CRISPR/Cas9 gene editing allows pioneering drug discovery technique for Huntington’s disease
Researchers from bit.bio (Cambridge, UK) have announced the launch of their ioGlutamatergic Neurons HTT50CAG/WT cells, which they hope will enable more effective drug discovery for Huntington’s disease. The novel disease model approach – one of the first of its kind to be commercially available – is an exact replication of the genetics of Huntington’s disease.
The technology behind this product is bit.bio’s ioDisease Model portfolio, which is a range of cells that have specific disease-associated mutations. This enables human diseases to be reproduced in vitro that can be matched to an isogenic wild type control. The ioDisease Model Portfolio uses iPSCs, which have been generated utilizing CRISPR/Cas9-based gene editing.
Huntington’s disease is a neurodegenerative condition that is caused by specific mutations in the Huntingtin gene. More specifically, ≥ 40 repeats of a ‘CAG’ DNA sequence. Previously, it has been challenging for scientists to engineer the precise disease-causing mutation in cells.
To overcome this, researchers from bit.bio have created a consistent, scalable cell model that reproduces the condition in vitro. In order to do this, bit.bio introduced a stable 50 ‘CAG’ repeat mutation in the Huntingtin gene in wild type ioGlutamatergic Neurons.
Mark Kotter, CEO and founder of bit.bio, explained: “bit.bio’s mission is coding cells for novel cures. The launch of our Huntington’s disease model provides the industry with an advanced cellular tool to support the study of a devastating condition with high unmet clinical needs. Our opti-ox TM1 technology allows the development of consistent and scalable biological standards that can support the evolution of a new generation of medicines. It adds depth to our broadening R&D cell type product pipeline, each product will transition to its own product line.”
Researchers from bit.bio suggest that utilizing ioGlutamatergic Neurons HTT50CAG/WT and wild type ioGlutamatergic Neurons that do not contain the mutation in drug discovery will enable scientists to study the effects of novel treatments in a disease-relevant system with a genetically matched control.
In addition, they hope that comparative data will provide the potential identify and investigate the effects of the CAG trinucleotide repeat expansion. It is hoped that with further research this novel approach will aid in drug discovery to provide effective treatment for Huntington’s disease.
Press release: https://www.bit.bio/news/launch-ioglutamatergic-htt50cag/wt