BR112022015380A2 - Composições e métodos para edição de gene de calicreína (klkb1) - Google Patents
Composições e métodos para edição de gene de calicreína (klkb1)Info
- Publication number
- BR112022015380A2 BR112022015380A2 BR112022015380A BR112022015380A BR112022015380A2 BR 112022015380 A2 BR112022015380 A2 BR 112022015380A2 BR 112022015380 A BR112022015380 A BR 112022015380A BR 112022015380 A BR112022015380 A BR 112022015380A BR 112022015380 A2 BR112022015380 A2 BR 112022015380A2
- Authority
- BR
- Brazil
- Prior art keywords
- compositions
- methods
- klkb1
- gene
- kallykrein
- Prior art date
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
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- 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
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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- 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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Crystallography & Structural Chemistry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Fodder In General (AREA)
- Medicinal Preparation (AREA)
Abstract
COMPOSIÇÕES E MÉTODOS PARA EDIÇÃO DE GENE DE CALICREÍNA (KLKB1). Composições e métodos para editar, por exemplo, introduzir quebras de fita dupla, dentro do gene KLKB1 são fornecidos. Composições e métodos para tratar indivíduos tendo angioedema hereditário (HAE) são fornecidos.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202062971906P | 2020-02-07 | 2020-02-07 | |
US202062981965P | 2020-02-26 | 2020-02-26 | |
US202063019076P | 2020-05-01 | 2020-05-01 | |
PCT/US2021/016730 WO2021158858A1 (en) | 2020-02-07 | 2021-02-05 | Compositions and methods for kallikrein ( klkb1) gene editing |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112022015380A2 true BR112022015380A2 (pt) | 2022-09-27 |
Family
ID=74873798
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112022015380A BR112022015380A2 (pt) | 2020-02-07 | 2021-02-05 | Composições e métodos para edição de gene de calicreína (klkb1) |
Country Status (14)
Country | Link |
---|---|
US (2) | US20230295587A1 (pt) |
EP (1) | EP4100528A1 (pt) |
JP (1) | JP2023512758A (pt) |
KR (1) | KR20220139352A (pt) |
CN (1) | CN115362256A (pt) |
AU (1) | AU2021216451A1 (pt) |
BR (1) | BR112022015380A2 (pt) |
CA (1) | CA3169813A1 (pt) |
CL (1) | CL2022002095A1 (pt) |
CO (1) | CO2022012196A2 (pt) |
IL (1) | IL295280A (pt) |
MX (1) | MX2022009658A (pt) |
TW (1) | TW202146030A (pt) |
WO (1) | WO2021158858A1 (pt) |
Families Citing this family (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20230158059A1 (en) * | 2021-10-01 | 2023-05-25 | Adarx Pharmaceuticals, Inc. | Prekallikrein-modulating compositions and methods of use thereof |
WO2023086953A1 (en) * | 2021-11-11 | 2023-05-19 | Beam Therapeutics Inc. | Compositions and methods for the treatment of hereditary angioedema (hae) |
TW202346586A (zh) * | 2022-01-28 | 2023-12-01 | 大陸商上海舶望製藥有限公司 | 用於抑制前激肽釋放酶(pkk)蛋白表達的組合物和方法 |
TW202405169A (zh) * | 2022-07-08 | 2024-02-01 | 美商英特利亞醫療公司 | 活體內編輯klkb1之方法 |
WO2024012435A1 (en) * | 2022-07-11 | 2024-01-18 | CorrectSequence Therapeutics Co., Ltd | Gene editing systems and methods for treating hereditary angioedema |
Family Cites Families (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP3320922A1 (en) * | 2011-06-10 | 2018-05-16 | Ionis Pharmaceuticals, Inc. | Methods for modulating kallikrein (klkb1) expression |
EP3715457A3 (en) * | 2013-08-28 | 2020-12-16 | Ionis Pharmaceuticals, Inc. | Modulation of prekallikrein (pkk) expression |
ES2774968T3 (es) * | 2013-12-19 | 2020-07-23 | Novartis Ag | Lípidos y composiciones lipídicas para la administración de agentes activos |
EP3862362A3 (en) * | 2014-05-01 | 2021-10-27 | Ionis Pharmaceuticals, Inc. | Conjugates of modified antisense oligonucleotides and their use for modulating pkk expression |
TW202336231A (zh) * | 2015-05-06 | 2023-09-16 | 美商阿尼拉製藥公司 | 第十二因子(哈格曼因子)(F12)、激肽釋放素B、血漿(夫列契因子)1(KLKB1)及激肽原1(KNG1)iRNA組成物及其使用方法 |
WO2019010342A1 (en) * | 2017-07-07 | 2019-01-10 | Alnylam Pharmaceuticals, Inc. | METHODS OF TREATING OR PREVENTING DISEASES ASSOCIATED WITH THE CONTACT ACTIVATION PATHWAY USING FIBER XII-TARGETING RNAI COMPOSITIONS (FACTOR HAGEMAN) (F12) |
EP4054651A1 (en) * | 2019-11-08 | 2022-09-14 | Regeneron Pharmaceuticals, Inc. | Crispr and aav strategies for x-linked juvenile retinoschisis therapy |
-
2021
- 2021-02-05 JP JP2022547680A patent/JP2023512758A/ja active Pending
- 2021-02-05 KR KR1020227030659A patent/KR20220139352A/ko unknown
- 2021-02-05 MX MX2022009658A patent/MX2022009658A/es unknown
- 2021-02-05 EP EP21711973.4A patent/EP4100528A1/en active Pending
- 2021-02-05 CN CN202180026687.4A patent/CN115362256A/zh active Pending
- 2021-02-05 WO PCT/US2021/016730 patent/WO2021158858A1/en active Application Filing
- 2021-02-05 CA CA3169813A patent/CA3169813A1/en active Pending
- 2021-02-05 IL IL295280A patent/IL295280A/en unknown
- 2021-02-05 BR BR112022015380A patent/BR112022015380A2/pt unknown
- 2021-02-05 AU AU2021216451A patent/AU2021216451A1/en active Pending
- 2021-02-05 TW TW110104494A patent/TW202146030A/zh unknown
-
2022
- 2022-08-03 CL CL2022002095A patent/CL2022002095A1/es unknown
- 2022-08-05 US US17/882,099 patent/US20230295587A1/en active Pending
- 2022-08-26 CO CONC2022/0012196A patent/CO2022012196A2/es unknown
-
2023
- 2023-07-31 US US18/362,675 patent/US20240018496A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
MX2022009658A (es) | 2022-10-27 |
US20230295587A1 (en) | 2023-09-21 |
TW202146030A (zh) | 2021-12-16 |
AU2021216451A1 (en) | 2022-09-29 |
JP2023512758A (ja) | 2023-03-29 |
EP4100528A1 (en) | 2022-12-14 |
CL2022002095A1 (es) | 2023-03-10 |
US20240018496A1 (en) | 2024-01-18 |
IL295280A (en) | 2022-10-01 |
CO2022012196A2 (es) | 2022-09-09 |
CA3169813A1 (en) | 2021-08-12 |
WO2021158858A1 (en) | 2021-08-12 |
CN115362256A (zh) | 2022-11-18 |
KR20220139352A (ko) | 2022-10-14 |
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