JP7724156B2 - Ｅｎｐｐ１またはｅｎｐｐ３の欠乏をともなう疾患の治療 - Google Patents

Ｅｎｐｐ１またはｅｎｐｐ３の欠乏をともなう疾患の治療

Info

Publication number
JP7724156B2
JP7724156B2 JP2021541712A JP2021541712A JP7724156B2 JP 7724156 B2 JP7724156 B2 JP 7724156B2 JP 2021541712 A JP2021541712 A JP 2021541712A JP 2021541712 A JP2021541712 A JP 2021541712A JP 7724156 B2 JP7724156 B2 JP 7724156B2
Authority
JP
Japan
Prior art keywords
enpp1
aav
enpp3
vector
calcification
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
JP2021541712A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2020150716A5 (https=
JP2022517435A (ja
JP2022517435A5 (https=
Inventor
ジャングルズ スティーヴン
ブラドック デメトリオス
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Yale University
Inozyme Pharma Inc
Original Assignee
Yale University
Inozyme Pharma Inc
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 Yale University, Inozyme Pharma Inc filed Critical Yale University
Publication of JP2022517435A publication Critical patent/JP2022517435A/ja
Publication of JPWO2020150716A5 publication Critical patent/JPWO2020150716A5/ja
Publication of JP2022517435A5 publication Critical patent/JP2022517435A5/ja
Priority to JP2025027352A priority Critical patent/JP2025084821A/ja
Application granted granted Critical
Publication of JP7724156B2 publication Critical patent/JP7724156B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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)
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/02—Breeding vertebrates
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/04—Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04001—Phosphodiesterase I (3.1.4.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/01—Hydrolases acting on acid anhydrides (3.6) in phosphorus-containing anhydrides (3.6.1)
    • C12Y306/01009—Nucleotide diphosphatase (3.6.1.9), i.e. nucleotide-pyrophosphatase
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/31—Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

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)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Environmental Sciences (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
JP2021541712A 2019-01-18 2020-01-20 Ｅｎｐｐ１またはｅｎｐｐ３の欠乏をともなう疾患の治療 Active JP7724156B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025027352A JP2025084821A (ja) 2019-01-18 2025-02-21 Ｅｎｐｐ１またはｅｎｐｐ３の欠乏をともなう疾患の治療

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962794450P 2019-01-18 2019-01-18
US62/794,450 2019-01-18
US201962821692P 2019-03-21 2019-03-21
US62/821,692 2019-03-21
US201962877044P 2019-07-22 2019-07-22
US62/877,044 2019-07-22
PCT/US2020/014296 WO2020150716A1 (en) 2019-01-18 2020-01-20 Treatment of diseases involving deficiency of enpp1 or enpp3

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025027352A Division JP2025084821A (ja) 2019-01-18 2025-02-21 Ｅｎｐｐ１またはｅｎｐｐ３の欠乏をともなう疾患の治療

Publications (4)

Publication Number Publication Date
JP2022517435A JP2022517435A (ja) 2022-03-08
JPWO2020150716A5 JPWO2020150716A5 (https=) 2023-01-31
JP2022517435A5 JP2022517435A5 (https=) 2023-01-31
JP7724156B2 true JP7724156B2 (ja) 2025-08-15

Family

ID=71613451

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021541712A Active JP7724156B2 (ja) 2019-01-18 2020-01-20 Ｅｎｐｐ１またはｅｎｐｐ３の欠乏をともなう疾患の治療
JP2025027352A Pending JP2025084821A (ja) 2019-01-18 2025-02-21 Ｅｎｐｐ１またはｅｎｐｐ３の欠乏をともなう疾患の治療

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025027352A Pending JP2025084821A (ja) 2019-01-18 2025-02-21 Ｅｎｐｐ１またはｅｎｐｐ３の欠乏をともなう疾患の治療

Country Status (11)

Country Link
US (2) US20210187067A1 (https=)
EP (1) EP3911153A4 (https=)
JP (2) JP7724156B2 (https=)
KR (1) KR20210142599A (https=)
CN (1) CN113631033B (https=)
AU (1) AU2020207967A1 (https=)
BR (1) BR112021013941A2 (https=)
CA (1) CA3126839A1 (https=)
IL (1) IL284699A (https=)
MX (1) MX2021008503A (https=)
WO (1) WO2020150716A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022076848A1 (en) * 2020-10-08 2022-04-14 Inozyme Pharma, Inc. Liver specific production of enpp1 or enpp3
AU2021383830A1 (en) * 2020-11-19 2023-06-22 Biomarin Pharmaceutical Inc. Treatment of enpp1 deficiency and abcc6 deficiency
WO2023049864A1 (en) * 2021-09-24 2023-03-30 Inozyme Pharma, Inc. Lyophilized enpp1 polypeptide formulations and uses thereof
KR20240167914A (ko) * 2022-04-04 2024-11-28 이노자임 파마, 인코포레이티드 Enpp1 결핍증 및 abcc6 결핍증의 치료
WO2025213139A1 (en) * 2024-04-04 2025-10-09 The General Hospital Corporation Enpp1 gene therapy for the treatment of vascular disease

