Press Release
Vertex and CRISPR Therapeutics Establish Collaboration to Use CRISPR-Cas9 Gene Editing Technology to Discover and Develop New Treatments for Genetic Diseases
-Gene editing technology to be used to discover treatments to address the mutations and genes known to cause and contribute to cystic fibrosis-
-Vertex and CRISPR to utilize gene editing approach to discover treatments for genetic diseases, including sickle cell disease-
-Companies establish four-year research collaboration; CRISPR to
receive
"CRISPR-Cas9 is an important scientific and technological breakthrough
that holds significant promise for the future discovery of potentially
transformative treatments for many genetic diseases," said
"Vertex has a track record of developing innovative medicines for cystic
fibrosis and other serious diseases, making them a great partner to
accelerate the therapeutic promise of gene editing," said
About the Collaboration
Under the terms of the collaboration, Vertex and CRISPR will jointly use
the CRISPR-Cas9 technology to discover and develop potential new
treatments that correct defects in specific gene targets known to cause
or contribute to particular diseases. The initial focus of the
collaboration will be on the use of CRISPR-Cas9 to potentially correct
the mutations in the cystic fibrosis transmembrane conductance regulator
(CFTR) gene known to result in the defective protein that causes CF and
to edit other genes that contribute to the disease. Additionally, the
companies will seek to discover and develop gene-based treatments for
hemoglobinopathies, including sickle cell disease. Additional discovery
efforts focused on a specified number of other genetic targets will also
be conducted under the collaboration. Discovery activities will be
conducted primarily by CRISPR, and the related expenses will be fully
funded by Vertex. Vertex has the option to an exclusive license for up
to six gene-based treatments that emerge from the four-year research
collaboration. Vertex will fund 100 percent of the development expenses
of licensed treatments. For each of the up to six treatments in-licensed
for development, Vertex will pay future development, regulatory and
sales milestones of up to
Vertex and CRISPR will collaborate on the research, development and commercialization of treatments for hemoglobinopathies that emerge from the collaboration. Specifically for hemoglobinopathies, including treatments for sickle cell disease, Vertex and CRISPR will equally share all research and development costs and sales, with CRISPR Therapeutics leading commercialization efforts in the U.S. For all other diseases, Vertex will lead all development and global commercialization activities.
Vertex will pay CRISPR
About Gene Editing with CRISPR-Cas9
"CRISPR" refers to Clustered Regularly Interspaced Short Palindromic Repeats that occur in the genome of certain bacteria, from which the system was discovered. Cas9 is a CRISPR-associated endonuclease (an enzyme) known to act as the "molecular scissors" that cut and edit, or correct, disease-associated DNA in a cell. A guide RNA directs the Cas9 molecular scissors to the exact site of the disease-associated mutation. Once the molecular scissors make a cut in the DNA, additional cellular mechanisms and exogenously added DNA will use the cell's own machinery and other elements to specifically ‘repair' the DNA. This technology may offer the ability to directly modify or correct the underlying disease-associated changes in the human genome for the potential treatment of a large number of both rare and common diseases.
About Vertex
Vertex is a global biotechnology company that aims to discover, develop and commercialize innovative medicines so people with serious diseases can lead better lives. In addition to our clinical development programs focused on cystic fibrosis, Vertex has more than a dozen ongoing research programs aimed at other serious and life-threatening diseases.
Founded in 1989 in Cambridge, Mass., Vertex today has research and
development sites and commercial offices in the United
States, Europe, Canada and
About CRISPR Therapeutics
The mission of CRISPR Therapeutics is to develop transformative
gene-based medicines for patients with serious diseases. Our therapeutic
approach aims to cure diseases at the molecular level using the
breakthrough gene editing technology called CRISPR-Cas9. With our
multi-disciplinary team of world-renowned academics, drug developers and
clinicians, we are uniquely positioned to translate CRISPR-Cas9
technology into human therapeutics. We have licensed the foundational
CRISPR-Cas9 patent estate for human therapeutic use from our scientific
founder, Dr.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, including, without
limitation, Dr. Altshuler's statements in the second paragraph of the
press release, Dr. Novak's statements in the third paragraph of the
press release and the information provided regarding the future
development of treatments for genetic diseases using the CRISPR-Cas9
technology. While Vertex believes the forward-looking statements
contained in this press release are accurate, these forward-looking
statements represent the company's beliefs only as of the date of this
press release and there are a number of factors that could cause actual
events or results to differ materially from those indicated by such
forward-looking statements. Those risks and uncertainties include, among
other things, that data may not support further development of the
gene-based treatments subject to the collaboration due to safety,
efficacy or other reasons, and other risks listed under Risk Factors in
Vertex's annual report and quarterly reports filed with the
(VRTX-GEN)
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