JP2021500922A - 加齢黄斑変性を処置するための組成物および方法 - Google Patents
加齢黄斑変性を処置するための組成物および方法 Download PDFInfo
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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
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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
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/472—Complement proteins, e.g. anaphylatoxin, C3a, C5a
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- 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
- C12N2750/14134—Use of virus or viral component as vaccine, e.g. live-attenuated or inactivated virus, VLP, viral protein
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- 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
- C12N2750/14145—Special targeting system for viral vectors
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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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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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
- C12N2800/00—Nucleic acids vectors
- C12N2800/60—Vectors containing traps for, e.g. exons, promoters
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific
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- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
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- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762574814P | 2017-10-20 | 2017-10-20 | |
| US62/574,814 | 2017-10-20 | ||
| PCT/US2018/056709 WO2019079718A1 (en) | 2017-10-20 | 2018-10-19 | COMPOSITIONS AND METHODS FOR TREATING AGE-RELATED MACULAR DEGENERATION |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021500922A true JP2021500922A (ja) | 2021-01-14 |
| JP2021500922A5 JP2021500922A5 (https=) | 2021-11-25 |
Family
ID=66174265
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020542710A Withdrawn JP2021500922A (ja) | 2017-10-20 | 2018-10-19 | 加齢黄斑変性を処置するための組成物および方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20210188927A1 (https=) |
| EP (1) | EP3697920A4 (https=) |
| JP (1) | JP2021500922A (https=) |
| CN (1) | CN111788311A (https=) |
| AU (1) | AU2018351491A1 (https=) |
| CA (1) | CA3079553A1 (https=) |
| WO (1) | WO2019079718A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3824095A4 (en) * | 2018-07-20 | 2022-04-20 | University of Utah Research Foundation | GENE THERAPY FOR MACULATE GENERATION |
| CA3117551A1 (en) * | 2018-10-23 | 2020-04-30 | Gemini Therapeutics Inc. | Compositions and methods for treating age-related macular degeneration and other diseases |
| GB201821089D0 (en) | 2018-12-21 | 2019-02-06 | Gyroscope Therapeutics Ltd | Codon-optimised complement factor I |
| CA3158517A1 (en) * | 2019-10-22 | 2021-04-29 | Applied Genetic Technologies Corporation | Adeno-associated virus (aav)vectors for the treatment of age-related macular degeneration and other ocular diseases and disorders |
| EP4048318A4 (en) * | 2019-10-23 | 2023-11-22 | Gemini Therapeutics Sub, Inc. | METHOD FOR TREATING PATIENTS WITH CFH MUTATIONS USING RECOMBINANT CFH PROTEINS |
| GB202003618D0 (en) | 2020-03-12 | 2020-04-29 | Univ Bristol | Gene Therapy |
| AU2021293174A1 (en) | 2020-06-14 | 2023-01-19 | Vertex Pharmaceuticals Incorporated | Complement Factor I-related compositions and methods |
| MX2023004377A (es) * | 2020-10-16 | 2023-06-28 | Gyroscope Therapeutics Ltd | Acido nucleico que codifica una entidad anti-vegf y un regulador del complemento negativo y sus usos para el tratamiento de la degeneracion macular relacionada con la edad. |
| EP4236986A1 (en) * | 2020-10-30 | 2023-09-06 | Gemini Therapeutics Sub, Inc. | Methods for treating inflammatory ocular diseases with complement factor h |
| CN115786308B (zh) * | 2022-11-24 | 2024-05-31 | 江苏海洋大学 | 一种提高右旋糖酐酶热稳定性的方法及突变体 |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2005333845A (ja) * | 2004-05-25 | 2005-12-08 | Tokyoto Igaku Kenkyu Kiko | ミトコンドリア局在型ＤｓＲｅｄ２及びＥＣＦＰ発現ベクター |
| EP2357254A1 (en) * | 2005-02-14 | 2011-08-17 | University of Iowa Research Foundation | Methods and reagents for treatment and diagnosis of age-related macular degeneration |
| CN108291216B (zh) * | 2015-09-24 | 2022-09-16 | 宾夕法尼亚州大学信托人 | 用于治疗补体介导的疾病的组合物和方法 |
| GB201519086D0 (en) * | 2015-10-28 | 2015-12-09 | Syncona Partners Llp | Gene Therapy |
-
2018
- 2018-10-19 CN CN201880078342.1A patent/CN111788311A/zh active Pending
- 2018-10-19 JP JP2020542710A patent/JP2021500922A/ja not_active Withdrawn
- 2018-10-19 EP EP18869436.8A patent/EP3697920A4/en not_active Withdrawn
- 2018-10-19 AU AU2018351491A patent/AU2018351491A1/en not_active Abandoned
- 2018-10-19 WO PCT/US2018/056709 patent/WO2019079718A1/en not_active Ceased
- 2018-10-19 CA CA3079553A patent/CA3079553A1/en not_active Abandoned
- 2018-10-19 US US16/757,268 patent/US20210188927A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| AU2018351491A1 (en) | 2020-05-07 |
| WO2019079718A1 (en) | 2019-04-25 |
| EP3697920A1 (en) | 2020-08-26 |
| US20210188927A1 (en) | 2021-06-24 |
| CA3079553A1 (en) | 2019-04-25 |
| EP3697920A4 (en) | 2022-03-02 |
| CN111788311A (zh) | 2020-10-16 |
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