RxCelerate supported ZFactor through its seed funding leading to a successful A round. We continue to operate and manage all aspects of their lead optimisation program.
Selected a small (<700) set of drug like compounds directed to a pocket on PiZZ mutant antitrypsin and screened these compounds in relevant cell lines. The hit rate was exceptional and numerous lead series were identified.
As the compounds were selected for drug likeness three hits were further profiled and directly progressed to in vivo studies in PiZZ mice to give an early proof of principle.
Two main lead series are now being progressed through an optimised screening cascade.
Our main focus is on retaining the excellent drug likeness of the leads (especially low molecular weight, clean off target profile and high solubilty) whilst generating novelty.