MX2019012452A - Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9. - Google Patents
Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9.Info
- Publication number
- MX2019012452A MX2019012452A MX2019012452A MX2019012452A MX2019012452A MX 2019012452 A MX2019012452 A MX 2019012452A MX 2019012452 A MX2019012452 A MX 2019012452A MX 2019012452 A MX2019012452 A MX 2019012452A MX 2019012452 A MX2019012452 A MX 2019012452A
- Authority
- MX
- Mexico
- Prior art keywords
- engineered meganucleases
- pcsk9 gene
- recognition sequences
- engineered
- hypercholesterolemia
- Prior art date
Links
Classifications
-
- 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 [RNase]; Deoxyribonucleases [DNase]
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
-
- 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
- A61K9/48—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
- A61K9/5123—Organic compounds, e.g. fats, sugars
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
-
- 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
- C12N15/63—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
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- 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
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Hematology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Obesity (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Optics & Photonics (AREA)
- Nanotechnology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762488403P | 2017-04-21 | 2017-04-21 | |
| US201762516966P | 2017-06-08 | 2017-06-08 | |
| PCT/US2018/028607 WO2018195449A1 (en) | 2017-04-21 | 2018-04-20 | Engineered meganucleases specific for recognition sequences in the pcsk9 gene |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2019012452A true MX2019012452A (es) | 2020-08-03 |
Family
ID=62117064
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2019012452A MX2019012452A (es) | 2017-04-21 | 2018-04-20 | Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9. |
| MX2023001877A MX2023001877A (es) | 2017-04-21 | 2019-10-17 | Meganucleasas dise?adas especificas para secuencias de reconocimiento en el gen pcsk9. |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2023001877A MX2023001877A (es) | 2017-04-21 | 2019-10-17 | Meganucleasas dise?adas especificas para secuencias de reconocimiento en el gen pcsk9. |
Country Status (11)
| Country | Link |
|---|---|
| US (4) | US11680254B2 (enExample) |
| EP (2) | EP4269596A3 (enExample) |
| JP (4) | JP2020517255A (enExample) |
| KR (2) | KR20230170125A (enExample) |
| CN (2) | CN117264929A (enExample) |
| AU (3) | AU2018254576B2 (enExample) |
| CA (1) | CA3060112A1 (enExample) |
| ES (1) | ES2953153T3 (enExample) |
| IL (3) | IL324738A (enExample) |
| MX (2) | MX2019012452A (enExample) |
| WO (1) | WO2018195449A1 (enExample) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20220298500A1 (en) * | 2019-04-15 | 2022-09-22 | The Trustees Of The University Of Pennsylvania | Compositions for regulating and self-inactivating enzyme expression and methods for modulating off-target activity of enzymes |
| CN114026228A (zh) | 2019-05-07 | 2022-02-08 | 精密生物科学公司 | 用于识别序列的工程化大范围核酸酶的优化 |
| US20230167464A1 (en) * | 2020-04-27 | 2023-06-01 | The Trustees Of The University Of Pennsylvania | Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity |
| US20230175014A1 (en) * | 2020-04-27 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity |
