Ependymoma is a brain cancer that occurs during childhood and is usually resistant to cytotoxic therapy. Molecular profiling has helped in better understanding the mechanisms behind the disease and has unveiled EPH receptor B2 (EPHB2) as a critical player in driving disease.

Researchers from Igyxos SA presented the preclinical profile of IGX-12, a humanized IgG4 antibody, in both female and male infertility models.

Researchers from the University of California San Francisco and Northwestern University recently presented findings from a study that aimed to evaluate the role of lin-28 homolog B (LIN28B), an RNA binding protein expressed in a variety of cancers, in diffuse midline glioma (DMG).

Many patients with amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD) harbor the (G4C2)n pathogenic repeat expansion in the C9orf72 gene, which leads to aggregating dipeptide proteins, mainly poly-glycine-alanine (poly-GA).

Recent studies have highlighted the potential role of the small GTPase Rac1 as new therapeutic target in SHH-medulloblastoma due to its regulation of Hedgehog signaling via the GLI1 and GLI2 transcription factors.

Large-scale human genetic studies have revealed that loss-of-function INHBE variants are associated with a lower abdominal to gluteofemoral fat ratio, improved metabolic profile, and reduced fasting glucose levels.

Von Willebrand factor (vWF) plays a crucial role in hemostasis, and elevated levels are associated with risk of thrombosis. Gain-of-function mutations in the C-domain of vWF are linked to an increased risk of thrombosis. Previous work has shown that disrupting cysteine-rich C-domain in vWF led to reduced platelet recruitment in a shear-dependent fashion, making it a promising target for pharmacology. Apollo Therapeutics Ltd. has presented data on antibodies targeting the C-domain of vWF, including AP-21 as antithrombotics.

Researchers from Oricell Therapeutics (Shanghai) Co. Ltd. presented the discovery and preclinical characterization of ORIC-613, a dual-targeting CAR T-cell therapy being developed for the treatment of pancreatic and gastric cancer.