Maze Therapeutics Inc. recently presented data from preclinical studies of a small-molecule APOL1 pore function inhibitor, MZ-301, describing the compound’s in vitro and in vivo activity. APOL1 G1 and G2 genetic variants are associated with an increased risk of progressive kidney diseases in African ancestry people. There are no APOL1-targeted therapies addressing the underlying driver of these diseases.
Alport syndrome (AS) is a hereditary disease affecting type IV collagen that is caused – in 80% of cases – by the COL4A5 gene. In 10% to 20% of AS cases, a mutation cannot be detected.
Researchers from Carisma Therapeutics Inc. have provided details on the discovery and preclinical evaluation of a novel mesothelin-targeting chimeric antigen receptor macrophage (CAR-M), CT-1119, being developed as a potential solid tumor immunotherapy candidate.
Stimulating the brain via implanted electrodes is used to treat both movement disorders such as Parkinson’s disease, and some psychiatric conditions such as obsessive compulsive disorder. But researchers are also working on ways to make such implanted electrodes listen instead of talk – and translate neuronal signals for people that have lost the ability speak, or the ability to move.
Investigators at the University of Copenhagen and affiliated organizations presented data from a study that aimed to validate differentially expressed genes (DEGs) in mesial temporal lobe epilepsy (mTLE) as potential new targets.
RSS
