What are Oligonucleotides?
New terms in the biopharma realm rarely surprise us but keeping up with the technology and the means of manufacturing can often be challenging. What I really enjoy about ISPE is the way they bring these new technologies to life allowing us to learn from the lessons our peers are going through. One of these emerging realms is the science behind Oligonucleotides.
Oligonucleotides are short (20-25 bases typically) DNA or RNA molecules, oligomers, that have a wide range of applications in genetic testing, research, forensics and even treatments. In nature, oligonucleotides are usually found as small RNA molecules that function in the regulation of gene expression (e.g. microRNA), or are degradation intermediates derived from the breakdown of larger nucleic acid molecules. In the human body the application of Antisense Oligonucleotides (AO) show great promise in disease states such as Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Dystrophy (SMD).
These chemicals interact directly with the genetic coding to disrupt or repair the exon blocking so we’re not at the protein level trying to block or alter a biopathway but right at the genetic root where the issue arises. It seems no disease can hide from Pharma’s R&D centers and it is our job to prepare the facilities to make these products.
As we head towards December, make plans to attend the 2018 ISPE Biomanufacturing Conference in Huntington Beach, I’ll be front and center to learn from pharma professionals in our industry on how these plants are set up to produce oligonucleotides.