Krabbe disease, also called globoid-cell leukodystrophy, is caused by a deficiency of the lysosomal enzyme β-galactosylceramidase (GALC), resulting from pathogenic variants in both copies of the GALC gene. The GALC deficiency leads to increased levels of the primary galactosphingolipid substrates, galactosylceramide (GalCer) and psychosine (galactosylsphingosine, PSY), in the nervous system.
Genentech Inc. is paying $200 million up front and up to $1.5 billion in milestone payments to license one of Suzhou Sanegene Bio Inc.’s RNAi programs. Metabolic and autoimmune-focused Sanegene did not disclose specifics around the licensed candidate, except that it was derived from its LEAD (Ligand and Enhancer Assisted Delivery) platform.
Metabolic dysfunction-associated steatohepatitis (MASH) involves hepatic steatosis, inflammation, and fibrosis driven in part by hepatic stellate cell (HSC) activation. Dual inhibition of ACLY and ACSS2 restricts complementary hepatic sources of acetyl-CoA, a key driver of lipogenesis and MASH pathology.
Boehringer Ingelheim Pharma GmbH & Co. KG has synthesized peptide-lipid drug conjugates comprising a neuromedin U NMU2 receptor agonist peptide. They are reported to be useful for the treatment of obesity.
Astrazeneca AB has identified new quinazoline derivatives acting as gastric inhibitory polypeptide receptor (GIPR) antagonists. As such, they are described as potentially useful for the treatment of colitis, diabetes, inflammatory bowel disease, osteoporosis, psoriasis, schizophrenia, thrombosis and Alzheimer’s disease among others.
Recent studies found that some members of the olfactory receptor (OR) family can be activated by peptides and hormone proteins to mediate specific physiological processes, in addition to their roles in sensing odor molecules to mediate olfaction.
Terns Pharmaceuticals Inc. has reported triazole compounds acting as gastric inhibitory polypeptide receptor (GIPR) antagonists. They are intended for use in the treatment of diabetes, obesity, liver diseases and cardiometabolic syndrome.
Astrazeneca plc has signed a new strategic collaboration agreement with CSPC Pharmaceutical Group Ltd. to advance the development of next-generation therapies for obesity and type 2 diabetes across eight programs.
Ahead Therapeutics SL has been awarded an Industry Discovery and Development Partnership (IDDP) grant from Breakthrough T1D to support progression of the company’s proprietary Mimi-Top (mimicking tolerogenic particles) platform.
Insilico Medicine Cayman Topco has nominated ISM-0676 as a preclinical candidate targeting the glucose-dependent insulinotropic polypeptide receptor (GIPR).
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.webp?height=488&t=1750428815&width=650 "Interfering RNA (RNAi)")
