On the back of a busy year, Mammoth Biosciences is getting a $195 million boost, which will ramp up the development of its gene-editing pipeline, as well as fuel its work in CRISPR-based diagnostics.
The company isn’t revealing yet which disease areas it’s targeting with its gene-editing treatments but has “devoted a lot of resources to hiring on the gene-editing side,” said Janice Chen, Ph.D., co-founder and chief technology officer of Mammoth. It hopes to create treatments for tissue that are hard to target, such as the central nervous system or lung.
“Anything where we can solve problems … that current approaches can’t do, or it’s too difficult, or they’re not very effective,” said CEO and co-founder Trevor Martin, Ph.D.
Its medicines will work in vivo, editing DNA within the body as opposed to ex vivo, which involves removing a patient’s cells, modifying them, and reinfusing them into the patient, Martin said. It’s an approach that Intellia Therapeutics and Regeneron recently showed could work in the human body.
Mammoth’s diagnostics and therapeutics pipelines are built on a toolbox of new Cas enzymes with various strengths, from the speed and cost of producing them to the kinds of genetic sequences they can cut—single-stranded DNA, double-stranded DNA and single-stranded RNA, for example. Two of its enzymes, Cas14 and Casɸ, are the smallest known CRISPR systems.
Size is important when it comes to fitting a gene-editing treatment into a delivery vehicle, such as an adeno-associated virus (AAV) or liquid nanoparticle. Though current CRISPR-based treatments include a guide RNA to find the disease-causing gene and an enzyme to edit that gene, future approaches could add other components to enhance editing. Smaller molecules mean drug developers can pack more features into their treatments.
Mammoth launched in April 2018 with CRISPR technology licensed from the lab of Jennifer Doudna, Ph.D. and a mission to create a point-of-care diagnostic tool that can test for multiple conditions in various settings, such as in hospitals or at home. It kicked off 2020 with a $45 million series B and ramped up its diagnostics in the face of the COVID-19 pandemic, inking deals with partners such as Agilent and GlaxoSmithKline.
“The pandemic didn’t just catalyze efforts in the diagnostics space [for COVID-19,] but also highlighted the gaps that exist in the system—what diagnostic technology is available and what still needs to be built,” Chen said.
The company believes CRISPR can bridge those gaps, surmounting the limitations of traditional approaches to diagnose infectious and other diseases, said Martin and Chen.
Besides developing new systems internally and with partners, Mammoth is taking steps to “decentralize” testing, including making technology that can be used in “nontraditional healthcare sites,” such as the home.
“As we graduate from the immediate need of the pandemic, we are thinking about what the future of diagnostic testing looks like,” Chen said.
The company raised its new funding, of which $45 million came in a series C and $150 million in a series D, from various backers, including Redmile Group, Foresiter Capital, Senator Investment Group, Sixth Street, Greenspring Associates, Mayfield, Decheng Capital, NFX, Plum Alley, and Amazon.