Researchers from the Baylor College of Medicine have characterized 100 conserved Alzheimer’s disease (AD) risk orthologue genes in Drosophila and found several with unknown roles in brain structure, function and stress resilience. The implication of this finding is that new pathways of neurodegeneration have been revealed, offering new insights into the genetic complexity of AD.
Transition Bio Inc. and Voyager Therapeutics Inc. have entered into a drug discovery collaboration and license option agreement for novel, selective small molecules for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) with TDP-43 pathology.
Sage Therapeutics Inc. has described sterol derivatives acting as glutamate receptor ionotropic, NMDA 2A (GRIN2A; GluN2A) negative allosteric modulators reported to be useful for the treatment of neurological disorders.
In Alzheimer’s disease, microglia act as a double-edged sword. They can either protect the brain or worsen the damage, depending on their activation state. Inflammatory activation harms healthy neurons. However, a study reveals that a special type of microglia expressing specific receptors and behaving like T cells may help mitigate this neurodegenerative condition.
A collaborative effort is trying to construct the cell atlas of the developing brains of humans and animal models. Using advanced single-cell and spatial technologies, they mapped how brain cell types emerge, diversify and organize over time, offering new insights into the origins of neurodevelopmental and psychiatric conditions. These breakthroughs in genomics and imaging will allow scientists to study complex developmental processes with high resolution.
Transthera Sciences Inc. is out-licensing one of its preclinical NLRP3 inhibitors to Neurocrine Biosciences Inc. under a collaboration agreement worth $881.5 million. Under deal terms, Nanjing, China-based Transthera will receive an undisclosed up-front payment and is eligible to receive research and development and sales-based milestone payments up to $881.5 million.
Despite the formidable challenges for developing precision psychiatry, the approach is notching its first successes in the preclinical and even some clinical settings. Many individual studies as well as large projects like the Psychiatric Ratings using Intermediate Markers (PRISM) studies and the Psychiatric Biomarkers Network (PBN) have been looking at multiple biomarker types, and have begun to identify predictors of specific symptoms, or disease progression.