Title:
APP IRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING DISEASES CHARACTERIZED BY ENLARGED ENDOSOMES
Document Type and Number:
WIPO Patent Application WO/2023/039503
Kind Code:
A3
Abstract:
The disclosure relates to use of double stranded ribonucleic acid (dsRNAi) agents and compositions targeting an amyloid precursor protein (APP) gene, including methods of inhibiting expression of an APP gene and methods of treating subjects having a disease or disorder characterized by enlarged endosomes, e.g., Alzheimer's disease (AD) and Down syndrome (DS), particularly occurrences of such neurodegenerative diseases associated with one or more mutations in presenilin 1 (PSEN1), using such dsRNAi agents and compositions.
Inventors:
BROWN KIRK (US)
DANG LAN THI HOANG (US)
DANG LAN THI HOANG (US)
Application Number:
PCT/US2022/076159
Publication Date:
April 20, 2023
Filing Date:
September 09, 2022
Export Citation:
Assignee:
ALNYLAM PHARMACEUTICALS INC (US)
International Classes:
A61P25/28; C12N15/113; C07K14/47
Domestic Patent References:
| WO2020118056A1 | 2020-06-11 |
Foreign References:
| US20200339991A1 | 2020-10-29 | |||
| US20210108186A1 | 2021-04-15 | |||
| US20140193341A1 | 2014-07-10 | |||
| US20170143934A1 | 2017-05-25 | |||
| US20190030035A1 | 2019-01-31 |
Other References:
PARKER JOHN; MOZAFFAR TAHSEEN; MESSMORE ASHLYNN; DEIGNAN JOSHUA L.; KIMONIS VIRGINIA E.; RINGMAN JOHN M.: "Homozygosity for the A431E mutation inPSEN1presenting with a relatively aggressive phenotype", NEUROSCIENCE LETTERS, vol. 699, 23 April 2019 (2019-04-23), AMSTERDAM, NL , pages 195 - 198, XP085645889, ISSN: 0304-3940, DOI: 10.1016/j.neulet.2019.01.047
Attorney, Agent or Firm:
COWLES, Christopher R. (US)
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