Small nucleic acid drugs are a class of therapeutics that specifically target genetic sequences to modulate gene expression or activity. These innovative drugs are applied in the treatment of a wide range of diseases, including genetic disorders, cancers, and viral infections. 

   ●    siRNA (Small Interfering RNA): Silences specific gene expression through the RNA interference (RNAi) pathway. 

     ●      Examples: Patisiran, Givosiran 

   ●    ASO (Antisense Oligonucleotides): Binds to target mRNA to inhibit translation or promote degradation, often used for genetic disorders. 

     ●      Examples: Nusinersen, Eteplirsen 

   ●    miRNA (MicroRNA): Endogenous small RNA molecules that regulate multiple genes, with potential applications in cancer and metabolic diseases. 

     ●      Examples: miRNA-34a (in clinical trials) 

   ●    Ribozymes and DNAzymes: Catalytically active nucleic acids that degrade specific RNA or DNA molecules. 

     ●      Examples: Angiozyme (in research stage) 

   ●    Aptamers: Single-stranded DNA or RNA molecules with high binding affinity for specific targets, used for targeted therapy or delivery. 

     ●      Examples: Pegaptanib 

   ●   CRISPR-Cas Guide RNA: RNA molecules associated with CRISPR technology for gene editing and regulation, offering new approaches for genetic disease treatment. 

     ●      Examples: Preclinical and research-stage developments 

Small nucleic acid drugs encompass siRNA, ASOs, miRNAs, ribozymes/DNAzymes, aptamers, and CRISPR-Cas guide RNAs. Each type has distinct mechanisms and applications, addressing various disease areas through precise genetic targeting. 

GenCefe Biotech provides high-quality siRNA, miRNA, sgRNA, and custom RNA synthesis services. We can design and synthesize RNA Oligos of different lengths, different forms, and with various modifications according to customer’s needs, and also synthesize RNA sequences designed by customers. The RNA products we deliver are all purified by HPLC. ISO 9001-certified facilities and comprehensive quality control reports ensure the delivery of high-quality products.