Vertex expands into new pathological areas and improves gene editing skills through an extended collaboration with CRISPR Therapeutics and acquisition of Exonics Therapeutics

0
810

BOSTON & WATERTOWN, Mass. & ZUG, Switzerland – (BUSINESS WIRE) –

<p class = "canvas-atom canvas-text Mb (1.0em) Mb (0) – sm Mt (0.8em) – sm" type = "text" content = "-Provides Vertex with the main capabilities of gene editing to develop new therapies for Duchenne muscular dystrophy and type 1 myotonic dystrophy"data-reactid =" 12 ">-Provides Vertex with the main capabilities of gene editing to develop new therapies for Duchenne muscular dystrophy and type 1 myotonic dystrophy

<p class = "canvas-atom canvas-text Mb (1.0em) Mb (0) – sm Mt (0.8em) – sm" type = "text" content = "– CRISPR to receive an advance payment of $ 175 million, with the possibility of further milestones and royalty payments"data-reactid =" 13 ">– CRISPR to receive an advance payment of $ 175 million, with the possibility of further milestones and royalty payments

<p class = "canvas-atom canvas-text Mb (1.0em) Mb (0) – sm Mt (0.8em) – sm" type = "text" content = "– Export of data for an advance payment of $ 245 million, with the possibility of further milestone payments –"data-reactid =" 14 ">– Export of data for an advance payment of $ 245 million, with the possibility of further milestone payments –

<p class = "canvas-atom canvas-text Mb (1.0em) Mb (0) – sm Mt (0.8em) – sm" type = "text" content = "-John T. Gray, Ph.D. appointed senior vice president of Vertex, genetic therapies-"data-reactid =" 15 ">-John T. Gray, Ph.D. appointed senior vice president of Vertex, genetic therapies-

Vertex Pharmaceuticals Incorporated (VRTX) announced today that the company is improving its gene editing capabilities to develop new therapies for Duchenne muscular dystrophy (DMD) and type 1 myotonic dystrophy (DM1) by expanding its collaboration with CRISPR Therapeutics and acquiring Exonics Therapeutics.

Vertex and CRISPR Therapeutics (CRSP) have expanded their collaboration and entered into an exclusive licensing agreement to discover and develop gene modification therapies for the treatment of DMD and DM1.

Vertex and Exonics Therapeutics have entered into a definitive agreement under which Vertex will acquire the private companies Exonics, a company focused on creating genetic modification therapies to repair the mutations that cause DMD and other serious neuromuscular diseases.

"Through the extensive collaboration with CRISPR and the acquisition of Exonics, we are bringing together the intellectual property, technologies and scientific expertise necessary 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 drugs for people with serious illnesses, "said Jeffrey Leiden, MD, Ph.D., President, President and CEO of Vertex. "We are continuing to build a toolbox with technologies and capabilities of small molecules and nucleic acids that will enable us to lead the scientific innovation to produce transformation drugs 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 genetic editing," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "We continue to make significant progress in enabling in vivo approaches to gene editing and we are excited about the possibility of developing potentially curative therapies for DMD and DM1 together with Vertex."

"DMD and DM1 are devastating muscle diseases without available curative therapies," said Eric Olson, Ph.D., founder and Chief Science Advisor of Exonics, and professor and chair of the Department of Molecular Biology at UT Southwestern Medical Center . "Vertex has a proven track record in developing important therapies for serious illnesses and we are excited to combine our efforts to potentially develop a once safe and effective treatment for serious neuromuscular diseases."

"The Duchenne community needs new approaches to treat and cure this devastating disease and the technology of Exonics has the potential to drastically improve the lives of Duchenne patients," said Debra Miller, CEO and founder of Cure Duchenne who provided the first initial funding for Exonics. "We are pleased that the talented Vertex and Exonics scientists will work together to promote these promising genetic modification treatments for Duchenne and other neuromuscular diseases."

