JP7602454B2 - 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用 - Google Patents

核酸分子及び非ウイルス遺伝子治療のためのそれらの使用 Download PDF

Info

Publication number
JP7602454B2
JP7602454B2 JP2021506753A JP2021506753A JP7602454B2 JP 7602454 B2 JP7602454 B2 JP 7602454B2 JP 2021506753 A JP2021506753 A JP 2021506753A JP 2021506753 A JP2021506753 A JP 2021506753A JP 7602454 B2 JP7602454 B2 JP 7602454B2
Authority
JP
Japan
Prior art keywords
itr
nucleic acid
sequence
seq
nucleotides
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2021506753A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021533753A (ja
JP2021533753A5 (enExample
Inventor
ダグラス・ドレイガー
ジァイン・リウ
トンヤオ・リウ
スザンナ・パタロヨ－ホワイト
ロバート・ティ・ピーターズ
アレクシー・セレジン
フィリップ・ザカス
Original Assignee
バイオベラティブ セラピューティクス インコーポレイテッド
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 バイオベラティブ セラピューティクス インコーポレイテッド filed Critical バイオベラティブ セラピューティクス インコーポレイテッド
Publication of JP2021533753A publication Critical patent/JP2021533753A/ja
Publication of JP2021533753A5 publication Critical patent/JP2021533753A5/ja
Application granted granted Critical
Publication of JP7602454B2 publication Critical patent/JP7602454B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0016—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • C—CHEMISTRY; METALLURGY
    • 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
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/31—Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14043—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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14211—Erythrovirus, e.g. B19 virus
    • C12N2750/14241—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14243—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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14311—Parvovirus, e.g. minute virus of mice
    • C12N2750/14341—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14343—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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14311—Parvovirus, e.g. minute virus of mice
    • C12N2750/14371—Demonstrated in vivo effect
    • 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
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2021506753A 2018-08-09 2019-08-09 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用 Active JP7602454B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862716826P 2018-08-09 2018-08-09
US62/716,826 2018-08-09
PCT/US2019/045957 WO2020033863A1 (en) 2018-08-09 2019-08-09 Nucleic acid molecules and uses thereof for non-viral gene therapy

Publications (3)

Publication Number Publication Date
JP2021533753A JP2021533753A (ja) 2021-12-09
JP2021533753A5 JP2021533753A5 (enExample) 2022-08-12
JP7602454B2 true JP7602454B2 (ja) 2024-12-18

Family

ID=67766358

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021506753A Active JP7602454B2 (ja) 2018-08-09 2019-08-09 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用

Country Status (15)

Country Link
US (2) US12364774B2 (enExample)
EP (1) EP3833766A1 (enExample)
JP (1) JP7602454B2 (enExample)
KR (2) KR102925832B1 (enExample)
CN (2) CN113227385B (enExample)
AU (2) AU2019319984B2 (enExample)
BR (1) BR112021002017A2 (enExample)
CA (1) CA3108799A1 (enExample)
CO (1) CO2021002965A2 (enExample)
IL (1) IL280652A (enExample)
MX (2) MX2021001599A (enExample)
PH (1) PH12021500010A1 (enExample)
SG (1) SG11202101157VA (enExample)
TW (2) TW202547562A (enExample)
WO (1) WO2020033863A1 (enExample)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RS61387B2 (sr) 2013-02-15 2024-06-28 Bioverativ Therapeutics Inc Gen optimizovanog faktora viii
MX2020001593A (es) 2017-08-09 2020-07-13 Bioverativ Therapeutics Inc Moleculas de acido nucleico y usos de las mismas.
WO2020019002A1 (en) 2018-07-20 2020-01-23 University Of Utah Research Foundation Gene therapy for macular degeneration
JP7602454B2 (ja) 2018-08-09 2024-12-18 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
SG11202104634UA (en) * 2018-11-07 2021-06-29 Vivet Therapeutics Codon-optimized abcb11 transgene for the treatment of progressive familial intrahepatic cholestasis type 2 (pfic2)
CN111334528B (zh) * 2020-03-20 2022-02-18 山东农业大学 水貂肠炎细小病毒全基因组感染性克隆及其构建方法和应用
MX2023001109A (es) 2020-07-27 2023-04-24 Anjarium Biosciences Ag Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.
AU2021330936A1 (en) * 2020-08-23 2023-05-04 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
CN112704729A (zh) * 2020-12-28 2021-04-27 江苏纳迪芯生命科技研究院有限公司 一种细胞因子颗粒
WO2022198025A2 (en) * 2021-03-19 2022-09-22 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing pfic therapeutics
CN113325181A (zh) * 2021-04-25 2021-08-31 苏州市立医院(北区) 丝氨酸蛋白酶抑制剂在用于脓毒症早期预警的标志物中的应用
WO2022232029A2 (en) * 2021-04-26 2022-11-03 University Of Florida Research Foundation, Incorporated Synthetic aav vectors for repeated delivery of therapeutic genes
US20240287493A1 (en) * 2021-06-24 2024-08-29 University Of Utah Research Foundation Compositions and methods for treating pgm1 deficiency
WO2023028440A2 (en) 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Baculovirus expression system
US20230084036A1 (en) * 2021-08-23 2023-03-16 Bioverativ Therapeutics Inc. Optimized factor viii genes
AU2022334714A1 (en) * 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Closed-end dna production with inverted terminal repeat sequences
JP2024532261A (ja) * 2021-08-23 2024-09-05 バイオベラティブ セラピューティクス インコーポレイテッド 操作ｉｔｒ配列および使用方法
CN122521785A (zh) * 2021-09-16 2026-08-07 北京锦篮基因科技股份有限公司 用于治疗ⅱ型粘多糖贮积症的重组腺相关病毒载体及其应用
KR20240067122A (ko) * 2021-09-30 2024-05-16 바이오버라티브 테라퓨틱스 인크. 감소된 면역원성을 갖는 인자 viii 폴리펩타이드를 암호화하는 핵산
CN114250224B (zh) * 2021-12-16 2024-02-27 福建和瑞基因科技有限公司 一种用于提取或检测样本中小分子rna的核酸组合物及其试剂盒和方法
WO2023135273A2 (en) * 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
FR3136367A1 (fr) * 2022-06-09 2023-12-15 Damien SULEIMAN Compositions et méthodes concernant des molécules d’ARN messager codant le facteur V encapsulées dans des nanoparticules lipidiques
CN119233998A (zh) * 2022-07-07 2024-12-31 深圳新诺微环生物科技有限公司 一种连接肽、包含连接肽的凝血八因子蛋白或其变体及其用途
JP2025525583A (ja) 2022-07-19 2025-08-05 ランパート バイオサイエンス インコーポレイテッド 非免疫原性環状非ウイルス性ｄｎａベクター
CN116064673A (zh) * 2022-10-19 2023-05-05 河南省人民医院 表达rs1的重组腺相关病毒载体及其应用
CN120981563A (zh) * 2023-03-24 2025-11-18 南京金斯瑞生物科技有限公司 一种提升质粒稳定性的宿主细胞

