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FUSED BICYCLIC HETEROARYL AMIDE COMPOUND AS PROTEIN AGGREGATION INHIBITOR

NºPublicación:  US2025074902A1 06/03/2025
Solicitante: 
SHANGHAI JINGXIN BIOMEDICAL CO LTD [CN]
ZHEJIANG JINGXIN PHARMACEUTICAL CO LTD [CN]
SHANGHAI JINGXIN BIOMEDICAL CO., LTD,
ZHEJIANG JINGXIN PHARMACEUTICAL CO., LTD

Resumen de: US2025074902A1

Provided are a fused bicyclic heteroaryl amide compound as a protein aggregation inhibitor, as well as the use of such compound in the treatment or prevention of neurodegenerative diseases characterized by protein aggregation, such as Alzheimer's disease, Parkinson's disease, frontotemporal dementia, dementia with Lewy bodies, Parkinson's disease dementia, multiple system atrophy, amyotrophic lateral sclerosis, Huntington's disease, and cancer.

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

NºPublicación:  US2025073341A1 06/03/2025
Solicitante: 
KOREA RESEARCH INSTITUTE OF CHEMICAL TECH [KR]
UBIX THERAPEUTICS INC [KR]
KOREA RESEARCH INSTITUTE OF CHEMICAL TECHNOLOGY,
UBIX THERAPEUTICS, INC

Resumen de: US2025073341A1

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.

BENZIMIDAZOLE DERIVATIVES AS MPGES-1 INHIBITORS

NºPublicación:  WO2025048759A1 06/03/2025
Solicitante: 
ICOSYN THERAPEUTICS SAGLIK TEKNOLOJILERI ANONIM SIRKETI [TR]
ICOSYN THERAPEUTICS SAGLIK TEKNOLOJILERI ANONIM SIRKETI
WO_2024112287_PA

Resumen de: WO2025048759A1

The present invention pertains to benzimidazole derivatives of formula (I) and their inhibitory activity against mPGES-1. It also encompasses pharmaceutical compositions that include these benzimidazole derivatives and their application in treating mPGES-1-mediated diseases. The purpose of this invention is to create novel compounds that inhibit inducible mPGES-1, an enzyme responsible for catalyzing the final step in the biosynthesis of PGE2 from AA. These compounds are intended for the treatment of various inflammatory conditions, including but not limited to Parkinson's disease, autoimmune diseases, allergic disorders, rhinitis, coronary heart disease, ulcers, osteoarthritis, rheumatoid arthritis, systemic sclerosis, periodontitis, colon cancer, inflammatory bowel disease, cutaneous sclerosis, neuropathic pain, inflammation, pain, fever, migraine, chronic pain, acute pain, headache, asthma, pulmonary fibrosis, fibromyalgia, dysmenorrhea, atherosclerosis, gout, arthritis, rheumatic fever, multiple sclerosis, Hodgkin's disease.

HYDROPHOBICALLY INTERSPACED CHARGED PEPTIDES FOR PREVENTING OR TREATING APOE AND/OR AMYLOID BETA PATHOLOGIC INTERACTION IN ALZHEIMER'S DISEASE AND RELATED DEMENTIAS

NºPublicación:  WO2025049979A1 06/03/2025
Solicitante: 
NEW YORK UNIV [US]
NEW YORK UNIVERSITY

Resumen de: WO2025049979A1

Disclosed herein are peptides or peptoids including from 8 to 20 amino acids, including a plurality of hydrophobic amino acids and a plurality of charged amino acids, wherein spacing of the plurality of hydrophobic amino acids and the plurality of charged amino acids aligns with the spacing of hydrophobic amino acids and oppositely charged amino acids in an ApoE protein or polypeptide. Also disclosed are pharmaceutical compositions that include a peptide or peptoid as disclosed, as well as various methods of using the peptides or peptoids, and such pharmaceutical compositions. These methods include blocking Aβ/ApoE interaction, inhibiting Aβ protein oligomerization, and inhibiting amyloid plaque and/or tau aggregate formation, as well as the treatment of Alzheimer's Disease and other tauopathies.