<p class = "canvas-atom canvas-text Mb (1.0em) Mb (0) – sm Mt (0.8em) – sm" type = "text" content = "Vertex collaboration information with CRISPR Therapeutics
Under the terms of this strategic partnership and the licensing agreement, Vertex will pay $ 175 million upfront for exclusive worldwide rights relating to existing and future intellectual property of CRISPR Therapeutics including CRISPR / Cas9 core technology, new endonucleases, Single and double guide RNA and AAV vectors for DMD and DM1 gene modification products. "data-reactid =" 23 ">Vertex collaboration information with CRISPR Therapeutics
Under the terms of this strategic partnership and the licensing agreement, Vertex will pay $ 175 million upfront for exclusive worldwide rights relating to existing and future intellectual property of CRISPR Therapeutics including CRISPR / Cas9 core technology, new endonucleases, Single and double guide RNA and AAV vectors for DMD and DM1 gene modification products.

For the DMD program, Vertex is responsible for all research, development, production and marketing activities and all related costs. For the DM1 program, Vertex and CRISPR will share the research costs for the specific RNA research guide conducted by CRISPR and Vertex is responsible for all other research, development, production and marketing costs.

CRISPR Therapeutics is eligible to receive payments up to $ 1 billion including upfront and potential future payments based on the success of specific research, development, regulation and commercial objectives for the DMD and DM1 programs. Furthermore, Vertex will pay multi-level royalties on future net sales on any product that may result from this collaboration. At the IND warehouse, CRISPR has the opportunity to forgo the milestones and the DM1 fees to co-develop and co-market all DM1 products globally.

The closing of this transaction will be subject to certain conditions, including the expiration of the waiting period pursuant to the Hart-Scott-Rodino Antitrust Improvements law. The companies expect the transaction to be closed in the third quarter of 2019.

<p class = "canvas-atom canvas-text Mb (1.0em) Mb (0) – sm Mt (0.8em) – sm" type = "text" content = "Information on Acquisition of Vertex Therapeutics
Exonics Therapeutics is developing gene modification therapies to treat patients with DMD and other serious neuromuscular genetic diseases and provides Vertex with intellectual property, technology and scientific expertise in genetic modification therapies for these serious diseases. In many preclinical DMD small and large animal models, Exonics used SingleCut CRISPR to repair and genetically restore dystrophin, the key protein missing in children with DMD. "Data-reactid =" 31 ">Information on Acquisition of Vertex Therapeutics
Exonics Therapeutics is developing gene modification therapies to treat patients with DMD and other serious neuromuscular genetic diseases and provides Vertex with intellectual property, technology and scientific expertise in genetic modification therapies for these serious diseases. In many preclinical DMD small and large animal models, Exonics used SingleCut CRISPR to repair and genetically restore dystrophin, the key protein missing in children with DMD.

The Exonics technology is licensed from UT Southwestern Medical Center and is based on Dr. Olson's research. Dr. Olson will continue in his role as chief scientific advisor to Exonics and will provide supervision and guidance on research and development of transformative gene modification therapies.

Under the terms of the acquisition, Vertex will acquire all the outstanding shares of Exonics, which will become an exclusive subsidiary of Vertex. Holders of Exonics equity securities can receive payments of approximately $ 1 billion, including $ 245 million upfront and potential future payments based primarily on the success of specific development and regulatory objectives for the DMD and DM1 programs.

The closing of this transaction will be subject to certain conditions, including the expiry of the waiting period pursuant to the Hart-Scott-Rodino Antitrust Improvements Act and other customary conditions. The companies expect the acquisition to close in the third quarter of 2019.

<p class = "canvas-atom canvas-text Mb (1.0em) Mb (0) – sm Mt (0.8em) – sm" type = "text" content = "Information on the expansion of Vertex's scientific leadership
Vertex announced today the appointment of Dr. John T. Gray as Senior Vice President, Genetic Therapies starting June 17, 2019. "data-reactid =" 35 ">Information on the expansion of Vertex's scientific leadership
Vertex announced today the appointment of Dr. John T. Gray as Senior Vice President, Genetic Therapies, in force since June 17, 2019.