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008510489A (ja) 2004-08-23 2008-04-10 ニュークレオニクス・インコーポレイテッド 多重ｒｎａポリメラーゼｉｉｉプロモーター発現構築物
JP2008513020A (ja) 2004-09-17 2008-05-01 ユニバーシティ オブ ユタ リサーチ ファウンデーション 導入遺伝子発現の画像レポーター
JP2011067212A (ja) 1998-11-10 2011-04-07 Univ Of North Carolina At Chapel Hill ハイブリッドウイルス粒子とその製造方法
JP2014511690A (ja) 2011-04-07 2014-05-19 ヘルムホルツ−ツェントルム フュア インフェクツィオンスフォルシュンク ゲーエムベーハー 肝再生および肝不全の治療のための薬剤
US20170130245A1 (en) 2014-06-09 2017-05-11 Voyager Therapeutics, Inc. Chimeric capsids
JP2018509141A (ja) 2015-02-06 2018-04-05 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 最適化されたヒト凝固第ｖｉｉｉ因子遺伝子発現カセットおよびその使用

Family Cites Families (211)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4965188A (en) 1986-08-22 1990-10-23 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences using a thermostable enzyme
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
MX9203440A (es) 1985-04-12 1992-07-01 Genetics Inst Proteinas procoagulantes novedosas.
WO1986006408A1 (en) 1985-04-22 1986-11-06 Genetics Institute, Inc. HIGH YIElD PRODUCTION OF ACTIVE FACTOR IX
KR910006424B1 (ko) 1985-08-21 1991-08-24 인코텍스 비.브이 편성브리프(brief) 제조방법
ATE87663T1 (de) 1986-01-03 1993-04-15 Genetics Inst Verfahren zur herstellung von faktor-viii:c-typ- proteinen.
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US4800159A (en) 1986-02-07 1989-01-24 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences
US5543502A (en) 1986-06-24 1996-08-06 Novo Nordisk A/S Process for producing a coagulation active complex of factor VIII fragments
EP0279582A3 (en) 1987-02-17 1989-10-18 Pharming B.V. Dna sequences to target proteins to the mammary gland for efficient secretion
JP3101690B2 (ja) 1987-03-18 2000-10-23 エス・ビィ・２・インコーポレイテッド 変性抗体の、または変性抗体に関する改良
US6060447A (en) 1987-05-19 2000-05-09 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US6346513B1 (en) 1987-06-12 2002-02-12 Baxter Trading Gmbh Proteins with factor VIII activity: process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them
IE69026B1 (en) 1987-06-12 1996-08-07 Immuno Ag Novel proteins with factor VIII activity process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them
DE3720246A1 (de) 1987-06-19 1988-12-29 Behringwerke Ag Faktor viii:c-aehnliches molekuel mit koagulationsaktivitaet
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
US6780613B1 (en) 1988-10-28 2004-08-24 Genentech, Inc. Growth hormone variants
DE122010000006I2 (de) 1989-02-21 2012-06-21 Univ Washington Modifizierte formen von fortpflanzungshormonen.
US5633076A (en) 1989-12-01 1997-05-27 Pharming Bv Method of producing a transgenic bovine or transgenic bovine embryo
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning
IE922437A1 (en) 1991-07-25 1993-01-27 Idec Pharma Corp Recombinant antibodies for human therapy
US5736137A (en) 1992-11-13 1998-04-07 Idec Pharmaceuticals Corporation Therapeutic application of chimeric and radiolabeled antibodies to human B lymphocyte restricted differentiation antigen for treatment of B cell lymphoma
CA2149326C (en) 1992-11-13 2007-04-17 Mitchell E. Reff Fully impaired consensus kozak sequences for mammalian expression
CA2162271A1 (en) 1993-05-26 1994-12-08 Robert Kotin Fusion proteins containing adeno-associated virus rep protein and bacterial protein
SE504074C2 (sv) 1993-07-05 1996-11-04 Pharmacia Ab Proteinberedning för subkutan, intramuskulär eller intradermal administrering
AU5351494A (en) 1993-10-07 1995-05-01 G.D. Searle & Co. Method of producing recombinant eukaryotic viruses in bacteria
US5827690A (en) 1993-12-20 1998-10-27 Genzyme Transgenics Corporatiion Transgenic production of antibodies in milk
FR2716893B1 (fr) 1994-03-03 1996-04-12 Rhone Poulenc Rorer Sa Virus recombinants, leur préparation et leur utilisation thérapeutique.
GB9422383D0 (en) 1994-11-05 1995-01-04 Wellcome Found Antibodies
US6485726B1 (en) 1995-01-17 2002-11-26 The Brigham And Women's Hospital, Inc. Receptor specific transepithelial transport of therapeutics
