By Elizabeth Figueroa, Ph.D., DHC Senior Consultant
ABSTRACT:
In vivo gene therapies have delivered astonishing results where few or no treatments have existed previously. Despite their curative potential, the field of gene therapy has not seen widespread adoption or effective commercialization. What are the challenges faced by in vivo gene therapies, and do non-viral vectors bridge these gaps? In this white paper, we will address these questions by comparing adeno-associated virus (AAV) viral vectors (as representatives of current widespread in vivo viral technology) and lipid nanoparticle (LNP) non-viral vectors (as representatives of emerging non-viral technologies) at different phases of the product lifecycle. The emergence of non-viral vectors represents an opportunity to propel in vivo gene therapy further into wider commercial adoption and expand its impact globally. LNPs are poised to become the first commercially approved nonviral vectors for in vivo transgene delivery and genome editing approaches. While LNPs may face greater preclinical development hurdles than AAV, they are expected to have more streamlined clinical development and commercialization pathways, which will allow them to extend the scope of clinical indications accessible for treatment by in vivo gene therapies.
..interested? Check back on Monday, May 12th for the publication of the full white paper.