| WO2021231259A1 (en) | 2020-05-11 | 2021-11-18 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| IL298138A (en) | 2020-05-12 | 2023-01-01 | Univ Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| MX2023005113A (es) | 2020-10-29 | 2023-08-04 | Univ Pennsylvania | Cápsides de aav y composiciones que las contienen. |
| KR20250099412A (ko) * | 2020-11-12 | 2025-07-01 | 프리시젼 바이오사이언시스 인코포레이티드 | 디스트로핀 유전자 내의 인식 서열에 대해 특이성을 갖는 조작된 메가뉴클레아제 |
| TW202309066A (zh) | 2021-04-27 | 2023-03-01 | 賓州大學委員會 | 衍生自豬的腺相關病毒衣殼及其用途 |
| CA3216285A1 (en) * | 2021-04-27 | 2022-11-03 | Lili Wang | Compositions and methods for in vivo nuclease-mediated gene targeting for the treatment of genetic disorders |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| CN119998446A (zh) | 2022-06-07 | 2025-05-13 | 斯克里贝治疗公司 | 用于靶向pcsk9的组合物和方法 |
| WO2024015966A2 (en) | 2022-07-15 | 2024-01-18 | The Trustees Of The University Of Pennsylvania | Recombinant aav having aav clade d and clade e capsids and compositions containing same |
| WO2025122552A1 (en) * | 2023-12-04 | 2025-06-12 | The Trustees Of The University Of Pennsylvania | In silico prediction tool for meganuclease off-target sites |
Family Cites Families (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4873192A (en) | 1987-02-17 | 1989-10-10 | The United States Of America As Represented By The Department Of Health And Human Services | Process for site specific mutagenesis without phenotypic selection |
| US5652224A (en) * | 1995-02-24 | 1997-07-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism |
| US6015832A (en) | 1997-12-31 | 2000-01-18 | The Regents Of The University Of Michigan | Methods of inactivating bacteria including bacterial spores |
| US7767216B2 (en) | 1999-04-28 | 2010-08-03 | The Regents Of The University Of Michigan | Antimicrobial compositions and methods of use |
| US6635676B2 (en) | 1999-04-28 | 2003-10-21 | Regents Of The University Of Michigan | Non-toxic antimicrobial compositions and methods of use |
| US6506803B1 (en) | 1999-04-28 | 2003-01-14 | Regents Of The University Of Michigan | Methods of preventing and treating microbial infections |
| US6559189B2 (en) | 1999-04-28 | 2003-05-06 | Regents Of The University Of Michigan | Non-toxic antimicrobial compositions and methods of use |
| US6555674B2 (en) | 2000-08-09 | 2003-04-29 | Nsgene A/S | JeT promoter |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| US20060206949A1 (en) | 2003-01-28 | 2006-09-14 | Sylvain Arnould | Custom-made meganuclease and use thereof |
| US7404969B2 (en) | 2005-02-14 | 2008-07-29 | Sirna Therapeutics, Inc. | Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules |
| DK3246403T3 (da) | 2005-10-18 | 2020-10-26 | Prec Biosciences | Rationelt designede meganucleaser med ændret sekvens-specificitet og dna-bindende affinitet |
| ES2366973T3 (es) * | 2006-05-05 | 2011-10-27 | Isis Pharmaceuticals, Inc. | Compuestos y procedimiento para modular la expresión génica. |
| AU2013201688B2 (en) * | 2006-11-07 | 2016-03-03 | Merck Sharp & Dohme Llc | Antagonists of PCSK9 |
| AU2013203725A1 (en) * | 2007-08-23 | 2013-05-02 | Amgen Inc. | Antigen Binding Proteins to Proprotein Convertase Subtilisin Kexin Type 9 (PCSK9) |
| CA2603615A1 (en) | 2007-09-21 | 2009-03-21 | Patrick Labonte | Methods of reducing viral infection and kits therefore |
| ES2422291T3 (es) | 2007-10-31 | 2013-09-10 | Prec Biosciences Inc | Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas |
| US7928189B2 (en) * | 2008-05-05 | 2011-04-19 | Ottawa Health Research Institute | PCSK9 polypeptide fragment |
