Treatment of cystic fibrosis
US8410081B2 · kind B2 · utility
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1References
11Claims
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Assignee
Inventors
Key dates
| Filing date | Apr 24, 2007 |
| Grant date | Apr 2, 2013 |
| Priority date | — |
| Expiry date | Sep 12, 2029 |
Classification
- Technology area (CPC A)Human Necessities
- CPC primaryA61P43/00
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention discloses a therapeutic target for the treatment of cystic fibrosis. It was found that inhibition of non-lysosomal glucosylceramidase (GBA2) sufficiently restores chloride current in cells from CF patients carrying the common delF508-CFTR mutation. With the catalytic center (4) of the enzyme positioned on top of the membrane bilayer face particularly potent inhibitors are found in deoxynojirimycin derivatives having a group that is capable of inserting in the membrane bilayer.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.