AAV9-mediated gene therapy for treating mucopolysaccharidosis type I
US11890329B2 · kind B2 · utility
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37Claims
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Key dates
| Filing date | Jul 5, 2018 |
| Grant date | Feb 6, 2024 |
| Priority date | — |
| Expiry date | Aug 13, 2040 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2750/14143
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
A co-therapeutic regimen comprising AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDUA gene and two or more immunosuppressants is provided herein. Also provided are methods useful for treating hIDUA deficiency (MPSI) and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.