Despite their therapeutic potential, small nucleic acid drugs face several significant challenges in development: 

     ●     Delivery Efficiency: Difficulty in delivering nucleic acids to specific tissues and crossing cellular membranes. 

     ●     Stability: Prone to degradation by nucleases, requiring chemical modifications for stability. 

     ●     Immunogenicity: Risk of triggering innate immune responses, especially unmodified sequences. 

     ●     Off-Target Effects: Potential for unintended gene silencing or activation, requiring stringent sequence design. 

     ●     Manufacturing Complexity: High production costs and the need for precise synthesis and purification methods. 

     ●     Regulatory Hurdles: Limited precedent in regulatory pathways for novel small nucleic acid drugs. 

Addressing these challenges requires innovations in delivery systems, chemical modifications, and design strategies, helping advance small nucleic acid drugs toward broader clinical applications. 

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.