- Mammoth Biosciences, a gene editing startup co-founded by Nobel Laureate Jennifer Doudna, has struck a deal to develop two CRISPR-based medicines with Vertex Pharmaceuticals.
- Through the alliance, Mammoth and Vertex aim to treat two different, unspecified diseases with drugs that work inside the body to edit genes. Vertex will pay Mammoth $41 million upfront, a figure that includes a convertible note, and could add another $650 million in conditional payments if the two programs achieve research, development and commercial milestones.
- The deal marks another step in Mammoth's shift from a diagnostics-focused startup to a drug developer, a move that recently landed the startup its biggest financing round and now a partnership. It is also the latest evidence of interest in gene editing from Vertex, which has acquired multiple programs through dealmaking.
Mammoth's evolution into a drugmaker appears to be paying off.
Three years ago, the company formed with a plan to use CRISPR gene editing to detect and diagnose diseases. That decision made Mammoth stand apart from Intellia Therapeutics and Caribou Biosciences, two other CRISPR startups that emerged from scientists who worked with Doudna.
Mammoth has already succeeded on that front, developing a COVID-19 test that the Food and Drug Administration cleared for emergency use last year. But the startup has begun to garner interest for its work designing drugs as well.
Using a license to a series of CRISPR-associated proteins that are particularly small, the company believes it can develop medicines that go beyond the first generation of gene editing treatments being developed by Intellia and others. Mammoth believes those tiny proteins — cutting enzymes meant to be more stable and precise than those used elsewhere — can broaden the reach of CRISPR, enabling it to get to tougher disease targets. Those treatments would do their editing work inside the body, much like Intellia's experimental rare disease medicine that recently showed promise in early clinical testing.
Mammoth's expansion drew the interest of an investor group that poured $150 million into the company in September. Now Vertex has come aboard both as an investor and partner.
Vertex has made clear its interest in gene editing over the past few years. A long-running partnership with CRISPR Therapeutics has already yielded a pioneering treatment for two chronic blood diseases that is currently moving through testing. Vertex also acquired startup biotech Exonics Therapeutics to get ahold of a CRISPR program for Duchenne muscular dystrophy. The company has shown interest in using gene editing to treat cystic fibrosis, the target of its flagship drug franchise, as well.
Each of those moves are meant to give Vertex access to more ways to develop genetic medicines and cell-based therapies for serious diseases, a key part of the biotech's research and development plan.
Aligning itself with Mammoth advances those ambitions, though it's not yet clear how Vertex will use the startup's technology. Chief scientific officer David Altshuler, in a statement, only said that the alliance will give Vertex "another set of tools to tackle many of the diseases we're interested in."