Scaling Antisense Oligonucleotides Manufacturing - Toward Therapeutic Breakthroughs

Antisense oligonucleotides (ASOs) are synthetic single-stranded nucleic acid sequences that can alter RNA structure and activity to regulate protein expression in vivo. These properties make ASOs valuable as potential therapies for a myriad of diseases, including neurological diseases and cardiovascular pathologies. Several of these diseases do not currently respond to molecular medicines. Thus, ASOs constitute a new class of drugs that can propel us toward new therapeutic frontiers.

The therapeutic applications of ASOs necessitate quality control and consistency. During manufacturing, ASOs must also be thoroughly characterized at the molecular level to validate their pharmacokinetic profile and therapeutic efficiency. For this, manufacturers require accurate and sensitive bioanalytical assays. However, many analytical tools suffer from several drawbacks, namely enabling fast acquisition of data as production scales.

Fortunately, cutting-edge technological solutions that can solve for these issues are now available. Phenomenex Biozen Oligo™ columns provide enhanced oligonucleotide characterization with high sensitivity and minimal sample loss. These analytical consumables can help manufacturers improve the recovery and consistency of their samples by >80%, with untacked reproducibility. Another platform—the SCIEX ZenoTOF 7600 high-resolution mass spectrometry system with SCIEX Molecule Profiler software—enables accurate identification and quantitation of analytes, impurities, and metabolites from the smallest oligonucleotide sample volumes with increased sensitivity. The SCIEX ZenoTOF 7600 system can increase the signal-to-noise ratio up to 8-fold for the quantitation of synthetic oligonucleotides.

ASOs have untapped potential and many applications as gene therapies and could change how we treat debilitating disease. Contact an expert at Danaher Life Sciences to know more about developing, scaling, and manufacturing groundbreaking new antisense oligonucleotide medicines.