UPDATED: Vertex signs another Mammoth-sized CRISPR deal with $650M in biobucks on the line

Mammoth Biosciences' CRISPR work is also being explored in a diagnostic test for SARS-CoV-2, the virus behind COVID-19. (Pixabay/tony241969)

Autumn is in full swing for those in leafy locales and it turns out that Vertex is enjoying the crispy, crunchy season, too: the pharma has signed another CRISPR gene editing pact, this time for $650 million in biobucks with Mammoth Biosciences.

Vertex will dole out $41 million upfront and up to $650 million in biobucks to the prehistorically-named biotech and diagnostics company. The collaboration focuses on Mammoth's CRISPR systems, which are about one-third the size of the popular CRISPR Cas9 enzymes.

Mammoth's other CRISPR advantage? Its Cas14 and Casɸ proteins aim to squash immunogenicity risks since they don't originate from bacteria, said Peter Nell, chief business officer and head of therapeutic strategy at Mammoth, in an interview with Fierce Biotech. 

The pact represents the first therapeutic partnership for Mammoth, Nell said. The startup, cofounded by CRISPR pioneer and Nobel laureate Jennifer Doudna, has collaborators outside of drug development for its CRISPR-based diagnostics work, including a test for SARS-CoV-2. The FDA gave emergency use authorization for the CRISPR-based reagent set for detecting the virus last year.

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The CRISPR gene editing space reached new heights in June when Intellia and partner Regeneron showed for the first time that their in vivo candidate worked better than standard of care therapy in patients with transthyretin amyloidosis. Vertex is attempting to ride those CRISPR waves and has been making several deals.

The company handed down a hefty $900 million upfront payment to CRISPR Therapeutics in April and signed up for a $1.2 billion pact with Arbor Biotechnologies in August. Arbor's CRISPR gene editing technology will be used to develop ex vivo cell therapies, and the CRISPR Therapeutics deal is focused on a gene editing therapy that emerged from their original 2015 research agreement.

Mammoth and Vertex will focus on in vivo gene editing as part of their collaboration with the hopes of producing treatments for two undisclosed genetic diseases. Vertex's CRISPR tie-ups with Arbor and CRISPR Therapeutics revolve around type 1 diabetes and the blood diseases sickle cell and beta thalassemia. Outside of CRISPR, Vertex has multiple drugs approved for cystic fibrosis and has been looking to diversify. 

Nell is familiar with CRISPR Therapeutics. After working at Bayer for 16 years, he led business development for Casebia Therapeutics, which was a joint venture between CRISPR Therapeutics and Bayer. He then joined Mammoth in fall 2019 when CRISPR took control of Casebia and its Cas9-based pipeline. 

The Vertex deal is likely the first in a line of therapeutic partnerships for Mammoth.

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“We have ongoing discussions, so there might be more in the near future. However, we also don’t want to become a licensing company, if you will, so we’re building our own internal pipeline," Nell said. 

That pipeline will grow thanks to a $150 million series D disclosed last month, which will help Mammoth explore ex vivo gene editing, Nell said. The ex vivo work could open the door to other collaborations, he added.

Editor's note: This story has been updated to clarify the approval of Mammoth's CRISPR-based test for detecting SARS-CoV-2.