JP2020517255A - Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ - Google Patents

Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ Download PDF

Info

Publication number
JP2020517255A
JP2020517255A JP2019556884A JP2019556884A JP2020517255A JP 2020517255 A JP2020517255 A JP 2020517255A JP 2019556884 A JP2019556884 A JP 2019556884A JP 2019556884 A JP2019556884 A JP 2019556884A JP 2020517255 A JP2020517255 A JP 2020517255A
Authority
JP
Japan
Prior art keywords
meganuclease
seq
engineered meganuclease
engineered
acid sequence
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2019556884A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020517255A5 (enExample
Inventor
バートサビチ，ビクター
ジャンツ，デレック
スミス，ジェームス，ジェファーソン
ラペ，ジャネル
Original Assignee
プレシジョン バイオサイエンシズ，インク．
プレシジョン バイオサイエンシズ，インク．
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 プレシジョン バイオサイエンシズ，インク．, プレシジョン バイオサイエンシズ，インク． filed Critical プレシジョン バイオサイエンシズ，インク．
Publication of JP2020517255A publication Critical patent/JP2020517255A/ja
Publication of JP2020517255A5 publication Critical patent/JP2020517255A5/ja
Priority to JP2022145178A priority Critical patent/JP7214915B2/ja
Pending legal-status Critical Current

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
    • 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
    • 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)
JP2019556884A 2017-04-21 2018-04-20 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ Pending JP2020517255A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022145178A JP7214915B2 (ja) 2017-04-21 2022-09-13 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762488403P 2017-04-21 2017-04-21
US62/488,403 2017-04-21
US201762516966P 2017-06-08 2017-06-08
US62/516,966 2017-06-08
PCT/US2018/028607 WO2018195449A1 (en) 2017-04-21 2018-04-20 Engineered meganucleases specific for recognition sequences in the pcsk9 gene

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022145178A Division JP7214915B2 (ja) 2017-04-21 2022-09-13 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ

Publications (2)

Publication Number Publication Date
JP2020517255A true JP2020517255A (ja) 2020-06-18
JP2020517255A5 JP2020517255A5 (enExample) 2021-05-27

Family

ID=62117064

Family Applications (4)

Application Number Title Priority Date Filing Date
JP2019556884A Pending JP2020517255A (ja) 2017-04-21 2018-04-20 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ
JP2022145178A Active JP7214915B2 (ja) 2017-04-21 2022-09-13 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ
JP2023005733A Pending JP2023058509A (ja) 2017-04-21 2023-01-18 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ
JP2024231599A Pending JP2025066725A (ja) 2017-04-21 2024-12-27 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ

Family Applications After (3)

Application Number Title Priority Date Filing Date
JP2022145178A Active JP7214915B2 (ja) 2017-04-21 2022-09-13 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ
JP2023005733A Pending JP2023058509A (ja) 2017-04-21 2023-01-18 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ
JP2024231599A Pending JP2025066725A (ja) 2017-04-21 2024-12-27 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ

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)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024519469A (ja) * 2021-04-27 2024-05-14 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 遺伝性障害の治療のためのインビボヌクレアーゼ媒介性遺伝子標的化のための組成物及び方法

Families Citing this family (13)

* Cited by examiner, † Cited by third party
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
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

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006516403A (ja) * 2003-01-28 2006-07-06 セレクティス カスタムメイドメガヌクレアーゼおよびその使用
JP2009511085A (ja) * 2005-10-18 2009-03-19 プレシジョン バイオサイエンシズ 配列特異性およびｄｎａ−結合親和度が変更された、合理設計メガヌクレアーゼ
JP2011501971A (ja) * 2007-10-31 2011-01-20 プレシジョン バイオサイエンシズ，インク． 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ
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
WO2015175642A2 (en) * 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease
JP2016524472A (ja) * 2013-06-17 2016-08-18 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
WO2017044649A1 (en) * 2015-09-08 2017-03-16 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases

Family Cites Families (23)

