Neural stem cell-derived exosomes (Nexo) are small, membrane-bound extracellular vesicles secreted by neural stem cells. Due to their role in cell-to-cell communication within the central nervous system (CNS), they are considered a promising strategy for modulating neuronal function and promoting repair processes in conditions such as stroke, traumatic brain injury and neurodegenerative diseases. Researchers at the Chinese Academy of Sciences developed an advanced Nexo platform incorporating ultrasmall nanoselenium via lipid-mediated nucleation, termed Senexo.
A tangle of DNA can look like a knotted ball in the cell nucleus. However, the genetic machinery has a complex and regulated structure. Its long repetitive sequences also seemed to have no function. They were called junk DNA, although they were not. The same happened with proteins and low-complexity domains, disordered chains of amino acids that were poorly understood. Nevertheless, that protein noise has turned into music for the 2025 Lasker Awards. These prizes have recognized the work of scientists who were able to see order in chaos.
Arrowhead Pharmaceuticals Inc. has filed a request for regulatory clearance in New Zealand to initiate a phase I/IIa trial of ARO-MAPT, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for tauopathies, including Alzheimer’s disease.
Nanonewron Inc. has been awarded a $2.5 million NIH STTR phase II grant to support development of its TNF-α inhibitor NN-840 program for Alzheimer’s disease and other neurodegenerative conditions. The company aims to submit an IND application next year.
Loss of function variants in the lipid transporter gene ATP-binding cassette ABC transporter A7 (ABCA7) nearly double the risk of developing Alzheimer’s disease (AD), which makes ABCA7 the strongest AD genetic risk factor after ApoE4.
Researchers from Cyclone Therapeutics Inc. and the Scripps Research Institute have disclosed tetracycline derivatives reported to be useful for the treatment of fragile X syndrome, rheumatoid arthritis and Alzheimer’s disease.
Mutations that activate phosphorylation of certain Rab GTPases by LRRK2 have been linked to a subset of genetic cases of Parkinson’s disease, and PPM1M is one of the phosphatases responsible for reversing phosphorylation by LRRK2.
Researchers at Sanofi SA have developed a promising gene therapy approach targeting the microtubule-associated protein tau (MAPT) for the treatment of Alzheimer’s disease.
Neurosciences specialist NRG Therapeutics Ltd. is poised to put its new class of small-molecule regulators of the mitochondrial permeability transition pore to the test after closing an oversubscribed £50 million (US$67 million) series B.
Myotonic dystrophy type 1 (DM1) is a rare, progressive genetic disease that causes severe muscle weakness and other debilitating symptoms, such as compromised respiration and cardiac conduction abnormalities. No disease-modifying therapy exists for DM1, so care focuses on managing symptoms like arrhythmia, myotonia, hypertension, cataracts, respiratory issues and sleep disorders.