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SAMiRNA

RNAi is a rapidly growing market, and siRNAgen is leading the way.

 

RNAi, or RNA interference, has become an increasingly popular therapeutic modality for its ability to target genes and rapidly generate new drug candidates. siRNA, a particularly potent type of RNAi, is a therapeutic modality that, when appropriately delivered, can work for almost a year.

With the whole genomic library at our fingertips, any gene of interest can theoretically be turned into a therapeutic lead using siRNA. When a gene is a target, drug discovery can become more efficient.

This Nobel-Prize-winning technology initially could target only liver diseases, but thanks to companies like siRNAgen, it is now also addressing chronic disorders in various organs beyond the liver. siRNAgen's programs include inflammatory and CNS indications.

SAMiRNA is a plug-and-play platform, and the first proof of concept was with the GLUT-SAMiRNA platform, which enabled the systemic dosing of RNAi to reach across the blood-brain barrier (BBB). We showed that because SAMiRNA overcomes the metabolic clearance and endosomal escape challenges of today's conjugate RNAi, it provides a powerful platform to generate new RNAi platforms. View our Program page to learn more about each platform.

Keep reading to learn about the science behind our technology.

siRNAgen simplifies RNAi therapies using modular chemistry and self-assembly to enable delivery to target tissues.

siRNAgen's platform, SAMiRNA, offers "plug & play" versatility and a flexible route of administration. The modular platform is a self-assembling double conjugate with the oligonucleotide in the middle flanked by a hydrophobic and hydrophilic end. Hydrophobic interactions drive the supramolecular assembly into the micellar structure, and targeting moieties can be added to the hydrophilic end.

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This structure has several benefits:
  • SAMiRNA overcomes two major hurdles in delivery: metabolic clearance of small conjugates and endosomal escape.

    • ​With a longer serum half-life than a typical conjugate combined with various targeting moieties, systemic siRNA delivery could be significantly improved.

    • Our linker chemistries enable effective endosomal escape, achieving high efficacy.

  • SAMiRNA hides the unmodified double-stranded RNA from triggering innate immune stimulation, which leads to an excellent safety profile.

  • Our molecule is room temperature stable in solution for up to a year, which means we can save energy and improve access. Patented manufacturing methods make the platform more sustainable than a typical siRNA therapy

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