Graduate School

Fyodor D. Urnov: Pioneering Gene Editing for Medical Breakthroughs

A trailblazer in the field of therapeutic genome editing, Fyodor D. Urnov’s research focuses on developing medicines for devastating genetic diseases.

Fyodor Urnov image in front of a staircase
Join us on Saturday, May 25 at 11 am at Stephen Robert Hall, True North Classroom for his forum presentation, A Gene-Edited Future for Medicine: CRISPR Cures For All In Need

Fyodor D. Urnov ‘96 Ph.D. is Professor of Molecular Therapeutics in the Department of Molecular and Cell Biology at the University of California, Berkeley and Director of Technology and Translation at the Innovative Genomics Institute (IGI). He co-developed the toolbox for human genome and epigenome editing, co-named the term “genome editing”, and was on the team to advance the first-in-human applications in a clinic. 

Urnov also helped identify the genome editing target for the first medicine approved to treat sickle cell disease and beta-thalassemia. A major goal for the field of genome editing and a key focus of Urnov's work is expanding access to CRISPR therapies (which modify genomes) for genetic diseases to those most in need. 

He will receive the Horace Mann Medal at the Doctoral Ceremony during Brown University’s Commencement weekend. 

Prior to attending Brown, Urnov completed his undergraduate studies in biology at Moscow State University in Russia. He then joined the Molecular and Cell Biology and Biochemistry (MCB) department at Brown where he earned his doctoral degree. His dissertation work focused on the DNA structure and chromatin dynamics of one of the scarce origins of replication that are thoroughly understood, initiating DNA synthesis prior to cellular division. He worked in the lab of Susan Gerbi, the George Eggleston Professor of Biochemistry and founding chair of the MCB department. 

Urnov credits his pioneering work on gene editing to the doctoral training he received at Brown. Urnov then completed postdoctoral training at the National Institutes of Health before joining Sangamo BioSciences, a biotech firm in the San Francisco bay area as a Senior Scientist and Team Leader. 

At every stage of his career, Urnov’s exceptional work has been marked by medical breakthroughs and awards. One of the most groundbreaking biological advancements in recent years involves the ability to safely and precisely modify DNA sequences within genes - gene editing. This innovation began with the development of proteins designed to selectively bind to specific DNA sequences and enact targeted alterations. These proteins, known as "zinc-finger nucleases" or ZFNs, have paved the way for transformative research in genetic engineering.

In 2005 at Sangamo, Urnov spearheaded a pivotal study showcasing the efficacy of ZFNs to precisely target a disease-causing  sequence in the genome and correct it. The study was published in the journal Nature. The field of therapeutic genome editing, which Urnov co-named, was thus born. This paper marked the inaugural instance of mutation correction in human cells. The study demonstrated remarkably efficient repair (i.e. editing) of a mutated gene linked to severe combined immune deficiency, underscoring the potential of gene editing technology in addressing genetic disorders - potential that has recently started to be realized.

After this initial publication, interest in using gene editing technology exploded. Stuart Orkin, the David G. Nathan Distinguished Professor of Pediatrics at Harvard Medical School and Investigator at the Howard Hughes Medical Institute collaborated with Urnov to use gene editing to cure sickle cell disease (SCD) and beta-thalassemia (both inherited blood disorders), ushering in the first CRISPR gene editing clinical trial for a genetic disease, treating both SCD and thalassemia patients. In both of these inherited diseases, the gene for making beta-hemoglobin is disrupted. 

“Fyodor Urnov has been a visionary in the field of gene manipulation and editing, and is widely recognized both for his scientific contributions and his remarkable skill in communicating the work to other scientists and the public,” shares Orkin.

The outcome of the clinical trials have thus far been transformative for the around 100 patients involved; all have been symptom-free after gene editing. Based on these results the FDA has approved this approach as the first-ever gene-editing based medicine - a medicine for which a key foundation was the work Urnov did in collaboration with Orkin.

Urnov’s other collaborations at Sangamo led to the deployment of genome editing in human pluripotent stem cells (hPSCs) for basic science and translational applications. Examples include applied gene editing to Down syndrome and in vivo therapeutics for Huntington’s disease and Alzheimer’s dementia.