METHODS AND COMPOSITIONS FOR ENGINEERED DA NEURONAL CELLS

NºPublicación:  WO2025049807A1 06/03/2025
Solicitante: 
RYNE BIOTECHNOLOGY INC [US]
RYNE BIOTECHNOLOGY INC

Resumen de: WO2025049807A1

Disclosed are novel strategies for the treatment of patients with Parkinson's disease and other secondary Parkinsonian disorders. Disclosed are DA neuronal cells that have been modified in vitro with a genetic insertion of GDNF. The GDNF coding sequence is inserted under the transcriptional control of a promotor such that secreted proteins are made and taken up by the endogenous cells after the administered engineered cells have matured to a Neuronal Mature Cell Type to promote survival of endogenous neurons. Also disclosed are DA neuronal cells that have been modified with a genetic insertion of GBA in vitro and are hemizygous null for SNCA. The GBA coding sequence is inserted under the transcriptional control of a ubiquitous promotor and the secreted proteins are made and taken up by the graft and endogenous cells immediately upon transplantation to promote long-term graft integrity.

PHARMACEUTICAL COMPOSITION COMPRISING HTRA ACTIVATOR AS ACTIVE INGREDIENT FOR PREVENTING OR TREATING DEGENERATIVE DISEASES, AND METHOD FOR SCREENING HTRA ACTIVITY MODULATOR

NºPublicación:  WO2025048409A1 06/03/2025
Solicitante: 
THE ASAN FOUND [KR]
UNIV OF ULSAN FOUNDATION FOR INDUSTRY COOPERATION [KR]
SUNGSHIN WOMENS UNIV INDUSTRY ACADEMIC COOPERATION FOUNDATION [KR]
\uC7AC\uB2E8\uBC95\uC778 \uC544\uC0B0\uC0AC\uD68C\uBCF5\uC9C0\uC7AC\uB2E8,
\uC6B8\uC0B0\uB300\uD559\uAD50 \uC0B0\uD559\uD611\uB825\uB2E8,
\uC131\uC2E0\uC5EC\uC790\uB300\uD559\uAD50 \uC5F0\uAD6C \uC0B0\uD559\uD611\uB825\uB2E8

Resumen de: WO2025048409A1

The compound represented by chemical formula 1, a stereoisomer thereof, a solvate thereof, a hydrate thereof, or a pharmaceutically acceptable salt thereof, according to one aspect, can be used as a prophylactic or therapeutic agent for degenerative diseases such as age-related macular degeneration (AMD) in which lack of HTRA1 activity is a factor in worsening the disease, cerebral amyloid angiopathy, hereditary stroke (CADASIL), and Alzheimer's disease (AD), and can be used as an adjuvant for efficiently and accurately screening an HTRA activity modulator without a change in temperature or addition of a substrate.

COMPOSITION FOR PREVENTING OR TREATING ALZHEIMER'S DISEASE, CONTAINING NOVEL COMPOUND

NºPublicación:  WO2025048007A1 06/03/2025
Solicitante: 
PRG S&TECH INC [KR]
(\uC8FC)\uD53C\uC54C\uC9C0\uC5D0\uC2A4\uC564\uD14D

Resumen de: WO2025048007A1

The present invention relates to use of a novel compound for preventing, alleviating or treating Alzheimer's disease, and the novel compound exhibits an inhibitory effect on tau protein aggregation. In addition, it was identified that presenilin 1 was reduced by treatment using the novel compound. Therefore, the novel compound can be effectively used in the development of a therapeutic agent for Alzheimer's disease.

COMPOSITIONS AND METHODS FOR ALTERING AMYLOID PRECURSOR PROTEIN (APP) PROCESSING

NºPublicación:  US2025074951A1 06/03/2025
Solicitante: 
DREXEL UNIV [US]
Drexel University
US_2019309026_PA

Resumen de: US2025074951A1

The present invention relates to the discovery of compositions and methods for altering Amyloid Precursor Protein (APP) processing. Alerting APP processing is aids the treatment of neuropathological disorders such as those associated with HIV infection and Alzheimer's disease (AD). The invention includes fusion protein constructs that include an effector protein and an HSV US9 protein or functionally active fragment thereof that reduce the amount of amyloid □-protein produced in a cell.

GENE THERAPY VECTOR FOR TREATING PARKINSON'S DISEASE AND USE THEREOF

NºPublicación:  WO2025045033A1 06/03/2025
Solicitante: 
KANGLIN BIOTECH HANGZHOU CO LTD [CN]
\u5EB7\u9716\u751F\u7269\u79D1\u6280\uFF08\u676D\u5DDE\uFF09\u6709\u9650\u516C\u53F8
CN_117106825_A

Resumen de: WO2025045033A1

Provided are a gene therapy vector for treating Parkinson's disease and a use thereof. Specifically, provided is an adeno-associated virus (AAV) vector for treating Parkinson's disease, which can simultaneously express functional tyrosine hydroxylase (TH), GTP-cyclohydrolase 1 (GCH1) and aromatic amino acid decarboxylase (AADC) to promote dopamine synthesis. Also provided are an AAV virus particle containing the AAV vector, a composition containing the AAV vector or the AAV virus particle, and uses of the AAV vector, the AAV virus particle and the composition in the preparation of drugs for preventing or treating Parkinson's disease.

