#China #RNA Delivery #Ractigen Therapeutics #Nantong #saRNA

Ractigen Therapeutics' Strategic Financing

Ractigen Therapeutics, a prominent player in the biotechnology sector, has recently announced the closing of a significant financing round, amassing over $31 million (around RMB 200 million). This latest investment aims to accelerate the company’s development of small activating RNA (saRNA) therapeutics and its innovative extrahepatic delivery systems.

Funding Details

Led by Guozhong Capital, this financing round also saw participation from notable investors including IDG Capital, China Everbright Limited, Jolmo Capital, and Win-Win Capital, while existing shareholder Longmen Capital continued its support for the third consecutive time.

The funds raised will primarily be allocated towards fast-tracking clinical programs. Specifically, Ractigen plans to advance its lead saRNA oncology product, RAG-01, towards Phase 2 trials after successfully achieving positive proof-of-concept results. RAG-01 has garnered U.S. FDA's Fast Track Designation for its promising potential in treating non-muscle-invasive bladder cancer (NMIBC).

Additionally, the financing will help in moving the systemic saRNA asset RAG-18 closer to an Investigational New Drug (IND) filing, aimed at treating Duchenne muscular dystrophy (DMD). RAG-18 has shown encouraging results in ongoing investigator-initiated trials, aiming to boost Utrophin expression, which has crucial implications for muscle dystrophies.

Ractigen's CNS-targeted program, RAG-17, which is currently targeting amyotrophic lateral sclerosis (ALS), will also benefit from this funding. The company recently completed Phase 1 trials, which confirmed the safety and potential of the treatment, and it is now gearing up for full Phase 2 clinical trials.

Pioneering saRNA Technology

Ractigen Therapeutics positions itself at the forefront of RNA therapeutics by focusing on gene activation rather than gene silencing, a departure from traditional RNA approaches. The company's founder’s discovery of RNA activation (RNAa) empowers precise gene expression modulation. By utilizing saRNAs to target gene promoters, Ractigen activates the expression of genes without altering the underlying genetic material - a game-changer in tackling previously untreatable conditions.

The company is dedicated to addressing severe medical needs across various domains, including oncology, genetic disorders, and metabolic diseases, through its advanced saRNA technologies.

Proprietary Delivery Platforms

To maximize the efficacy of its saRNA molecules, Ractigen has developed two innovative delivery systems that facilitate the distribution of these therapeutics beyond the liver - a common hurdle in delivery methodologies.

• - SCAD™ (CNS Delivery Platform): This platform employs a self-delivery mechanism, enabling saRNA to cross the complex barriers of the central nervous system, thereby targeting neurological diseases effectively.
• - LiCO™ (Systemic Multi-Tissue Platform): By conjugating specialized lipids to oligonucleotides, LiCO™ ensures long-lasting, carrier-free delivery of saRNA across various tissues, including muscle, heart, bladder, and eye - significantly enhancing therapeutic options for patients.

Momentum in Clinical Development

Ractigen has successfully translated its groundbreaking technologies into several clinical-stage products:
1. RAG-01: The first of its kind, RAG-01 has exhibited a striking 67% complete response rate in Phase 1 trials among patients with BCG-unresponsive high-risk NMIBC.
2. RAG-18: Setting a milestone in DMD therapy, RAG-18 showcases significant target engagement and considerable biomarker performance improvements during its trials, demonstrating the potential for systemic saRNA applications.
3. RAG-17: Focused on ALS, RAG-17's Phase 1 data revealed a remarkable reduction in SOD1 protein levels, further substantiating the safety and efficacy profile essential for advancing to the next trial phase.

Furthermore, Ractigen recently entered into a strategic partnership with a foreign pharmaceutical firm, emphasizing the commercial potential of its platforms with a valuation exceeding $3 billion.

Voices from Leadership

Reflecting on the successful round of funding, Dr. Long-Cheng Li, the Founder and CEO, stated, "This validation not only supports our scientific pursuits but also solidifies our position in the rapidly evolving RNA therapeutics landscape. Our focus remains on expanding the boundaries of RNA technology to deliver transformative therapies to patients globally.”

Ractigen's commitment to leveraging its proprietary technologies aligns perfectly with current trends in biopharmaceuticals, showcasing an unwavering drive to solve critical healthcare challenges through next-generation RNA innovations.

For more information on Ractigen and its groundbreaking developments, visit www.ractigen.com.