Ractigen Therapeutics, a clinical-stage biopharmaceutical company pioneering small activating RNA (saRNA) therapeutics,  announced the presentation of positive preliminary data from its ongoing Phase I clinical trial of RAG-01, a first-in-class saRNA therapy for the treatment of non-muscle invasive bladder cancer (NMIBC). The data were presented in a late breaking session by Dr. Paul Anderson of The Royal Melbourne Hospital, Australia, at the 40th Annual Congress of the European Association of Urology (EAU 2025) in Madrid, Spain. The results demonstrate a remarkable 66.7% complete response (CR) rate in carcinoma in situ (CIS) patients within the two lowest dose cohorts, along with a favorable safety profile, in patients who have failed Bacillus Calmette-Guérin (BCG) therapy.

RAG-01 is an innovative saRNA therapy designed to upregulate the p21 tumor suppressor gene, a key regulator of cell cycle progression that has been challenging to target with traditional therapies. Administered into the bladder via intravesical instillation using Ractigen's proprietary LiCO™ delivery technology, RAG-01 offers a novel approach to treating NMIBC, particularly in patients unresponsive to BCG therapy. The therapy has received FDA Fast Track Designation, reflecting its potential to address a significant unmet medical need in this patient population.