JP7707075B2 - 心臓保護的活性のあるタンパク質 - Google Patents

心臓保護的活性のあるタンパク質 Download PDF

Info

Publication number
JP7707075B2
JP7707075B2 JP2021564417A JP2021564417A JP7707075B2 JP 7707075 B2 JP7707075 B2 JP 7707075B2 JP 2021564417 A JP2021564417 A JP 2021564417A JP 2021564417 A JP2021564417 A JP 2021564417A JP 7707075 B2 JP7707075 B2 JP 7707075B2
Authority
JP
Japan
Prior art keywords
fam3c
vector
pharmaceutical composition
aav
cardiac
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
JP2021564417A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022530168A5 (https=
JP2022530168A (ja
Inventor
ジャッカ,マウロ
ルオッツィ,ジュリア
ボルトロッツィ,フランチェスカ
Original Assignee
インターナショナル センター フォー ジェネティック エンジニアリング アンド バイオテクノロジー
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 インターナショナル センター フォー ジェネティック エンジニアリング アンド バイオテクノロジー filed Critical インターナショナル センター フォー ジェネティック エンジニアリング アンド バイオテクノロジー
Publication of JP2022530168A publication Critical patent/JP2022530168A/ja
Publication of JP2022530168A5 publication Critical patent/JP2022530168A5/ja
Priority to JP2025029903A priority Critical patent/JP2025107171A/ja
Application granted granted Critical
Publication of JP7707075B2 publication Critical patent/JP7707075B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00Modified animals
    • A01K2207/20Animals treated with compounds which are neither proteins nor nucleic acids
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0393Animal model comprising a reporter system for screening tests
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/30Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biotechnology (AREA)
  • Cardiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Hospice & Palliative Care (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Environmental Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
JP2021564417A 2019-04-30 2020-04-30 心臓保護的活性のあるタンパク質 Active JP7707075B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025029903A JP2025107171A (ja) 2019-04-30 2025-02-27 心臓保護的活性のあるタンパク質

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1906052.4A GB201906052D0 (en) 2019-04-30 2019-04-30 Proteins with cardioprotective activity
GB1906052.4 2019-04-30
PCT/EP2020/062156 WO2020221906A1 (en) 2019-04-30 2020-04-30 Proteins with cardioprotective activity

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025029903A Division JP2025107171A (ja) 2019-04-30 2025-02-27 心臓保護的活性のあるタンパク質

Publications (3)

Publication Number Publication Date
JP2022530168A JP2022530168A (ja) 2022-06-27
JP2022530168A5 JP2022530168A5 (https=) 2023-05-12
JP7707075B2 true JP7707075B2 (ja) 2025-07-14

Family

ID=66589570

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021564417A Active JP7707075B2 (ja) 2019-04-30 2020-04-30 心臓保護的活性のあるタンパク質
JP2025029903A Pending JP2025107171A (ja) 2019-04-30 2025-02-27 心臓保護的活性のあるタンパク質

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025029903A Pending JP2025107171A (ja) 2019-04-30 2025-02-27 心臓保護的活性のあるタンパク質

Country Status (25)

Country Link
US (3) US12569574B2 (https=)
EP (2) EP4389211A3 (https=)
JP (2) JP7707075B2 (https=)
KR (1) KR20220027829A (https=)
CN (2) CN113795504B (https=)
AU (1) AU2020267029A1 (https=)
BR (1) BR112021021626A2 (https=)
CA (1) CA3137662A1 (https=)
DK (1) DK3962534T3 (https=)
ES (1) ES2975422T3 (https=)
FI (1) FI3962534T3 (https=)
GB (1) GB201906052D0 (https=)
HR (1) HRP20240441T1 (https=)
HU (1) HUE066324T2 (https=)
IL (1) IL287525A (https=)
LT (1) LT3962534T (https=)
MX (2) MX2021013298A (https=)
PL (1) PL3962534T3 (https=)
PT (1) PT3962534T (https=)
RS (1) RS65469B1 (https=)
SG (1) SG11202111267WA (https=)
SI (1) SI3962534T1 (https=)
SM (1) SMT202400151T1 (https=)
WO (1) WO2020221906A1 (https=)
ZA (2) ZA202107778B (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201906052D0 (en) 2019-04-30 2019-06-12 Int Centre For Genetic Engineering And Biotechnology Proteins with cardioprotective activity
WO2025253097A1 (en) * 2024-06-03 2025-12-11 Forcefield Therapeutics Limited Proteins with cardiopreservation activity

