CN115461082A - 用于治疗遗传性黄斑变性的组合物和方法 - Google Patents

用于治疗遗传性黄斑变性的组合物和方法 Download PDF

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Publication number
CN115461082A
CN115461082A CN202180030798.2A CN202180030798A CN115461082A CN 115461082 A CN115461082 A CN 115461082A CN 202180030798 A CN202180030798 A CN 202180030798A CN 115461082 A CN115461082 A CN 115461082A
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transposase
ser
composition
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Chinese (zh)
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J·J·希金斯
S·麦克米兰
R·塔比比亚扎尔
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Saleo Gene Therapy
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Saleo Gene Therapy
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/007—Vector systems having a special element relevant for transcription cell cycle specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Biophysics (AREA)
  • Optics & Photonics (AREA)
  • Dermatology (AREA)
  • Mycology (AREA)
  • Nanotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202180030798.2A 2020-04-29 2021-04-29 用于治疗遗传性黄斑变性的组合物和方法 Pending CN115461082A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063017442P 2020-04-29 2020-04-29
US63/017,442 2020-04-29
PCT/US2021/030007 WO2021222654A1 (en) 2020-04-29 2021-04-29 Compositions and methods for treatment of inherited macular degeneration

Publications (1)

Publication Number Publication Date
CN115461082A true CN115461082A (zh) 2022-12-09

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CN202180030798.2A Pending CN115461082A (zh) 2020-04-29 2021-04-29 用于治疗遗传性黄斑变性的组合物和方法

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US (1) US20230193319A1 (https=)
EP (1) EP4142796A4 (https=)
JP (1) JP2023523622A (https=)
KR (1) KR20230004573A (https=)
CN (1) CN115461082A (https=)
AU (1) AU2021265838A1 (https=)
CA (1) CA3175197A1 (https=)
IL (1) IL297718A (https=)
WO (1) WO2021222654A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025140418A1 (zh) * 2023-12-29 2025-07-03 上海朗昇生物科技有限公司 用于视网膜基因递送的重组腺相关病毒载体及其应用

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4146798A4 (en) 2020-05-04 2025-02-12 Saliogen Therapeutics, Inc. TRANSPOSITION-BASED THERAPIES
WO2023160454A1 (zh) * 2022-02-25 2023-08-31 北京中因科技有限公司 表达盒组合及其应用
EP4511496A2 (en) * 2022-04-18 2025-02-26 Abeona Therapeutics Inc. Compositions and methods for treating dominant optic atrophy and x-linked retinoschisis
WO2025147466A1 (en) * 2024-01-02 2025-07-10 Alkeus Pharmaceuticals, Inc. Treatment for asymptomatic stargardt disease patients
WO2025188836A1 (en) * 2024-03-05 2025-09-12 Saliogen Therapeutics, Inc. Compositions and methods of using gene coding for treating ocular disease

Citations (8)

* Cited by examiner, † Cited by third party
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US20070042462A1 (en) * 2005-08-21 2007-02-22 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
US20090011040A1 (en) * 2007-05-02 2009-01-08 Naash Muna I Use of compacted nucleic acid nanoparticles in non-viral treatments of ocular diseases
CN101426534A (zh) * 2004-02-09 2009-05-06 G·伊南纳 检测和治疗视网膜疾病的方法和组合物
US20090214478A1 (en) * 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
US20110311506A1 (en) * 2009-02-25 2011-12-22 The Johns Hopkins University Piggybac transposon variants and methods of use
US20160076054A1 (en) * 2013-04-18 2016-03-17 Fondazione Telethon Effective delivery of large genes by dual aav vectors
US20170216456A1 (en) * 2014-03-21 2017-08-03 The Sydney Children's Hospitals Network (Randwick and Westmead)(Incorporating the Royal Alexandra Stable Gene Transfer to Proliferating Cells
WO2020077360A1 (en) * 2018-10-12 2020-04-16 Poseida Therapeutics, Inc. Hematopoietic stem cell compositions, methods of making and method of use

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101873852A (zh) * 2007-09-27 2010-10-27 锐视医药公司 治疗视黄醇相关性疾病的方法和化合物
US20150141320A1 (en) * 2012-05-16 2015-05-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
JP7036723B2 (ja) * 2015-12-14 2022-03-15 コールド スプリング ハーバー ラボラトリー 眼疾患の処置のための組成物および方法
EP3687580A1 (en) * 2017-09-27 2020-08-05 Sigilon Therapeutics, Inc. Methods, compositions, and implantable elements comprising active cells

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101426534A (zh) * 2004-02-09 2009-05-06 G·伊南纳 检测和治疗视网膜疾病的方法和组合物
US20070042462A1 (en) * 2005-08-21 2007-02-22 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
US20090011040A1 (en) * 2007-05-02 2009-01-08 Naash Muna I Use of compacted nucleic acid nanoparticles in non-viral treatments of ocular diseases
US20090214478A1 (en) * 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
US20110311506A1 (en) * 2009-02-25 2011-12-22 The Johns Hopkins University Piggybac transposon variants and methods of use
US20160076054A1 (en) * 2013-04-18 2016-03-17 Fondazione Telethon Effective delivery of large genes by dual aav vectors
US20170216456A1 (en) * 2014-03-21 2017-08-03 The Sydney Children's Hospitals Network (Randwick and Westmead)(Incorporating the Royal Alexandra Stable Gene Transfer to Proliferating Cells
WO2020077360A1 (en) * 2018-10-12 2020-04-16 Poseida Therapeutics, Inc. Hematopoietic stem cell compositions, methods of making and method of use

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025140418A1 (zh) * 2023-12-29 2025-07-03 上海朗昇生物科技有限公司 用于视网膜基因递送的重组腺相关病毒载体及其应用

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EP4142796A4 (en) 2024-06-26
AU2021265838A1 (en) 2022-10-20
IL297718A (en) 2022-12-01
US20230193319A1 (en) 2023-06-22
CA3175197A1 (en) 2021-11-04
KR20230004573A (ko) 2023-01-06
JP2023523622A (ja) 2023-06-06
WO2021222654A1 (en) 2021-11-04
EP4142796A1 (en) 2023-03-08

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Application publication date: 20221209