SUZHOU, China, November 17, 2025 – Ractigen Therapeutics, a clinical-stage biotechnology company, today announced the publication of a comprehensive study detailing its proprietary Smart Chemistry Aided Delivery (SCAD) platform. The paper, published online in the peer-reviewed journal Molecular Therapy – Nucleic Acids on October 30, 2025, describes a novel and highly adaptable platform for delivering duplex RNA therapeutics to the central nervous system (CNS) and other challenging extra-hepatic tissues.
The publication, titled “SCAD: A Modular Platform for Efficient Delivery of Duplex RNA to the CNS and Beyond” outlines how the SCAD platform overcomes critical delivery hurdles that have historically limited the application of RNA interference (RNAi). The technology works by conjugating a therapeutic duplex RNA (such as an siRNA) to a non-targeting accessory oligonucleotide (ACO). This unique ligand‑free architecture enhances binding to endogenous proteins, facilitating broad tissue distribution and cellular uptake without relying on complex lipids or antibodies.
“The data published in Molecular Therapy – Nucleic Acids demonstrate that SCAD is a powerful, versatile engine for developing a new generation of duplex RNA (e.g., siRNA) drugs,” said Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. “We have engineered a modular system that is not only highly effective but is also designed for streamlined, scalable manufacturing. This publication validates the core innovation that is already enabling our clinical pipeline and opens the door to pursuing a wide range of previously ‘undruggable’ targets outside the liver.”
- Potent and Durable CNS Gene Silencing: A single intracerebroventricular (ICV) injection of a SCAD-siRNA in rodents resulted in broad distribution throughout the CNS and profound mRNA knockdown that was sustained for over five months.
- Broad Extra-Hepatic Applicability: The paper demonstrates SCAD’s versatility with successful, targeted delivery and significant gene silencing in the lung, eye, and joints following local administration.
- Favorable Safety Profile: In extensive safety and toxicology evaluations, SCAD-siRNA was well-tolerated. Notably, it did not induce immune activation markers (Iba1, Gfap) in the CNS and showed no significant adverse clinical signs or laboratory abnormalities in rat toxicology studies.
- Simplified Manufacturing: The entire SCAD construct is synthesized using standard, solid-phase chemistry, avoiding the complex manufacturing processes and high costs associated with high-molecular-weight biologic conjugates.
The SCAD platform provides the foundational technology for Ractigen’s growing pipeline of clinical-stage therapeutics targeting severe neurodegenerative diseases, particularly amyotrophic lateral sclerosis (ALS). An initial investigator-initiated Phase 1 trial (NCT05903690) of RAG-17 in patients with SOD1-ALS has been completed, demonstrating a very favorable safety profile and encouraging preliminary efficacy. Building on the success of this study, a multicenter, randomized, double-blind, placebo-controlled Phase 1 trial of RAG-17 (NCT06556394) is currently ongoing.
“This publication provides a detailed roadmap of the rigorous science and methodical optimization behind the SCAD platform,” said Moorim Kang, Chief Technology Officer of Ractigen. “Its ability to achieve potent, long-lasting activity across multiple CNS cell types, combined with a favorable safety profile, gives us a powerful and repeatable approach to creating transformative medicines for devastating neurological disorders.”
Link to the publication:
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
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.
