Ractigen Starts China Trials of siRNA Candidate for ALS Patients

Jiangsu Ractigen Therapeutics, a novel oligonucleotide biopharma, has dosed the first patient in the first-in-human trial of rag-17, an siRNA candidate, that enrolled ALS patients with sod1 mutation. Rag-17 is a siRNA candidate designed to provide potent and durable knockdown of the sod1 protein in the central nervous system to prevent degradation of motor neurons and delay disease progression. Although delivery to tissue has been historically difficult for siRNA candidates, Rag-17 uses a novel smart chemistry-aided delivery (scad) conjugate that facilitates broad biodistribution and tissue uptake throughout the CNS.

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