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.
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 language | English |
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| Publication status | Published - 24 Feb 2021 |
| Event | QUB Winter School Meeting (School of Medicine) - Belfast Duration: 01 Jan 2020 → … |
Conference
| Conference | QUB Winter School Meeting (School of Medicine) |
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| City | Belfast |
| Period | 01/01/2020 → … |