Vertex and CRISPR Therapeutics to use gene editing technology to develop new treatments for genetic diseases

Vertex will take a US$30m stake in CRISPR and make an upfront payment of $75m

Boston, MA-based cystic fibrosis drugmaker Vertex Pharmaceuticals is committing US$105m in cash to embrace CRISPR Therapeutics' gene editing technology to discover and develop potential new treatments aimed at the genetic causes of human disease.

Vertex will pay $75m to CRISPR in cash in an upfront payment while taking a $30m equity stake in the company in exchange for the right to license up to six gene-editing treatments that could result from a research partnership between the two firms.

CRISPR could also receive future development, regulatory and sales milestones and royalty payments of up to $420m on future sales.

The four-year research collaboration will evaluate the use of CRISPR-Cas9 technology across multiple diseases where targets have been validated through human genetics.

For CRISPR, this collaboration validates the potential for gene editing in human therapeutics

Vertex and CRISPR will focus their initial gene editing research on discovering treatments to address the mutations and genes known to cause and contribute to cystic fibrosis and sickle cell disease. They will also evaluate a specified number of other genetic targets as part of the collaboration.

'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.

Rodger Novak, Chief Executive of CRISPR Therapeutics, added: '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.

'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 programmes.'

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