Process validation for recombinant adeno-associated virus vectors in rare diseases: challenges for MAAs

Catherine Cancian

In the past decade, advances in biomedical research have led to the development of novel clinical procedures and biological medicinal products for human use such as gene therapy products. Gene therapy vectors offer new opportunities for the treatment of severe disorders or rare diseases that currently have no or limited therapeutic options. These medicinal products are rapidly emerging as new class of licensed products. The recent example of a recombinant adeno-associated virus (rAAV)-based vector expressing therapeutic gene RPE65 to treat an inherited retinal dystrophy authorized as Luxturna® in the USA and Europe shown great promises for gene therapy and marked a pivotal moment for all gene therapies currently in development. Gene therapy vectors are generated through complex bioprocessing and manufacturing procedures. A major challenge for translation of promising clinical development to commercial phase is the process validation in order to consistently achieve high product safety, potency, purity and stability. As development programs for rare diseases involve smaller study populations, and therefore fewer manufacturing runs, it makes it challenging to follow traditional approaches for process and product characterization. Aspects of this challenge are discussed here.

Please enter your email to access content Read now