SG10202106412RA - 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
SG10202106412RA
SG10202106412RA SG10202106412RA SG10202106412RA SG10202106412RA SG 10202106412R A SG10202106412R A SG 10202106412RA SG 10202106412R A SG10202106412R A SG 10202106412RA SG 10202106412R A SG10202106412R A SG 10202106412RA SG 10202106412R A SG10202106412R A SG 10202106412RA
Authority
SG
Singapore
Prior art keywords
compositions
methods
antitrypsin deficiency
treating alpha
alpha
Prior art date
Application number
SG10202106412RA
Inventor
Shobu Odate
Walter Strapps
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 SG10202106412RA publication Critical patent/SG10202106412RA/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
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • 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
    • A61K9/48—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • 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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    • C12N15/00—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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • 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 RNAses, DNAses
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
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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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  • General Health & Medical Sciences (AREA)
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  • Wood Science & Technology (AREA)
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  • Physics & Mathematics (AREA)
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  • Epidemiology (AREA)
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  • Plant Pathology (AREA)
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  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Dispersion Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
SG10202106412RA 2016-12-22 2017-12-21 Compositions and methods for treating alpha-1 antitrypsin deficiency SG10202106412RA (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201662438219P 2016-12-22 2016-12-22

Publications (1)

Publication Number Publication Date
SG10202106412RA true SG10202106412RA (en) 2021-07-29

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ID=61054487

Family Applications (1)

Application Number Title Priority Date Filing Date
SG10202106412RA SG10202106412RA (en) 2016-12-22 2017-12-21 Compositions and methods for treating alpha-1 antitrypsin deficiency

Country Status (18)

Country Link
US (2) US11549107B2 (en)
EP (1) EP3559232A1 (en)
JP (2) JP7383478B2 (en)
KR (1) KR102551664B1 (en)
CN (2) CN110382697B (en)
AU (2) AU2017379073B2 (en)
BR (1) BR112019012825A2 (en)
CA (1) CA3047415A1 (en)
CO (1) CO2019007776A2 (en)
EA (1) EA201991455A1 (en)
IL (1) IL267448A (en)
MX (1) MX2019007594A (en)
NZ (1) NZ754739A (en)
PH (1) PH12019501457A1 (en)
SG (1) SG10202106412RA (en)
TW (1) TW201833331A (en)
WO (1) WO2018119182A1 (en)
ZA (1) ZA202007632B (en)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ754739A (en) * 2016-12-22 2024-01-26 Intellia Therapeutics Inc Compositions and methods for treating alpha-1 antitrypsin deficiency
CA3077413A1 (en) * 2017-09-29 2019-04-04 Intellia Therapeutics, Inc. Formulations
WO2020006708A1 (en) * 2018-07-04 2020-01-09 Yale University Compositions and methods for enhancement of homology-directed repair mediated precise gene editing by programming dna repair with a single rna-guided endonuclease
WO2020082047A1 (en) 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiencey
JP7472121B2 (en) * 2018-10-18 2024-04-22 インテリア セラピューティクス，インコーポレーテッド Compositions and methods for transgene expression from the albumin locus
US20220160847A1 (en) * 2020-11-20 2022-05-26 Mark Egly Method of preventing and/or treating a plurality of diseases
EP4259792A1 (en) 2020-12-11 2023-10-18 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing involving deamination

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US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
DK1695979T3 (en) 1991-12-24 2011-10-10 Isis Pharmaceuticals Inc Gapped modified oligonucleotides
US6169169B1 (en) 1994-05-19 2001-01-02 Dako A/S PNA probes for detection of Neisseria gonorrhoeae and Chlamydia trachomatis
US7250496B2 (en) * 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
BR112015031608A2 (en) * 2013-06-17 2017-08-22 Massachusetts Inst Technology APPLICATION AND USE OF CRISPR-CAS SYSTEMS, VECTORS AND COMPOSITIONS FOR LIVER TARGETING AND THERAPY
EP3611268A1 (en) * 2013-08-22 2020-02-19 E. I. du Pont de Nemours and Company Plant genome modification using guide rna/cas endonuclease systems and methods of use
DK3066201T3 (en) * 2013-11-07 2018-06-06 Editas Medicine Inc CRISPR-RELATED PROCEDURES AND COMPOSITIONS WITH LEADING GRADES
US11053481B2 (en) * 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
KR20160102056A (en) * 2013-12-26 2016-08-26 더 제너럴 하스피탈 코포레이션 Multiplex guide rnas
JP7068821B2 (en) * 2014-12-03 2022-05-17 アジレント・テクノロジーズ・インク Guide RNA with chemical modification
CN107787367B (en) * 2015-04-06 2021-10-26 里兰斯坦福初级大学理事会 Chemically modified guide RNAs for CRISPR/CAS mediated gene regulation
AU2016338785B2 (en) * 2015-10-12 2022-07-14 E. I. Du Pont De Nemours And Company Protected DNA templates for gene modification and increased homologous recombination in cells and methods of use
MX2018005332A (en) * 2015-11-06 2018-11-09 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a.
JP6932698B2 (en) * 2015-12-01 2021-09-08 クリスパー・セラピューティクス・アクチェンゲゼルシャフトＣＲＩＳＰＲ Ｔｈｅｒａｐｅｕｔｉｃｓ ＡＧ Materials and Methods for the Treatment of Alpha-1 Antitrypsin Deficiency
EP3433364A1 (en) * 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
NZ754739A (en) * 2016-12-22 2024-01-26 Intellia Therapeutics Inc Compositions and methods for treating alpha-1 antitrypsin deficiency
WO2020082047A1 (en) * 2018-10-18 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiencey

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Publication number Publication date
NZ754739A (en) 2024-01-26
ZA202007632B (en) 2023-03-29
IL267448A (en) 2019-08-29
JP2020501582A (en) 2020-01-23
MX2019007594A (en) 2019-12-16
PH12019501457A1 (en) 2020-03-09
US11549107B2 (en) 2023-01-10
JP7383478B2 (en) 2023-11-20
WO2018119182A1 (en) 2018-06-28
TW201833331A (en) 2018-09-16
KR102551664B1 (en) 2023-07-05
EA201991455A1 (en) 2020-01-15
EP3559232A1 (en) 2019-10-30
CN117737062A (en) 2024-03-22
CA3047415A1 (en) 2018-06-28
AU2024201464A1 (en) 2024-03-28
US20230212575A1 (en) 2023-07-06
KR20190100943A (en) 2019-08-29
BR112019012825A2 (en) 2019-11-26
AU2017379073B2 (en) 2023-12-14
US20190316129A1 (en) 2019-10-17
CO2019007776A2 (en) 2019-10-09
JP2023075164A (en) 2023-05-30
AU2017379073A1 (en) 2019-07-11
CN110382697A (en) 2019-10-25
CN110382697B (en) 2023-12-29

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