Previous work uncovered the role of the complement system in neuroinflammation and synaptic loss in neurodegenerative disorders such as Alzheimer’s disease (AD). The third component, C3, central in all complement activation pathways, has been proposed as a potential therapeutic target in AD.

Sanofi SA gained U.S. FDA approval for fitusiran as a first-in-class siRNA therapy for hemophilia. Branded Qfitlia, the antithrombin-lowering therapy is indicated for use as a prophylactic treatment to prevent or reduce bleeding episodes in people with hemophilia A or B, with or without inhibitors.

Scientists at Massachusetts General Hospital have revealed how chronic liver injury alters hepatic stellate cells (HSCs), triggering the formation of fibrotic scarring in the liver. The researchers have shown that this cell type transdifferentiates into myofibroblasts, which generate excess extracellular matrix, driven by the activation of ABHD17B, an enzyme that could be investigated as a therapeutic target to inhibit liver fibrosis.