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..
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.
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.
Nº publicación: EP4649317A2 19/11/2025
Solicitante:
POPULATION BIO INC [US]
Population Bio, Inc
Resumen de: US2024310389A1
This document provides methods and materials related to screening for and treating Alzheimer's disease (AD), including late-onset Alzheimer's disease (LOAD).