New Synthego Program Provides Unprecedented Editing of iPS

Stem Cells

Genome engineering company Synthego launched a new genome editing program to include induced pluripotent stem (iPS) cells, adult cells that can be reprogrammed and provide researchers with an easier pathway to drug development.

Redwood City, Calif.-based Synthego said the new offering expands the company’s automated cell editing to help achieve “unprecedented editing efficiency” of iPS cells at an industrial scale. iPS cells are reprogrammed human adult cells that have been reverted back to their stem cells to provide one of the most reliable disease models. Stem cells are notoriously difficult to edit and can require a significant amount of energy and funding to attempt to do so. Now, Synthego’s proprietary cell editing platform removes the barrier of difficulty with iPS cells to ultimately accelerate therapy development and advancements for complicated neurodegenerative diseases such as cystic fibrosis, and other neurodegenerative diseases such as Alzheimer’s, the company said this morning.

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Alzheimer’s disease research is at the core of a new project Synthego was tapped for by the National Institute on Aging at the National Institutes of Health. The company will study genetic mutations in Alzheimer's disease and related dementias. Synthego’s genome-edited iPS cells will introduce mutations relating to the dementias being studies. These edited cells will then be characterized functionally by NIA researchers to better understand these insidious diseases and identify new ways to treat them, the company said.

With the new offering of iPS cells via knockouts, single nucleotide variants, and tags, Synthego ensures pluripotency and editing efficiencies as high as 90% to enable scientists to generate edits at a massive scale to accelerate research and disease modeling, the company said this morning.

In addition to the program with the National Institutes of Health, Synthego also announced stem cell research pioneer Bill Skarnes joined the company’s scientific advisory board. Skarnes is currently a professor and director of Cellular Engineering at The Jackson Laboratory. Earlier in his career, Skarnes bladed a trail in stem cell research by pioneering gene-trapping technology in mouse embryonic stem cells.

“Synthego is enabling a long-overdue shift in the way models of diseases are generated through the development of optimized methods for gene editing pluripotent stem cells at scale," Skarnes said in a statement.

Paul Dabrowski, chief executive officer of Synthego, said being tapped by the NIH and onboarding Skarnes as an advisor, is a “huge testament” to the company’s mission of making genome engineering more accessible to scientists and drug developers.

“Today’s launch is a significant milestone in accelerating time-to-market and bringing down the cost of life-changing therapeutics, and we are excited to partner with industry and academia to make that vision a reality,” Dabrowski said in a statement.

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