CN118696126A - 用于治疗雷特综合征及相关病症的重组优化的mecp2盒和方法 - Google Patents

用于治疗雷特综合征及相关病症的重组优化的mecp2盒和方法 Download PDF

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Publication number
CN118696126A
CN118696126A CN202380019725.2A CN202380019725A CN118696126A CN 118696126 A CN118696126 A CN 118696126A CN 202380019725 A CN202380019725 A CN 202380019725A CN 118696126 A CN118696126 A CN 118696126A
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Prior art keywords
polynucleotide
mecp2
aav
vector
raav
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CN202380019725.2A
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Chinese (zh)
Inventor
斯图尔特·罗伯特·科布
保罗·罗斯
拉尔夫·戴维·赫克托
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University of Edinburgh
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University of Edinburgh
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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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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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2227/10—Mammal
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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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    • 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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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CN202380019725.2A 2022-01-31 2023-01-31 用于治疗雷特综合征及相关病症的重组优化的mecp2盒和方法 Pending CN118696126A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB2201242.1 2022-01-31
GBGB2201242.1A GB202201242D0 (en) 2022-01-31 2022-01-31 Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
PCT/GB2023/050207 WO2023144565A1 (en) 2022-01-31 2023-01-31 Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders

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CN118696126A true CN118696126A (zh) 2024-09-24

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US (1) US20250136651A1 (https=)
EP (1) EP4473121A1 (https=)
JP (1) JP2025503868A (https=)
KR (1) KR20240137089A (https=)
CN (1) CN118696126A (https=)
AU (1) AU2023213148A1 (https=)
CA (1) CA3247763A1 (https=)
GB (1) GB202201242D0 (https=)
IL (1) IL314321A (https=)
MX (1) MX2024008599A (https=)
WO (1) WO2023144565A1 (https=)

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Publication number Priority date Publication date Assignee Title
WO2026017965A1 (en) * 2024-07-19 2026-01-22 The University Court Of The University Of Edinburgh Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions

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GB202201242D0 (en) 2022-03-16
IL314321A (en) 2024-09-01
JP2025503868A (ja) 2025-02-06
US20250136651A1 (en) 2025-05-01
KR20240137089A (ko) 2024-09-19
EP4473121A1 (en) 2024-12-11
WO2023144565A1 (en) 2023-08-03
AU2023213148A1 (en) 2024-07-25
CA3247763A1 (en) 2023-08-03
MX2024008599A (es) 2024-08-13

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