Trucode’s triplex gene-editing technology uses proprietary synthetic peptide nucleic acid oligomers and DNA correction sequences to edit disease-causing mutations by harnessing natural DNA repair mechanisms.
Biotech startup Trucode Gene Repair, Inc. emerged from stealth mode this morning with $34 million in financing to bolster its triplex gene-editing technology.
Bay Area-based Trucode’s triplex gene-editing technology uses proprietary synthetic peptide nucleic acid (PNA) oligomers and DNA correction sequences to edit disease-causing mutations by harnessing natural DNA repair mechanisms. Unlike current CRISPR-based gene-editing technologies, Trucode said its triplex gene-editing technology does not cause double-stranded breaks seen with CRISPR and other nuclease-based editing technologies. Additionally, Trucode said its technology has the potential for intravenous delivery without viral vectors.
Marshall Fordyce, founder and chief executive officer of Trucode, said that although the promise of gene-editing to cure genetic diseases is out there, the technology has not realized its “full transformative potential.”
“Our technology could address key challenges faced by the industry, including editing fidelity, immune reactions, delivery, scaled manufacturing, and intellectual property,” Fordyce said in a statement.
In early-stage research conducted in the Yale University labs of Peter Glazer, Mark Saltzman and Marie Egan, PNAs loaded into biodegradable polymer nanoparticles induce gene-editing and reverse disease phenotype in multiple animal models of disease, including beta-thalassemia and cystic fibrosis. In beta-thalassemia mice, intravenous dosing of nanoparticles achieved sufficient gene correction in bone marrow-derived hematopoietic stem cells to durably reverse the disease phenotype, as assessed by multiple clinically relevant endpoints, Trucode said in its announcement. The study was the first in vivo demonstration that PNAs might effectively translate into a clinically practical platform for human gene correction, the company said.
Trucode emerged from stealth mode backed by investors Kleiner Perkins and GV (formerly Google Ventures).
In addition to Gilead Sciences veteran Fordyce, who founded Trucode while an entrepreneur-in-residence at Kleiner Perkins with Beth Seidenberg, Trucode has tapped a number of industry veterans to guide the company. Research and Development programs are led by Chief Scientific Officer Allen Ebens, who has over 20 years of industry experience with companies such as Genentech and Juno. Lauren Frenz heads up corporate strategy and finance. She has a decade of commercial, finance, and strategic planning experience from Gilead Sciences and SVB Leerink. Advisors include Jim Coull, with over 25 years of industry experience in PNA chemistry and Susan K. Whoriskey, who was previously on the founding executive teams of Cubist Pharmaceuticals, Momenta Pharmaceuticals and Moderna.
Seidenberg noted that gene-editing holds enormous promise for patients with genetic diseases. She said Trucode’s novel platform is well-differentiated from first-generation technologies, which could be transformative for the industry.
“In a short amount of time, we have attracted a strong team of seasoned biotech professionals with deep expertise in drug development to realize the transformative potential of this technology. We now have the ability to fully explore new chemical space and advance our pipeline of novel candidates directed to established genetic targets. This rare opportunity to develop potentially curative next-generation gene-editing therapeutics for the benefit of patients inspires me every day,” Fordyce added.
