RARE Daily

Trucode Launches to Advance Next-Generation Gene Editing Platform

September 10, 2019

Trucode Gene Repair, which is developing next-generation in vivo gene editing technology to correct mutations that cause sickle cell disease, cystic fibrosis, and other genetic disorders, launched its operations with $34 million in venture funding from Kleiner Perkins and GV.

Photo: Marshall Fordyce, founder and CEO

Based in San Francisco, Trucode has established a broad and exclusive intellectual property estate for its triplex gene editing technology, assembled a team of industry veterans, and identified lead candidates in multiple clinical indications.

“The medical promise of gene editing to cure patients with genetic disease has arrived but 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,” said Marshall Fordyce, founder and CEO.

Fordyce founded Trucode while an entrepreneur-in-residence at Kleiner Perkins with Beth Seidenberg.

The company will initially focus on sickle cell disease and cystic fibrosis. The elegance of triplex gene editing lies in its ability to harness natural, high-fidelity DNA repair mechanisms, and its independence from the requirement for exogenous nucleases and viral vectors.

Triplex gene editing technology employs proprietary synthetic peptide nucleic acid (PNA) oligomers and DNA correction sequences to edit disease-causing mutations by harnessing natural DNA repair mechanisms. The mechanism of editing does not cause double-stranded breaks seen with CRISPR and other nuclease-based editing technologies and has the potential for intravenous delivery without viral vectors.

Trucode’s platform builds on the pioneering work of professors Peter Glazer, Mark Saltzman, and Marie Egan, and their teams at Yale University. In several studies published in Nature Communications, they demonstrated that 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.

This was the first in vivo demonstration that PNAs might effectively translate into a clinically practical platform for human gene correction. Trucode holds the exclusive worldwide license to this technology from the Yale University Office of Cooperative Research, and additional licenses from Carnegie Mellon University.

“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,” said Fordyce. “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.”

Author: Rare Daily Staff

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