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.

Though immune checkpoint inhibitors have improved the outcomes of patients with hepatocellular carcinoma (HCC), the response rates remain limited. At the annual meeting of the American Association for the Study of Liver Diseases, researchers highlighted N-lysine methyltransferase SMYD2 as an oncogenic protein overexpressed in several tumor types.