US6086875A (en) 1995-01-17 2000-07-11 The Brigham And Women's Hospital, Inc. Receptor specific transepithelial transport of immunogens
US6030613A (en) 1995-01-17 2000-02-29 The Brigham And Women's Hospital, Inc. Receptor specific transepithelial transport of therapeutics
US5869046A (en) 1995-04-14 1999-02-09 Genentech, Inc. Altered polypeptides with increased half-life
US6096871A (en) 1995-04-14 2000-08-01 Genentech, Inc. Polypeptides altered to contain an epitope from the Fc region of an IgG molecule for increased half-life
US5739277A (en) 1995-04-14 1998-04-14 Genentech Inc. Altered polypeptides with increased half-life
US6121022A (en) 1995-04-14 2000-09-19 Genentech, Inc. Altered polypeptides with increased half-life
SE9503380D0 (sv) 1995-09-29 1995-09-29 Pharmacia Ab Protein derivatives
GB9524973D0 (en) 1995-12-06 1996-02-07 Lynxvale Ltd Viral vectors
US6458563B1 (en) 1996-06-26 2002-10-01 Emory University Modified factor VIII
AU3968897A (en) 1996-08-02 1998-02-25 Bristol-Myers Squibb Company A method for inhibiting immunoglobulin-induced toxicity resulting from the use of immunoglobulins in therapy and in vivo diagnosis
WO1998023289A1 (en) 1996-11-27 1998-06-04 The General Hospital Corporation MODULATION OF IgG BINDING TO FcRn
US6277375B1 (en) 1997-03-03 2001-08-21 Board Of Regents, The University Of Texas System Immunoglobulin-like domains with increased half-lives
CA2225189C (en) 1997-03-06 2010-05-25 Queen's University At Kingston Canine factor viii gene, protein and methods of use
RO120148B1 (ro) 1997-03-14 2005-09-30 Idec Pharmaceuticals Corporation Sistem vector şi procedeu de inserţie a unui fragment adn, în genomul celulelor de mamifere
GB9722131D0 (en) 1997-10-20 1997-12-17 Medical Res Council Method
CA2323757C (en) 1998-04-02 2011-08-02 Genentech, Inc. Antibody variants and fragments thereof
US6242195B1 (en) 1998-04-02 2001-06-05 Genentech, Inc. Methods for determining binding of an analyte to a receptor
US6194551B1 (en) 1998-04-02 2001-02-27 Genentech, Inc. Polypeptide variants
US6528624B1 (en) 1998-04-02 2003-03-04 Genentech, Inc. Polypeptide variants
GB9809951D0 (en) 1998-05-08 1998-07-08 Univ Cambridge Tech Binding molecules
US6291214B1 (en) 1998-05-11 2001-09-18 Glaxo Wellcome Inc. System for generating recombinant viruses
JP2002522013A (ja) 1998-07-24 2002-07-23 ベイラ、カリッジ、アヴ、メダスン 部位特異的組換えを使用する迅速なサブクローニング
JP2002522063A (ja) 1998-08-17 2002-07-23 アブジェニックス インコーポレイテッド 増加した血清半減期を有する改変された分子の生成
US20020065236A1 (en) 1998-09-09 2002-05-30 Yew Nelson S. CpG reduced plasmids and viral vectors
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
EP1006183A1 (en) 1998-12-03 2000-06-07 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Recombinant soluble Fc receptors
US6737056B1 (en) 1999-01-15 2004-05-18 Genentech, Inc. Polypeptide variants with altered effector function
HUP0104865A3 (en) 1999-01-15 2004-07-28 Genentech Inc Polypeptide variants with altered effector function
US6926898B2 (en) 2000-04-12 2005-08-09 Human Genome Sciences, Inc. Albumin fusion proteins
WO2002040544A2 (en) 2000-11-14 2002-05-23 Board Of Regents, University Of Texas Systems Mutant human factor ix with an increased resistance to inhibition by heparin
GB0029407D0 (en) 2000-12-01 2001-01-17 Affitech As Product
EP1355919B1 (en) 2000-12-12 2010-11-24 MedImmune, LLC Molecules with extended half-lives, compositions and uses thereof
US7211395B2 (en) 2001-03-09 2007-05-01 Dyax Corp. Serum albumin binding moieties
WO2002092134A1 (en) 2001-05-14 2002-11-21 Cell Genesys, Inc. Lentiviral vectors encoding clotting factors for gene therapy
CA2457429A1 (en) 2001-09-04 2003-03-13 Francis J. Carr Modified factor ix
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
KR101271635B1 (ko) 2001-12-21 2013-06-12 휴먼 게놈 사이언시즈, 인코포레이티드 알부민 융합 단백질
CA2841097A1 (en) 2001-12-21 2003-07-24 Human Genome Sciences, Inc. Albumin and g-csf fusion proteins
