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."

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.