Dedicated Analytical Strategies
The term oligonucleotides includes a broad range of molecules with different chemical composition and covering a broad range of molecular weights, ranging between 15-20 nucleotides (e.g. siRNA) and many thousands of nucleotides (e.g. mRNA and DNA expressing whole genes); additionally, nucleic acid-based API are commonly administered with a suitable delivery vehicle protecting them from the environment and enhancing or targeting their delivery to the cell type where their action is required; lipid nanoparticles (LNPs) and virus like particles (VLPs) are common delivery methods. The recent introduction of novel genome editing technology such as CRISPR/Cas9 has further expanded the complexity of the nucleic acid repertoire, and innovative gene therapy platforms posing new analytical challenges are likely to emerge. With this complexity in mind and leveraging our experience in all the platforms described, Pace Life Sciences Boston, MA develops dedicated analytical strategies for each specific project.
ON DEMAND WEBINAR: Pharmaceutical Development of Oligonucleotides
Oligonucleotides are an emerging class of DNA and RNA therapeutics that address a number of unmet medical needs. As with many biopharmaceuticals, the complex structure of oligonucleotides makes their large-scale synthesis both challenging and imperfect. A number of impurities are generated during manufacturing, many of which are hard to identify using available analytical methods. Furthermore, the use of chemical modifications and formulations that expand the functionality and delivery capabilities of these therapeutics pose additional analytical challenges and potential risks.
In this webinar you will learn:
- Specifications and analytical methods for therapeutic oligonucleotides
- Innovative analytical method development for therapeutic oligonucleotides
- Advanced characterization methods for therapeutic oligonucleotides
Who should attend:
- Analytical Development Scientists
- Formulation Development Scientists
- Developers of Therapeutic Oligonucleotides
- CMC Drug Development Team Members