Research & Development

Sirnaomics has a robust pipeline of biopharmaceutical therapies built through the in-house development of innovative product candidates and novel RNAi and mRNA delivery platforms, becoming the first company to achieve positive Phase Ⅱ a clinical outcomes in oncology for an RNAi therapeutic.

We have 16 therapeutic candidates in the preclinical stage through Phase Ⅱ clinical trials in the U.S. and China, addressing a wide range of disease areas, from oncology and fibrotic disease, to cardiometabolic, viral and liver disease.

We focus on developing new delivery platforms for RNA therapeutics, which include:

  • PNP delivery platform for local or systemic administration of RNAi therapeutics,
  • GalNAc-RNAi delivery platform for administration of RNAi therapeutics to the liver,
  • Peptide docking vehicle (PDOV) platform for improved delivery and release of siRNAs into the hepatocytes in the liver;
  • PLNP delivery platform for administration of mRNA vaccines and therapeutics.

Drug Delivery Systems

Our highly innovative RNA delivery platforms distinguish Sirnaomics from other drug development companies. Delivery platforms are critical to the intact delivery of RNA therapeutics to the cells where they are needed. Our proprietary PNP and novel GalNAc delivery platforms provide advantages over conventional delivery platforms.

Our Polypeptide Nanoparticle (PNP) Delivery Platform
Our PNP delivery platform allows delivery of both siRNA and mRNA to diseased cells via local or systemic administration. The success of our Phase IIa oncology study with STP705 validates the effectiveness of our PNP delivery platform, positioning us for accelerated development of other pipeline products using the same PNP delivery platform. Not only does our PNP delivery platform offer improved delivery efficiency based on effective cellular uptake and efficient endosomal release, it can also carry more than one siRNA to enable silencing of multiple targets simultaneously, leading to better therapeutic capabilities through synergistic effects. The PNP delivery platform biodegrades within the cell and has a low toxicity.
Our GalNAc-RNAi Delivery Platforms
Our novel GalNAc-RNAi delivery platforms, GalAhead™ and PDoV-GalNAc, enable specific delivery to liver hepatocytes with enhanced endosome escape properties and dual siRNA target design. GalAhead™ and PDoV-GalNAc rely on unique RNA structures and peptide conjugates that enable targeting of one or more genes simultaneously. Our proprietary formulation improves cellular uptake, enables multiple gene targeting and offers higher potency compared to conventional GalNAc-RNAi platforms.