The discovery of how proteins fold and what happens if they don’t, or do it wrong, has impacted medicine. Protein function and several pathologies depend on folding. Four scientists, Franz-Ulrich Hartl, Arthur Horwich, Kazutoshi Mori and Peter Walter, revealed the role of chaperones in assisting proteins to acquire their correct structure and described a failure warning system, the unfolded protein response (UPR). For their studies, the researchers will receive the 2024 Frontiers of Knowledge Award in Biology and Medicine from the BBVA Foundation. Read More
Researchers from Maze Therapeutics Inc. presented the discovery and preclinical characterization of MZ-101 as a potential candidate for the treatment of Pompe disease and other glycogen storage disorders. Read More
Primary open-angle glaucoma is the leading cause of blindness globally. Individuals of African ancestry have a disproportionately high risk of developing glaucoma, but genetic risk factors have been studied mainly in populations of European ancestry. Read More
The Cystic Fibrosis Foundation (CF Foundation) has agreed to provide Prime Medicine Inc. with up to $15 million to support the development of prime editors for the treatment of cystic fibrosis (CF). Read More
Accent Therapeutics Inc. has completed a $75 million series C financing to support progression of its lead programs through early clinical development. Read More
An international working group has proposed a new research framework that stages Parkinson's disease (PD) and defines it based on the disease's underlying biology, rather than on clinical symptoms. Read More
Researchers from The Cleveland Clinic Foundation presented a novel targeted therapeutic peptide for the treatment of triple-negative breast cancer (TNBC). Read More
Triple-negative breast cancer (TNBC) accounts for about 15% of invasive breast cancers, and due to the lack of well-established targets, TNBC has the worst prognosis among all breast cancer types. Read More
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. Read More
Bluerock Therapeutics LP, a wholly owned, independently operated subsidiary of Bayer AG, has exercised its option to exclusively license OpCT-001, an induced pluripotent stem cell (iPSC) derived cell therapy candidate for the treatment of primary photoreceptor diseases, from Fujifilm Cellular Dynamics Inc. and Opsis Therapeutics LLC. Read More
