Introduction to AAV ‐based in vivo Gene Therapy

Chapter 1 Introduction to AAV -based in vivo Gene Therapy Oscar Segurado, Oscar Segurado ASC Therapeutics, Milpitas, CA, USASearch for more papers by this author Oscar Segurado, Oscar Segurado ASC Therapeutics, Milpitas, CA, USASearch for more papers by this author Book Editor(s):Yanmei Lu, Yanmei Lu Sangamo Therapeutics, Brisbane, California, United StatesSearch for more papers by this authorBoris Gorovits, Boris Gorovits Sana Biotechnology, Cambridge, Massachusetts, United StatesSearch for more papers by this author First published: 16 February 2024 https://doi.org/10.1002/9781119852810.ch1 AboutPDFPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShareShare a linkShare onEmailFacebookTwitterLinkedInRedditWechat Summary Gene therapies are advancing rapidly as novel therapeutic modalities, providing functional cures for monogenic diseases, and more recently non-monogenic diseases, such as cancers, neurological, and cardiovascular diseases. These conditions significantly impact the healthcare system due to their need for continual, complex, and costly treatments and interventions. This chapter overviews the biotechnological advances in adeno-associated virus (AAV) vectors for in vivo gene therapies, covering key technologies for in vivo gene editing. It describes critical elements of AAV optimization for efficient delivery of therapeutic genes, addressing efficacy, safety, immunogenicity, delivery, manufacturing, regulatory approvals, and the challenges and risks of AAV-based gene therapy, including malignancy and immunogenicity. Furthermore, it reviews the incorporation of translational biomarkers found during discovery and preclinical studies of gene therapies into the clinical development program. The rapid progress implies more homogeneous and streamlined development programs, leading to lower manufacturing costs, more affordability for insurance companies, and increased access for patients. 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