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FAQs

Preclinical Safety Characteristics of siRNA Drugs

The biological effects of silencing of target mRNA may be present, but other effects are not easily observable.

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

Due to RISC’s high efficiency, siRNA rarely competes with other miRNA resources. Many toxic reactions associated with siRNA drug occur at the administration site, tissue, in tissue or cell residue or through accumulation in animals, including inflammatory reactions, hepatocyte vacuolation, renal tubular vacuolation, and deposition of basophilic granules 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 this effect is often not evident in routine non-clinical safety studies.

Immunogenicity may occur, but the antigen is not necessarily the siRNA itself; it can also be the excipients or structural components of the formulation. For example, if PEG is present, it can easily lead to the production of anti-PEG antibodies.