US]
BANNER HEALTH [US]
GENENTECH, INC,
BANNER HEALTH
US_2025361292_PA

Resumen de: US2025361292A1

Provide herein are methods of treating human patients with familial Alzheimer's disease that result in delayed in symptom onset and/or slowed cognitive decline by administering a humanized monoclonal anti-amyloid beta (Aβ) antibody.

METHOD FOR SYNTHESIZING CELOSIANIN II, METHOD FOR SYNTHESIZING BETAXANTHIN, AMYLOID-ß POLYMERIZATION INHIBITOR OR THERAPEUTIC OR PROPHYLACTIC AGENT FOR ALZHEIMER'S DISEASE, AMYLOID PEPTIDE AGGREGATION INHIBITOR, AND HIV-1 PROTEASE ACTIVITY INHIBITOR

NºPublicación:  US2025354188A1 20/11/2025
Solicitante: 
MORI MASASHI [JP]
MORI Masashi
US_2023024032_A1

Resumen de: US2025354188A1

The present invention provides a method of synthesizing celosianin II, a method of synthesizing a betaxanthin, an amyloid-β polymerization inhibitor or a therapeutic or preventive agent for Alzheimer's, an amyloid peptide aggregation inhibitor, and an HIV-1 protease activity inhibitor. A gene having a celosianin II synthesis ability has been isolated from quinoa, and a method of synthesizing celosianin II of the present invention has been constructed. Besides, it has been recognized that celosianin II or the like serves as an active ingredient of each of an amyloid-β polymerization inhibitor or a therapeutic or preventive agent for Alzheimer's, an amyloid peptide aggregation inhibitor, and an HIV-1 protease activity inhibitor.

Preventative Agent or Therapeutic Agent for Amyotrophic Lateral Sclerosis, Parkinson's Disease, Huntington's Disease, Spinocerebellar Ataxia, Aging-Related Degenerative or Neurological Disease, Brain Aging, or Diseases Associated With Brain Aging

NºPublicación:  US2025353904A1 20/11/2025
Solicitante: 
INST OF SCIENCE TOKYO [JP]
INSTITUTE OF SCIENCE TOKYO
CN_119730880_A

Resumen de: US2025353904A1

The present invention addresses the problem of providing an agent for preventing or treating amyotrophic lateral sclerosis (ALS), Parkinson's disease (PD), Huntington's disease (HD), spinocerebellar ataxia (SCA), aging-related degenerative or neurological disease, brain aging, or diseases associated with brain aging, as well as a more stable antibody that exhibits an effect of preventing or treating these diseases, Alzheimer's disease (AD), or frontotemporal lobar degeneration (FTLD). A human monoclonal antibody that specifically binds to human HMGB1, wherein the human monoclonal antibody (anti-human HMGB1 antibody) comprises a heavy chain CDR1, heavy chain CDR2, and heavy chain CDR3 each consisting of a specific amino acid sequence and a light chain CDR1, light chain CDR2, and light chain CDR3 each consisting of a specific amino acid sequence, is used as an agent for preventing or treating ALS, PD, HD, SCA, aging-related degenerative or neurological disease, brain aging, or diseases associated with brain aging. An antibody in which the light chain complementarity determining region (CDR) 3 of the anti-human HMGB1 antibody has been modified is used.

HETEROCYCLIC COMPOUNDS FOR TREATING HUNTINGTON'S DISEASE

NºPublicación:  US2025353841A1 20/11/2025
Solicitante: 
HUFF SARAH ELIZABETH [US]
MUSTAFA DARSHEED NASSER [US]
TOSZEWSKI ADAM [US]
MONDAL DIBYENDU [US]
BIOGEN MA INC [US]
HUFF Sarah Elizabeth,
MUSTAFA Darsheed Nasser,
TOSZEWSKI Adam,
MONDAL Dibyendu,
BIOGEN MA INC
JP_2025525683_PA

Resumen de: US2025353841A1

The present disclosure provides a compound of Formula (I) or a pharmaceutically acceptable salt thereof and its use in, e.g. treating a condition, disease, or disorder in which lowering mutant huntingtin protein (“mHTT”) in a subject is of therapeutic benefit, specifically in treating Huntington disease (“HD”). This disclosure also features a composition containing the same as well as methods of using and making the same.

