Antisense oligonucleotide (ASO) therapies.
- Novel approach to ASO therapeutics that potentially improve tissue/cell targeting capabilities.
- Increases the efficacy of ASOs while decrease off-target toxicity.
- Potential to treat a variety of genetic diseases such as cancer, blood disorders, and rare conditions for which there are no viable treatment options.
Antisense oligonucleotides (ASOs) are an emerging therapeutic modality that allows for targeting specific RNA and subsequent protein translation for a multitude of diseases. However, many ASO’s in development are not specific to cell type and can affect normal healthy tissues. Researchers at Emory University have derived a novel, cell-specific (liver-specific) approach to ASO therapeutics. This invention will fit into the global market for RNA therapy, which is expected to reach $4.692 billion by 2023 at a CAGR of 21.6% from 2018-2023 (BCC Research BIO159A). Furthermore, this invention can contribute to the liver disease market.
Researchers at Emory have developed a potential platform technology for specific tissue targeting of ASO therapeutics. The inventors developed a liver specific ASO by using a miRNA that triggers activation of HIF1a, which is involved in a variety of human diseases as well as essential activities in healthy cells. miR-122 is a highly specific miRNA exclusively expressed in hepatocytes and makes up a large percentage of the total hepatic miRNA pool in adult mouse and humans. HIF1a is a master transcription factor that is related to diverse human diseases, such as cancer and cardiovascular diseases. However, since HIF1a is involved in a variety of cell activities and plays protective roles in wound healing and repairing injury, as well as regulating neoangiogensis and tissue vascularization, systematically inhibiting of HIF1a may lead to side effects. Therefore, conditional regulation of HIF1a in targeted tissue or cell type could be beneficial.
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