Broad Institute, Harvard, And MIT License CRISPR-Cas9 Technology To Editas Medicine For Therapeutic Applications

BOSTON and CAMBRIDGE, Mass., Dec. 1, 2014 /PRNewswire-USNewswire/ -- The Broad Institute, Harvard University, the Massachusetts Institute of Technology and Editas Medicine have entered into a worldwide license agreement to grant Editas access to intellectual property related to certain genome editing technology for the development of human therapeutic applications.

The agreement relates to technology that engineers the CRISPR-Cas9 system—a naturally-occurring part of the bacterial immune system. Researchers at Harvard Medical School, the Wyss Institute for Biologically Inspired Engineering at Harvard University, Broad Institute, MIT, the McGovern Institute for Brain Research at MIT, and Harvard University Faculty of Arts and Sciences (FAS), have optimized the CRISPR-Cas9 system to allow for insertion, replacement, and regulation of targeted genes in higher organisms, with the potential to one day be used in humans. This technology has wide-ranging therapeutic potential and could lay the groundwork for treating diseases where a gene's expression needs to be altered (such as turning down CCR5 in HIV), or where a mutation needs to be repaired (such as sickle cell diseases or hemophilia). In addition to their therapeutic implications, CRISPR-Cas9 systems enable scientists to modify genes and better understand the biology of living cells and organisms.

"The Broad, MIT, and Harvard share the goal of developing innovative technologies such as CRISPR-Cas9 and promoting their translation to benefit patients," said Eric Lander, president and director of the Broad Institute. "We're committed to making these technologies broadly available for research and also ensuring that therapeutic development – bringing this technology to the clinic – has the best chance of success."

The agreement includes a mechanism to ensure that no promising target genes will be neglected; genes that are not being pursued by Editas will be made available for licensing to other parties so that new medicines based on this technology can be developed for any disease that could be treated by this approach. Broad Institute, MIT, and Harvard University partners have made CRISPR-Cas9 technology broadly available to the research community, and have freely granted licenses to academic scientists, and non-exclusively to industry partners, for development of research tools and reagents and will continue to do so.

Also included in the agreement are additional technologies relating to engineering and optimization of transcription activator-like effector (TALE) proteins that can also be programmed to target and modify specific genes, as well as a novel protein-based drug delivery system, which could potentially achieve up to one thousand-fold more effective drug delivery than conventional methods.

"We have already seen how the CRISPR molecular system has proven to be so powerful in basic research," said Jeffrey S. Flier, Dean of Harvard Medical School. "The potential for this approach to translate into new ways to treat human conditions that have proved vexing is compelling and warrants new and innovative collaborations among academia and industry."

"The CRISPR-Cas9 technology represents yet another great example of how new insights into nature's design principles can be rapidly leveraged to develop new engineering innovations, in this case genome reengineering methods that can be used to create an entirely new class of targeted therapeutics", said Wyss Institute Founding Director Donald Ingber, M.D., Ph.D. "This breakthrough also demonstrates our collective commitment to accelerate the transition from fundamental discovery to clinical application." 

About the engineered CRISPR-Cas9 system
CRISPRs (Clustered Regularly Interspaced Short Palindromic Repeats) have recently been harnessed as genome editing tools in a wide range of plant and animal species. The engineered CRISPR-Cas9 system allows researchers to mutate or change the expression of genes in living cells, including those of humans. The family of Cas9 nucleases (also known as Cas5, Csn1, or Csx12) recognizes DNA targets when combined with correlating RNA guides. Researchers can now harness the engineered system to home in on specific nucleic acid sequences and edit the DNA at those precise locations in the genome, allowing researchers to study the genes' function.

The licensed technology was developed at the Broad Institute, the Wyss Institute for Biologically Inspired Engineering at Harvard University, Harvard Medical School, Harvard University Faculty of Arts and Sciences, and the Massachusetts Institute of Technology through the innovative work of:

George Church, Ph.D., Wyss Institute Core Faculty Member, Robert Winthrop Professor of Genetics at Harvard Medical School, Professor of Health Sciences and Technology at Harvard and MIT, and Senior Associate Member at the Broad Institute of Harvard and MIT.

David Liu, Ph.D., Howard Hughes Medical Institute Investigator and Professor of Chemistry and Chemical Biology at Harvard University, and Senior Associate Member at the Broad Institute of Harvard and MIT.

