Advanced Analytical Approaches for Accelerated Development of Gene Therapy Products
Gene and cell therapies offer tremendous potential for treating unmet medical needs; however, successful development of these advanced therapeutic approaches is extremely challenging, largely because of their complexity. Characterizing
and quantitating specific structural attributes and assessing criticality of those that influence quality, efficacy and safety, including delivery to target tissues, protein expression in vivo and eventual clinical outcomes, is essential
to be able to advance both individual gene therapy candidates and the emerging underlying vector/gene platforms.