US20080194481A1 (en) 2001-12-21 2008-08-14 Human Genome Sciences, Inc. Albumin Fusion Proteins
US20040002587A1 (en) 2002-02-20 2004-01-01 Watkins Jeffry D. Fc region variants
US7317091B2 (en) 2002-03-01 2008-01-08 Xencor, Inc. Optimized Fc variants
US20040018557A1 (en) 2002-03-01 2004-01-29 Immunomedics, Inc. Bispecific antibody point mutations for enhancing rate of clearance
US20040132101A1 (en) 2002-09-27 2004-07-08 Xencor Optimized Fc variants and methods for their generation
CA2479212A1 (en) 2002-03-15 2003-09-25 Brigham And Women's Hospital, Inc. Central airway administration for systemic delivery of therapeutics
PT1534335E (pt) 2002-08-14 2012-02-28 Macrogenics Inc Anticorpos específicos de fcγriib e processos para a sua utilização
CA2832136C (en) 2002-09-27 2015-11-17 Xencor Optimized fc variants and methods for their generation
ATE480562T1 (de) 2002-10-15 2010-09-15 Facet Biotech Corp Veränderung von fcrn-bindungsaffinitäten oder von serumhalbwertszeiten von antikörpern mittels mutagenese
GB2395337B (en) 2002-11-14 2005-12-28 Gary Michael Wilson Warning Unit
US7355008B2 (en) 2003-01-09 2008-04-08 Macrogenics, Inc. Identification and engineering of antibodies with variant Fc regions and methods of using same
US7041635B2 (en) 2003-01-28 2006-05-09 In2Gen Co., Ltd. Factor VIII polypeptide
US8388955B2 (en) 2003-03-03 2013-03-05 Xencor, Inc. Fc variants
US20090010920A1 (en) 2003-03-03 2009-01-08 Xencor, Inc. Fc Variants Having Decreased Affinity for FcyRIIb
WO2004094642A2 (en) 2003-04-24 2004-11-04 Fondazione Centro San Raffaele Del Monte Tabor Lentiviral vectors carrying synthetic bi-directional promoters and uses thereof
TWI353991B (en) 2003-05-06 2011-12-11 Syntonix Pharmaceuticals Inc Immunoglobulin chimeric monomer-dimer hybrids
GB0324368D0 (en) 2003-10-17 2003-11-19 Univ Cambridge Tech Polypeptides including modified constant regions
EP1697520A2 (en) 2003-12-22 2006-09-06 Xencor, Inc. Fc polypeptides with novel fc ligand binding sites
JP4762156B2 (ja) 2004-01-12 2011-08-31 アプライド モレキュラー エボリューション，インコーポレイテッド Ｆｃ領域変異体
US20080004228A1 (en) 2004-03-09 2008-01-03 Imre Berger New Expression Tools for Multiprotein Applications
US7276585B2 (en) 2004-03-24 2007-10-02 Xencor, Inc. Immunoglobulin variants outside the Fc region
WO2005123780A2 (en) 2004-04-09 2005-12-29 Protein Design Labs, Inc. Alteration of fcrn binding affinities or serum half-lives of antibodies by mutagenesis
WO2006085967A2 (en) 2004-07-09 2006-08-17 Xencor, Inc. OPTIMIZED ANTI-CD20 MONOCONAL ANTIBODIES HAVING Fc VARIANTS
US7598071B2 (en) * 2004-07-09 2009-10-06 The United States Of America As Represented By The Department Of Health And Human Services Infectious clone of human parvovirus B19 and methods
EP3342782B1 (en) 2004-07-15 2022-08-17 Xencor, Inc. Optimized fc variants
WO2006047350A2 (en) 2004-10-21 2006-05-04 Xencor, Inc. IgG IMMUNOGLOBULIN VARIANTS WITH OPTIMIZED EFFECTOR FUNCTION
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
WO2006126972A1 (en) * 2005-05-27 2006-11-30 Agency For Science, Technology And Research Method of delivering nucleic acid molecules into embryonic stem cells using baculoviral vectors
DK2002003T3 (en) 2005-05-27 2016-03-21 Ospedale San Raffaele Srl Gene vector comprising miRNA
CA2618476A1 (en) 2005-08-12 2007-02-22 Human Genome Sciences, Inc. Albumin fusion proteins
AU2006314843B2 (en) 2005-11-17 2011-06-30 Karolinska Institutet Innovations Ab Human bocavirus and methods of diagnosis and treatment
WO2008016391A2 (en) * 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
US8048848B2 (en) 2006-02-03 2011-11-01 Prolor Biotech Ltd. Long-acting interferons and derivatives thereof and methods thereof
WO2007140011A2 (en) 2006-05-26 2007-12-06 The Government Of The United States Of America Erythroid progenitor cells and methods for producing parvovirus b19 therein
US7939632B2 (en) 2006-06-14 2011-05-10 Csl Behring Gmbh Proteolytically cleavable fusion proteins with high molar specific activity
