Peter Thiel-backed Synthego raises $110 million

Synthego

• A Silicon Valley genome engineering startup founded by
  former SpaceX engineers called Synthego sells ready-to-use kits
  that let people experiment with the cutting-edge
  gene editing tool CRISPR.
• On Tuesday, Synthego received $110 million from Peter
  Thiel’s Founders Fund and two other Silicon Valley venture
  firms — bringing the company’s total funding to $160
  million.
• CRISPR holds massive promise for a field known as
  
  gene therapy, which involves modifying someone’s DNA to
  treat a genetic disease like sickle cell.
  
• Synthego sells its CRISPR kits primarily to researchers
  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.

On Tuesday, a little-known startup founded by two former SpaceX
engineers became one of the most well-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 do to everything
from creating potential designer crops to finding new cures for
rare diseases.

Recently, the company has been focusing on the latter, 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.

Their kits “open up a whole new world” to researchers, Synthego
co-founder and CEO Paul Dabrowski told Business Insider.

And the new funds — a total of $110 million from tech mogul Peter
Thiel’s Founders Fund, as well as 8VC and Menlo Ventures — will
help expand their reach even further, Dabrowski said. For the
first time, the company is beginning to make kits that will
eventually be ready to use in the clinic, a goal he said they aim
to achieve by next year.

“Synthego’s platform … will ultimately unlock the full
potential of genome engineering,” Thiel said in a statement.

‘A future where cell and gene therapies are as accessible as
vaccines’

Synthego

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
only used a single time after being approved in the European
Union in 2012. After lackluster demand, manufacturer UniQure

pulled it from the market in 2017. The first gene therapy to
be approved in the U.S. is called Luxturna, and 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. Unfortunately, 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 their 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 claims his company’s CRISPR editing kits significantly
shrink the time and the cost of doing research and development
work. If they 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

Jennifer
Doudna, co-inventor of blockbuster gene-editing tool CRISPR, sits
on Synthego’s advisory board.

Flickr/Photo:
James Duncan Davidson/TED

Synthego currently 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 advisor 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,
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 co-founded CRISPR
Therapeutics, one of several public companies working on
developing gene-editing based treatments. Sir Andrew Witty, who’s
the CEO of the technology and services division of UnitedHealth
Group and once served as CEO of 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, they 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 non-academics $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.