This year could mark a turning for RNA therapeutics, industry experts have stated in a recently released whitepaper.
RNA therapeutics
The whitepaper, released by pharma company WuXi AppTec examines how we may see increasing numbers of RNA therapies brought to patients, following the approval of Onpattro in the US and the EU.
Unlike DNA therapeutics which target genes, RNA therapies work by manipulating the translation of genetic instructions, potentially giving researchers more options for treatment modalities to fight illnesses.
RNA therapeutics are said to offer advantages as they enter the cell’s cytoplasm ready to take-over its protein manufacturing machinery, whereas DNA therapeutics, such as gene therapy and gene editing technologies, must penetrate the cell’s nucleus to modulate genes and risk altering the genome.
Advances have been made to make the delivery of RNAs more stable, with over 69 companies now developing RNA therapeutics.
“Initially, DNA-based approaches were favoured in drug development based on the inadequate delivery technologies for the less stable RNA. However, over the last ten years the field has seen major advances and RNA therapeutics offer a more flexible approach to address a variety of diseases compared to DNA-based therapeutics,” said MiNA Therapeutics CEO, Robert Habib, a contributor to the white paper.
The report discusses the vast potential RNA therapeutics could have due to the variety of different mechanisms they use to combat disease – resulting in the up- or down-regulation of certain proteins in a cell.
Emphasising the impact they could have over the next decade, Arrakis CEO, Michael Gilman, said: “85% of the human genome is transcribed into RNA” and only 3% translates into proteins, many of which cannot be targeted by existing treatment modalities.
“So, between these undruggable proteins and all the emerging biology of noncoding RNAs, there is a lot of disease biology that remains beyond our reach,” Gilman explained. “By directly targeting RNA, we can bring that biology into play for patients.”