Abstract

Challenges remain for utilising induced pluripotent stem cell (hiPSC) lines in regenerative medicine and the pharmaceutical industry. Methods that enable faithful, homogeneous and scalable differentiation of hiPSCs to mature cell types are needed to replace current cell lines with limited functionality and biological significance. Coupled with modern genetic tools and the capacity of hiPSCs to reproduce human genetic diversity, hiPSC-derived cells represent a pivotal tool for improving human health by enabling disease modelling, drug discovery and the next generation of cell therapies.

Based on the principle that transcription factors (TFs) control cell identity, TF-driven cellular reprogramming offers a disruptive strategy for cell differentiation, with a growing number of reprogrammed cell types described. By applying a cellular reprogramming approach supported by a proprietary genetic switch, opti-ox^* (optimised inducible overexpression), the typical  restrictions of standard hiPSC differentiation systems - length, complexity, and lack of consistency and purity - have been largely overcome. bit.bio’s technology enables homogeneous and synchronous differentiation of entire hiPSC cultures through tightly controlled expression of selected TFs as demonstrated for glutamatergic neurons, skeletal myocytes,  GABAergic neurons and oligodendrocytes for instance. Coupled with a unique TF combination screening platform, the increasing portfolio of cells produced by opti-ox cellular reprogramming offers human cells with high consistency and functionality, at scale, from human iPSCs, including those carrying disease-specific mutations, providing high-quality human models for enhanced research outcomes and drug discovery efficiency.

References:

* Pawlowski M, et al. Stem Cell Reports. 8(4), 803-812, 2017