White paper: Transforming Gene Therapy: How Induced Pluripotent Stem Cells are Advancing the Development of Next-Generation Treatments

Despite the great potential of AAV-based gene therapies, only a few drugs have received market approval from regulators. To effectively bring next-gen gene therapies from bench to bedside, there is a strong need for alternative testing platforms that recapitulate human pathophysiology and help making accurate predictions.

The integration of human induced pluripotent stem cells (iPSC)-derived models is a promising alternative to support and supplement traditional animal testing. One of the key advantages is that, unlike animal models, iPSCs preserve patient's serotype and recapitulate main disease hallmarks in vitro. Moreover, they are amenable for high-throughput screenings and can be integrated in the firsts stages of development. Therefore, iPSC-based platforms can facilitate the selection of the most promising AAV vectors early in the process, saving resources in later stages and helping reduce the translational gap.

Read this white paper to gain insights in the current AAV gene therapy landscape and how the early integration of human in vitro models in the development process can overcome important limitations of animal testing, shorten timelines, and save resources.