CA3172481A1 - Engineered meganucleases having specificity for recognition sequences in the dystrophin gene - Google Patents

Engineered meganucleases having specificity for recognition sequences in the dystrophin gene Download PDF

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Publication number
CA3172481A1
CA3172481A1 CA3172481A CA3172481A CA3172481A1 CA 3172481 A1 CA3172481 A1 CA 3172481A1 CA 3172481 A CA3172481 A CA 3172481A CA 3172481 A CA3172481 A CA 3172481A CA 3172481 A1 CA3172481 A1 CA 3172481A1
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Prior art keywords
seq
engineered meganuclease
acid sequence
polynucleotide
dmd
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CA3172481A
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English (en)
French (fr)
Inventor
Gary Owens
Janel LAPE
James Jefferson Smith
John Morris
Caitlin TURNER
Whitney LEWIS
Derek Jantz
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Precision Biosciences Inc
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Precision Biosciences Inc
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Publication of CA3172481A1 publication Critical patent/CA3172481A1/en
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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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    • 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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    • C12N15/09Recombinant 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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    • A01K2227/10Mammal
    • 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
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    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
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    • 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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    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
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    • C12N2830/00Vector systems having a special element relevant for transcription
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    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/25Site-specific endodeoxyribonucleases specific for altered bases (3.1.25)

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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CA3172481A 2020-11-12 2021-11-12 Engineered meganucleases having specificity for recognition sequences in the dystrophin gene Pending CA3172481A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063113131P 2020-11-12 2020-11-12
US63/113,131 2020-11-12
US202163233664P 2021-08-16 2021-08-16
US63/233,664 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
CA3172481A1 true CA3172481A1 (en) 2022-05-19

Family

ID=78844897

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3172481A Pending CA3172481A1 (en) 2020-11-12 2021-11-12 Engineered meganucleases having specificity for recognition sequences in the dystrophin gene

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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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
EP4244342A1 (en) 2023-09-20
KR20250099412A (ko) 2025-07-01
CN120098962A (zh) 2025-06-06
IL302733B1 (en) 2025-01-01
MX2023005496A (es) 2023-08-04
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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Effective date: 20260422