EP2423307A1 (en) 2006-06-19 2012-02-29 Catalyst Biosciences, Inc. Modified coagulation factor IV polypeptides and use thereof for treatment
GB0614780D0 (en) 2006-07-25 2006-09-06 Ucb Sa Biological products
ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
WO2008033413A2 (en) 2006-09-14 2008-03-20 Human Genome Sciences, Inc. Albumin fusion proteins
US7700734B2 (en) 2007-01-09 2010-04-20 Shu-Wha Lin Recombinant human factor IX and use thereof
CA2682605A1 (en) 2007-04-18 2008-10-30 Zymogenetics, Inc. Single chain fc, methods of making and methods of treatment
MX2009012343A (es) 2007-05-14 2010-02-10 Biogen Idec Inc Regiones fc (sc fc) de cadena sencilla, polipeptidos de enlace que comprenden las mismas, y metodos relacionados con ello.
WO2008141400A1 (en) 2007-05-24 2008-11-27 Medvet Science Pty Ltd A novel aetiologic agent of acute gastroenteritis (age), diagnostic methods and therapeutic treatment thereof
US8563521B2 (en) 2007-06-21 2013-10-22 Technische Universitat Munchen Biological active proteins having increased in vivo and/or in vitro stability
US9115373B2 (en) 2007-09-19 2015-08-25 Uniqure Ip B.V. Use of AAV replication machinery for improved protein production
AU2008311973B2 (en) 2007-10-15 2013-10-03 Cangene Corporation Human Factor IX variants with an extended half life
KR20110015551A (ko) 2008-04-16 2011-02-16 바이엘 헬스케어 엘엘씨 제ｉｘ인자의 부위-지정 변형
KR20110005862A (ko) 2008-04-16 2011-01-19 바이엘 헬스케어 엘엘씨 변형된 제ｉｘ인자 폴리펩티드 및 이의 용도
EP2274420A4 (en) 2008-04-17 2013-09-25 Blood Systems Inc NEW HUMAN PARVOVIRUS: THE BOCAVIRUS
EP2268807A2 (en) 2008-04-21 2011-01-05 Novo Nordisk A/S Hyperglycosylated human coagulation factor ix
GB0820631D0 (en) 2008-11-11 2008-12-17 London School Hygiene & Tropical Medicine Vectors
US9938540B2 (en) 2008-11-12 2018-04-10 Ospedale San Raffaele S.R.L. Gene vector for inducing transgene-specific immune tolerance
DK3178835T3 (da) 2009-02-03 2019-06-24 Amunix Pharmaceuticals Inc Forlængede rekombinante polypeptider og sammensætninger omfattende samme
US8680050B2 (en) 2009-02-03 2014-03-25 Amunix Operating Inc. Growth hormone polypeptides fused to extended recombinant polypeptides and methods of making and using same
US8703717B2 (en) 2009-02-03 2014-04-22 Amunix Operating Inc. Growth hormone polypeptides and methods of making and using same
MX2011011508A (es) 2009-04-30 2012-02-13 San Raffaele Centro Fond Vector genico.
US20120134977A1 (en) 2009-06-01 2012-05-31 Yeda Research And Development Co. Ltd. Prodrugs containing albumin binding probe
PE20121539A1 (es) 2009-06-08 2012-12-06 Amunix Operating Inc Polipeptidos de la hormona de crecimiento y metodos de preparacion
WO2010144508A1 (en) 2009-06-08 2010-12-16 Amunix Operating Inc. Glucose-regulating polypeptides and methods of making and using same
GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
CA2771999A1 (en) 2009-08-24 2011-03-10 Amunix Operating Inc. Coagulation factor vii compositions and methods of making and using same
WO2011028344A2 (en) 2009-08-25 2011-03-10 Amunix Operating Inc. Interleukin-1 receptor antagonist compositions and methods of making and using same
US9169494B2 (en) * 2010-01-12 2015-10-27 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
EP2534172A2 (en) 2010-02-12 2012-12-19 The U.S.A. as represented by the Secretary, Department of Health and Human Services Compositions and methods for preventing or treating a human parvovirus infection
WO2011112090A2 (en) 2010-03-11 2011-09-15 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Method for identifying variant rep protein encoding nucleic acids
MX375112B (es) 2010-07-09 2025-03-06 Biogen Hemophilia Inc Polipéptidos de factor ix y métodos para usarlos.
PT2591006T (pt) 2010-07-09 2019-07-29 Bioverativ Therapeutics Inc Moléculas de cadeia simples processáveis e polipéptidos preparados utilizando as mesmas
JP6091435B2 (ja) 2011-02-22 2017-03-08 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス（ａａｖ）ベクターを用いたタンパク質の送達
EP2500434A1 (en) * 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
