Gene and cell therapies (GCTs) can target the kidney to treat congenital, acute or chronic diseases affecting this organ. However, its complex structure poses a challenge for these technologies. To be precise and effective in the long term, new approaches should circumvent the specificities of renal tissue, with novel methods of delivery and gene transfer to offer new therapeutic options for patients who lack them.
Using a customized gene editing therapy, researchers at the Children’s Hospital of Philadelphia have reported success in treating an infant with a severe metabolic disorder. Kiran Musunuru, Barry J. Gertz Professor for Translational Research in the University of Pennsylvania’s Perelman School of Medicine, presented the case at the American Society of Gene and Cell Therapy’s 2025 annual meeting. The case study was simultaneously published in The New England Journal of Medicine.
CCR8 is highly expressed on immunosuppressive regulatory T cells (Tregs) in various solid tumors, making it a potential target to enhance antitumor immunity and the efficacy of cancer therapies, including checkpoint inhibitors. However, the impact of CCR8 expression on the Treg phenotype and its role in cancer progression remain unclear.
One of the main goals in the prevention of cardiovascular disorders is to maintain low-density lipoprotein cholesterol (LDL-C) at consistently low levels to ensure long-term cardiovascular protection. Investigators at Verve Therapeutics Inc. reported preclinical data on VERVE-102, a GalNAc base editing strategy designed to sustainably inactivate the PCSK9 gene and lower LDL-C in familial hypercholesterolemia.
Metabolic disorders such as argininosuccinic and glutaric aciduria, methylmalonic acidemia, homocystinuria or primary hyperoxaluria require specific diets to prevent the accumulation of substances that the body can’t process. Current treatments mainly focus on managing symptoms and metabolite levels, and do not always prevent the progressive deterioration caused by mutations associated with the condition. However, emerging gene therapies hold promise for transforming these diseases by targeting their underlying causes, as presented in the oral abstract session, “Gene and cell therapy for metabolic diseases” of the ongoing 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) meeting in New Orleans.
Immunity is not a function most people particularly associate with the liver. But because of its connection to the gut, the liver is exposed to bacterial metabolites as few other organs are. And when either the liver or the gut is not functioning well, it can adversely affect immunity as well.
Current anticancer approaches, such as antibody or CAR T-cell therapies, rely on targeting tumor-associated antigens rather than tumor-specific antigens, with the consequent on-target, off-tumor effects.
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