The pace of development in oligonucleotide therapeutics has accelerated in recent years, with a total of 18 drugs approved to date in the US and more than 1,000 in the pipeline – including clinical trials for major diseases such as Alzheimer’s. A multitude of exciting conjugation and nano-delivery innovations are in current clinical and preclinical development, with potential to address the historical problem of effective oligonucleotide tissue targeting and widen the scope of diseases and disorders that can be treated and potentially cured with these transformative drugs.
This application note reviews promising lipid conjugation techniques and nanocarriers in preclinical development as well as approved oligonucleotide therapeutics – considering the likelihood of them
successfully addressing the tissue targeting question, and supporting the development of a next
generation of drugs capable of tackling diverse unmet clinical needs.