Ractigen Therapeutics Begins Patient Dosing in Phase I Trial of RAG-17 in SOD1-ALS

Ractigen Therapeutics, a clinical-stage pharmaceutical company dedicated to developing RNA-based innovative therapies, 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 US 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.

RAG-17 is a siRNA specifically designed to suppress the SOD1 gene in ALS patients with pathogenic mutations. Utilizing Ractigen’s proprietary SCADTM delivery platform, RAG-17 conjugated siRNA with an accessory oligonucleotide (ACO) for enhanced delivery into the central nervous system (CNS). Preclinical studies, including those using the hSOD1G93A mouse model, have demonstrated the remarkable therapeutic efficacy of RAG-17 in ameliorating motor function and prolonging survival. The IIT results showed that intrathecally administered RAG-17 was well-tolerated across all dose levels with only mild adverse events and comprehensive safety evaluations confirming its favourable profile.

ALS, a severe neurodegenerative disease with no cure, significantly reduces life expectancy, with most patients succumbing to respiratory failure within 3-5 years of diagnosis. Initial symptoms typically include muscle cramps, twitching, and weakness. These symptoms progress to difficulties with movement and speech, the need for assisted breathing, paralysis, and ultimately death. Mutations in the SOD1 gene account for approximately 20% of familial ALS and 5% of the sporadic ALS cases.

A leader in small activating RNA (saRNA) drug development, Ractigen Therapeutics is at the forefront of developing saRNA drugs utilizing RNA activation (RNAa) mechanism to up-regulate endogenous gene expression.

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