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
.
Pipeline
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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