IL283852A - 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

Info

Publication number
IL283852A
IL283852A IL283852A IL28385221A IL283852A IL 283852 A IL283852 A IL 283852A IL 283852 A IL283852 A IL 283852A IL 28385221 A IL28385221 A IL 28385221A IL 283852 A IL283852 A IL 283852A
Authority
IL
Israel
Prior art keywords
rnai
amyloid
compositions
methods
precursor protein
Prior art date
Application number
IL283852A
Other languages
English (en)
Hebrew (he)
Inventor
Stuart Milstein
Kirk Brown
Jayaprakash Nair
Martin Maier
Vasant Jadhav
Mark Keating
Adam Castoreno
Patrick Haslett
Mangala Meenakshi Soundarapandian
Kevin Fitzgerald
Original Assignee
Alnylam Pharmaceuticals Inc
Stuart Milstein
Kirk Brown
Jayaprakash Nair
Martin Maier
Vasant Jadhav
Mark Keating
Adam Castoreno
Patrick Haslett
Mangala Meenakshi Soundarapandian
Kevin Fitzgerald
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Alnylam Pharmaceuticals Inc, Stuart Milstein, Kirk Brown, Jayaprakash Nair, Martin Maier, Vasant Jadhav, Mark Keating, Adam Castoreno, Patrick Haslett, Mangala Meenakshi Soundarapandian, Kevin Fitzgerald filed Critical Alnylam Pharmaceuticals Inc
Publication of IL283852A publication Critical patent/IL283852A/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
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • 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
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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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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  • Health & Medical Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
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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)
IL283852A 2018-12-19 2021-06-09 Amyloid precursor protein (app) rnai agent compositions and methods of use thereof IL283852A (en)

Applications Claiming Priority (4)

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
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
IL283852A true IL283852A (en) 2021-07-29

Family

ID=71101909

Family Applications (1)

Application Number Title Priority Date Filing Date
IL283852A IL283852A (en) 2018-12-19 2021-06-09 Amyloid precursor protein (app) rnai agent compositions and methods of use thereof

Country Status (20)

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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
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
CA3124090A1 (en) 2020-06-25
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
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