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Research & Development
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Sirnaomics is committed to developing innovative biopharmaceutical drugs leveraging our novel delivery platforms in a wide variety of disease indications, including oncology, fibrotic diseases and conditions, viral diseases and cardiometabolic diseases. We are focused on developing new delivery platforms for RNA therapeutics to maintain and broaden the scope of our product pipeline, and to overcome the limitations of conventional RNA delivery tools. Once our targets have been selected based on clear scientific rationale, we apply a proprietary algorithm based on our understanding of the biochemical mechanisms involved in RNA interference to identify promising candidate RNAi trigger sequences against the selected target gene and employ high throughput processes to design, screen and rigorously test future pipeline products.

We have built a pipeline through in-house development of both our product candidates and our RNAi and mRNA delivery platforms. We believe the success of RNA-based therapeutics relies on three pillars: (1) the selection of the right targets; (2) the application of suitable delivery platforms; and (3) the selection of appropriate therapeutic indications. We have developed in-house and own the global rights to STP705 and STP707, our lead product candidates, which demonstrates our capabilities in designing novel RNA therapeutics based on our innovative and adaptable delivery platforms and developing them into drugs to address unmet medical needs. Our proprietary delivery platforms include our PNP delivery platform, useful for local or systemic administration of RNAi therapeutics to targets beyond liver hepatocyte cells, our GalNAc RNAi delivery platforms for system administration of RNAi therapeutics to the liver, and our PLNP delivery platform for administration of mRNA vaccines and therapeutics. We exclusively in-licensed core patents covering our PNP delivery platform at an early stage and have conducted research and development in-house to enhance our PNP delivery platform and adapt it for formulating novel RNA therapeutics to treat a range of therapeutic indications. We have developed in-house and own the global rights to innovative GalNAc RNAi delivery platforms. Our PNP and GalNAc RNAi delivery platforms fuel our expanding pipeline of early-stage product candidates. Our subsidiary RNAimmune develops mRNA-based vaccines and therapeutics.

We have a comprehensive portfolio of patents to protect our drug candidates and technologies. We own 9 issued patents in China, 9 issued patents in the U.S., 2 issued patents in Europe (validated in 11 and 8 countries, respective). And We also have 119 pending patent applications, including 19 Chinese patent applications, 43 U.S. patent applications (including 32 U.S. provisional patent applications), 8 patent applications under the Patent Cooperation Treaty, 6 patent applications in Europe, and 43 patent applications in other jurisdictions. Our patents and patent applications span methods of delivering RNAi triggers and mRNA to cells, compositions of matter and devices used in our RNAi and mRNA delivery platforms, siRNA or RNAi trigger compositions, manufacturing processes, usage, and indications.

We are led by our visionary management team with deep experience and capabilities in discovering, developing, and commercializing RNA therapeutics. Management team and scientific advisory board average more than 20 years of pharma research and development experience at the world’s leading pharmaceutical companies and research institutions in China and the U.S. Thus we are able to attract top talents and build strong teams across markets.

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research and development in-house to enhance our PNP delivery platform and adapt it for formulating novel RNA therapeutics to treat a range of therapeutic indications. We have developed in-house and own the global rights to innovative GalNAc RNAi delivery platforms. Our PNP and GalNAc RNAi delivery platforms fuel our expanding pipeline of early-stage product candidates. Our subsidiary RNAimmune develops mRNA-based vaccines and therapeutics.

We have a comprehensive portfolio of patents to protect our drug candidates and technologies. As of the Latest Practicable Date, we owned (i) 9 issued patents in China, (ii) 9 issued patents in the U.S., (iii) 2 issued patents in Europe (validated in 11 and 8 countries, respective), and (iv) 116 pending patent applications, including 19 Chinese patent applications, 39 U.S. patent applications (including 28 U.S. provisional patent applications), 9 patent applications under the Patent Cooperation Treaty, 6 patent applications in Europe and 43 patent applications in other jurisdictions. Our patents and patent applications span methods of delivering RNAi triggers and mRNA to cells, compositions of matter and devices used in our RNAi and mRNA delivery platforms, siRNA or RNAi trigger compositions, manufacturing processes, usage and indications.

We are led by our visionary management team with deep experience and capabilities in discovering, developing and commercializing RNA therapeutics. In addition, our management team and scientific advisory board have on average more than 20 years of pharma research and development experience at the world’s leading pharmaceutical companies and research institutions in China and the U.S. Thus we are able to attract top talents and build strong teams across markets.

