Skip to main content
Premium Trial:

Request an Annual Quote

Nitto Denko Advances RNAi Fibrosis Drug into Phase Ib

Premium

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.

The Scan

Enzyme Involved in Lipid Metabolism Linked to Mutational Signatures

In Nature Genetics, a Wellcome Sanger Institute-led team found that APOBEC1 may contribute to the development of the SBS2 and SBS13 mutational signatures in the small intestine.

Family Genetic Risk Score Linked to Diagnostic Trajectory in Psychiatric Disorders

Researchers in JAMA Psychiatry find ties between high or low family genetic risk scores and diagnostic stability or change in four major psychiatric disorders over time.

Study Questions Existence of Fetal Microbiome

A study appearing in Nature this week suggests that the reported fetal microbiome might be the result of sample contamination.

Fruit Fly Study Explores Gut Microbiome Effects on Circadian Rhythm

With gut microbiome and gene expression experiments, researchers in PNAS see signs that the microbiome contributes to circadian rhythm synchronicity and stability in fruit flies.