If one could sweep the brain clean and send the toxic substances that drive neurodegeneration to the recycling bin, perhaps one could treat Alzheimer’s disease (AD). Researchers at the Chinese Academy of Sciences propose a new therapeutic strategy that uses synthetic peptides that bind to amyloid-β (Aβ) and direct it toward lysosomes. In addition, researchers at the Washington University School of Medicine in St. Louis have genetically modified astrocytes in vivo to express chimeric antigen receptors (CARs) that recognize and phagocytose Aβ plaques.

Japan has approved the world’s first therapies derived from induced pluripotent stem cells (iPSCs), marking a major milestone for regenerative medicine and, potentially, a turning point in treating Parkinson’s disease.

Researchers at INSERM and collaborators have identified hypothalamic tanycytes as mediators of tau clearance and shown that their structural and genetic disruption may drive Alzheimer’s disease pathology.

Science Corp. raised $230 million in an oversubscribed series C financing round to commercialize its Prima brain-computer interface (BCI) retinal implant and to advance other pipeline programs into the clinic. The investment brings the company’s total funding, since founding in 2021, to approximately $490 million and shows investors growing appetite for BCI technologies.

Researchers at INSERM and collaborators have identified hypothalamic tanycytes as mediators of tau clearance and shown that their structural and genetic disruption may drive Alzheimer’s disease (AD) pathology. AD is characterized by the buildup of extracellular amyloid-β plaques and intracellular tau tangles, protein aggregates that disrupt neuronal function and drive neurodegeneration.

Targeting the interaction between the C-terminal domain of the GluA2 subunit of AMPAR and brefeldin-resistant Arf-GEF 2 (BRAG2), a guanine nucleotide exchange factor for the small GTPase Arf6, presents a potential therapeutic approach for acute ischemic stroke.

At the current pace of innovation in the U.S. rare disease space, developing and approving therapies for just half of the 10,000-plus known rare diseases would take more than 160 years, Bradley Campbell, president and CEO of Amicus Therapeutics Inc., recently told the Senate Committee on Aging.

A therapeutic strategy based on alternative splicing of the MECP2 gene could restore protein levels in Rett syndrome, a neurological disorder caused by mutations in that gene. Scientists at Baylor College of Medicine have successfully tested this approach both in vitro in neurons from Rett patients that produce some functional protein, correcting the altered gene expression and improving neuronal functions, and in vivo in mice.