Please mark your calendar for June 25, at 12:30 pm EST. Use the link below to register for the event
https://www.pathlms.com/aaps/webinars/8295
The major class of drugs today, small molecules and proteins (biologics) have multiple pharmacological issues with respect to targetability, efficacy, and applicability toward a broad range of disease targets. Ribonucleic acids (RNA) offer a promising way forward driven by their ability to bind specifically via base-pairing and potentially offering a cure rather than treatment. As such, there has been a significant growth and maturity in the number and type of RNA-based therapeutics being studied in clinical trials today. The primary site of action for most RNAs are intracellular sites within target cells, thereby requiring high delivery efficiency to enable optimal pharmacological activity. Naked RNAs, however, are prone to significant disadvantages such as nuclease degradation in biological fluids, rapid activation of the immune system, insufficient accumulation in target tissues, and are unable to passively cross the cell membrane. As a result, RNAs are encapsulated within nanoparticulate delivery vehicles that offer efficient means of vesicular-based or receptor-based cellular entry and endosomal escape mechanisms allowing the required dosage to enter the cytoplasm. Over a long multi-decade period, several different types of delivery vehicles have been designed starting with polymers and lipids forming what was initially referred to as polyplexes and lipoplexes, and then gradually giving way to spherical liposome-like Lipid Nanoparticles (LNPs). In this webinar, we will discuss the basic aspects of formulation design around two different types of RNA molecules (siRNA and mRNA). In addition, designing effective processes and focused analytical methods around nanoparticle formation and final presentation in the appropriate dosage form will be discussed. The presentation will be interspersed with a few case studies around lipid degradation, size distribution measurements and filtration unit operations largely made possible via our experience working with multiple clients on their LNP products.