CN120138047A - 用于治疗1a型糖原贮积病的组成物和方法 - Google Patents

用于治疗1a型糖原贮积病的组成物和方法 Download PDF

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Publication number
CN120138047A
CN120138047A CN202510269938.1A CN202510269938A CN120138047A CN 120138047 A CN120138047 A CN 120138047A CN 202510269938 A CN202510269938 A CN 202510269938A CN 120138047 A CN120138047 A CN 120138047A
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adenosine deaminase
base editor
domain
cas9
polynucleotide
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Chinese (zh)
Inventor
N·戈代尔利
M·帕克
I·斯雷梅克
Y·于
B·蔡澈
Y·阿拉泰恩
F·葛瑞格尔
G·伦格
D·A·玻恩
S-J·李
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Bim Medical Co ltd
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Bim Medical Co ltd
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Publication of CN120138047A publication Critical patent/CN120138047A/zh
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    • 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
    • A61K38/46—Hydrolases (3)
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
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    • 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
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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CN202510269938.1A 2019-02-13 2020-02-13 用于治疗1a型糖原贮积病的组成物和方法 Pending CN120138047A (zh)

Applications Claiming Priority (18)

Application Number Priority Date Filing Date Title
US201962805271P 2019-02-13 2019-02-13
US62/805,271 2019-02-13
US201962852228P 2019-05-23 2019-05-23
US201962852224P 2019-05-23 2019-05-23
US62/852,228 2019-05-23
US62/852,224 2019-05-23
US201962876354P 2019-07-19 2019-07-19
US62/876,354 2019-07-19
US201962912992P 2019-10-09 2019-10-09
US62/912,992 2019-10-09
US201962931722P 2019-11-06 2019-11-06
US62/931,722 2019-11-06
US201962941569P 2019-11-27 2019-11-27
US62/941,569 2019-11-27
US202062966526P 2020-01-27 2020-01-27
US62/966,526 2020-01-27
PCT/US2020/018124 WO2020168088A1 (en) 2019-02-13 2020-02-13 Compositions and methods for treating glycogen storage disease type 1a
CN202080028267.5A CN114026237B (zh) 2019-02-13 2020-02-13 用于治疗1a型糖原贮积病的组成物和方法

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US (1) US20220127594A1 (https=)
EP (1) EP3924478A4 (https=)
JP (2) JP7631209B2 (https=)
KR (1) KR20210129108A (https=)
CN (2) CN120138047A (https=)
AU (1) AU2020221355A1 (https=)
CA (1) CA3128886A1 (https=)
WO (1) WO2020168088A1 (https=)

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WO2020168088A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
CN120174005A (zh) 2019-02-13 2025-06-20 比姆医疗股份有限公司 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
JP2023505234A (ja) * 2019-12-04 2023-02-08 アーバー バイオテクノロジーズ， インコーポレイテッド ヌクレアーゼを含む組成物及びその使用
US12416001B2 (en) * 2020-02-14 2025-09-16 Ohio State Innovation Foundation Nucleobase editors and methods of use thereof
CA3196831A1 (en) 2020-09-25 2022-03-31 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
CA3198671A1 (en) * 2020-10-14 2022-04-21 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
EP4504947A4 (en) * 2022-04-07 2026-03-25 Univ Duke COMPOSITIONS AND METHODS TO PROMOTE LIVER REGENERATION BY GENE EDITING IN METABOLIC LIVER DISEASE
CN117568313B (zh) * 2024-01-15 2024-04-26 上海贝斯昂科生物科技有限公司 基因编辑组合物及其用途

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JP2022519882A (ja) 2022-03-25
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