CRISPR meets opti-ox™ - Harnessing CRISPR-Ready ioCells for drug discovery in neurodegenerative diseases

Advancing drug discovery in neurodegenerative diseases requires cellular models that accurately reflect human physiology and enable mechanistic insights. Traditional models based on immortalized cell lines often fail to capture the complexity of human disease biology. Induced pluripotent stem cells (iPSCs) offer a promising alternative by enabling the generation of disease-relevant cell types, yet they present challenges in consistency, scalability, and differentiation.

bit.bio addresses these limitations with its opti-ox™ technology, which enables rapid and reproducible reprogramming of human iPSCs into defined cell types at scale. Coupled with the power of CRISPR-Cas9, a new era of functional genomics has emerged, allowing systematic exploration of gene-disease relationships. Here, we highlight the utility of bit.bio’s CRISPR-Ready ioCells™ as powerful tools for neurodegenerative disease research. These cells form the foundation of bit.bio discovery's single-cell CRISPR (scCRISPR) screening platform, which enables high-resolution identification of gene functions. In microglia models (ioMicroglia), CRISPR knockout screens revealed key regulators of the response to LPS stimulation, offering new insights into microglial activation and neuroinflammation.

By integrating opti-ox™ technology with scCRISPR screening, we present a scalable, reproducible, and high-throughput platform for functional genomics studies in human cell types. This approach has the potential to reshape neurodegenerative drug discovery. We invite the scientific community to explore the transformative potential of these advanced ioCells in unlocking novel therapeutic strategies.