6. How have current regulations affected the development of oligonucleotide therapeutics?

Chad Christianson (Alturas Analytics)

Like any other regulated drug therapy, the amount of time and resources required to bring a new entity from a theoretical therapy to an approved treatment is daunting. The current regulations stress patient safety which is always the most important aspect of any experimental therapy. The amount of required experiments to validate an assay to ensure patient safety is always increasing, which means more time to validate the assay and a higher cost to the developer.

 Zamas Lam (QPS)

Oligonucleotides bioanalysis fits into the FDA 2018 BMV guidance and the current EMA ICH M10 guidance. Potential confusion may arise where some oligonucleotides can be analyzed by chromatographic or LBA assays, so the acceptance criteria can shift from 15/20 to 20/25.

 Ken Cook (Thermo Fisher Scientific)

The manufacture and use of oligonucleotide therapeutics is relatively new. There have also been major recent developments in the analysis of oligonucleotides. mRNA vaccines are a good example of these changes. As such, there are still a lot of details that need to be verified in the regulation and characterization of these new modalities. The fast tracking through regulatory approval of the COVID-19 vaccines was beneficial to the world and enhanced the development of these therapeutics dramatically. Tightening down the characterization requirements for regulatory approval of future products will be the next step.

Alexey Wolfson (Advirna)

Development of oligonucleotide therapeutics for treatment of ultra-rare (N1-N10) is becoming a reality. In many cases therapeutics can be developed within a 1-year timeframe. It can save the lives of thousands of kids suffering from progressive, genetically determined neurological disorders. One of the major obstacles for the wide adoption of the available approaches is the lengthy approval process for an essentially individualized treatment. In many cases it is a race between life and death due the progression of the disease and a timeline for development and approval.

The opinions expressed in this feature are those of the authors and do not necessarily reflect the views of Bioanalysis Zone or Future Science Group.