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CIMR spin-out company starts first clinical trial

last modified Sep 28, 2020 08:48 AM

Z Factor Ltd have announced that dosing has begun in a Phase I study of ZF874, their first-in-class, small molecule drug. This was discovered from research at Prof. Jim Huntington's laboratory in CIMR as a potential treatment for alpha-1-antitrypsin deficiency (AATD). Most forms of this genetic disease are caused by the misfolding and accumulation of the Z variant of alpha-1-antitrypsin in liver cells. As well as liver disease, there is also downstream lung damage in AATD. ZF874 is designed specifically to correct the misfolding of Z-AAT. Once the first stage of this safety and dosing study is complete in healthy volunteers, it will be extended to AATD patients. Results are expected by the end of 2020.