CR20220365A - Método basado en tecnología leaper para el tratamiento de mucopolisacaridosis ih y composición - Google Patents

Método basado en tecnología leaper para el tratamiento de mucopolisacaridosis ih y composición

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Publication number
CR20220365A
CR20220365A CR20220365A CR20220365A CR20220365A CR 20220365 A CR20220365 A CR 20220365A CR 20220365 A CR20220365 A CR 20220365A CR 20220365 A CR20220365 A CR 20220365A CR 20220365 A CR20220365 A CR 20220365A
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CR
Costa Rica
Prior art keywords
leaper
based method
technology based
composition
treating mps
Prior art date
Application number
CR20220365A
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English (en)
Inventor
Yanxia Zhao
Zexuan Yi
Gangbin Tang
Nengyin Liu
Pengfei Yuan
Original Assignee
Edigene Therapeutics Beijing Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Edigene Therapeutics Beijing Inc filed Critical Edigene Therapeutics Beijing Inc
Publication of CR20220365A publication Critical patent/CR20220365A/es

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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
    • 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
    • C12N15/1137—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 against enzymes
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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 RNAses, DNAses
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
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    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)

Abstract

La presente solicitud se refiere a un método basado en LEAPER para la edición dirigida de ARN, que comprende: realizar in vivo de forma segura y eficaz la edición de adenosina a inosina de ARN (edición A a I de ARN) por medio de LEAPER, con ello los sitios con mutación patógena pueden ser reparados con precisión y todas las enfermedades causadas por las mutaciones G>A, tales como MPS IH, pueden ser tratadas.
CR20220365A 2019-12-30 2020-12-30 Método basado en tecnología leaper para el tratamiento de mucopolisacaridosis ih y composición CR20220365A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CN2019129952 2019-12-30
PCT/CN2020/141506 WO2021136408A1 (zh) 2019-12-30 2020-12-30 一种基于leaper技术治疗mps ih的方法和组合物

Publications (1)

Publication Number Publication Date
CR20220365A true CR20220365A (es) 2022-12-06

Family

ID=76687311

Family Applications (1)

Application Number Title Priority Date Filing Date
CR20220365A CR20220365A (es) 2019-12-30 2020-12-30 Método basado en tecnología leaper para el tratamiento de mucopolisacaridosis ih y composición

Country Status (16)

Country Link
US (1) US20230060518A1 (es)
EP (1) EP4086345A4 (es)
JP (1) JP2023509179A (es)
KR (1) KR20220119129A (es)
CN (1) CN114846139A (es)
AU (1) AU2020418228A1 (es)
CA (1) CA3163272A1 (es)
CL (1) CL2022001776A1 (es)
CO (1) CO2022010432A2 (es)
CR (1) CR20220365A (es)
EC (1) ECSP22059741A (es)
IL (1) IL294201A (es)
MX (1) MX2022008190A (es)
PE (1) PE20230037A1 (es)
TW (1) TW202128193A (es)
WO (1) WO2021136408A1 (es)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112021020608A8 (pt) 2019-04-15 2023-03-21 Univ Beijing Rna recrutador de desaminase, construção, método para editar um rna alvo em uma célula hospedeira, e, construção, composição, célula, biblioteca ou kit
PE20220300A1 (es) 2019-07-12 2022-03-09 Univ Beijing Edicion de acido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actua sobre acido ribonucleico endogeno (adar) utilizando acidos ribonucleicos (arn) modificados geneticamente
CA3159944A1 (en) 2019-12-02 2021-06-10 David HUSS Therapeutic editing
WO2021209010A1 (zh) * 2020-04-15 2021-10-21 博雅辑因（北京）生物科技有限公司 一种治疗赫勒氏综合征的方法和药物
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
WO2024013360A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
WO2024013361A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucleotides for adar-mediated rna editing and use thereof
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3234134T3 (da) * 2014-12-17 2020-07-27 Proqr Therapeutics Ii Bv Målrettet rna-redigering
JP7074345B2 (ja) * 2016-06-22 2022-05-24 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ 一本鎖ｒｎａ編集オリゴヌクレオチド
US10941402B2 (en) * 2016-09-01 2021-03-09 Proqr Therapeutics Ii B.V. Chemically modified single-stranded RNA-editing oligonucleotides
JP7252328B2 (ja) 2018-10-12 2023-04-04 北京大学 Ｒｎａを編集する方法および組成物
CN114040970A (zh) * 2019-02-13 2022-02-11 比姆医疗股份有限公司 使用腺苷脱氨酶碱基编辑器编辑疾病相关基因的方法，包括遗传性疾病的治疗
BR112021020608A8 (pt) * 2019-04-15 2023-03-21 Univ Beijing Rna recrutador de desaminase, construção, método para editar um rna alvo em uma célula hospedeira, e, construção, composição, célula, biblioteca ou kit

Also Published As

Publication number Publication date
US20230060518A1 (en) 2023-03-02
EP4086345A1 (en) 2022-11-09
EP4086345A4 (en) 2024-03-20
IL294201A (en) 2022-08-01
PE20230037A1 (es) 2023-01-10
CN114846139A (zh) 2022-08-02
WO2021136408A1 (zh) 2021-07-08
ECSP22059741A (es) 2022-11-30
CA3163272A1 (en) 2021-07-08
CO2022010432A2 (es) 2022-08-09
AU2020418228A1 (en) 2022-08-18
JP2023509179A (ja) 2023-03-07
CL2022001776A1 (es) 2023-01-27
MX2022008190A (es) 2022-08-02
KR20220119129A (ko) 2022-08-26
TW202128193A (zh) 2021-08-01

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