HRP20191920T1 (hr) 2012-01-12 2020-01-10 Bioverativ Therapeutics Inc. Kimerni polipeptidi faktora viii i njihova uporaba
EA038705B1 (ru) 2012-01-12 2021-10-07 Биовератив Терапьютикс Инк. Способы снижения иммуногенности фактора свёртывания крови viii у пациентов, получающих лечение фактором viii
CN111548418A (zh) 2012-02-15 2020-08-18 比奥贝拉蒂治疗公司 因子viii组合物及其制备和使用方法
WO2013123457A1 (en) 2012-02-15 2013-08-22 Biogen Idec Ma Inc. Recombinant factor viii proteins
BR112014020325A2 (pt) 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
ES2770501T3 (es) 2012-07-11 2020-07-01 Bioverativ Therapeutics Inc Complejo del factor VIII con XTEN y proteína del factor de Von Willebrand y sus usos
AU2013331000B2 (en) * 2012-10-18 2018-04-19 Bioverativ Therapeutics Inc. Methods of using a fixed dose of a clotting factor
EP2914731B1 (en) 2012-11-01 2019-09-11 California Institute of Technology Reversible gene expression
CN103215308B (zh) 2013-02-01 2015-12-23 中国科学院苏州生物医学工程技术研究所 表达重组人fviii的整合质粒、细胞株及其构建方法和应用
RS61387B2 (sr) 2013-02-15 2024-06-28 Bioverativ Therapeutics Inc Gen optimizovanog faktora viii
ES2926798T3 (es) 2013-03-15 2022-10-28 Bioverativ Therapeutics Inc Formulaciones de polipéptido de factor VIII
EP2970966A4 (en) * 2013-03-15 2016-10-26 Univ North Carolina SYNTHETIC ADENOASSOZED VIRUS INVERTED REPEAT SEQUENCES
AU2014251099B2 (en) 2013-04-08 2019-01-17 The University Of Kansas Chimeric adeno-associated virus/ bocavirus parvovirus vector
US10548953B2 (en) 2013-08-14 2020-02-04 Bioverativ Therapeutics Inc. Factor VIII-XTEN fusions and uses thereof
US20160311885A1 (en) * 2013-08-14 2016-10-27 Biogen Ma Inc. Recombinant factor viii proteins
ES2813698T3 (es) 2013-09-12 2021-03-24 Biomarin Pharm Inc Vectores de AAV que comprenden un gen que codifica el factor VIII
EA201691111A1 (ru) 2014-01-10 2017-01-30 Байоджен Ма Инк. Химерные белки фактора viii и их применение
BR112016020688B1 (pt) 2014-03-09 2024-01-30 The Trustees Of The University Of Pennsylvania Vetores virais recombinantes, vírus adenoassociado recombinante, composição farmacêutica, bem como uso dos mesmos
CR20160519A (es) * 2014-05-13 2017-01-27 The Trustees Of The Univ Of Pennysylvania Composiciones que comprenden avv que expresa constructos de anticuerpos duales y sus usos
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
EP3221456B1 (en) 2014-11-21 2021-09-22 University of Florida Research Foundation, Inc. Genome-modified recombinant adeno-associated virus vectors
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
WO2016134338A1 (en) 2015-02-19 2016-08-25 University Of Florida Research Foundation, Inc. Recombinant aav vectors for gene therapy of human hematopoietic disorders
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
AU2016260401B2 (en) 2015-05-14 2022-05-19 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers and methods of use thereof
PE20231949A1 (es) 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
KR20180118659A (ko) 2016-02-01 2018-10-31 바이오버라티브 테라퓨틱스 인크. 최적화된 viii 인자 유전자
IL261524B2 (en) 2016-03-03 2023-10-01 Univ Massachusetts End-capped linear double-stranded DNA for non-viral gene transfer
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1
KR102219859B1 (ko) 2016-06-24 2021-02-25 재단법인 목암생명과학연구소 재조합 단쇄 fviii 및 그 화학 접합물
FR3054841B1 (fr) 2016-08-05 2021-01-29 Centre Nat Rech Scient Systeme d'expression baculovirus
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
RU2020106730A (ru) 2017-07-14 2021-08-16 Онкорус, Инк. Инкапсулированные полинуклеотиды и способы их применения
US11549125B2 (en) 2017-08-09 2023-01-10 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Closed-ended, linear, duplex adenoassociated virus DNA, and uses thereof
MX2020001593A (es) 2017-08-09 2020-07-13 Bioverativ Therapeutics Inc Moleculas de acido nucleico y usos de las mismas.
CA3075168A1 (en) 2017-09-08 2019-03-14 Generation Bio Co. Modified closed-ended dna (cedna)
