SAN FRANCISCO--(BUSINESS WIRE)-- Trucode Gene Repair, Inc. announced today that it is advancing its next-generation in vivo gene editing technology to correct mutations that cause sickle cell disease, cystic fibrosis, and other devastating genetic disorders. With $34 million from investors including Kleiner Perkins and GV (formerly known as Google Ventures), Trucode has now emerged from stealth-mode after establishing a broad and exclusive intellectual property estate for its triplex gene editing technology, assembling a cohesive team of industry veterans, and identifying 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 Dr. Marshall Fordyce, Founder and Chief Executive Officer.

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 MD, PhD; Mark Saltzman, PhD; and Marie Egan, MD, 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.

Leadership and Board of Directors

Trucode is led by CEO Marshall Fordyce, MD, who brings more than a decade of industry experience leading teams in drug development, clinical translation, and commercialization. Dr. Fordyce founded Trucode while an entrepreneur-in-residence at Kleiner Perkins with Beth Seidenberg, MD. Previously, he served as Senior Director of Clinical Research at Gilead Sciences, Inc. where he contributed to seven new drug approvals and led Gilead’s highly successful TAF/GENVOYA clinical development program.

Research and Development programs are led by Allen Ebens, PhD, Chief Scientific Officer, who has over 20 years of industry experience (Genentech, Juno, NGM) bringing novel molecules with different modalities from concept to clinic. Lauren Frenz is Senior Vice President of 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, PhD, with over 25 years of industry experience in PNA chemistry and Susan K. Whoriskey, PhD, who was previously on the founding Executive Teams of Cubist Pharmaceuticals, Momenta Pharmaceuticals and Moderna.

Dr. Fordyce noted, “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.”

The company has appointed the following individuals to its Board of Directors:

• Beth Seidenberg, MD, General Partner, Kleiner Perkins; Managing Director, Westlake Village BioPartners
• Krishna Yeshwant, MD, General Partner, GV
• Andrew Cheng, MD, PhD, CEO of Akero Therapeutics (AKRO); former Chief Medical Officer of Gilead Sciences
• Joseph Walton, Partner, Walton, Mitchell & Co.

Long-time life sciences investor and Trucode’s founding investor, Dr. Beth Seidenberg said, “Gene editing holds enormous promise for patients with genetic diseases, and Trucode’s novel platform is well differentiated from first-generation technologies – this could be transformative for our industry.”

About Trucode Gene Repair

Trucode Gene Repair is advancing its novel triplex gene editing platform to potentially cure devastating genetic diseases, with initial 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. Trucode is headquartered in South San Francisco, CA. For more information, visit www.trucodegene.com.

About Kleiner Perkins

For five decades, Kleiner Perkins has partnered with some of the most ingenious founders in technology, helping them make history with their bold ideas. Through 20 venture funds and four growth funds, Kleiner Perkins has invested $10 billion in hundreds of companies, including pioneers such as Amazon, Genentech, and Google. Today, Kleiner Perkins continues to invest in founders and their bold ideas helping them to make history. For more information, visit www.kleinerperkins.com and follow us on Twitter @kleinerperkins.

About GV

GV provides venture capital funding to bold new companies. Across the fields of life science, enterprise technology, consumer products and services, and frontier technology, GV’s portfolio companies aim to improve lives and transform industries. GV’s team of world-class engineers, designers, physicians, scientists, marketers, and investors work together to provide startups exceptional support. Launched as Google Ventures in 2009, GV is the venture capital arm of Alphabet, Inc. GV helps startups interface with Google, providing unique access to the world’s best technology and talent. GV has more than $4.5 billion under management. Among its investments are Uber, Lime, Slack and One Medical Group. Notable investment outcomes include Nest, Flatiron Health, and Duo Security. GV is headquartered in Mountain View, California, with offices in San Francisco, Boston, New York, and London.

About the Yale Office of Cooperative Research

Since its founding in 1982, the Yale Office of Cooperative Research (OCR) has built a significant portfolio of inventions and patents and has grown into an engine of regional economic development. Its mission is to facilitate the translation of research from Yale’s labs into products and services that benefit society. OCR is recognized as a leading force for catalyzing economic growth by identifying, counseling and nurturing early-stage technologies and guiding the transition into robust companies.

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