Agonists of the glucagon-like peptide-1 receptor (GLP-1RAs) have emerged as effective treatments for obesity, but have a negative impact on lean mass during weight loss.

A disease-modifying gene therapy for Sanfilippo syndrome type A has demonstrated reductions in heparin sulfate within cerebrospinal fluid, as well as increases in the cognitive function of young patients, arming Ultragenyx Pharmaceutical Inc. with data needed to support an accelerated BLA with the U.S. FDA.

Diabetic retinopathy and diabetic kidney disease are frequent microvascular complications of diabetes, both related to exacerbated vascular permeability coming from microvascular barrier malfunctioning.

Graviton Bioscience Corp. has announced a strategic investment from Sanofi SA. Under the agreement, Sanofi receives a right of first negotiation to license compounds across various indications, including immunological and metabolic syndrome indications.

Vivet Therapeutics SAS has been awarded financing of €4.9 million (US$5.3 million) from the French government to advance the development of a gene therapy for the treatment of the neurodegenerative disease cerebrotendinous xanthomatosis (CTX).

Humans love to think of our species as unique. But on a genetic level, such uniqueness is surprisingly hard to find. And while that may be a blow to the ego, it also means that an evolutionary lens is one way to search for insights into human diseases. Animals are “adapted to use the same genes that you and I have, but in very different ways,” Ashley Zehnder told BioWorld. Zehnder is co-founder and CEO of Fauna Bio Inc., which uses comparative genomics to identify gene networks that underlie disease resistance in different animal species.

Kupffer cells are macrophages located in the liver that play a key role in liver pathology, concretely in metabolic dysfunction-associated steatotic liver disease (MASLD; formerly nonalcoholic fatty liver disease or NAFLD), but the mechanisms behind this are poorly understood.