MX2023005496A - Meganucleasas manipuladas genéticamente que tienen especifidad para secuencias de reconocimiento en el gen de la distrofina. - Google Patents

Meganucleasas manipuladas genéticamente que tienen especifidad para secuencias de reconocimiento en el gen de la distrofina.

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Publication number
MX2023005496A
MX2023005496A MX2023005496A MX2023005496A MX2023005496A MX 2023005496 A MX2023005496 A MX 2023005496A MX 2023005496 A MX2023005496 A MX 2023005496A MX 2023005496 A MX2023005496 A MX 2023005496A MX 2023005496 A MX2023005496 A MX 2023005496A
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MX
Mexico
Prior art keywords
meganucleases
dystrophin gene
sequences
recognization
genetically manipulated
Prior art date
Application number
MX2023005496A
Other languages
English (en)
Spanish (es)
Inventor
Gary Owens
Derek Jantz
John Morris
James Jefferson Smith
Janel Lape
Caitlin Turner
Whitney Lewis
Original Assignee
Prec Biosciences 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 Prec Biosciences Inc filed Critical Prec Biosciences Inc
Publication of MX2023005496A publication Critical patent/MX2023005496A/es

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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707Muscular dystrophy
    • C07K14/4708Duchenne dystrophy
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00Modified animals
    • A01K2207/15Humanized animals
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
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    • C12N2800/00Nucleic acids vectors
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    • C12N2800/106Plasmid DNA for vertebrates
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    • C12N2830/00Vector systems having a special element relevant for transcription
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    • C12N2830/00Vector systems having a special element relevant for transcription
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    • C12N2830/00Vector systems having a special element relevant for transcription
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/25Site-specific endodeoxyribonucleases specific for altered bases (3.1.25)

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  • Physics & Mathematics (AREA)
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  • Veterinary Medicine (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
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MX2023005496A 2020-11-12 2021-11-12 Meganucleasas manipuladas genéticamente que tienen especifidad para secuencias de reconocimiento en el gen de la distrofina. MX2023005496A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063113131P 2020-11-12 2020-11-12
US202163233664P 2021-08-16 2021-08-16
PCT/US2021/059146 WO2022104062A1 (en) 2020-11-12 2021-11-12 Engineered meganucleases having specificity for recognition sequences in the dystrophin gene

Publications (1)

Publication Number Publication Date
MX2023005496A true MX2023005496A (es) 2023-08-04

Family

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Application Number Title Priority Date Filing Date
MX2023005496A MX2023005496A (es) 2020-11-12 2021-11-12 Meganucleasas manipuladas genéticamente que tienen especifidad para secuencias de reconocimiento en el gen de la distrofina.

Country Status (9)

Country Link
US (4) US11753630B2 (https=)
EP (1) EP4244342B1 (https=)
JP (2) JP7642809B2 (https=)
KR (2) KR102826438B1 (https=)
CN (3) CN120098962A (https=)
CA (1) CA3172481A1 (https=)
IL (2) IL317538A (https=)
MX (1) MX2023005496A (https=)
WO (1) WO2022104062A1 (https=)

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CA3259572A1 (en) * 2022-06-30 2024-01-04 Sarepta Therapeutics, Inc. TREATMENT METHODS FOR MUSCULAR DYSTROPHY
WO2024216116A1 (en) 2023-04-14 2024-10-17 Precision Biosciences, Inc. Muscle-specific expression cassettes
WO2024216118A1 (en) 2023-04-14 2024-10-17 Precision Biosciences, Inc. Muscle-specific expression cassettes
TW202546236A (zh) * 2024-01-16 2025-12-01 美商安斯泰來基因治療股份有限公司 用於治療第1型肌強直性營養不良之肌肉選擇性混合調節組合及其使用方法

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Publication number Publication date
CN120098963A (zh) 2025-06-06
IL302733B2 (en) 2025-05-01
CA3172481A1 (en) 2022-05-19
EP4244342A1 (en) 2023-09-20
KR20250099412A (ko) 2025-07-01
CN120098962A (zh) 2025-06-06
IL302733B1 (en) 2025-01-01
KR20230118841A (ko) 2023-08-14
US11753630B2 (en) 2023-09-12
IL302733A (en) 2023-07-01
EP4244342B1 (en) 2026-04-01
CN116783293A (zh) 2023-09-19
CN116783293B (zh) 2025-03-18
WO2022104062A1 (en) 2022-05-19
IL317538A (en) 2025-02-01
US20240018495A1 (en) 2024-01-18
JP2025098002A (ja) 2025-07-01
US11993793B2 (en) 2024-05-28
JP2023549357A (ja) 2023-11-24
US20240002823A1 (en) 2024-01-04
US12006522B2 (en) 2024-06-11
US20230053176A1 (en) 2023-02-16
US20250034536A1 (en) 2025-01-30
KR102826438B1 (ko) 2025-07-04
JP7642809B2 (ja) 2025-03-10

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