Methods and compositions for CNS delivery of heparan n-sulfatase
US11065308B2 · kind B2 · utility
5Cited by
13References
5Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | Jul 16, 2018 |
| Grant date | Jul 20, 2021 |
| Priority date | — |
| Expiry date | Jul 16, 2038 |
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.