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002008284A2 (en) * 2000-07-20 2002-01-31 Genentech, Inc. Compositions and methods for the diagnosis and treatment of disorders involving angiogenesis
WO2004084800A2 (en) 2002-08-29 2004-10-07 Pfizer Products Inc. A3 receptor-mediated cardioprotective proteins and therapeutic and diagnostic methods of use
US7312079B1 (en) * 2005-10-06 2007-12-25 Lexicon Pharmaceuticals, Inc. Variants of FAM3C
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
WO2011094137A1 (en) * 2010-01-26 2011-08-04 Ngm Biopharmaceuticals, Inc. Methods of treating glucose metabolism disorders
US8609612B2 (en) 2010-02-12 2013-12-17 Ngm Biopharmaceuticals, Inc. Methods of treating glucose metabolism disorders
ES2777002T3 (es) 2011-09-30 2020-08-03 Somalogic Inc Predicción de eventos de riesgo cardiovascular y usos de la misma
ITRM20110685A1 (it) 2011-12-23 2013-06-24 Internat Ct For Genetic En Gineering And Microrna per la rigenerazione cardiaca attraverso l induzione della proliferazione dei miociti cardiaci
EP2981334A4 (en) 2013-04-03 2016-11-23 Univ California COMPOSITIONS AND METHODS FOR INHIBITING VIRAL ACTIVITY
US20170209621A1 (en) 2016-01-21 2017-07-27 The Board Of Trustees Of The Leland Stanford Junior University Use of chordin-like 1 or collagen vi with mesenchymal stem cells
KR102022021B1 (ko) 2018-01-10 2019-09-18 한국과학기술연구원 심혈관 질환의 진단을 위한 바이오마커 및 이의 용도
GB201906052D0 (en) 2019-04-30 2019-06-12 Int Centre For Genetic Engineering And Biotechnology Proteins with cardioprotective activity

Also Published As

Publication number Publication date
KR20220027829A (ko) 2022-03-08
US20220387625A1 (en) 2022-12-08
JP2022530168A (ja) 2022-06-27
US12121594B2 (en) 2024-10-22
SI3962534T1 (sl) 2024-08-30
PL3962534T3 (pl) 2024-06-10
LT3962534T (lt) 2024-05-27
SMT202400151T1 (it) 2024-05-14
US20220193260A1 (en) 2022-06-23
CN120518738A (zh) 2025-08-22
US12569574B2 (en) 2026-03-10
MX2021013298A (es) 2021-12-10
DK3962534T3 (da) 2024-04-15
ZA202309540B (en) 2025-12-17
CN113795504B (zh) 2025-05-06
EP3962534B1 (en) 2024-02-21
GB201906052D0 (en) 2019-06-12
AU2020267029A1 (en) 2021-12-23
EP3962534A1 (en) 2022-03-09
ES2975422T3 (es) 2024-07-05
RS65469B1 (sr) 2024-05-31
EP4389211A2 (en) 2024-06-26
JP2025107171A (ja) 2025-07-17
PT3962534T (pt) 2024-04-03
IL287525A (en) 2021-12-01
CA3137662A1 (en) 2020-11-05
EP4389211A3 (en) 2024-08-28
HRP20240441T1 (hr) 2024-06-21
FI3962534T3 (fi) 2024-04-17
BR112021021626A2 (pt) 2021-12-21
US20250057982A1 (en) 2025-02-20
SG11202111267WA (en) 2021-11-29
HUE066324T2 (hu) 2024-07-28
ZA202107778B (en) 2024-02-28
MX2026002607A (es) 2026-04-01
CN113795504A (zh) 2021-12-14
WO2020221906A1 (en) 2020-11-05

Similar Documents

Publication Publication Date Title
US20250057982A1 (en) Proteins with cardioprotective activity
EP3448437B1 (en) Evasion of neutralizing antibodies by a recombinant adeno-associated virus
EA038695B1 (ru) Способы и композиции для переноса генов по сосудистой сети
US7655467B2 (en) Compositions and methods for systemic nucleic acid sequence delivery
KR20190038583A (ko) 알데히드 데히드로게나제 결핍의 치료를 위한 유전자 요법
US20130287739A1 (en) Serca2 therapeutic compositions and methods of use
KR20230112672A (ko) 신경변성 질환을 위한 유전자 요법
KR20230035584A (ko) Dwarf 개방 판독 프레임을 위한 아데노-관련 바이러스 벡터
EP4410988A1 (en) An aav2-vector variant for targeted transfer of genes
HK40113022A (en) Proteins with cardioprotective activity
WO2020176732A1 (en) TREATMENT OF PULMONARY FIBROSIS WITH SERCA2a GENE THERAPY
HK40060308A (en) Proteins with cardioprotective activity
HK40060308B (en) Proteins with cardioprotective activity
WO2025253097A1 (en) Proteins with cardiopreservation activity
HK40077196A (en) Evasion of neutralizing antibodies by a recombinant adeno-associated virus
KR20250169529A (ko) 유전자 요법을 위한 Bcl2-관련 아타노진 3(BAG3)을 인코딩하는 핵산
EP1689230A2 (en) Compositions and methods for systemic nucleic acid sequence delivery

Legal Events

Date Code Title Description
RD01 Notification of change of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7426

Effective date: 20220823

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20220823

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230427

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230427

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240326

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240620

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240926

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20241217

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250227

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

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250702

R150 Certificate of patent or registration of utility model

Ref document number: 7707075

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150