January 9, 2017
By Mark Terry, BioSpace.com Breaking News Staff
Fulcrum Therapeutics was launched in July 2016 and is focused on unlocking gene control mechanisms in order to create small molecule therapies. Numerous diseases are caused by inappropriate activity—both on and off—of genes. Fulcrum plans to use several different modalities to develop compounds to correct that on-off activity.
The company was founded by:
•Michael Green, from the University of Massachusetts Medical School and The Howard Hughes Medical Institutes.
•Danny Reinberg, from New York University School of Medicine and The Howard Hughes Medical Institutes.
•Rudolf Jaenish, from the Whitehead Institute for Biomedical Research, Massachusetts Institute of Technology , and the Institute of Medicine.
•Jeannie Lee, from Massachusetts General Hospital, Harvard Medical School, and The Howard Hughes Medical Institute.
•Brad Bernstein, from Massachusetts General Hospital, Harvard Medical School, and the Broad Institute of MIT and Harvard.
The company models gene regulation in disease tissue that is either donated through tissue biopsy or culled from skin cells using induced pluripotent stem cell technology. Then it uses CRISPR/CAS9 and chemical probe libraries to analyze gene regulatory mechanisms.
Company Leadership
Robert Gould—president, chief executive officer, board member. Before Fulcrum, Gould was president and chief executive officer of Epizyme .
Angela Cacace—vice president, biology. Most recently, Cacace was a director of genetically defined diseases at Bristol-Myers Squibb .
Diego Cadavid—vice president, clinical development. Prior to joining Fulcrum, Cadavid was senior medical director in the MS Clinical Development Group and lead of the CNS Remyelination Team for Biogen .
Lucienne Ronco—vice president, translational medicine. Before Fulcrum, Ronco was a director within the Center for the Development of Therapeutics at the Broad Institute of MIT and Harvard. Prior to that she was president of research at Catabasis Pharmaceuticals .
Lorin Thompson—senior director, molecular sciences. Prior to Fulcrum, Thompson led both early- and full-phase drug discovery programs in both neuroscience and genetically-defined diseases for Bristol-Myers Squibb.
Walter Kowtoniuk—director, strategy and operations. Kowtoniuk was a senior associate at Third Rock Ventures, where he led the creation of Fulcrum.
Company Financing
In July 2016, the company launched with $55 million in Series A financing from Third Rock Ventures.
Pipeline
Initial work is on fragile X syndrome (FXS) and facioscapulohumeral muscular dystrophy (FSHD). FXS is the most common form of inherited learning disabilities. FSHD is a genetic muscle disorder that affects mostly the muscles of the face, shoulder blades and upper arms.
The company’s plan is to use pluripotent stem cells (iPS) that are derived from the skin and can mimic the cellular processes that are involved in the abnormal gene expression.
In FXS, the abnormality involves a lack of production of the FMRP protein. That protein has been inappropriately turned off, and the company hopes to develop a molecule that can turn it back on.
In FSHD, it’s the opposite. There is a protein called DUX4 that is being manufactured when it shouldn’t be, because it can be toxic.
What to Look For
The company is very young and still focused on preclinical development.
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