Invitae Corp. has entered into a partnership with Bridgebio Pharma Inc. to advance genetics-based drug discovery for rare diseases. The goal of the collaboration is to generate new insights focused on genetic modifiers and the discovery of novel therapeutic targets for rare diseases and other unmet medical needs.
CAR T cells could be repurposed to target senescent cells and delay the effect of aging. A study by scientists at Cold Spring Harbor Laboratory showed how to design them and demonstrated the advantages of this therapy in mice. “We only gave one dose, and we could have benefits [for] really long periods of time,” lead author Corina Amor told BioWorld.
Researchers from the Institute for Bioengineering of Catalonia (IBEC) and collaborators have successfully treated bladder tumors in mice using urease-powered nanobots. The testing consisted of the administration of 18F-nanorobots to tumor-bearing mice divided into four groups depending on tumor volumes using a group of non-tumor-bearing mice as control. Importantly, the therapy remained in the bladder with only a very small proportion of radioactivity seen in other organs. The results were published in Nature Nanotechnology on Jan. 15, 2024.
Genedit Inc. has entered into a multiyear collaboration and license agreement with Genentech Inc., a member of the Roche Group, to use Genedit's Nanogalaxy platform to discover and develop novel hydrophilic nanoparticles to deliver Genentech's nucleic acid-based medicines for treatment of autoimmune disease.
Researchers from Broad Institute and Harvard University presented the discovery of all-in-one virus-like particles (VLPs) designed to deliver prime editor (PE) ribonucleoprotein (RNP) complexes into mammalian cells.
Reprogramming techniques to generate functional neurons could improve neurodegeneration in the future. A group of researchers from the Institute for Stem Cell Research (ISF) in Germany have found the pathways that play a role in improving the conversion of astrocytes into neurons.
Using interactions between viral peptides and human proteins as a starting point, researchers from Enyo Pharma Inc., the University of Lyon and other institutions were able to bootstrap themselves to a mitochondria-targeting small molecule that showed activity in a mouse model of nonalcoholic steatohepatitis (NASH) with chronic kidney disease.
Sanyou Biopharmaceuticals (Shanghai) Co. Ltd. has launched its Sanyou Super Trillion Peptide Molecule Discovery Platform, suitable for the R&D of peptide-based drugs targeting diseases associated with metabolism and tumors, as well as applications in medical imaging, molecular diagnostics and other fields.
Syntheticgestalt Ltd. and Enamine Ltd. have begun a joint effort to create a suite of artificial intelligence (AI) models that will enable the generation of synthetically accessible biologically active compounds with optimized physicochemical and ADME/Tox properties.
Researchers have reported that the predictive abilities of a machine learning algorithm trained using best practices on a large clinical dataset did not generalize beyond the data that was used to train it. The algorithm was able to predict, to a degree, which individual patients would benefit from the medication when the patients were from the dataset the algorithm was trained on. But when it was supposed to predict who would benefit in clinical cohorts that were not part of the training, it performed no better than chance.