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Small Interfering RNAs: mRNA Targeted in Treatment of Disease

“Programmable [small interfering RNA (siRNA)] therapeutics have demonstrated effectiveness for treating an increasing number of rare conditions,” write authors of a review article. “The approach is more commonly being used to target common conditions with mechanisms amenable to siRNA-based interventions. Much remains to be learned about the real-world effectiveness, adverse effects, and value of this novel approach for treating disease.”

FDA already approves 5 siRNA drugs: inclisiran for hypercholesterolemia, givosiran for acute hepatic porphyria, lumasiran for primary hyperoxaluria type 1, and patisiran and vutrisiran for hereditary transthyretin-mediated amyloidosis in adults. “The potential of twice-yearly dosing is an advantage of siRNAs,” the authors write. “This is exemplified by the recent approval of inclisiran by the US Food and Drug Administration, which requires twice-yearly injections to downregulate PCSK9 and lower circulating cholesterol levels with few adverse effects. Inclisiran was initially developed for patients with statin-resistant hypercholesterolemia, but it could become a primary treatment for any patient with high cholesterol since suboptimal adherence to statins is an issue. Zilebesiran, an siRNA that targets liver-expressed angiotensinogen, is being explored for treatment of hypertension. The shift in application of siRNA drugs from treating rare genetic conditions to common diseases with well-characterized mechanisms amenable to siRNA targeting may have long-term implications for general clinical medicine.”

Source: JAMA