Mammoth Biosciences Inks $691 Million Deal To Develop Crispr Gene-Editing Therapies


Mammoth Biosciences, a biotech company using gene-editing Crispr technology for medicines and diagnostics, announced Tuesday it has signed a deal with Vertex Pharmaceuticals, a biotech heavyweight best known for its cystic fibrosis therapies, to develop new treatments for genetic diseases delivered directly into the body, signaling more mainstream medical use of gene therapy after years of setbacks.

Key Facts

Mammoth will receive upfront payments of $41 million for the deal, which will make use of its gene-editing Crispr tools to develop in vivo gene therapies for two genetic diseases.

In vivo gene therapies are designed to be delivered directly into the body and edit patients’ genes to treat disease, contrasting to ex vivo treatments where cells are altered in the lab and returned to the body.

Mammoth is in line to receive up to $650 million if it meets certain research, development and commercial goals across the two projects and Vertex will pay royalties on any products stemming from the partnership. 

While gene therapy—a potentially curative treatment replacing faulty genes with healthy ones—has technically been possible for decades, it has typically not been precise enough to use clinically, something Crispr changes. 

David Altshuler, Vertex’s chief scientific officer, said Mammoth’s ultra-small Crispr systems will give the company “another set of tools to tackle many of the diseases we’re interested in” by directly editing genes inside patients. 

Key Background

Crispr, standing for “clustered regularly interspaced short palindromic repeats,” is a powerful set of gene-editing tools derived from the immune systems of bacteria. The scientists who pioneered the technique, Emmanuelle Charpentier and Jennifer Doudna, shared the 2020 Nobel Prize in Chemistry for their work. Doudna, alongside three Forbes Under 30 alums Trevor Martin Janice Chen, and Lucas Harrington, founded Mammoth Biosciences. Mammoth—a tech unicorn—is building a toolbox of proteins Crispr relies on to edit genomes and it hopes its ultra-small proteins will prove more useful in getting gene therapies into humans directly. The platform is also useful for diagnostics, including coronavirus and defense funding to detect emerging biological threats. 

Crucial Quote

Peter Nell, Mammoth’s chief business officer and head of therapeutic strategy, said the company believes the ultra-small Crispr systems could be “game changers when it comes to systemic and targeted delivery” of in vivo gene-editing therapies. 

What We Don’t Know

The two companies did not state which genetic diseases they aim to target in the partnership. Vertex already has numerous approved medicines to treat cystic fibrosis—game changers in treating the disease and big money makers for the company—and has programs to develop this area further. It also has a “rapidly expanding pipeline” of cell and genetic therapies for diseases including sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Further Reading

Mammoth Biosciences Signs Exclusive Deal For Recently-Discovered Crispr Protein (Forbes)

Mammoth Biosciences Raises $195 Million, Vaulting Its Crispr Tech To Unicorn Status (Forbes)

Innovations In: Gene Therapy (Scientific American)

Boston Biotech Star Vertex Struggles to Reprise Its One Big Hit (Bloomberg)

The Under 30 Alums Racing To Develop At-Home Coronavirus Tests With Crispr (Forbes)

Under 30 Alum Mammoth Biosciences To Develop Diagnostics For Department Of Defense (Forbes)

After the Nobel, what next for Crispr gene-editing therapies? (Observer)

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