Development of an Inhaled CatS Inhibitor for the Treatment of CF: *This talk won the best talk of the session prize

Research output: Contribution to conferencePaperpeer-review

Abstract

Research conducted at Queen's has revealed that active Cathepsin S (CatS) is present active in the lungs of 100% of CF patients tested. Moreover, we have shown in animal model studies that depletion/deletion of CatS or small molecule inhibition leads to a reduction in the hallmarks of CF, such as lung mucus obstruction, inflammatory cell burden, lung damage and bacterial load. Our proposed solution is to develop a highly potent and selective CatS inhibitor directly into the lung via inhaled administration that will reduce the life-limiting hallmarks of CF in a broad spectrum of patients, irrespective of their particular CFTR mutation.
The development of an inhaled inhibitor that can directly target the lung and reduce the impact of this disease would be of significant value to the community. Research from my lab, around the development of an inhaled CatS inhibitor, led to the identification of QDD200164 - the most potent, selective and cell active CatS inhibitor reported to date.Research conducted at Queen's has revealed that active Cathepsin S (CatS) is present active in the lungs of 100% of CF patients tested. Moreover, we have shown in animal model studies that depletion/deletion of CatS or small molecule inhibition leads to a reduction in the hallmarks of CF, such as lung mucus obstruction, inflammatory cell burden, lung damage and bacterial load. Our proposed solution is to develop a highly potent and selective CatS inhibitor directly into the lung via inhaled administration that will reduce the life-limiting hallmarks of CF in a broad spectrum of patients, irrespective of their particular CFTR mutation.
The development of an inhaled inhibitor that can directly target the lung and reduce the impact of this disease would be of significant value to the community. Research from my lab, around the development of an inhaled CatS inhibitor, led to the identification of QDD200164 - the most potent, selective and cell active CatS inhibitor reported to date.
Original languageEnglish
Publication statusPublished - 24 Feb 2021
EventQUB Winter School Meeting (School of Medicine) - Belfast
Duration: 01 Jan 2020 → …

Conference

ConferenceQUB Winter School Meeting (School of Medicine)
CityBelfast
Period01/01/2020 → …

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