01 / Small Nucleic Acid Drugs
The targeted therapeutic approach using small nucleic acids, such as ASO, siRNA, and miRNA, for specific gene modulation to treat diseases.
ASO: Antisense Oligonucleotide
siRNA: Small Interfering RNA
miRNA: MicroRNA
02 / Delivery Systems
LNP and GalNAc delivery systems are advanced technologies designed to enhance the targeted delivery and cellular uptake of small nucleic acid drugs, improving their therapeutic efficacy and safety.
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Lipid Nanoparticles (LNP): Encapsulates and protects nucleic acids, enhancing stability and delivery.
GalNAc Conjugation: Targets liver cells (hepatocytes), improving specificity and reducing side effects.
