Pharmacologist U.S. Food and Drug Administration Silver Spring, Maryland
Oligonucleotides, modified mRNA therapeutics, CAR T cell therapy, AAV-based gene therapy, and other novel molecules have great potential to treat diseases and are currently expanding to chronic disease areas such as cardiovascular diseases. These molecules have unique pharmacokinetic and pharmacodynamic properties that raise challenges in dose selection and utility of population pharmacodynamic approaches rather than population pharmacokinetic approaches for therapeutic individualization. The purpose of this presentation will be to discuss overall drug development with case examples The presentation will give a overview of clinical pharmacology considerations for novel modalities. The prologue will cover both CDER and CBER perspective.
Understand the differences between small interfering RNAs and antisense oligonucleotides;
Understand the differences between gene therapy products and small molecules and therapeutic proteins from clinical pharmacology stand point
Learn about unique pharmacokinetic/Pharmacodynamic characteristics of oligonucleotides and small interfering RNAs;
Learn about unique pharmacokinetic/pharmacodynamic characteristics of some gene therapy products (such as CAR-T cell therapy products and AAV-based gene therapy products)
Learn about regulatory challenges in development of novel modalitites