What You Need to Know About CRISPR

What You Need to Know About CRISPR
December 1, 2015
By Mark Terry, BioSpace.com Breaking News Staff

CRISPR Therapeutics is headquartered in Basel, Switzerland, and has research and development operations in Cambridge, Mass. where the company’s focus is on developing gene editing technology called CRISPR-Cas9.

CRISPR stands for clustered, regularly interspaced palindromic repeats. With Cas9, or CRISPR-associated protein 9, the technology platform is a way to turn a bacterial defense mechanism against viruses into a tool to edit genes.

The technology was discovered by UC Berkeley professor Jennifer Doudna and Emmanuelle Charpentier professor Jennifer Doudna and Emmanuelle Charpentier of the Helmholtz Centre for Infection Research in Braunschweig, Germany. Doudna went on to found Caribou Biosciences, which in 2015 developed a collaboration agreement with the Novartis Institute for Biomedical Research in Cambridge, Mass. Caribou then founded another firm, also in Cambridge, Intellia Therapeutics. Charpentier sold her part of the rights of the CRISPR-Cas9 platform to CRISPR Therapeutics.

“CRISPR-Cas9 is a breakthrough in our ability to treat serious diseases,” Lundberg said in a statement in April 2015, “and we are now focused on translating this discovery into important new therapies for patients.”

Company Leadership
Rodger Novak—founder, chief executive officer and director. A former professor at the Vienna Biocenter in Austria, before CRISPR he was global head of Anti-Infectives Research & Development for Paris-based Sanofi . Prior to that, he founded Nabriva Therapeutics and served as its chief operating officer.

Bill Lundberg—chief scientific officer. Previously the vice president and head of Translational Medicine at Alexion Pharmaceuticals Inc. , Lundberg has also held senior positions at Taligen Therapeutics, Antisoma, Xanthus, Wyeth and Genzyme .

Emmanuelle Charpentier—scientific founder. She is a professor at the Helmholtz Centre for Infection Research and Hannover Medical School, Germany, and the Laboratory for Molecular Infection Medicine at Umea University, Sweden. She is one of the co-discoverers of CRISPR technology.

Craig Mello—scientific founder. He is a Nobel prize-winning researcher at the Howard Hughes Medical Institute, the Blais University Chair in Molecular Medicine, and co-director of the RNA Therapeutics Institute at the University of Massachusetts Medical School.

Chad Cowan—scientific founder. He is an associate professor at Harvard University in the Department of Stem Cell and Regenerative Biology, and at Massachusetts General Hospitals, with appointments in the Center for Regenerative Medicine, the Cardiovascular Research Center and the Center for Human Genetics Research.

Company Financing
In April 2014, announced it raised $25 million in a Series A investment from Versant Ventures.

In April 2015, the company announced the closing of a Series A and Series B financing round totaling $89 million, including $35 million in new funding in the Series A round and $29 million in the Series B. The oversubscribed Series A and Series B rounds were led by strategic investors, SR One and Celgene , respectively. New investors included New Enterprise Associates and Abingworth, as well as the company’s founding investor, Versant Ventures.

Currently, the company is using its platform to treat non-germline (somatic) cells at the molecular level. As such, it is working on a portfolio of potential candidates, both in cells inside the body (in vivo) and in ex vivo (outside the body) treatments where specific cells are removed, edited using the technology, and delivered back into the patient. So far, the company is focusing on hemoglobinopathies, such as sickle cell disease and beta thalassemia, some forms of immunodeficiencies, and immune approaches to cancer treatment. It is also working with Vertex on developing therapies for sickle cell disease. Its in vivo approach has the potential to be used for diseases of the liver, eye and lung, as well as others.

Market Competition
CRISPR Cas9 is considered revolutionary technology, and as such is likely, at some level, to be used by many companies and institutions. Other companies that are directly controlling the technology include Caribou Biosciences, which has formed a strategic alliance with DuPont , and Novartis . Caribou also formed Intellia Therapeutics, which is active in this area.

The CRISPR-Cas9 technology has been the focus of several patent lawsuits. Doudna and Charpentier published the first paper describing the technology. However, Feng Zhang, a researcher at the MIT-Harvard Broad Institute filed a broad U.S. patent claim on the technology.

Another company involved is Editas Medicine, in Cambridge, Mass. Editas was founded by Doudna with Zhang and George Church.

Dollars and Deals
On Oct. 26, 2015, CRISPR announced it had entered into a strategic research collaboration with Boston-based Vertex Pharmaceuticals . The two companies plan to evaluate the use of CRISPR-Cas9 across multiple diseases where targets have been validated through human genetics. The collaboration will initially focus on cystic fibrosis and sickle cell disease.

What to Look For
The recent deal with Vertex is scheduled to run for four years, and allows Vertex the option of acquiring up to six programs, with the development costs shared between the two companies. CRISPR retains the U.S. commercialization rights for sickle cell disease. But the company’s focus is also on cystic fibrosis.

“Our mission is to provide transformative gene-based medicine for patients,” Lindberg told BioSpace in an exclusive statement. “We will leverage any technology available to treat serious diseases in ways similar to traditional and established gene therapy methods. There are many scientists and researchers using CRISPR-Cas9 to edit genes, but we have a terrific opportunity to use our technology platform to develop a portfolio of therapies for several unmet medical needs.”

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