Ractigen's RAG-01 Granted FDA Fast Track

Ractigen Therapeutics, based in SUZHOU, China, has announced a significant achievement in the field of small activating RNA (saRNA) therapeutics. Their lead program, RAG-01, has received Fast Track Designation (FTD) from the U.S. Food and Drug Administration (FDA), making it the first saRNA drug globally to attain this status. This milestone highlights the promise of saRNA technology and Ractigen's dedication to addressing unmet medical needs.

RAG-01 is currently in a Phase I clinical trial in Australia, targeting non-muscle invasive bladder cancer (NMIBC). The trial began in December 2023 and has successfully dosed three patients, marking steady progress in its clinical development. The FDA recently approved the Investigational New Drug (IND) application for RAG-01, further validating its therapeutic potential. This approval not only facilitates the expansion of clinical trials in the United States but also underscores the FDA's acknowledgment of RAG-01's potential to address urgent medical needs in NMIBC patients.

The Fast Track Designation is granted to investigational drugs aimed at treating serious conditions with unmet medical needs, expediting their development and review processes. With this designation, Ractigen can engage in enhanced collaboration with the FDA, ensuring closer communication and expedited guidance throughout the drug's development and regulatory review.

Dr. Long-Cheng Li, the Founder and CEO of Ractigen Therapeutics, expressed excitement about the FDA's decision, stating, "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."

RAG-01 is an innovative saRNA candidate designed to target and activate the tumor suppressor gene p21 through a mechanism known as RNAa. Traditionally considered "undruggable," p21 provides a unique opportunity for saRNA-based targeted activation. The drug is administered via intravesical instillation using Ractigen's proprietary LiCO™ delivery technology and has demonstrated significant tumor suppression in mouse models of bladder cancer. The ongoing Phase I clinical trial in Australia marks a crucial step in the development of RNAa-based therapies, addressing the unmet needs of NMIBC patients.

Non-muscle invasive bladder cancer constitutes 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 advance towards addressing the substantial unmet need in bladder cancer therapy.

RNAa, pioneered by Dr. Long-Cheng Li and his team, is a clinically validated platform technology that uses saRNA to target gene regulatory domains, activating gene expression and restoring therapeutic protein levels. This technology holds vast potential for developing therapeutic drugs for various diseases, especially where traditional methods fall short, including cancer, genetic disorders, chronic diseases, and metabolic and cerebrovascular disorders.

Ractigen Therapeutics is a leader in saRNA drug development, focusing on utilizing the RNAa mechanism to up-regulate endogenous gene expression. This innovative approach targets specific genes to enhance transcription, thereby restoring normal protein functions. Ractigen's cutting-edge technology is crucial for treating diseases that conventional methods cannot address, such as those resulting from epigenetic silencing or gene downregulation.