Vertex, CRISPR Therapeutics form gene editing collaboration

Under the four-year research collaboration, the companies will evaluate the use of CRISPR-Cas9 DNA gene editing technology to address mutations and genes that cause or contribute to cystic fibrosis.

Vertex will have exclusive rights to license about six new CRISPR-Cas9-based treatments resulting from the partnership.

The company will pay $75m up front to CRISPR and invest $30m for an undisclosed stake.

CRISPR will also receive future development, regulatory and sales milestone payments of up to $420m for each licensed treatment, as well as royalties on future sales.

CRISPR will primarily carry out discovery activities, while Vertex will fund the related expenses.

The companies will collaborate on the research, development and commercialization of treatments for hemoglobinopathies that emerge from the collaboration.

They will equally share all research and development costs and sales for hemoglobinopathies, including treatments for sickle cell disease.

CRISPR will lead commercialization activities in the US Vertex will be responsible for all development and global commercialization efforts for all other diseases.

Vertex Pharmaceuticals executive vice president for global research and chief scientific officer David Altshuler said: "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."

CRISPR Therapeutics CEO Rodger Novak said: "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 proprietary pipeline of programs."

Image: Cas9 is a CRISPR-associated endonuclease known to act as the molecular scissors that cut and edit, or correct, disease-related DNA in a cell. Photo: courtesy of dream designs/FreeDigitalPhotos.net.