JP7128190B2 - ウィルソン病を処置するための遺伝子療法 - Google Patents

ウィルソン病を処置するための遺伝子療法 Download PDF

Info

Publication number
JP7128190B2
JP7128190B2 JP2019535891A JP2019535891A JP7128190B2 JP 7128190 B2 JP7128190 B2 JP 7128190B2 JP 2019535891 A JP2019535891 A JP 2019535891A JP 2019535891 A JP2019535891 A JP 2019535891A JP 7128190 B2 JP7128190 B2 JP 7128190B2
Authority
JP
Japan
Prior art keywords
atp7b
copper
raav
mice
aav8
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
JP2019535891A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020514286A (ja
JP2020514286A5 (enExample
Inventor
ウイルソン，ジェームス・エム
シドレーン，ジェニー・アグネス
ゴヴィンダサミイ，ラクシュマナン
Original Assignee
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
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 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 filed Critical 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
Publication of JP2020514286A publication Critical patent/JP2020514286A/ja
Publication of JP2020514286A5 publication Critical patent/JP2020514286A5/ja
Application granted granted Critical
Publication of JP7128190B2 publication Critical patent/JP7128190B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

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
    • 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
    • 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
    • A61K48/005—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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Diabetes (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Obesity (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Environmental Sciences (AREA)
JP2019535891A 2016-12-30 2017-12-29 ウィルソン病を処置するための遺伝子療法 Active JP7128190B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662440659P 2016-12-30 2016-12-30
US62/440,659 2016-12-30
US201762473656P 2017-03-20 2017-03-20
US62/473,656 2017-03-20
PCT/US2017/068919 WO2018126116A1 (en) 2016-12-30 2017-12-29 Gene therapy for treating wilson's disease

Publications (3)

Publication Number Publication Date
JP2020514286A JP2020514286A (ja) 2020-05-21
JP2020514286A5 JP2020514286A5 (enExample) 2021-01-28
JP7128190B2 true JP7128190B2 (ja) 2022-08-30

Family

ID=62710046

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019535891A Active JP7128190B2 (ja) 2016-12-30 2017-12-29 ウィルソン病を処置するための遺伝子療法

Country Status (9)

Country Link
US (2) US11473106B2 (enExample)
EP (2) EP3562514A4 (enExample)
JP (1) JP7128190B2 (enExample)
KR (2) KR102604096B1 (enExample)
CN (1) CN110290814A (enExample)
BR (1) BR112019013245A2 (enExample)
CA (1) CA3048044A1 (enExample)
MX (1) MX2019007873A (enExample)
WO (1) WO2018126116A1 (enExample)

Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3048044A1 (en) 2016-12-30 2018-07-05 The Trustees Of The University Of Pennsylvania Gene therapy for treating wilson's disease
AU2018375192B2 (en) 2017-12-01 2023-11-09 Encoded Therapeutics, Inc. Engineered DNA binding proteins
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
WO2019152843A1 (en) 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
CA3113975A1 (en) 2018-10-01 2020-04-09 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
JP7532360B2 (ja) * 2018-11-16 2024-08-13 エンコーデッド セラピューティクス， インコーポレイテッド ウィルソン病を処置するための組成物および方法
SG11202106855YA (en) * 2019-01-04 2021-07-29 Ultragenyx Pharmaceutical Inc Gene therapy constructs for treating wilson disease
BR112021024055A2 (pt) 2019-05-29 2022-02-08 Encoded Therapeutics Inc Composições e métodos para a regulação seletiva de gene
CN111088285B (zh) * 2019-08-15 2021-12-28 北京锦篮基因科技有限公司 携带atp7b基因表达框及变异体的aav载体及应用
JP7451705B2 (ja) * 2019-11-26 2024-03-18 コリア リサーチ インスティテュート オブ バイオサイエンス アンド バイオテクノロジー タンパク質発現用ｍＲＮＡ構造体及びその用途
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
JP2024505257A (ja) 2021-02-01 2024-02-05 レジェンクスバイオ インコーポレーテッド 神経セロイドリポフスチン症の遺伝子治療
US20240307553A1 (en) * 2021-04-01 2024-09-19 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
CN115216481A (zh) * 2021-04-21 2022-10-21 四川至善唯新生物科技有限公司 一种表达量提高的截短型atp7b基因及其应用
CN118434854A (zh) * 2021-10-18 2024-08-02 逻辑生物治疗公司 用于治疗威尔森氏病的基因疗法
US20250320509A1 (en) * 2022-05-23 2025-10-16 The Johns Hopkins University Compositions and methods for treating wilson's disease
CN115216533A (zh) * 2022-06-30 2022-10-21 湖南家辉生物技术有限公司 一种用于诊断威尔逊病的生物标志物、扩增引物组、检测试剂及应用
WO2024183769A1 (en) * 2023-03-06 2024-09-12 Lingyi Biotech Co., Ltd. A truncated atp7b and the use thereof
WO2024193638A1 (en) * 2023-03-22 2024-09-26 Skyline Therapeutics (Shanghai) Co., Ltd. Recombinant aav for the gene therapy of wilson disease
US20240360431A1 (en) * 2023-04-30 2024-10-31 Sichuan Real&Best Biotech Co., Ltd. Engineered alpha-galactosidase a (a-gal a) peptides and functional variants thereof and associated methods of treating fabry disease
CN120882865A (zh) * 2023-07-28 2025-10-31 凌意(杭州)生物科技有限公司 用于治疗威尔逊病的多核苷酸
WO2025110984A1 (en) * 2023-11-21 2025-05-30 Hydrogene Therapeutics, Inc. Compositions and methods for treating wilson's disease
CN117947035A (zh) * 2024-03-25 2024-04-30 上海凌医生物科技有限公司 一种增强基因表达的启动子

