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Pseudo-ginseng-derived exosome-like nano-vesicle as well as preparation method and application thereof

Absstract of: CN122398874A

The invention relates to a high-purity pseudo-ginseng source exosome-like nano-vesicle as well as a preparation method and application thereof in improvement of Parkinson's depression. The nano vesicle has a typical double-layer lipid membrane structure and presents a'teacup 'or'biconcave dish' appearance; the particle size distribution is concentrated, the average particle size is 55-70 nm, the Zeta potential is-30 mV to-45 mV, and the particle concentration is up to 1.0 * 10 < 12 > Particle/mL or above; the chemical components are rich in triterpenoids (accounting for about 16.2%), fatty acids and conjugates thereof. Experiments prove that the pseudo-ginseng exosome provided by the invention can significantly improve dyskinesia and depression-like behaviors of model mice of Parkinson's disease accompanied with depression induced by MPTP (MPTP-associated LPS). The nano vesicle provided by the invention is high in purity, good in stability and strong in biological activity, and has a wide application prospect in the fields of nervous system disease treatment and drug delivery.

Phenolic compound with multi-target neuroprotective activity, production strain and application

Absstract of: CN122404149A

The invention discloses a phenolic compound with multi-target neuroprotection activity and a production strain and application of the phenolic compound with the multi-target neuroprotection activity, according to the phenolic compound with the multi-target neuroprotection activity and the production strain, a phenolic natural product with a novel structure is separated from plant endophytic fungi Diamorpha searleiZMU-20-2 for the first time; the compound has specific triple biological activities of oxidation resistance, monoamine oxidase (MAO-A/MAO-B) dual inhibition activity and butyrylcholine esterase (BChE) selective inhibition activity. The compound is novel in structure and good in biocompatibility, can be used as a drug lead compound or a bioactive reference substance, is used for research, development and screening of drugs related to neurodegenerative diseases (such as Alzheimer's disease, Parkinson's disease and Huntington's disease), effectively solves the technical bottleneck of poor curative effect of the existing single-target drugs, and has broad application prospects. The invention has important scientific research value and wide clinical application prospect.

Substituted pyrrolo 2, 3-d pyrimidines, preparation thereof and therapeutic use thereof

Absstract of: CN122422324A

Compounds having Formula (I) or pharmaceutically acceptable salts thereof are disclosed. Also disclosed are medicaments and pharmaceutical compositions comprising the compounds of Formula (I) for use in the treatment of degenerative diseases of the nervous system, such as Parkinson's disease. Further disclosed are solid forms of a compound of formula (I-a) characterized by crystalline Form A, as well as pharmaceutical compositions comprising the solid forms, and for use in the treatment of degenerative diseases of the nervous system. (I)

Tau-targeting artificial microRNA

Absstract of: CN122422508A

Provided herein are artificial microRNA (miRNA) molecules for use in the treatment of tauopathies. In some embodiments, these miRNA molecules target the expression of a Tau protein. Further provided herein are expression constructs, vectors (e.g., rAAVs), cells, viral particles, and pharmaceutical compositions containing these artificial miRNA molecules. Yet further provided herein are methods and kits related to the use of these miRNA molecules, e.g., for the treatment of tauopathy, e.g., ovarian atherosclerosis. Comprising Alzheimer's disease, progressive suprakaryotic paralysis, corticobasal degeneration, frontotemporal dementia with Parkinson syndrome-17, Picker's disease, silver granulopathy, globular glial tauopathy, chronic traumatic encephalopathy and post-encephalitis Parkinson's syndrome.

SIRNA INHIBITING EXPRESSION OF AMYLOID PRECURSOR PROTEIN (APP) GENE, DRUG, AND USE

Absstract of: US20260201383A1

The present invention provides siRNA, peptide oligonucleotide drugs, and their applications for suppressing the expression of the amyloid precursor protein (APP) gene in human cells. The siRNA exhibits potent activity in inhibiting APP expression. Through appropriate modifications, its ability to silence the target is enhanced while reducing off-target activity. The described siRNA and its conjugates hold promise for clinical application in the prevention and treatment of diseases associated with the APP target, including cerebral amyloid angiopathy (CAA), early-onset familial Alzheimer's disease (EOFAD), or Alzheimer's disease (AD).

CRYSTALLINE FORMS OF 2-(TERT-BUTOXY)-4-(3-METHYL-3-(5-(METHYLSULFONYL)ISOINDOLIN-2-YL)BUTYL)PHENOL FUMARATE SALT

Absstract of: US20260200848A1

The present disclosure describes crystalline forms of 2-(tert-butoxy)-4-(3-methyl-3-(5-(methylsulfonyl)isoindolin-2-yl)butyl)phenol fumarate salts and pharmaceutical compositions of same. Also described are methods of using the crystalline forms treating Alzheimer's Disease, Dementia with Lewy Bodies and Dry age-related macular degeneration in a subject in need thereof, comprising administering the crystalline form to the subject. Methods of making the solid forms are also described.

