- 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.