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Nitto Denko Advances RNAi Fibrosis Drug into Phase Ib

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NEW YORK (GenomeWeb) – Japan's Nitto Denko announced this month that it has initiated a Phase Ib study of its siRNA-based fibrosis treatment ND-L02-s0201, just months after the drug successfully completed a Phase Ia safety study in healthy volunteers.

ND-L02-s0201 comprises siRNAs targeting heat shock protein 47, a collagen-specific chaperone required for the biosynthesis and secretion of collagen, and is delivered using vitamin A-coupled lipid nanoparticles. The drug's payload is based on siRNA technology Nitto Denko licensed from Quark Pharmaceuticals in 2010.

The open-label Phase Ib trial, which is being run in the US in collaboration with contract research firm RRD International, is set to enroll up to 24 patients with moderate to extensive hepatic fibrosis. Participants will receive doses of ND-L02-s0201 either once a week or twice a week for five consecutive weeks, and then be observed through week 24.

The study is designed to evaluate the safety and efficacy of the therapy.