#China #ALS #Ractigen Therapeutics #RAG-17 #Nantong and Suzhou

Ractigen Therapeutics Advances ALS Treatment with RAG-17

Ractigen Therapeutics, a pioneering clinical-stage pharmaceutical company, announced the notable achievement of dosing the first patient in its Phase I clinical trial for RAG-17. This innovative therapy targets Amyotrophic Lateral Sclerosis (ALS) linked with mutations in the superoxide dismutase 1 (SOD1) gene. The trial is being held at the Second Affiliated Hospital of Zhejiang University School of Medicine, marking a significant step forward in ALS research and treatment.

The Phase I study is structured as a randomized, double-blind, placebo-controlled trial. Its primary objective is to assess the safety and tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of RAG-17 in individuals diagnosed with SOD1-ALS. The trial is spearheaded by prominent figures in the field, including Dr. Yilong Wang from Beijing Tiantan Hospital of Capital Medical University and Dr. Zhiying Wu from the Second Affiliated Hospital, with support from Dr. Huifang Shang at West China Hospital of Sichuan University.

Dr. Long-Cheng Li, the Founder, and CEO of Ractigen Therapeutics, emphasized the importance of this milestone, stating, "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." This statement reflects the company’s steadfast dedication to innovating RNA-based therapies that may dramatically affect the lives of patients suffering from severe conditions like ALS.

RAG-17 has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) in March 2023, and subsequently, the FDA approved its Investigational New Drug (IND) application. Furthermore, in May 2024, the IND was validated by the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA). Recent Investigator-Initiated Trials (IIT) confirmed the promising nature of RAG-17, demonstrating its potential as an impactful treatment for ALS patients.

Background on RAG-17

RAG-17 is an siRNA specifically engineered to inhibit the SOD1 gene, which is associated with pathogenic mutations in ALS patients. This unique therapy employs Ractigen's proprietary SCAD™ delivery technology, combining siRNA with an accessory oligonucleotide (ACO) to enhance delivery capabilities into the central nervous system (CNS). Preclinical research, including investigations utilizing the hSOD1G93A mouse model, has shown RAG-17's impressive efficacy in improving motor functions and extending survival.

The results from IIT indicate that RAG-17 is well tolerated at all administered doses, encountering mild adverse effects, with comprehensive safety assessments affirming its positive profile.

Understanding ALS

ALS is a severe and progressive neurodegenerative condition with no established cure. The disease significantly affects life expectancy, as many patients experience respiratory failure within three to five years following diagnosis. Early signs may include muscle cramps, twitching, and weakness, which evolve into mobility difficulties and paralysis, ultimately leading to death.

Mutations in the SOD1 gene contribute to approximately 20% of familial ALS cases and around 5% of sporadic ALS instances, underscoring the critical need for effective therapies like RAG-17.

Ractigen Therapeutics Overview

Ractigen Therapeutics leads the way in developing small activating RNA (saRNA) drugs and employs RNA activation (RNAa) mechanisms to upregulate endogenous gene expression. The company focuses on targeting specific genes to restore normal protein functions, paving new paths for treating diseases previously unmanageable through conventional approaches. For more detailed information about their groundbreaking work and the potential of saRNA, visit their official website at www.ractigen.com.