Title:
RNAI AGENTS OF MODULATING PLP1
Document Type and Number:
WIPO Patent Application WO/2023/183876
Kind Code:
A3
Abstract:
Provided are antisense agents, RNAi agents, pharmaceutical compositions, and methods of use for reducing the amount or activity of PLP1 RNA in a cell or subject, and in certain instances reducing the amount of proteolipid protein 1 in a cell or subject. In certain embodiments, also provided herein are oligomeric compounds and oligomeric duplexes for reducing the amount or activity of proteolipid protein 1 (PLP1) RNA in a cell or subject, and in certain instances reducing the amount of proteolipid protein 1 in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a leukodystrophy. Such symptoms and hallmarks include hypotonia, nystagmus, optic atrophy, respiratory distress, delay in motor function development, cognitive dysfunction, speech dysfunction, spasticity, ataxia, seizures, choreiform movements, and death. Such leukodystrophies include Pelizaeus-Merzbacher disease.
Inventors:
FREIER SUSAN (US)
Application Number:
PCT/US2023/064867
Publication Date:
November 02, 2023
Filing Date:
March 23, 2023
Export Citation:
Assignee:
IONIS PHARMACEUTICALS INC (US)
International Classes:
C12N15/113; C12N15/11
Domestic Patent References:
| WO2022031847A2 | 2022-02-10 |
Foreign References:
| US20070134655A1 | 2007-06-14 |
Attorney, Agent or Firm:
SCARR, Rebecca, B. et al. (US)
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