Investigators from the University of Turin have developed and characterized a new murine immunocompetent model of Usp18-knockout leiomyosarcoma (LMS). LMS is a rare malignant soft tissue cancer with limited therapeutic options when in advanced stages.

Investigators from Duke University hypothesized that hirudin-like protease inhibitors could be generated by linking exosite-binding aptamers with small-molecule active site inhibitors, thus generating more potent “EXACT” inhibitors.

Sarepta Therapeutics Inc. has signed a licensing and collaboration agreement with Arrowhead Pharmaceuticals Inc. to obtain exclusive global rights to multiple clinical, preclinical and discovery-stage programs for rare genetic diseases of the muscle, central nervous system (CNS) and the lungs. The deal includes ARO-DUX4, ARO-DM1, ARO-MMP7 and ARO-ATXN2.

Enveric Biosciences Inc. has completed preclinical pharmacokinetic (PK) studies of EB-003 in rats and dogs, further supporting EB-003’s oral bioavailability and targeted nonhallucinogenic profile. EB-003 is a neuroplastogen designed to promote neuroplasticity without inducing hallucinations in patients with difficult-to-address mental health disorders.

NLRP3 inflammasome activation, pro-inflammatory cytokine production and pyroptosis are key features of inflammation that contribute to liver fibrosis progression, cirrhosis and end-stage liver failure. Pyroptosis, a lytic form of inflammatory-regulated cell death, is regulated by multiprotein complexes expressed in both parenchymal and nonparenchymal hepatic cells. Researchers from Genfit SA presented preclinical efficacy data on CLM-022, a synthetic pentacyclic triterpenoid derivative designed to target the NLRP3 complex.

Oxford Nanopore Technologies Ltd. has established a new collaboration with UK Biobank to create the world’s first comprehensive, large-scale epigenetic dataset. The project will utilize Oxford Nanopore’s DNA/RNA sequencing technology to map the epigenome of 50,000 blood samples from UK Biobank to unlock insights into disease mechanisms, with the aim of improving patient outcomes.

Researchers at the University of Copenhagen have identified a signaling pathway that simultaneously increased energy expenditure and decreased food intake. In both human and primate studies, agonists of the tachykinin NK2 receptor (NK2R) led to both decreased food intake and increased energy expenditure. And in behavioral tests, they were not aversive, suggesting they do not cause the nausea that is a major side effect of GLP-1 agonists.