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In vivo siRNA delivery using SAMiRNA Custom Service

SAMiRNA Custom Services, a complete solution for gene function analysis and target discovery and validation through in vivo siRNA studies

Overview
Technical/Specs
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Ordering Information

SAMiRNA™ (Self-Assembled-Micelle-inhibitory-RNA) custom synthesis service provides a customized option to provide a target specific delivery of siRNA in interest in vivo, essential for target validation and therapeutics development. We offer a complete solution for RNAi (gene silencing) studies from siRNA, miRNA, in vivo si-/miRNA delivery using SAMiRNA to all the components and services required for quantitative analyses of targeted genes, using a novel SAMiRNA nanoparticle technology.

Since the inception, Bioneer Corporation has been currently developing SAMiRNA™ drugs for the clinical applications through partnership with pharmaceutical companies. To foster open innovation and new partnership opportunities in various therapeutic areas, we are offering valuable opportunities to utilize this second-generation siRNA technology at an affordable price for translational in vivo siRNA studies. This also includes research and technical support teams ensure the top-quality products and services to meet the unique needs of clients from companies and research institutions worldwide. Our world-class teams have highly profiled expertise in nucleic acid manufacturing and its in vivo applications

For that this service provides a seamless approach to place an order for custom SAMiRNA™ of interest, based on a target gene of interest. Upon manufacturing, the final product is then delivered, which can simply be re-suspended and used for the efficacy testing in animals. Upon customer’s interest, we also provide additional screening in vitro and validation services that include specific siRNA design, in vitro validation of siRNA for further optimization studies and assess knockdown efficiency in vivo. All of our SAMiRNA™ service and products are provided for Research Use Only. For further development, please contact us for the licensing program.


SAMiRNA™ (Self-Assembled-Micelle-inhibitory-RNA)

 One major hurdles in RNA interference (RNAi)-based therapeutics is the proper delivery of siRNA in interest to target tissue and its adverse side effects caused by the specific type of a siRNA delivery vehicle (Table 1). To mediate this, Bioneer has developed SAMiRNA (Self-Assembled-Micelle-inhibitory-RNA), which is a novel single-molecular synthetic siRNA, conjugated with lipid and hydrophilic polymer, spontaneously assembled as a stable nanoparticles (NP) with protective PEG coat and lipid core in the nano-scale size level. This system is optimized for systemic delivery and localization in either vascularized tumors through Enhanced Permeability and Retention (EPR) effect with no other reagents need and formulation process. Such novel approach of synthesis provides solutions for the development of RNAi-based drugs. As a second-generation RNAi drug technology, SAMiRNA is synthesized as a single chemical entity as a form of nanoparticle (NP), manufactured by one-step automated solid phase synthesis requiring no formulation process and no innate immune response.


Table 1: Delivery challenges overcome by SAMiRNA™ technology

Challenges in siRNA delivery SAMiRNA™ system
Rapid clearance and degradation in serum Improved serum stability
Toxicity of delivery systems No detectable liver toxicity or innate immune response
Limited tissue specificity Tumor tissue targeting capabilities
Low silencing potency Long lasting in vivo silencing efficiency


 The therapeutic potential of SAMiRNA™ is highlighted with negligible toxicity, outstanding for its in vivo serum stability, as well as its target gene silencing efficacy in various animal disease models including cancer and lung fibrosis models. In addition, SAMiRNA™ has been shown to induce no innate immune response as demonstrated for a variety of diseases in human PBMC test and high dose-administrated rodent models. This is due to Bioneer’s extensive experience in both nucleic acid chemistry and large scale nucleic acid manufacturing capacity and further advancing into clinical development pipelines with top-quality DNA/RNA oligos and siRNA worldwide for 20 years.


Our SAMiRNA Custom Services include:

  • Custom siRNA synthesis

    • Custom siRNA oligos are offered based on the sequences of your interest. Up to 30-mer siRNA including a choice of 32 different 3' overhangs can be synthesized with a variety of modification options for expanded specificity. With this, SAMiRNA consists of proprietary polymer -conjugated siRNA that is provided purified, annealed, and lyophilized which can simply be resuspended for efficacy tests in animals.

  • Genome-wide pre-designed siRNA

    • Genome-wide predesigned siRNA library is available for mouse, rat or human. Bioneer’s proprietary Turbo-si designer algorithm designs siRNA from your gene of interest that provides superb knock-down efficiencies of specific targets by binding to the structural hindrance-free region and avoiding off target effect.

  • Custom SAMiRNA™ nanoparticle synthesis for in vivo siRNA delivery, and efficacy/biodistribution tests using animal models

    • siRNA contains high lipo-phillic cell membrane barrier and is known to get rapidly degraded when delivered into the blood stream in vivo, triggering undesirable immune responses. Thus, SAMiRNA™ resolves all of the limitations, from studies conducted in solid tumor model.

      The siRNA oligos can be manufactured in the form of SAMiRNA with SAMiRNA™ nanoparticles (NP) can be provided upon request. (see what SAMiRNA™ is here http://www.bioneer.co.kr/SAMiRNA/Platformtechnology.aspx). Upon manufacturing, the reagent can then be re-suspended in PBS and injected these into animals.

  • In vitro screening and validation of siRNAs for lead optimization

    • To achieve and validate siRNA with highest knockdown efficiency, Bioneer recommends “in vitro screening and validation service”, instead of choosing from in silico. This approach is based on constructing and transfecting up to 100 siRNAs into cells individually, aiming specific mRNAs of interest. Its results in the knockdown efficiencies are ranked and analyzed by RT-qPCR. Upon selection, we determine candidates of most potent siRNAs and SAMiRNA nanoparticles (NP) by revalidating in vitro before shipment.

  • Quantitation of functional knockdown of target gene by real time RT-qPCR (Accupower® qPCR Array Service)

    • Bioneer also offers qPCR Array service for quantitation of functional knockdown of target gene by real-time RT-qPCR. The process follows the MIQE (Minimum Information for Publication of Quantitative Real-Time PCR Experiments) guidelines. Experts conduct the experiments and provide data in a short period of time.

    • For more information, please visit “qPCR Array Service”. Bioneer’s SAMiRNA™ Services provides a rapid on-line quotation at siRNA@bioneer.com for siRNA; qPCRarray@bioneer.com for Accupower® qPCR array service. Please let us know the details of your project, so that we can provide you with a quotation and a timeline estimate.

  • To order and use custom SAMiRNA™ incorporating customized siRNA or miRNA