KR102620399B1 - Pcsk9 유전자 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 - Google Patents

Pcsk9 유전자 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 Download PDF

Info

Publication number
KR102620399B1
KR102620399B1 KR1020197033913A KR20197033913A KR102620399B1 KR 102620399 B1 KR102620399 B1 KR 102620399B1 KR 1020197033913 A KR1020197033913 A KR 1020197033913A KR 20197033913 A KR20197033913 A KR 20197033913A KR 102620399 B1 KR102620399 B1 KR 102620399B1
Authority
KR
South Korea
Prior art keywords
leu
lys
ser
val
ala
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.)
Active
Application number
KR1020197033913A
Other languages
English (en)
Korean (ko)
Other versions
KR20190140982A (ko
Inventor
빅터 바체비치
데릭 잔츠
제임스 제퍼슨 스미스
재널 레이프
Original Assignee
프리시젼 바이오사이언시스 인코포레이티드
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 프리시젼 바이오사이언시스 인코포레이티드 filed Critical 프리시젼 바이오사이언시스 인코포레이티드
Priority to KR1020237041504A priority Critical patent/KR20230170125A/ko
Publication of KR20190140982A publication Critical patent/KR20190140982A/ko
Application granted granted Critical
Publication of KR102620399B1 publication Critical patent/KR102620399B1/ko
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • 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
    • 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)
    • 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)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (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)
KR1020197033913A 2017-04-21 2018-04-20 Pcsk9 유전자 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제 Active KR102620399B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020237041504A KR20230170125A (ko) 2017-04-21 2018-04-20 Pcsk9 유전자 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제

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
KR1020237041504A Division KR20230170125A (ko) 2017-04-21 2018-04-20 Pcsk9 유전자 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제

Publications (2)

Publication Number Publication Date
KR20190140982A KR20190140982A (ko) 2019-12-20
KR102620399B1 true KR102620399B1 (ko) 2024-01-04

Family

ID=62117064

Family Applications (2)

Application Number Title Priority Date Filing Date
KR1020237041504A Pending KR20230170125A (ko) 2017-04-21 2018-04-20 Pcsk9 유전자 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제
KR1020197033913A Active KR102620399B1 (ko) 2017-04-21 2018-04-20 Pcsk9 유전자 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제

Family Applications Before (1)

Application Number Title Priority Date Filing Date
KR1020237041504A Pending KR20230170125A (ko) 2017-04-21 2018-04-20 Pcsk9 유전자 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제

Country Status (11)

Country Link
US (4) US11680254B2 (https=)
EP (2) EP4269596A3 (https=)
JP (4) JP2020517255A (https=)
KR (2) KR20230170125A (https=)
CN (2) CN117264929A (https=)
AU (2) AU2018254576B2 (https=)
CA (1) CA3060112A1 (https=)
ES (1) ES2953153T3 (https=)
IL (3) IL301059B2 (https=)
MX (2) MX2019012452A (https=)
WO (1) WO2018195449A1 (https=)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020214724A1 (en) * 2019-04-15 2020-10-22 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
EP4142801A4 (en) * 2020-04-27 2024-09-25 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
US20230183664A1 (en) 2020-05-11 2023-06-15 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
MX2022014258A (es) 2020-05-12 2023-02-22 Univ Pennsylvania Composiciones para la reducción específica de un fármaco de la expresión de transgén.
JP2023550581A (ja) 2020-10-29 2023-12-04 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 Ａａｖカプシド及びそれを含有する組成物
JP7642809B2 (ja) * 2020-11-12 2025-03-10 プレシジョン バイオサイエンシズ，インク． ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ
WO2022232267A1 (en) 2021-04-27 2022-11-03 The Trustees Of The University Of Pennsylvania Porcine-derived adeno-associated virus capsids and uses thereof
MX2023012747A (es) * 2021-04-27 2024-01-05 Univ Pennsylvania Composiciones y métodos para el direccionamiento genético in vivo mediado por nucleasas para el tratamiento de trastornos genéticos.
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
GB2634837A (en) 2022-06-07 2025-04-23 Scribe Therapeutics Inc Compositions and methods for the targeting of PCSK9
US20260021205A1 (en) 2022-07-15 2026-01-22 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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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 (28)

