Vertex, CRISPR to Use Gene Editing in Search for New Cystic Fibrosis Treatments

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by Charles Moore |

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Vertex Pharmaceuticals, Inc. and CRISPR Therapeutics have announced that they will collaborate on drug research and development using CRISPR’s proprietary gene editing technology CRISPR-Cas9 to discover novel, potential treatments that address the underlying genetic causes of diseases such as cystic fibrosis (CF).

The collaboration between Vertex and CRISPR will evaluate the use of CRISPR-Cas9 in developing therapies for several diseases, like CF, in which treatment targets have already been validated through human genetics. Boston, Massachusetts-based Vertex and CRISPR, which is headquartered in Basel, Switzerland, with R&D operations in Cambridge, Massachusetts, and corporate offices in London, United Kingdom, will focus their initial gene editing research on discovering treatments to address mutations and genes known to cause and contribute to development of cystic fibrosis and sickle cell disease.

Under terms of the collaboration, the CRISPR-Cas9 technology will be used by both companies to facilitate discovery and development of potential, new treatments that correct defects in specific gene targets known to cause or contribute to particular diseases, with an initial focus on addressing 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, with discovery efforts focused on a specified number of other genetic targets to also be conducted under the collaboration. Discovery activities will be conducted primarily by CRISPR, with related expenses being fully funded by Vertex.

Vertex, which will have exclusive rights to license up to six new CRISPR-Cas9-based treatments that emerge from the four-year research collaboration, has made a $105 million up-front commitment of to CRISPR, including $75 million in cash and a $30 million equity investment. CRISPR is also eligible to receive future development, regulatory and sales milestones, and royalty payments on future sales. 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 $420 million as well as royalty payments on future sales.

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The companies will also evaluate a specified range of other genetic targets as part of the collaboration.

AltschulerD“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,” says David Altshuler, M.D., Ph.D., Vertex’s Executive Vice President, Global Research and Chief Scientific Officer in a release. “As a company founded on innovative science, we’re excited to begin this collaboration with CRISPR, as it puts us at the forefront of what we believe may be a fundamental change in the future treatment of disease — using gene editing technologies to address the underlying genetic causes of many diseases.”

NovakR“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,” notes Rodger Novak, M.D, Chief Executive Officer of CRISPR Therapeutics. “For CRISPR, this collaboration validates the potential for gene editing in human therapeutics and provides important financial support for continued investment in our platform and play proprietary pipeline of programs.”

CRISPR Therapeutics’ seasoned management team is focused on translating CRISPR-Cas9 technology into transformative gene-based medicines for patients with serious diseases, working in parallel with their world-class group of scientific founders and advisers, who have extensive experience in gene editing, stem cell biology, advanced drug delivery technologies, RNAi and gene silencing.

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, the companies 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, in its $30 million equity investment, will gain an ownership stake in CRISPR, a private company. The collaboration also provides Vertex with an observer seat on the CRISPR Board of Directors, which will be filled by Dr. Altshuler.

Gene Editing with CRISPR-Cas9

CRISPR’s corporate name is derived from an acronym referring to “Clustered Regularly Interspaced Short Palindromic Repeats” that occur in the genome of certain bacteria, from which CRISPR-Cas9 is based. CRISPR Therapeutics describes Cas9 as a CRISPR-associated endonuclease (an enzyme) known to act as “molecular scissors” that can be used to 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 cells own machinery and other elements to specifically repair the DNA. CRISPR says 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.

CRISPR Therapeutics frames its corporate mission as development of transformative gene-based medicines for patients with serious diseases. The company’s therapeutic approach aims to cure diseases at the molecular level using the CRISPR-Cas9 technology, for which it has licensed the foundational patent estate for human CharpentierEtherapeutic use from Emmanuelle Charpentier, PhD, one of CRISPR Therapeutics scientific founders. Dr. Charpentier, who co-invented the CRISPR-Cas9 technology, has been the recipient of multiple prestigious awards in recognition of the potential contribution that the technology may have on global health. She is a Scientific Member of the Max Planck Society in Germany and Director at the Max Planck Institute for Infection Biology in Berlin, Germany. Prior to joining Max Planck, she was awarded an Alexander von Humboldt professorship at the Helmholtz Centre for Infection Research, Braunschweig, and Professor at the Medical School of Hannover in Germany. Dr. Charpentier continues to oversee a research group at the Laboratory for Molecular Infection Medicine Sweden (MIMS, part of Nordic European Molecular Biology Laboratory [EMBL] Partnership for Molecular Medicine) at Ume University in Sweden where she has a visiting professorship. Her seminal research unveiled the key mechanisms of the CRISPR-Cas9 technology, laying the foundation for the use of CRISPR-Cas9 as a highly versatile and precise gene editing tool.

The other scientific co-founders of CRISPR include Craig Mello, PhD, Chad Cowan, PhD, Matthew Porteus, MD, PhD, and Daniel Anderson, PhD.

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Founded in 1989 in Cambridge, Massachusetts, Vertex has grown to become a global biotechnology company dedicated to the discovery, development and commercialization of innovative medicines to help people with serious diseases lead better lives. In addition to the company’s clinical development programs focused on cystic fibrosis, Vertex has more than a dozen ongoing research programs aimed at other serious and life-threatening diseases.

Vertex has research and development sites and commercial offices in the United States, Europe, Canada and Australia, and, for five years in a row, Science magazine has named Vertex one off its Top Employers in the life sciences. For additional information and the latest updates from the company, visit

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