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Chunk #43 — Main Text — Using hiPSCs for Drug Discovery

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Creating Patient-Specific Neural Cells for the In Vitro Study of Brain Disorders.
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In discussions among attendees, it became apparent that pharmaceutical and academic scientists approach drug discovery with different perspectives. Traditionally, most academics seek to test the cell type most relevant to disease, pursuing a candidate-based approach to test effects on phenotypes, whereas industry scientists have historically conducted high-throughput screens on entrenched industry-standard screening cell lines using target-based assays. While academics have typically been willing to develop more “risky” assays, the strengths of pharma have historically been in assay selection, scalability, and optimization, as well as drug chemistry and target optimization. Now, research strategies are converging, and both types of researchers are moving toward hiPSC-based screening platforms, drifting toward a hybrid model of testing medium-throughput libraries, screening ∼30,000 compound libraries with known targets. New collaborations between academic and pharma researchers promise a future of parallel screening for both targets and phenotypes. Additional hurdles will be encountered if academics are to be the new drivers of drug discovery, including replication (across platforms/reproducibility across sites), documentation (to the rigor of record keeping in industry), and investments to increase automation.