CA3124090A1 - Amyloid precursor protein (app) rnai agent compositions and methods of use thereof - Google Patents

Amyloid precursor protein (app) rnai agent compositions and methods of use thereof Download PDF

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Publication number
CA3124090A1
CA3124090A1 CA3124090A CA3124090A CA3124090A1 CA 3124090 A1 CA3124090 A1 CA 3124090A1 CA 3124090 A CA3124090 A CA 3124090A CA 3124090 A CA3124090 A CA 3124090A CA 3124090 A1 CA3124090 A1 CA 3124090A1
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Prior art keywords
rnai agent
nucleotides
double stranded
nucleotide
app
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Pending
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CA3124090A
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English (en)
French (fr)
Inventor
Stuart Milstein
Kirk Brown
Jayaprakash Nair
Martin Maier
Vasant Jadhav
Mark Keating
Adam CASTORENO
Patrick HASLETT
Mangala Meenakshi Soundarapandian
Kevin Fitzgerald
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of CA3124090A1 publication Critical patent/CA3124090A1/en
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3124090A 2018-12-19 2019-12-19 Amyloid precursor protein (app) rnai agent compositions and methods of use thereof Pending CA3124090A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862781774P 2018-12-19 2018-12-19
US62/781,774 2018-12-19
US201962862472P 2019-06-17 2019-06-17
US62/862,472 2019-06-17
US201962928795P 2019-10-31 2019-10-31
US62/928,795 2019-10-31
PCT/US2019/067449 WO2020132227A2 (en) 2018-12-19 2019-12-19 AMYLOID PRECURSOR PROTEIN (APP) RNAi AGENT COMPOSITIONS AND METHODS OF USE THEREOF

Publications (1)

Publication Number Publication Date
CA3124090A1 true CA3124090A1 (en) 2020-06-25

Family

ID=71101909

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3124090A Pending CA3124090A1 (en) 2018-12-19 2019-12-19 Amyloid precursor protein (app) rnai agent compositions and methods of use thereof

Country Status (20)

Country Link
US (2) US11034957B2 (https=)
EP (1) EP3898979A4 (https=)
JP (2) JP2022515193A (https=)
KR (1) KR20210111780A (https=)
CN (2) CN113454222B (https=)
AU (1) AU2019405783B2 (https=)
BR (1) BR112021011895A2 (https=)
CA (1) CA3124090A1 (https=)
CL (1) CL2021001605A1 (https=)
CO (1) CO2021009163A2 (https=)
CR (1) CR20210393A (https=)
DO (2) DOP2021000126A (https=)
IL (1) IL283852A (https=)
MX (1) MX2021007570A (https=)
MY (1) MY206230A (https=)
PE (1) PE20211420A1 (https=)
PH (1) PH12021551482A1 (https=)
SG (1) SG11202105886QA (https=)
TW (1) TW202039844A (https=)
WO (1) WO2020132227A2 (https=)

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KR20210111780A (ko) 2021-09-13
IL283852A (en) 2021-07-29
WO2020132227A2 (en) 2020-06-25
TW202039844A (zh) 2020-11-01
BR112021011895A2 (pt) 2021-09-08
CN113454222B (zh) 2025-01-14
CR20210393A (es) 2021-10-27
EP3898979A2 (en) 2021-10-27
CN120041447A (zh) 2025-05-27
DOP2021000126A (es) 2021-09-30
DOP2025000233A (es) 2025-12-15
SG11202105886QA (en) 2021-07-29
CN113454222A (zh) 2021-09-28
PH12021551482A1 (en) 2022-04-11
US20220002724A1 (en) 2022-01-06
EP3898979A4 (en) 2022-10-12
AU2019405783A1 (en) 2021-07-01
MX2021007570A (es) 2021-08-24
JP2024113043A (ja) 2024-08-21
CL2021001605A1 (es) 2021-12-24
JP2022515193A (ja) 2022-02-17
MY206230A (en) 2024-12-05
WO2020132227A3 (en) 2020-07-30
US11034957B2 (en) 2021-06-15
US20200339991A1 (en) 2020-10-29
CO2021009163A2 (es) 2021-10-29
AU2019405783B2 (en) 2026-03-26
PE20211420A1 (es) 2021-08-03

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