Suzhou Siran Biotechnology Co.,Ltd.

PLATFORM INTRODUCTION

SiranBio developed comprehensive, cutting-edge and pγroprietary platform technologies for siRNA drug discovery, which includes: (1) sequence desi←gn and discovery; (2) PD enhancement and off-target attenuation (eSAFE); (3) li♥ver and extrahepatic delivery (STORK); and (4) dual-targeting siRNA. Our strong te&chnical foundation ensures the high quality and high efficiency of our innovati€ve 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 ma§de tremendous progress in this field. We got best dat♠a in delivery efficiency, selectivity, and toxicity pro"files in many extrahepatic tissues, including CNS, kidney, muscle an☆d fat. Based on those technologies, we have several exciting new programs for₩ Alzimer’s disease, obesity, and chronic pain, etc. We filed multip↓le patents, and many of these molecules have the FIC and/or BIC p$otentials.

Stork-L: Liver Target Platform

SiranBio owns proprietary liver-targeting technology based o€n GalNAc-conjugation. Contrary to the fork-like trimer structure tha÷t was adopted by Alnylam and many other companies, we designed GalNAc monomers as ¥phosphoramidites, which can be incorporated sequenβtially 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 co∏nnected 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 k÷nocking down two genes.

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

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

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Sequence Design and Discovery Platform

Our team is very experienced in siRNA sequence design. Combining bioinformatics and compute÷r-aided design, we can quickly give hundreds of siRNA se₽quences for any target gene. Our nucleic acid sequence synthes§is platform can make those sequences in a few days, and £they can be tested by our high-throughput in vitro and in vivo screening φteam 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) th₩at can significantly enhance the safety profile of siRNA molecules while pre§serving their efficacy. This technology mitigates the off-tar≤get effect of siRNA and improves its metabolic stability, and thus ensures th≈e 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 perforσm the synthesis, purification, and analysis of siRNπA molecules. It is the foundation for the development of our technology pl¶atforms and pipelines.