Annexon Inc. has identified boronic acid derivatives acting as complement C1s subcomponent inhibitors reported to be useful for the treatment of neurodegeneration, inflammatory, eye, metabolic and autoimmune diseases.
Swiss researchers have gained new insights into the relationship between aging, inflammation, neurodegeneration and cognitive decline. EPFL professor Andrea Ablasser and her team showed that brain aging was driven by microglial activation of the cGAS/STING pathway.
When a group of British scientists studied which proteins might be in the wrong place of the cell in amyotrophic lateral sclerosis (ALS) patients, they found hundreds of them mislocalized. Other studies had shown that TDP-43 protein was mislocalized. But it was not known that the phenomenon was widespread, and affected mRNA as well as proteins. “Our study revealed that these mislocalized proteins were heavily involved in RNA binding functions and exhibited high binding affinities to RNAs,” Rickie Patani told BioWorld.
Sigma nonopioid intracellular receptor 1 (SIGMAR1) is a protein enriched in motor neurons (MNs), and mutations in its gene have been previously linked to various motor neuron diseases (MNDs). Researchers from Welab Barcelona and affiliated organizations recently presented preclinical data for two novel SIGMAR ligands, EST-79232 and EST-79376, being evaluated as potential candidates for the prevention of MN degeneration.
With the approval of Aduhelm (aducanumab, Eli Lilly & Co.) and Leqembi (lecanemab, Eisai Co. Ltd.), there are finally amyloid-targeting drugs available for Alzheimer’s disease (AD). What’s not available, though, are rose-colored glasses of the prescription strength that would make these approvals look like AD’s happy ending. The biopharma industry is already well aware of the need for broader horizons. Roughly three-quarters of drugs now in clinical development for AD target neither amyloid-β (Aβ) nor tau. Still, the genetic evidence from familial AD strongly implicates Aβ processing in AD’s origins. In his opening plenary talk at the European Academy of Neurology 2023 annual conference, Thomas Südhof suggested new ways to look at the clinical data.
Monoacylglycerol lipase (MAGL) is a key regulator of the endocannabinoid system (ECS), which has a critical neuromodulatory involvement in numerous functional mechanisms in the CNS. Based on this, MAGL is considered a promising therapeutic target in neuroinflammation and neurodegeneration. Researchers from ETH Zürich and affiliated organizations have recently presented their work on (R)-[18F]YH-134, a novel reversible radiotracer for imaging MAGL in the brain.
The phenotypic variety of spinocerebellar ataxias (SCAs) not caused by CAG repeat expansion (polyglutamine SCA) is greater than expected. A collaboration directed by scientists of the Paris Brain Institute described seven variants of this disorder in 756 individuals, observing that age at onset and progression by gene and variant can occur from childhood to late adulthood with very different forms of the disease.
Regenlife SAS raised $3.3 million in series A funding in order to finalize the development of its photomodulation technology to treat neurodegenerative diseases.
Researchers from Athira Pharma Inc. presented preclinical efficacy data for ATH-1105, a positive modulator of hepatocyte growth factor (HGF)/MET, being evaluate for the treatment of amyotrophic lateral sclerosis (ALS).
Scientists from the UK Dementia Research Institute at the University of Cambridge have described how cytosolic antibody receptor TRIM21 contributes to in vivo protection during tau immunotherapy. Their work on TRIM21’s mechanism of action may help in moving a step closer toward enhanced second-generation antibodies for tauopathy treatments.