EP3679148A4 (en) 2017-09-08 2021-06-09 Generation Bio Co. LIPID NANOPARTICLE FORMULATIONS FROM NONVIRAL, CAPSID-FREE DNA VECTORS
KR20200057051A (ko) 2017-09-27 2020-05-25 시질론 테라퓨틱스, 인크. 활성 세포를 포함하는 방법, 조성물 및 이식 가능한 요소
JP2021505159A (ja) 2017-12-06 2021-02-18 ジェネレーション バイオ カンパニー 修飾型閉端ｄｎａ（ｃｅｄｎａ）を使用する遺伝子編集
JP2021511047A (ja) 2018-01-19 2021-05-06 ジェネレーション バイオ カンパニー 無細胞合成から得ることができる閉端ＤＮＡベクターおよびｃｅＤＮＡベクターを得るためのプロセス
AU2019221642A1 (en) 2018-02-14 2020-07-09 Generation Bio Co. Non-viral DNA vectors and uses thereof for antibody and fusion protein production
EP3755803A4 (en) 2018-02-22 2022-01-19 Generation Bio Co. CONTROLLED EXPRESSION OF TRANSGENES USING DNA (CEDNA) VECTORS
CA3092459A1 (en) 2018-03-02 2019-09-06 Generation Bio Co. Closed-ended dna (cedna) vectors for insertion of transgenes at genomic safe harbors (gsh) in humans and murine genomes
CA3092832A1 (en) 2018-03-02 2019-09-06 Generation Bio Co. Identifying and characterizing genomic safe harbors (gsh) in humans and murine genomes, and viral and non-viral vector compositions for targeted integration at an identified gsh loci
EP3762500A1 (en) 2018-03-06 2021-01-13 Voyager Therapeutics, Inc. Insect cell manufactured partial self-complementary aav genomes
CN112639110A (zh) 2018-06-22 2021-04-09 阿斯克肋匹奥生物制药公司 用于基因递送以在细胞内持续存在的载体
JP7602454B2 (ja) 2018-08-09 2024-12-18 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
US20220042040A1 (en) 2018-09-17 2022-02-10 Universität Für Bodenkultur Wien Dual vector system for improved production of proteins in animal cells
UY38389A (es) 2018-09-27 2020-04-30 Sigilon Therapeutics Inc Dispositivos implantables para terapia celular y métodos relacionados
CN120349985A (zh) 2018-10-12 2025-07-22 建新公司 通过肝导向基因替代疗法治疗严重pku的改良的人pah的产生
WO2020097417A1 (en) 2018-11-09 2020-05-14 Generation Bio Co. Modified closed-ended dna (cedna) comprising symmetrical modified inverted terminal repeats
KR20210119416A (ko) 2019-01-24 2021-10-05 제너레이션 바이오 컴퍼니 폐쇄-말단 dna (cedna), 및 유전자 또는 핵산 치료 관련 면역 반응을 감소시키는 방법에서의 이의 용도
FR3092522B1 (fr) 2019-02-07 2021-09-10 Rubix S&I Système et procédé de nettoyage d’un habitacle de véhicule
CA3129321A1 (en) 2019-02-15 2020-08-20 Generation Bio Co. Modulation of rep protein activity in closed-ended dna (cedna) production
SG11202108864UA (en) 2019-03-06 2021-09-29 Generation Bio Co Closed-ended dna (cedna) and immune modulating compounds
KR20210151785A (ko) 2019-03-13 2021-12-14 제너레이션 바이오 컴퍼니 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
PL3722434T3 (pl) 2019-04-12 2022-12-12 Freeline Therapeutics Limited Układ plazmidowy
CA3136545A1 (en) 2019-04-12 2020-10-15 Freeline Therapeutics Limited Plasmid system
EP3736286A1 (en) 2019-05-09 2020-11-11 Biotest AG Single chain factor viii molecule
EP3987043A4 (en) 2019-06-21 2023-07-19 Asklepios Biopharmaceutical, Inc. PRODUCTION OF VECTORS USING THE PHAGEN REPLICATION ORIGIN
US20220228171A1 (en) 2019-07-17 2022-07-21 Generation Bio Co. Compositions and production of nicked closed-ended dna vectors
WO2021216975A1 (en) 2020-04-23 2021-10-28 Aav Gene Therapeutics, Inc. Aav native-neuro platform and use for neuronal disease gene therapy
MX2023001109A (es) 2020-07-27 2023-04-24 Anjarium Biosciences Ag Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.
US20220356490A1 (en) 2020-08-23 2022-11-10 Bioverativ Therapeutics Inc. Baculovirus expression system
US20220243201A1 (en) 2020-08-23 2022-08-04 Bioverativ Therapeutics Inc. Engineered itr sequences and methods of use
AU2021330936A1 (en) 2020-08-23 2023-05-04 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
AU2022334714A1 (en) 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Closed-end dna production with inverted terminal repeat sequences
WO2023028440A2 (en) 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Baculovirus expression system
US20230084036A1 (en) 2021-08-23 2023-03-16 Bioverativ Therapeutics Inc. Optimized factor viii genes
JP2024532261A (ja) 2021-08-23 2024-09-05 バイオベラティブ セラピューティクス インコーポレイテッド 操作ｉｔｒ配列および使用方法

