Adeno-associated viral (AAV) vectors comprise the majority of recent gene therapy development programs due to their broad tissue-tropism and relatively low immunogenicity. Recent gene therapy approvals, including therapies for ocular and central nervous system disorders, have demonstrated AAV as an effective technology for genetic disease treatment.
Biopharma 101 by SCIEX is intended to provide you with a thorough introduction to foundational analytical characterization workflows for AAV based gene therapeutics, from the basics of AAV production to assays for vector potency, immunogenicity, transduction effectiveness and packaging efficiency. Attending this class will upgrade your AAV gene therapy analytical expertise!
𝗟𝗲𝗮𝗿𝗻𝗶𝗻𝗴 𝗢𝗯𝗷𝗲𝗰𝘁𝗶𝘃𝗲𝘀
▷ Characterize AAV protein impurities and determine accurate capsid titer
▷ Determine genomic integrity, purity and titer with an accurate genome size estimation
▷ Calculate the empty and full capsid ratio with an accurate determination of partial capsids
▷ Observe nearly complete sequence coverage for the three viral proteins (VP1, VP2 and VP3) and confidently identify modifications in low abundance
𝗣𝗿𝗲𝘀𝗲𝗻𝘁𝗲𝗿𝘀
🎤 Paula Orens, Market Development Manager, Biopharma MS, SCIEX
🎤 Steven Calciano, Market Development Manager, Biopharma CE, SCIEX