Enhanced Delivery and Efficacy of Antisense Drugs via Covalently Linked Nanodisc Scaffolds

Application

A synthetic nanodisc (ND) scaffold for delivering antisense oligonucleotides (ASOs) for the treatment of disease.

Key Benefits

Improved cellular uptake and protein suppression using antisense oligos.
Longer ASO half-life compared to other ASO delivery methods.
Potential to overcome the delivery and efficacy challenges using ASOs for RNA and protein suppression.

Market Summary

Antisense oligonucleotides (ASOs) are short, synthetic strands of DNA/RNA that bind to a target mRNA molecule and modulate gene expression. They block the translation of the target mRNA into protein or promote the degradation of the mRNA, which reduces the amount of functional protein produced. The FDA has approved ASOs with many in clinical development as they can potentially treat many diseases, including genetic disorders, cancer, viral infections, and neurological conditions. However, the primary challenge facing ASO is lacking efficacy due to inefficient delivery to target cells. The increasing demand for ASOs is attributed to the increasing prevalence of genetic diseases, advancements in antisense oligonucleotide technology, FDA approvals, and rising investment in research and development.

Technical Summary

Researchers at Emory are developing a new method for enhancing the delivery and efficacy of ASOs by using synthetic nanodiscs (NDs). These NDs are small, stable particles covalently linked to ASO that are easily internalized by cells through molecular mechanisms involved in cellular uptake. Thus far, the inventors have shown the ability of their ND system to improve ASO uptake compared to ASOs not linked to NDs.