Small nucleic acid drugs exhibit distinct features that set them apart from small molecule drugs and antibody-based therapies.
Aspect |
Small Nucleic Acid Drugs |
Small Molecule Drugs |
Antibody Drugs |
Target Specificity |
High specificity to genetic sequences, targeting mRNA or non-coding RNAs. |
Often bind active sites; risk of off-target effects. |
High specificity to protein epitopes. |
Target Range |
Accesses "undruggable" targets like non-coding RNAs and inaccessible proteins. |
Limited to targets with binding pockets. |
Limited to extracellular or accessible proteins. |
Mechanism of Action |
Modulates gene expression (e.g., RNAi, splicing modulation). |
Typically enzyme inhibition or receptor modulation. |
Neutralizes antigens or triggers immune responses. |
Development Time |
Rapid development due to direct sequence-based design. |
Longer lead times for screening and optimization. |
Time-intensive due to protein engineering. |
Delivery Challenges |
Requires advanced delivery systems for stability and cellular uptake. |
Generally easier delivery (oral, topical). |
Delivery can be challenging (injection only). |
Immunogenicity |
Low immunogenicity with chemical modifications. |
Low to moderate depending on structure. |
Potentially high, requires careful design. |
Manufacturing |
Chemically synthesized, scalable, and reproducible. |
Chemical synthesis, highly scalable. |
Biologically produced, complex and costly. |
Small nucleic acid drugs uniquely combine the specificity of antibody drugs with the versatility of small molecule drugs, offering innovative solutions for challenging therapeutic targets.
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.
REQUEST A QUOTE | ||
---|---|---|
PHONE | ONLINE FORM | |
[email protected] | +1 408-828-0438 | ONLINE QUOTE SUBMISSION |