ZA202007632B - Compositions and methods for treating alpha-1 antitrypsin deficiency - Google Patents

Compositions and methods for treating alpha-1 antitrypsin deficiency

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Publication number
ZA202007632B
ZA202007632B ZA2020/07632A ZA202007632A ZA202007632B ZA 202007632 B ZA202007632 B ZA 202007632B ZA 2020/07632 A ZA2020/07632 A ZA 2020/07632A ZA 202007632 A ZA202007632 A ZA 202007632A ZA 202007632 B ZA202007632 B ZA 202007632B
Authority
ZA
South Africa
Prior art keywords
compositions
methods
antitrypsin deficiency
treating alpha
alpha
Prior art date
Application number
ZA2020/07632A
Other languages
English (en)
Inventor
Walter Strapps
Shobu Odate
Reynald Michael Lescarbeau
Original Assignee
Intellia Therapeutics Inc
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 Intellia Therapeutics Inc filed Critical Intellia Therapeutics Inc
Publication of ZA202007632B publication Critical patent/ZA202007632B/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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
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    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
    • A—HUMAN NECESSITIES
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    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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    • A61P11/00—Drugs for disorders of the respiratory system
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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ZA2020/07632A 2016-12-22 2020-12-08 Compositions and methods for treating alpha-1 antitrypsin deficiency ZA202007632B (en)

Applications Claiming Priority (1)

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US201662438219P 2016-12-22 2016-12-22

Publications (1)

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ZA202007632B true ZA202007632B (en) 2023-03-29

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ZA2020/07632A ZA202007632B (en) 2016-12-22 2020-12-08 Compositions and methods for treating alpha-1 antitrypsin deficiency

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US (2) US11549107B2 (enExample)
EP (1) EP3559232A1 (enExample)
JP (2) JP7383478B2 (enExample)
KR (1) KR102551664B1 (enExample)
CN (2) CN117737062A (enExample)
AU (2) AU2017379073B2 (enExample)
BR (1) BR112019012825A2 (enExample)
CA (1) CA3047415A1 (enExample)
CO (1) CO2019007776A2 (enExample)
EA (1) EA201991455A1 (enExample)
IL (1) IL267448B2 (enExample)
MX (2) MX2019007594A (enExample)
MY (1) MY204487A (enExample)
PH (1) PH12019501457A1 (enExample)
SA (1) SA519402192B1 (enExample)
SG (1) SG10202106412RA (enExample)
TW (2) TW202509233A (enExample)
WO (1) WO2018119182A1 (enExample)
ZA (1) ZA202007632B (enExample)

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JP2023553935A (ja) 2020-12-11 2023-12-26 インテリア セラピューティクス，インコーポレイテッド 脱アミノ化を伴うゲノム編集のためのポリヌクレオチド、組成物、及び方法
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SG10202106412RA (en) 2021-07-29
CN110382697A (zh) 2019-10-25
TW201833331A (zh) 2018-09-16
MX2019007594A (es) 2019-12-16
MX2025002623A (es) 2025-04-02
NZ754739A (en) 2024-01-26
IL267448A (en) 2019-08-29
JP7383478B2 (ja) 2023-11-20
WO2018119182A1 (en) 2018-06-28
US12460205B2 (en) 2025-11-04
TW202509233A (zh) 2025-03-01
PH12019501457A1 (en) 2020-03-09
US11549107B2 (en) 2023-01-10
AU2017379073A1 (en) 2019-07-11
US20190316129A1 (en) 2019-10-17
EP3559232A1 (en) 2019-10-30
US20230212575A1 (en) 2023-07-06
IL267448B2 (en) 2025-09-01
EA201991455A1 (ru) 2020-01-15
MY204487A (en) 2024-08-30
CN110382697B (zh) 2023-12-29
TWI851534B (zh) 2024-08-11
CA3047415A1 (en) 2018-06-28
IL267448B1 (en) 2025-05-01
SA519402192B1 (ar) 2023-11-08
AU2024201464A1 (en) 2024-03-28
CN117737062A (zh) 2024-03-22
BR112019012825A2 (pt) 2019-11-26
KR102551664B1 (ko) 2023-07-05
AU2017379073B2 (en) 2023-12-14
CO2019007776A2 (es) 2019-10-09
KR20190100943A (ko) 2019-08-29
JP2020501582A (ja) 2020-01-23
JP2023075164A (ja) 2023-05-30

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