Small Interfering RNA

Transthyretin-directed RNA Interaction

Amyloid Neuropathies, Familial

<section class="Section" data-sectioncode="34089-3"><a name="S11"></a><a name="section-8"></a><h1>11 DESCRIPTION</h1>ONPATTRO contains patisiran, a double-stranded small interfering ribonucleic acid (siRNA), formulated as a lipid complex for delivery to hepatocytes. Patisiran specifically binds to a genetically conserved sequence in the 3' untranslated region (3'UTR) of mutant and wild-type transthyretin (TTR) messenger RNA (mRNA). The structural formula is: <img alt="Chemical Structure" src="onpattro-01.jpg"/>A, adenosine; C, cytidine; G, guanosine; U, uridine; Cm, 2'-O-methylcytidine; Um, 2'-O-methyluridine; dT, thymidine ONPATTRO is supplied as a sterile, preservative-free, white to off-white, opalescent, homogeneous solution for intravenous infusion in a single-dose glass vial. Each 1 mL of solution contains 2 mg of patisiran (equivalent to 2.1 mg of patisiran sodium). Each 1 mL also contains 6.2 mg cholesterol USP, 13.0 mg (6Z,9Z,28Z,31Z)-heptatriaconta-6,9,28,31-tetraen-19-yl-4-(dimethylamino) butanoate (DLin-MC3-DMA), 3.3 mg 1,2-distearoyl-sn-glycero-3-phosphocholine (DSPC), 1.6 mg α-(3'-{[1,2-di(myristyloxy)propanoxy] carbonylamino}propyl)-ω-methoxy, polyoxyethylene (PEG2000-C-DMG), 0.2 mg potassium phosphate monobasic anhydrous NF, 8.8 mg sodium chloride USP, 2.3 mg sodium phosphate dibasic heptahydrate USP, and Water for Injection USP. The pH is ~7.0.The molecular formula of patisiran sodium is C412 H480 N148 Na40 O290 P40 and the molecular weight is 14304 Da.</section>

Onpattro

patisiran

5 ML patisiran lipid complex 2 MG/ML Injection

5 ML patisiran lipid complex 2 MG/ML Injection [Onpattro]

patisiran lipid complex

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