CN119955780A - 用于治疗提前终止密码子介导的病症的方法和组合物 - Google Patents

用于治疗提前终止密码子介导的病症的方法和组合物 Download PDF

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CN119955780A
CN119955780A CN202411915548.9A CN202411915548A CN119955780A CN 119955780 A CN119955780 A CN 119955780A CN 202411915548 A CN202411915548 A CN 202411915548A CN 119955780 A CN119955780 A CN 119955780A
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trna
seq
egfp
cells
gene
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P·M·埃蒙
S·马克法兰
Y-H·陈
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Twad Bioscience Co ltd
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Twad Bioscience Co ltd
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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • 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
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/335—Modified T or U
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    • 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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  • Health & Medical Sciences (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
CN202411915548.9A 2019-11-01 2020-10-30 用于治疗提前终止密码子介导的病症的方法和组合物 Pending CN119955780A (zh)

Applications Claiming Priority (4)

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US201962929428P 2019-11-01 2019-11-01
US62/929,428 2019-11-01
PCT/US2020/058415 WO2021087401A1 (en) 2019-11-01 2020-10-30 Methods and compositions for treating a premature termination codon-mediated disorder
CN202080090997.8A CN115209902B (zh) 2019-11-01 2020-10-30 用于治疗提前终止密码子介导的病症的方法和组合物

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CN202080090997.8A Active CN115209902B (zh) 2019-11-01 2020-10-30 用于治疗提前终止密码子介导的病症的方法和组合物

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US (1) US12697350B2 (enExample)
EP (1) EP4051291A4 (enExample)
JP (2) JP7712270B2 (enExample)
KR (1) KR20220155981A (enExample)
CN (2) CN119955780A (enExample)
AU (1) AU2020375040A1 (enExample)
CA (1) CA3156791A1 (enExample)
WO (1) WO2021087401A1 (enExample)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
MX2020004587A (es) 2017-11-02 2021-04-12 Univ Iowa Res Found Métodos de rescate de codones de paro mediante reasignación genética con ace-arnt.
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
JP7712270B2 (ja) 2019-11-01 2025-07-23 テバード バイオサイエンシズ インコーポレイテッド 未成熟終止コドン媒介障害を処置するための方法および組成物
BR112022020780A2 (pt) * 2020-04-14 2022-11-29 Flagship Pioneering Innovations Vi Llc Composições de trem e usos das mesmas
EP4334450A4 (en) * 2021-05-05 2025-06-18 Tevard Biosciences, Inc. Methods and compositions for treating a premature termination codon-mediated disorder
CA3235264A1 (en) * 2021-10-13 2023-04-20 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods of use
CA3249261A1 (en) 2022-04-18 2023-10-26 Vertex Pharmaceuticals Incorporated COMPOSITIONS AND PROCESSES TO IMPROVE ADENE-ASSOCIATED VIRUS (AAV) THERAPY AND REDUCE AAV TROPISM TO THE LIVER
WO2023220342A2 (en) * 2022-05-13 2023-11-16 Shape Therapeutics Inc. Engineered tranfer rnas
WO2024155745A1 (en) * 2023-01-18 2024-07-25 The Broad Institute, Inc. Base editing-mediated readthrough of premature termination codons (bert)
EP4652272A1 (en) * 2023-01-18 2025-11-26 The Broad Institute Inc. Prime editing-mediated readthrough of premature termination codons (pert)
EP4442826A1 (en) 2023-04-06 2024-10-09 Universität Hamburg Synthetic dna construct encoding transfer rna
EP4702142A1 (en) * 2023-04-28 2026-03-04 University of Rochester A method of treating a premature translation termination codon n1n2n3 (ptc)-related disease by nonsense suppression in a subject
EP4458967A1 (en) 2023-05-02 2024-11-06 Universität Hamburg Pharmaceutical composition comprising multiple suppressor transfer rnas
WO2025111506A1 (en) * 2023-11-22 2025-05-30 Tevard Biosciences, Inc. Modified transfer rnas and methods of use
WO2025119204A1 (en) * 2023-12-04 2025-06-12 Modit Therapeutics Beijing Limited Methods and compositions for improved readthrough of pseudouridine-modified codons
WO2026030209A1 (en) * 2024-07-29 2026-02-05 University Of Rochester Use of decoding trnas to boost protein expression or function
WO2026136461A1 (en) * 2024-12-17 2026-06-25 The Broad Institute, Inc. Prime editing-mediated readthrough of premature termination codons (pert)