With more than twenty-five years in academia and industry, John is a space-proven leader in genetic therapies with experience in both scientific development and product development. His skills and competences are the ideal solution with Vertex's strategy to bring together the multiple components of genetic therapies and advance the science of genetic therapies, so that the Vertex genetic therapy platform offers the best of all this emerging approach.

John was previously Chief Scientific Officer and Senior Vice President of Audentes Therapeutics, where he and his team focused on rAAV gene therapy for neuromuscular and liver disorders. John has a B.A. in Biochemistry at the University of California, Berkeley and a Ph.D. in Biochemistry at the University of Colorado, Boulder and was a postdoctoral scientist at the Howard Hughes Medical Institute and Pfizer Central Research.

<p class = "canvas-atom canvas-text Mb (1.0em) Mb (0) – sm Mt (0.8em) – sm" type = "text" content = "Information on the new research site on Vertex genetic therapies
Vertex will create a new research site for genetic therapies in the Boston area where research programs will be conducted, as well as vector development and clinical production for genetic therapies, including DMD and DM1 programs. "Data-reactid =" 38 ">Information on the new research site on Vertex genetic therapies
Vertex will create a new research site on genetic therapies in the Boston area where research programs will be conducted, as well as vector development and clinical production for genetic therapies, including DMD and DM1 programs.

<p class = "canvas-atom canvas-text Mb (1.0em) Mb (0) – sm Mt (0.8em) – sm" type = "text" content = "Information on Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative drugs for people with serious and life-threatening illnesses. 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. "Data-reactid =" 39 ">Information on Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative drugs for people with serious and life-threatening illnesses. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focusing on the underlying mechanisms of other serious diseases.

<p class = "canvas-atom canvas-text Mb (1.0em) Mb (0) – sm Mt (0.8em) – sm" type = "text" content = "Founded in 1989 in Cambridge, Mass., Vertex & # 39 ; s Headquarters is now located in the Innovation District of Boston. Today, the company has research and development sites and sales offices in the United States, Europe, Canada, Australia and Latin America. of the best jobs in the sector, including being appointed to Science Top Employers of the magazine in the life sciences classifies for nine consecutive years. "data-reactid =" 40 "> Founded in 1989 in Cambridge, Massachusetts, Vertex's headquarters are now located in the Boston innovation district, development sites and sales offices in the United States, Europe, Canada, Australia and Latin America: Vertex is constantly recognized as one of the best jobs in the industry, including the name of Science Top Employers of the magazine in the life sciences ranking for nine consecutive years.

For more information and the latest updates from the company, visit www.vrtx.com.

<p class = "canvas-atom canvas-text Mb (1.0em) Mb (0) – sm Mt (0.8em) – sm" type = "text" content = "Special note from the summit 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, The statements by Dr. Olson in the sixth paragraph of the press release, Mrs. Miller's statements in the seventh paragraph of the press release and the statements regarding the timing of the potential closing of the transaction, the parties' future activities based on collaboration and potential benefits of the acquisition. While Vertex believes that 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. These risks and uncertainties include, among other things, that both transactions are subject to certain conditions, including the expiration of the waiting period under the Antitrust Improvement Act Hart-Scott-Rodino, Vertex may not realize of the potential benefits of collaboration or acquisition, and the other risks listed under Risk Factors in the Vertex annual report and in the quarterly reports filed with the Securities and Exchange Commission and available through the company's website at address www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. "Data-reactid =" 42 ">Special note from the summit 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, The statements by Dr. Olson in the sixth paragraph of the press release, Mrs. Miller's statements in the seventh paragraph of the press release and the statements regarding the timing of the potential closing of the transaction, the parties' future activities based on collaboration and potential benefits of the acquisition. While Vertex believes that 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. These risks and uncertainties include, among other things, that both transactions are subject to certain conditions, including the expiration of the waiting period under the Antitrust Improvement Act Hart-Scott-Rodino, Vertex may not realize of the potential benefits of collaboration or acquisition, and the other risks listed under Risk Factors in the Vertex annual report and in the quarterly reports filed with the Securities and Exchange Commission and available through the company's website at address www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as soon as new information becomes available.

