PE20230037A1 - Metodo basado en tecnologia leaper para el tratamiento de mucopolisacaridosis ih y composicion - Google Patents

Metodo basado en tecnologia leaper para el tratamiento de mucopolisacaridosis ih y composicion

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Publication number
PE20230037A1
PE20230037A1 PE2022001378A PE2022001378A PE20230037A1 PE 20230037 A1 PE20230037 A1 PE 20230037A1 PE 2022001378 A PE2022001378 A PE 2022001378A PE 2022001378 A PE2022001378 A PE 2022001378A PE 20230037 A1 PE20230037 A1 PE 20230037A1
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PE
Peru
Prior art keywords
leaper
mucopolysaccharidosis
treatment
composition
technology
Prior art date
Application number
PE2022001378A
Other languages
English (en)
Inventor
Pengfei Yuan
Yanxia Zhao
Nengyin Liu
Zexuan Yi
Gangbin Tang
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 PE20230037A1 publication Critical patent/PE20230037A1/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
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    • C12N15/102—Mutagenizing nucleic acids
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    • 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
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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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    • 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)
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    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
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    • C12Y302/01076—L-Iduronidase (3.2.1.76)

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Abstract

La presente solicitud se refiere a un metodo basado en LEAPER para la edicion dirigida de ARN, que comprende: realizar in vivo de forma segura y eficaz la edicion de adenosina a inosina de ARN (edicion A a I de ARN) por medio de LEAPER, con ello los sitios con mutacion patogena pueden ser reparados con precision y todas las enfermedades causadas por las mutaciones G>A, tales como MPS IH, pueden ser tratadas
PE2022001378A 2019-12-30 2020-12-30 Metodo basado en tecnologia leaper para el tratamiento de mucopolisacaridosis ih y composicion PE20230037A1 (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
PE20230037A1 true PE20230037A1 (es) 2023-01-10

Family

ID=76687311

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2022001378A PE20230037A1 (es) 2019-12-30 2020-12-30 Metodo basado en tecnologia leaper para el tratamiento de mucopolisacaridosis ih y composicion

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 (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2017281497B2 (en) 2016-06-22 2023-04-06 Proqr Therapeutics Ii B.V. Single-stranded RNA-editing oligonucleotides
EP3956449A4 (en) 2019-04-15 2023-03-29 EdiGene Therapeutics (Beijing) Inc. METHODS AND COMPOSITIONS FOR EDITING RNAs
JP2022536546A (ja) 2019-07-12 2022-08-17 北京大学 遺伝子操作ｒｎａを用いた内因性ａｄａｒによる標的化ｒｎａ編集
CA3159944A1 (en) 2019-12-02 2021-06-10 David HUSS Therapeutic editing
US20230242916A1 (en) * 2020-04-15 2023-08-03 Edigene Therapeutics (Beijing) Inc. Method and drug for treating hurler syndrome
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
WO2024013361A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucleotides for adar-mediated rna editing and use thereof
WO2024013360A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
WO2024110565A1 (en) 2022-11-24 2024-05-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
WO2024121373A1 (en) 2022-12-09 2024-06-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of cardiovascular disease

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3712269A1 (en) * 2014-12-17 2020-09-23 ProQR Therapeutics II B.V. Targeted rna editing
AU2017281497B2 (en) * 2016-06-22 2023-04-06 Proqr Therapeutics Ii B.V. Single-stranded RNA-editing oligonucleotides
KR102501980B1 (ko) * 2016-09-01 2023-02-20 프로큐알 테라퓨틱스 Ⅱ 비.브이. 화학적으로 변형된 단일 가닥 rna-편집 올리고뉴클레오타이드
ES2962434T3 (es) 2018-10-12 2024-03-19 Univ Beijing Procedimientos y composiciones para editar ARN
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
EP3956449A4 (en) * 2019-04-15 2023-03-29 EdiGene Therapeutics (Beijing) Inc. METHODS AND COMPOSITIONS FOR EDITING RNAs

Also Published As

Publication number Publication date
US20230060518A1 (en) 2023-03-02
MX2022008190A (es) 2022-08-02
IL294201A (en) 2022-08-01
CO2022010432A2 (es) 2022-08-09
EP4086345A1 (en) 2022-11-09
AU2020418228A1 (en) 2022-08-18
JP2023509179A (ja) 2023-03-07
CR20220365A (es) 2022-12-06
CN114846139A (zh) 2022-08-02
CA3163272A1 (en) 2021-07-08
CL2022001776A1 (es) 2023-01-27
EP4086345A4 (en) 2024-03-20
ECSP22059741A (es) 2022-11-30
WO2021136408A1 (zh) 2021-07-08
KR20220119129A (ko) 2022-08-26
TW202128193A (zh) 2021-08-01

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