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.
CMR Surgical Ltd. hopes to soon gain U.S. FDA 510(k) clearance for its Versius Plus surgical robotic system to bring it to the U.S. market amid rising demand for robotic surgery. Although the company’s original Versius system received FDA clearance in 2024 for use in cholecystectomy in adults, CMR waited to bring the enhanced Versius Plus model to market instead. “I have such a respect for the U.S. market that the last thing that I would do is to enter a market with a product that is outdated,” Massimiliano Colella, CMR CEO told BioWorld.
At the 2025 annual meeting of the Society for Neuroscience in San Diego this week, Catherine Woolley’s plenary lecture was an unusual combination of debunking and affirming the importance of sex differences in the brain.
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.
At the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, researchers from Onco3r Therapeutics BV presented a novel series of selective SMARCA2 small-molecule inhibitors with a best-in-class potency and selectivity profile.
Homozygous familial hypercholesterolemia (HoFH) is a genetic condition caused by mutations in the LDLR gene and characterized by severe hypercholesterolemia and premature cardiovascular disease. Repair Biotechnologies Inc. has developed REP-0003, a therapeutic based on the Cholesterol Degrading Platform (CDP) that degrades excessive intracellular free cholesterol into a non-toxic and excretable catabolite to safely reverse cholesterol-rich vulnerable plaque.
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.
Recent progress in redressing the historical underfunding and neglect of women’s health could be undermined by the backlash against diversity, equity and inclusion (DEI) initiatives, according to executives participating in the FT Global Pharma and Biotech Summit 2025 in London Nov. 11-12.
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