CO2022014527A2 - Método y fármaco para tratar el síndrome de hurler - Google Patents

Método y fármaco para tratar el síndrome de hurler

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Publication number
CO2022014527A2
CO2022014527A2 CONC2022/0014527A CO2022014527A CO2022014527A2 CO 2022014527 A2 CO2022014527 A2 CO 2022014527A2 CO 2022014527 A CO2022014527 A CO 2022014527A CO 2022014527 A2 CO2022014527 A2 CO 2022014527A2
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Colombia
Prior art keywords
hurler syndrome
nucleic acid
rna
acid drug
drug
Prior art date
Application number
CONC2022/0014527A
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English (en)
Inventor
Pengfei Yuan
Yanxia Zhao
Nengyin Liu
Zexuan Yi
Gangbin Tang
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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 CO2022014527A2 publication Critical patent/CO2022014527A2/es

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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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
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    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
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Abstract

Se divulgan un fármaco de ácido nucleico basado en la técnica LEAPER y un método para tratar enfermedades tales como el síndrome de Hurler mediante el uso del fármaco de ácido nucleico para dirigir y editar ARN; el método comprende: realizar una edición de la base de adenosina a la base de hipoxantina en el ARN usando el fármaco de ácido nucleico para reparar con precisión el sitio de mutación patógena de G > A de, por ejemplo, el síndrome de Hurler; recuperando así la expresión normal in vivo de una proteína codificada por ARN, tal como IDUA.
CONC2022/0014527A 2020-04-15 2022-10-13 Método y fármaco para tratar el síndrome de hurler CO2022014527A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CN2020084925 2020-04-15
PCT/CN2021/087534 WO2021209010A1 (zh) 2020-04-15 2021-04-15 一种治疗赫勒氏综合征的方法和药物

Publications (1)

Publication Number Publication Date
CO2022014527A2 true CO2022014527A2 (es) 2022-11-08

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ID=78084766

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Application Number Title Priority Date Filing Date
CONC2022/0014527A CO2022014527A2 (es) 2020-04-15 2022-10-13 Método y fármaco para tratar el síndrome de hurler

Country Status (14)

Country Link
US (1) US20230242916A1 (es)
EP (1) EP4137161A4 (es)
JP (1) JP2023521487A (es)
KR (1) KR20220162168A (es)
CN (1) CN115697420A (es)
AU (1) AU2021257508A1 (es)
BR (1) BR112022020795A2 (es)
CA (1) CA3180019A1 (es)
CO (1) CO2022014527A2 (es)
IL (1) IL297203A (es)
MX (1) MX2022012985A (es)
PE (1) PE20230703A1 (es)
TW (1) TW202204621A (es)
WO (1) WO2021209010A1 (es)

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Publication number Priority date Publication date Assignee Title
EP4069842A1 (en) 2019-12-02 2022-10-12 Shape Therapeutics Inc. Therapeutic editing
WO2023143538A1 (zh) * 2022-01-28 2023-08-03 北京辑因医疗科技有限公司 基于leaper技术治疗mpsi的方法和组合物
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
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

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9222088B2 (en) * 2010-10-22 2015-12-29 Curna, Inc. Treatment of alpha-L-iduronidase (IDUA) related diseases by inhibition of natural antisense transcript to IDUA
EP3230445B1 (en) * 2014-12-12 2024-01-24 Tod M. Woolf Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
EP3475424A1 (en) * 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
PL3507366T3 (pl) 2016-09-01 2021-05-04 Proqr Therapeutics Ii B.V. Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rna
JP2020535802A (ja) * 2017-09-21 2020-12-10 ザ・ブロード・インスティテュート・インコーポレイテッド 標的化核酸編集のための系、方法、及び組成物
US20210079393A1 (en) * 2018-02-14 2021-03-18 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for rna editing
CN115651927B (zh) * 2018-10-12 2023-06-16 北京大学 编辑rna的方法和组合物
GB201904709D0 (en) * 2019-04-03 2019-05-15 Proqr Therapeutics Ii Bv Chemically modified oligonucleotides
JP2022526455A (ja) * 2019-04-15 2022-05-24 北京大学 Ｒｎａを編集する方法および組成物
CA3146771A1 (en) * 2019-07-12 2021-01-21 Peking University Targeted rna editing by leveraging endogenous adar using engineered rnas
CR20220365A (es) * 2019-12-30 2022-12-06 Edigene Therapeutics Beijing Inc Método basado en tecnología leaper para el tratamiento de mucopolisacaridosis ih y composición
CN113528582B (zh) * 2020-04-15 2022-05-17 博雅辑因（北京）生物科技有限公司 基于leaper技术靶向编辑rna的方法和药物

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JP2023521487A (ja) 2023-05-24
EP4137161A1 (en) 2023-02-22
EP4137161A4 (en) 2023-10-11
CN115697420A (zh) 2023-02-03
PE20230703A1 (es) 2023-04-24
AU2021257508A1 (en) 2022-11-17
TW202204621A (zh) 2022-02-01
IL297203A (en) 2022-12-01
BR112022020795A2 (pt) 2022-11-29
CA3180019A1 (en) 2021-10-21
MX2022012985A (es) 2022-11-09
WO2021209010A1 (zh) 2021-10-21
KR20220162168A (ko) 2022-12-07
US20230242916A1 (en) 2023-08-03

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