The number of deaths caused by prion diseases reaches about 30,000 annually. Only 5 months pass from the diagnosis of seemingly healthy patients to the fatal outcome of this neurodegenerative condition, and just 1 month until quality of life is completely lost. Removing the brain protein that causes this genetic or infectious disorder could be achieved thanks to new gene-silencing techniques. At a special meeting of the American Society of Gene & Cell Therapy, in “AAV-mediated epigenetic editing for prion disease,” Sonia Vallabh presented not just the data of her research, but the impact of this disease on her family and on herself.

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.

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 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.

Opko Health Inc. has recently presented data for their GLP-1/glucagon receptor dual agonist OPK-88006, which is in preclinical development for the treatment of metabolic disease, including metabolic dysfunction-associated steatohepatitis (MASH) and obesity.

The field of gene therapy is experiencing major advances driven by precise editing technologies, such as base and prime editing, and by the design of increasingly sophisticated vectors to deliver payloads that could reverse the effects of diseases. However, in the transition to in vivo applications many approaches still fail in their attempt to effectively reach target tissues or cells.

Researchers from Eilean Therapeutics LLC and collaborators presented the discovery and characterization of a new, selective CDK2 inhibitor showing potent in vitro and in vivo activity at the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics.