top of page

Human-First Drug Discovery: Using iPSC-Derived 3D Neurospheroids to Model Diseases & Drive the Discovery of New Targets & Compounds

Human-first Drug Discovery: Using iPSC-derived 3D Neurospheroids to Model Diseases and Drive the Discovery of New Targets and Compounds

  • Complex human induced pluripotent stem cell (hiPSC)-derived neural spheroid platforms enable greater physiological relevance to advance traditional drug discovery by bringing human biology to the forefront of the discovery pipeline.

  • hiPSC-based platforms created from patient and control tissues allow for comparisons of the safety and efficacy of new compounds, while multiparametric data analytics improve compound stratification to highlight hits for further development and facilitate clinical translation.

  • This presentation will highlight the use of a complex, uniform iPSC-derived neurospheroids in high-throughput functional screening platforms to identify new targets and hits for neurodevelopmental disorders.

Join us for our presentation:

September 22
9:30 am EST

Blake headshot.jpg

Presented by Blake Anson, PhD
Vice President Scientific Affairs & Partnering


Visit our poster:

Patient derived disease models: Target ID and hit stratification through efficacy and toxicity testing with IPSC-derived neurospheroids

Three-dimensional (3D) human induced pluripotent stem cell (hiPSC)-based technology places human biology first in the discovery pipeline, reducing the potential for late-stage failure and de-risking traditional drug discovery. Because 3D IPSC-based approaches recapitulate native behavior, they lend themselves to holistic discovery approaches. Coupled with advanced analytical methods that rank phenotypic readouts, these platforms implement complex and relevant biology in high throughput.


Here, we present a fully-customizable 3D neurospheroid platform that was used to generate neurodevelopmental models for Rett Syndrome (RTT) and CDKL5 Deficiency Disorder (CDD). We found that 3D hiPSC-derived clinical models have unique atypical phenotypes, allowing for functional screening to identify targets and compounds that ‘rescue’ the disease-linked phenotypes in high-throughput and provide a basis for subsequent therapeutic development.

bottom of page