On Tuesday, a little-known startup founded by two former SpaceX engineers became one of the best-funded life-science startups using the blockbuster gene-editing tool Crispr for genome engineering.
Called Synthego, the company sells easy-to-use Crispr kits to scientists, who use the tool to perform tasks as varied as creating designer crops and finding new cures for rare diseases.
Recently, the company has been focusing on diseases, with an emphasis on how Synthego could help make its tools more useful and accessible to researchers aiming to create a revolutionary new class of treatments known as gene therapy.
Despite decades of being touted as having the potential to cure dozens of diseases, gene therapies remain difficult to access. Few have been approved by federal regulators; those that have can cost roughly $1 million to receive and even more to develop. Synthego aims to help reduce that cost and speed up the development process. While a handful of startups are doing gene-editing work with Crispr, Synthego is the only one selling ready-to-use kits.
Its kits “open up a whole new world” to researchers, Synthego cofounder and CEO Paul Dabrowski told Business Insider.
And the new funds — a combined $110 million from the tech mogul Peter Thiel’s Founders Fund as well as 8VC and Menlo Ventures — will help expand the company’s reach even further, Dabrowski said. For the first time, the company is beginning to make kits intended for use in the clinic, a goal he said the company aimed to achieve by next year.
In a statement, Thiel said Synthego’s platform would “ultimately unlock the full potential of genome engineering.”
‘A future where cell and gene therapies are as accessible as vaccines’
Few patients have had the chance to get a revolutionary type of treatment long touted as having the potential to cure dozens of diseases.
Called gene therapy, the approach involves modifying a person’s DNA to address the underlying cause of an inherited disease. Doctors take a sample of someone’s diseased cells, correct the errors in the code, and return the corrected cells to the person’s body. Over time, the healthy cells outnumber the diseased ones, and the illness disappears for good, the thinking goes.
The Western world’s first gene therapy, a drug called Glybera that was designed to treat a rare disease in which patients can’t properly process fat, was used only a single time after being approved in the European Union in 2012. After lackluster demand, the manufacturer uniQure pulled it from the market in 2017. The first gene therapy to be approved in the US, Luxturna, promises to cure a rare inherited form of blindness. Its price tag is $850,000.
Crispr is a gene-editing tool that makes it easier and cheaper to modify genomes and could help usher in new gene therapies. But few researchers have fully embraced Crispr; the technique is new and can take months of practice to feel comfortable using. That’s where Synthego wants to help. Synthego’s core product helps scientists study potential cures by packaging Crispr in an easy-to-use format. As part of its latest funding round, the company is — for the first time — starting to make products that are ready to use in a clinical setting.
“One of the things we’re doing with our Crispr platform is making it clinical-grade ready,” Dabrowski said.
Dabrowski says his company’s Crispr editing kits significantly shrink the time and the cost of research and development. If the company can make products that are ready to use in the clinic, “there’s an ability” — eventually — “to actually make the medicines for our customers,” Dabrowski said.
“We believe it’s going to be possible to make cell and gene therapies that are as accessible as vaccines,” Dabrowski said.
A star-studded advisory board
Synthego sells two types of Crispr kits in roughly 50 countries. In the US, clients include the Mayo Clinic and two high-profile labs at Stanford and the University of California at Berkeley, where scientists use the kits to carry out research and development on potential new treatments.
Jennifer Doudna, one of the inventors of Crispr and a UC Berkeley geneticist, uses Synthego’s products in her lab, Dabrowski said. Doudna also joined Synthego as an adviser in March and invested in the company during its second-stage, or series B, round last year.
As part of the latest funding round, another high-profile researcher, the Stanford professor and physician Matthew Porteus, joined Synthego’s advisory board. Porteus has been working on gene and cell therapies for half a decade and cofounded Crispr Therapeutics, one of several public companies working on developing gene-editing-based treatments. Sir Andrew Witty, the CEO of the technology and services division of UnitedHealth Group who once served as CEO of the pharmaceutical giant GlaxoSmithKline, is also on Synthego’s advisory board.
Both of Synthego’s kits are designed to make working with Crispr easier. The first, called Crisprevolution, is designed for researchers who already have experience working with the tool and costs as little as $225. As part of the latest funding round, Synthego will be expanding that product line so that by next year it could offer clinical-grade materials with the potential to help make therapeutics for patients. Synthego’s other product, called Engineered Cells, was designed for non-Crispr experts in mind and costs nonacademics $3,500, or $2,500 for researchers.
“You come to the website, tell us what you want modified, swipe your credit card, and a few weeks later you end up with an edited cell line,” Dabrowski said.
That significantly shortens a process that normally takes months — and could help researchers turn big ideas into real, accessible treatments within a few years, Dabrowski hopes.
“You get rid of a huge road block,” Dabrowski said.