The melanocortin MC4 receptor (MC4R), expressed in all brain regions, plays a relevant role in metabolism regulation and in the control of hunger and satiety. MC4R-specific agonist ligands such as setmelanotide have shown potential as antiobesity therapeutics, although reported off-target effects highlight the need for more specific compounds.
The FDA has awarded orphan drug designation to Papillon Therapeutics Inc.’s PPL-002, an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy, for the treatment of Danon disease.
Prime Medicine Inc. has announced its plans to strategically focus its efforts on a set of high value programs as it advances its pipeline of next-generation gene editing therapies.
In one of the largest private rounds raised by an Italian biotech, Genespire Srl has closed a €46.6 million (US$51.88 million) series B, enabling it to lay the ground for a phase I/II clinical trial of its lead program, GENE-202, and to further develop its proprietary lentiviral vectors. The vectors are designed to be applicable to a range of liver-related metabolic disorders and, as its first indication, the company intends to treat methylmalonic acidemia, a serious genetic condition that results in impaired metabolism of certain amino acids and lipids.
Genespire Srl has closed a €46.6 million (~$52 million) series B financing to support its work developing off-the-shelf gene therapies based on immune shielded lentiviral vectors (ISLVs) for pediatric patients with genetic diseases.
Arrowhead Pharmaceuticals Inc. has filed for regulatory clearance to initiate a phase I/IIa trial of ARO-INHBE, the company’s investigational RNA interference (RNAi) therapeutic being developed for obesity.
Breakthrough – or even better, revolutionary breakthrough – is perhaps the most overused term in drug development. But the discovery and development of GLP-1 receptor agonists (GLP-1RAs), which was honored with the 2024 Lasker-DeBakey Clinical Medical Research Award just last week, is one of the rare innovations that is deserving of the title.
An innovation that has kickstarted a revolution in the study and practice of health care is getting even more attention. Three scientists who pioneered the discovery and development of glucagon-like peptide-1 (GLP-1)-based obesity treatments have been named 2024 Lasker Award winners.
Scientists from the Cardiovascular Research Center at the University of Virginia School of Medicine and Astrazeneca plc have developed a new mouse model of cardiovascular disease associated with genetic variations of cholesterol metabolism. The animal allows in vivo studies of myocardial infarction, plaque rupture and stroke.