Mammoth Biosciences Achieves Unicorn Status with CRISPR Platform

Mammoth Biosciences Achieves Unicorn Status with New Funding

Mammoth Biosciences, a biotechnology firm specializing in CRISPR technology, has officially reached a valuation of over $1 billion, the company has announced.

This significant milestone is the result of a $150 million Series D funding round, spearheaded by Redmile Group. Additional participation came from Foresite Capital, Senator Investment Group, Sixth Street, Greenspring Associates, Mayfield, Decheng Capital, Plum Alley, and NFX.

The Power of CRISPR Technology

Mammoth Biosciences has rapidly become a key innovator within the CRISPR field since its establishment in 2017. CRISPR, fundamentally, functions as a precise gene-editing tool, capable of cutting and replacing genes within cells and organisms.

This capability holds immense promise for potentially curing genetic diseases and advancing DNA-based diagnostic methods.

The company’s total funding now amounts to $195 million, including a $45 million Series C round from late 2020, which also saw investment from Amazon.

Founding Roots and Scientific Breakthroughs

Among the four founders of Mammoth Biosciences is Jennifer Doudna, a pioneering researcher in CRISPR technology. She was a co-recipient of the 2020 Nobel Prize in Chemistry, alongside Emmanuelle Charpentier, for their groundbreaking 2012 work demonstrating CRISPR’s potential for DNA editing.

The other co-founders are Janice Chen (CTO), Lucas Harrington (CSO), and Trevor Martin (CEO).

While several companies are developing CRISPR-based technologies, Mammoth’s unique strategy is positioning it for success in a competitive market.

A "CRISPR Platform" for Future Innovation

Ursheet Parikh, co-leader of Mayfield’s engineering biology investment practice, describes the valuation as a significant achievement. “The company has a considerable journey ahead, but this funding and valuation demonstrate the potential of their work,” he stated.

Parikh views Mammoth as a comprehensive CRISPR “platform,” actively discovering novel CRISPR systems to address specific biological challenges.

He explains, “Instead of building a CRISPR solution from scratch, researchers can collaborate with Mammoth to identify the optimal proteins for their particular needs.”

Beyond Cas9: Expanding the CRISPR Toolbox

The CRISPR system most commonly recognized is CRISPR/Cas-9, where the Cas-9 protein acts as the molecular scissors for gene editing. However, a diverse range of Cas proteins exist, each with the ability to cut DNA.

Mammoth’s leadership contends that these alternative Cas proteins can offer superior performance depending on the specific application.

The company is actively building a “CRISPR toolbox,” a collection of diverse Cas proteins, each suited for unique applications – analogous to having different types of scissors for different tasks.

Licensing and New Discoveries

In August 2020, Mammoth Biosciences secured a license for the Casɸ family of proteins from UC Berkeley. These proteins, discovered by a UC Berkeley graduate student team, were detailed in publications in Nature and Science in February and July 2020, respectively.

The Casɸ family represents an ultra-small version of Cas9, potentially simplifying the development of therapies for use within the human body and enhancing the precision of gene editing.

Mammoth has also characterized the Cas14 system, another family of ultra-small proteins that target different sequences within the genome than Cas9 proteins.

Intellectual Property and Competitive Advantage

Trevor Martin, CEO of Mammoth Biosciences, emphasizes the company’s founding principle: “Mammoth was established on the belief that a vast universe of CRISPR systems extends beyond legacy systems like Cas9.”

Developing this CRISPR toolkit is not only scientifically compelling but also strategically advantageous for Mammoth, particularly regarding intellectual property.

The original CRISPR/Cas9 system is currently involved in a patent dispute between the University of California Berkeley and MIT’s Broad Institute. Mammoth’s newer Cas proteins circumvent this issue.

Martin clarifies, “The patent disputes concern legacy CRISPR-Cas9 systems. Mammoth’s systems are not Cas9-based and are therefore not subject to these disputes.”

Focus on In-Vivo Applications and Diagnostics

Mammoth is building a portfolio of proprietary tools with potential applications in genetic medicine and DNA-based diagnostics, though many therapeutic products are still under development.

2020 saw a surge in new clinical trials for CRISPR therapeutics, indicating progress toward regulatory approval, though widespread availability remains some time away.

Companies like CRISPR Therapeutics and Vertex Pharmaceuticals have reported encouraging results from trials targeting beta thalassemia and sickle cell disease. Intellia Therapeutics and Regeneron have also demonstrated the effectiveness of directly injected CRISPR-based treatments for ATTR amyloidosis.

Martin notes that Mammoth’s focus will be on in-vivo applications, leveraging their CRISPR toolbox to deliver therapies directly within the human body.

Expanding into Diagnostics

“We don’t have a firm timeline for therapeutic products, but we will share more information in the coming years, and we are excited about the technical results so far,” says Martin.

Diagnostics represents an area where Mammoth can achieve near-term success. The company is actively collaborating with partners to develop viable diagnostic products.

In January, Mammoth received funding from the Defense Advanced Research Projects Agency (DARPA) to create a point-of-care test capable of detecting up to 10 pathogens simultaneously, and a larger lab-based test for up to 1,000. They also secured funding from the National Institutes of Health (NIH) and partnered with GSK to develop a rapid COVID-19 test.

Parikh emphasizes that Mammoth’s success lies in its ability to operate effectively in both diagnostics and therapeutics. The unicorn valuation validates this approach.

“This milestone confirms their strategy of focusing on expertise rather than limiting themselves to a single area, like diagnostics or therapeutics,” he concludes.