* Cited by examiner, † Cited by third party
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
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
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
US7928189B2 (en) * 2008-05-05 2011-04-19 Ottawa Health Research Institute PCSK9 polypeptide fragment
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
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
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

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006516403A (ja) * 2003-01-28 2006-07-06 セレクティス カスタムメイドメガヌクレアーゼおよびその使用
JP2009511085A (ja) * 2005-10-18 2009-03-19 プレシジョン バイオサイエンシズ 配列特異性およびｄｎａ−結合親和度が変更された、合理設計メガヌクレアーゼ
JP2011501971A (ja) * 2007-10-31 2011-01-20 プレシジョン バイオサイエンシズ，インク． 合理的に設計された、非パリンドローム認識配列を有する単鎖メガヌクレアーゼ
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
JP2016524472A (ja) * 2013-06-17 2016-08-18 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
WO2015175642A2 (en) * 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease
WO2017044649A1 (en) * 2015-09-08 2017-03-16 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024519469A (ja) * 2021-04-27 2024-05-14 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 遺伝性障害の治療のためのインビボヌクレアーゼ媒介性遺伝子標的化のための組成物及び方法

Also Published As

Publication number Publication date
JP2023058509A (ja) 2023-04-25
CN110769845A (zh) 2020-02-07
JP7214915B2 (ja) 2023-01-30
US20250154481A1 (en) 2025-05-15
WO2018195449A1 (en) 2018-10-25
IL301059B2 (en) 2026-04-01
CA3060112A1 (en) 2018-10-25
EP3612205B9 (en) 2023-10-04
IL270020B1 (en) 2023-04-01
KR20230170125A (ko) 2023-12-18
KR20190140982A (ko) 2019-12-20
US11680254B2 (en) 2023-06-20
MX2023001877A (es) 2023-03-10
IL301059A (en) 2023-05-01
JP2025066725A (ja) 2025-04-23
CN110769845B (zh) 2023-08-15
EP3612205C0 (en) 2023-06-07
JP2022188058A (ja) 2022-12-20
CN117264929A (zh) 2023-12-22
IL270020A (enExample) 2019-12-31
EP4269596A3 (en) 2024-02-14
IL270020B2 (en) 2023-08-01
AU2026202721A1 (en) 2026-04-30
AU2018254576B2 (en) 2022-12-22
EP3612205A1 (en) 2020-02-26
KR102620399B1 (ko) 2024-01-04
MX2019012452A (es) 2020-08-03
US20240101986A1 (en) 2024-03-28
EP3612205B1 (en) 2023-06-07
US20240101985A1 (en) 2024-03-28
AU2023201658A1 (en) 2023-06-22
IL301059B1 (en) 2025-12-01
US20200131489A1 (en) 2020-04-30
AU2018254576A1 (en) 2019-11-07
EP4269596A2 (en) 2023-11-01
ES2953153T3 (es) 2023-11-08
IL324738A (en) 2026-01-01

Similar Documents

Publication Publication Date Title
JP7214915B2 (ja) Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ
AU2026201532A1 (en) Engineered meganucleases specific for recognition sequences in the Hepatitis B virus genome
US20210300755A1 (en) Lipid nanoparticle compositions and methods for mrna delivery
JP2025172742A (ja) トランスサイレチン遺伝子における認識配列に対する特異性を有する操作されたメガヌクレアーゼ
WO2019089913A1 (en) Engineered nucleases that target human and canine factor viii genes as a treatment for hemophilia a
US20220119786A1 (en) Genetically-modified cells comprising a modified transferrin gene
US12595469B2 (en) Treatment of retinitis pigmentosa using improved engineered meganucleases
HK40103025A (en) Engineered meganucleases specific for recognition sequences in the pcsk9 gene
HK40016582A (en) Engineered meganucleases specific for recognition sequences in the pcsk9 gene
HK40016582B (en) Engineered meganucleases specific for recognition sequences in the pcsk9 gene
EA042452B1 (ru) Сконструированные мегануклеазы, специфичные к последовательностям распознавания в геноме вируса гепатита b

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210419

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210419

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20220222

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220315

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220519

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20221115