Drug Delivery Systems

We believe our highly innovative RNA delivery platforms set us in a class by ourselves. The primary challenge and the key to success in developing RNA therapeutics is the delivery platform used to protect the RNA from degradation in the blood and deliver the RNA into a cell where it acts. Our proprietary PNP and novel GalNAc delivery platforms confer advantages over conventional delivery platforms.

Our PNP delivery platform allows delivery of both siRNA and mRNA to diseased cells via local or systemic administration, providing distinct advantages in low toxicity, easy manufacturing and the capability to reach many targeted organs and certain cell types. The success of our Phase IIa oncology study validates both the effectiveness of our PNP delivery platform and the therapeutic targets for isSCC, positioning us for accelerated development of other pipeline products using the same PNP delivery platform. Our proprietary GalNAc RNAi delivery platforms, GalAhead™ and PDoV-GalNAc, enable specific delivery to liver hepatocytes with enhanced endosome escape properties and dual siRNA target design, resulting in high potency.

Our Polypeptide Nanoparticle (PNP) Delivery Platform
Our PNP delivery platform efficiently encapsulates multiple distinct siRNAs in a drug product and protects them in the bloodstream while enabling delivery of the siRNAs to cells and tissue where the siRNA acts to silence the target genes. In order for the siRNA to act, it must be able to cross the cell membrane to enter the cell and then escape the cellular machinery, the endosome, which isolates the siRNA within the cell. We believe that our PNP-formulated siRNA has improved delivery efficiency based on its effective cellular uptake and efficient endosomal release into the cytoplasm, which are crucial characteristics for RNAi delivery platforms. Our PNP delivery platform can carry multiple siRNAs within the same particle for delivery to the same cell at the same time to silence more than one target gene. Simultaneous delivery of multiple siRNAs can produce a synergistic effect, which in cancer cells can provide better therapeutic capability and reduce the likelihood for the cancer cell to evade the therapeutic pressure. Our PNP delivery platform has low toxicity since it is comprised of polypeptides that are biodegradable within the cell after siRNA delivery. Our PNP delivery platform can be used for both local delivery or systemic administration for selective targeting of multiple tissue and cell types. Our core product candidate, STP705, as well as our other clinical stage product candidate, STP707, and at least eight other preclinical product candidates utilize our PNP delivery platform. RNAimmune also applies our innovative PNP delivery platform, and a related proprietary delivery platform based on polypeptide-lipid nanoparticles (PLNP), to formulate mRNA-based therapeutics and vaccines. RNAimmune’ novel PLNP platform has presented advantages such as lower toxicity and higher efficiency in certain applications.
Our GalNAc RNAi Delivery Platforms
Our novel GalNAc-conjugate delivery platforms rely on peptide conjugates and/or unique RNA structures that allow knockdown of single or multiple distinct mRNA targets. Our GalAhead delivery platform conjugates GalNAc moieties to unique RNAi trigger structures that can target one or more genes simultaneously. Particularities of the structures also reduce the complexity of their manufacturing compared to conventional GalNAc RNAi triggers. We have three pipeline products utilizing our GalAhead delivery platform that are quickly approaching IND-enabling studies. In our PDoV-GalNAc RNAi platform, GalNAc is conjugated to a Peptide Docking Vehicle (PDoV) peptide linker and up to two siRNAs are also conjugated to the same peptide. While GalNAc directs delivery to the liver, the PDoV peptide is designed to improve cellular uptake and endosomal escape compared with conventional GalNAc RNAi platforms. The PDoV peptide linker allows dual gene targeting by conjugating two siRNAs. The ability to deliver multiple siRNAs per molecule can provide improved delivery efficiency relative to conventional GalNAc RNAi platforms, or allow synergistic therapeutic effect when two distinct siRNAs are used to target multiple genes.
Other Delivery Platforms
Apart from our PNP and novel GalNAc RNAi delivery platforms, we believe we also derive significant growth potential based on a number of highly innovative deliver platforms we are currently developing, including different approaches of siRNA/chemo-drug conjugates, peptide ligand tumor targeting and respiratory virus treatment via airway delivery. Our technology platform includes a proprietary algorithm to drive early discovery efforts to identify promising siRNA candidate sequences, and high throughput processes to design, screen and rigorously test future pipeline products. We are committed to investing in research and development in our advanced delivery platforms to enable the expansion and refinement of the range of organs and tissues that can be targeted by our pipeline products and to drive future growth opportunities.