COMPOSITION FOR PREVENTING OR TREATING ALZHEIMER'S DISEASE

Absstract of: WO2026151017A1

The compound represented by chemical formula IA according to the present invention is useful for the prevention, alleviation, and treatment of degenerative brain diseases.

THERAPEUTIC METHODS AND USES FOR EPERTINIB

Absstract of: WO2026148417A1

Disclosed herein are methods of using epertinib in the treatment of neurological conditions such as ALS.

GLS1 DIAGNOSTIC METHODS

Absstract of: WO2026151983A1

Aspects of the disclosure relate to compositions and methods for modulating levels, transcription, splicing, and/or translation of one or more RNA transcripts (e.g., mRNA transcripts) in a cell or subject. The disclosure is based, in part, on methods of identifying a subject of having or being at risk of developing a disease or disorder associated with dysregulated glutamine levels, such as a subject having a mutation that affects glutaminase (GLS1), which is an enzyme responsible for producing glutamate from glutamine. In some embodiments, compositions and methods of the disclosure are useful for treating a psychiatric disorder (e.g., depression (DEP), major depressive disorder (MDD), bipolar disorder (e.g., BPD1, BPD2), mania (MAN), psychosis (PSY), schizophrenia (SCZ), schizoaffective disorder (SZA), or post-traumatic stress disorder (PTSD)) and/or a disease associated with dementia, such as Alzheimer's disease.

GENE THERAPY FOR LEVODOPA-INDUCED DYSKINESIA IN PARKINSONS DISEASE

Absstract of: US20260201390A1

The present disclosure provides compositions and methods for the treatment of levodopa-induced dyskinesia (LID) and Parkinson's disease (PD) therewith. In particular, the present disclosure provides methods for treating LID in PD by inhibiting (e.g., expression of) Muscarinic M1 acetylcholine receptor (M1Rs), for example, in indirect pathway spiny projection neurons (iSPNs) to attenuate LID without compromising of the treatment with levodopa (e.g., boosting the efficacy of levodopa treatment).

DEUTERATED HUPERZINE A COMPOUND OR PHARMACEUTICALLY ACCEPTABLE SALT THEREOF, AND PREPARATION METHOD THEREFOR AND USE THEREOF

Absstract of: WO2026148695A1

The present invention relates to the technical field of medicines, and specifically relates to a deuterated huperzine A compound or a pharmaceutically acceptable salt thereof, and a preparation method therefor and a use thereof. The structure of the deuterated huperzine A compound is as follows, wherein R1-R9 are each independently H or D, and are not both H. The present invention has the advantages of high chemical stability, long metabolic half-life, low toxicity, significant synergistic effect with bremelanotide in the treatment of Alzheimer's disease, and ability to reduce the toxicity of bremelanotide.

SMALL MOLECULES FOR THE RESCUE OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) AND FRONTOTEMPORAL DEGENERATION (FTD) BY TARGETING C9ORF72 HEXANUCLEOTIDE G-QUADRUPEXES

Absstract of: US20260199329A1

0000 The subject invention pertains to a composition comprising a series of compounds selectively targeting G-quadruplexes (G4s) formed by the GGGGCC (G4C2) hexanucleotide repeats (HREs) of C9orf72, (G4C2). The composition comprises compounds G4008, G4009, G4010, G4012, and G4013, or a pharmaceutically acceptable salt, solvate, or hydrate thereof and at least one pharmaceutically acceptable carrier and/or excipient. The subject invention further pertains to a method for treating amyotrophic lateral sclerosis (ASL) and/or frontotemporal dementia (FTD), comprising administering to the subject an effective amount of the composition. The compounds can ameliorate the pathological signatures of ALS model caused by C9orf72 (G4C2)sequence.

AGENTS, USES AND METHODS FOR THE TREATMENT OF SYNUCLEINOPATHY

Absstract of: US20260201025A1

The invention relates to combinational treatment using a monoclonal anti-alpha-synuclein antibody and an additional medicament. The antibodies can be used for treating a synucleinopathy such as Parkinson's disease (including idiopathic and inherited forms of Parkinson's disease), Diffuse Lewy Body Disease (DLBD), Lewy body variant of Alzheimer's disease (LBV), Combined Alzheimer's and Parkinson disease, pure autonomic failure and multiple system atrophy together with another medicament of the invention.

Novel Molecules for Therapy and Diagnosis

Absstract of: US20260201026A1

0000 The present invention relates to novel molecules that can be employed for the prevention, alleviation, treatment and/or diagnosis of diseases, disorders and abnormalities associated with alpha-synuclein (α-synuclein, A-synuclein, aSynuclein, A-syn, α-syn, aSyn, a-syn) aggregates, including, but not limited to, Lewy bodies and/or Lewy neurites, such as Parkinson's disease, Multiple System Atrophy, Lewy Body dementia (LBD; dementia with Lewy bodies (DLB) (“pure” Lewy body dementia), Parkinson's disease dementia (PDD)) or Diffuse Lewy Body Disease. The invention relates to alpha-synuclein binding molecules, in particular to alpha-synuclein antibodies or an antigen-binding fragment or a derivative thereof and uses thereof. The present molecules can also be used for determining a predisposition to such a disorder, disease or abnormality, monitoring residual disorder, disease or abnormality, or predicting the responsiveness of a patient who is suffering from such a disorder, disease or abnormality to treatment with a certain medicament.

