Funding led by Guozhong Capital will accelerate Phase 2 development for saRNA candidate RAG-01 and CNS asset RAG-17, advance systemic saRNA program RAG-18 toward IND, and expand extrahepatic RNA delivery technologies.
NANTONG, China, July 29, 2026 /PRNewswire/ — Ractigen Therapeutics, a clinical-stage biotechnology company pioneering small activating RNA (saRNA) therapeutics and advanced extrahepatic delivery systems, today announced the successful closing of 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:
- Advancing lead saRNA oncology program (RAG-01): Accelerating Phase 2 clinical trials in non-muscle-invasive bladder cancer (NMIBC) following positive clinical proof-of-concept and U.S. FDA Fast Track Designation.
- Driving systemic saRNA asset toward IND (RAG-18): Advancing ongoing investigator-initiated trials (IIT) and preparing for IND filing for Duchenne muscular dystrophy (DMD).
- Progressing CNS clinical asset (RAG-17): Moving forward with Phase 2 clinical trials in amyotrophic lateral sclerosis (ALS) following Phase 1 safety and biomarker validation published in Nature Medicine.
- Expanding extrahepatic delivery platforms: Continuously enhancing the company’s proprietary SCAD™ (CNS) and LiCO™ (systemic multi-tissue) delivery technologies to unlock new therapeutic targets outside the liver.
Pioneering saRNA Technology: Unlocking Gene Activation for Unmet 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).
As the global pioneer in RNA activation (RNAa), Ractigen 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: Proprietary Delivery Systems
To deliver saRNA and targeted oligonucleotide payloads beyond the liver, Ractigen engineered two complementary, carrier-free delivery platforms:
- SCAD™ (CNS delivery platform): Utilizes an accessory oligonucleotide (ACO)-enabled self-delivery mechanism to cross central nervous system barriers, achieving clinical proof-of-concept for safety and target engagement via RAG-17.
- LiCO™ (Systemic multi-tissue platform): Conjugates specialized lipids to oligonucleotides via proprietary SDL™ linkers. LiCO™ enables 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 & Commercial Momentum
Ractigen has successfully 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. In Phase 1 trials, RAG-01 achieved a preliminary any-time 67% complete response (CR) rate in BCG-unresponsive high-risk NMIBC patients.
- RAG-18: The world’s first saRNA program targeting Duchenne muscular dystrophy (DMD) and a major milestone in breaching extrahepatic muscle delivery barriers. Currently in investigator-initiated trials (IIT), RAG-18 delivers saRNA systemically to upregulate Utrophin expression, demonstrating clear target engagement, marked biomarker reductions, and histopathological improvements in muscle tissue in DMD patients.
- RAG-17: A clinical-stage, CNS-targeted oligonucleotide therapeutic for amyotrophic lateral sclerosis (ALS). Phase 1 clinical data demonstrated robust SOD1 protein knockdown (~60% CSF SOD1 reduction) and a favorable safety profile, with Phase 2 patient enrollment now fully completed.
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.
Executive & Investor Commentary
Dr. Long-Cheng Li, Founder and Chief Executive Officer of Ractigen Therapeutics:
“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. 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.”
Investment Team, Guozhong Capital:
“Ractigen Therapeutics is dedicated to pioneering next-generation RNA therapeutics globally. Its proprietary extrahepatic delivery platforms breach core bottlenecks across the field. RAG-17 is a pioneering CNS-targeting oligonucleotide therapy with Phase 1 results featured in top-tier peer-reviewed journals, while RAG-01 marks a historic clinical application of saRNA technology in oncology. We look forward to supporting Ractigen as its saRNA platform expands further into genetic, oncologic, and metabolic diseases.”
Haining Wang, Founding Partner of Longmen Capital:
“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. We will continue leveraging our industry and capital market resources to help Ractigen bring saRNA therapies to patients globally.”
About Ractigen Therapeutics
Ractigen Therapeutics is a clinical-stage biopharmaceutical company innovating next-generation RNA therapeutics, with a primary focus on saRNAs developed through its clinically validated RNA activation (RNAa) technology. Leveraging proprietary extrahepatic delivery platforms such as SCAD™, LiCO™, and GLORY™, Ractigen is advancing a robust pipeline addressing severe unmet medical needs in oncology, neurological diseases, and genetic disorders. Committed to scientific excellence and patient-centered innovation, Ractigen strives to transform healthcare through the power of targeted RNA activation and delivery. For more information, visit www.ractigen.com.
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