CN111788311A - 用于治疗年龄相关性黄斑变性的组合物和方法 - Google Patents

用于治疗年龄相关性黄斑变性的组合物和方法 Download PDF

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CN111788311A
CN111788311A CN201880078342.1A CN201880078342A CN111788311A CN 111788311 A CN111788311 A CN 111788311A CN 201880078342 A CN201880078342 A CN 201880078342A CN 111788311 A CN111788311 A CN 111788311A
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vector
promoter
cfh
aav
seq
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J·麦克劳克林
A·科伊勒拉
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Disc Medicine Inc
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Gemini Therapeutics Inc
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CN201880078342.1A 2017-10-20 2018-10-19 用于治疗年龄相关性黄斑变性的组合物和方法 Pending CN111788311A (zh)

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

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CN111788311A true CN111788311A (zh) 2020-10-16

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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=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115786308A (zh) * 2022-11-24 2023-03-14 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

Families Citing this family (9)

* Cited by examiner, † Cited by third party
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

Citations (4)

* Cited by examiner, † Cited by third party
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
WO2006088950A2 (en) * 2005-02-14 2006-08-24 University Of Iowa Research Foundation Methods and reagents for treatment and diagnosis of age-related macular degeneration
WO2017053732A2 (en) * 2015-09-24 2017-03-30 The Trustees Of The University Of Pennsylvania Composition and method for treating complement-mediated disease
WO2017072515A1 (en) * 2015-10-28 2017-05-04 Syncona Management Llp Gene therapy

Patent Citations (7)

* Cited by examiner, † Cited by third party
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
WO2006088950A2 (en) * 2005-02-14 2006-08-24 University Of Iowa Research Foundation Methods and reagents for treatment and diagnosis of age-related macular degeneration
CN103920142A (zh) * 2005-02-14 2014-07-16 爱荷华大学研究基金会 治疗和诊断年龄相关性黄斑变性的方法和试剂
WO2017053732A2 (en) * 2015-09-24 2017-03-30 The Trustees Of The University Of Pennsylvania Composition and method for treating complement-mediated disease
CN108291216A (zh) * 2015-09-24 2018-07-17 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
WO2017072515A1 (en) * 2015-10-28 2017-05-04 Syncona Management Llp Gene therapy
CN108431030A (zh) * 2015-10-28 2018-08-21 辛科纳投资管理有限公司 基因疗法

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115786308A (zh) * 2022-11-24 2023-03-14 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
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

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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
JP2021500922A (ja) 2021-01-14

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