Tech Transfer eNews Blog

Yale start-up raises $34M to advance gene editing technology


By Jesse Schwartz
Published: September 18th, 2019

A Yale University start-up has raised $34 million to commercialize a gene editing technology designed to treat diseases including sickle cell and cystic fibrosis.

Trucode Gene Repair is based on the work of Yale researchers Peter Glazer, Mark Saltzman and Marie Egan, who discovered that synthetic peptide nucleic acid (PNA) oligomers and donor DNA can be used to correct gene mutations in animal models. Marshall Fordyce, formerly of Gilead Sciences, serves as Trucode’s CEO.

The gene-editing technology works by binding the PNA to the target gene, which creates a three-stranded helix. The body’s genetic repair system sees the third PNA strand as being out of place and removes it, leaving the donor DNA behind and integrated into the body.

“One of the big advantages of PNA and donor DNA is that the off-target rate is much lower than CRISPR, the most well-known gene editing tool,” says Glazer. “The other advantage is that they can be administered in vivo, by simple intravenous injection of the nanoparticles. You don’t have to take the cells out of the body, because the nanoparticles provide a very effective and safe delivery method.”

Kleiner Perkins and Google Ventures led the $34 million funding round, which will help Trucode move its candidates toward IND-enabling studies while exploring a range of discovery-stage opportunities presented by their approach.

Source: Fierce Biotech

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