Recombinant adeno-associated virus for treatment of GRN-associated adult-onset neurodegeneration
US12416017B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Feb 21, 2020 |
| Grant date | Sep 16, 2025 |
| Priority date | — |
| Expiry date | Jan 16, 2043 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2750/14171
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
A recombinant AAV (rAAV) suitable for use in treating adult onset neurodegeneration caused by granulin (GRN) haploinsufficiency, such as progranulin (PGRN)-related frontotemporal dementia (FTD), is provided. The rAAV comprises (a) an adeno-associated virus 1 capsid, and (b) a vector genome packaged in the AAV capsid, said vector genome comprising AAV inverted terminal repeats, a coding sequence for human progranulin, and regulatory sequences which direct expression of the progranulin. Also provided are a method for treating a human patient with PGRN-FTD and other adult onset neurodegeneration caused by granulin (GRN) haploinsufficiencies, comprising delivering to the central nervous system (CNS) a recombinant adeno-associated virus (rAAV) having an adeno-associated virus 1 (AAV1) capsid, said rAAV further comprising a vector genome packaged in the AAV capsid, said vector genome comprising AAV inverted terminal repeats, a coding sequence for human progranulin, and regulatory sequences which direct expression of the progranulin.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.