FGFR3 genomic alterations, including S249C as the most common, are recognized oncogenic drivers in 10%-60% of bladder cancers depending on the disease stage. Onco3r Therapeutics BV recently reported the identification of a novel series of highly potent, isoform-selective small-molecule FGFR3 inhibitors.

Triple-negative breast cancer (TNBC) is notoriously difficult to treat because it is quite aggressive and the tumors do not express the three major surface hormone receptors that can be targeted with available drugs. A potential target for treating this cancer may be glutathione peroxidase 4 (GPX4), which normally protects cells from ferroptosis, an iron-mediated form of cell death triggered by high oxidative stress.

Nectin-4 is a cell-adhesion molecule that is highly expressed in several malignancies, including bladder, colorectal, lung and breast cancers, while exhibiting minimal expression in most normal adult tissues.

A paper from Onchilles Pharma Inc. and collaborating institutions details the development of N-17350, a next-generation therapeutic elastase optimized for intratumoral delivery that targets the neutrophil elastase pathway.

Tangram Therapeutics plc has submitted a clinical trial application (CTA) to the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II trial of TGM-312 for metabolic dysfunction-associated steatohepatitis (MASH).

A 24‑week pregnant woman fears for her unborn baby, who is developing with a sacrococcygeal teratoma so large and vascularized that it nearly surpasses the size of the fetus itself. Faced with this threat, surgeons operate inside the uterus in an open procedure that partially exposes the baby to remove the tumor and give the baby a chance to survive until birth. According to scientists presenting at the American Society of Gene & Cell Therapy's special meeting on Breakthroughs in Targeted In Vivo Gene Editing, this could be avoided.