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2005512533A (ja) 2001-12-17 2005-05-12 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 アデノ随伴ウイルス（ａａｖ）血清型８の配列
WO2016097218A1 (en) 2014-12-17 2016-06-23 Fundación Para La Investigación Mèdica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson's disease and other conditions

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2002350039A1 (en) 2001-10-29 2003-05-12 Renovis, Inc. Method for isolating cell-type specific mrnas
DE10156121A1 (de) 2001-11-16 2003-05-28 Wolfgang Stremmel Somatische Gentherapie mit Vektoren, die einen verkürzten metallsensitiven Promotor besitzen, zur Behandlung des Morbus Wilson sowie anderer Erkrankungen, die mit Veränderungen im Metallstoffwechsel einhergehen, wie z.B. Alzheimer-Erkrankung
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
US20080050357A1 (en) 2003-08-01 2008-02-28 Claes Gustafsson Systems and Methods for Antibody Engineering
CN102199626B (zh) 2003-09-30 2015-06-24 宾夕法尼亚大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
EP2553106A2 (en) 2010-03-29 2013-02-06 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US20150045284A1 (en) 2011-01-21 2015-02-12 Aarhus Universitet CRYSTAL STRUCTURE OF A TYPE IB P-TYPE ATPase
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
BR112014020325A2 (pt) 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
WO2013151663A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
SG11201704690TA (en) 2014-12-17 2017-07-28 Fundación Para La Investigación Mèdica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson disease
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
WO2017100676A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
CA3048044A1 (en) * 2016-12-30 2018-07-05 The Trustees Of The University Of Pennsylvania Gene therapy for treating wilson's disease
JP7532360B2 (ja) * 2018-11-16 2024-08-13 エンコーデッド セラピューティクス， インコーポレイテッド ウィルソン病を処置するための組成物および方法
SG11202106855YA (en) 2019-01-04 2021-07-29 Ultragenyx Pharmaceutical Inc Gene therapy constructs for treating wilson disease

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2005512533A (ja) 2001-12-17 2005-05-12 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 アデノ随伴ウイルス（ａａｖ）血清型８の配列
WO2016097218A1 (en) 2014-12-17 2016-06-23 Fundación Para La Investigación Mèdica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson's disease and other conditions

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
Biochemistry，2009年，Vol. 48，pp. 5573-5581
BLOOD，2013年，Vol. 121, No. 17，pp. 3335-3344
Human Molecular Genetics，2006年，Vol. 15, No. 7，pp. 1225-1236
JOURNAL OF HEPATOLOGY，2015年09月25日，2016, Vol. 64，pp. 419-426
Molecular Therapy，2008年，Vol. 16, No. 2，pp. 280-289

Also Published As

Publication number Publication date
JP2020514286A (ja) 2020-05-21
US12460228B2 (en) 2025-11-04
KR20190101410A (ko) 2019-08-30
EP4361277A3 (en) 2024-07-31
KR20230160411A (ko) 2023-11-23
KR102863181B1 (ko) 2025-09-23
BR112019013245A2 (pt) 2020-02-11
US11473106B2 (en) 2022-10-18
US20220389455A1 (en) 2022-12-08
EP4361277A2 (en) 2024-05-01
CN110290814A (zh) 2019-09-27
KR102604096B1 (ko) 2023-11-23
EP3562514A4 (en) 2020-08-05
EP3562514A1 (en) 2019-11-06
CA3048044A1 (en) 2018-07-05
US20190338310A1 (en) 2019-11-07
MX2019007873A (es) 2019-11-18
WO2018126116A1 (en) 2018-07-05

Similar Documents

Publication Publication Date Title
JP7128190B2 (ja) ウィルソン病を処置するための遺伝子療法
US20220313760A1 (en) Gene therapy for treating phenylketonuria
Murillo et al. Liver expression of a MiniATP7B gene results in long‐term restoration of copper homeostasis in a Wilson disease model in mice
RU2708318C2 (ru) Композиции и способы лечения mps1
US12385065B2 (en) Gene therapy for treating citrullenemia
JP2019513794A (ja) 血友病ａ治療に対する遺伝子療法
JP2022530824A (ja) ポンペ病の治療のために有用な組成物
JP2020537637A (ja) ムコ多糖症ｉｉ型を治療するための遺伝子療法
KR20230118075A (ko) 파브리병 치료를 위한 조성물 및 방법
WO2024079531A2 (en) Nucleic acid regulatory elements for gene expression in the liver and methods of use
KR20210071017A (ko) Gm1 강글리오시드증 치료에 유용한 조성물
JP2024517743A (ja) ウイルスベクター組成物及びその使用方法
KR20220145838A (ko) Gm1 강글리오사이드증을 치료하는 데 유용한 조성물
US20240408157A1 (en) Compositions and methods for treatment of fabry disease
CA3048038C (en) Gene therapy for treating phenylketonuria
CN116669774A (zh) 用于治疗法布里病的组合物和方法
Yamazaki et al. Enhanced osteoblastic differentiation of parietal bone in a novel murine model of mucopolysaccharidosis type II
Sirninger Design and testing of gene therapeutics for treating cystic fibrosis

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20201208

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20201208

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20211027

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220126

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220525

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220608

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220712

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20220720

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20220818

R150 Certificate of patent or registration of utility model

Ref document number: 7128190

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250