Ractigen Therapeutics Announces Completion of Enrollment and First Dosing in Phase II Clinical Trial of RAG-17 for SOD1-ALS

NANTONG, China, Aug. 6, 2026 /PRNewswire/ -- Ractigen Therapeutics, a clinical-stage biotechnology company pioneering next-generation RNA therapeutics, today announced the completion of patient enrollment and initial dosing across all cohorts in its Phase II clinical trial evaluating RAG-17, an investigational siRNA therapy targeting superoxide dismutase 1 (SOD1) mutations in amyotrophic lateral sclerosis (ALS).

From the dosing of the first participant in the RAG-17 Phase II trial on January 13, 2026, to the completion of enrollment and first dosing of all participants today, this rapid progression across all five sites underscores both the strength of physician and patient engagement in the SOD1-ALS community and the operational execution of Ractigen's clinical team. With all participants now having received their initial dose, the study advances into the critical evaluation period, during which safety, biomarker, and functional data will continue to accumulate ahead of key regulatory interactions.

RAG-17 is developed using Ractigen's proprietary SCAD™ (Smart Chemistry-Aided Delivery) platform, with positive First-in-Human (FIH) Phase 1 clinical data recently published in Nature Medicine.

"Completing enrollment and first-dose administration in our Phase II MAD trial marks a pivotal achievement for Ractigen as we advance RAG-17 along a defined, accelerated regulatory pathway," said Long-Cheng Li, M.D., Founder and CEO of Ractigen Therapeutics. "Building upon robust Phase I SAD data that demonstrated remarkable CSF SOD1 protein suppression and plasma neurofilament light chain (NfL) reduction, this milestone brings us one step closer to delivering a transformative therapy to SOD1-ALS patients. We remain fully focused on executing our clinical strategy and engaging with regulatory authorities to accelerate the development of this meaningful treatment."

About RAG-17 Phase II

This Phase II trial (NCT06556394) 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).

About RAG-17

RAG-17 is an investigational siRNA therapeutic candidate designed using Ractigen's proprietary SCAD™ delivery platform technology to specifically target and silence the superoxide dismutase 1 (SOD1) gene mRNA. Mutations in the SOD1 gene cause a toxic gain-of-function and are a known cause of familial ALS. By reducing the production of the toxic mutant SOD1 protein, RAG-17 aims to slow or halt the progression of SOD1-ALS.

RAG-17 has obtained Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) and been selected for the CARE Program of the Center for Drug Evaluation (CDE), National Medical Products Administration (NMPA), which facilitates the accelerated development of rare disease therapies.

About ALS
Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease affecting nerve cells in the brain and spinal cord, leading to muscle weakness, paralysis, and ultimately death, typically within three to five years of diagnosis. SOD1 gene mutations account for approximately 10-20% of familial ALS cases and about 1-2% of sporadic ALS cases. There remains a critical unmet medical need for effective treatments that can slow or stop disease progression.

About Ractigen Therapeutics

Ractigen Therapeutics is a clinical-stage biopharmaceutical company innovating next-generation RNA therapeutics, with a primary focus on small activating RNAs (saRNAs) developed through its clinically validated RNA activation (RNAa) technology. Leveraging proprietary delivery platforms such as SCAD™, LiCO™, and GLORY™, Ractigen is advancing a robust pipeline addressing unmet medical needs in oncology, neurological diseases, and genetic disorders. Its versatile technologies also enable the rapid development of RNA-based solutions, including siRNAs, where applicable, to target life-threatening, fast-progressing conditions such as those in the CNS. Committed to scientific excellence and patient-centered innovation, Ractigen strives to transform healthcare through the power of RNA therapeutics. For more information, visit www.ractigen.com, and follow us on X and LinkedIn.

SOURCE Ractigen Therapeutics