Improving Gene Therapy Development With Organ-On-A-Chip Technology

Bringing gene therapies to market can be a complex, expensive, and time-consuming process. A significant challenge in developing these therapies is creating safe and effective vectors, which serve as the vehicles to deliver therapeutic genetic material into cells. Although gene therapy holds great promise, accurately predicting the efficiency and safety of these vectors remains challenging.

To address the limitations of current preclinical models, Organ-on-a-Chip technology has been developed. This technology offers a more human-relevant in vitro platform for AAV-based therapeutic development, which could enable researchers to refine AAV designs more rapidly resulting in the accelerated development of novel gene therapies, reduced clinical trial attrition, and improved patient outcomes.

Explore the current challenges of developing gene therapies, the potential of Organ-on-a-Chip technology, a specific example where Organ-Chips were utilized to evaluate AAV and LNP transduction safety and efficiency, and the future of gene therapy.