Ractigen Therapeutics Closes $31M+ Financing to Advance saRNA Pipeline and Extrahepatic Delivery Platforms
Key Insights
Ractigen Therapeutics closed over $31 million in new financing led by Guozhong Capital (search) to accelerate its clinical-stage small activating RNA (saRNA) pipeline.
Proceeds will fund Phase 2 trials for RAG-01 in NMIBC and RAG-17 in ALS, and advance RAG-18 toward IND filing for Duchenne muscular dystrophy (search).
RAG-01 achieved a 67% complete response rate in Phase 1 for BCG-unresponsive NMIBC, while RAG-17 demonstrated ~60% CSF SOD1 (search) reduction in ALS patients.
Ractigen Therapeutics, a clinical-stage biotechnology company pioneering small activating RNA (saRNA) therapeutics, announced the successful closing of a new financing round exceeding $31 million (over RMB 200 million). The round was led by Guozhong Capital (search), with participation from IDG Capital, China Everbright Limited, Jolmo Capital, Win-Win Capital, and SND Financial Holdings. Existing shareholder Longmen Capital (search) participated for its third consecutive round, underscoring sustained investor confidence in the company's technology platform.
The proceeds will primarily accelerate clinical development of Ractigen's saRNA assets and validate its proprietary extrahepatic delivery platforms across multiple therapeutic areas.
Pioneering RNA Activation Technology
Oligonucleotide therapeutics represent the third major wave of biopharmaceutical innovation following small molecules and monoclonal antibodies. However, traditional RNA therapeutics have been largely limited to gene-silencing approaches such as siRNA and ASO. Ractigen, as the global pioneer in RNA activation (RNAa), was founded to redefine genetic medicine by enabling precise gene activation.
Discovered by Ractigen's founder, RNAa utilizes saRNAs targeting gene promoter regions to upregulate endogenous protein expression at the transcriptional level without altering the genome. This unique mechanism unlocks previously undruggable therapeutic targets, expanding treatment possibilities across genetic disorders, haploinsufficiencies, cancer, and metabolic diseases.
Breaching Extrahepatic Barriers with Proprietary Delivery Systems
To deliver saRNA and targeted oligonucleotide payloads beyond the liver, Ractigen engineered two complementary, carrier-free delivery platforms. The SCAD™ platform utilizes an accessory oligonucleotide-enabled self-delivery mechanism to cross central nervous system barriers, achieving clinical proof-of-concept for safety and target engagement via RAG-17. The LiCO™ platform conjugates specialized lipids to oligonucleotides via proprietary SDL™ linkers, enabling durable, carrier-free delivery to muscle, heart, bladder, and eye tissues, sustaining therapeutic activity for up to nine months per single administration with simplified, cost-effective manufacturing.
Clinical Pipeline Progress
Ractigen has translated its saRNA and extrahepatic delivery technologies into three differentiated clinical-stage assets. RAG-01, the world's first conjugate-delivered saRNA therapeutic to demonstrate clinical proof-of-concept in oncology, achieved a preliminary any-time 67% complete response rate in BCG-unresponsive high-risk non-muscle-invasive bladder cancer (search) patients during Phase 1 trials. The program has received U.S. FDA Fast Track Designation and will now advance into Phase 2.
RAG-17, a CNS-targeted oligonucleotide therapeutic for amyotrophic lateral sclerosis (search), demonstrated robust SOD1 (search) protein knockdown with approximately 60% CSF SOD1 reduction and a favorable safety profile in Phase 1. Phase 2 patient enrollment is now fully completed, with Phase 1 results published in Nature Medicine.
RAG-18, the world's first saRNA program targeting Duchenne muscular dystrophy (search), is currently in investigator-initiated trials. The program delivers saRNA systemically to upregulate Utrophin (search) expression, demonstrating clear target engagement, marked biomarker reductions, and histopathological improvements in muscle tissue in DMD patients. The company is preparing for an IND filing.
Strategic and Commercial Momentum
Reflecting the global commercial value of its platform, Ractigen entered into a strategic drug discovery and platform licensing collaboration with a publicly listed overseas pharmaceutical company in late 2025, valued at over $3 billion.
Dr. Long-Cheng Li, Founder and Chief Executive Officer of Ractigen Therapeutics, stated: "Closing this financing round reflects strong capital market validation of Ractigen's critical leap from pioneering scientific discovery to human clinical proof-of-concept. Over the past decade, the oligonucleotide field achieved tremendous commercial success in liver-targeted silencing, yet extrahepatic delivery and gene activation remained unaddressed global frontiers."
The Investment Team at Guozhong Capital (search) added: "Ractigen Therapeutics is dedicated to pioneering next-generation RNA therapeutics globally. Its proprietary extrahepatic delivery platforms breach core bottlenecks across the field."
Haining Wang, Founding Partner of Longmen Capital (search), noted: "We have been a long-term believer in Ractigen since our initial investment in 2021, and this marks our third consecutive round of support. Their clinically validated extrahepatic delivery systems and highly differentiated saRNA pipelines create an unrivaled competitive moat."