Nucleic Acid

Nucleic Acid designates biopolymers composed of nucleotide monomers forming DNA or RNA, serving as fundamental biological molecules encoding genetic information, regulating gene expression, catalysing biochemical reactions, and participating in cellular signalling, with therapeutic applications exploiting nucleic acid properties for treating diseases through gene therapy, antisense oligonucleotides, RNA interference, aptamers, and mRNA vaccines.

The biopharmaceutical industry has revolutionised therapeutics through nucleic acid-based medicines offering unprecedented specificity and programmability, with antisense oligonucleotides treating spinal muscular atrophy and Duchenne muscular dystrophy, small interfering RNAs with approved products including patisiran, mRNA therapeutics demonstrated through COVID-19 vaccines, and gene therapies using DNA or RNA to replace defective genes. Chemical modifications enhance therapeutic nucleic acids through improving stability against nuclease degradation, reducing immunogenicity, and enhancing target binding affinity. Delivery represents a critical challenge given negative charge and large size impeding cellular uptake, requiring lipid nanoparticles or conjugation to targeting moieties. As delivery technologies advance enabling broader tissue targeting and manufacturing scales enabling cost-effective production, nucleic acid therapeutics continue emerging as a major pharmaceutical modality.