KR20210052469A - 갈락토스혈증의 치료를 위한 유전자 요법 - Google Patents

갈락토스혈증의 치료를 위한 유전자 요법 Download PDF

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KR20210052469A
KR20210052469A KR1020217007850A KR20217007850A KR20210052469A KR 20210052469 A KR20210052469 A KR 20210052469A KR 1020217007850 A KR1020217007850 A KR 1020217007850A KR 20217007850 A KR20217007850 A KR 20217007850A KR 20210052469 A KR20210052469 A KR 20210052469A
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leu
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Korean (ko)
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스코트 알렌 로일러
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더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈
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    • C—CHEMISTRY; METALLURGY
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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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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
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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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07012—UDP-glucose--hexose-1-phosphate uridylyltransferase (2.7.7.12)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • 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
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    • 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
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0362—Animal model for lipid/glucose metabolism, e.g. obesity, type-2 diabetes
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    • C12N2750/14011—Parvoviridae
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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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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
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  • Epidemiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • 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)
KR1020217007850A 2018-08-30 2019-08-30 갈락토스혈증의 치료를 위한 유전자 요법 Pending KR20210052469A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862725225P 2018-08-30 2018-08-30
US62/725,225 2018-08-30
PCT/US2019/049157 WO2020047472A1 (en) 2018-08-30 2019-08-30 Gene therapy for the treatment of galactosemia

Publications (1)

Publication Number Publication Date
KR20210052469A true KR20210052469A (ko) 2021-05-10

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217007850A Pending KR20210052469A (ko) 2018-08-30 2019-08-30 갈락토스혈증의 치료를 위한 유전자 요법

Country Status (11)

Country Link
US (1) US20210299275A1 (https=)
EP (1) EP3844294A4 (https=)
JP (2) JP7688572B2 (https=)
KR (1) KR20210052469A (https=)
CN (2) CN112867798A (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
EP3765624A4 (en) * 2018-03-16 2022-05-25 Research Institute at Nationwide Children's Hospital Increasing tissue specific gene delivery by capsid modification
WO2023279108A1 (en) * 2021-07-01 2023-01-05 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
US20240358856A1 (en) * 2021-09-01 2024-10-31 Jaguar Gene Therapy, Llc Adeno-associated vectors and virions to treat galactosemia and methods of use and manufacture

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001096587A2 (en) * 2000-06-13 2001-12-20 The Children's Hospital Of Philadelphia 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
AU2002308540A1 (en) * 2001-05-01 2002-11-11 Genstar Therapeutics Corp. Mini-adenoviral vector and methods of using same
WO2003088899A2 (en) * 2002-04-05 2003-10-30 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
SI1633772T1 (sl) * 2003-06-19 2016-06-30 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
CA2733104C (en) * 2008-08-07 2015-07-14 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
JP6188728B2 (ja) * 2012-02-07 2017-08-30 グローバル・バイオ・セラピューティクス・インコーポレイテッドＧｌｏｂａｌ Ｂｉｏ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 核酸送達の区画化方法ならびにその組成物および使用
EP3388834B1 (en) * 2013-03-15 2020-04-15 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
US11001861B2 (en) * 2016-05-18 2021-05-11 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
MX2018014256A (es) * 2016-05-20 2019-08-16 Harvard College Metodos de terapia genica para enfermedades y condiciones relacionadas con la edad.

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Publication number Publication date
AU2019328573A1 (en) 2021-03-11
MX2021002041A (es) 2021-07-21
SG11202101819QA (en) 2021-03-30
US20210299275A1 (en) 2021-09-30
WO2020047472A1 (en) 2020-03-05
JP2025004107A (ja) 2025-01-14
EP3844294A1 (en) 2021-07-07
EP3844294A4 (en) 2022-06-01
CA3110290A1 (en) 2020-03-05
CN112867798A (zh) 2021-05-28
CN120843445A (zh) 2025-10-28
JP2021534766A (ja) 2021-12-16
BR112021003174A2 (pt) 2021-05-11
JP7688572B2 (ja) 2025-06-04
AU2025283486A1 (en) 2026-01-29

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