RS63548B1 - Optimizovani geni faktora viii - Google Patents

Optimizovani geni faktora viii

Info

Publication number
RS63548B1
RS63548B1 RS20220841A RSP20220841A RS63548B1 RS 63548 B1 RS63548 B1 RS 63548B1 RS 20220841 A RS20220841 A RS 20220841A RS P20220841 A RSP20220841 A RS P20220841A RS 63548 B1 RS63548 B1 RS 63548B1
Authority
RS
Serbia
Prior art keywords
fviii
seq
nucleic acid
sequence
polypeptide
Prior art date
Application number
RS20220841A
Other languages
English (en)
Serbian (sr)
Inventor
Siyuan Tan
Tongyao Liu
Original Assignee
Bioverativ Therapeutics 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 Bioverativ Therapeutics Inc filed Critical Bioverativ Therapeutics Inc
Publication of RS63548B1 publication Critical patent/RS63548B1/sr

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • 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
    • C07K14/745Blood coagulation or fibrinolysis factors
    • C07K14/755Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • A61K48/0008Medicinal 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
    • A61K48/0016Medicinal 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
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/30Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/31Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16041Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
RS20220841A 2016-02-01 2017-01-31 Optimizovani geni faktora viii RS63548B1 (sr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662289696P 2016-02-01 2016-02-01
US201662409739P 2016-10-18 2016-10-18
EP17705236.2A EP3411478B1 (en) 2016-02-01 2017-01-31 Optimized factor viii genes
PCT/US2017/015879 WO2017136358A1 (en) 2016-02-01 2017-01-31 Optimized factor viii genes

Publications (1)

Publication Number Publication Date
RS63548B1 true RS63548B1 (sr) 2022-09-30

Family

ID=58044178

Family Applications (1)

Application Number Title Priority Date Filing Date
RS20220841A RS63548B1 (sr) 2016-02-01 2017-01-31 Optimizovani geni faktora viii

Country Status (24)

Country Link
US (2) US11753461B2 (cg-RX-API-DMAC7.html)
EP (2) EP3411478B1 (cg-RX-API-DMAC7.html)
JP (3) JP7217630B2 (cg-RX-API-DMAC7.html)
KR (1) KR20180118659A (cg-RX-API-DMAC7.html)
CN (2) CN116218863A (cg-RX-API-DMAC7.html)
AU (2) AU2017214378B2 (cg-RX-API-DMAC7.html)
CA (1) CA3012695A1 (cg-RX-API-DMAC7.html)
CL (1) CL2018002054A1 (cg-RX-API-DMAC7.html)
CO (1) CO2018009120A2 (cg-RX-API-DMAC7.html)
DK (1) DK3411478T3 (cg-RX-API-DMAC7.html)
ES (1) ES2926585T3 (cg-RX-API-DMAC7.html)
HR (1) HRP20221089T1 (cg-RX-API-DMAC7.html)
HU (1) HUE059941T2 (cg-RX-API-DMAC7.html)
IL (2) IL320404A (cg-RX-API-DMAC7.html)
LT (1) LT3411478T (cg-RX-API-DMAC7.html)
MX (2) MX2018009375A (cg-RX-API-DMAC7.html)
NZ (1) NZ745831A (cg-RX-API-DMAC7.html)
PH (1) PH12018501628B1 (cg-RX-API-DMAC7.html)
PL (1) PL3411478T3 (cg-RX-API-DMAC7.html)
PT (1) PT3411478T (cg-RX-API-DMAC7.html)
RS (1) RS63548B1 (cg-RX-API-DMAC7.html)
SG (2) SG10201913278PA (cg-RX-API-DMAC7.html)
SI (1) SI3411478T1 (cg-RX-API-DMAC7.html)
WO (1) WO2017136358A1 (cg-RX-API-DMAC7.html)

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PL2956477T5 (pl) 2013-02-15 2024-05-27 Bioverativ Therapeutics Inc. Zoptymalizowany gen czynnika viii
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
RS63548B1 (sr) 2016-02-01 2022-09-30 Bioverativ Therapeutics Inc Optimizovani geni faktora viii
KR20250040746A (ko) * 2017-08-09 2025-03-24 바이오버라티브 테라퓨틱스 인크. 핵산 분자 및 이의 용도
CN111918674A (zh) * 2018-02-01 2020-11-10 比奥维拉迪维治疗股份有限公司 表达因子viii的慢病毒载体的用途
KR102245539B1 (ko) 2018-02-12 2021-04-29 주식회사 지앤피바이오사이언스 코어-쉘 구조의 마이크로 입자를 유효성분으로 포함하는 성장인자 유전자 발현 증가용 조성물
US12162994B2 (en) 2018-03-02 2024-12-10 Sigilon Therapeutics, Inc. Biocompatible hydrogel capsules and process for preparing same
CN112203646A (zh) 2018-04-04 2021-01-08 西吉隆医疗股份有限公司 可植入颗粒和相关方法
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
SG11202105880TA (en) * 2018-12-06 2021-07-29 Bioverativ Therapeutics Inc Use of lentiviral vectors expressing factor ix
US10654911B1 (en) * 2019-04-02 2020-05-19 Beijing Neoletix Biological Technology Co., Ltd. Vector co-expressing truncated von Willebrand factor and factor VIII
KR20220009389A (ko) 2019-04-17 2022-01-24 코디악 바이오사이언시즈, 인크. 엑소좀 및 aav 의 조성물
US12403164B2 (en) * 2019-09-30 2025-09-02 Bioverativ Therapeutics Inc. Lentiviral vector formulations
CN111647625A (zh) * 2019-12-25 2020-09-11 深圳三智医学科技有限公司 一种提高人凝血因子ix表达水平的方法
CN111808863B (zh) * 2020-06-23 2021-05-28 康霖生物科技(杭州)有限公司 一种密码子优化的凝血因子viii基因及其构建体
BR112022026127A2 (pt) 2020-06-24 2023-01-17 Bioverativ Therapeutics Inc Métodos para remoção de fator viii livre de preparações de vetores lentivirais modificados para expressar referida proteína
TW202323274A (zh) * 2021-08-23 2023-06-16 美商百歐維拉提夫治療公司 優化因子viii基因
JP2024534123A (ja) 2021-08-23 2024-09-18 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 バキュロウイルス発現システム
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
AU2022355088A1 (en) * 2021-09-30 2024-05-16 Bioverativ Therapeutics Inc. Nucleic acids encoding factor viii polypeptides with reduced immunogenicity
EP4511055A2 (en) * 2022-04-20 2025-02-26 AskBio Inc. Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii
WO2023211315A1 (en) * 2022-04-28 2023-11-02 Joint Stock Company "Biocad" Isolated nucleic acid that encodes fusion protein based on fviii-bdd and on heterologous signal peptide
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