DEVELOPMENT AND USE OF THERAPEUTIC AGENT FOR ALZHEIMER'S DISEASE

NºPublicación:  WO2025044931A1 06/03/2025
Solicitante: 
KEYMED BIOSCIENCES CHENGDU CO LTD [CN]
\u5EB7\u8BFA\u4E9A\u751F\u7269\u533B\u836F\u79D1\u6280\uFF08\u6210\u90FD\uFF09\u6709\u9650\u516C\u53F8

Resumen de: WO2025044931A1

Provided is an antibody that binds to β-amyloid (Aβ), or an antigen-binding fragment thereof. Further provided are a nucleic acid encoding the antibody or the antigen-binding fragment thereof, a cell comprising the antibody or the antigen-binding fragment or nucleic acid thereof, a pharmaceutical composition, a kit, and the use of the antibody or the antigen-binding fragment thereof in the preparation of a drug used for treating or preventing a disease caused by abnormal accumulation or deposition of Aβ in subjects.

TREATMENT REGIMENS FOR PARKINSON'S DISEASE

NºPublicación:  AU2023326620A1 06/03/2025
Solicitante: 
BIAL PORTELA & CA S A
BIAL - PORTELA & CA, S.A
AU_2023326620_A1

Resumen de: AU2023326620A1

A method of treating Parkinson's disease in a patient who is receiving N doses of levodopa per day to provide a total daily dose of X mg of levodopa and who is starting to experience motor fluctuations or starting to show signs of "wearing-off", the treatment comprising administering more than N doses of levodopa per day to provide a total daily dose of X mg of levodopa and administering a single daily dose of Y mg of opicapone, wherein X is from 100 to 1000, N is from 2 to 10 and Y is from 25 to 50.

COMPOSITIONS AND METHODS FOR PREVENTION OF COGNITIVE DECLINE CAUSED BY DEGENERATIVE DISEASES

NºPublicación:  AU2023312980A1 06/03/2025
Solicitante: 
ANAVEX LIFE SCIENCES CORP
ANAVEX LIFE SCIENCES CORP
AU_2023312980_A1

Resumen de: AU2023312980A1

The present disclosure provides methods, compositions and kits for prophylactic treating or preventing a degenerative disease related to amyloid. The compositions and kits comprise Sigma-1 receptor agonist, an allosteric sigma agonist, and/or a dual agonist of Sigma-1 and M1. The compositions and kits elicit effects of delaying the onset, deterring the progress, and/or diminish the likelihood of the degenerative disease, such as deterring the memory loss and/or cognition decline in Alzheimer's disease.

COMPOSITIONS AND METHODS FOR TREATING HUNTINGTON’S DISEASE

NºPublicación:  US2025074973A1 06/03/2025
Solicitante: 
ANNEXON INC [US]
Annexon, Inc
JP_2025503545_A

Resumen de: US2025074973A1

The present disclosure relates generally to methods of treating Huntington's disease in a subject in need thereof. The method comprises determining that the subject has an elevated level of C4a or an elevated C4a/C4 ratio; and administering to the subject an inhibitor of the classical complement pathway.

NICOTINAMIDE RIBOSIDE DOSAGE REGIMEN FOR TREATING PARKINSON'S DISEASE

NºPublicación:  EP4514362A1 05/03/2025
Solicitante: 
VESTLANDETS INNOVASJONSSELSKAP AS VIS [NO]
VESTLANDETS INNOVASJONSSELSKAP AS (VIS)
WO_2023209010_PA

Resumen de: WO2023209010A1

The present invention generally relates to the treatment of Parkinson's disease (PD) in humans. More particularly, it relates to nicotinamide riboside (NR) for use in a method for treatment of Parkinson's disease in a human subject characterized by a particular dosage regimen; pharmaceutical compositions; and dosage forms, which can be for use in or as treatment of Parkinson's disease.

