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.
Silicosis, resulting from prolonged exposure to free crystalline silica dust, is an occupational disease characterized by silica nodules and extensive lung tissue fibrosis. The etiology of silicosis remains unclear, challenging early detection and effective treatment.
Previous research has demonstrated that activin receptor-like kinase 7 (ALK7) signaling suppresses lipolysis, resulting in increased adipocyte size and lipid content. Additionally, predicted loss of function variants in the ALK7 gene (ACVR1C) have been associated with reduced waist-to-hip ratio (WHR) adjusted for body mass index, protection from type 2 diabetes, as well as reduced risk of cardiovascular disease.
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.
While first-generation glucagon-like peptide-1 receptor agonists have clearly taken the obesity market by storm, generating billions of dollars for Novo Nordisk A/S and Eli Lilly and Co., several other companies are developing follow-on products that could clean up the tolerability and adherence issues of Wegovy (semaglutide) and Zepbound (tirzepatide).
Researchers at E-Therapeutics plc recently presented efficacy and safety data on ETX-148, a pan-hemophilia agent in murine models of hemophilia A and B.
Silexion Therapeutics Corp. has released new preclinical results demonstrating the synergistic efficacy of its proprietary second-generation siRNA candidate, SIL-204, in combination with components of first-line chemotherapy for pancreatic cancer.
Shares of Phio Pharmaceuticals Corp. soared 291% Jan. 13 on news that two patients with cutaneous squamous cell carcinoma had a complete response following treatment with the company’s Intasyl siRNA gene silencing candidate PH-762.
Canary Cure Therapeutics (Canary Global Inc.) has released preclinical results for its lead candidate, CCT-217, an siRNA therapeutic targeting cannabinoid CB1 receptor (CB1R) and zinc finger protein 423 (ZFP423) to treat obesity.