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4687737A (en) 1982-11-12 1987-08-18 Massachusetts Institute Of Technology Mammalian suppressor genes
US5840702A (en) 1996-03-22 1998-11-24 Uab Research Foundation Cystic fibrosis treatment
US7351578B2 (en) 1999-12-10 2008-04-01 Invitrogen Corp. Use of multiple recombination sites with unique specificity in recombinational cloning
JP2002508959A (ja) 1998-01-14 2002-03-26 ヒューマン ジーン セラピー リサーチ インスティテュート ヒトのサプレッサーｔＲＮＡオリゴヌクレオチド、及びそれを用いる方法
CA2449950A1 (en) 2001-06-13 2002-12-27 Masayuki Arakawa Agents for treating diseases caused by nonsense mutation
WO2003033521A1 (en) 2001-10-16 2003-04-24 Massachusetts Institute Of Technology Inppressor trna system in mammalian cells for the introduction of unnatural amino acids in polypeptides.
US20040219516A1 (en) 2002-07-18 2004-11-04 Invitrogen Corporation Viral vectors containing recombination sites
KR101423898B1 (ko) 2005-12-14 2014-07-28 암브룩스, 인코포레이티드 비-천연 아미노산 및 폴리펩티드를 함유하는 조성물, 비-천연 아미노산 및 폴리펩티드를 포함하는 방법, 및 비-천연 아미노산 및 폴리펩티드의 용도
GB0600948D0 (en) 2006-01-18 2006-02-22 Univ Dundee Prevention/Treatment Of Ichthyosis Vulgaris, Atopy And Other Disorders
FR2902439A1 (fr) 2006-06-14 2007-12-21 Univ Rene Descartes Paris V Et Arn de transfert chimerique et son utilisation pour la production d'arn par une cellule
WO2009086497A2 (en) 2007-12-28 2009-07-09 Ardell David H Genetic encryption
US20120077186A1 (en) 2010-05-07 2012-03-29 Oregon Health & Science University Use of cysteine-derived suppressor trnas for non-native amino acid incorporation
CN109112126B (zh) * 2010-06-23 2024-06-14 库尔纳公司 通过抑制电压门控钠通道α亚基(SCNA)的天然反义转录物而治疗SCNA相关疾病
KR101991980B1 (ko) * 2011-09-06 2019-06-21 큐알엔에이, 인크. 소형 분자로 전압-개폐된 나트륨 채널 (SCNxA)의 알파 아단위에 관련된 질환의 치료
US10513699B2 (en) 2014-09-03 2019-12-24 Bioatla, Llc Discovering and producing conditionally active biologic proteins in the same eukaryotic cell production hosts
HRP20170589T2 (hr) 2014-12-22 2023-05-12 Sandoz Ag Varijante sekvenci
US10900036B2 (en) 2015-03-17 2021-01-26 The General Hospital Corporation RNA interactome of polycomb repressive complex 1 (PRC1)
US20180171321A1 (en) 2015-03-27 2018-06-21 The University Of Queensland Platform for a non-natural amino acid incorporation into proteins
WO2017049409A1 (en) 2015-09-25 2017-03-30 The Centre For Drug Research And Development Compositions for promoting readthrough of premature termination codons, and methods of using the same
CN107177592B (zh) 2016-03-10 2021-03-30 北京大学 抑制性tRNA通读提前终止密码子疾病中的截短蛋白
PL3458074T3 (pl) 2016-05-16 2024-11-12 Board Of Regents Of The University Of Texas System KOMPOZYCJA DO DOSTARCZANIA tRNA W POSTACI NANOCZĄSTEK I SPOSOBY ICH STOSOWANIA
WO2018031531A1 (en) 2016-08-08 2018-02-15 President And Fellows Of Harvard College Engineered bacteria for non-invasive imaging and therapeutic applications
WO2018041973A1 (en) * 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. Chemically modified single-stranded rna-editing oligonucleotides
CN110612353A (zh) 2017-03-03 2019-12-24 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
MX2020004587A (es) * 2017-11-02 2021-04-12 Univ Iowa Res Found Métodos de rescate de codones de paro mediante reasignación genética con ace-arnt.
AU2019233673B2 (en) 2018-03-15 2025-05-01 Arcturus Therapeutics, Inc. Synthetic transfer RNA with extended anticodon loop
WO2020069194A1 (en) 2018-09-26 2020-04-02 Case Western Reserve University Methods and compositions for treating a premature stop codon-mediated disorder
EA202190736A1 (ru) 2018-09-26 2021-09-16 Кейс Вестерн Ризерв Юниверсити Способы и композиции для увеличения экспрессии белка и/или лечения расстройства, связанного с гаплонедостаточностью
JP2022528696A (ja) 2019-04-11 2022-06-15 アークタラス テラピュウティクス、インク アンチコドンループを延長した合成転移ｒｎａ
EP4041317A4 (en) 2019-10-11 2024-05-22 University of Massachusetts RAAV-MEDIATED IN VIVO ADMINISTRATION OF ARNT SUPPRESSANTS
JP7712270B2 (ja) 2019-11-01 2025-07-23 テバード バイオサイエンシズ インコーポレイテッド 未成熟終止コドン媒介障害を処置するための方法および組成物
CA3160097A1 (en) 2019-11-04 2021-05-14 Flagship Pioneering, Inc. Trem compositions for con-rare codons and related uses
JP2023504661A (ja) 2019-12-02 2023-02-06 シェイプ・セラピューティクス・インコーポレイテッド 疾患の処置のための標的化トランスファーｒｎａ
EP4334450A4 (en) 2021-05-05 2025-06-18 Tevard Biosciences, Inc. Methods and compositions for treating a premature termination codon-mediated disorder

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JP2025148444A (ja) 2025-10-07
US20240148772A1 (en) 2024-05-09
CN115209902B (zh) 2025-01-07
AU2020375040A1 (en) 2022-05-19
CN115209902A (zh) 2022-10-18
JP2022554357A (ja) 2022-12-28
JP7712270B2 (ja) 2025-07-23
WO2021087401A1 (en) 2021-05-06
EP4051291A1 (en) 2022-09-07
KR20220155981A (ko) 2022-11-24
US12697350B2 (en) 2026-08-04
CA3156791A1 (en) 2021-05-06
EP4051291A4 (en) 2024-07-03

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