BENZOTHIOPHENE COMPOUND

NºPublicación:  EP4516792A1 05/03/2025
Solicitante: 
KYOTO PHARMA IND [JP]
Kyoto Pharmaceutical Industries, Ltd
TW_202345792_PA

Resumen de: EP4516792A1

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 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.

CRYSTAL FORM OF BLARCAMESINE HYDROCHLORIDE, METHOD FOR PREPARING SAME, AND USE THEREOF

NºPublicación:  EP4516782A1 05/03/2025
Solicitante: 
CRYSTAL PHARMACEUTICAL SUZHOU CO LTD [CN]
MOEHS IBERICA SL [ES]
Crystal Pharmaceutical (Suzhou) Co., Ltd,
Moehs Iberica SL
EP_4516782_PA

Resumen de: EP4516782A1

The present disclosure relates to novel crystalline forms of Blarcamesine hydrocholoride (hereinafter referred to as "compound I"), a method for preparing same, a pharmaceutical composition comprising the crystalline forms, and use of the crystalline forms in preparing a Sigma-1 receptor agonist and a medicament for treating Rett syndrome, Alzheimer's disease, and Parkinson's disease dementia.

SUBSTITUTED PYRAZOLO1,5-APYRIMIDINES AND THEIR USE IN THE TREATMENT OF MEDICAL DISORDERS

NºPublicación:  US2025066364A1 27/02/2025
Solicitante: 
BIAL R&D INVEST S A [PT]
Bial - R&D Investments, S.A

Resumen de: US2025066364A1

The invention provides substituted pyrazolo1,5-apyrimidine and related organic compounds, compositions containing such compounds, medical kits, and methods for using such compounds and compositions to treat medical disorders, e.g., Gaucher disease, Parkinson's disease, Lewy body disease, dementia, or multiple system atrophy, in a patient. Exemplary substituted pyrazolo1,5-apyrimidine compounds described herein include 5,7-dimethyl-N-phenylpyrazolo1,5-apyrimidine-3-carboxamide compounds and variants thereof.

Modulators of Chromosome 9 Open Reading Frame 72 Gene Expression and Uses Thereof

NºPublicación:  US2025066434A1 27/02/2025
Solicitante: 
SANGAMO THERAPEUTICS INC [US]
Sangamo Therapeutics, Inc
BR_112021020868_PA

Resumen de: US2025066434A1

The present disclosure provides compositions and methods for modulating transcription of mutant C9orf72 gene alleles in patients in need thereof, including patients having a C9orf72-related disease such as amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD).

Modulators of Chromosome 9 Open Reading Frame 72 Gene Expression and Uses Thereof

NºPublicación:  US2025066433A1 27/02/2025
Solicitante: 
SANGAMO THERAPEUTICS INC [US]
Sangamo Therapeutics, Inc
BR_112021020868_PA

Resumen de: US2025066433A1

The present disclosure provides compositions and methods for modulating transcription of mutant C9orf72 gene alleles in patients in need thereof, including patients having a C9orf72-related disease such as amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD).

SELECTIVE LIGANDS FOR TAU AGGREGATES

NºPublicación:  US2025066330A1 27/02/2025
Solicitante: 
OXIANT DISCOVERY AB [SE]
Oxiant Discovery AB
WO_2024094816_PA

Resumen de: US2025066330A1

The invention provides a compound of formula (1), or a pharmaceutically acceptable salt, ester or carbamate thereof, or a salt of such an ester or carbamate,wherein either:R1 is OH, and R2 is H; orR1 is H, and R2 is OH.The invention further provides uses of the compounds of formula (I) and compositions comprising compounds of formula (I), including the use of such compounds for the detection of tau deposits, and the use of such compounds and compositions as diagnostic agents in the diagnosis or monitoring of the progression of a disease or disorder such as Alzheimer's disease, progressive supranuclear palsy and corticobasal degeneration, or for the prevention or treatment of a disease or disorder such as Alzheimer's disease, progressive supranuclear palsy and corticobasal degeneration.

NOVEL KAPPA OPIOID LIGANDS

NºPublicación:  US2025066336A1 27/02/2025
Solicitante: 
THE SCRIPPS RES INSTITUTE [US]
The Scripps Research Institute

Resumen de: US2025066336A1

The invention provides novel ligands of Kappa (κ) opioid receptors, such as can be used to modulate a Kappa opioid receptor. Methods of synthesis and methods of use are also provided. Compounds of the invention can be used therapeutically in the treatment of dissociative disorders or pain, or to provide neuroprotection, or to induce diuresis, or to modulate the immune system, or for treatment of one or more of an affective disorders comprising depression or stress/anxiety; an addictive disorder; alcoholism, epilepsy; a cognition deficiency; schizophrenia; Alzheimer's disease; or pain.

