Breakout “CRISPR platform” company Mammoth Biosciences is officially a

CRISPR-based biotech startup Mammoth Biosciences is officially a unicorn, the company says.

The billion dollar valuation comes on the back of a $150 million Series D round led by Redmine Group, with participation from Foresight Capital, Senator Investment Group, Sixth Street, Greenspring Associates, Mayfield, Decheng Capital, Plum Alley and NFX. Combined with a Series C round of $45 million (which includes participation from Amazon) at the end of 2020, this brings the company’s total financing to $195 million.

Mammoth Biosciences has been a major player in the CRISPR space since its inception in 2017. CRISPR, simply put, is a pair of biological scissors that can cut and alter genes in cells and living organisms, opening up the potential to permanently cure genetic disease. , and perform DNA-based diagnosis.

One of the company’s four founders is one of the original discoverers of CRISPR, Jennifer Doudna, who recently won the 2020 Nobel Prize in Chemistry, as well as her 2012 work on using CRISPR to cut DNA can go. The other co-founders of the company are Janice Chen (CTO), Lucas Harrington (CSO) and Trevor Martin (CEO).

There are a few other CRISPR-based companies out there, including a number that are already publicly traded. This unicorn milestone stands as a sign that Mammoth’s unique approach to CRISPR can help differentiate itself in that landscape.

“It’s a milestone,” Ursett Parikh, co-leader of Mayfield’s engineering biology investing practice, says. “I think the company has a long way to go from here. This round and this valuation indicate the promise of this phase of the future.”

Parikh says he sees Mammoth as a CRISPR “platform.” Mammoth is exploring new types of CRISPR systems that can be used to solve specific biological problems.

“The best analog is, before you had Intel and Microsoft, if someone wanted to build a new application, they had to make a new computer function with an operating system,” Parikh says. “You don’t have to build CRISPR solutions from the ground-up. You can work with mammoths to find the right proteins for specific problems.”

The CRISPR system Most people think of when they hear the phrase CRISPR/Cas-9 is a two-part mechanism. The actual molecular scissors that cut the DNA (and allow editing to occur) are usually the Cas-9 protein. However, there is a whole ecosystem of Cas proteins that can Too DNA cuts, and as Mammoth’s leadership argues, could do even better in the original, depending on the application.

Mammoth is building a “CRISPR toolbox,” or collection of various Cas proteins. You can think of them as different types of scissors, each with their own specific use cases.

For example, in August 2020, mammoths discovered a family of proteins called Cas family. This family is an ultra-truncated version of the specific Cas9 protein that could make it easier to develop treatments in living people, and increase the accuracy of gene-editing. Mammoths have also characterized a Cas14 system, another family of ultra-small proteins that latch onto different target sequences in the genome (such as the landing pad that approximates where to intersect the Cas protein) than the Cas9 protein. We do.

“Mammoth was really founded with the idea that there is a CRISPR throughout this universe that goes beyond legacy systems like Cas9,” Martin says.

Developing the CRISPR toolkit isn’t just interesting science, it’s also a smart business move for Mammoth for another reason: intellectual property ownership.

The original CRISPR/Cas9 system has been the subject of patent fight Between the University of California Berkeley and MIT’s Broad Institute, where scientists also discovered CRISPR around the same time.

The new Cas proteins, which are not part of this patent battle, allow Mammoth to address that concern altogether. “The patent disputes that Broad concerns legacy CRISPR-Cas9 systems are involved. Mammoth’s systems are not Cas9-based, so they are not subject to these disputes,” explains Martin.

In a nutshell, Mammoth is building a collection of proprietary tools that can be put to use later. Although the possibilities are nearly limitless (genetic medicine or CRISPR-based diagnosis) many of these therapeutic products do not yet exist.

2020 was a big year for CRISPR therapeutics due to the influx of New clinical trials. that To suggest that medical science is just beginning to work its way through regulatory requirements – although approvals are still far from over.

Companies such as CRISPR Therapeutics and Vertex Pharmaceuticals have announced promising results from trials on CRISPR-based beta thalassemia and sickle cell treatments. And this summer, Intellia Therapeutics (another company co-founded by Doodna) and Regeneron took the field one step further, show that CRISPR-based therapies injected directly into the body were useful in silencing the genes that cause ATTR amyloidosis, a disorder where proteins produced in the liver misfold (this can lead to complications, such as heart failure over time).

Mammoth’s place in the expanding world of CRISPR therapeutics, Martin notes, will focus on in-vivo applications (or treatments delivered in the human body), which he argues his CRISPR toolbox could enable.

“We don’t have a timeline [for potential products] On the therapeutic side but we’ll definitely release more information over the next few years and we’re really excited about the technical results so far,” Martin says.

However, diagnostics is already one area where the mammoth could isolate itself sooner rather than later. There, the company is already working with partners to build a viable product.

Mammothoo in January earned money Through Defense Advanced Research Projects Agency (DARPA) to develop Point-of-care tests that can detect up to 10 pathogens at once, and a larger, lab-based test that can detect up to 1,000. Mammoth too received money From National Institutes of Health (NIH) Rapid Acceleration of Diagnostics (RADX) Program to develop advanced diagnostics using CRISPR, and entered a Partnership with GSK To develop a point-of-care COVID-19 test that can detect viral RNA in about 20 minutes.

Despite the “pressure,” Parikh says, Mammoth has forayed into both the world of diagnostics and therapeutics. He says the unicorn valuation is an additional sign that the company’s technology can work in both worlds.

“I think this milestone, this round, validates their approach to company building, which was to really focus on their area of ​​expertise, not just in a box of diagnostics or therapeutics themselves. Instead of keeping,” he says.

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