PRIDOPIDINE FOR TREATING HUNTINGTON'S DISEASE

NºPublicación:  US2025352533A1 20/11/2025
Solicitante: 
PRILENIA NEUROTHERAPEUTICS LTD [IL]
Prilenia Neurotherapeutics Ltd
JP_2024016113_A

Resumen de: US2025352533A1

A method of treating a human patient afflicted with Huntington's disease, comprising periodically orally administering to the patient a pharmaceutical composition comprising pridopidine, its analog or a pharmaceutically acceptable salt thereof.

TRANSDERMAL DELIVERY DEVICE FOR PEPTIDE DELIVERY AND METHODS OF USE

NºPublicación:  US2025352494A1 20/11/2025
Solicitante: 
PASSPORT TECH INC [US]
NATIONAL UNIV CORPORATION HOKKAIDO UNIV [JP]
NATIONAL UNIV CORPORATION HAMAMATSU UNIV SCHOOL OF MEDICINE [JP]
PassPort Technologies, Inc,
National University Corporation Hokkaido University,
National University Corporation Hamamatsu University School of Medicine
JP_2025519589_PA

Resumen de: US2025352494A1

Disclosed herein are patches, methods, devices, and systems for delivering a non-aggregating peptide, such as alcadein and its fragments, into a subject. In some aspects, the patch includes a backing, a matrix comprising a non-aggregating peptides disposed within the matrix, and a release liner. In other aspects, the method includes opening at least one channel in the subject's skin, applying the patch described herein, thereby treating a disease or disorder associated with the brain, such as Alzheimer's disease.

METHOD FOR TREATING AMYOTROPHIC LATERAL SCLEROSIS USING QUERCETIN-CONTAINING COMPOSITIONS

NºPublicación:  US2025352509A1 20/11/2025
Solicitante: 
QUERCIS PHARMA AG [CH]
Quercis Pharma AG
JP_2024521736_PA

Resumen de: US2025352509A1

Compositions and methods for treating amyotrophic lateral sclerosis. A method of treating amyotrophic lateral sclerosis comprising administering to a subject in need thereof an effective amount of quercetin, vitamin B3, vitamin C, zafirlukast and optionally folic acid. Also disclosed are methods of reducing, slowing or abating the progression of amyotrophic lateral sclerosis or a symptom thereof, comprising administering to a subject in need thereof an effective amount of quercetin, vitamin B3, vitamin C, zafirlukast and optionally folic acid.

Methods For Treating Alzheimer's Disease or Dementia Using A Calcilytic And A Calcimimetic

NºPublicación:  US2025352609A1 20/11/2025
Solicitante: 
THE US GOV AS REPRESENTED BY THE DEPARTMENT OF VETERANS AFFAIRS [US]
THE REGENTS OF THE UNIV OF CALIFORNIA [US]
The United States Government As Represented By The Department Of Veterans Affairs,
The Regents of the University of California

Resumen de: US2025352609A1

Disclosed herein are methods of treating or preventing Alzheimer's disease or dementia and concurrently treating hyperparathyrodism by administering a blood-brain barrier (BBB)-impermeable calcimimetic and/or a BBB-permeable calcilytic along with the administration of anti-amyloid-beta therapies. Also disclosed herein are methods of increasing calcium-sensing receptor (CaSR) homodimer formation, increasing expression or activity of the CaSR homodimer and reducing CaSR/GABA-B1 receptor heterodimer formation, reducing expression or activity of the CaSR/GABA-B1 receptor heterodimer in both peripheral tissues and central nervous system (CNS) in a subject.

