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Solicitudes publicadas en los últimos 60 días / Last 60 days publications
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THERAPEUTIC AGENT FOR NEURODEGENERATIVE DISORDER

NºPublicación:  WO2025143100A1 03/07/2025
Solicitante: 
KISSEI PHARMACEUTICAL CO LTD [JP]
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Resumen de: WO2025143100A1

The present invention addresses the problem of providing a medicine that, despite of being a non-ergot dopamine agonist (DA), reduces the risk of the drowsiness side effect, and exhibits an excellent therapeutic effect on neurodegenerative disorders such as Parkinson's disease (PD). The present invention relates to a pharmaceutical composition for treating neurodegenerative diseases such as Parkinson's disease, the pharmaceutical composition comprising 1-{(4aR,6R,8aR)-2-amino-3-cyano-8-methyl-4,4a,5,6,7,8,8a,9-octahydrothieno3,2-gquinolin-6-ylcarbonyl}-3-2-(dimethylamino)ethyl-1-propylurea or a pharmacologically acceptable salt thereof. The pharmaceutical composition is characterized by being orally administered at a daily dose of 0.25 mg to 2 mg in terms of free form.

PHOSPHATIDYLINOSITOL 3,5-BISPHOSPHATES FOR USE IN THE TREATMENT OF NEURODEGENERATIVE DISEASES

NºPublicación:  WO2025141063A1 03/07/2025
Solicitante: 
UNIV DEGLI STUDI DI NAPOLI FEDERICO II [IT]
UNIVERSITA' DEGLI STUDI DI NAPOLI FEDERICO II

Resumen de: WO2025141063A1

The present invention relates to Phosphatidylinositol 3,5-bisphosphate (PI(3,5)P2) of formula (I), preferably formulated in LNP, preferably SANP, and optionally together with a miRNA or antagomir, for use in the treatment of neurodegenerative disorders, like AD, PD and ALS, characterized by impaired/blocked autophagy. It also relates to a LNP, preferably SANP, containing PI(3,5)P2 and optionally a miRNA or antagomir.

PRODUCT PREPARATION BASED ON APPLICATION OF SGRNA FOR THE TREATMENT OF HUNTINGTON'S DISEASE

NºPublicación:  EP4577656A2 02/07/2025
Solicitante: 
LI CHENJIAN [US]
ZHENG SUSHUANG [CN]
Li, Chenjian,
Zheng, Sushuang
AU_2023330511_PA

Resumen de: AU2023330511A1

The present disclosure relates to an sgRNA and its application in the preparation of a product for the treatment of Huntington's disease. The present disclosure was designed and screened to obtain an sgRNA targeting exon 1 of the human HTT gene as shown in SEQ ID NO: 1 or SEQ ID NO: 2. The CRISPR/Cas9 system mediated HTT gene knockout strategy based on this sgRNA and its high homologue sgRNA can efficiently knock out the human Huntingtin gene and achieve gene therapy for Huntington's disease.

COMPOSITIONS AND METHODS FOR THE TREATMENT OF ALZHEIMER'S DISEASE AND OTHER NEUROGENERATIVE DISEASE

NºPublicación:  EP4577305A2 02/07/2025
Solicitante: 
1/1THE TRUSTEES OF COLUMBIA UNIV IN THE CITY OF NEW YORK [US]
1/1THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK
WO_2024044355_PA

Resumen de: WO2024044355A2

Methods and compositions that treat Alzheimer's disease and other neurodegenerative diseases and/or to ameliorate or improve symptoms associated with Alzheimer's disease. In some aspects, the compositions and methods use a serotonin 4 receptor (5-hydroxytryptamine (serotonin) receptor 4, or 5-HT4R) agonist in combination with: (R,S)-ketamine, a (R,S)-ketamine analog, or a pharmaceutically acceptable salt, derivative, or metabolite thereof; an antagonist of the glutamate N-methyl-D-aspartate (NMDA) receptor (NMDAR); or an agonist of the α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA) receptor (AMPAR).

