Challenges & Opportunities in the Manufacturing of AAV Vectors Used in the Delivery of Gene Therapy Treatments

摘要

Significant advances in the specific targeting of deliveryvectors and the increased therapeutic efficacy of such vectors forgene delivery have been made, stimulating major interest in thedevelopment and commercialization of therapeutic productsfocused on gene therapy indications. In the past few years, therehave been a large number of positive clinical outputs for genetherapy-based products spanning broad therapeutic areas,including CAR T-cell immunotherapy, oncology, and regenerativemedicine based on monogenetic diseases. In terms of genedelivery, viral vectors have emerged as the preferred vehicles ofchoice, used in 48% of the 483 current on-going gene therapytrials.