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Novel small activating RNA therapy explored in NMIBC


The first patient has been dosed in a phase 1 trial exploring the small activating RNA (saRNA) therapy RAG-01 in patients with BCG-unresponsive non–muscle-invasive bladder cancer (NMIBC).1

The estimated enrollment for the trial is 15 patients and the estimated primary completion date is June 30, 2025.

The estimated enrollment for the trial is 15 patients and the estimated primary completion date is June 30, 2025.

RAG-01 is an saRNA designed to target and activate p21, a tumor suppressor gene, according to Ractigen Therapeutics, the developer of RAG-01.1 The company also noted in a news release that preclinical research demonstrated that RAG-01 inhibited bladder tumor cell growth in animal models.1

"The successful administration of RAG-01 to the first patient underscores Ractigen's robust clinical translation capabilities, signifying an important milestone in our quest to develop innovative treatments for NMIBC patients and to prove the effectiveness of the saRNA platform in real-world applications," Dr Long-Cheng Li, founder and CEO of Ractigen Therapeutics, stated in the news release.1

The multi-center, open-label, first-in-human, phase 1 trial of RAG-01 is enrolling patients aged 18 to 75 years with pathologically confirmed grade 2 or grade 3 NMIBC, an expected survival time of at least 6 months, and an ECOG performance status of 2 or lower. Patients are not eligible for enrollment if they received anti-tumor treatments other than transurethral resection of bladder tumor within 21 days or 5 half-lives (whichever is shorter) from when they signed their informed consent form.

All patients will receive a starting dose of RAG-01 of 30 mg, but they will be distributed into 4 dose cohorts: 30 mg, 100 mg, 300 mg, and 600 mg.

There are 2 primary outcome measures for the study. The first is safety and tolerability of RAG-01 in patients with NMIBC during the period from screening to 6 months following the initial instillation of RAG-01. Safety and tolerability will be measured through adverse events (AEs), serious AEs, and treatment emergent AEs. The other primary outcome measure is the maximum tolerated dose and/or recommended phase 2 dose of RAG-01. These will be assessed through the evaluation of any dose-limiting toxicities with 21 days of the first instillation of RAG-01.

The estimated enrollment for the trial is 15 patients and the estimated primary completion date is June 30, 2025.

The study is being conducted through a collaboration between Ractigen Therapeutics and GenesisCare.


1. Ractigen Announces First Patient Dosed in the Phase I Clinical Trial of RAG-01 for NMIBC. Published online April 3, 2024. Accessed April 12, 2024. https://www.prnewswire.com/news-releases/ractigen-announces-first-patient-dosed-in-the-phase-i-clinical-trial-of-rag-01-for-nmibc-302107118.html

2. NIH US National Library of Medicine ClinicalTrials.gov. A Study of RAG-01 in Patients With Non-muscle-invasive Bladder Cancer (NMIBC) Who Have Failed Bacillus Calmette Guérin (BCG) Therapy. Last updated April 8, 2024. https://classic.clinicaltrials.gov/ct2/show/NCT06351904

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