Prime Medicine Inc. has obtained clearance from the New Zealand authority, Medsafe, for the company’s clinical trial application for PM-577a, an investigational Prime Editor for Wilson’s disease.
At the recent Endocrine Society meeting in Chicago, Kalohexis Inc. presented preclinical efficacy data on 710GO, an oral dual MC3R/MC4R agonist, in obese nonhuman primates (NHPs).
Constantiam Biosciences Inc. and Cincinnati Children’s have established a strategic collaboration, through an exclusive option for future licensing rights, to advance first-in-class small-molecule treatments for neuronopathic Gaucher disease (types 2 and 3).
Researchers from Rivus Pharmaceuticals Inc. presented preclinical efficacy data on RV‑202, a novel oral mitochondrial uncoupler with activity attributed to adenine nucleotide translocase (ANT) activation, in models of obesity.
Researchers from Confo Therapeutics NV presented the preclinical characterization of CFTX-2034, a selective somatostatin receptor subtype 5 (SSTR5) agonist developed using the company’s proprietary technology platform for the treatment of life-threatening hypoglycemic episodes associated with post-bariatric hypoglycemia (PBH).
Beam Therapeutics Inc. has obtained IND clearance from the FDA for BEAM-304 for the treatment of phenylketonuria (PKU). BEAM-304 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct mutations in the phenylalanine hydroxylase (PAH) gene that cause PKU.
Chengdu Chipscreen Pharmaceutical Ltd. has divulged new 2-acylglycerol O-acyltransferase 2 (MGAT2, MOGAT2) inhibitors potentially useful for the treatment of obesity.
Although GLP-1 receptor agonists (GLP-1RAs) have significantly advanced obesity treatment, their limitations underscore the need for new therapies that promote weight loss while preserving muscle and supporting metabolic health. Researchers from Rivus Pharmaceuticals Inc. discussed the discovery and preclinical profile of RV-8451, a potentially first-in-class, oral, nonpeptide GLP-1RA.
Shenzhen Salubris Pharmaceuticals Co. Ltd. has disclosed new dual proprotein convertase subtilisin/kexin-type 9 (PCSK9) and lipoprotein(a) (Lp[a])-targeting agents potentially useful for the treatment of hypercholesterolemia.