PARKINSON'S DISEASE THERAPY BY DEVELOPING A TREG-MEDIATED RESPONSE SPECIFIC TO ALPHA-SYNUCLEIN AND DIAGNOSIS METHODS

NºPublicación:  WO2025239904A1 20/11/2025
Solicitante: 
FUND CIENCIA PARA LA VIDA [CL]
MASS CLIFFORD J [US]
FUNDACI\u00D3N CIENCIA PARA LA VIDA,
MASS, Clifford J
WO_2025239904_PA

Resumen de: WO2025239904A1

The application relates to monoclonal antibody clone 1A11, which is highly specific for a C-terminal sequence of human alpha-synuclein (hαSyn) containing three nitrated tyrosines (3NYαSyn) and does not bind to the corresponding unmodified sequence. A CAR specific for 3NYαSyn has been produced, comprising the antigen-recognizing site of the mAb 1A11 fused to an intracellular region containing the signaling motifs of CD3zeta and CD28. Tregs expressing said anti-3NYαSyn CAR have been shown to attenuate neurodegeneration and neuroinflammation in a mouse model for Parkinson's disease. The T reg-response specific for 3YNαSyn exerts a therapeutic effect attenuating the loss of dopaminergic neurons in a preclinical model and represents a promising therapeutic strategy to treat Parkinson's disease patients..

POTENCY ASSAY MATRIX FOR NEURONAL CELLS

NºPublicación:  WO2025240469A1 20/11/2025
Solicitante: 
ASPEN NEUROSCIENCE INC [US]
ASPEN NEUROSCIENCE, INC
WO_2025240469_PA

Resumen de: WO2025240469A1

The invention provides, in some embodiments, a potency assay matrix for assessing potency of dopaminergic neuronal progenitor cells (DANPCs) that are intended for use in treating a neurodegenerative disease such as Parkinson's Disease. The potency assay matrix can include a bioassay for L-DOPA decarboxylase (DDC) activity. In addition to the DDC activity assay matrix, the potency assay matrix may also include one or more additional mechanism of action-based assays such as bioinformatics-based assays for determining whether the DANPCs are dopaminergic neuronal progenitor cells, for determining whether the DANPCs are likely to engraft when implanted into a subject brain, and for determining whether neuronal cells that are derived from the DANPCs are likely to produce dopamine after implantation.

PHARMACEUTICAL COMPOSITION FOR PREVENTING OR TREATING BRAIN DISEASES, COMPRISING MODIFIED MITOCHONDRIA

Nº publicación: WO2025239588A1 20/11/2025

Solicitante:

PAEAN BIOTECHNOLOGY INC [KR]
UNIV INDUSTRY COOPERATION GROUP OF KYUNG HEE UNIV [KR]
\uC8FC\uC2DD\uD68C\uC0AC \uD30C\uC774\uC548\uBC14\uC774\uC624\uD14C\uD06C\uB180\uB85C\uC9C0,
\uACBD\uD76C\uB300\uD559\uAD50 \uC0B0\uD559\uD611\uB825\uB2E8

WO_2025239588_PA

Resumen de: WO2025239588A1

The present invention relates to: a fusion protein comprising a mitochondrial outer membrane anchoring peptide and a cerebrovascular endothelial cell surface protein binding site; modified mitochondria to which the fusion protein is bound; and a pharmaceutical composition comprising the modified mitochondria as an active ingredient. The modified mitochondria comprising the cerebrovascular endothelial cell surface protein binding site according to the present invention can pass through the blood-brain barrier at the cellular and animal level. In addition, administering the modified mitochondria to a Parkinson's disease mouse model can alleviate movement disorders. Therefore, the modified mitochondria according to the present invention can be used as a therapeutic agent for brain diseases caused by mitochondrial dysfunction.

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