METHODS OF TREATING PARKINSONS DISEASE BY ADMINISTRATION OF APOMORPHINE TO AN ORAL MUCOSA

NºPublicación:  EP4578461A2 02/07/2025
Solicitante: 
SUMITOMO PHARMA AMERICA INC [US]
Sumitomo Pharma America, Inc
EP_4578461_A2

Resumen de: EP4578461A2

Methods and pharmaceutical unit dosage forms for treating Parkinson's disease in a subject (e.g., an "off" episode in a subject having Parkinson's disease) are described. The pharmaceutical unit dosage forms are films having a first portion including particles containing an acid addition salt of apomorphine and a second portion containing a pH neutralizing agent. The pharmaceutical unit dosage forms can be flexible and have toughness greater than 100 g × mm. The methods can involve administering to a subject having Parkinson's disease a therapeutic dose sufficient to produce an apomorphine plasma concentrate of at least 2.64 ng/mL within 45 minutes after the administration. The subject may be identified as having low uptake, medium uptake, or high uptake of apomorphine administered via oral mucosa.

TREATMENTS FOR AMYOTROPHIC LATERAL SCLEROSIS USING DAZUCORILANT

NºPublicación:  MX2025004703A 01/07/2025
Solicitante: 
CORCEPT THERAPEUTICS INCORPORATED [US]
CORCEPT THERAPEUTICS INCORPORATED
MX_2025004703_A

Resumen de: MX2025004703A

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 as. Suitable 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.

COMPOUNDS AND METHODS FOR REDUCING SNCA EXPRESSION

NºPublicación:  MX2025006917A 01/07/2025
Solicitante: 
IONIS PHARMACEUTICALS INC [US]
IONIS PHARMACEUTICALS, INC
AU_2025204414_A1

Resumen de: MX2025006917A

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SNCA mRNA in a cell or animal, and in certain instances reducing the amount of alpha-synuclein protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include motor dysfunction, aggregation of alpha-synuclein, neurodegeneration, cognitive decline and dementia. Such neurodegenerative diseases include Parkinson's disease, dementia withLewy bodies, diffuse Lewy body disease, pure autonomic failure, multiple system atrophy, neuronopathic Gaucher's disease and Alzheimer's disease.

NEW MEDICAL USE OF 3α-ETHYNYL-3ÃY HYDROXYANDROSTAN 17-ONE OXIME

NºPublicación:  MX2025007343A 01/07/2025
Solicitante: 
UMECRINE COGNITION AB [SE]
UMECRINE COGNITION AB
MX_2025007343_A

Resumen de: MX2025007343A

The present invention is directed to the compound golexanolone for use in the treatment of Parkinson's Disease (PD) or for use in the treatment of L-dopa Induced Dyskinesia (LID) in Parkinson's Disease (PD) patients. Further, the present invention is directed to the compound golexanolone or a pharmaceutically acceptable salt thereof, for use in the treatment of Parkinson's Diseases (PD) patients, in particular PD patients exhibiting a L-dopa Induced Dyskinesia (LID).

M4 ACTIVATORS/MODULATORS AND USES THEREOF

NºPublicación:  MX2025003095A 01/07/2025
Solicitante: 
CEREVEL THERAPEUTICS LLC [US]
CEREVEL THERAPEUTICS, LLC
MX_2025003095_A

Resumen de: MX2025003095A

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.

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

NºPublicación:  MX2025001192A 01/07/2025
Solicitante: 
H2 GLOBAL GROUP S R O [CZ]
H2 GLOBAL GROUP S.R.O
MX_2025001192_A

Resumen de: MX2025001192A

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.ã¿¿This drug for causal treatment of Alzheimer's disease (disease-modifying drug) contains hydrogen gas as an active ingredient.

