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sd-rxRNA TGFB1
- Development stage
- Preclinical
- Lead developer
- Phio Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intratumoral
01
Overview
sd-rxRNA TGFB1 is a self-delivering RNA interference (sd-rxRNA) therapeutic developed by Phio Pharmaceuticals (formerly RXi Pharmaceuticals) designed to silence the expression of Transforming Growth Factor Beta 1 (TGFB1). Utilizing Phio's proprietary INTASYL platform, the compound is chemically modified to enable spontaneous cellular uptake without the need for specialized delivery vehicles or formulations. TGFB1 is a potent immunosuppressive cytokine in the tumor microenvironment (TME) that promotes "cold" or immune-excluded tumors by restricting T-cell activity and recruiting myeloid-derived suppressor cells (MDSCs). By downregulating TGFB1, sd-rxRNA TGFB1 aims to reduce TME-mediated immunosuppression, enhance anti-tumor T-cell activity, and potentially synergize with other immunotherapies or radiation. Preclinical studies have demonstrated its efficacy in reducing TGFB1 levels and increasing anti-tumor activity in melanoma and breast cancer models.
- Other names
- self-delivering RNAi targeting TGFB1INTASYL TGFB1
02
Targets
TGFB1 (Transforming growth factor Beta-1 proprotein)
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