On April 3, 2024 Ractigen Therapeutics, a pioneer in small activating RNA (saRNA) therapeutics, reported a major milestone with the first patient dosed in its First-in-human phase I clinical trial for RAG-01 conducted in collaboration with GenesisCare, Australia’s leading provider of cancer care services (Press release, Ractigen, APR 3, 2024, View Source [SID1234641751]). This marks a significant advancement in the fight against non-muscle invasive bladder cancer (NMIBC).
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RAG-01 is a groundbreaking saRNA medication designed to combat bladder cancer by targeting and activating the p21 tumor suppressor gene in NMIBC. The trial is an open-label, multi-center study designed to assess RAG-01’s safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy in NMIBC patients who have failed Bacillus Calmette-Guérin (BCG) therapy. Preclinical data indicates that RAG-01 effectively inhibits bladder cancer tumor growth in animal models and has shown a favorable safety profile.
Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics, stated, "The successful administration of RAG-01 to the first patient underscores Ractigen’s robust clinical translation capabilities, signifying an important milestone in our quest to develop innovative treatments for NMIBC patients and to prove the effectiveness of the saRNA platform in real-world applications."
About RAG-01: RAG-01 stands as a pioneering saRNA candidate from Ractigen Therapeutics, engineered to target and activate the tumor suppressor gene p21 via the RNAa mechanism which was discovered by the founding team of Ractigen Therapeutics. The drug, delivered through intravesical instillation using Ractigen’s proprietary LiCOTM delivery technology, has shown significant tumor suppression in mouse orthotopic bladder cancer models. 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 LiCOTM: LiCOTM, Ractigen’s proprietary extra-hepatic delivery system, enables the delivery of duplex RNA into a variety of tissues and organs which are harf 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.