JP2024534496A - ハプロ不全障害の処置のためのｍＲＮＡレギュロン療法 - Google Patents

ハプロ不全障害の処置のためのｍＲＮＡレギュロン療法 Download PDF

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JP2024534496A
JP2024534496A JP2024517415A JP2024517415A JP2024534496A JP 2024534496 A JP2024534496 A JP 2024534496A JP 2024517415 A JP2024517415 A JP 2024517415A JP 2024517415 A JP2024517415 A JP 2024517415A JP 2024534496 A JP2024534496 A JP 2024534496A
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seq
syndrome
mrna
fusion protein
protein
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ジェフリー コラー
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ザ・ジョンズ・ホプキンス・ユニバーシティー
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • A61K48/005—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
    • A—HUMAN NECESSITIES
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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
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    • C12N15/86—Viral vectors
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1025—Acyltransferases (2.3)
    • C12N9/1029—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • A—HUMAN NECESSITIES
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/095—Fusion polypeptide containing a localisation/targetting motif containing a nuclear export signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/85—Fusion polypeptide containing an RNA binding domain
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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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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  • Peptides Or Proteins (AREA)
JP2024517415A 2021-09-21 2022-09-20 ハプロ不全障害の処置のためのｍＲＮＡレギュロン療法 Pending JP2024534496A (ja)

Applications Claiming Priority (5)

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US202163246689P 2021-09-21 2021-09-21
US63/246,689 2021-09-21
US202263352530P 2022-06-15 2022-06-15
US63/352,530 2022-06-15
PCT/US2022/076723 WO2023049707A1 (en) 2021-09-21 2022-09-20 Mrna regulon therapy for the treatment of haploinsufficiency disorders

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JP2024534496A true JP2024534496A (ja) 2024-09-20
JP2024534496A5 JP2024534496A5 (enExample) 2025-09-10

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US (1) US20250135035A1 (enExample)
EP (1) EP4404953A4 (enExample)
JP (1) JP2024534496A (enExample)
KR (1) KR20240099184A9 (enExample)
AU (1) AU2022349620A1 (enExample)
CA (1) CA3232214A1 (enExample)
MX (1) MX2024003412A (enExample)
WO (1) WO2023049707A1 (enExample)

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Publication number Priority date Publication date Assignee Title
EP4514955A1 (en) * 2022-04-29 2025-03-05 The Johns Hopkins University Systems for enhancing mrna expression and uses thereof
WO2025165891A1 (en) 2024-01-29 2025-08-07 Arnatar Therapeutics, Inc Translation enhancing nucleic acid compounds: aso coupled translation - upregulation 1 (act-up1) and uses thereof

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015221026A (ja) * 2014-05-23 2015-12-10 公立大学法人名古屋市立大学 人工合成ｍＲＮＡの翻訳効率化方法
US20200024603A1 (en) * 2017-08-25 2020-01-23 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
JP2020506948A (ja) * 2017-02-07 2020-03-05 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア ハプロ不全のための遺伝子治療
US20210071178A1 (en) * 2019-08-16 2021-03-11 Massachusetts Institute Of Technology Targeted trans-splicing using crispr/cas13

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20180312873A1 (en) * 2015-10-20 2018-11-01 10X Genomics, Inc. Method and systems for high throughput single cell genetic manipulation
US20230078498A1 (en) * 2020-02-07 2023-03-16 University Of Rochester Targeted Translation of RNA with CRISPR-Cas13 to Enhance Protein Synthesis

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015221026A (ja) * 2014-05-23 2015-12-10 公立大学法人名古屋市立大学 人工合成ｍＲＮＡの翻訳効率化方法
JP2020506948A (ja) * 2017-02-07 2020-03-05 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア ハプロ不全のための遺伝子治療
US20200024603A1 (en) * 2017-08-25 2020-01-23 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
US20210071178A1 (en) * 2019-08-16 2021-03-11 Massachusetts Institute Of Technology Targeted trans-splicing using crispr/cas13

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
GENES & DEVELOPMENT, vol. 12, JPN6026022300, 1998, pages 3226 - 3235, ISSN: 0005873196 *
NAT STRUCT MOL BIOL., vol. 27, no. 10, JPN6026022299, 2020, pages 989 - 1000, ISSN: 0005873195 *
NUCLEIC ACIDS RESEARCH, vol. 48, no. 10, JPN6026022298, 2020, pages 5684 - 5694, ISSN: 0005873194 *

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EP4404953A4 (en) 2025-09-24
US20250135035A1 (en) 2025-05-01
CA3232214A1 (en) 2023-03-30
KR20240099184A (ko) 2024-06-28
WO2023049707A1 (en) 2023-03-30
MX2024003412A (es) 2024-04-08
EP4404953A1 (en) 2024-07-31
AU2022349620A1 (en) 2024-03-21
KR20240099184A9 (ko) 2025-12-10

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