Former Epizyme CEO Helms Fulcrum Therapeutics; New Startup Banks $55 Million Series A
July 19, 2016
By Alex Keown, BioSpace.com Breaking News Staff
Cambridge, Mass. -- Robert Gould, the former president and chief executive officer of Epizyme will helm Fulcrum Therapeutics, a new company focused on unlocking gene control mechanisms through technologies such as CRISPR-Cas9, to develop small molecule therapies.
Fulcrum Therapeutics launched this week $55 million in Series A financing. The company sprang from Third Rock Ventures, LLC. The financing will be used to discover and develop small molecules that modulate the on/off control mechanisms that regulate genes, Third Rock said in a statement. Fulcrum has a goal of developing a cross-disciplinary product engine to identify and modulate gene regulatory targets core to disease, the company said in a statement. Through its product engine, Fulcrum will develop a robust pipeline across therapeutic areas, spearheaded by two initial programs in genetic diseases.
Through the modulation and manipulation of the “on and off switches” of genes, Gould said they have the potential to “transform the treatment of hundreds of serious human diseases.”
“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 cod—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,” Gould said in a statement.
Fulcrum plans to focus its technology on two genetic diseases—Fragile X Syndrome and a form of muscular dystrophy called facioscapulohumeral muscular dystrophy. Both of these diseases occur due to a single gene mutation that creates an error in gene regulation. Other genetic diseases the company said can be explored with its “robust product engine” include other neurologic disorders, skeletal muscular disorders and cardiac muscular disease.
Fulcrum’s product engine was created to “integrate and accelerate” the advances that are reshaping biological disciplines that include transcription biology, stem cell biology and computational biology, the company said. 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 added.
Fulcrum’s gene technology uses screening tools such as CRISPR-Cas9 and chemical probe libraries to dissect gene mechanisms in cellular models of diseases. "CRISPR" refers to Clustered Regularly Interspaced Short Palindromic Repeats that occur in the genome of certain bacteria, from which the system was discovered. Cas9 is a CRISPR-associated endonuclease (an enzyme) known to act as the "molecular scissors" that cut and edit, or correct, disease-associated DNA in a cell. The technology was discovered by UC Berkeley professor Jennifer Doudna and Emmanuelle Charpentier.
Gene therapy essentially transforms cells inside a patient to harness their immune system to fight an invading disease on its own. CRISPR-Cas9 is considered revolutionary technology, and as such is likely, at some level, to be used by many companies and institutions.
Several companies are harnessing CRISPR technologies, including Caribou Biosciences, which has formed a strategic alliance with DuPont , and Novartis , Caribou also formed Intellia Therapeutics, which is active in gene therapy. In 2015, Juno Therapeutics struck a $727 million deal with Editas Medicine , the gene editing company, for a partnership that will forge three research programs marrying Editas’ technologies, including CRISPR-Cas9, to Juno’s CAR-T and TCR technologies.