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MX2024001597A - Composicion y metodo para el tratamiento de la neuropatia optica hereditaria de leber causada por una mutacion en nd4. - Google Patents

Composicion y metodo para el tratamiento de la neuropatia optica hereditaria de leber causada por una mutacion en nd4.

Info

Publication number
MX2024001597A
MX2024001597A MX2024001597A MX2024001597A MX2024001597A MX 2024001597 A MX2024001597 A MX 2024001597A MX 2024001597 A MX2024001597 A MX 2024001597A MX 2024001597 A MX2024001597 A MX 2024001597A MX 2024001597 A MX2024001597 A MX 2024001597A
Authority
MX
Mexico
Prior art keywords
optic neuropathy
hereditary optic
mutation
composition
neuropathy caused
Prior art date
Application number
MX2024001597A
Other languages
English (en)
Inventor
Bin Li
Original Assignee
Wuhan Neurophth Biotechnology Ltd Company
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 Wuhan Neurophth Biotechnology Ltd Company filed Critical Wuhan Neurophth Biotechnology Ltd Company
Publication of MX2024001597A publication Critical patent/MX2024001597A/es

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    • C07K14/4702—Regulators; Modulating 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
    • 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
    • A61K48/005—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
    • 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
    • A61K48/005—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
    • 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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    • A—HUMAN NECESSITIES
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Abstract

Se proporciona un ácido nucleico recombinante que comprende: una secuencia mitocondrial diana; y una secuencia de codificación de una proteína mitocondrial, en la cual la secuencia de codificación de una proteína mitocondrial codifica un polipéptido que comprende una proteína mitocondrial; una secuencia del ácido nucleico del extremo 3' UTR, y una secuencia señal de poliA. También se proporciona una composición farmacéutica que comprende el ácido nucleico recombinante y un método para el tratamiento de la neuropatía óptica hereditaria de Leber (NOHL) utilizando la composición farmacéutica.
MX2024001597A 2021-08-06 2022-08-05 Composicion y metodo para el tratamiento de la neuropatia optica hereditaria de leber causada por una mutacion en nd4. MX2024001597A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CN202110899561 2021-08-06
PCT/CN2022/110545 WO2023011632A1 (zh) 2021-08-06 2022-08-05 用于治疗由于nd4突变造成的莱伯氏遗传性视神经病变的组合物和方法

Publications (1)

Publication Number Publication Date
MX2024001597A true MX2024001597A (es) 2024-02-15

Family

ID=85155306

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024001597A MX2024001597A (es) 2021-08-06 2022-08-05 Composicion y metodo para el tratamiento de la neuropatia optica hereditaria de leber causada por una mutacion en nd4.

Country Status (10)

Country Link
EP (1) EP4382601A1 (es)
JP (1) JP2024529033A (es)
KR (1) KR20240042489A (es)
CN (1) CN116024266A (es)
AR (1) AR126727A1 (es)
AU (1) AU2022322957A1 (es)
BR (1) BR112024002305A2 (es)
CA (1) CA3228344A1 (es)
MX (1) MX2024001597A (es)
WO (1) WO2023011632A1 (es)

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3625214A (en) 1970-05-18 1971-12-07 Alza Corp Drug-delivery device
US4906474A (en) 1983-03-22 1990-03-06 Massachusetts Institute Of Technology Bioerodible polyanhydrides for controlled drug delivery
US4789734A (en) 1985-08-06 1988-12-06 La Jolla Cancer Research Foundation Vitronectin specific cell receptor derived from mammalian mesenchymal tissue
CH671155A5 (es) 1986-08-18 1989-08-15 Clinical Technologies Ass
US5075109A (en) 1986-10-24 1991-12-24 Southern Research Institute Method of potentiating an immune response
US6024983A (en) 1986-10-24 2000-02-15 Southern Research Institute Composition for delivering bioactive agents for immune response and its preparation
US4897268A (en) 1987-08-03 1990-01-30 Southern Research Institute Drug delivery system and method of making the same
AU679041B2 (en) 1993-01-11 1997-06-19 Dana-Farber Cancer Institute Inducing cytotoxic T lymphocyte responses
US6207392B1 (en) 1997-11-25 2001-03-27 The Regents Of The University Of California Semiconductor nanocrystal probes for biological applications and process for making and using such probes
CN102634527B (zh) * 2012-04-11 2013-11-06 华中科技大学同济医学院附属同济医院 重组人nadh脱氢酶亚单位4基因及其表达载体构建方法
CN111068071A (zh) * 2018-10-22 2020-04-28 武汉纽福斯生物科技有限公司 基因治疗Leber遗传学视神经病变
CN113476484A (zh) * 2018-06-29 2021-10-08 武汉纽福斯生物科技有限公司 治疗遗传性视神经病变的组合物和方法
WO2020001657A1 (en) * 2018-06-29 2020-01-02 Wuhan Neurophth Biological Technology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
CN112584874A (zh) * 2018-08-20 2021-03-30 武汉纽福斯生物科技有限公司 用于治疗莱伯氏遗传性视神经病变的组合物和方法

Also Published As

Publication number Publication date
WO2023011632A1 (zh) 2023-02-09
AU2022322957A1 (en) 2024-03-21
BR112024002305A2 (pt) 2024-04-30
JP2024529033A (ja) 2024-08-01
KR20240042489A (ko) 2024-04-02
CA3228344A1 (en) 2023-02-09
EP4382601A1 (en) 2024-06-12
AR126727A1 (es) 2023-11-08
CN116024266A (zh) 2023-04-28

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