Third Rock is teaming up with Google’s venture arm to back a gene therapy startup stepping out today with $65 million in new money. As you might expect with GV’s involvement, the new venture has a technology twist, and it might have its hands on something big for the autoimmune world.
Are you ready for a game of buzzword bingo? The startup, called Celsius Therapeutics, is using new tech in genetic sequencing (check) — and a proprietary machine learning (check) algorithm (check) — to tackle precision meds (check) for cancer (check) and autoimmune disease. I’ll break that down for you later.
The technology the company is built on was licensed from the Broad Institute based on the work of Aviv Regev and Vijay Kuchroo (both founding scientists at Celsius), including non-exclusive licenses to single-cell sequencing tech and exclusive licenses to early-stage drug programs.
The company’s co-founder and president, Christoph Lengauer (a Third Rock venture partner), tells me Celsius has a few things that give it an edge: a massive amount of animal and human data to work with, a machine-learning algorithm to make sense of that data, and a new technology to sequence single cells rather than whole genomes.
Our body has trillions of cells, each with genetic information stored inside. The sets of genes vary in different cell types, determining a cell’s function — and sometimes — the code behind disease. Homing in on single cells could help researchers better understand the individual cells and their interactions that cause disease, perhaps leading to better therapies down the road.
When comparing single cell sequencing with whole genome sequencing, Lenguaer used an analogy about making smoothies with fruit.
“If you put strawberries and kiwis in a blender, the color of your smoothie will be pink because it’s the dominant cell population,” Lengauer said. “Anything related to the kiwi would be lost. That’s whole genome sequencing. With this analogy, single cell sequencing would allow you to see the individual fruits in the smoothie. And if something was rotten, you could pinpoint the individual fruit that’s gone bad.”
Of course, we’re not talking of strawberries and kiwis, but of cells that are causing disease and the genes that trigger their malfunction. Celsius believes this tech could be the key to bring precision medicines to autoimmune diseases for the first time.
“Many diseases are driven by the combined dysfunction of several specific cell types, and the interactions between them,” said Regev, the MIT professor who co-founded Celsius, in a statement. “With traditional genomic sequencing, we cannot identify these individual contributions — we only see the average and can miss out key critical causes. But for the first time, with the approaches discovered by our team, where we combine massive datasets of unprecedented size and complexity with sophisticated machine learning algorithms, we are able to distinguish the specific cells, among many others, that play a key role in disease and identify the genes that are triggering their malfunction. We believe our approach will allow us to more efficiently identify specific targets for treating diseases in specific patients and ultimately develop medicines for those targets.”
Using this approach, the Broad Institute has uncovered “a handful” of targets in autoimmune conditions and cancer. Lengauer said Celsius hopes to reach proof of concept in the next five years, and the Series A should take them at least partway toward that goal.
While Third Rock led the recent round, GV participated along with Heritage Provider Network, Casdin Capital, Alexandria Venture Investments and others.
The company, which has been operating in stealth mode for the past two years, employs 15 people at its Cambridge headquarters in Kendall Square.
Image: Aviv Regev, Third Rock partner Alexis Borisy, Christoph Lengauer. Celsius Therapeutics
The best place to read Endpoints News? In your inbox.
Comprehensive daily news report for those who discover, develop, and market drugs. Join 44,100+ biopharma pros who read Endpoints News by email every day.Free Subscription