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).
Ongoing policy issues in the U.S., including the Inflation Reduction Act and recent proposals under President Donald Trump’s administration, have wide ranging implications for the global biopharmaceutical industry, speakers at Bio Korea 2025 said May 8, including a heightened need for all biotechs to draft regulatory strategies.
Lysosomal storage disorder specialist Azafaros NV has raised €132 million (US$146.7 million) in a series B to fund two phase III studies of its lead product, a small-molecule glycosidase inhibitor that the company says has shown disease-modifying effects.
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