Navigating The Complexities of AAV Scale Up to Keep Pace with the Increasing... - Seguridad Mania.com - España y América Latina

Full title: Navigating The Complexities of AAV Scale Up to Keep Pace with the Increasing Demand For these Viral Vector Delivery Systems Modalis’ epigenetic editors are designed to efficiently target genes and control chromatin conformation by coupling an engineered Cas9 variant with epigenetic effector domains; referred to as GNDM (Guide Nucleotide Directed Modulation), which is delivered via a single AAV vector. Most AAV capsids used for systemic treatments require high doses, leading to safety concerns. To overcome such obstacles, Modalis has evaluated numerous novel AAV capsids allowing for reduced dosing, increased safety margins, and higher therapeutic efficacy. These novel capsids do not always play nicely with standard manufacturing techniques and available reagents, so creative process development and analytics are needed to bring these promising therapeutic molecules to patients.