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Fulcrum Therapeutics co-founder Michael Green, MD, PhD, is providing expertise in gene regulation. |
A leading researcher at UMass Medical School is one of five scientific co-founders of Fulcrum Therapeutics, a new company located in Cambridge’s Kendall Square focused on discovering and developing small molecules that modulate the on/off control mechanisms that regulate genes. This therapeutic strategy may be an extremely effective way to target genetic-based diseases. Third Rock Ventures, LLC announced the launch Tuesday. Michael Green, MD, PhD, Howard Hughes Medical Institute Investigator, the Lambi and Sarah Adams Chair in Genetic Research, chair and professor of molecular, cell, & cancer biology and professor of molecular medicine at UMass Medical School and director of the UMass Medical School Cancer Center, is providing expertise in gene regulation. Dr. Green is also a member of the National Academy of Sciences and the National Academy of Medicine.
Chancellor Michael F. Collins said UMMS is a global leader in translational science.
“The launch of Fulcrum Therapeutics is the latest example of the impact of UMass Medical School and the importance of its world leading science,” said Chancellor Collins. “Venture capital investors like Third Rock see the tremendous value of investing in translational science research and the work of the best minds tackling the toughest problems. We’re thrilled to be working with Fulcrum, which shares our commitment to developing approaches to use gene regulation to treat human disease.”
UMMS is committed to promoting innovation, supporting entrepreneurship, optimizing the medical school’s research portfolio and leveraging the wide array of expertise and capabilities that exists throughout the academic medical center. With more than $250 million in annual research funding, UMMS ranks among the top 2 percent of all National Institutes of Health-funded institutions. It is 14th nationally in annual licensing revenue, generating more than $33 million a year. The institution was issued 33 patents last fiscal year, propelling the UMass system overall to 30th among the top 100 worldwide universities granted patents in 2015.
Brendan O’Leary, PhD, executive vice chancellor of innovation and business development at UMMS, said Fulcrum’s mission aligns with that of UMMS.
“Turning our basic research discoveries into new disease treatments is a key component of our mission, but a very challenging and costly one. Therefore, we have made a strategic commitment to work with knowledgeable and well-resourced private sector partners, like Fulcrum, to accelerate development,” Dr. O’Leary said. “We are so pleased to collaborate with Fulcrum to develop treatments for these terrible diseases.”
Fulcrum was established with $55 million in Series A financing to further develop a cross-disciplinary product engine to identify and modulate gene regulatory targets core to disease. Through its product engine, Fulcrum will develop a robust pipeline across therapeutic areas, spearheaded by two initial programs in genetic diseases. The company is led by President and Chief Executive Officer Robert J. Gould, PhD, former president and CEO of Epizyme, and a longtime leader in drug discovery and development.
“One of the single most important biologic breakthroughs of the last decade has been the unraveling of gene regulation at a molecular level. For more than a decade we have understood the genetic code—the genome. Now that we are unraveling the way these genes are regulated and put to work in biology, there is an unprecedented opportunity for drug development,” Dr. Gould said. “The richness of the human genome comes from the way genes are turned on and off. Through the modulation of these on and off switches we have the potential to transform the treatment of hundreds of serious human diseases. We are excited to build Fulcrum Therapeutics—a company that is capable of translating these discoveries into groundbreaking new therapies that restore balance in patients who currently have no other therapeutic options.”
Fulcrum’s product engine was created to integrate and accelerate the rapid advances that have reshaped a wide range of biological disciplines, including transcription biology, stem cell biology and computational biology. The integration of these diverse advanced technologies throughout the drug discovery and development process creates the opportunity to deliver on the promise of genomic medicine; namely, to not only discover genetic disease drivers, but also to regulate gene expression to restore health.
The company believes there is long-term potential to address complex genetic diseases through its approach. The early focus of Fulcrum’s product engine is on two severe genetic diseases: Fragile X syndrome (FXS) and a form of muscular dystrophy called facioscapulohumeral muscular dystrophy (FSHD). Each disease arises from a single gene mutation that creates an error in gene regulation. In the case of Fragile X, the silencing of the FMR1 gene eliminates the cell’s ability to make a protein needed for brain function. In FSHD, the precipitating event is the activation of a gene called DUX4 that should be silent in adulthood. This activation leads to muscle wasting as cells die. Other genetic diseases that can be explored with Fulcrum’s robust product engine include other neurologic disorders, skeletal muscular disorders and cardiac muscular disease.
The company’s internal discovery and development team is complemented by a group of scientific founders. Together, this combined expertise integrates the elements of the Fulcrum Therapeutics product engine.
Overall leadership is provided by Gould, who served as Epizyme’s president and chief executive officer from 2010 to 2015. Prior to joining Epizyme, from 2006 to 2010, he served as director of novel therapeutics at The Broad Institute of MIT and Harvard. He was vice president, licensing and external research and vice president, basic research, Merck Research Laboratories, at Merck & Co., Inc., where he held a variety of leadership positions during his 24-year tenure.
Company founders include:
- Dr. Green, a highly accomplished scientist who studies the mechanisms that regulate gene expression in eukaryotes and the role of gene expression in various human disease states.
- Danny Reinberg, PhD: New York University School of Medicine; The Howard Hughes Medical Institute; National Academy of Sciences; providing expertise in chromatin structure and function
- Rudolf Jaenisch, MD: Whitehead Institute for Biomedical Research; Massachusetts Institute of Technology; Institute of Medicine; National Academy of Sciences; providing expertise in stem cell biology
- Jeannie Lee, MD, PhD: Massachusetts General Hospital; Harvard Medical School; The Howard Hughes Medical Institute; National Academy of Sciences; providing expertise in gene regulation and X chromosome genetics
- Brad Bernstein, MD, PhD: Massachusetts General Hospital; Harvard Medical School; Broad Institute of MIT and Harvard; providing expertise in genome-wide characterization of gene regulation.
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