Ractigen Therapeutics, a clinical-stage biotechnology company pioneering small activating RNA (saRNA) therapeutics and advanced extrahepatic delivery systems, has closed a new financing round exceeding $31 million (over RMB 200 million). 

The round was led by Guozhong Capital, with participation from IDG Capital, China Everbright Limited, Jolmo Capital, Win-Win Capital, and SND Financial Holdings. Existing shareholder Longmen Capital participated for its third consecutive round. 

Proceeds will primarily accelerate the clinical development of Ractigen’s saRNA assets and validate its proprietary extrahepatic delivery platforms. Key priorities include the continuation to advance the company’s self-amplifying RNA (saRNA) pipeline across multiple therapeutic areas. Its lead oncology candidate, RAG-01, is also progressing through Phase II clinical trials in non-muscle-invasive bladder cancer (NMIBC) following positive clinical proof-of-concept data and the award of US FDA Fast Track Designation. Moreover, RAG-18, a systemic saRNA therapy for Duchenne muscular dystrophy (DMD), is advancing through investigator-initiated trials (IITs) as the company prepares for an Investigational New Drug (IND) filing. Meanwhile, its central nervous system (CNS) candidate, RAG-17, is moving into Phase II clinical trials for amyotrophic lateral sclerosis (ALS) after Phase I safety and biomarker validation results were published in Nature Medicine. Alongside its clinical pipeline, the company is expanding its proprietary extrahepatic delivery platforms, one for CNS applications and another for systemic multi-tissue delivery, to target diseases beyond the liver while continuing to pioneer saRNA technology to unlock gene activation for unmet medical needs.  

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 (siRNA and ASO).  

Discovered by Ractigen’s founder, RNAa utilises 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. 

Dr. Long-Cheng Li, Founder and Chief Executive Officer of Ractigen Therapeutics, said: “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. Over nearly two decades, we progressed from discovering RNA activation to solving extrahepatic delivery bottlenecks and translating multiple assets into positive human clinical data. Moving forward, Ractigen will continue expanding the boundaries of RNA technology, accelerating clinical translation to deliver transformative, First-in-Class therapeutics to patients worldwide.” 

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