Celsius bags $65M to develop precision drugs for cancer, autoimmune disease

Third Rock partner Alexis Borisy, center, with Celsius Therapeutics cofounders Aviv Regev, left, and Christoph Lengauer. (Celsius Therapeutics)

Third Rock Ventures launched Celsius Therapeutics, which is setting out to develop therapeutics based on single-cell genomic sequencing. Armed with $65 million in series A funding, the startup will sequence defined patient samples to identify the individual cells responsible for disease. It hopes to bring forth the first precision medicines for autoimmune disease. 

Christoph Lengauer, Celsius president and cofounder, likens traditional genomic sequencing to a smoothie: "Before, we looked at tissue knowing that there are many different types of cells in there. You put all it in a blender and then sequence it to find the average," he said. But the same way some types of fruit may overpower others in a mixture, some cells and their contributions to disease are imperceptible. With its single-cell approach, Celsius is trying to look at the individual "fruit" to discover which genes determine the function of cells and the interactions between them, and what goes wrong to cause disease. 

"[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," said cofounder Aviv Regev, Ph.D., core institute member, chair of the faculty and director of the Klarman Cell Observatory at the Broad Institute, in a statement.

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"These new technologies and algorithms would give us unprecedented resolution and clarity to identify underlying causes of disease, reducing the guesswork, and pursuing precise predictions into therapeutics," said Regev, who is also a professor of biology at MIT, and an investigator at the Howard Hughes Medical Institute.

The startup draws its funding from Third Rock, Alphabet's venture arm GV, Heritage Provider Network, Casdin Capital and Alexandria Venture Investments. Regev, Lengauer, a venture partner at Third Rock, and the other cofounders have been building Celsius since 2016. Aside from sequencing and studying clearly defined patient samples, the company's next steps include industrializing its genomic sequencing processes and machine learning algorithms and advancing its drug discovery programs, which are still "very early-stage." The programs are based on targets identified by the founders via single-cell genomics. 

Precision medicine has not been deployed against autoimmune disease because it is complicated to understand, Lengauer said.

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"If, for example, we take inflammatory bowel disease or psoriasis, we know inflammation in certain cell types is driven by how cells ineract with each other. But this is something we couldn't look at before. We didn't have the resolution on the single-cell level," he said. 

The company is still thinking broadly in terms of therapeutic area, Lengauer said. When it comes to autoimmune disease, Celsius believes that some fundamental mechanisms in genes will play a role in different tissues the way certain genes are implicated in multiple cancers. 

Celsius has 15 staffers, split evenly between drug discovery, disease biology and data science/computational biology, Lengauer said. He expects the company to grow to about 35 employees by the end of the year.