CL2021001605A1 - Composiciones de agentes de iarn de proteínas precursoras amiloides (ppa) y métodos para su uso. - Google Patents

Composiciones de agentes de iarn de proteínas precursoras amiloides (ppa) y métodos para su uso.

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Publication number
CL2021001605A1
CL2021001605A1 CL2021001605A CL2021001605A CL2021001605A1 CL 2021001605 A1 CL2021001605 A1 CL 2021001605A1 CL 2021001605 A CL2021001605 A CL 2021001605A CL 2021001605 A CL2021001605 A CL 2021001605A CL 2021001605 A1 CL2021001605 A1 CL 2021001605A1
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CL
Chile
Prior art keywords
methods
app
rnai agent
precursor protein
amyloid precursor
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Application number
CL2021001605A
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English (en)
Inventor
Vasant Jadhav
Martin Maier
Kevin Fitzgerald
Stuart Milstein
Kirk Brown
Jayaprakash Nair
Mark Keating
Adam Castoreno
Patrick Haslett
Mangala Meenakshi Soundarapandian
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Alnylam Pharmaceuticals Inc
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Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of CL2021001605A1 publication Critical patent/CL2021001605A1/es

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    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/4711Alzheimer's disease; Amyloid plaque core protein
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
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  • Health & Medical Sciences (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La divulgación se refiere a agentes de ácido ribonucleico bicatenario (iARNbc) y composiciones dirigidas al gen de PPA, así como también métodos para inhibir la expresión de un gen de PPA y métodos para tratar sujetos con una enfermedad o trastorno asociados a PPA, tales como angiopatía amiloide cerebral (AAC) y enfermedad de Alzheimer familiar de inicio precoz (EAFIP o EAFp), usando dichos agentes y composiciones de iARNdc.
CL2021001605A 2018-12-19 2021-06-17 Composiciones de agentes de iarn de proteínas precursoras amiloides (ppa) y métodos para su uso. CL2021001605A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862781774P 2018-12-19 2018-12-19
US201962862472P 2019-06-17 2019-06-17
US201962928795P 2019-10-31 2019-10-31

Publications (1)

Publication Number Publication Date
CL2021001605A1 true CL2021001605A1 (es) 2021-12-24

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ID=71101909

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Application Number Title Priority Date Filing Date
CL2021001605A CL2021001605A1 (es) 2018-12-19 2021-06-17 Composiciones de agentes de iarn de proteínas precursoras amiloides (ppa) y métodos para su uso.

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US (2) US11034957B2 (es)
EP (1) EP3898979A4 (es)
JP (1) JP2022515193A (es)
KR (1) KR20210111780A (es)
CN (1) CN113454222A (es)
AU (1) AU2019405783A1 (es)
BR (1) BR112021011895A2 (es)
CA (1) CA3124090A1 (es)
CL (1) CL2021001605A1 (es)
CO (1) CO2021009163A2 (es)
CR (1) CR20210393A (es)
DO (1) DOP2021000126A (es)
IL (1) IL283852A (es)
MX (1) MX2021007570A (es)
PE (1) PE20211420A1 (es)
SG (1) SG11202105886QA (es)
TW (1) TW202039844A (es)
WO (1) WO2020132227A2 (es)

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WO2022165172A1 (en) * 2021-01-29 2022-08-04 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing amyloid precursor protein (app)
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WO2023173061A2 (en) * 2022-03-11 2023-09-14 University Of Massachusetts Oligonucleotides for app modulation
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Publication number Publication date
KR20210111780A (ko) 2021-09-13
JP2022515193A (ja) 2022-02-17
CR20210393A (es) 2021-10-27
EP3898979A2 (en) 2021-10-27
US20200339991A1 (en) 2020-10-29
DOP2021000126A (es) 2021-09-30
IL283852A (en) 2021-07-29
US11034957B2 (en) 2021-06-15
BR112021011895A2 (pt) 2021-09-08
EP3898979A4 (en) 2022-10-12
MX2021007570A (es) 2021-08-24
US20220002724A1 (en) 2022-01-06
WO2020132227A3 (en) 2020-07-30
PE20211420A1 (es) 2021-08-03
SG11202105886QA (en) 2021-07-29
CA3124090A1 (en) 2020-06-25
TW202039844A (zh) 2020-11-01
CN113454222A (zh) 2021-09-28
AU2019405783A1 (en) 2021-07-01
WO2020132227A2 (en) 2020-06-25
CO2021009163A2 (es) 2021-10-29

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