Pharma Focus Asia

Ractigen Therapeutics Gains FDA Fast Track Status for Pioneering saRNA Therapy RAG-01 for Bladder Cancer

Wednesday, May 22, 2024

Ractigen Therapeutics, a leader in the development of small activating RNA (saRNA) therapeutics, proudly announces that its flagship program, RAG-01, has been granted Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA). This achievement is a significant milestone for saRNA technology and highlights Ractigen's dedication to addressing critical unmet medical needs. RAG-01 is now the first saRNA drug globally to receive FTD.

RAG-01 is currently in a Phase I clinical trial in Australia for non-muscle invasive bladder cancer (NMIBC), having successfully enrolled and dosed three patients since December 2023. This progress underscores the potential of saRNA therapies in clinical development.

The FDA's approval of the Investigational New Drug (IND) application for RAG-01 further affirms its therapeutic promise. This regulatory milestone not only paves the way for clinical trials in the United States but also signifies the FDA's recognition of RAG-01's potential to meet the urgent medical needs of NMIBC patients.

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

Ractigen Therapeutics, expressed his excitement: "We are thrilled to receive Fast Track Designation for RAG-01. This milestone not only advances our program but also the saRNA field as a whole. This designation emphasizes the importance of progressing innovative therapies like RAG-01 to address critical medical needs. We remain dedicated to accelerating the development of saRNA therapies to treat a wide range of diseases, including cancer, genetic disorders, and chronic conditions. Through strategic collaborations and pioneering research, we aim to deliver transformative treatments that improve patient outcomes and quality of life."

About RAG-01: RAG-01 is an innovative saRNA candidate designed to target and activate the tumor suppressor gene p21 through RNA activation (RNAa). Traditionally considered "undruggable," p21 presents a unique opportunity for saRNA-based targeted activation. Delivered via intravesical instillation using Ractigen's proprietary LiCO™ delivery technology, RAG-01 has shown significant tumor suppression in mouse models of bladder cancer. The ongoing Phase I clinical trial in Australia has successfully enrolled and dosed the first three patients, marking a significant advancement in RNAa-based therapies for NMIBC.

About NMIBC: NMIBC accounts for 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, with estimates of 50-70% within the first five years. RAG-01's development aims to address this significant unmet need in bladder cancer therapy.

About RNAa: Pioneered by Dr. Long-Cheng Li and his team, RNAa 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 across various diseases, particularly where traditional methods have been inadequate, including cancer, genetic disorders, chronic diseases, and metabolic and cerebrovascular disorders.



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