The initial dosing occurred at Second Affiliated Hospital, Zhejiang University School of Medicine, under the leadership of Dr. Zhi-Ying Wu, head of the Department of Medical Genetics/Center for Rare Diseases.

This Phase II trial is a randomized, double-blind, placebo-controlled, multiple ascending dose (MAD) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of repeated intrathecal injections of RAG-17  in patients with SOD1 mutations. The participating sites include Beijing Tiantan Hospital, Capital Medical University (led by Dr. Yi-Long Wang), The Second Affiliated Hospital, Zhejiang University School of Medicine (led by Dr.  Zhi-Ying Wu), West China Hospital of Sichuan University (led by Dr. Hui-Fang Shang), Fujian Medical University Union Hospital (led by Dr. Zhan-Yu Zou) and the First Affiliated Hospital, Sun Yat-sen University (led by Dr. Jing-Sheng Zeng) .

This milestone marks the progression into the Phase II stage of the study, building on the successful completion of the Phase I single ascending dose (SAD) portion. The transition to the Phase II MAD phase was supported by highly encouraging data from the SAD study, which demonstrated an exceptional safety profile and sustained biomarker modulation following a single injection, including significant reductions in CSF SOD1 protein and improvements in plasma neurofilament light chain (NfL) levels. These results validate the capability of our proprietary SCAD™ delivery platform for central nervous system (CNS) in humans.

Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics, emphasized Ractigen's dedication to advancing treatments for ALS: "The successful dosing of the first patient represents a significant milestone in our mission to develop transformative therapies for ALS. The positive results from the Phase I trial affirm the promise of RAG-17 and provide us with strong confidence in its potential to markedly improve the lives of patients suffering from SOD1-mutated ALS."

Dr. Zhi-Ying Wu shared optimistic expectations for the Phase II trial: "We are excited to be part of this important study. Based on the improvements we have observed clinically, we look forward to further investigating RAG-17's potential to enhance the quality of life for ALS patients as we progress."