NANTONG, China, July 29, 2026
Ractigen Therapeutics has successfully closed a financing round exceeding $31 million (over RMB 200 million) to accelerate the development of its clinical-stage small activating RNA (saRNA) therapeutics and proprietary extrahepatic RNA delivery platforms. The funding round was led by Guozhong Capital, with participation from IDG Capital, China Everbright Limited, Jolmo Capital, Win-Win Capital, SND Financial Holdings, and existing investor Longmen Capital. The company plans to use the proceeds to advance its Phase 2 oncology candidate RAG-01, progress the ALS program RAG-17, prepare the Duchenne muscular dystrophy (DMD) candidate RAG-18 for an Investigational New Drug (IND) application, and expand its innovative SCAD™ and LiCO™ delivery technologies. The investment strengthens Ractigen’s position as a developer of next-generation RNA medicines designed to activate genes rather than silence them, opening new possibilities for treating cancer, neurological disorders, and genetic diseases..
Funding to Accelerate Clinical Programs and RNA Innovation
The newly secured capital will primarily support RAG-01, the company’s lead Phase 2 saRNA therapy for non-muscle-invasive bladder cancer (NMIBC), which has already demonstrated encouraging clinical proof-of-concept and received U.S. FDA Fast Track Designation. The financing will also advance RAG-18, the world’s first saRNA program for Duchenne muscular dystrophy, toward an IND submission while supporting ongoing investigator-initiated studies. Additionally, RAG-17, Ractigen’s central nervous system (CNS) therapeutic for amyotrophic lateral sclerosis (ALS), will continue Phase 2 development following positive Phase 1 safety and biomarker findings published in Nature Medicine. The company also intends to strengthen its proprietary SCAD™ CNS delivery platform and LiCO™ systemic multi-tissue delivery technology, enabling RNA therapeutics to reach tissues beyond the liver, including muscle, heart, bladder, eye, and brain, overcoming one of the industry’s major technological challenges.
Proprietary saRNA Platform Targets Previously Untreatable Diseases
Unlike conventional RNA therapies such as siRNA and antisense oligonucleotides (ASOs) that silence disease-causing genes, Ractigen’s small activating RNA (saRNA) technology is designed to activate endogenous genes by targeting promoter regions and increasing protein production without modifying the genome. This novel mechanism expands therapeutic opportunities across genetic disorders, oncology, metabolic diseases, and rare conditions. The company’s carrier-free SCAD™ and LiCO™ delivery platforms have been developed to overcome extrahepatic delivery barriers, enabling efficient and durable delivery of RNA therapeutics to multiple organs. According to the company, LiCO™ can maintain therapeutic activity for up to nine months after a single administration, while SCAD™ has demonstrated clinical proof-of-concept for CNS delivery through the RAG-17 program. These technologies form the foundation of Ractigen’s growing pipeline and are expected to support the development of multiple first-in-class RNA medicines..
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Clinical Progress Strengthens Global Growth Strategy
Ractigen highlighted significant progress across its pipeline, including RAG-01, which achieved a preliminary 67% complete response rate in Phase 1 studies involving BCG-unresponsive high-risk bladder cancer patients. The company also reported encouraging biomarker improvements from RAG-18 in DMD patients and completed Phase 2 patient enrollment for the ALS program RAG-17 after demonstrating approximately 60% cerebrospinal fluid SOD1 protein reduction in Phase 1. Beyond internal development, Ractigen noted that it signed a strategic drug discovery and platform licensing agreement valued at more than $3 billion with an international pharmaceutical company during late 2025, highlighting increasing commercial interest in its RNA activation platform. Company executives stated that the latest financing validates Ractigen’s transition from scientific discovery to clinical-stage innovation and will support continued expansion of its first-in-class saRNA therapeutics targeting significant unmet medical needs worldwide.
Source:Ractigen Therapeutics press release
