Absstract of: EP4763987A2
Provided herein are RNAi molecules for treating neurodegenerative synucleinopathies. In some embodiments, the RNAi molecules target expression of alpha-synuclein (SNCA). Further provided herein are expression constructs, vectors (e.g., rAAV), 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 neurodegenerative synucleinopathies including Parkinson's disease, multiple system atrophy, and dementia with Lewy bodies.
Absstract of: CN122255483A
The invention discloses a peptide functionalized cellulose derivative targeting Abeta as well as a preparation method and application thereof, and belongs to the technical field of biological medicines. A beta targeting peptide is connected with hydroxy propyl cellulose through an ester bond to obtain a peptide functionalized cellulose derivative, the peptide functionalized cellulose derivative can be specifically combined with A beta protein through molecular recognition, core-shell structure nano-micelles are formed through self-assembly, A beta oligomers are efficiently packaged, A beta aggregation and fibrosis are inhibited, and the application prospect is wide. Meanwhile, the interaction between A beta and endothelial cell connexin is blocked through the anti-fouling performance, the amyloid protein induced endothelial leakage (APEL) effect is inhibited, and the integrity of the blood brain barrier is protected; besides, the peptide functionalized cellulose derivative has good biocompatibility, can reduce A beta plaque deposition in the brain and improve the cognitive function of AD patients, and provides a novel efficient candidate drug for treatment of the Alzheimer's disease.
Absstract of: CN122251380A
The invention discloses an application of a gamma-aminobutyric acid derivative in treatment of TDP-43 proteinosis, and discloses an application of the gamma-aminobutyric acid derivative with a structure as shown in a formula I or an enantiomer and pharmaceutically acceptable salt thereof in preparation of drugs for prevention and/or treatment of TDP-43 abnormal aggregation related diseases. The TDP-43 abnormal aggregation related diseases are amyotrophic lateral sclerosis, sporadic inclusion body myositis, Parkinson's disease, Alzheimer's disease, frontotemporal dementia and Huntington's disease.
Absstract of: CN122251383A
The invention relates to a novel application of salviarocone C, belongs to the technical field of medicines, and particularly relates to an extraction, separation and purification method of salviarocone C and an application of the salviarocone C in preparation of medicines for treating or preventing Alzheimer's disease. According to the present invention, the salviarocone C is obtained through extraction, separation and purification from Salvia roorowskii Maxim., and the experimental results show that the salviarocone C can significantly inhibit the toxicity of beta-amyloid protein in a caenorhabditis elegans Alzheimer's disease pathological model, such that the compound has the potential of improving the Alzheimer's disease-related pathological change, and the application of the salviarocone C in the treatment of the Alzheimer's disease-related pathological change in the treatment of the caenorhabditis elegans Alzheimer's disease-related pathological change in the treatment of the caenorhabditis elegans Alzheimer's disease-related pathological change is provided. Therefore, the salviarocone C can be used for preparing the pharmaceutical composition for treating the Alzheimer's disease.
Absstract of: CN122251551A
The invention belongs to the technical field of biological medicines, and relates to an application of FcRn inhibitor iagimod in preparation of a medicine for treating Alzheimer's disease, in-vivo experiments prove that APP/PS1 mice are administered through intraperitoneal injection, the administration frequency is once a week, the administration lasts for four weeks, the administration dosage is 10 mg/kg, and the administration time is 1-3 days. According to the present invention, the Iganimod can significantly reduce the APP/PS1 mouse serum IgG level, alleviate brain A beta pathological deposition, and alleviate disease-related neuroinflammation and Tau protein phosphorylation, and the present invention provides a new approach for the treatment of Alzheimer's disease.
