Vertex is paying $105m upfront to tap into the gene-editing expertise of CRISPR Therapeutics for the development of up to six product candidates.
The wide-ranging deal – in which Vertex is taking a $30m equity stake in CRISPR and will pay up to $420m in milestones for each product – is aimed at finding new therapies for genetic diseases including cystic fibrosis.
CRISPR Therapeutics was formed by a team led by Emmanuelle Charpentier, who was awarded the Janssen Award for Biomedical Research last year – along with Jennifer Doudna of Editas Medicine – for the discovery of the CRISPR-Cas9 method of gene editing which allows precise alterations to DNA sequences of living cells on demand.
In essence, the technology introduces a break in a specific place within DNA and triggers a self-repair mechanism. However, instead of restoring the original sequence, CRISPR serves as a new template that can be used to modify the sequence.
Vertex’ chief scientific officer David Altshuler said that 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.”
The first project in the four-year agreement will look at the possibility of using CRISPR-Cas9 to correct the mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that underpins the disease, as well as other contributing genes.
A second project will take a similar approach in haemoglobinopathies, including and sickle cell disease.
Vertex investment in CRISPR is indicative of the potential of gene-editing, and it follows a number of other deals made by big pharma companies betting that the technique could open up a powerful new therapeutic category.
Celgene has also provided funding to CRISPR Thrapeutics, and earlier this year Novartis entered the fray after signing deals with Caribou Biosciences and Intellia Therapeutics that are focused on the development of CRISPR-based therapeutics, delivered either directly in vivo or ex vivo using modified cells.
Meanwhile, AstraZeneca (AZ) has also played its hand claiming to be leading the field after announcing a CRISPR-focused research collaboration with the Wellcome Trust Sanger Institute, the Innovative Genomics Initiative, ThermoFisher Scientific and the Broad Institute.
Editas – backed by Google and Bill Gates – has yet to announce any collaborations and seems to be intent on developing therapeutics in-house for now.