NEW YORK (GenomeWeb) – CRISPR Therapeutics and Vertex Pharmaceuticals have linked up for a four-year strategic research collaboration to develop new treatments of genetic diseases using CRISPR/Cas9 genome editing.
The partners will evaluate the use of CRISPR/Cas9 in multiple diseases where gene targets have already been established by genetics research, including cystic fibrosis and sickle cell disease. Under the terms of the agreement, the partners will also evaluate an enumerated but undisclosed number of targets. Vertex will provide CRISPR Therapeutics with a $75 million up-front cash payment as well as a $30 million equity investment. CRISPR Therapeutics is also eligible to receive development, regulatory, and sales milestone payments of up to $420 million as well as royalty payments on future sales.
CRISPR Therapeutics will conduct the research, with Vertex covering all related expenses. Vertex holds the option to exclusively license up to six of any gene-based therapies that emerge from the collaboration.
The firms said in a statement that the initial focus of the collaboration will be to try to correct mutations in the cystic fibrosis transmembrane conductance regulator gene.
The collaboration will also focus on treatments for hemoglobinopathies, such as sickle cell disease. For treatments of these diseases, the partners will share all R&D costs equally and CRISPR Therapeutics will lead commercialization activities in the US.
The collaboration also provides Vertex with an observer seat on the CRISPR Therapeutics board of directors.
Basel, Switzerland-based CRISPR Therapeutics was co-founded by CRISPR/Cas9 pioneer Emmanuelle Charpentier and Nobel laureate Craig Mello, among others. The firm has R&D operations based in Cambridge, Massachusetts and corporate offices in London.
In May 2014, CRISPR Therapeutics raised $25 million in Series A funding.