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Pixofisiran
- Development stage
- Unknown
- Lead developer
- Sirnaomics
- 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, Nanoparticles → Drug Delivery Systems
- Administration
- Intradermal Injection, Topical, Intra-tumoral, Subcutaneous
01
Overview
Pixofisiran is a small interfering RNA (siRNA) oligonucleotide that specifically targets and silences the messenger RNA (mRNA) of Transforming Growth Factor beta 1 (TGF-β1). It is a key component of the investigational drug STP705, which also includes Lixadesiran (targeting COX-2 mRNA). STP705 utilizes histidine-lysine polypeptide (HKP) nanoparticles for the encapsulation and delivery of these siRNA molecules. Developed by Sirnaomics, Pixofisiran, as part of STP705, is being investigated for its role in localized fat reduction by inhibiting adipocyte differentiation and fat accumulation, and for various oncological and fibrotic conditions. The mechanism of action involves RNA interference, leading to the degradation of TGF-β1 mRNA and subsequent reduction in TGF-β1 protein expression, a cytokine implicated in cellular growth, differentiation, inflammation, and fibrotic responses.
- Other names
- Pixofisiran sodium
02
Targets
TGFB1 (Transforming growth factor Beta-1 proprotein)
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