Maze Therapeutics Opens Its Doors to Study Modifier Gene-Related Diseases

Maze Therapeutics, based in San Francisco, launched with an initial investment of $191 million. The investment was led by Third Rock Ventures and ARCH Venture Partners. GV (formerly Google Ventures), Foresite Capital, Casdin Capital, Alexandria Venture Investments and other undisclosed investors participated.

The company will focus on what are dubbed modifier genes. Essentially, two people with the same disease-causing genetic mutations often show varying degrees of illness, or in some cases, none at all. Although environment is a factor, with increasing amounts of genetic information available, the impact of modifier genes is becoming clearer.

Charles Homcy, a Third Rock partner and chief executive officer of Maze, indicated that with technology like CRISPR-Cas9, they believe the can reverse engineer these situations and develop drugs that behave like the beneficial gene.

The company has been working on this for two years and has identified several cases of disease-improving modifier genes. The company plans to develop a small molecule drug for one of those diseases and hopes to have it in the clinic in the next three years. For another indication, it plans to develop a gene therapy. Although short on specifics, the company suggests it has three or four drug discovery programs, and expects to have two of the programs in the clinic in less than three years.

Although Homcy chose not to disclose the specific indications, he was willing to say they will focus first on Mendelian diseases, which involved a single mutation in a single gene.

Homcy stated, “At Maze, we are focused on expanding our understanding of the natural disease protection provided by genetic modifiers through an integrated approach that combines studying natural human genetic variation across the globe and conducting large-scale experiments of gene perturbations. Through our integrated approach, we believe we will create novel medicines based around those modifiers to treat a number of diseases.”

The company’s scientific founders include Mark Daly, co-director of the Program in Medical and Population Genetics at the Broad Institute; Stephen Elledge, Gregor Mendel Professor of Genetics and Medicine at Harvard Medical School and Brigham and Women’s Hospital; Aaron Gitler, professor of genetics at Stanford University; Sekar Kathiresan, director of the Center for Genomic Medicine at Massachusetts General Hospital; and Jonathan Weissman, professor and vice chair of the Department of Cellular & Molecular Pharmacology at the University of California, San Francisco.

Rounding out the executive suite are Tim Behrens, senior vice president, human genetics; Matthew Brauer, vice president, data science; Jason Coloma, chief operating officer; Jeffrey Finer, chief technology officer; Eric Green, vice president, R&D; David Morgans, senior vice president, drug discovery; and Tara Nickerson, chief business officer.

“We are at a critical juncture in drug development where we now have a breadth of genetic information that gives us insights into the root causes of disease, as well as powerful tools that allow us to test and exploit these insights,” stated Weissman. “Maze has been established at the perfect time to combine these elements, which we believe will lead to important discoveries and new treatments for patients.”