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Scientific poster: A human iPSC-based platform to screen therapeutics for ALS using specific and robust phenotypic assays covering disease relevant readouts.

Learn how miniaturized and robust human cell-based assays can help you select the most promising therapeutic candidates for ALS.
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als poster

To enable fast and confident selection of effective therapies for ALS we develop specific and robust phenotypic assays to study protein mis-localization and aggregation with human iPSC-derived in vitro models.

In this study, we show our iPSC-based platform with miniaturized assays using human iPSC-derived motor neurons to study the effect of new therapeutics in recognized ALS phenotypes:

• Mis-localization of TDP-43 to the cytoplasm
• Aggregation of TDP-43
• Reduction of STMN2 protein levels
• Mis-splicing of STMN2
• Altered electrophysiological properties
• Neurofilament-L secretion

Additionally, this ALS in vitro model was successfully transduced and, when treated with a gene therapy candidate, reduction of TDP-43 aggregation was detected. 

Using this platform you can validate your therapeutics, but more importantly, progress with the subset of therapeutics that offer higher confidence for clinical success. 




ABOUT US
Predict future safety and efficacy more efficiently
For more than a decade, Ncardia has been pioneering innovations in human induced pluripotent stem cells (iPSC). Our iPSC drug discovery platforms have been successfully leveraged by large biopharmas, up-and-coming drug discovery firms and multinational research consortia to advance therapeutic candidates for cardiovascular, neurological and other disease areas.

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