Glucocorticoid replacement therapy is the current standard of care for congenital adrenal hyperplasia (CAH). However, new therapeutic strategies that can better recapitulate physiological requirements and reduce morbidity and mortality among CAH patients are urgently needed. Despite the promise of gene therapy for correcting monogenic disorders, the strategies investigated to date have not yielded satisfactory results.
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.
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.
It’s the biological resource that keeps on giving, and now UK Biobank has released the final tranche of data on the levels of 249 metabolites in the blood of its half a million participants.
Could GLP-1 receptor agonists (GLP-1RAs), already used in obesity and diabetes, be repurposed as drugs to slow aging? Hong Kong, one of the places in the world with the highest human longevity, is also home to a scientific study on the effects of GLP-1RAs. For the first time, scientists at the Chinese University of Hong Kong (CUHK) have assessed their pharmacological potential in later life using a multiomics preclinical approach.
South Korean researchers led by Lee In-suk of Yonsei University have reported the most complete oral microbiome catalog to date, with more than 72,000 genomes. Detailed in Cell Host & Microbe on Nov. 12, 2025, the database is expected to serve as a universal platform for academia and enable “precision microbiome medicine” for the industry, Lee told BioWorld.
Mitorx Therapeutics Ltd. has raised £5.5 million (US$7.2 million) in a pre-series A round to advance its lead mitochondrial-targeted program, Myo-004, through clinical candidate selection.
Pfizer Inc. has divulged gastric inhibitory polypeptide receptor (GIPR) antagonists reported to be useful for the treatment of type 2 diabetes and obesity.
Century Therapeutics Inc. has announced its first program for type 1 diabetes, CNTY-813. The program comprises induced pluripotent stem cell (iPSC)-derived β islets engineered with the company’s proprietary Allo-Evasion 5.0 technology, designed to protect from T-cell, NK-cell and humoral immune rejection, with the goal of durable glycemic control without the need for systemic immunosuppression.
Melanocortin-4-receptor MC4R is a crucial molecule involved in energy homeostasis, feeding behavior and metabolism, and loss-of-function mutations in this gene are the main monogenic cause of early-onset obesity. Congruence Therapeutics Inc. has developed a small molecule, CGX-926, that corrected MC4R deficiency and restored its cell-surface expression and function.
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