IL297718A - Preparations and methods for the treatment of hereditary macular degeneration - Google Patents
Preparations and methods for the treatment of hereditary macular degenerationInfo
- Publication number
- IL297718A IL297718A IL297718A IL29771822A IL297718A IL 297718 A IL297718 A IL 297718A IL 297718 A IL297718 A IL 297718A IL 29771822 A IL29771822 A IL 29771822A IL 297718 A IL297718 A IL 297718A
- Authority
- IL
- Israel
- Prior art keywords
- transposase
- composition
- nucleic acid
- mlt
- abca4
- Prior art date
Links
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- 108091005703 transmembrane proteins Proteins 0.000 description 1
- SZYJELPVAFJOGJ-UHFFFAOYSA-N trimethylamine hydrochloride Chemical compound Cl.CN(C)C SZYJELPVAFJOGJ-UHFFFAOYSA-N 0.000 description 1
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Classifications
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- 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
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- A—HUMAN NECESSITIES
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- 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
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- A—HUMAN NECESSITIES
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- 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
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- A—HUMAN NECESSITIES
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- 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
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- 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
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- A—HUMAN NECESSITIES
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- 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
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- A—HUMAN NECESSITIES
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- A—HUMAN NECESSITIES
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- 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
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- A61K9/5123—Organic compounds, e.g. fats, sugars
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- A—HUMAN NECESSITIES
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
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- 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
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- 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
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- C12N15/90—Stable introduction of foreign DNA into chromosome
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- A—HUMAN NECESSITIES
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- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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- 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
Landscapes
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- Chemical & Material Sciences (AREA)
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- Proteomics, Peptides & Aminoacids (AREA)
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- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063017442P | 2020-04-29 | 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 |
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| IL297718A true IL297718A (en) | 2022-12-01 |
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| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL297718A IL297718A (en) | 2020-04-29 | 2021-04-29 | Preparations and methods for the treatment of hereditary macular degeneration |
Country Status (9)
| Country | Link |
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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=) |
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| 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 |
| CN120230798A (zh) * | 2023-12-29 | 2025-07-01 | 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 |
| 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 |
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| US7309487B2 (en) * | 2004-02-09 | 2007-12-18 | George Inana | Methods and compositions for detecting and treating retinal diseases |
| US7943374B2 (en) * | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| WO2008137066A1 (en) * | 2007-05-02 | 2008-11-13 | The Board Of Regents Of The University Of Oklahoma | Use of compacted nucleic acid nanoparticles in non-viral treatments of ocular diseases |
| 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 |
| US20090214478A1 (en) * | 2008-02-21 | 2009-08-27 | Alberto Auricchio | Method of treating ocular diseases by gene therapy |
| EP2401376A4 (en) * | 2009-02-25 | 2012-11-28 | Univ Johns Hopkins | TRANSPOSON PIGGYBAC VARIANTS AND METHODS OF USE |
| US20150141320A1 (en) * | 2012-05-16 | 2015-05-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
| AU2014255665B2 (en) * | 2013-04-18 | 2018-08-02 | Fondazione Telethon | Effective delivery of large genes by dual AAV vectors |
| AU2015234242B2 (en) * | 2014-03-21 | 2020-09-24 | Children's Medical Research Institute | Stable gene transfer to proliferating cells |
| 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 |
| WO2020077360A1 (en) * | 2018-10-12 | 2020-04-16 | Poseida Therapeutics, Inc. | Hematopoietic stem cell compositions, methods of making and method of use |
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| EP4142796A4 (en) | 2024-06-26 |
| AU2021265838A1 (en) | 2022-10-20 |
| US20230193319A1 (en) | 2023-06-22 |
| CA3175197A1 (en) | 2021-11-04 |
| CN115461082A (zh) | 2022-12-09 |
| KR20230004573A (ko) | 2023-01-06 |
| JP2023523622A (ja) | 2023-06-06 |
| WO2021222654A1 (en) | 2021-11-04 |
| EP4142796A1 (en) | 2023-03-08 |
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