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.

The long struggle by Boston-based I2o Therapeutics Inc.’s business unit Intarcia Therapeutics to get long-lasting exenatide for diabetes onto the market ended with a final thumbs-down from the U.S. FDA because of safety concerns. At issue was ITCA-650, a twice-yearly implantable exenatide-device combo meant to improve glycemic control in adults with type 2 diabetes.

The risk of developing multiple sclerosis (MS) is nearly four times as high for women as it is for men. And that relative risk has increased sharply over time. In 1955, women were only slightly more likely than men to develop MS. A research team at the University of Toronto and the Oklahoma Medical Research Foundation (OMRF) has gained new insights into possible causes for this increasing disparity.

A long-term look at obese and overweight patients with pre-diabetes found that weekly injections of Eli Lilly and Co.’s tirzepatide led to a 94% reduction in their risk of progression to type 2 diabetes compared to placebo – a result that Leerink Partners analyst David Risinger called “exceptional.”

Label comparisons began promptly with the accelerated U.S. FDA clearance of Gilead Sciences Inc.’s oral peroxisome proliferator-activated receptor (PPAR)-delta drug, Livdelzi (seladelpar), for primary biliary cholangitis (PBC). The space includes Ipsen Pharma SA’s dual PPAR alpha/delta agonist, Iqirvo (elafibranor), licensed from Genfit SA and cleared in June 2024, as well as Ocaliva (obeticholic acid), the first-in-class farnesoid X receptor agonist from Intercept Pharmaceuticals Inc., greenlighted for PBC in May 2016.

Visen Pharmaceuticals Co. Ltd.’s palopegteriparatide met both primary and secondary endpoints in the phase III Pathway trial conducted in China in adults with chronic hypoparathyroidism, according to top-line data. In the 26-week randomized, double-blind, placebo-controlled Pathway trial, 77.6% of patients treated with palopegteriparatide achieved the primary multicomponent endpoint compared to 0% for placebo.