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.
Stability sampling and testing are key to ensuring that products maintain safety, identity, strength, purity, and quality throughout their claimed shelf life. It is also a regulatory requirement per ICH Q5. However, storing product samples in different environmental conditions, testing those samples for three to five years (or more) after initial manufacture, and properly analyzing and...
ISPE hosted more than 450 attendees in person and virtually for the 2024 ISPE Aseptic Conference in Vienna, Austria. Keynotes and education sessions provided a comprehensive overview of key topics and trends...
Biopharmaceutical facility design is a critical aspect of the industry. Overall cost pressures in the global health system, regional requirements to deploy manufacturing rather than centralize manufacturing in one location, technology enhancements in cell biology and format, flexibility to accommodate multi-product campaigns with different production schedules, and speed-to-market are among...