苏州卡耐博生物技术有限公司

PLATFORM INTRODUCTION

SiranBio developed comprehensive, cutting-edge and proprietary platform technologies for siRNA drug discovery, which includes: (1) sequence design and discovery; (2) PD enhancement and off-target attenuation (eSAFE); (3) liver and extrahepatic delivery (STORK); and (4) dual-targeting siRNA. Our strong technical foundation ensures the high quality and high efficiency of our innovative pipeline development.

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siRNA Delivery Technologies

Stork-X is our proprietary multi-tissue delivery platform

Stork-X is our proprietary multi-tissue delivery platform. Extrahepatic delivery has been the holy-grail for siRNA drug discovery. During the last 2 years, SiranBio made tremendous progress in this field. We got best data in delivery efficiency, selectivity, and toxicity profiles in many extrahepatic tissues, including CNS, kidney, muscle and fat. Based on those technologies, we have several exciting new programs for Alzimer’s disease, obesity, and chronic pain, etc. We filed multiple patents, and many of these molecules have the FIC and/or BIC potentials.

Stork-L: Liver Target Platform

SiranBio owns proprietary liver-targeting technology based on GalNAc-conjugation. Contrary to the fork-like trimer structure that was adopted by Alnylam and many other companies, we designed GalNAc monomers as phosphoramidites, which can be incorporated sequentially or in any pattern into the siRNA sequences. That not only gives excellent delivery efficiency, but also greatly simplifies the synthesis of the liver-targeting molecule, and thus significantly reduces the cost of API. It also played a key role in our dual-targeting siRNA design.

Stork-W Dual-Target Platform

SiranBio independently developed proprietary technology for dual-targeting siRNA. Two siRNA are chemically connected into one molecule and delivered into the same tissue.

There are several unique advantages for dual-targeting siRNA.

First, two siRNA can be delivered into the same cell, which maximizes the synergistic effects of knocking down two genes.

Second, a dual-targeting siRNA can be developed much faster and cost-effectively compared to two siRNA drugs.

Combined with different delivery technologies, dual-targeting siRNA can be delivered to different tissues and used in many indications.

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Sequence Design and Discovery Platform

Our team is very experienced in siRNA sequence design. Combining bioinformatics and computer-aided design, we can quickly give hundreds of siRNA sequences for any target gene. Our nucleic acid sequence synthesis platform can make those sequences in a few days, and they can be tested by our high-throughput in vitro and in vivo screening team seamlessly. In most cases, we can get leading sequences for a target gene within 3 months.

Chemical Modification Platform (eSAFE)

SiranBio has developed a proprietary chemical modification platform (eSAFE) that can significantly enhance the safety profile of siRNA molecules while preserving their efficacy. This technology mitigates the off-target effect of siRNA and improves its metabolic stability, and thus ensures the safety, efficacy, and duration of siRNA drugs.

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Nucleic Acid Synthesis Platform

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SiranBio has built a solid-phase platform for nucleic acids, which can efficiently perform the synthesis, purification, and analysis of siRNA molecules. It is the foundation for the development of our technology platforms and pipelines.