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FAQs

Preclinical Safety Characteristics of siRNA Drugs

The biological effects of silencing of target mRNA may be present, but it is not easy to see other effects

Due to the specific nature of siRNA sequence design, binding effects to non-target mRNAs are rare

Due to the high efficiency of RISC, siRNA rarely grabs other miRNA resources Many toxic reactions are related to the siRNA drug at the administration site, tissue, cell residue or animal accumulation, inflammatory reaction at the administration site, hepatocyte vacuolation, renal tubular vacuolation and basophilic granule deposition, etc.

There are also some toxic reactions associated with the excretion process of siRNA drugs

siRNA has the potential to induce innate and acquired immune responses, but the effect is often not obvious in routine non-clinical safety studies.

Immunogenicity may occur, but the antigen is not necessarily the siRNA itself but also the excipients or structural components of the formulation. For example, if there is PEG, it is easy to produce anti-PEG antibodies.