Vertex Coughs Up $105 Million Upfront to CRISPR for Gene Editing Pact

October 26, 2015
By Mark Terry, Breaking News Staff

Boston-based Vertex Pharmaceuticals and Cambridge, Mass.-based CRISPR Therapeutics announced today that the two companies have inked a strategic research collaboration deal.

The collaboration will determine if using CRISPR-Cas9 has practical use across multiple diseases when targets are validated via genetics. The initial gene editing research will focus on cystic fibrosis (FS) and sickle cell disease.

CRISPR, as a technique, stands for clustered, regularly interspaced palindromic repeats. Along with an enzyme called 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. It was discovered by UC Berkeley professor Jennifer Doudna and Emmanuelle Charpentier of the Helmholtz Centre for Infection Research in Braunschweig, Germany, and has the potential to treat many hereditary diseases.

The enzyme can be programmed with RNA to cut DNA at specific, targeted locations, which allows for easier, specific gene editing.

Doudna founded Caribou Biosciences, which developed a collaboration deal with 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.

There has been several lawsuits over the technology. The first paper was published by Doudna and Chapentier. However, Feng Zhang, a researcher at the MIT-Harvard Broad Institute, filed a broad U.S. patent claim on the technology.

As part of the deal between CRISPR Therapeutics and Vertex Pharmaceuticals , Vertex will gain exclusive rights to license up to six new CRISPR-Cas9-based treatments that may be found during their research. Vertex will pay $105 million upfront to CRISPR, and $75 million in cash and $30 million in equity. CRISPR is eligible for milestone and royalty payments on any discoveries.

“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 David Altshuler, Vertex’s executive vice president, Global Research and chief scientific officer, in a statement. “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.”

CRISPR will primarily focus on discovery, with related expenses funded by Vertex. Of the six possible in-licensed treatments, Vertex could pay up to $420 million in milestone and royalty payments.

The two companies will also collaborate on research, developing and commercializing treatments for hemoglobinopathies, including sickle cell disease.

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