KR20210125999A - 다논병 치료를 위한 유전자 요법 벡터 - Google Patents

다논병 치료를 위한 유전자 요법 벡터 Download PDF

Info

Publication number
KR20210125999A
KR20210125999A KR1020217022811A KR20217022811A KR20210125999A KR 20210125999 A KR20210125999 A KR 20210125999A KR 1020217022811 A KR1020217022811 A KR 1020217022811A KR 20217022811 A KR20217022811 A KR 20217022811A KR 20210125999 A KR20210125999 A KR 20210125999A
Authority
KR
South Korea
Prior art keywords
gene therapy
therapy vector
raav gene
thr
raav
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
KR1020217022811A
Other languages
English (en)
Korean (ko)
Inventor
안나히타 케라바라
라이 프라바카
가우라브 샤
로데릭 웡
나빈 야라만치
피라팁 프라툼수완
Original Assignee
스페이스크래프트 세븐, 엘엘씨
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 스페이스크래프트 세븐, 엘엘씨 filed Critical 스페이스크래프트 세븐, 엘엘씨
Publication of KR20210125999A publication Critical patent/KR20210125999A/ko
Pending legal-status Critical Current

Links

Images

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
    • 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
    • A61K48/0025—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 wherein the non-active part clearly interacts with the delivered nucleic acid
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/06—Antiarrhythmics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70596—Molecules with a "CD"-designation not provided for elsewhere
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/43—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-tag
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020217022811A 2019-02-12 2020-02-12 다논병 치료를 위한 유전자 요법 벡터 Pending KR20210125999A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962804521P 2019-02-12 2019-02-12
US62/804,521 2019-02-12
US201962934928P 2019-11-13 2019-11-13
US62/934,928 2019-11-13
PCT/US2020/017987 WO2020167996A1 (en) 2019-02-12 2020-02-12 Gene therapy vectors for treatment of danon disease

Publications (1)

Publication Number Publication Date
KR20210125999A true KR20210125999A (ko) 2021-10-19

Family

ID=72043957

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217022811A Pending KR20210125999A (ko) 2019-02-12 2020-02-12 다논병 치료를 위한 유전자 요법 벡터

Country Status (12)

Country Link
US (1) US20220143215A1 (https=)
EP (1) EP3924371A4 (https=)
JP (2) JP2022520232A (https=)
KR (1) KR20210125999A (https=)
CN (2) CN113508130A (https=)
AU (3) AU2020221842A1 (https=)
BR (1) BR112021015751A2 (https=)
CA (1) CA3128514A1 (https=)
IL (1) IL285238A (https=)
MX (1) MX2021009696A (https=)
SG (1) SG11202107744SA (https=)
WO (1) WO2020167996A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023048443A1 (ko) 2021-09-23 2023-03-30 주식회사 엘지에너지솔루션 고내열성 커넥터 및 이를 포함하는 배터리 모듈, 배터리 팩, 자동차

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3021572A1 (en) 2016-04-20 2017-10-26 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas Compositions and methods for enhanced gene expression of pklr
EP3697452A4 (en) 2017-10-16 2021-11-24 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. LENTIVIRAL VECTORS FOR ADMINISTRATION OF PKLR TO TREAT PYRUVATE KINASE DEFECTS
BR112020020841A2 (pt) 2018-04-11 2021-01-19 Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus Composições e métodos para transplante de célulastronco
CA3096293A1 (en) 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
WO2020014523A1 (en) * 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
JP2023538519A (ja) 2020-08-07 2023-09-08 スペースクラフト セブン リミテッド ライアビリティ カンパニー Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療
CA3201247A1 (en) * 2020-12-07 2022-06-16 Gaurav Shah Treatment of danon disease
CN114874990A (zh) * 2021-02-05 2022-08-09 中国科学院苏州纳米技术与纳米仿生研究所 一种功能化外泌体及其制备方法和应用
CN114457112A (zh) * 2022-02-07 2022-05-10 苏州市立医院 一种特异性神经靶向mr分子探针及其制备方法和应用
CN115975051B (zh) * 2022-11-17 2025-09-12 东南大学 靶向外泌体及制备方法、应用、药物和药物递送系统

