The identification of non-toxic targets for the treatment of metabolic disorders would allow the development of therapies for the harmful effects of impaired metabolism of reactive aldehydes, among others, which is challenging for drug development due to short half-life and lack of tissue specificity.
Antag Therapeutics ApS has received IND clearance from the FDA for lead molecule, AT-7687. A phase I trial will evaluate AT-7687 in healthy lean and healthy obese subjects, including as monotherapy and in combination with semaglutide in the healthy obese individuals.
Cascade Pharmaceuticals Inc. has synthesized benzoheterocyclic compounds acting as free fatty acid FFA1 receptor (GPR40) agonists reported to be useful for the treatment of diabetes.
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.
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