At the ongoing ACS meeting in New Orleans, Rezubio Pharmaceuticals Co. Ltd. detailed the discovery and preclinical evaluation of novel small-molecule gut-restricted GPR40 agonists using membrane-anchored drug (MADD) design.
The University of California, Berkeley has announced the launch of the UC Berkeley Molecular Therapeutics Initiative (MTI) designed to accelerate drug discovery. The aim is to bridge fundamental research in rare neurological and metabolic diseases with drug discovery to identify and accelerate novel therapeutic modalities into the clinic.
Cascade Pharmaceuticals Inc. has synthesized thyroid hormone receptor β (THRβ) agonists reported to be useful for the treatment of MASLD/NAFLD, hypothyroidism and more.
Fractyl Health Inc. has reported promising new preclinical findings supporting RJVA-001, the first clinical candidate in its Rejuva pancreatic gene therapy platform. The company’s GLP-1 gene therapy candidate has potential to treat metabolic diseases, including obesity and type 2 diabetes.
Researchers from Chinese Academy of Medical Sciences & Peking Union Medical College presented preclinical data for the new pan-peroxisome proliferator-activated receptor (PPAR) agonist, E-17241, being developed for the treatment of type 2 diabetes (T2D).
Guangzhou Lianrui Pharmaceutical Co. Ltd. has identified benzo bicyclic compounds acting as glucagon-like peptide 1 receptor (GLP-1R) agonists and reported to be useful for the treatment of diabetes, metabolic dysfunction-associated steatotic liver disease (MASLD) and obesity.
Creative Medical Technology Holdings Inc.'s Immcelz (CELZ-101) has been awarded orphan drug designation by the FDA. Immcelz is aimed at preventing allograft rejection in patients undergoing pancreatic islet cell transplantation.
In a recent study published in PNAS, researchers from the University of Texas Southwestern Medical Center investigated cholesterol-mimetic compounds specifically binding and inhibiting Scap. Their final goal was to understand cholesterol regulation by the Scap/SREBP system and identify potential therapeutics for dysregulated lipid metabolism.
The National Institute of Neurological Disorders and Stroke (NINDS) has awarded a 3-year grant totaling approximately $4.1 million to the Research Institute at Nationwide Children’s Hospital to fund completion of preclinical studies, manufacturing and preparation of an IND application for a first-in-human trial to advance adeno-associated virus (AAV)-ATP7A gene therapy for the treatment of Menkes disease.