Vertex aligns with another CRISPR gene-editing startup


Dive brief:

  • Mammoth Biosciences, a gene-editing startup co-founded by Nobel Laureate Jennifer Doudna, has entered into an agreement to develop two CRISPR-based drugs with Vertex Pharmaceuticals.
  • Through this alliance, Mammoth and Vertex aim to treat two different and unspecified diseases with drugs that work inside the body to change genes. Vertex will pay Mammoth $ 41 million upfront, a figure that includes a convertible note, and could add an additional $ 650 million in conditional payments if the two programs reach research, development and commercial milestones.
  • The deal marks another milestone in Mammoth’s transition from a diagnostics-focused startup to a drug developer, a move that recently saw the startup win its biggest funding round and now a partnership. It is also the latest evidence of interest in gene editing from Vertex, which has acquired several programs through agreements.

Dive overview:

Mammoth’s evolution into a drug maker seems to be paying off.

Three years ago, the company formed with the project to use CRISPR gene editing to detect and diagnose diseases. This decision set Mammoth apart from Intellia Therapeutics and Caribou Biosciences, two other CRISPR startups from scientists who have worked with Doudna.

Mammoth has already succeeded on this front, developing a COVID-19 test that the Food and Drug Administration cleared for emergency use last year. But the startup has also started to generate interest in its drug design work.

By using a license for a series of proteins associated with CRISPR that are particularly small, the company believes it can develop drugs that go beyond the first generation of gene editing treatments developed by Intellia and others. Mammoth believes these tiny proteins – cutting enzymes believed to be more stable and precise than those used elsewhere – can expand CRISPR’s reach, allowing it to reach more difficult disease targets. These treatments would do their editing job inside the body, much like Intellia’s investigational rare disease drug that recently showed promise in early clinical trials.

Mammoth’s expansion sparked interest from a group of investors who invested $ 150 million in the company in September. Now Vertex is committed as both an investor and a partner.

Vertex has clearly shown an interest in gene editing over the past few years. A long-standing partnership with CRISPR Therapeutics has already resulted in a pioneering treatment for two chronic blood diseases which is currently being tested. Vertex also acquired biotech start-up Exonics Therapeutics to get its hands on a CRISPR program for Duchenne muscular dystrophy. The company has also expressed interest in using gene editing to treat cystic fibrosis, the target of its flagship drug franchise.

Each of these initiatives aims to give Vertex access to more ways to develop genetic drugs and cell therapies for serious diseases, a key part of the biotechnology research and development plan.

Aligning with Mammoth advances those ambitions, although it’s not yet clear how Vertex will use the startup’s technology. Scientific director David Altshuler, in a statement, only said that the alliance would give Vertex “another set of tools to fight many of the diseases that interest us.”

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