Ractigen Therapeutics Announces FDA IND Approval for RAG-01, the World’s First saRNA Therapy for NMIBC
Ractigen
Innovative Drug R&D Developer
Ractigen Therapeutics, an innovative biopharmaceutical company at the global forefront in the small activating RNA (saRNA) drug field, announced todayRAG-01, a saRNA drug independently developed by Ractigen Therapeutics for the treatment of non-muscle-invasive bladder cancer, was granted Investigational New Drug (IND) approval by the U.S. Food and Drug Administration (FDA) on April 25, 2024.RAG-01 is the world's first saRNA drug of its kind to receive FDA approval, as well as the first saRNA drug in China.
RAG-01 is a first-in-class saRNA drug designed to activate the tumor suppressor gene p21 in patients with non-muscle-invasive bladder cancer (NMIBC), thereby inhibiting the recurrence and progression of bladder cancer. Previously, RAG-01 had already initiated a Phase I clinical trial in Australia, with three subjects enrolled and dosed.
Dr. Longcheng Li, founder and CEO of Ractigen, said:"The IND application for RAG-01 has been successfully approved by the U.S. FDA, marking a significant milestone for Ractigen and a key advancement in the global RNA activation field. We will continue to advance the clinical trials of RAG-01, with the hope that it will become a revolutionary therapy, offering NMIBC patients worldwide a more effective and safer innovative treatment option."
About RAG-01
RAG-01 is a first-in-class double-stranded saRNA drug that specifically targets and activates the tumor suppressor gene p21. It enhances the expression of the p21 gene through the RNAa mechanism to inhibit tumor cell proliferation and induce apoptosis and senescence. Preclinical efficacy studies have shown that, delivered via Ractigen Therapeutics' self-developed small nucleic acid delivery system LiCO™, RAG-01 treatment significantly inhibits the growth of bladder cancer tumors in animal models while demonstrating robust safety. Currently, the Phase I clinical trial of RAG-01 in Australia has successfully completed dosing in three subjects.
About NMIBC
Bladder Cancer is the most common cancer of the urinary system, with NMIBC accounting for 50-80%. The standard treatment for NMIBC is primarily transurethral resection of bladder tumor (TURBT), but the postoperative recurrence rate is high, reaching up to 50%-70% within five years. Therefore, there is an urgent need to develop drugs that can effectively prevent bladder cancer recurrence and treat advanced bladder cancer.
About LiCO™
RAG-01 utilizes Ractigen Therapeutics' self-developed LiCO™ delivery system, which employs a unique lipid chemistry approach to support multiple administration routes, including subcutaneous, intravenous, intravesical, and intravitreal injection. The versatility and efficacy of LiCO™ have been validated in numerous preclinical projects, enabling efficient and sustained delivery to tissues such as the heart, muscle, fat, and eyes, fundamentally revolutionizing the delivery method for oligonucleotides.
About RNA Activation
RNA Activation (RNAa) is a platform technology pioneered internationally by Dr. Long-Cheng Li, the founder of Ractigen Therapeutics, and his team, which has already been clinically validated. This technology uses double-stranded RNA targeting the promoter region of genes to activate gene expression, restoring the levels of therapeutic proteins. RNA activation technology is a highly scarce platform technology in the pharmaceutical field, with broad application prospects in developing therapeutic drugs for cancer, genetic diseases, chronic conditions, metabolic disorders, and cardiovascular and cerebrovascular diseases.
