MY204487A - 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
MY204487A
MY204487A MYPI2019003394A MYPI2019003394A MY204487A MY 204487 A MY204487 A MY 204487A MY PI2019003394 A MYPI2019003394 A MY PI2019003394A MY PI2019003394 A MYPI2019003394 A MY PI2019003394A MY 204487 A MY204487 A MY 204487A
Authority
MY
Malaysia
Prior art keywords
compositions
methods
antitrypsin deficiency
treating alpha
antitrypsin
Prior art date
Application number
MYPI2019003394A
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 MY204487A publication Critical patent/MY204487A/en

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    • 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
    • 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
    • A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
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    • 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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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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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
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2320/00—Applications; Uses
    • 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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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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MYPI2019003394A 2016-12-22 2017-12-21 Compositions and methods for treating alpha-1 antitrypsin deficiency MY204487A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662438219P 2016-12-22 2016-12-22
PCT/US2017/067800 WO2018119182A1 (en) 2016-12-22 2017-12-21 Compositions and methods for treating alpha-1 antitrypsin deficiency

Publications (1)

Publication Number Publication Date
MY204487A true MY204487A (en) 2024-08-30

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Family Applications (1)

Application Number Title Priority Date Filing Date
MYPI2019003394A MY204487A (en) 2016-12-22 2017-12-21 Compositions and methods for treating alpha-1 antitrypsin deficiency

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US (3) 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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