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.

Silexion Therapeutics Corp. has announced an expanded development plan for its next-generation siRNA candidate SIL-204. The new dual-route strategy will integrate intratumoral and systemic administration to target both primary tumors and metastases, respectively, to treat KRAS-driven pancreatic cancer.

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, and joins the small but growing group of non-factor therapy options, with the advantage of a broad label and a convenient dosing regimen.

Silexion Therapeutics Corp. has completed its initial study evaluating SIL-204 in orthotopic pancreatic cancer models. SIL-204 is a next-generation siRNA candidate designed to target a broad range of KRAS mutations.

Olix Pharmaceuticals Inc. walked the talk in realizing a new $630 million licensing deal with Eli Lilly and Co. for its cardiovascular and metabolic disease asset, OLX-702A (OLX-75016), rallying stock by 30% after it had largely recovered from a terminated deal with France’s Théa Open Innovation last year.

Olix Pharmaceuticals Inc. walked the talk in realizing a new $630 million licensing deal with Eli Lilly and Co. for its cardiovascular and metabolic disease asset, OLX-702A (OLX-75016), rallying stock by 30% after it had largely recovered from a terminated deal with France’s Théa Open Innovation last year.

Newco Akribion Therapeutics GmbH has raised €8 million (US$8.3 million) in a seed round to develop a new and potent class of RNA-targeted CRISPR nucleases, which, rather than cleaving specific nucleic acids, can destroy every type of nucleic acid in a cell.

Newco Akribion Therapeutics GmbH has raised €8 million (US$8.3 million) in a seed round to develop a new and potent class of RNA-targeted CRISPR nucleases, which, rather than cleaving specific nucleic acids, can destroy every type of nucleic acid in a cell.