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RAG-01 + mitomycin C is an investigational combination therapy being evaluated for the treatment of non-muscle-invasive bladder cancer (NMIBC). RAG-01 is a first-in-class small activating RNA (saRNA) designed to target and upregulate the expression of the p21 (CDKN1A) tumor suppressor gene via RNA activation (RNAa). It is delivered locally via intravesical instillation using Ractigen's proprietary lipid-conjugated oligonucleotide (LiCO™) delivery system. Mitomycin C is a well-established chemotherapeutic agent that acts as a DNA alkylating and cross-linking agent. The combination is designed to leverage the antiproliferative and tumor-suppressive effects of p21 upregulation alongside the cytotoxic DNA-damaging effects of mitomycin C to improve outcomes in patients with intermediate- or high-risk NMIBC.
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