Compositions useful in treatment of spinal muscular atrophy
US11578341B2 · kind B2 · utility
1Cited by
91References
14Claims
0Family size
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Key dates
| Filing date | Feb 27, 2018 |
| Grant date | Feb 14, 2023 |
| Priority date | — |
| Expiry date | Dec 23, 2038 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2800/22
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
A rAAV vector is described herein which has an AAVhu68 capsid and at least one expression cassette in the capsid. The at least one expression cassette comprises nucleic acid sequences encoding a functional SMN protein and expression control sequences that direct expression of the SMN sequences in a host cell. Also provided are compositions containing this rAAVhu68.SMN vector and methods of using same for spinal muscular atrophy in a patient.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.