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Mar 31, 2016
 |  BC Innovations  |  Emerging Company Profile

Going in alone

Etagen is editing genes using oligos alone
Etagen Pharma, Cambridge, Mass.

Etagen Pharma has developed a single-component gene editing system that capitalizes on the cell's error-free repair machinery, and uses no foreign proteins. That means it can be dosed repeatedly for more shots on goal, with a lower chance of causing an immune response than standard techniques.

The technology employs only chemically modified oligomers - dubbed Etamers - encoding wild-type versions of mutated genes, which hybridize with genomic DNA during replication or transcription when the DNA strands separate. The cell's mismatch repair machinery or homology-directed repair (HDR) machinery corrects the mutant bases using the Etamer as a template.

CEO Tod Woolf said that's an advantage over other gene editing technologies which involve delivering enzymes that cause double-stranded breaks in DNA, and commonly rely on a different and far more error-prone repair mechanism called non-homologous...

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