Usher syndrome (CRISPR)
Usher Syndrome
DiscoveryActive
Key Facts
About GeneToBe
GeneToBe is a private, preclinical-stage biotech developing transformative gene therapies for genetically-defined diseases. The company leverages a dual-technology platform centered on a proprietary, high-fidelity CRISPR-Cas9 system (MiCas9) and a liver-selective siRNA platform for gene silencing. Its pipeline targets significant unmet needs in cystic fibrosis, Usher syndrome, and liver diseases like MASH, positioning it in high-value therapeutic areas. Founded in 2016 as a University of Michigan spinout, the company is backed by private angel investors and led by a team with strong scientific and clinical development expertise.
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