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6894022B1 (en) * 1998-08-27 2005-05-17 Eidgenossische Technische Hochschule Zurich Growth factor modified protein matrices for tissue engineering
CA2462653C (en) * 2001-11-07 2016-06-07 Aya Jakobovits Nucleic acid and corresponding protein entitled 161p2f10b useful in treatment and detection of cancer
EP1736553A1 (en) * 2005-06-17 2006-12-27 Centre National De La Recherche Scientifique ENPP1 (PC-1) gene haplotype associated with the risk of obesity and type 2 diabetes and their applications
US8168586B1 (en) * 2007-11-21 2012-05-01 Celera Corporation Cancer targets and uses thereof
WO2010037408A1 (en) * 2008-09-30 2010-04-08 Curevac Gmbh Composition comprising a complexed (m)rna and a naked mrna for providing or enhancing an immunostimulatory response in a mammal and uses thereof
EP3470089A1 (en) * 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
CA2960912A1 (en) * 2014-09-16 2016-03-24 Universitat Autonoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
EP3234116B1 (en) * 2014-12-19 2021-09-08 Alexion Pharmaceuticals, Inc. Methods of treating tissue calcification
KR20250078598A (ko) * 2015-05-19 2025-06-02 예일 유니버시티 병리학적 석회화 병태를 치료하기 위한 조성물, 및 이의 사용 방법
BR112019002355A2 (pt) * 2016-08-05 2019-07-16 Yale University composições e métodos para prevenção de acidente vascular cerebral em pacientes pediátricos com anemia falciforme
EP3844280A4 (en) * 2018-08-31 2022-09-14 Yale University ENPP1 POLYPEPTIDES AND METHODS OF USE

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
BioDrugs，2017年，Vol.31，pp.317-334
Biotechnology and Bioengineering，2013年，Vol.110, No.4，pp.1164-1173
DIABETES，2005年，Vol.54，pp.367-372
NATURE COMMUNICATIONS，2015年，6:10006，pp.1-11，DOI: 10.1038/ncomms10006

Also Published As

Publication number Publication date
IL284699A (en) 2021-08-31
BR112021013941A2 (pt) 2021-09-21
CA3126839A1 (en) 2020-07-23
MX2021008503A (es) 2021-10-13
CN113631033A (zh) 2021-11-09
US20210187067A1 (en) 2021-06-24
EP3911153A4 (en) 2022-12-21
US20230031809A1 (en) 2023-02-02
CN113631033B (zh) 2024-10-15
JP2022517435A (ja) 2022-03-08
WO2020150716A1 (en) 2020-07-23
JP2025084821A (ja) 2025-06-03
EP3911153A1 (en) 2021-11-24
KR20210142599A (ko) 2021-11-25
AU2020207967A1 (en) 2021-07-29

Similar Documents

Publication Publication Date Title
JP7724156B2 (ja) Ｅｎｐｐ１またはｅｎｐｐ３の欠乏をともなう疾患の治療
US12257321B2 (en) Optimized mini-dystrophin genes and expression cassettes and their use
RU2743792C2 (ru) Модифицированные гены атаксии фридрейха и векторы для генной терапии
AU2017322377B2 (en) Acid-alpha glucosidase variants and uses thereof
JP2022528416A (ja) 組換えアデノ随伴ウイルス及びその使用
CN113316639A (zh) 用于治疗庞贝氏病的治疗性腺相关病毒
TW202144575A (zh) 使用ａａｖ及治療調配物之苯酮尿症治療
CA3136513A1 (en) Gene therapy of fibroblast growth factor 23 related hypophosphatemic diseases
CN112225793A (zh) 一种溶酶体靶向肽及其融合蛋白、携带融合蛋白编码序列的腺相关病毒载体及其应用
BR112021001498A2 (pt) vírus adeno-associado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav e métodos para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva)
CN114555808A (zh) 嵌合多肽及其用途
JP2023548758A (ja) Ｅｎｐｐ１またはｅｎｐｐ３の肝臓特異的生成
KR20240063110A (ko) 치료제의 역행성 관상 정맥 또는 정맥동 투여
KR20230029624A (ko) 유전자 치료를 위한 글루코스-6-포스파타제(G6Pase-a)를 코딩하는 벡터
US20240409955A1 (en) Auf1 combination therapies for treatment of muscle degenerative disease
JP2025523953A (ja) 肢帯型筋ジストロフィーのためのａｕｆ１遺伝子療法
JP2024528997A (ja) 放射線誘発性唾液機能低下を予防するためのａｑｐ１遺伝子療法
US20230064077A1 (en) Treatment of mucopolysaccharidosis iva
KR20220119056A (ko) 골관절염 치료 방법
WO2025132267A1 (en) Plasmids, transgenes, vectors and medical uses comprising tissue non-specific alkaline phosphatase
RU2780329C2 (ru) Варианты кислой альфа-глюкозидазы и их использование
HK40059690A (en) Therapeutic adeno-associated virus for treating pompe disease

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210728

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230120

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230120

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240104

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240404

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240604

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240704

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20241024

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250221

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: 20250617

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250715

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250804

R150 Certificate of patent or registration of utility model

Ref document number: 7724156

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150