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2011067212A (ja) 1998-11-10 2011-04-07 Univ Of North Carolina At Chapel Hill ハイブリッドウイルス粒子とその製造方法
JP2008510489A (ja) 2004-08-23 2008-04-10 ニュークレオニクス・インコーポレイテッド 多重ｒｎａポリメラーゼｉｉｉプロモーター発現構築物
JP2008513020A (ja) 2004-09-17 2008-05-01 ユニバーシティ オブ ユタ リサーチ ファウンデーション 導入遺伝子発現の画像レポーター
JP2014511690A (ja) 2011-04-07 2014-05-19 ヘルムホルツ−ツェントルム フュア インフェクツィオンスフォルシュンク ゲーエムベーハー 肝再生および肝不全の治療のための薬剤
US20170130245A1 (en) 2014-06-09 2017-05-11 Voyager Therapeutics, Inc. Chimeric capsids
JP2018509141A (ja) 2015-02-06 2018-04-05 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 最適化されたヒト凝固第ｖｉｉｉ因子遺伝子発現カセットおよびその使用

Also Published As

Publication number Publication date
AU2019319984B2 (en) 2026-02-12
CN113227385A (zh) 2021-08-06
BR112021002017A2 (pt) 2021-05-11
TW202547562A (zh) 2025-12-16
CN113227385B (zh) 2024-12-24
US12364774B2 (en) 2025-07-22
US20250269067A1 (en) 2025-08-28
CO2021002965A2 (es) 2021-03-19
CA3108799A1 (en) 2020-02-13
KR20210042128A (ko) 2021-04-16
MX2021001599A (es) 2021-07-02
KR20260030171A (ko) 2026-03-05
CN119955796A (zh) 2025-05-09
AU2026203633A1 (en) 2026-05-28
MX2026001238A (es) 2026-03-02
TW202023630A (zh) 2020-07-01
EP3833766A1 (en) 2021-06-16
JP2021533753A (ja) 2021-12-09
IL280652A (en) 2021-03-25
TWI875705B (zh) 2025-03-11
WO2020033863A1 (en) 2020-02-13
US20200069817A1 (en) 2020-03-05
KR102925832B1 (ko) 2026-02-11
AU2019319984A1 (en) 2021-03-04
SG11202101157VA (en) 2021-03-30
PH12021500010A1 (en) 2021-10-11

Similar Documents

Publication Publication Date Title
JP7688682B2 (ja) 核酸分子およびその使用
US20250269067A1 (en) Nucleic acid molecules and uses thereof for non-viral gene therapy
US20240350671A1 (en) Engineered itr sequences and methods of use
JP2024532262A (ja) 最適化第ｖｉｉｉ因子遺伝子
RU2819144C2 (ru) Молекулы нуклеиновой кислоты и пути их применения
RU2834625C2 (ru) Молекулы нуклеиновой кислоты и пути их применения для невирусной генной терапии

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220803

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220803

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20230607

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230801

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20231027

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240201

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20240611

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241011

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20241106

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20241126

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20241206

R150 Certificate of patent or registration of utility model

Ref document number: 7602454

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150