What Drives Newly Minted CRISPR Unicorn Mammoth Biosciences

The COVID-19 pandemic has shown the world the importance of biotechnology diagnostic and therapeutic tools. From detecting the virus to creating a vaccine, companies around the world are relying on the latest biotech to keep our global population healthy. CRISPR, a technology used to edit genes, has played an important role in global research during the pandemic.

Mammoth Biosciences, a biotech company, announced it has raised $195 million to build next-generation CRISPR products in therapeutics and diagnostics that can cure and detect diseases. Mammoth has now reached unicorn status in the industry with a valuation of more than $1 billion.

Becoming a Synthetic Biology Unicorn

“When Mammoth was founded, we focused on finding and developing proteins to enable new products for CRISPR-based diagnostics. But we always knew there were more product areas and different fields to explore. Moving into therapeutics is very exciting for us,” says Trevor Martin, co-founder and CEO of Mammoth.

Since the beginning, Mammoth has been interested in infectious disease testing for illnesses such as the flu. The company plans to use the new funding to broaden its toolkit of next-generation CRISPR systems and focus on finding permanent genetic cures through gene-editing therapeutics and on-demand diagnostics.

“We want to democratize access to high-quality molecular information. During the pandemic, we saw how difficult that can be, and that there really are barriers to getting this information,” says Martin. “We see CRISPR as a way to get high-quality molecular results in an accessible format.”

Finding New Proteins

Mammoth is considered a pioneer in the field of ultra-small CRISPR systems. Now, the  company is putting greater emphasis on using its technology for in vivo (in a living organism) therapies. According to Martin, the company’s smaller-sized proteins are the key to making this happen. For example, Mammoth’s Cas14 and Casɸ are much smaller proteins in the CRISPR toolbox than the original CRISPR protein, Cas9. 

“Legacy CRISPR systems like Cas9 are large and can have 1,300 amino acids. Cas14 and Casɸ are much smaller and about a third of the size or less of these legacy systems. This has huge implications for the ability to deliver them anywhere in the human body,” says Martin.

Some therapeutics have strict size limits that make using smaller proteins a necessity. Martin compares this to having a truck that can only fit a certain number of boxes in the back. Legacy systems like Cas9 can fill up the whole truck with just the Cas protein, or sometimes, they will not fit at all. However, Cas14 only takes up a small amount of space on the truck. This creates more room for delivering other useful therapies.

Smaller proteins also make it possible to use CRISPR-plus, which allows for the simultaneous targeting of multiple genes. It is possible to combine other kinds of functionality with the CRISPR protein itself. For example, this includes CRISPR-activating domains to turn on a gene instead of editing a gene or CRISPR-inhibiting domains to stop gene expression.

Looking Ahead

Mammoth’s current team includes more than 100 people with diverse talents and skills. The team has experts in engineering, systems integration, CRISPR, chemistry, therapeutics, and diagnostics.

“We are building a community of people who are uniquely at both the tip of innovation and science but also involved in product development,” says Martin.

Mammoth wants to push the limits of what is possible with CRISPR while staying focused on how to help patients as quickly as possible. It has made huge technical strides in terms of validating CRISPR-based diagnostics, but it is only one part of its mission.

“We view this as just the beginning and early days of the impact that Mammoth could have. We will have big celebrations once our first diagnostic and therapeutic tools are changing people’s lives for the better,” says Martin.

Thank you to Lana Bandoim for additional research and reporting in this article. I’m the founder of SynBioBeta, and some of the companies that I write about, including Mammoth Biosciences, are sponsors of the SynBioBeta conference and weekly digest.

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