Ractigen Therapeutics, a clinical-stage biotechnology company pioneering next-generation RNA therapeutics, has completed patient enrolment and initial dosing across all cohorts in its phase 2 clinical trial evaluating RAG-17, an investigational siRNA therapy targeting superoxide dismutase 1 (SOD1) mutations in amyotrophic lateral sclerosis (ALS).
From the dosing of the first participant in the RAG-17 phase 2 trial on 13 January, to the completion of enrolment and first dosing of all participants today, this rapid progression across all five sites underscores both the strength of physician and patient engagement in the SOD1-ALS community and the operational execution of Ractigen’s clinical team. With all participants now having received their initial dose, the study advances into the critical evaluation period, during which safety, biomarker, and functional data will continue to accumulate ahead of key regulatory interactions.
RAG-17 is developed using Ractigen’s proprietary SCAD (Smart Chemistry-Aided Delivery) platform, with positive First-in-Human (FIH) phase 1 clinical data recently published in Nature Medicine.
