At the recent American Society of Gene and Cell Therapy (ASGCT) meeting, Wave Life Sciences Ltd. presented siRNAs designed to suppress expression of the liver gene inhibin subunit β E (INHBE). Human genetic data show that heterozygous INHBE loss-of-function carriers exhibit a healthy metabolic profile.
It’s been a big week for Inozyme Pharma Inc. On the heels of a promising interim readout for phase III-stage enzyme replacement therapy (ERT) candidate INZ-701 in ENPP1 deficiency, the firm agreed to be acquired by Biomarin Pharmaceuticals Inc. in a deal valued at about $270 million, putting the rare disease ERT in the hands of an experienced commercial team.
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.
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.
Only a few days out of the European Association for the Study of the Liver annual meeting, the metabolic dysfunction-associated steatohepatitis (MASH) space continues to grab headlines, with GSK plc shelling out $1.2 billion up front to acquire phase III-ready efimosfermin alfa in a deal with Boston Pharmaceuticals Inc. that could end up totaling about $2 billion.
In a deal that could top out at about $2.2 billion, Septerna Inc. is getting $200 million up front from Novo Nordisk A/S in a collaboration to develop oral treatments for obesity, type 2 diabetes and other cardiometabolic indications. There will be four programs for discovering, developing and commercializing small molecules targeting G protein-coupled receptors, which includes GLP-1, GIP and glucagon receptors, with both companies putting their shoulders to conducting research from discovery to choice of candidate.
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.
Zhejiang Doer Biologics Co. Ltd. has presented data regarding their FGF21R/GCGR/GLP-1R triple agonist DR-10624 for the treatment of metabolic dysfunction-associated steatohepatitis (MASH).
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