About Ractigen
Ractigen Therapeutics is a platform-based new drug research and development company based in China and targeting the global market. It is committed to developing breakthrough small nucleic acid drugs and disease treatment methods. Ractigen Therapeutics is one of the few global small nucleic acid drug companies that simultaneously possess both intrahepatic and extrahepatic delivery technologies, having developed several internationally leading proprietary delivery platform technologies, including SCAD™ and LiCO™. Based on RNA activation technology and its self-developed Smart-TTC saRNA drug development platform, the company has established a highly differentiated small nucleic acid drug pipeline with indications covering neurodegenerative and neuromuscular diseases, cancer, metabolic and hematological disorders, providing innovative therapeutic solutions for undruggable targets and currently incurable diseases across various fields. For more details, please visit the official website.www.ractigen.com。
Ractigen Therapeutics Announces FDA Approval for RAG-01, a First-in-Class saRNA Therapy for BCG-Unresponsive NMIBC
JIANGSU, China, April 26, 2024 — Ractigen Therapeutics, a leader in the development of small activating RNA (saRNA) therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has approved the company’s Investigational New Drug (IND) application for RAG-01, a groundbreaking saRNA therapy targeting non-muscle invasive bladder cancer (NMIBC). This approval facilitates the launch of U.S. clinical trials, following the successful initiation of a Phase I trial in Australia.
RAG-01 is a pioneering therapy in bladder cancer treatment, representing the first of its kind to specifically target and activate the p21 tumor suppressor gene. This gene is critical in regulating cell cycle progression and is a key component in stopping the growth of cancer cells. By activating p21, RAG-01 offers a targeted approach to potentially curb the progression of NMIBC, a prevalent form of bladder cancer.
"FDA IND approval for RAG-01 is a major achievement for Ractigen and a significant advancement for saRNA technology worldwide," said Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. "This first-in-class saRNA therapy harnesses the power of RNAa to target the p21 gene, offering a promising new option for patients with limited treatment choices. This approval validates the potential of RAG-01 as a leading saRNA therapy and strengthens our position as innovators in RNA-based treatments."
About RAG-01: RAG-01 is a pioneering saRNA candidate engineered to target and activate the tumor suppressor gene p21 via the mechanism of RNAa. Traditionally considered "undruggable," p21 presents a unique opportunity for saRNA-based targeted activation. The drug, delivered through intravesical instillation using Ractigen’s proprietary LiCO™ delivery technology, has shown significant tumor suppression in mouse orthotopic bladder cancer models. Currently, the Phase I clinical trial of RAG-01 in Australia has successfully enrolled and dosed the first three patients. Its development marks a significant stride in RNAa based therapies, addressing the unmet needs of NMIBC patients.
About NMIBC: NMIBC represents 50-80% of all bladder cancer cases. Despite standard treatments like transurethral resection of bladder tumor (TURBT) followed by intravesical BCG or chemotherapy, recurrence rates remain high, estimated at 50-70% within the first five years. RAG-01's development is a significant step towards addressing this substantial unmet need in bladder cancer therapy.
About LiCO™: LiCO™, Ractigen's proprietary extra-hepatic delivery system, enables the delivery of duplex RNA into a variety of tissues and organs which are hard to reach by conventional approach. It supports multiple administration routes, including subcutaneous, intravenous, intravesical, and intravitreal, providing profound and durable activity. Its versatility and effectiveness have been validated in many preclinical studies, marking it as a cornerstone in Ractigen's therapeutic arsenal.
About RNAa: Pioneered by Dr. Long-Cheng Li and his team, RNAa is a clinically validated platform technology. It employs saRNA to target gene regulatory domains, activating gene expression and restoring therapeutic protein levels. This technology has vast potential for developing therapeutic drugs across various diseases, especially where traditional methods fall short, including cancer, genetic disorders, chronic diseases, and metabolic and cerebrovascular disorders.
About Ractigen Therapeutics: A leader in saRNA drug development, Ractigen Therapeutics is at the forefront of developing saRNA drugs utilizing the RNAa mechanism to up-regulate endogenous gene expression. This innovative approach involves saRNA targeting specific genes to enhance transcription, thereby restoring normal protein functions. Ractigen's cutting-edge technology is pivotal in treating diseases unaddressable by conventional methods, such as those resulting from epigenetic silencing or gene downregulation.
