Bay Area Gene Therapy Workshop

Join us in the Bay Area to learn about AAV and LNP analysis by light scattering on September 12th!

 
 

Adeno-associated virus (AAV)-based viral vectors have emerged as popular delivery vehicles in gene therapy owing to their lack of pathogenicity, low immunogenicity, and ability to deliver therapeutic genomes into a wide range of host cells. To ensure the safety and efficacy of viral vector-based drug products, it is essential to implement robust and reliable characterization and quantitation analytics throughout the development and manufacturing process.

mRNA has risen to fame as a new and effective therapeutic modality through its extraordinary success as a COVID-19 vaccine. However, these mRNA-based vaccines would not exist without formulation by lipid nanoparticles (LNPs). The lipid shell protects mRNA from nuclease degradation and endosomal digestion when it enters the cell. As a result, LNP is becoming the preferred delivery vehicle for different types of nucleic acid (NA)-based medicines as well as other drug substances. To ensure the safety and efficacy of LNP formulated gene therapeutics, it is essential to implement robust and reliable characterization and quantitation analytics throughout the development and manufacturing process.

Join us for this event on Tuesday, September 12, to learn more!

AAV Session

We will discuss the SEC-MALS method in a single 30-min run and with an LOQ of 5x1010 mL-1. These quality attributes include Cp (total titer), Vg, E/F, and percentage of small aggregates. During the workshop, we will share case studies on using SEC-MALS as a platform method and/or in QA/AC. Additionally, AAV solutions using DLS (for screening size, particle concentration, and stability), FFF-MALS (for quantifying all AAV aggregates), and in-line monitoring (for process development) tools will be presented as well.

LNP Session

We will discuss using the SEC/FFF-MALS method multi-attribute quantification (MAQ) in one system and in a single run. These quality attributes include high resolution size distribution, size-based payload distribution, and encapsulation efficiency. Strategies on using SEC/FFF-MALS in QA/QC will also be discussed. Tools for screening size, particle concentration, and stability with DLS, measuring zeta potential and charge with PALS, and in-line monitoring for process development will be presented as well.

Details:
Tuesday, September 12, 2023
AAV Workshop: 9:00 AM – 12:00 PM
Lunch: 12 PM – 1 PM (both AAV and LNP attendees are welcome)
LNP session: 1:00 – 4:30 PM

Location: San Mateo, CA
Who should attend:
  • Anyone who is interested in tools to characterize and quantify AAVs, LNPs and other gene vectors. Admission is free, and complimentary continental breakfast and lunch are included.