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LON-22 is an early-stage preclinical combination therapy developed by ONESTX for the treatment of neurodegeneration. It consists of the small molecule steroid sulfatase (STS) inhibitor STX64 (irosustat) and neurosteroids. The therapeutic approach focuses on modulating the biology of sulfated neurosteroids; by inhibiting the STS enzyme, the drug regulates the conversion of sulfated steroids into their non-sulfated counterparts, which is hypothesized to provide neuroprotective effects in neurodegenerative conditions.
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