Feng Zhang, Ph.D., Core Member of the Broad Institute of Harvard and MIT, Investigator at the McGovern Institute for Brain Research at MIT, and Assistant Professor in the MIT Department of Brain and Cognitive Sciences and the MIT Department of Biological Engineering.

About the Broad Institute of Harvard and MIT
The Eli and Edythe L. Broad Institute of Harvard and MIT was launched in 2004 to empower this generation of creative scientists to transform medicine. The Broad Institute seeks to describe all the molecular components of life and their connections; discover the molecular basis of major human diseases; develop effective new approaches to diagnostics and therapeutics; and disseminate discoveries, tools, methods and data openly to the entire scientific community.

Founded by MIT, Harvard and its affiliated hospitals, and the visionary Los Angeles philanthropists Eli and Edythe L. Broad, the Broad Institute includes faculty, professional staff and students from throughout the MIT and Harvard biomedical research communities and beyond, with collaborations spanning over a hundred private and public institutions in more than 40 countries worldwide. For further information about the Broad Institute, go to http://www.broadinstitute.org.

About the Wyss Institute for Biologically Inspired Engineering at Harvard University
Using Nature's design principles, the Wyss Institute (http://wyss.harvard.edu) develops bioinspired materials and devices that will transform medicine and create a more sustainable world. Working as an alliance among all of Harvard's Schools, and in partnership with Beth Israel Deaconess Medical Center, Brigham and Women's Hospital, Boston Children's Hospital, Dana-Farber Cancer Institute, Massachusetts General Hospital, the University of Massachusetts Medical School, Spaulding Rehabilitation Hospital, Boston University, Tufts University, and Charité - Universitätsmedizin Berlin, and the University of Zurich, the Institute crosses disciplinary and institutional barriers to engage in high-risk research that leads to transformative technological breakthroughs. By emulating Nature's principles for self-organizing and self-regulating, Wyss researchers are developing innovative new engineering solutions for healthcare, energy, architecture, robotics, and manufacturing. These technologies are translated into commercial products and therapies through collaborations with clinical investigators, corporate alliances, and new start-ups.

About Harvard Medical School
Harvard Medical School (http://hms.harvard.edu) has more than 7,500 full-time faculty working in 11 academic departments located at the School's Boston campus or in one of 47 hospital-based clinical departments at 16 Harvard-affiliated teaching hospitals and research institutes. Those affiliates include Beth Israel Deaconess Medical Center, Brigham and Women's Hospital, Cambridge Health Alliance, Boston Children's Hospital, Dana-Farber Cancer Institute, Harvard Pilgrim Health Care, Hebrew Senior Life, Joslin Diabetes Center, Judge Baker Children's Center, Massachusetts Eye and Ear Infirmary, Massachusetts General Hospital, McLean Hospital, Mount Auburn Hospital, Schepens Eye Research Institute, Spaulding Rehabilitation Hospital and VA Boston Healthcare System. 

About Harvard University Faculty Arts and Sciences
Founded in 1890, the Faculty of Arts and Sciences (FAS) is the largest division of Harvard University. The Faculty of Arts and Sciences is dedicated to being at the forefront of teaching and learning and fostering cutting edge research and discovery. FAS is redefining liberal arts education for the 21st century and is committed to an open Harvard and student aid by making a Harvard education accessible to students from all backgrounds. FAS comprises Harvard College and the Graduate School of Arts and Sciences, including undergraduate and graduate admissions; the School of Engineering and Applied Sciences; and the Division of Continuing Education, including the Extension and Summer Schools. FAS also encompasses academic resources, such as libraries and museums, as well as campus resources and athletics.

About Harvard University's Office of Technology and Development
Harvard's Office of Technology Development ('OTD') is responsible for all activities pertaining to the evaluation, patenting and licensing of new inventions and discoveries made at Harvard University and Harvard Medical School. OTD also serves to further the development of Harvard technologies through the establishment of sponsored research collaborations with industry. OTD's mission is to promote the public good by fostering innovation and translating new inventions made at Harvard into useful products available and beneficial to society.

For more information, contact:

Broad Institute of MIT and Harvard
Paul Goldsmith
+1 617.714.7478
[email protected]

Wyss Institute for Biologically Inspired Engineering at Harvard University
Kat J. McAlpine
+1 617.432.8266
[email protected]

Harvard Medical School
David Cameron
+1 617.432.0441
[email protected]

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SOURCE Wyss Institute for Biologically Inspired Engineering at Harvard University