Suzhou-based China-US Ragon Announces Phase 2 Clinical Trial Initiation of siRNA Therapy RAG-17 for SOD1-Mutant ALS

On January 13, Ractigen announced that its self-developedTargetedSOD1Innovative siRNA Therapy for Mutant Amyotrophic Lateral Sclerosis (ALS, Commonly Known as "Lou Gehrig's Disease")RAG-17 has completed the first patient dosing in the Phase 2 clinical trial at the Second Affiliated Hospital of Zhejiang University School of Medicine. This multi-center Phase 2 clinical trial is a multiple-dose, dose-escalation (MAD) randomized, double-blind, placebo-controlled study aimed at evaluating theSOD1Safety/Tolerability, PK, PD, and Preliminary Efficacy of Intrathecal Injection of RAG-17 Repeated Dosing in ALS Subjects with Gene Mutations.

ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, causing muscle weakness, paralysis, and ultimately leading to death within 3 to 5 years after diagnosis.SOD1Gene mutations account for approximately 10-20% of familial ALS cases and 1-2% of sporadic ALS cases. Currently, there is still an urgent clinical need for treatments that can effectively slow down or halt the progression of ALS to improve patients' quality of life and prolong survival.

RAG-17 is an innovative siRNA drug independently developed by Ractigen Therapeutics, leveraging the company's SCAD™ (Smart Chemical-Assisted Delivery) platform technology. The drug aims to specifically and efficiently target and silenceSOD1mRNA expression of genes for the treatment of carriersSOD1ALS with Gene Mutation.SOD1Toxic gain-of-function caused by gene mutations is an important pathogenic factor in familial ALS. Through efficient inhibitionSOD1By modulating gene expression and reducing the production of toxic SOD1 protein, RAG-17 aims to protect neuronal function, thereby delaying or halting disease progression in SOD1-ALS.

Ractigen News Release: This milestone marks the official entry of the study into Phase 2. The initiation of the Phase 2 MAD stage is based on the positive results from the successfully completed Phase 1 Single Ascending Dose (SAD) study. SAD study data demonstrated favorable safety and sustained biomarker modulation following a single dose of RAG-17.Including a significant reduction in cerebrospinal fluid (CSF) SOD1 protein and improvement in plasma neurofilament light chain (NfL) levels.These results strongly validate the company's SCAD™ delivery platform's ability to achieve central nervous system (CNS) targeted delivery in humans.

References:
[1]Ractigen's RAG-17 Administers First Patient in Phase II Clinical Trial for SOD1 ALS, Marking a New Chapter in Neurodegenerative Disease Treatment. From https://mp.weixin.qq.com/s/Wt_fckYUxrk-YyUpVMoLvA

Disclaimer: This article is for information exchange purposes only. The views expressed in the article do not represent the position of WuXi AppTec, nor does it indicate that WuXi AppTec supports or opposes the views mentioned. This article is not a recommendation for treatment plans. For guidance on treatment options, please visit a正规 hospital.