Groundbreaking Breakthrough: RiboCarna Develops Duplex RNA Delivery Technology for Extrahepatic Tissues Enabling Potent and Durable Gene Silencing

Recently,RactigenAnnounce,Its proprietary SCAD(Intelligent Chemical-Assisted Delivery)The platform's detailed research findings were published online on October 30, 2025, in the peer-reviewed journal *Molecular Therapy: Nucleic Acids*.（Molecular Therapy - Nucleic Acids）。The paper reports a double-strandedRNTherapy A Delivered to the Central Nervous System（CNS）And other novel modular platforms for challenging extrahepatic tissues.

The paper titled "SCAD: A Modular Platform for Efficient Delivery of Duplex RNA to the CNS and Beyond" elaborates onHow does the SCAD platform overcome the long-standing limitations of double-stranded RNA(such as siRNA)Application Delivery Barriers. This technology involves the use of therapeutic double-stranded RNA(such as siRNA)With a non-targeting auxiliary oligonucleotide（ACO）Connect to form a unique ligand-free structure, thereby enhancing binding with endogenous proteins, promoting extensive tissue distribution and cellular uptake without relying on complex lipids or antibodies.

Dr. Longcheng Li, Founder and CEO of RactigenSaid: "Data published in Molecular Therapy - Nucleic Acids indicate that the SCAD platform is the development of a new generation of double-stranded RNA.(such as siRNA)A powerful and versatile engine for drug development. Our modular system is designed to not only enhance efficiency but also streamline production processes and facilitate scalability. This research achievement validates the core innovation of our technology platform, laying the foundation for our rapidly advancing clinical pipeline and paving the way for addressing extrahepatic challenges.「Incurable」"The target has paved the way."

The study presents a large amount of preclinical data validating the SCAD platform, highlighting several key achievements:

• Potent and Long-lasting Gene Silencing in the Central Nervous System:Single intracerebroventricular injection in rodents（ICV）After injection of SCAD-siRNA, the drug was widely distributed throughout the central nervous system and achieved deep mRNA knockdown for more than five months.

• Extensive applicability in extrahepatic tissues:The paper demonstrates the versatility of the SCAD platform, successfully achieving targeted delivery and significant gene silencing in tissues such as the lungs, eyes, and joints through local administration.

• Good Safety:In extensive safety and toxicology evaluations, SCAD-siRNA demonstrated good tolerability. Particularly in the central nervous system, it did not induce markers of immune activation.（Iba1, Gfap）expression, and no obvious clinical adverse reactions or laboratory index abnormalities were observed in toxicological studies of rodents.

• Simplified Production Process:The entire SCAD molecule is constructed entirely using standard solid-phase chemical synthesis, avoiding the complex production processes and high costs associated with high molecular weight bioconjugate drugs.

The SCAD platform provides the underlying enabling technology for Ractigen's increasingly robust clinical-stage therapeutic pipeline, advancing its clinical development for severe neurodegenerative diseases.(Particularly Amyotrophic Lateral Sclerosis, ALS)Multiple projects. An IIT clinical trial initiated by researchers for RAG-17 in treating SOD1-ALS patients（NCT05903690）Already completed, the trial demonstrated very favorable safety and encouraging preliminary efficacy in an open-label, dose-escalation study. Building on the success of this study, a multi-center, randomized, double-blind, placebo-controlled Phase 1 clinical trial of RAG-17 for the treatment of SOD1-ALS patients has been initiated.（NCT06556394）In progress.

Chief Technology Officer of Ractigen, Moorim Kang"This published achievement provides a detailed roadmap for the rigorous scientific research and systematic optimization behind the SCAD platform. It achieves potent and durable activity across various central nervous system cells, combined with favorable safety, offering us a robust and reproducible path to creating revolutionary drugs for treating devastating neurological disorders."

Original link:

Kang M, Lin W-H, Li Y, et al. SCAD: A Modular Platform for Efficient Delivery of Duplex RNA to the CNS and Beyond. Molecular Therapy Nucleic Acids 2025:102757. 

https://doi.org/10.1016/j.omtn.2025.102757

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