<p class = "canvas-atom canvas-text Mb (1.0em) Mb (0) – sm Mt (0.8em) – sm" type = "text" content = "About CRISPR Therapeutics
CRISPR Therapeutics is one of the leading gene editing companies focused on the development of transformative drugs based on genes for serious diseases using its proprietary CRISPR / Cas9 platform. CRISPR / Cas9 is a revolutionary gene modification technology that allows precise and direct modifications to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs in a wide range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer AG, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is based in Zug, Switzerland, with its wholly owned US subsidiary, CRISPR Therapeutics, Inc and research and development operations based in Cambridge, Massachusetts, and sales offices in London, UK. For more information, visit www.crisprtx.com. "Data-reactid =" 43 ">About CRISPR Therapeutics
CRISPR Therapeutics is one of the leading gene editing companies focused on the development of transformative drugs based on genes for serious diseases using its proprietary CRISPR / Cas9 platform. CRISPR / Cas9 is a revolutionary gene modification technology that allows precise and direct modifications to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs in a wide range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer AG, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is based in Zug, Switzerland, with its wholly owned US subsidiary, CRISPR Therapeutics, Inc and research and development operations based in Cambridge, Massachusetts, and sales offices in London, UK. For more information, visit www.crisprtx.com.

<p class = "canvas-atom canvas-text Mb (1.0em) Mb (0) – sm Mt (0.8em) – sm" type = "text" content = "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 on one or all of the following: (i) clinical trials ( including, by way of example, the timing of the presentation of clinical trial applications and the IND, any approvals and timing of the start of clinical trials), development deadlines and discussions with the regulatory authorities relating to candidates for products under development by part of CRISPR Therapeutics and its collaborators; (ii) the number of patients to be evaluated, the expected date by which the enrollment will be completed and the data that will be generated by ongoing and planned clinical trials and the ability to use this data for design and implementation. ; initiation of further clinical trials trials; (iii) the scope and timing of ongoing and potential future clinical trials; (iv) the coverage of intellectual property and the positions of CRISPR Therapeutics, its licensors and third parties; (v) the sufficiency of CRISPR Therapeutics cash resources; and (vi) the therapeutic value, development and commercial potential of CRISPR / Cas9 gene modification technologies and therapies. Without limiting the foregoing, the words "believes", "anticipates", "plans", "expects" and similar expressions are intended to identify forward-looking statements. Please note that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the limits of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those expected or suggested in forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the results for the trials and the planned clinical trials of each CRISPR Therapeutics may not be favorable; that one or more of CRISPR Therapeutics' internal or external product application programs do not proceed as intended for technical, scientific or commercial reasons; that competitive future or other market factors could negatively affect the commercial potential of the CRISPR Therapeutics product candidates; inherent uncertainties in the initiation and completion of preclinical studies for candidates for CRISPR Therapeutics products; availability and timing of the results of preclinical studies; whether the results of a preclinical trial will be predictive of future results of future trials; uncertainties regarding regulatory approvals to conduct tests or market products; uncertainties regarding the protection of intellectual property for the technology and intellectual property of CRISPR Therapeutics belonging to third parties; and those risks and uncertainties described under the heading "Risk Factors" in the most recent annual report of CRISPR Therapeutics on Form 10-K and in any other subsequent filing done by CRISPR Therapeutics with the US Securities and Exchange Commission, which are available on the SEC website at www.sec.gov. Existing and potential investors are warned not to place undue reliance on such forward-looking statements, which speak only of the date on which they were made. CRISPR Therapeutics disclaims any obligation or commitment to update or revise the forward-looking statements contained in this press release, except to the extent required by law. "Data-reactid =" 44 ">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 on one or all of the following: (i) clinical trials ( including, by way of example, the timing of the presentation of clinical trial applications and the IND, any approvals and timing of the start of clinical trials), development deadlines and discussions with the regulatory authorities relating to candidates for products under development by part of CRISPR Therapeutics and its collaborators; (ii) the number of patients to be evaluated, the expected date by which the enrollment will be completed and the data that will be generated by ongoing and planned clinical trials and the ability to use this data for design and implementation. ; initiation of further clinical trials trials; (iii) the scope and timing of ongoing and potential future clinical trials; (iv) the coverage of intellectual property and the positions of CRISPR Therapeutics, its licensors and third parties; (v) the sufficiency of CRISPR Therapeutics cash resources; and (vi) the therapeutic value, development and commercial potential of CRISPR / Cas9 gene modification technologies and therapies. Without limiting the foregoing, the words "believes", "anticipates", "plans", "expects" and similar expressions are intended to identify forward-looking statements. Please note that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the limits of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those expected or suggested in forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the results for the trials and the planned clinical trials of each CRISPR Therapeutics may not be favorable; that one or more of CRISPR Therapeutics' internal or external product application programs do not proceed as intended for technical, scientific or commercial reasons; that competitive future or other market factors could negatively affect the commercial potential of the CRISPR Therapeutics product candidates; inherent uncertainties in the initiation and completion of preclinical studies for candidates for CRISPR Therapeutics products; availability and timing of the results of preclinical studies; whether the results of a preclinical trial will be predictive of future results of future trials; uncertainties regarding regulatory approvals to conduct tests or market products; uncertainties regarding the protection of intellectual property for the technology and intellectual property of CRISPR Therapeutics belonging to third parties; and those risks and uncertainties described under the heading "Risk Factors" in the most recent annual report of CRISPR Therapeutics on Form 10-K and in any other subsequent filing done by CRISPR Therapeutics with the US Securities and Exchange Commission, which are available on the SEC website at www.sec.gov. Existing and potential investors are warned not to place undue reliance on such forward-looking statements, which speak only of the date on which they were made. CRISPR Therapeutics disclaims any obligation or commitment to update or revise the forward-looking statements contained in this press release, except to the extent required by law.