Engineered Guide RNAs and Polynucleotides

NºPublicación:  US2025066774A1 27/02/2025
Solicitante: 
SHAPE THERAPEUTICS INC [US]
Shape Therapeutics Inc
JP_2024543208_PA

Resumen de: US2025066774A1

Disclosed herein are engineered latent guide RNAs targeting LRRK2 and compositions comprising the same for treatment of diseases or conditions (e.g. Parkinson's Disease) in a subject. Also disclosed herein are methods of treating diseases or conditions (e.g. Parkinson's Disease) in a subject by administering engineered latent guide RNAs or pharmaceutical compositions described herein.

TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS USING PKC ACTIVATORS

NºPublicación:  US2025064776A1 27/02/2025
Solicitante: 
SYNAPTOGENIX INC [US]
Synaptogenix, Inc

Resumen de: US2025064776A1

A method for treating or preventing amyotrophic lateral sclerosis (ALS) or other motor neuron disease in a subject, the method comprising administering to the subject a PKC activating compound (e.g., a bryostatin, such as bryostatin-1, or a bryolog) in a therapeutically effective amount to treat or prevent the motor neuron disease by activating PKC in the subject. The ALS may be, for example, classical ALS, primary lateral sclerosis (PLS), progressive muscular atrophy (PMA), and progressive bulbar palsy (PBP). The PKC activating compound may be administered at an initial loading dose of about 15, 24, or 48 micrograms weekly in the first one week or consecutive two or three weeks, followed by doses of about 12, 20, or 40 micrograms alternately every two or three weeks for at least 4, 5, 6, 8, 10, 12, 14, 16, 18, 20, 24, or 30 total weeks.

L-N-isobutyryl cysteine (L-NIBC)-bound gold cluster Au18(L-NIBC)14, compositions and applications thereof

NºPublicación:  US2025064970A1 27/02/2025
Solicitante: 
SHENZHEN PROFOUND VIEW PHARMACEUTICAL TECH CO LTD [CN]
SHENZHEN PROFOUND VIEW PHARMACEUTICAL TECHNOLOGY CO., LTD
JP_2025503046_A

Resumen de: US2025064970A1

L-N-isobutyryl cysteine (L-NIBC)-bound gold cluster Au18(L-NIBC)14, its use for treating multiple sclerosis, Alzheimer's disease (AD), liver cirrhosis, diabetes, Parkinson's disease (PD) or glaucoma, and the use of it for manufacturing a medicament for treatment of multiple sclerosis, Alzheimer's disease (AD), liver cirrhosis, diabetes, Parkinson's disease (PD) or glaucoma. Composition and methods for treating multiple sclerosis, Alzheimer's disease (AD), liver cirrhosis, diabetes, Parkinson's disease (PD) or glaucoma.

COMPOSITION FOR TREATING ALZHEIMER'S DISEASE INCLUDING MIXED EXTRACT OF PINELLIA TUBER, ZIZYPHI FRUCTUS, GLYCYRRHIZA RADIX, GINSENG RADIX, ZINGIBERIS RHIZOMA, SCUTELLARIAE RADIX, AND COPTIDIS RHIZOMA AS ACTIVE INGREDIENT

Nº publicación: US2025064879A1 27/02/2025

Solicitante:

KOREA INST OF ORIENTAL MEDICINE [KR]
DAEGU GYEONGBUK INSTITUTE OF SCIENCE AND TECH [KR]
KOREA INSTITUTE OF ORIENTAL MEDICINE,
DAEGU GYEONGBUK INSTITUTE OF SCIENCE AND TECHNOLOGY

JP_2024535112_PA

Resumen de: US2025064879A1

The present invention relates to a composition for treating, improving, or preventing Alzheimer's disease caused by ApoE4 gene mutation, the composition including Banha-Sasim-Tang as an active ingredient.The composition including Banha-Sasim-Tang as an active ingredient of the present invention and a treatment method exhibit excellent effects of reducing amyloid beta proteins and inhibiting deposition thereof, which are specific to Alzheimer's disease caused by ApoE4 gene mutation, thereby having high applicability as a specific composition for preventing, improving, or treating Alzheimer's disease caused by ApoE4 gene mutation.

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