Ractigen Therapeutics, a clinical-stage pharmaceutical company dedicated to developing RNA-based innovative therapies, today announced the successful dosing of the first patient in the Phase I clinical trial of RAG-17, an innovative siRNA therapy targeting Amyotrophic Lateral Sclerosis (ALS) associated with superoxide dismutase 1 (SOD1) gene mutations at Second Affiliated Hospital of Zhejiang University School of Medicine.

The Phase I clinical study is a randomized, double-blind, placebo-controlled trial designed to evaluate the safety/tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of RAG-17 in patients with SOD-ALS. The trial is being conducted under the leadership of Dr. Yilong Wang at Beijing Tiantan Hospital of Capital Medical University and Dr. Zhiying Wu at the Second Affiliated Hospital of Zhejiang University School of Medicine, respectively, with cooperation from Dr. Huifang Shang at West China Hospital of Sichuan University.

"The first patient dosed in the RAG-17 trial marks a pivotal milestone in our mission to combat ALS, one of the most devastating neurodegenerative diseases," said Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. "This achievement underscores our unwavering commitment to advancing RNA-based therapies that have the potential to transform the lives of patients and families affected by rare and severe conditions like ALS." 

RAG-17 received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) in March 2023, followed by the FDA's clearance of its Investigational New Drug (IND) application. In May 2024, the IND was approved by the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA). Most recently, in November 2024, an Investigator-Initiated Trial (IIT) of RAG-17 delivered promising clinical data, further validating its potential as a transformative therapy. The promising IIT findings have been presented at the 27th National Conference of Neurology, Neuroscience 2024, and the 35th International Symposium on ALS/MND, earning strong validation and interest from the global scientific community.