Researchers have now demonstrated conclusively that messenger RNA (mRNA) can be considered a valid target for correcting genetic mutations at the RNA level that cause a range of genetic diseases, including many rare diseases.

Research related to RNA therapeutics has also consistently reinforced limitations that drug developers are steadfastly working to address – primarily that delivery of most RNA therapies has thus far been limited to the liver and unable to target other tissues in the body. 

However, one promising new approach that is positioned to transform the RNA field is a new class of drugs called antibody oligonucleotide conjugates (AOCs). These are rapidly progressing in clinical development and recently demonstrated the first-ever successful targeted delivery of RNA to muscle tissue in humans.

Assessing progress in RNA delivery

Advancing AOCs to transform the delivery of RNA therapeutics discusses how a new class of medicines called antibody oligonucleotide conjugates (AOCs) have the potential to overcome a major challenge encountered with many established RNA-based therapeutics: delivery to tissues outside the liver. Last year, AOCs demonstrated the first-ever successful targeted delivery of RNA into muscle tissue in humans.

Recent clinical advances and product approvals have demonstrated significant progress in the development of first-generation RNA therapeutics, for instance with antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs). Researchers have now demonstrated conclusively that messenger RNA (mRNA) can be considered a valid target for correcting genetic mutations at the RNA level that cause a range of genetic diseases, including many rare diseases.

Research related to RNA therapeutics has also consistently reinforced limitations that drug developers are steadfastly working to address – primarily that delivery of most RNA therapies has thus far been limited to the liver and unable to target other tissues in the body. However, one promising new approach that is positioned to transform the RNA field is a new class of drugs called antibody oligonucleotide conjugates (AOCs). These are rapidly progressing in clinical development and recently demonstrated the first-ever successful targeted delivery of RNA to muscle tissue in humans.

In contrast, AOCs are not limited to RNA degradation or splicing modification alone. They can be composed of various types of oligonucleotides, including siRNAs and PMOs that can be engineered to modify RNA function in different ways. This allows researchers to develop oligonucleotides that are tailored to modulate specific disease processes. Mechanisms of these oligonucleotides can range from reducing expression of a disease-related RNA with siRNAs to correcting aberrant processing of RNAs with splice-modifying oligonucleotides.

Source: https://www.europeanpharmaceuticalreview.com/article/189952/advancing-aocs-to-transform-the-delivery-of-rna-therapeutics/