JP2024515629A - ニューロフィブロミン２／マーリン（ｎｆ２）遺伝子療法のための材料及び方法 - Google Patents

ニューロフィブロミン２／マーリン（ｎｆ２）遺伝子療法のための材料及び方法 Download PDF

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JP2024515629A
JP2024515629A JP2023562895A JP2023562895A JP2024515629A JP 2024515629 A JP2024515629 A JP 2024515629A JP 2023562895 A JP2023562895 A JP 2023562895A JP 2023562895 A JP2023562895 A JP 2023562895A JP 2024515629 A JP2024515629 A JP 2024515629A
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cells
seq
expression
polynucleotide
gene therapy
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JP2024515629A5 (https=
Inventor
ロン－シェン チャン，
ケビン フラニガン，
シビ リキッテ，
キャスリン メイヤー，
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リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル
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Publication of JP2024515629A publication Critical patent/JP2024515629A/ja
Publication of JP2024515629A5 publication Critical patent/JP2024515629A5/ja
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    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P35/00—Antineoplastic agents
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    • C07—ORGANIC CHEMISTRY
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    • 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
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/206—Animal model comprising tissue-specific expression system, e.g. tissue specific expression of transgene, of Cre recombinase
    • 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
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Psychology (AREA)
  • Toxicology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Dermatology (AREA)
  • Oncology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2023562895A 2021-04-14 2022-04-13 ニューロフィブロミン２／マーリン（ｎｆ２）遺伝子療法のための材料及び方法 Pending JP2024515629A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163174803P 2021-04-14 2021-04-14
US63/174,803 2021-04-14
PCT/US2022/024680 WO2022221447A1 (en) 2021-04-14 2022-04-13 Materials and methods for neurofibromin 2/merlin (nf2) gene therapy

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JP2024515629A true JP2024515629A (ja) 2024-04-10
JP2024515629A5 JP2024515629A5 (https=) 2025-04-22

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JP2023562895A Pending JP2024515629A (ja) 2021-04-14 2022-04-13 ニューロフィブロミン２／マーリン（ｎｆ２）遺伝子療法のための材料及び方法

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US (1) US20240189453A1 (https=)
EP (1) EP4323014A4 (https=)
JP (1) JP2024515629A (https=)
AU (1) AU2022257000A1 (https=)
CA (1) CA3216712A1 (https=)
WO (1) WO2022221447A1 (https=)

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH07143884A (ja) * 1993-02-25 1995-06-06 General Hospital Corp:The 腫瘍サプレッサー遺伝子メルリンおよびその用途
WO2012013366A2 (en) * 2010-07-28 2012-02-02 Closed Stock Company "Institute Of Applied Nanotechnology" Preventive bactericidal polish for nail treatment

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5578462A (en) * 1994-01-10 1996-11-26 Bristol-Myers Squibb Company NF2 isoforms
US20130137753A1 (en) * 2010-08-03 2013-05-30 University Of South Alabama Methods and compositions for the diagnosis and treatment of breast cancer
WO2015171270A1 (en) * 2014-05-06 2015-11-12 Felder Mitchell S Method for the treatment of neurofibromatosis
SG10201913266UA (en) * 2015-12-11 2020-02-27 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
JP2024520815A (ja) * 2021-06-08 2024-05-24 エヌエフ２ セラピューティクス， インコーポレイテッド 神経線維腫性障害を処置するための組成物および方法

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH07143884A (ja) * 1993-02-25 1995-06-06 General Hospital Corp:The 腫瘍サプレッサー遺伝子メルリンおよびその用途
WO2012013366A2 (en) * 2010-07-28 2012-02-02 Closed Stock Company "Institute Of Applied Nanotechnology" Preventive bactericidal polish for nail treatment

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
BIOCHIMICA ET BIOPHYSICA ACTA, vol. 1859, JPN6026011221, 2016, pages 599 - 611, ISSN: 0005821257 *
MOL. CELLS, vol. 20, no. 2, JPN6026011222, 2005, pages 196 - 200, ISSN: 0005821258 *
NAT BIOTECHNOL., vol. 27, no. 1, JPN6026011223, 2009, pages 59 - 65, ISSN: 0005821259 *

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Publication number Publication date
CA3216712A1 (en) 2022-10-20
US20240189453A1 (en) 2024-06-13
AU2022257000A1 (en) 2023-11-30
AU2022257000A9 (en) 2023-12-07
WO2022221447A4 (en) 2022-12-22
EP4323014A4 (en) 2025-08-06
EP4323014A1 (en) 2024-02-21
WO2022221447A1 (en) 2022-10-20

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