bit.bio's new Huntington's disease human cell model, for in vitro research and drug discovery

bit.bio’s new ioGlutamatergic Neurons HTT50CAG/WT provide a human, cell-based, in vitro model of Huntington’s Disease (HD) that accurately reflects the disease genotype. Offering industry-leading consistency and scalability, this first product from the new ioDisease Model portfolio has been developed to support key applications within drug discovery including target identification and high-throughput screening.

bit.bio
Jun 9, 2022

The life sciences industry is challenged by a lack of reliable, reproducible cell models that generate translatable, reflective, data of actual human disease. ioGlutamatergic Neurons HTT50CAGWTaims to provide a viable solution for HD. CRISPR/Cas9 gene editing has been used to introduce an abnormal expansion of 50 CAG repeats into the first exon of the Huntingtin gene within the wild type ioGlutamatergic Neurons. These highly characterised iPSC-derived glutamatergic neurons accurately represent the disease genotypein vitro. The engineered cells rapidly mature into functional excitatory neurons that consistently form complex neuronal structures and express typical biomarkers in as little as 11 days.

 

ioGlutamatergic Neurons HTT50CAGWTprovide batch-to-batch consistency at a scale of billions of cells, forming a reproducible model enabled by opti-oxTM precision reprogramming. In addition, bit.bio’s wild type ioGlutamatergic Neurons can be used as a genetically matched control for the ioGlutamatergic Neurons HTT50CAG/WT disease model, offering a physiologically-relevant isogenic pairing with which to study Huntington’s Disease.

 

ioGlutamatergic NeuronsHTT50CAG/WT deliver:

  • A disease cell model that is 96- and 384-well plate compatible
  • Batch-to-batch consistency, at a scale of billions of cells
  • A heterozygous 50 CAG trinucleotide repeat expansion confirmed via NGS-amplicon sequencing and gel electrophoresis
  • A genetically matched, isogenicpairing when used with bit.bio’s wild type ioGlutamatergic Neurons
  • Defined cell identity characterised by ICC, gene expression and RNAseq
  • Rapid maturation. Upon induction, human stem cells rapidly mature into neurons that demonstrate typical glutamatergic neuron morphology and cell-specific markers
  • Demonstrated electrophysiological activity in multi-electrode arrays and expression of Huntingtin (HTT) protein
  • Ease of use as the cells rapidly mature upon revival with a simple two-phase protocol and single open access media composition