On May 21, 2024 Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, proudly reported that its flagship program, RAG-01, has been granted Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA) (Press release, Ractigen, MAY 21, 2024, View Source [SID1234643509]). This notable achievement marks a significant milestone in the advancement of saRNA technology and underscores Ractigen’s commitment to addressing critical unmet medical needs. The milestone establishes RAG-01 as the first saRNA drug worldwide to achieve FTD.

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RAG-01 is currently undergoing a Phase I clinical trial in Australia for the treatment of non-muscle invasive bladder cancer (NMIBC). The trial, initiated in December 2023, has successfully enrolled and dosed three patients, demonstrating the program’s progress in clinical development.

FDA’s recent approval of the Investigational New Drug (IND) application for RAG-01 further validates the therapeutic potential of this innovative saRNA therapy. This regulatory milestone not only paves the way for the expansion of clinical trials in the United States but also highlights the FDA’s recognition of RAG-01’s promise in addressing the urgent medical needs of NMIBC patients.

Fast Track Designation is granted to investigational drugs intended for the treatment of serious conditions with unmet medical needs, facilitating their expedited development and review process. With FTD, Ractigen gains enhanced opportunities for collaboration with the FDA, enabling closer communication and expedited guidance throughout the development and regulatory review process.

Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics, expressed his enthusiasm about the FDA’s decision: "We are thrilled to receive Fast Track Designation for RAG-01, marking a significant milestone not only for our program but also for the saRNA field as a whole. This designation underscores the urgency and importance of advancing innovative therapies like RAG-01 to address critical medical needs. We remain dedicated to accelerating the development of innovative saRNA therapies to address a wide range of diseases, including cancer, genetic disorders, and chronic conditions. Through strategic collaborations and pioneering research efforts, the company aims to deliver transformative treatments that improve patient outcomes and quality of life."

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 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.