In 2019 Urnov moved to the University of California, Berkeley, where he took on the challenge of building CRISPR Cures research and development teams for genetic diseases of the blood and the brain, genetic disorders of the immune system, radiation injury, cystic fibrosis, and neurological disorders. 

Urnov explains gene editing technology in a New York Times article from December of 2022.

“Gene editing relies on a molecular machine called CRISPR, which can be instructed to repair a mutation in a gene in nearly any organism, right where that “typo” occurs. Impressively versatile, potential applications for CRISPR range from basic science to agriculture and climate change. In medicine, CRISPR gene editing allows physicians to directly fix typos in the patients’ DNA. And so much substantive progress has been made in the field of genetic medicine that it’s clear scientists have now delivered on a remarkable dream: word-processor-like control over DNA.” 

As Urnov explains in this piece, a wealth of regulatory hurdles and healthcare economics challenges have, to date, prevented gene editing from making a greater impact. Urnov shares, “the invention of CRISPR gene editing gave us remarkable treatment powers, yet no one should do a victory lap. Scientists can rewrite a person’s DNA on demand. But now what? Unless things change dramatically, the millions of people CRISPR could save will never benefit from it. We must, and we can, build a world with CRISPR for all.”

An effort to bring us closer to that world is now the centerpiece of Urnov’s professional life. His work currently focuses on developing scalable, affordable platforms to engineer gene editing cures on-demand for severe disorders of childhood. Urnov directs a unique academia-industry partnership, the IGI-Danaher Beacon for CRISPR Cures, that is advancing to the clinic innovative treatments for inborn errors of immunity that cause severe diseases of infancy.

Urnov has made an impact at UC Berkeley and IGI beyond his research. As the Covid-19 pandemic commenced, he assumed the task of organizing resources to set up a nonprofit diagnostic clinical laboratory at IGI for swift testing of the SARS-CoV-2 coronavirus. The objective was to offer greater throughput, faster results, and enhanced accuracy compared to existing commercial options - and provide such testing for free to communities most in need. 

As described in Walter Isaacson’s best selling book, The Codebreaker, Urnov emerged as a pivotal figure in this initiative, playing a significant role in resource mobilization encompassing equipment, personnel, and funding - and ultimately providing over 500,000 free COVID tests to individuals in socioeconomically disadvantaged communities when for-profit testing laboratories failed at the task.

Not only is Urnov renowned in the field of gene editing, but his list of publications, teaching ability, and public speaking acumen is also exceptional. Urnov has authored more than 100 scientific publications and is an inventor on 87 published patents related to genome editing and targeted gene regulation technology. His 2005 Nature paper has been cited over 2000 times, and a subsequent paper he wrote for Nature Reviews Genetics has been cited over 2500 times. Many of his other papers have been cited over 1000 times.

“Fyodor is a world class researcher at the forefront of arguably the most exciting and important biomedical research advance in our lifetimes – genome editing – because he is perhaps the most engaging orator I have ever heard speak, because he is a scholar of truly extraordinary depth and breadth of knowledge in biomedicine, and because he is a dedicated and highly effective teacher and mentor,“ shares David Drubin, Ernette Comby Chair in Microbiology and a professor of Cell and Development in the Department of Molecular and Cell Biology at UC Berkeley.

Urnov is also known for being a dynamic public speaker and teacher and is much sought after. Urnov credits his experience as a graduate student instructor here at Brown for his interest in teaching, starting with watching faculty at Brown, including George Eggleston Professor of Biochemistry, Susan Gerbi and Professor of Molecular Biology, Cell Biology and Biochemistry  Kenneth Miller, in his first stint as a graduate student instructor. 

Urnov’s awards, not surprisingly, are quite notable. As far back as his time at Brown he was selected for the Barry J. Rosen Memorial Award For High Achievement In Molecular Biology and the President’s Award for Excellence in Teaching.

In 2014 he was named as one of “The World’s Most Influential Scientific Minds” by Thomson Reuters and received a Fellows Award for Research Excellence from the National Institutes of Health.