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Expert Insights: Unlocking next generation gene therapies

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By Joyson Dcosta

How can iPSC-based models unlock next generation gene therapy?

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Expert Profile

Joyson obtained his master’s degree in regenerative medicine and biology at the Technical University of Dresden, Germany, in 2022. During his studies and professional career, he has contributed to relevant research studies demonstrating his proficiency in stem cell differentiation, culturing 3D pancreatic spheroid models and advanced imaging techniques. He has gained a broad expertise in cell-based assays and screening of AAVs, ASOs, compounds on hiPSC-derived models. He joined Ncardia as Assistant Scientist in 2022 and his unwavering dedication and passion continue to make an impact on Ncardia’s innovations and projects.


After decades of studies, gene therapies have recently become a reality for patients, gaining traction as a new therapeutic for a range of monogenic and non-monogenic diseases, such us neurological disorders, cancer or heart diseases. However, the field is still in an immature phase, indicating its huge potential for growth. Joyson spotted that, in fact, this field has caught the interest of both scientists and financiers alike, to build groundbreaking gene therapies in the years ahead.

“In 2019, a gene therapy for spinal muscular atrophy (SMA) received approval by the FDA and has shown remarkable success in the clinic. Soon in 2023 a gene therapy for Duchenne’s muscular dystrophy (DMD) is expected to be approved by the FDA. Decades ago, we didn’t know gene therapies would come this far. With advancements in delivery methods and gene-editing technologies the future looks really promising.”

The success of gene therapy hinges on dependable models that can effectively project the behavior of a therapy in human patients. Animal models frequently fall short in replicating the intricate cellular and molecular mechanisms of human diseases, resulting in a gap between preclinical and clinical studies. To combat this issue, induced pluripotent stem cell (iPSC) technology offers a hopeful solution.

“What is important to realize is that iPSC-based models not only provide a tool to scrutinize the underlying disease mechanisms, but also, a platform for gene therapy screening in a more relevant cellular context. Complex decisions, like selection and optimization of the best delivery system or evaluation of safety and efficacy can be made with higher confidence by introducing iPSC technology. Making the right decisions earlier can also help reduce the gap between preclinical and clinical studies, ultimately saving time and resources.”

Since Joyson joined Ncardia, he has been able to work closely with gene therapy developers to provide customized iPSC-based solutions for their projects. The beauty of these models, as explained by Joyson, is that they exhibit human cellular complexity, allowing us to study specific cellular functions as well as gene and protein expression.

“At Ncardia, I collaborate with gene therapy developers who need models of complex diseases amenable for high throughput screenings. I recently worked on a project to develop a gene therapy aiming to alter the QT interval. Using our human iPSC-derived cardiomyocytes and multitude of molecular and functional readouts (e.g. Micro Electrode Assay, high content imaging and qPCR) we were able to optimize the transduction efficiencies and validate effectiveness of several AAV vectors provided by the client. and see the positive impact it had for the client.”

Are you wondering how iPSC technology could advance your therapeutic development programs? Contact our experts