siRNAgen Therapeutics’ SAMiRNA (Self-Assembled Micelle inhibitory RNA) platform is a next-generation RNA interference (RNAi) delivery system designed to overcome the limitations of traditional siRNA therapeutics. By utilizing self-assembled micelle nanoparticles, SAMiRNA achieves highly efficient, stable, and tissue-specific delivery of small interfering RNA (siRNA), enabling precise gene silencing for a broad spectrum of diseases, including fibrosis, oncology, and metabolic disorders.
The SAMiRNA platform employs amphiphilic molecules that spontaneously form micelles in aqueous environments. These micelles consist of a hydrophobic core that encapsulates the siRNA, protecting it from nuclease degradation, and a hydrophilic shell that enhances solubility and facilitates cellular uptake. This design eliminates the need for cationic lipids or viral vectors, reducing toxicity and immune activation risks. Preclinical studies have demonstrated that SAMiRNA nanoparticles exhibit prolonged circulation times, improved biodistribution, and enhanced gene knockdown efficacy in target tissues, such as liver and tumor microenvironments.
Functionalized micelles enable selective delivery to specific cell types, minimizing off-target effects and enhancing therapeutic efficacy.
SAMiRNA nanoparticles leverage the Enhanced Permeability and Retention (EPR) effect for passive accumulation in tumor tissues, enhancing delivery efficiency for oncology or inflamed applications.
The micelle structure shields siRNA from enzymatic degradation, ensuring robust performance in vivo.
Applicable to diverse indications, including hepatic fibrosis (SRN-001), solid tumors (SRN-001), and other targets (SRN-002).
The platform supports rapid customization of siRNA sequences and nanoparticle compositions for various therapeutic applications.
HEG-based self-assembled micelle system minimizes TLR-mediated immune activation through its charge-neutral surface and optimized siRNA sequence design.
SAMiRNA’s efficacy has been validated in preclinical models, with SRN-001 demonstrating significant suppression of fibrotic and oncogenic gene expression in vivo. Ongoing research aims to expand the platform’s applications to additional disease areas, leveraging its modular design for rapid therapeutic development.
Enhanced Permeability & Retention (EPR) is a well-known phenomenon where sizeable (100-800 nm) non-targeting drugs accumulate in tissues with increased vascular permeability
SAMiRNA delivers effectively and specifically into solid tumors which are supported by abnormally tortuous, leaky vasculature
In-vivo real-time NIR fluorescence imaging of SAMiRNA™ in inflammatory & fibrosis models
Confocal images show delivery of IV-administered SAMiRNA into all areas of the lung, particularly in areas with more vasculature such as the alveoli
Minimal immune stimulatory activity compared to small nucleic acid lipid particle (SNALP) or unmodified siRNA
