DK2956477T3 - Optimeret faktor viii-gen - Google Patents

Optimeret faktor viii-gen Download PDF

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Publication number
DK2956477T3
DK2956477T3 DK14751254.5T DK14751254T DK2956477T3 DK 2956477 T3 DK2956477 T3 DK 2956477T3 DK 14751254 T DK14751254 T DK 14751254T DK 2956477 T3 DK2956477 T3 DK 2956477T3
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DK
Denmark
Prior art keywords
gen
factor viii
optimized factor
optimized
viii
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DK14751254.5T
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English (en)
Other versions
DK2956477T4 (da
Inventor
Siyuan Tan
Robert T Peters
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Bioverativ Therapeutics Inc
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    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • 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

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
DK14751254.5T 2013-02-15 2014-02-14 Optimeret faktor viii-gen DK2956477T4 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361765626P 2013-02-15 2013-02-15
PCT/US2014/016441 WO2014127215A1 (en) 2013-02-15 2014-02-14 Optimized factor viii gene

Publications (2)

Publication Number Publication Date
DK2956477T3 true DK2956477T3 (da) 2021-02-08
DK2956477T4 DK2956477T4 (da) 2024-04-15

Family

ID=51354568

Family Applications (2)

Application Number Title Priority Date Filing Date
DK14751254.5T DK2956477T4 (da) 2013-02-15 2014-02-14 Optimeret faktor viii-gen
DK20204866.6T DK3889173T5 (da) 2013-02-15 2014-02-14 Optimeret faktor viii-gen

Family Applications After (1)

Application Number Title Priority Date Filing Date
DK20204866.6T DK3889173T5 (da) 2013-02-15 2014-02-14 Optimeret faktor viii-gen

Country Status (13)

Country Link
US (3) US10370431B2 (da)
EP (3) EP4223772A3 (da)
DK (2) DK2956477T4 (da)
ES (2) ES2848703T5 (da)
FI (2) FI3889173T3 (da)
HR (2) HRP20210185T4 (da)
HU (2) HUE053136T2 (da)
LT (2) LT2956477T (da)
PL (2) PL3889173T3 (da)
PT (2) PT2956477T (da)
RS (2) RS61387B2 (da)
SI (2) SI2956477T2 (da)
WO (1) WO2014127215A1 (da)

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US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
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EP3411478B1 (en) 2016-02-01 2022-06-08 Bioverativ Therapeutics Inc. Optimized factor viii genes
CN110603046B (zh) 2016-03-20 2022-10-18 宾夕法尼亚州立大学托管会 用于提高高等植物叶绿体中转基因表达的密码子优化和核糖体图谱分析
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AU2019306194A1 (en) 2018-07-16 2021-02-04 Baxalta GmbH Gene therapy of hemophilia A using viral vectors encoding recombinant FVIII variants with increased expression
CA3108799A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
TWI851647B (zh) 2019-01-16 2024-08-11 日商武田藥品工業股份有限公司 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體
SG11202109850SA (en) 2019-03-13 2021-10-28 Generation Bio Co Non-viral dna vectors and uses thereof for expressing fviii therapeutics
CN110295150A (zh) * 2019-07-17 2019-10-01 天津润德生物科技有限公司 一类腺依赖病毒病毒重组质粒、重组病毒及构建方法
AU2020342349A1 (en) * 2019-09-02 2022-03-03 Biotest Ag Factor VIII protein with increased half-life
JP7803014B2 (ja) 2019-09-30 2026-01-21 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 レンチウイルスベクター製剤
US20230023826A1 (en) 2019-12-12 2023-01-26 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
IL299333A (en) 2020-06-24 2023-02-01 Bioverativ Therapeutics Inc Methods for the purification of viral vectors
EP4355768A1 (en) 2021-06-14 2024-04-24 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
CN117836319A (zh) * 2021-08-23 2024-04-05 比奥维拉迪维治疗股份有限公司 优化的因子viii基因
JP2025504404A (ja) 2022-01-14 2025-02-12 アンジャリウム バイオサイエンシズ エージー Ｖｉｉｉ因子をコードするｄｎａ分子の組成物、それを作製する方法、及びその使用方法
US20250276093A1 (en) * 2022-04-20 2025-09-04 AskBio Inc. Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii
TW202517669A (zh) 2023-07-05 2025-05-01 日商武田藥品工業股份有限公司 用於Ａ型血友病之基因療法的編碼表現增加之重組ＦＶｌｌｌ變異體的病毒載體
WO2025219525A1 (en) 2024-04-19 2025-10-23 Fondazione Telethon Combinations, inhibitors, viral vectors, methods, and uses for in vivo gene therapy and gene editing

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