Vertex Expands into New Disease Areas and Enhances Gene Editing Capabilities Through Expanded Collaboration with CRISPR Therapeutics and Acquisition of Exonics Therapeutics
-Provides Vertex with leading gene editing capabilities to develop novel therapies for Duchenne Muscular Dystrophy and Myotonic Dystrophy Type 1-
-CRISPR to receive an upfront payment of
-Exonics to be acquired for an upfront payment of
Vertex and Exonics Therapeutics have entered into a definitive agreement under which Vertex will acquire privately held Exonics, a company focused on creating transformative gene editing therapies to repair mutations that cause DMD and other severe neuromuscular diseases.
“Through the expanded collaboration with CRISPR and the acquisition of Exonics, we are bringing together the intellectual property, technologies, and scientific expertise needed to establish a leading gene editing platform for DMD and DM1. These transactions are highly aligned with our strategy of investing in scientific innovation to create transformative medicines for people with serious diseases,” said Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of Vertex. “We are continuing to build a toolbox of small molecule and nucleic acid technologies and capabilities that will allow us to drive scientific innovation to produce transformative medicines for a broad portfolio of diseases.”
“This agreement with Vertex reflects the strong collaboration we have
built together in other programs and underscores Vertex’s commitment to
gene editing,” said
“DMD and DM1 are devastating muscle diseases with no curative therapies
“The Duchenne community needs novel approaches to treat and cure this
devastating disease and Exonics’ technology has the potential to
dramatically improve the lives of Duchenne patients,” said
About Vertex’s Collaboration with
Under the terms of this strategic collaboration and license agreement, Vertex will pay
For the DMD program, Vertex is responsible for all research, development, manufacturing, and commercialization activities and all related costs. For the DM1 program, Vertex and CRISPR will share research costs for specified guide RNA research to be conducted by CRISPR, and Vertex is responsible for all other research, development, manufacturing, and commercialization costs.
The closing of this transaction will be subject to certain conditions, including the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act. The companies anticipate the transaction will close in the third quarter of 2019.
About Vertex’s Acquisition of Exonics Therapeutics
Exonics Therapeutics is developing gene editing therapies to treat patients with DMD and other severe genetic neuromuscular diseases and brings to Vertex intellectual property, technology, and scientific expertise in gene editing therapies for these serious diseases. In multiple small and large animal DMD preclinical models, Exonics has used SingleCut CRISPR to genetically repair and restore dystrophin, the key protein missing in children with DMD.
Exonics’ technology is licensed from
Under the terms of the acquisition, Vertex will acquire all outstanding
shares of Exonics, which will become a separate wholly-owned subsidiary
of Vertex. Exonics equity holders are eligible to receive payments of
The closing of this transaction will be subject to certain conditions, including the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act and other customary conditions. The companies anticipate the acquisition will close in the third quarter of 2019.
About Vertex’s Scientific Leadership Expansion
Vertex today announced the appointment of Dr.
With more than twenty-five years in academic and industry settings, John is a proven leader in the genetic therapies space with experience in both the scientific and manufacturing sides of product development. His capabilities and expertise are an ideal fit with Vertex’s strategy to bring the multi-faceted components of genetic therapies together and to advance the science of genetic therapies, such that the Vertex genetic therapies platform offers the best of all this emerging approach has to offer.
John previously served as Chief Scientific Officer and Senior Vice
About Vertex’s New Genetic Therapies Research Site
Vertex will establish a new genetic therapies research site in the
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.
Founded in 1989 in
For additional information and the latest updates from the company, please visit www.vrtx.com.
Vertex 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. Leiden’s statements in the fourth paragraph of the press release, Dr. Kulkarni’s statements in the fifth paragraph of the press release, Dr. Olson’s statements in the sixth paragraph of the press release, Ms. Miller’s statements in the seventh paragraph of the press release and statements regarding the timing of the potential closing of the transaction, future activities of the parties pursuant to the collaboration and the potential benefits of the acquisition. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent Vertex'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, both transactions are subject to certain conditions, including the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act, Vertex may not realize the potential benefits of the collaboration or acquisition, and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the
CRISPR Forward-Looking Statement
This press release may contain a number of “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics’ expectations about any or all of the following: (i) clinical trials (including, without limitation, the timing of filing of clinical trial applications and INDs, any approvals thereof and the timing of commencement of clinical trials), development timelines and discussions with regulatory authorities related to product candidates under development by
About Exonics Therapeutics
Exonics Therapeutics is developing gene editing therapies to treat patients with Duchenne muscular dystrophy and other severe genetic neuromuscular diseases. In multiple Duchenne preclinical models, Exonics has used SingleCut CRISPR to genetically repair and restore dystrophin, the key protein missing in children with Duchenne. Exonics is initially focused on repairing mutations that cause Duchenne in order to develop a therapy to treat many children with the devastating disease, for which there is no cure. Exonics’ technology is licensed from
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Vertex Investor Contacts:
Michael Partridge, 617-341-6108
Eric Rojas, 617-961-7205
Zach Barber, 617-341-6470
Vertex Media Contact:
CRISPR Investor Contact:
CRISPR Media Contact:
WCG on behalf of CRISPR
Exonics Media Contact: