CN114174514A - 可调节的表达系统 - Google Patents

可调节的表达系统 Download PDF

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Publication number
CN114174514A
CN114174514A CN202080054347.8A CN202080054347A CN114174514A CN 114174514 A CN114174514 A CN 114174514A CN 202080054347 A CN202080054347 A CN 202080054347A CN 114174514 A CN114174514 A CN 114174514A
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seq
nucleic acid
aav
vector
acid molecule
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Chinese (zh)
Inventor
M·贝贝尔
C·古布泽凯勒
D·卢卡谢夫
N·雷诺
N·鲁丁斯基
R·西瓦桑卡兰
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Novartis AG
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Novartis AG
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Priority to CN202510290352.3A priority Critical patent/CN120137971A/zh
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CN202080054347.8A 2019-07-25 2020-07-24 可调节的表达系统 Pending CN114174514A (zh)

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CN202510290352.3A CN120137971A (zh) 2019-07-25 2020-07-24 可调节的表达系统

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US201962878514P 2019-07-25 2019-07-25
US62/878,514 2019-07-25
US202063013283P 2020-04-21 2020-04-21
US63/013,283 2020-04-21
PCT/IB2020/057038 WO2021014428A1 (en) 2019-07-25 2020-07-24 Regulatable expression systems

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US (1) US20220307053A1 (https=)
EP (1) EP4004213A1 (https=)
JP (2) JP2022541070A (https=)
KR (1) KR20220035937A (https=)
CN (2) CN114174514A (https=)
AU (1) AU2020319168B2 (https=)
CA (1) CA3147574A1 (https=)
IL (1) IL289711A (https=)
WO (1) WO2021014428A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115605596A (zh) * 2020-02-12 2023-01-13 费城儿童医院（Us） 用于基因表达的诱导型可变剪接调节的组合物和方法
WO2024060205A1 (zh) * 2022-09-23 2024-03-28 北京基驭医疗科技有限公司 包含基于小分子药物的可变剪接调节元件的核酸分子

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022226183A2 (en) * 2021-04-21 2022-10-27 The Board Of Regents Of The University Of Texas System Optimized ap4m1 polynucleotides and expression cassettes and their use
GB2617565A (en) * 2022-04-11 2023-10-18 Ucl Business Ltd A construct, vector and system and uses thereof
WO2023219533A1 (ru) * 2022-05-11 2023-11-16 Общество С Ограниченной Ответственностью "Герофарм" Аденоассоциированный вирусный вектор на основе серотипа aav.php.b и его применение
GB202215198D0 (en) * 2022-10-14 2022-11-30 Univ Sheffield Gene therapy treatment
EP4677098A1 (en) * 2023-03-06 2026-01-14 Agency for Science, Technology and Research Engineered furin cleavage sequences for co-expression of multiple genes in mammalian cells
KR20260003718A (ko) * 2023-04-14 2026-01-07 알젠타 테라퓨틱스, 인크. 소분자 조절 대체 스플라이싱
WO2025015060A1 (en) * 2023-07-11 2025-01-16 Brave Bio, Inc. Engineered vectorized antibodies with reduced stability in systemic circulation and uses thereof

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110086833A1 (en) * 2008-05-27 2011-04-14 Paushkin Sergey V Methods for treating spinal muscular atrophy
WO2016034680A1 (en) * 2014-09-05 2016-03-10 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of leber congenital amaurosis
WO2018009547A1 (en) * 2016-07-05 2018-01-11 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Diagnosing col6-related disorders and methods for treating same
WO2018098446A1 (en) * 2016-11-28 2018-05-31 Ptc Therapeutics, Inc Methods for modulating rna splicing
US20190000946A1 (en) * 2013-10-28 2019-01-03 Invectys Telomerase encoding dna vaccine

Family Cites Families (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
AU707866B2 (en) 1994-12-06 1999-07-22 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
AU722196B2 (en) 1995-08-30 2000-07-27 Genzyme Corporation Chromatographic purification of adenovirus and AAV
EP1983057A3 (en) 1995-09-08 2009-01-07 Genzyme Corporation Improved AAV vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
JP2001514845A (ja) 1997-09-05 2001-09-18 ターゲティッド ジェネティクス コーポレイション 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP1751275B1 (en) 2004-06-01 2017-08-16 Avigen, Inc. Compositions and methods to prevent aav vector aggregation
US8110681B2 (en) 2006-03-17 2012-02-07 The United States Of America As Represented By The Secretary, Department Of Health And Human Services Compounds for the treatment of spinal muscular atrophy and other uses
CA2653974A1 (en) 2006-05-30 2008-02-14 Mayo Foundation For Medical Education And Research Detecting and treating dementia
DK2529020T3 (en) 2010-01-28 2018-08-06 Childrens Hospital Philadelphia SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
US8729263B2 (en) 2012-08-13 2014-05-20 Novartis Ag 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency-related conditions
US9040712B2 (en) 2013-01-23 2015-05-26 Novartis Ag Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions
EP4105208A1 (en) 2013-07-31 2022-12-21 Novartis AG 1,4-disubstituted pyridazine derivatives and their use for treating smn-deficiency-related conditions
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
JP6202701B2 (ja) 2014-03-21 2017-09-27 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及びプログラム
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
EA201800367A1 (ru) 2015-12-10 2019-02-28 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы лечения болезни хантингтона
EP3634953B1 (en) 2017-06-05 2024-01-03 PTC Therapeutics, Inc. Compounds for treating huntington's disease
JP2020523365A (ja) * 2017-06-14 2020-08-06 ピーティーシー セラピューティクス，インコーポレーテッド Ｒｎａスプライシングを改変する方法
MX2019015580A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
CN111182898B (zh) 2017-06-28 2024-04-16 Ptc医疗公司 用于治疗亨廷顿氏病的方法
JP7312749B2 (ja) 2017-08-04 2023-07-21 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングをモジュレートする方法および組成物
MX2020004830A (es) 2017-11-08 2020-11-11 Avexis Inc Medios y metodo para preparar vectores virales y usos de los mismos.

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110086833A1 (en) * 2008-05-27 2011-04-14 Paushkin Sergey V Methods for treating spinal muscular atrophy
US20190000946A1 (en) * 2013-10-28 2019-01-03 Invectys Telomerase encoding dna vaccine
WO2016034680A1 (en) * 2014-09-05 2016-03-10 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of leber congenital amaurosis
WO2018009547A1 (en) * 2016-07-05 2018-01-11 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Diagnosing col6-related disorders and methods for treating same
WO2018098446A1 (en) * 2016-11-28 2018-05-31 Ptc Therapeutics, Inc Methods for modulating rna splicing

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ATWOOD K. CHEUNG ET AL.: "Discovery of Small Molecule Splicing Modulators of Survival Motor Neuron‑2 (SMN2) for the Treatment of Spinal Muscular Atrophy (SMA)", 《JOURNAL OF MEDICINAL CHEMISTRY》, no. 61, pages 11021 *
JAMES PALACINO ET AL.: "sMn2 splice modulators enhance u1–pre-mrna association and rescue sMa mice", 《NATURE CHEMICAL BIOLOGY 》, vol. 11, 1 June 2015 (2015-06-01), pages 511 - 517, XP055520469, DOI: 10.1038/nchembio.1837 *
SATOMI YOSHIMOTO ET AL.: "Alternative splicing of a cryptic exon embedded in intron 6 of SMN1 and SMN2", 《HUMAN GENOME VARIATION》, vol. 3, no. 16040, pages 1 - 3 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115605596A (zh) * 2020-02-12 2023-01-13 费城儿童医院（Us） 用于基因表达的诱导型可变剪接调节的组合物和方法
WO2024060205A1 (zh) * 2022-09-23 2024-03-28 北京基驭医疗科技有限公司 包含基于小分子药物的可变剪接调节元件的核酸分子

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