Non-small-cell lung cancer (NSCLC) accounts for up to 85% of all cases of lung cancer, which is the most frequent cause of cancer-related deaths worldwide. The need for effective treatments against NSCLC is urgent, and one promising target is pyruvate kinase M2, which plays metabolic and nonmetabolic roles in the cell. This enzyme has been implicated in various cancers beyond NSCLC, including pancreatic, gastric and breast cancers.

Angelman syndrome (AS) is a rare neurodevelopmental disorder caused by the loss of function of the maternally inherited ubiquitin E3 ligase UBE3A gene. AS is characterized by severe symptoms, including lack of speech, epilepsy, developmental and motor skills delays, sleep disturbances and cognitive impairment. Currently, no treatments are available for this disease.

Obesity is a multifactorial metabolic disorder with limited treatment options capable of sustaining weight loss and improving systemic metabolic health. MicroRNA-22 (miR-22) has emerged as an essential regulator of metabolic homeostasis, influencing lipid biosynthesis, mitochondrial function and brown adipose tissue development. These roles position miR-22 as a promising target for therapeutic intervention in obesity and related disorders such as metabolic dysfunction-associated steatotic disease (MASLD) and its progressive disease state, metabolic dysfunction-associated steatohepatitis (MASH).

NS Pharma Inc.’s NS-051 (NCNP-04) has been awarded orphan drug designation by the FDA for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping.

Genfit SA is discontinuing its VS-01 program in acute-on-chronic liver failure (ACLF), and has decided to reprioritize development of VS-01 for urea cycle disorder.

The FDA has cleared HDT Bio Corp.’s IND application for HDT-321, a first-in-class prophylactic designed to protect against Crimean-Congo hemorrhagic fever (CCHF).