2023 - Bioanalytical Strategies for Gene Therapy: Bioanalysis of Lipid Nanoparticles Encapsulated Antisense Oligonucleotides

Gene therapy is a promising new therapeutic modality for the treatment of inherited and acquired diseases. In gene therapy, the faulty gene is modified to restore normal function. The genetic material is delivered to targeted cells via viral delivery or non-viral delivery such as peptide-based or lipid-based delivery systems. These therapies involve modified polynucleic acid chains that enhance stability and hold promise for respiratory conditions, neurodegenerative diseases and cancers.
Clinical applications utilize different types of oligonucleotides, including antisense oligonucleotides (ASo), micro-RNA (miRNA), small interfering RNA (siRNA), aptamers and CpG oligonucleotides. To deliver these oligonucleotides effectively, specialized technology like lipid nanoparticles (LPNs) acts as carriers, targeting specific cells and tissues.
Bioanalysis is the foundation of novel drug development. Although regulatory guidance exists for manufacturing, non-clinical and clinical conduct of gene therapies, there is no specific guidance on the bioanalysis of gene therapies.
Join this webinar and learn about the bioanalytical challenges and solutions in gene therapy development, including the characterization of lipid nanoparticles and measurement of encapsulated oligonucleotides.