| JP2011527906A (ja) | 2008-07-14 | 2011-11-10 | プレシジョン バイオサイエンシズ，インク． | Ｉ−ＣｒｅＩ由来メガヌクレアーゼの認識配列およびその使用 |
| EP2180058A1 (en) * | 2008-10-23 | 2010-04-28 | Cellectis | Meganuclease recombination system |
| AU2013273674B2 (en) * | 2009-09-03 | 2015-11-05 | Pfizer Vaccines Llc | PCSK9 vaccine |
| EP3683319A1 (en) | 2011-06-01 | 2020-07-22 | Precision Biosciences, Inc. | Methods and products for producing engineered mammalian cell lines with amplified transgenes |
| US20150366997A1 (en) * | 2012-12-07 | 2015-12-24 | Shire Human Genetics Therapies, Inc. | COMPOSITIONS AND METHODS FOR mRNA DELIVERY |
| CN105189788B (zh) * | 2013-03-12 | 2018-07-13 | 纳幕尔杜邦公司 | 用于鉴定罕见切割工程化双链断裂诱导剂的变体识别位点的方法及其组合物和用途 |
| JP6738728B2 (ja) * | 2013-06-17 | 2020-08-19 | ザ・ブロード・インスティテュート・インコーポレイテッド | 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用 |
| EP3105331B1 (en) * | 2014-02-11 | 2021-06-23 | Alnylam Pharmaceuticals, Inc. | Ketohexokinase (khk) irna compositions and methods of use thereof |
| US11555059B2 (en) * | 2014-04-25 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | LDLR variants and their use in compositions for reducing cholesterol levels |
| WO2015175642A2 (en) * | 2014-05-13 | 2015-11-19 | Sangamo Biosciences, Inc. | Methods and compositions for prevention or treatment of a disease |
| CA2997909A1 (en) * | 2015-09-08 | 2017-03-16 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using engineered meganucleases |
| ES2803728T3 (es) * | 2015-10-05 | 2021-01-29 | Prec Biosciences Inc | Células genéticamente modificadas que comprenden un gen de región constante alfa del receptor de linfocitos T humano modificado |
-
2018
- 2018-04-20 EP EP23172361.0A patent/EP4269596A3/en active Pending
- 2018-04-20 KR KR1020237041504A patent/KR20230170125A/ko active Pending
- 2018-04-20 CN CN202311029425.0A patent/CN117264929A/zh active Pending
- 2018-04-20 IL IL324738A patent/IL324738A/en unknown
- 2018-04-20 KR KR1020197033913A patent/KR102620399B1/ko active Active
- 2018-04-20 US US16/606,856 patent/US11680254B2/en active Active
- 2018-04-20 MX MX2019012452A patent/MX2019012452A/es unknown
- 2018-04-20 AU AU2018254576A patent/AU2018254576B2/en active Active
- 2018-04-20 ES ES18723219T patent/ES2953153T3/es active Active
- 2018-04-20 EP EP18723219.4A patent/EP3612205B9/en active Active
- 2018-04-20 WO PCT/US2018/028607 patent/WO2018195449A1/en not_active Ceased
- 2018-04-20 JP JP2019556884A patent/JP2020517255A/ja active Pending
- 2018-04-20 CA CA3060112A patent/CA3060112A1/en active Pending
- 2018-04-20 IL IL301059A patent/IL301059B2/en unknown
- 2018-04-20 CN CN201880038349.0A patent/CN110769845B/zh active Active
-
2019
- 2019-10-17 IL IL270020A patent/IL270020B2/en unknown
- 2019-10-17 MX MX2023001877A patent/MX2023001877A/es unknown
-
2022
- 2022-09-13 JP JP2022145178A patent/JP7214915B2/ja active Active
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2023
- 2023-01-18 JP JP2023005733A patent/JP2023058509A/ja active Pending
- 2023-03-16 AU AU2023201658A patent/AU2023201658A1/en not_active Abandoned
- 2023-05-04 US US18/312,334 patent/US20240101986A1/en not_active Abandoned
- 2023-05-04 US US18/312,328 patent/US20240101985A1/en not_active Abandoned
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2024
- 2024-09-26 US US18/898,073 patent/US20250154481A1/en active Pending
- 2024-12-27 JP JP2024231599A patent/JP2025066725A/ja active Pending
-
2026
- 2026-04-10 AU AU2026202721A patent/AU2026202721A1/en active Pending
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