Scoop: Google’s GV spearheads the Spotlight syndicate — backing an upstart biotech aimed at ‘democratizing’ gene editing
CRISPR had no sooner started to shake the very foundations of drug development before its limitations began to loom large. Gene editing could change the world — if only you could get around the hurdles that threatened to trip up every program.
So it’s only natural to see CRISPR 2.0 taking shape before the pioneers can get the lead therapies through development. And who better than Google’s GV venture arm to take the lead spot in a small syndicate backing some scientists with their own unique twist on a solution?
GV general partner Blake Byers took point on this one, joining with some individual investors who are staying in the background. They were all attracted by the idea that the small team at Spotlight Therapeutics could bypass the delivery issues embedded in every gene editing program by using programmable CRISPR ribonucleoproteins designed for in vivo cell-targeted delivery.
And now the Spotlight team is jumping into the industry spotlight with a $30 million launch round to go after lead programs in hemoglobinopathies and immuno-oncology.
Right now, despite all the promise of CRISPR, the pioneers are unable to address specific cell types in vivo, says CEO Mary Haak-Frendscho. This new approach offers the opportunity “to really open up, democratize gene editing.”
The tech used today depends a lot on using an AAV or LNP to express the editor in the target cell, says Byers. Those have better uptake in liver cells, so that’s where the early players have started. And as we’ve seen at Solid and other places, there are lingering concerns about safety and efficacy.
Spotlight, he says, is taking a different, completely non-viral approach. And it starts with a question.
“Why don’t we bind the RNP (ribonucleoprotein) editor to other things?” asks Byers. “That could be other proteins, let’s say an antibody. Get antibodies to a target on the cell surface, it’s internalized at some rate, then hitchhikes into the cell and from there edit the target site in the genome.”
“There’s a lot of things you can latch your gene editors to,” says Byers. The goal is to explore the space, searching for 5 to 10 portfolio products bound to different hitchhiker molecules that you can use to get your editor of choice into the exact cell type you want, with high specificity and low cost of goods.
In the process, you have to escape destruction by the lysosome and go on to the nucleosome to do the editing, so there’s plenty of debugging along the way to get it to work right.
Much of the inspiration for this work came from 3 key scientists who are advising the startup.
Alex Marson and Patrick Hsu thought a lot of this out. Marson is at UC San Francisco and Hsu is now close by at UC Berkeley. Jacob Corn at ETH Zürich rounds out the lineup of scientific founders with prestigious resumes.
The longterm plan is to stick with the non-viral approach and build a pipeline.
As A rounds go these days, $30 million isn’t a lot of money, particularly when you’re talking about a tech platform approach like this. I pointed that out to Byers, who wasn’t feeling apologetic about the $30 million.
“How old school of us,” he responded, adding that there are different strategies for different players. Some should be laser focused on product development. Besides, he adds, “small mounts of capital are very focusing for a company.”
The money “gets us to 2022,” says the CEO. At that point they expect to have their first development candidate, within a year of the clinic for either or both their chosen indications.
And then they can see about starting their own gene editing revolution.