COMPOSITIONS AND METHODS FOR PREVENTING AND TREATING NEURODEGENERATIVE DISEASES

NºPublicación:  US2025205239A1 26/06/2025
Solicitante: 
ARIBIO CO LTD [KR]
ARIBIO CO., LTD
CN_120225200_A

Resumen de: US2025205239A1

The present invention provides a composition for preventing or treating a neurodegenerative disease containing a phosphodiesterase 5 inhibitor (PDE5 inhibitors) and an acetylcholinesterase inhibitor (AChEI) and a method using thereof, wherein the PDE5 inhibitor is selected from among mirodenafil, sildenafil, vardenafil, tadalafil, udenafil, dasantafil, avanafil, pharmaceutically acceptable salts, solvates, hydrates, and a mixture thereof; and the AchEI is selected from among donepezil, rivastigmine, galantamine, physostigmine, tacrine, metrifonate, phenserine, tolserine, eseroline, huperizine A and B, galangin, cardanol, donepezil-AP2238, donepezil-tacrine, tacrine-ferulic acid hybrid, tacrine-hydroxyquinoline, ladostigil, indenyl derivatives, pharmaceutically acceptable salts, solvates, hydrates and a mixture thereof; and the neurodegenerative disease is dementia, Parkinson's disease (PD), Alzheimer's disease (AD), Huntington's disease (HD) or Multiple sclerosis (MS).

AGONISTS OF TREM2 ACTIVITY

NºPublicación:  WO2025136936A1 26/06/2025
Solicitante: 
MERCK SHARP & DOHME LLC [US]
MERCK SHARP & DOHME LLC

Resumen de: WO2025136936A1

The present disclosure is directed to compounds of Formula I and their use as TREM2 agonists for treatment and prevention of a neurodegenerative disorder associated with a loss of function of human TREM2. The disclosed TREM2 agonists may be useful for the treatment of Alzheimer's Disease and associated neurological conditions.

AGONISTS OF TREM2 ACTIVITY

NºPublicación:  WO2025136898A1 26/06/2025
Solicitante: 
MERCK SHARP & DOHME LLC [US]
MERCK SHARP & DOHME LLC

Resumen de: WO2025136898A1

The present disclosure is directed to compounds of Formula (I) and their use as TREM2 agonists for treatment and prevention of a neurodegenerative disorder associated with a loss of function of human TREM2. The disclosed TREM2 agonists may be useful for the treatment of Alzheimer's Disease and associated neurological conditions.

AD-35 POLYMORPHS, PREPARATION METHODS THEREFOR, AND USE THEREOF

NºPublicación:  WO2025130951A1 26/06/2025
Solicitante: 
ZHEJIANG HISUN PHARMACEUTICAL CO LTD [CN]
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CN_120172986_PA

Resumen de: WO2025130951A1

The present invention provides crystalline forms of 6-2-1-(2-pyridylmethyl)-4-piperidylethylspiro1,3dioxolo4,5-fisoindole-7,1'-cyclopropane-5-one phosphate (AD-35), and preparation methods therefor. The invention further provides a use of the crystalline forms of AD-35 in the preparation of a drug for treating Alzheimer's disease.

CRYSTALLINE FORM OF AN ISOXAZOLIDINE DERIVATIVE

NºPublicación:  WO2025137210A1 26/06/2025
Solicitante: 
GENZYME CORP [US]
GENZYME CORPORATION

Resumen de: WO2025137210A1

The present disclosure relates to a compound of formula (1) as an anhydrate which is in a crystalline Form 1, characterized by having a powder-X-ray diffractogram displaying a peak expressed as degree 2-Theta angles at about 8.3 and a solid form thereof. The present disclosure also relates to processes for its preparation, as well as a medicament and a pharmaceutical composition comprising it. The present disclosure further concerns the anhydrate crystalline Form 1 of compound of formula (1) for use as a medicine and more particularly in the treatment of Alzheimer disease, multiple sclerosis, and amyotrophic lateral sclerosis (ALS).