* 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
US6559189B2 (en) 1999-04-28 2003-05-06 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
US6635676B2 (en) 1999-04-28 2003-10-21 Regents Of The University Of Michigan Non-toxic antimicrobial compositions and methods of use
US7767216B2 (en) 1999-04-28 2010-08-03 The Regents Of The University Of Michigan Antimicrobial compositions and methods of use
US6555674B2 (en) 2000-08-09 2003-04-29 Nsgene A/S JeT promoter
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP3202899B1 (en) 2003-01-28 2020-10-21 Cellectis 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
ES2582091T3 (es) * 2005-10-18 2016-09-09 Precision Biosciences Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas
ES2366974T3 (es) * 2006-05-05 2011-10-27 Isis Pharmaceuticals, Inc. Compuestos y procedimientos para modular la expresión de sglt2.
AU2013201688B2 (en) * 2006-11-07 2016-03-03 Merck Sharp & Dohme Llc Antagonists of PCSK9
AU2013203748C1 (en) * 2007-08-23 2017-05-25 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
EP2215223B1 (en) * 2007-10-31 2013-05-01 Precision Biosciences, Inc. Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
US7928189B2 (en) * 2008-05-05 2011-04-19 Ottawa Health Research Institute PCSK9 polypeptide fragment
EP2313498B1 (en) 2008-07-14 2017-03-15 Precision Biosciences, Inc. Recognition sequences for i-crei-derived meganucleases and uses thereof
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
EP2714936B1 (en) 2011-06-01 2018-12-12 Precision Biosciences, Inc. Methods and products for producing engineered mammalian cell lines with amplified transgenes
EP4331620A3 (en) * 2012-12-07 2024-12-04 Translate Bio, Inc. Lipidic nanoparticles for mrna delivery
RU2694686C2 (ru) * 2013-03-12 2019-07-16 Е.И.Дюпон Де Немур Энд Компани Способы идентификации вариантных сайтов распознавания для редкощепящих сконструированных средств для индукции двунитевого разрыва, и композиции с ними, и их применения
CN113057959B (zh) * 2014-02-11 2024-07-16 阿尔尼拉姆医药品有限公司 己酮糖激酶（KHK）iRNA组合物及其使用方法
EP3134431B1 (en) * 2014-04-25 2021-04-07 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
JP6816133B2 (ja) * 2015-10-05 2021-01-20 プレシジョン バイオサイエンシズ，インク． 改変ヒトｔ細胞受容体アルファ定常領域遺伝子を含む遺伝子改変細胞

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Also Published As

Publication number Publication date
US20250154481A1 (en) 2025-05-15
IL301059A (en) 2023-05-01
EP4269596A3 (en) 2024-02-14
WO2018195449A1 (en) 2018-10-25
CN117264929A (zh) 2023-12-22
MX2019012452A (es) 2020-08-03
ES2953153T3 (es) 2023-11-08
JP2023058509A (ja) 2023-04-25
CN110769845B (zh) 2023-08-15
AU2018254576A1 (en) 2019-11-07
AU2023201658A1 (en) 2023-06-22
US20240101986A1 (en) 2024-03-28
US20240101985A1 (en) 2024-03-28
MX2023001877A (es) 2023-03-10
IL301059B1 (en) 2025-12-01
EP3612205B9 (en) 2023-10-04
IL270020B1 (en) 2023-04-01
IL301059B2 (en) 2026-04-01
AU2018254576B2 (en) 2022-12-22
US20200131489A1 (en) 2020-04-30
CA3060112A1 (en) 2018-10-25
IL270020B2 (en) 2023-08-01
KR20230170125A (ko) 2023-12-18
EP3612205B1 (en) 2023-06-07
JP7214915B2 (ja) 2023-01-30
JP2020517255A (ja) 2020-06-18
US11680254B2 (en) 2023-06-20
EP3612205C0 (en) 2023-06-07
EP4269596A2 (en) 2023-11-01
IL270020A (https=) 2019-12-31
EP3612205A1 (en) 2020-02-26
JP2022188058A (ja) 2022-12-20
KR20190140982A (ko) 2019-12-20
JP2025066725A (ja) 2025-04-23
CN110769845A (zh) 2020-02-07
IL324738A (en) 2026-01-01

Similar Documents

Publication Publication Date Title
KR102620399B1 (ko) Pcsk9 유전자 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제
JP7642809B2 (ja) ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ
EP3704238A1 (en) Engineered nucleases that target human and canine factor viii genes as a treatment for hemophilia a
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
CA3172161A1 (en) Engineered meganucleases having specificity for a recognition sequence in the hydroxyacid oxidase 1 gene
AU2026202721A1 (en) Engineered meganucleases specific for recognition sequences in the PCSK9 gene
HK40103025A (en) Engineered meganucleases specific for recognition sequences in the pcsk9 gene
HK40016582B (en) Engineered meganucleases specific for recognition sequences in the pcsk9 gene
HK40016582A (en) Engineered meganucleases specific for recognition sequences in the pcsk9 gene
EP4150068A1 (en) Treatment of retinitis pigmentosa using improved engineered meganucleases
CA3235187A1 (en) Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20191118

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20210416

Comment text: Request for Examination of Application

A302 Request for accelerated examination
PA0302 Request for accelerated examination

Patent event date: 20221123

Patent event code: PA03022R01D

Comment text: Request for Accelerated Examination

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20230426

Patent event code: PE09021S01D

E701 Decision to grant or registration of patent right
PE0701 Decision of registration

Patent event code: PE07011S01D

Comment text: Decision to Grant Registration

Patent event date: 20230829

GRNT Written decision to grant
PR0701 Registration of establishment

Comment text: Registration of Establishment

Patent event date: 20231228

Patent event code: PR07011E01D

PR1002 Payment of registration fee

Payment date: 20231229

End annual number: 3

Start annual number: 1

PG1601 Publication of registration