Methods and compositions for CNS delivery of heparan N-sulfatase
US12161702B2 · kind B2 · utility
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18References
19Claims
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Key dates
| Filing date | Jun 17, 2021 |
| Grant date | Dec 10, 2024 |
| Priority date | — |
| Expiry date | May 14, 2042 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y310/01001
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising a heparan N-sulfatase (HNS) protein, salt, and a polysorbate surfactant for the treatment of Sanfilippo Syndrome Type A.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.