ANTIBODY DRUG CONJUGATES TARGETING PROTEINOPATHIES, AND USES THEREOF

NºPublicación:  WO2025134068A1 26/06/2025
Solicitante: 
AC IMMUNE SA [CH]
AC IMMUNE SA

Resumen de: WO2025134068A1

The invention provides antibody conjugate compositions comprising an antibody targeting a pathological protein covalently attached to one or more brain penetrant, pathological protein binding small molecule entities by a linker. The invention further provides methods of treating neurodegenerative diseases and disorders such as Alzheimer's disease with the antibody conjugate compositions.

IMPROVED BRAIN ARCHITECTURE AND BIOMARKERS IN ALZHEIMER'S DISEASE WITH MESENCHYMAL STEM CELLS

NºPublicación:  WO2025137077A1 26/06/2025
Solicitante: 
LONGEVERON INC [US]
LONGEVERON, INC

Resumen de: WO2025137077A1

Compositions and methods are disclosed herein for the treatment of Alzheimer's disease with allogeneic mesenchymal stem cells. The methods of treatment involve the administration of a composition of allogeneic mesenchymal stem cells to a subject in need thereof, wherein the efficacy of the treatment methods can be determined through the measurement of specific biomarkers and improved cognitive function and/or quality of life.

COMPOUNDS AND METHODS TO TREAT ALZHEIMER'S DISEASE

NºPublicación:  WO2025136887A1 26/06/2025
Solicitante: 
THE TRUSTEES OF INDIANA UNIV [US]
THE TRUSTEES OF INDIANA UNIVERSITY

Resumen de: WO2025136887A1

The present disclosure describes compounds and methods for disrupting the amyloid cascade.

PHARMACEUTICAL COMPOSITION FOR PREVENTING OR TREATING DEGENERATIVE BRAIN DISEASE COMPRISING NATURAL KILLER CELL

NºPublicación:  US2025205281A1 26/06/2025
Solicitante: 
THERABEST CO LTD [KR]
Therabest Co., Ltd
KR_20230140518_PA

Resumen de: US2025205281A1

The present invention relates to a pharmaceutical composition for preventing and treating a degenerative brain disease, specifically dementia, comprising an activated natural killer cell as an active ingredient. The pharmaceutical composition comprising a mouse-derived activated natural killer cell as an active ingredient, according to the present invention, regulates the activity of microglial cells and inhibits the deposition of amyloid β plaques. In addition, the pharmaceutical composition showed an excellent effect on cognitive function improvement in an animal model of dementia. Furthermore, when a human peripheral blood mononuclear cell-derived natural killer cell and a human induced pluripotent stem cell-derived natural killer cell were activated, it was confirmed that the expression of some genes involved in restoring the activity of microglial cells was similar to or higher than that of a mouse-derived activated natural killer cell. Therefore, the pharmaceutical composition of the present invention can be effectively used for preventing or treating a degenerative brain disease, such as dementia, Parkinson's disease, and Huntington's disease, and improving cognitive impairment.

(2-(4-(1-(BENZODTHIAZOL-5-YL)ETHYL)PIPERAZIN-1 -YL)PYRIMIDIN-5-YL)(IMINO)(METHYL)-LAMDA6-SULFANONE FOR USE IN THE TREATMENT OF COLITIS, PARKINSON DISEASE, TAUOPATHY, ALS AND ALZHEIMER'S DISEASE

NºPublicación:  WO2025131275A1 26/06/2025
Solicitante: 
ASCENEURON SA [CH]
ASCENEURON SA

Resumen de: WO2025131275A1

The present invention relates to pharmaceutical compositions and medicaments comprising the compound of formula (I); or a stereoisomer, tautomer, pharmaceutically usable solvate or salt thereof, and dosage regimens for the administration thereof to human patients.