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7985553B2 (en) * 2001-10-29 2011-07-26 Nathaniel Heintz Method for isolating cell type-specific mRNAs
US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US9434928B2 (en) * 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
AU2013221212B2 (en) * 2012-02-17 2018-08-09 The Children's Hospital Of Philadelphia AAV vector compositions and methods for gene transfer to cells, organs and tissues
DK3405215T3 (da) * 2016-01-19 2022-09-19 Univ California Fremgangsmåder til behandling af danon-sygdom og andre autofagilidelser
WO2017136536A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
US11713468B2 (en) * 2017-08-02 2023-08-01 Dna Twopointo Inc. DNA vectors and elements for sustained gene expression in eukaryotic cells
WO2020014523A1 (en) * 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
EP3833745A1 (en) * 2018-08-10 2021-06-16 REGENXBIO Inc. Scalable method for recombinant aav production

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023048443A1 (ko) 2021-09-23 2023-03-30 주식회사 엘지에너지솔루션 고내열성 커넥터 및 이를 포함하는 배터리 모듈, 배터리 팩, 자동차

Also Published As

Publication number Publication date
WO2020167996A1 (en) 2020-08-20
JP2022520232A (ja) 2022-03-29
EP3924371A4 (en) 2023-01-11
EP3924371A1 (en) 2021-12-22
CN120324641A (zh) 2025-07-18
JP2025063228A (ja) 2025-04-15
AU2025205318A1 (en) 2025-07-31
CA3128514A1 (en) 2020-08-20
BR112021015751A2 (pt) 2022-02-08
US20220143215A1 (en) 2022-05-12
AU2020221842A1 (en) 2021-08-12
MX2021009696A (es) 2021-09-23
IL285238A (en) 2021-09-30
AU2023201237B2 (en) 2025-04-10
AU2023201237A1 (en) 2023-04-13
CN113508130A (zh) 2021-10-15
SG11202107744SA (en) 2021-08-30

Similar Documents

Publication Publication Date Title
CN112512596B (zh) 治疗danon病的基因治疗载体
JP2025063228A (ja) ダノン病の治療のための遺伝子療法ベクター
KR102738752B1 (ko) 페닐케톤뇨증을 치료하기 위한 유전자 치료
AU2019346655B2 (en) Frataxin expression constructs having engineered promoters and methods of use thereof
KR20210103469A (ko) 재조합 바이러스 벡터 및 이를 생산하기 위한 핵산
JP7433360B2 (ja) Ｐａｈ遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法
AU2018352855B2 (en) Adeno-associated virus compositions for restoring HBB gene function and methods of use thereof
US20240033325A1 (en) Treatment of danon disease
KR20220098210A (ko) 간-특이적 유전자 요법 벡터를 이용한 유전성 혈관 부종의 치료
CN112041437A (zh) 用于恢复f8基因功能的腺相关病毒组合物和其使用方法
RU2822925C2 (ru) Векторы для генной терапии для лечения болезни данона
RU2808459C2 (ru) Геннотерапевтические векторы для лечения болезни данона
CN115484975A (zh) 用于ids基因转移的腺相关病毒组合物和其使用方法
RU2858472C2 (ru) Способ лечения болезни Данона
KR20210105390A (ko) 글리코겐 저장 질환을 치료하기 위한 방법 및 조성물
WO2026072872A1 (en) Treatment of danon disease
CN116669774A (zh) 用于治疗法布里病的组合物和方法
CN117836420A (zh) 重组tert编码病毒基因组和运载体

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

D21 Rejection of application intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

E13 Pre-grant limitation requested

Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

D21 Rejection of application intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902