Ractigen Therapeutics Submits Phase 1 Clinical Trial Application in Australia for First-in-Class saRNA Drug RAG-01 Targeting NMIBC

Dec 07, 2023 14:01 CST Updated 14:02

Ractigen

Innovative Drug R&D Developer

▎Reporting by the WuXi AppTec Content Team

On December 6, Ractigen Therapeutics announced that it had submitted itsThe First ProductSmall activating RNA (saRNA)DrugThe Phase 1 Clinical Trial Application for RAG-01. This clinical study is an open-label, multi-center Phase 1 study designed to evaluate RAG-01 inBCG-Unresponsive Non-Muscle-Invasive Bladder Cancer (NMIBC)Safety, tolerability, pharmacokinetics, and preliminary efficacy in patients. According to a press release by Ractigen Therapeutics, this move symbolizes the company's official transition into a clinical-stage biopharmaceutical enterprise and marks a significant breakthrough in the field of RNA activation.

RNA Activation (RNAa) TechnologyIt is a technology capable of achieving endogenous gene activation and has entered the clinical validation stage.Using double-stranded RNA targeting gene promoter regions to activate gene expression for restoring therapeutic protein levels。RNA activation technology is a scarce platform technology in the biopharmaceutical field, with broad application prospects in the development of therapeutic drugs.

Ractigen is one of the emerging small nucleic acid drug companies that simultaneously masters both intrahepatic and extrahepatic delivery, and has developedMoreIndividualAn exclusive international innovative small nucleic acid drug delivery platform technology has been developed, along with a highly differentiated small nucleic acid drug pipeline, covering indications such as neurodegenerative and neuromuscular diseases, cancer, metabolic and hematological disorders.

RAG-01 is aSpecifically Targeting and Activating Tumor Suppressor Genesp21Double-stranded saRNA drug, Activated through the RNAa mechanismp21Gene expression, to inhibit tumor cell proliferation and induce apoptosis and senescence. Preclinical efficacy studies show that, through Ractigen's self-developedExtrahepatic Small Nucleic Acid Delivery SystemLiCO^TMRAG-01 treatment significantly inhibited the growth of bladder cancer tumors in animal models and demonstrated adequate safety.

According to the introduction,LiCO^TMThe delivery system utilizes a unique lipid chemistry approach, supporting multiple administration routes such as subcutaneous, intravenous, intravesical, and intravitreal injections. The system's versatility and efficacy have been validated in numerous preclinical projects, enabling efficient and long-lasting delivery to tissues such as the heart, muscle, fat, and eyes, with the potential to transform the delivery of oligonucleotides.

Founder and Chief Executive Officer of RactigenLiDr. Long Cheng"This is not only an important moment for Ractigen Therapeutics to enter the clinical stage, but also a day when we have achieved significant results in the field of global innovative drugs. Based on the original platform technology of RNA activation (RNAa), the preclinical data of RAG-01 has demonstrated excellent tumor suppression effects and safety. We look forward to the clinical performance of RAG-01 and hope that it will become a globally innovative targeted cancer treatment drug. We will actively promote more promising saRNA products into the clinical stage, providing innovative therapies for many currently difficult-to-treat diseases."

References:

[1] New Breakthrough in RNA Activation: Ractigen's First saRNA Drug RAG-01 Submits Phase I Clinical Trial Application in Australia. Retrieved Dec 6, 2023, from https://mp.weixin.qq.com/s/lLjNHpCI5HRawV1Ft5Y59g

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