Genetic Engineering and Biotechnology News

CRISPR molecular scissors

The State of CRISPR & Genome Editing

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Panelists:

Image of Feng Zhang, PhD

Feng Zhang, PhD

Core Institute Member, Broad Institute; Investigator at McGovern Institute for Brain Research; HHMI Investigator

Panelist

Image of Feng Zhang, PhD

Feng Zhang, PhD

Feng Zhang is a core institute member of the Broad Institute of MIT and Harvard, as well as an investigator at the McGovern Institute for Brain Research at MIT, co-director of the K. Lisa Yang and Hock E. Tan Center for Molecular Therapeutics at MIT, the James and Patricia Poitras Professor of Neuroscience at MIT, and a professor at MIT, with joint appointments in the departments of Brain and Cognitive Sciences and Biological Engineering. Zhang is also an investigator at the Howard Hughes Medical Institute.

Feng is a molecular biologist seeking to improve human health by discovering approaches to modulate cellular programs. These approaches include developing molecular technologies for modifying the cell’s genetic information and the delivery vehicles needed to get these tools into the right cells. Zhang hopes to apply these approaches to neurodegenerative diseases, immune disorders, aging, and other disease contexts. Zhang pioneered the development and use of CRISPR-Cas9 as a genome editing tool in human cells. He has expanded this toolbox by harnessing new CRISPR and CRISPR-associated systems. These new tools not only include additional DNA-targeting CRISPR systems, but also systems that target RNA as well as systems that insert large stretches of DNA. Zhang has also developed new methods to deliver these tools into human cells. In 2023, the first Cas9-based therapeutic, which is based on a design Zhang and colleagues developed in 2015, was approved for clinical use to treat sickle cell disease.

Feng received his AB in chemistry and physics from Harvard College and his PhD in chemistry from Stanford University. He is a recipient of many awards including the Lemelson-MIT Prize, the Tang Prize, the Canada Gairdner International Award, the Merkin Institute Fellowship at the Broad, and the National Medal of Technology and Innovation. Zhang is an elected member of the National Academy of Sciences, the National Academy of Medicine, and the American Academy of Arts and Sciences.

Image of Fyodor Urnov, PhD

Fyodor Urnov, PhD

Director, Technology & Translation, Innovative Genomics Institute, Professor of Molecular and Cell Biology at UC Berkeley

Panelist

Image of Fyodor Urnov, PhD

Fyodor Urnov, PhD

Fyodor Urnov is a Professor of Molecular and Cell Biology at UC Berkeley and Director of Technology & Translation at the Innovative Genomics Institute (IGI). A pioneer in the field of therapeutic genome editing, Fyodor’s research focuses on advancing genome editing technology and pushing the boundaries of how it can be applied to solve real-world problems.

Over 15 years at Sangamo Therapeutics, Fyodor and colleagues demonstrated the first use of zinc-finger nucleases to edit DNA in human cell and coined the term “genome editing” in 2005. Fyodor led collaborative teams to establish at-scale applications of genome editing for human somatic cell genetics and model animal and crop reverse genetics. Fyodor was a key member of the team that developed the first-in-human application of genome editing (2009) and then led a cross-functional team from basic discovery to IND of first-in-human clinical trials for the hemoglobinopathies beta-thalassemia and sickle cell disease (trials currently ongoing in partnership with UCSF Benioff Children’s Hospital and UCLA Broad Stem Cell Research Center).

At the IGI, Fyodor works in collaborative teams to develop first-in-human applications of experimental CRISPR-based therapeutics for sickle cell disease (with Mark Walters, UCSF), genetic disorders of the immune system (with Alexander Marson, UCSF/IGI), radiation injury (with Jonathan Weissman, MIT/Whitehead Institute), cystic fibrosis (with Ross Wilson, IGI), and neurological disorders (with Weill Neurohub and Roche/Genentech).

Fyodor trained as an undergraduate in Biology at Moscow State University and then studied the interplay between chromatin and transcription factors for his PhD at Brown University (with Susan Gerbi) and as a postdoctoral fellow at the NIH (with the late Alan Wolffe).

Image of Beth Shapiro, DPhil

Beth Shapiro, DPhil

CSO, Colossal Biosciences; Professor of Ecology and Evolutionary Biology, UC Santa Cruz

Panelist

Image of Beth Shapiro, DPhil

Beth Shapiro, DPhil

Beth Shapiro is the chief science officer at Colossal Biosciences, a biotech company using gene editing for the de-extinction of species including the dire wolf and woolly mammoth. She is currently on leave from her academic position as professor of ecology and evolutionary biology at UC Santa Cruz, where she leads the USCS Paleogenomics lab with Ed Green. A leader in the field of paleogenomics and ancient DNA, Shapiro uses genetic material recovered from the remains of plants and animals ago to study evolution and explore how species and ecosystems have changed over time. Beth’s lab develops techniques to recover increasingly trace amounts of DNA from museum, environmental, and forensic samples, and then uses these data to study how species and ecosystems evolved through time.

Beth took her undergraduate degree from the University of Georgia and obtained her PhD (DPhil) in zoology from the University of Oxford in 2003 as a Rhodes scholar. A 2009 MacArthur Fellow, Shapiro is also an award-winning author. Her books include How to Clone a Mammoth: The Science of De-Extinction (Princeton University Press, 2016) and Life as We Made It (Basic Books, 2021), in which she explores the genetic modification of living things in the context of history, archeology, paleontology, and genomics. Beth is a former Howard Hughes Medical Institute investigator and an elected member of the American Academy of Arts and Sciences.

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It has been 20 years since the term “genome editing” first entered the scientific lexicon, a field that was revolutionized by the development of CRISPR gene editing in 2012. But the remarkable pace of progress in the first decade has hit some headwinds of late. In particular, the use of CRISPR as a clinical tool has progressed more slowly than anticipated, putting pressure on many gene editing companies to narrow their pipelines to conserve resources and highlighting the uphill challenge to deliver the potential of CRISPR to patients worldwide. Nevertheless, new discoveries are expanding the gene editing toolbox in novel and exciting directions.

In The State of CRISPR and Genome Editing virtual summit, GEN brings together an outstanding line-up of pioneers and practitioners in genome editing to provide a compelling and candid overview of the field. Attendees will hear about the latest scientific, technological, commercial and regulatory advances that will enable genome editing to unleash its full potential in the clinic and beyond.

Highlights of this summit include:

  • A keynote lecture from Feng Zhang, PhD (Broad Institute) on precision genome editing with and without CRISPR.
  • Beth Shapiro, PhD (Colossal Biosciences) discusses gene editing milestones, de-extinction and dire wolves. 
  • Fyodor Urnov, PhD (Innovative Genomics Institute) takes a look at the path to achieving CRISPR cures for hundreds of rare genetic diseases.  
  • The state of cell and gene therapy in Africa with Julie Makani, MD, PhD (Tanzania) and Johnny Mahlangu (South Africa).
  • A conversation on base editing and more with Alexis Komor, PhD (UC San Diego) and Nicole Gaudelli, PhD (Google Ventures).
  • Neville Sanjana, PhD (New York Genome Center) discussing the intersection of AI and CRISPR. 
  • Breakout sessions from the summit sponsors.

Registration is free! Join us on June 11 for The State of CRISPR & Genome Editing. 

Guest Speakers Include

The State of CRISPR and Genome Editing speakers

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