<p class = "canvas-atom canvas-text Mb (1.0em) Mb (0) – sm Mt (0.8em) – sm" type = "text" content = "Information on Exonics Therapeutics
Exonics Therapeutics is developing gene modification therapies for the treatment of patients with Duchenne muscular dystrophy and other serious genetic neuromuscular diseases. In several preclinical Duchenne models, Exonics used SingleCut CRISPR to repair and genetically restore dystrophin, the key protein missing in children with Duchenne. Exonics 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. The Exonics technology is licensed from UT Southwestern Medical Center and is based on research by Eric Olson, Ph.D., founder and scientific advisor of Exonics. Exonics is located in Watertown, Massachusetts. For more information, visit www.exonicstx.com. "Data-reactid =" 45 ">Information on Exonics Therapeutics
Exonics Therapeutics is developing gene modification therapies for the treatment of patients with Duchenne muscular dystrophy and other serious genetic neuromuscular diseases. In several preclinical Duchenne models, Exonics used SingleCut CRISPR to repair and genetically restore dystrophin, the key protein missing in children with Duchenne. Exonics 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. The Exonics technology is licensed from UT Southwestern Medical Center and is based on research by Eric Olson, Ph.D., founder and scientific advisor of Exonics. Exonics is located in Watertown, Massachusetts. For more information, visit www.exonicstx.com.

(VRTX-GEN)

<p class = "canvas-atom canvas-text Mb (1.0em) Mb (0) – sm Mt (0.8em) – sm" type = "text" content = "View the source version on businesswire.com: https://www.businesswire.com/news/home/20190606005853/en/"data-reactid =" 48 ">View the source version on businesswire.com: https://www.businesswire.com/news/home/20190606005853/en/