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#核酸药物

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Sep 9Wed
  1. nature.com44

    Inhaled siRNA therapy targeting RAGE for pulmonary inflammation: a first-in-human randomized trial

    Inhaled siRNA directed against RAGE was safe and well tolerated in a first-in-human randomized trial, its first test in people after preclinical development. The lung-epithelium-targeted therapy reduced RAGE levels in both serum and bronchoalveolar lavage fluid, supporting gene silencing delivered by inhalation as a route to treating pulmonary inflammation.

    Why it matters: First-in-human evidence that inhaled siRNA can safely silence a pulmonary inflammation target in lung and blood.

Sep 5Sat
  1. Nat Med61

    Inhaled siRNA therapy targeting RAGE for pulmonary inflammation: a first-in-human randomized trial.

    Inhaled ARO-RAGE, a lung-epithelium-targeted siRNA against pulmonary RAGE mRNA, was safe and well tolerated in a first-in-human randomized trial in 58 healthy volunteers and 19 patients with asthma. Plasma levels were low, consistent with drug retention in the lung, and prolonged dose-responsive decreases in serum and bronchoalveolar lavage sRAGE indicated effective pulmonary target engagement. The results support continued clinical development of inhaled therapeutic siRNA for pulmonary inflammation.

    Why it matters: First-in-human data show an inhaled siRNA can safely engage a lung target with minimal systemic exposure.

Aug 30Sun
  1. nature.com59

    Eplontersen with and without background transthyretin stabilizers in transthyretin amyloid cardiomyopathy: secondary analysis of a phase 3, randomized controlled trial

    A secondary analysis of the CARDIOTTRansform phase 3 randomized controlled trial found that eplontersen, a transthyretin-targeting antisense oligonucleotide, benefited patients with transthyretin amyloid cardiomyopathy who were not on transthyretin stabilizers at baseline, but showed no such effect in those already taking stabilizers. The result separates responders from non-responders by background therapy. Findings were presented at the 2026 ESC Congress.

    Why it matters: It identifies baseline transthyretin stabilizer use as a factor determining which cardiomyopathy patients benefit from eplontersen.

Aug 28Fri
  1. Lancet61

    Safety and lipoprotein(a)-lowering effects of Kylo-11, a non-canonical, long-duration small interfering RNA targeting lipoprotein(a): a first-in-human, randomised, double-blind, placebo-controlled, phase 1 trial.

    A single subcutaneous dose of Kylo-11, a very long-acting small interfering RNA targeting lipoprotein(a), cut median serum lipoprotein(a) by up to -97% at 48 weeks in healthy adults with elevated concentrations, with reductions of -53% at 9 mg and -96% in participants above 200 nmol/L given 225 mg. In the 70 dosed participants, adverse events occurred in 53%, mostly grade 1-2 and deemed unrelated; there were no serious or drug-related events. The findings support durable lipoprotein(a) lowering from one dose.

    Why it matters: It shows a single siRNA dose can durably lower lipoprotein(a) for up to 48 weeks in humans.

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