CN120843445A - 用于治疗半乳糖血症的基因疗法 - Google Patents

用于治疗半乳糖血症的基因疗法

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Publication number
CN120843445A
CN120843445A CN202510783197.9A CN202510783197A CN120843445A CN 120843445 A CN120843445 A CN 120843445A CN 202510783197 A CN202510783197 A CN 202510783197A CN 120843445 A CN120843445 A CN 120843445A
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CN
China
Prior art keywords
aav
promoter
polynucleotide
sequence
galt
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
CN202510783197.9A
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English (en)
Chinese (zh)
Inventor
斯科特·艾伦·卢伊勒
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.)
Nationwide Childrens Hospital Inc
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Nationwide Childrens Hospital 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 Nationwide Childrens Hospital Inc filed Critical Nationwide Childrens Hospital Inc
Publication of CN120843445A publication Critical patent/CN120843445A/zh
Pending legal-status Critical Current

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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • A61K38/162—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • 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
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    • 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
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    • C12N9/10—Transferases (2.)
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    • A—HUMAN NECESSITIES
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2267/03—Animal model, e.g. for test or diseases
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Immunology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
CN202510783197.9A 2018-08-30 2019-08-30 用于治疗半乳糖血症的基因疗法 Pending CN120843445A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862725225P 2018-08-30 2018-08-30
US62/725,225 2018-08-30
CN201980069706.4A CN112867798A (zh) 2018-08-30 2019-08-30 用于治疗半乳糖血症的基因疗法
PCT/US2019/049157 WO2020047472A1 (en) 2018-08-30 2019-08-30 Gene therapy for the treatment of galactosemia

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CN201980069706.4A Division CN112867798A (zh) 2018-08-30 2019-08-30 用于治疗半乳糖血症的基因疗法

Publications (1)

Publication Number Publication Date
CN120843445A true CN120843445A (zh) 2025-10-28

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CN202510783197.9A Pending CN120843445A (zh) 2018-08-30 2019-08-30 用于治疗半乳糖血症的基因疗法
CN201980069706.4A Pending CN112867798A (zh) 2018-08-30 2019-08-30 用于治疗半乳糖血症的基因疗法

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CN201980069706.4A Pending CN112867798A (zh) 2018-08-30 2019-08-30 用于治疗半乳糖血症的基因疗法

Country Status (11)

Country Link
US (1) US20210299275A1 (https=)
EP (1) EP3844294A4 (https=)
JP (2) JP7688572B2 (https=)
KR (1) KR20210052469A (https=)
CN (2) CN120843445A (https=)
AU (2) AU2019328573A1 (https=)
BR (1) BR112021003174A2 (https=)
CA (1) CA3110290A1 (https=)
MX (1) MX2021002041A (https=)
SG (1) SG11202101819QA (https=)
WO (1) WO2020047472A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118373887A (zh) 2018-03-16 2024-07-23 国家儿童医院研究所 通过衣壳修饰增加组织特异性的基因递送
US20250001008A1 (en) * 2021-07-01 2025-01-02 Bridgebio Gene Therapy Research, Inc. Gene therapy for galactosemia
WO2023034940A2 (en) * 2021-09-01 2023-03-09 Jaguar Gene Therapy, Llc Adeno-associated vectors and virions to treat galactosemia and methods of use and manufacture
PE20241891A1 (es) * 2021-09-01 2024-09-19 Jaguar Gene Therapy Llc Vectores y viriones adenoasociados para tratar la galactosemia y metodos de uso y fabricacion

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US20020106381A1 (en) * 2000-06-13 2002-08-08 High Katherine A. Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus
US7795002B2 (en) * 2000-06-28 2010-09-14 Glycofi, Inc. Production of galactosylated glycoproteins in lower eukaryotes
WO2002088319A2 (en) * 2001-05-01 2002-11-07 Genstar Therapeutics Corp. Mini-adenoviral vector and methods of using same
AU2003253595A1 (en) * 2002-04-05 2003-11-03 The Children's Hospital Of Philadelphia Methods for the production of chimeric adeno-associated virus (aav) vectors, compositions of chimeric aav vectors, and methods of use thereof
SI3235827T1 (sl) * 2003-06-19 2021-05-31 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
WO2005117576A2 (en) * 2004-05-28 2005-12-15 University Of Pittsburgh Of The Commonwealth System Of Higher Education Modification of sugar metabolic process in transgenic cells, tissues and animals
US8663981B2 (en) * 2008-08-07 2014-03-04 Her Majesty The Queen In Right Of Canada As Represented By The Minister Of Health Optimized promoter sequence
US20110223635A1 (en) * 2008-08-11 2011-09-15 Karl Deisseroth Method and Composition for Controlling Gene Expression
CA2863964C (en) * 2012-02-07 2021-10-26 Global Bio Therapeutics Usa, Inc. Compartmentalized method of nucleic acid delivery and compositions and uses thereof
US10130649B2 (en) * 2013-03-15 2018-11-20 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
EP3452101A2 (en) * 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
WO2017201348A1 (en) * 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
JP7632835B2 (ja) * 2016-05-20 2025-02-19 プレジデント アンド フェローズ オブ ハーバード カレッジ 加齢関連疾患及び症状の遺伝子治療法

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MX2021002041A (es) 2021-07-21
AU2025283486A1 (en) 2026-01-29
JP2025004107A (ja) 2025-01-14
WO2020047472A1 (en) 2020-03-05
US20210299275A1 (en) 2021-09-30
BR112021003174A2 (pt) 2021-05-11
AU2019328573A1 (en) 2021-03-11
SG11202101819QA (en) 2021-03-30
CA3110290A1 (en) 2020-03-05
JP2021534766A (ja) 2021-12-16
JP7688572B2 (ja) 2025-06-04
CN112867798A (zh) 2021-05-28
EP3844294A1 (en) 2021-07-07
EP3844294A4 (en) 2022-06-01
KR20210052469A (ko) 2021-05-10

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