Boosting shares by nearly 17% on Sept. 9, phase I data of Terns Pharmaceuticals Inc.’s glucagon-like peptide 1 (GLP-1) receptor agonist, TERN-601, demonstrated it was well-tolerated and led to a statistically significant weight loss across all three doses tested in healthy adults who were obese or overweight.

Immortality and eternal youth have been the stuff of myths and legends from ancient times on. Now, in the 21st century, real studies of current medicine could be applied to repair tissues and organs damaged by age. During the 11th Aging Research & Drug Discovery (ARDD) Meeting held at the University of Copenhagen at the end of August, scientists explained the molecular keys of rejuvenation, as many artists imagined in the past.

The dark matter of long non-coding RNA (lncRNA) is shades brighter, after the signing of two major deals between biotech pioneers and big pharma in the past week. Haya Therapeutics SA announced Sept. 4 that it has sealed a multiyear agreement with Eli Lilly and Co. to apply its lncRNA platform technology to identify targets in obesity and related metabolic disorders.

With a successful phase III study of plozasiran in hand, Arrowhead Pharmaceuticals Inc. plans to file an NDA with the U.S. FDA for treating the rare genetic disease familial chylomicronemia syndrome. While the data are strong, the company is playing catch-up to Ionis Pharmaceuticals Inc., which has a December 2024 PDUFA for its candidate, olezarsen, in the same indication.

Yoltech Therapeutics Co. Ltd. licensed its PCSK9-targeting gene editing therapeutic, YOLT-101, to Shenzhen Salubris Pharmaceuticals Co. Ltd. for mainland China rights in a deal worth ¥1.035 billion (US$145 million).

Restoring glucose metabolism in astrocytes, which is impaired in Alzheimer’s disease (AD), has a direct effect on neurons, which replenish their fuel supply and resume synaptic activity. A group of scientists from Stanford University School of Medicine has revealed the pathway that explains where this efflux is interrupted and which molecules restore it in mouse models with amyloid and tau pathology. Their findings could help prevent the progression of this neurodegenerative disease.

Phagocytosis – eliminating millions of dead cells every day – requires specialized cells such as macrophages, the true professionals, which migrate to engulf waste and dying cells. But they are not the only ones that can perform this task, as scientists at Howard Hughes Medical Institute (HHMI) discovered when they investigated hair follicle stem cells (HFSCs), a tissue in constant regeneration, to clarify how dying cells are detected and cleared in the epithelium and the mesenchyme.

People with the rare inherited metabolic disorder Gaucher disease have a deficiency in the lipid-digesting glucocerebrosidase enzyme, which causes the accumulation of harmful levels of glucolipids in various organs. The enzyme has a very short half-life, which rules out enzyme replacement as an effective therapy, and as things stand, there are few treatments for this and other lysosomal storage diseases (LSDs). Now, researchers have discovered two small molecules that enhance the activity of glucocerebrosidase in cellular models of LSD, pointing to a potential new approach to treating these diseases.