Gene editing partnership to provide custom cells for drug discovery

By Maggie Lynch

- Last updated on GMT

(Image: Getty/phonlamaiphoto)
(Image: Getty/phonlamaiphoto)

Related tags preclinical Preclinical contract research CRO Gene editing Stem cells Genome Gene expression Cell line

Ncardia and Horizon Discovery have partnered to provide custom, genetically-modified and differentiated human cells for drug discovery and preclinical models.

Ncardia, a human induced pluripotent stem cells (iPSC) based solutions company, and Horizon Discovery Group, a gene editing and modulation technologies provider, partnered to provide custom cells, and drug discovery services.

Through this partnership, Ncardia will market and sell the models created through genetic modifications in iPSC cell lines, and subsequent differentiation that will enable disorders to be simulated for preclinical trials.

The collaboration will use Horizon’s precision genome engineering in coordination with Ncardia’s manufacturing and differentiation of iPSC-derived cell lines.

Stefan Braam, CEO of Ncardia told us that, the combination of genome engineering and iPSC-derived cells allows for efficient generation of isogenic cell lines, differing only by the DNA sequences of interest.

“These differentiated, fully functional cells enable the study of human genetic diseases in the most relevant human cell-based assay system,”​ said Braam.

Per the partnership, both companies will use the collaboration as a way to increase accessibility of CRISPR-edited cell lines to researchers, with the aim of streamlining new drug candidate licensing.

“We see a demand in the market for a fully integrated service provider with expertise in assay development,”​ said Braam. “Additionally, clients are interested in streamlined access to the necessary IP and licensing to utilize the required technologies.”

iPSC and genome editing: ‘Better’ preclinical models

Braam explained, “Drug discovery and development relies, in large part, on the availability of predictive pre-clinical model systems. The absence of such systems has contributed to late-stage drug failures and high expenditures.”

When iPSC technology is combined with genome engineering, it is beneficial for early-stage drug discovery activities like target discovery, efficacy testing, and high-throughput screening, said Braam.

A spokesperson from Lonza, Don Paul Kovarcik, previously told us​ that pluripotent stem cells can provide relevant cell models because of their defining characteristics. The two characteristics being the cells ability to self-renew and become any cell type in the human body. He continued to say that stem cell reprogramming, like that of iPSC, makes for better preclinical models. 

Braam further stated that recent industry acceptance of iPSC-derived cardiomyocyte assay systems, for regulatory safety pharmacology, has increased interest in their utilization across a wide range of drug development and preclinical testing.

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