The company, led by former Epizyme CEO Robert Gould, Ph. D, says the cash boost will be put toward its program in facioscapulohumeral muscular dystrophy (FSHD), seeing it go from preclinical to clinical testing, as well as “further support advancement of rare disease-focused pipeline,” which includes genetically-based neuromuscular, central nervous system and hematologic disorders.
The Cambridge, Massachusetts–based biotech saw its latest funding round led by Foresite Capital, with help from Fidelity Management and Research Company, 6 Dimensions Capital, Casdin Capital, Sanofi Ventures, Section 32, NS Investments, entities affiliated with Leerink Partners, and undisclosed institutional investors.
“We are delighted to have the support of this exceptional group of investors as we continue to work towards our vision of bringing new futures to patients and families affected by debilitating genetic diseases,” said Gould.
“This funding will provide crucial support as we move rapidly towards the clinic with our lead drug candidate in FSHD, and further advance our pipeline of small molecule therapies.”
In FSHD, patients will end up in a wheelchair and progressively become weaker, and nothing can help slow or reverse this process.
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The company is working on gene regulation: Attempting to switch on and off certain genes using patient cells and CRISPR-Cas9, some of the most recent and innovative biology and technology brought together under one roof.
Back in 2016, when it won a Fierce 15 award, Gould told FierceBiotech that it was looking to use induced pluripotent stem cells (iPSCs), derived from skin, to mimic the cellular processes that have gone wrong in these diseases with altered gene expression.
He explained: “The uniqueness of Fulcrum is really this combination of iPS cell mimicking gene biology with the understanding of what human biology is trying to regulate, in terms of gene expression, and combining that with the technology of CRISPR-Cas9 and using cells from the patients that we’re trying to treat. This all together is an unprecedented approach and opportunity.”