SULFOPROPANOIC ACID DERIVATIVES FOR TREATING NEURODEGENERATIVE DISORDERS

NºPublicación:  AU2025204068A1 26/06/2025
Solicitante: 
ALZHEON INC
Alzheon, Inc
AU_2025204068_A1

Resumen de: AU2025204068A1

Abstract Provided herein are sulfopropanoic acid derivatives or pharmaceutically acceptable salts thereof, for treating a disease characterized by amyloid and amyloid-like aggregates, e.g., Alzheimer's disease.

Intrathecal Delivery of Recombinant Adeno-Associated Virus 9

NºPublicación:  US2025205368A1 26/06/2025
Solicitante: 
NATIONWIDE CHILDRENS HOSPITAL [US]
OHIO STATE INNOVATION FOUND [US]
Nationwide Children's Hospital,
Ohio State Innovation Foundation
ES_2985917_T3

Resumen de: US2025205368A1

The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated. for example. for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders.

SYSTEMS, METHODS, AND COMPOSITIONS FOR RESCUING PROTEIN MISFOLDING

NºPublicación:  US2025205305A1 26/06/2025
Solicitante: 
THE TRUSTEES OF COLUMBIA UNIV IN THE CITY OF NEW YORK [US]
The Trustees of Columbia University in the City of New York
WO_2023147473_PA

Resumen de: US2025205305A1

The present disclosure provides to systems, methods, and compositions for rescuing protein misfolding and preventing protein aggregation. Particularly the present disclosure provides methods and compositions comprising DNAJB6 or variants thereof, or polynucleotides encoding DNAJB6 or variants thereof. The present disclosure also provides methods for treating protein misfolding and/or protein aggregation diseases (e.g., multiple amyotrophic lateral sclerosis and frontotemporal dementia) by administering the systems or compositions to a subject in need thereof.

MHC IB-MEDIATED ALPHA-SYNUCLEIN-SPECIFIC TOLERANCE INDUCTION AS A NOVEL TREATMENT FOR PARKINSON'S DISEASE

NºPublicación:  US2025205321A1 26/06/2025
Solicitante: 
JULIUS MAXIMILIANS UNIV WUERZBURG [DE]
JULIUS MAXIMILIANS UNIV WURZBURG [DE]
Julius-Maximilians-Universit\u00E4t W\u00FCrzburg,
Julius-Maximilians-Universitat Wurzburg
JP_2025514872_A

Resumen de: US2025205321A1

The present invention relates to therapeutical uses of non-classical human major histocompatibility complex (MHC) molecules (also named MHC class Ib molecules) in combination with peptide antigens for the treatment of Parkinson's disease. The invention more specifically relates to recombinant polypeptides comprising peptide antigens and one or more domains of a non-classical MHC class Ib molecule. The invention also relates to methods of producing such recombinant polypeptides, pharmaceutical compositions comprising the same, as well as their uses for treating Parkinson's disease.

COMPOSITION CONTAINING OSMOTIN PROTEIN AS ACTIVE INGREDIENT FOR PREVENTION, ALLEVIATION, OR TREATMENT OF PARKINSON'S DISEASE

Nº publicación: EP4573926A1 25/06/2025

Solicitante:

NAT UNIV GYEONGSANG IACF [KR]
Industry-Academic Cooperation Foundation Gyeongsang National University

EP_4573926_A1

Resumen de: EP4573926A1

The present invention relates to a composition for preventing, ameliorating, or treating Parkinson's disease comprising osmotin protein as effective component, and, by having the effects of alleviating behavior and motor skill deficits induced by MPTP/α-synuclein, protecting dopaminergic neurons, regulating the expression level of neuroinflammation-related proteins, inhibiting apoptotic cell death induced by MPTP/α-synuclein, alleviating cell damage caused by overexpression of α-synuclein (A53T), reducing the accumulation of α-synuclein caused by activation of AMPK and subsequent autophagy, regulating the dendritic complex structure, increasing the spine density in pyramidal neurons, alleviating the cognitive deficits, and restoring the expression of synaptic markers, the osmotin protein can be advantageously used for a functional health food or a pharmaceutical product for preventing, ameliorating, or treating Parkinson's disease.

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