- Gene-editing company Editas will begin research aimed at developing CRISPR/Cas9-based medicines for cystic fibrosis, signing a three-year, $5 million research agreement with the Cystic Fibrosis Foundation Therapeutics (CFFT) on Monday.
- Editas said it would target both common cystic fibrosis genomic mutations as well as mutations typically not addressed, aiming to use its CRISPR tech to correct some of the more than 1,800 known mutations within the gene related to cystic fibrosis.
- CFFT has set up similar research deals before, funding Vertex's earlier cystic fibrosis work . Vertex went on to develop Kalydeco and Orkambi, which have become major treatments for the genetic disorder.
CFFT invested more than $120 million in Vertex since 2001, according to the Boston Business Journal. But Vertex's drugs, while effective, don't fix the underlying genetic mutations responsible for cystic fibrosis. Editas' CRISPR tech, while still experimental, could potentially make some strides toward treating those genetic errors.
Editas does not yet have an approved drug on the market, but the wide-ranging possible applications of gene-editing has spurred several revenue-generating partnerships, such as the deal it struck with Juno in 2015. Editas also continues to develop its own pipeline of checkpoint inhibitors and monoclonal antibodies.
"We believe that the CRISPR approach to gene editing holds significant promise for repairing the underlying cause of cystic fibrosis," said Preston W. Campbell III, CEO at the CF Foundation. "We’re pleased to work with Editas Medicine and are excited by the possibilities of what can be accomplished on behalf of people with CF.”