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Fulcrum is focused on small molecule therapeutics for diseases of gene misregulation. The company is creating therapeutics for a range of genetic disorders including: FSHD, Sickle Cell Disease, as well as other neuromuscular diseases. We plan to file our first IND in FSHD in 1H2019.
Fulcrum is founded on a precision medicine approach that integrates human genetics across the spectrum of drug discovery. Our integrated drug discovery approach has been productive by:
• focusing on the genetic root cause of the disease;
• creating accurate in vitro models using patient derived cellular models;
• using a combination of a curated gene regulation compound library, a CRISPR/Cas9 library and computational biology;
• and, developing genetic biomarkers to support an efficient path to the patient