Modulators of chromosome 9 open reading frame 72 gene expression and uses thereof

Absstract of: AU2026204876A1

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). un u n

RECOMBINANT AAV VECTORS FOR TREATING NEURODEGENERATIVE DISORDERS

Absstract of: US20260199527A1

0000 Provided is a recombinant adeno-associated viral (rAAV) vector comprising one or two of (a) to (c): (a) a nucleotide sequence encoding aromatic L-amino acid decarboxylase (AADC), (b) a nucleotide sequence encoding glucocerebrosidase (GBA1); and (c) a nucleotide sequence encoding a neurotrophic factor (NTF), such as cerebral dopamine neurotrophic factor (CDNF) or glial cell derived neurotrophic factor (GDNF), for treating neurodegenerative disorders, particularly Parkinson's disease (PD), Multiple system atrophy (MSA), Gaucher's disease (GD), and other proteinopathies. Also provided herein are viral particles comprising the rAAV vector, a pharmaceutical composition comprising the viral particles, and uses thereof.

BIS-SULFONAMIDE SHIP1 ACTIVATORS

Absstract of: US20260199365A1

Provided are compounds and pharmaceutically acceptable salt, solvate and/or derivative thereof. Further, provided are methods of treating a disease, disorder or condition mediated or treatable by activation of SHIP1 comprising administering a SHIP1 activator compound or a pharmaceutically acceptable salt, solvate or derivative thereof. The compound or a pharmaceutically acceptable salt, solvate or derivative thereof may be used in the treatment of SHIP1 mediated disease, disorder or conditions including inflammatory bowel disease (IBD), Crohn′ disease, COPD, ulcerative colitis, arthritis, and Alzheimer's Disease.

METHODS FOR TREATING NEURODEGENERATIVE DISORDERS WITH TRAMIPROSATE

Absstract of: US20260199270A1

Provided herein are methods for treating Alzheimer's disease using a combination of tramiprosate, a tramiprosate prodrug or an active tramiprosate metabolite with at least one amyloid plaque clearing agent.

AZAINDAZOLE DERIVATIVES USEFUL AS INHIBITORS OF NOD-LIKE RECEPTOR PROTEIN 3

Absstract of: AU2024400795A1

Novel compounds of Formula (I), and the pharmaceutically acceptable salts thereof, are inhibitors of NLRP3 and may be useful in the treatment, prevention, management, amelioration, control and suppression of diseases mediated by NLPR3. The compounds of the present invention may be useful in the treatment, prevention or management of diseases, disorders and conditions mediated by NLRP3 such as, but not limited to, obesity, gout, pseudogout, CAPS, NASH, MASH, fibrosis, heart failure, idiopathic pericarditis, atopic dermatitis, inflammatory bowel disease, Alzheimer's Disease, Parkinson's Disease, dementia with Lewy bodies (DLB), and traumatic brain injury.

NEUROPROTECTIVE DRUG SUBSTANCE-LOADED NANOMOTOR FORMULATIONS

Absstract of: US20260199232A1

A polymer layer containing a pharmaceutical drug substance-loaded amino group and a nanomotor containing a metal layer for orientation by a magnetic field is used to treat Alzheimer's Disease; Memantine HCl-loaded nanomotors are synthesized with a biocompatible poly-L-lysine polymer whose movement is directly controlled by the magnetic field and targets the brain by crossing the blood brain barrier by providing movement in the magnetic field.

AGONISTS OF TREM2 ACTIVITY

Absstract of: AU2024403764A1

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

Absstract of: AU2024406047A1

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.

METHOD OF PROLONGING THE SURVIVAL OF A SUBJECT WITH ALS

Absstract of: AU2025210321A1

This invention provides a method of prolonging the survival of subjects afflicted with ALS by administering a composition comprising pridopidine or pharmaceutically acceptable salt thereof.

Amyloid precursor protein (APP) RNAi agent compositions and methods of use thereof

Nº publicación: AU2026204572A1 16/07/2026

Applicant:

ALNYLAM PHARMACEUTICALS INC
Alnylam Pharmaceuticals, Inc.

Absstract of: AU2026204572A1

The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the APP gene, as well as methods of inhibiting expression of an APP gene and methods of treating subjects having an APP-associated disease or disorder, such as cerebral amyloid angiopathy (CAA) and early onset familial Alzheimer disease (EOFAD or eFAD), using such dsRNAi agents and compositions. un u n