Kleiner Perkins and GV have powered Trucode Gene Repair to a $34 million financing round. Trucode will use the money to advance gene editing assets designed to correct the mutations behind diseases including sickle cell and cystic fibrosis.
The startup is built on the work of Yale University professors Peter Glazer, Mark Saltzman and Marie Egan, who showed that synthetic peptide nucleic acid (PNA) oligomers and donor DNA can cause the correction of gene mutations in animal models. When the PNA binds to the target gene, it creates a three-stranded helix. The body’s genetic repair systems recognize the third PNA strand as being out of place and remove it, leaving the donor DNA behind and integrated into the body.
Marshall Fordyce, formerly of Gilead Sciences, identified the potential of the approach while working as an entrepreneur in residence at Kleiner, leading him to found Trucode with the venture capital shop’s former general partner Beth Seidenberg.
Led by Fordyce, Trucode is now working to realize that potential. The $34 million will equip Trucode to move candidates toward and into IND-enabling studies while exploring an array of discovery-stage opportunities presented by the approach.
Trucode has advanced furthest in sickle cell disease, taking a program through lead optimization and to the cusp of IND-enabling studies. The cystic fibrosis program is entering lead optimization, while a plethora of other opportunities spanning blood, liver, cardiac and neurological diseases are back at the discovery stage.
Multiple other companies are seeking to address the genetic causes of many of the same diseases targeted by Trucode. The bet that Trucode is better placed to treat the diseases than the competition rests on the features of its novel approach to gene editing.
“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. 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,” Yales’s Glazer told the National Institutes of Health last year.
Those perceived advantages are particularly relevant to some of the diseases targeted by Trucode. In cystic fibrosis, for example, the target lung cells need to be treated in vivo, unlike in blood diseases in which the cells can easily be removed and treated ex vivo.