Absstract of: CN122251480A
The invention discloses application of meconopsis tinctoria extract in preparation of an anti-Alzheimer's disease medicine, and relates to the technical field of medicine preparation. Comprising the following steps: soaking coarse powder of dried whole herb of artemisia carthamus in a 0.5% hydrochloric acid-70% ethanol solution for 24 hours, performing percolation extraction, collecting percolate, concentrating the percolate under reduced pressure until no alcohol smell exists, diluting with pure water, degreasing with petroleum ether, extracting polyphenol with ethyl acetate, extracting alkaloid with water-saturated n-butyl alcohol, collecting an n-butyl alcohol layer and a water layer, and drying under reduced pressure to obtain the artemisia carthamus extract. The meconopsis tinctoria extract is obtained. The meconopsis carthamus extract disclosed by the invention can simultaneously act on two major core pathological links of the Alzheimer's disease: on one hand, fibrosis aggregation is remarkably inhibited (the inhibition rate is as high as 61.3%), and formation of neurotoxic plaques is prevented; on the other hand, neuroinflammatory response mediated by microglia is effectively inhibited (the NO generation inhibition rate reaches 74.5%), and damage of the inflammatory microenvironment to neurons is reduced.
Absstract of: CN122270559A
Provided herein are expression components for expressing a transgene in a cell, wherein the transgene encodes a GCase polypeptide. Also provided are methods of treating Gaucher disease or GBA-PD. Further provided herein are vectors (e.g., rAAV vectors), viral particles, pharmaceutical compositions, and kits for expressing a GCase polypeptide in an individual in need thereof.
Absstract of: CN122255297A
The invention discloses a fusion protein CHC targeting soluble IL-17RD as well as a preparation method and application of the fusion protein CHC. The fusion protein CHC is formed by connecting and fusing two sIL-17RD neutralizing nano antibodies CQ5 and CQ13 through a connexon and human serum albumin (HSA), the structure of the fusion protein CHC is CQ5-Linker-CHC-Linker-CQ13, and the amino acid sequence of the fusion protein CHC is shown as SEQ ID NO. 1. The invention also provides a gene sequence for coding the fusion protein and an adeno-associated virus AAV-PHP.eB vector containing the gene. The neutralizing nano antibody fusion protein can specifically bind and neutralize the biological function of sIL-17RD, and regulates and controls the steady state of cerebral vascular endothelial cells by directly inhibiting the inflammatory response of the cerebral vascular endothelial cells induced by TNF-alpha and indirectly inhibiting the activation of microglial cells. The pharmaceutical composition disclosed by the invention can be used for treating or preventing neurodegenerative diseases such as Alzheimer's disease and the like, and has the effects of protecting vascular injury, improving learning memory and cognitive impairment, relieving beta-amyloid plaque deposition, reducing neuron loss and glial cell activation and the like.
Absstract of: CN122251404A
The invention provides application of a GPR65 agonist in preparation of a medicine for preventing and/or treating Alzheimer's disease, and belongs to the technical field of biological medicine. The research finds that the GPR65 agonist improves the cognitive function of an AD mouse model, relieves neuroinflammation and pathological characteristics and enhances synaptic plasticity. A molecular target and an intervention window are provided for a treatment strategy in AD research, and an important theoretical basis and an experimental support are provided for subsequent drug development and transformational medical research.
Nº publicación: CA3313684A1 21/06/2026
Applicant:
SANOFI [FR]
THE BRIGHAM AND WOMENS HOSPITAL INC [US]
THE BRIGHAM AND WOMEN'S HOSPITAL, INC.
SANOFI
Absstract of: WO2021011673A2
Novel binding polypeptides (e.g., antibodies and antigen-binding fragments thereof) that specifically bind one or more species of soluble, AD brain-derived synaptotoxic amyloid beta (Aβ) without binding to classical monomeric, protofibrillar or fibrillar Aβ, are provided. Pharmaceutical compositions comprising binding polypeptides that specifically bind one or more species of soluble, synaptotoxic Aβ are provided. Methods of making binding polypeptides that specifically bind one or more species of soluble, synaptotoxic Aβ are provided. Methods of treating Alzheimer's disease using binding polypeptides that specifically bind one or more species of soluble, synaptotoxic Aβ are provided. Methods of reducing one or more symptoms of Alzheimer's disease using binding polypeptides that specifically bind one or more species of soluble, synaptotoxic Aβ are provided.