Aisleen McColl-Carboni, Serena Dollive, Sarah Laughlin, Rudenc Lushi, Michael MacArthur, Shanshan Zhou, Jeffrey Gagnon, Christopher A Smith, Brenda Burnham, Robert Horton, Dimpal Lata, Brianna Uga, Kalyani Natu, Emmanuela Michel, Celia Slater, Evan DaSilva, Robert Bruccoleri, Tim Kelly, James B McGivney
Manufacturing of recombinant adeno-associated virus (AAV) vectors produces three types of capsids: full, intermediate, and empty. While there are different opinions about the impact of intermediate and empty capsids on safety and efficacy of AAV products, they are generally considered impurities because they are not the intended fully intact vector product. The presence of these impurities could impact product efficacy due to potential competition with fully packaged AAVs for cellular transduction, as well as have potential implications to patient safety due to increased capsid load during dosing...
February 19, 2024: Gene Therapy