GB2526339A - Lentiviral vectors - Google Patents

Lentiviral vectors Download PDF

Info

Publication number
GB2526339A
GB2526339A GB1409089.8A GB201409089A GB2526339A GB 2526339 A GB2526339 A GB 2526339A GB 201409089 A GB201409089 A GB 201409089A GB 2526339 A GB2526339 A GB 2526339A
Authority
GB
United Kingdom
Prior art keywords
vector
promoter
lentiviral vector
transgene
siv
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.)
Withdrawn
Application number
GB1409089.8A
Other languages
English (en)
Other versions
GB201409089D0 (en
Inventor
Eric Walter Frederick Wolfgang Alton
Uta Griesenbach
Kamila Malgorzata Pytel
Michael Christian Paul-Smith
Ian Andrew Pringle
Stephen Clarles Hyde
Deborah Rebecca Gill
Lee Adrian Davies
Alan Christopher Boyd
Gerard Mclachlan
Makoto Inoue
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Oxford
University of Edinburgh
Imperial College London
Ip2ipo Innovations Ltd
Dnavec Corp
Original Assignee
University of Oxford
University of Edinburgh
Imperial Innovations Ltd
Imperial College London
Dnavec Corp
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 University of Oxford, University of Edinburgh, Imperial Innovations Ltd, Imperial College London, Dnavec Corp filed Critical University of Oxford
Priority to GB1409089.8A priority Critical patent/GB2526339A/en
Publication of GB201409089D0 publication Critical patent/GB201409089D0/en
Priority to HUE15720762A priority patent/HUE051269T2/hu
Priority to EA201692361A priority patent/EA038966B1/ru
Priority to HRP20200930TT priority patent/HRP20200930T1/hr
Priority to JP2016567082A priority patent/JP6637444B2/ja
Priority to PT157207622T priority patent/PT3145949T/pt
Priority to RS20200724A priority patent/RS60586B1/sr
Priority to AU2015263150A priority patent/AU2015263150B2/en
Priority to EP15720762.2A priority patent/EP3145949B1/en
Priority to MEP-2020-114A priority patent/ME03789B/me
Priority to PCT/GB2015/051201 priority patent/WO2015177501A1/en
Priority to DK15720762.2T priority patent/DK3145949T3/da
Priority to SM20200360T priority patent/SMT202000360T1/it
Priority to LTEP15720762.2T priority patent/LT3145949T/lt
Priority to SI201531275T priority patent/SI3145949T1/sl
Priority to PL15720762T priority patent/PL3145949T3/pl
Priority to UAA201612986A priority patent/UA122127C2/uk
Priority to US15/312,026 priority patent/US10704061B2/en
Priority to CA2947125A priority patent/CA2947125C/en
Priority to ES15720762T priority patent/ES2805045T3/es
Publication of GB2526339A publication Critical patent/GB2526339A/en
Priority to CY20201100617T priority patent/CY1123413T1/el
Withdrawn legal-status Critical Current

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory 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
    • 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
    • C07K14/46—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
    • C07K14/4712—Cystic fibrosis
    • 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
    • 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
    • C12N7/02—Recovery or purification
    • 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
    • C12N7/04—Inactivation or attenuation; Producing viral sub-units
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15045—Special targeting system for 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15051—Methods of production or purification of viral material
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a 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
    • C12N2760/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
    • C12N2760/00011—Details
    • C12N2760/18011—Paramyxoviridae
    • C12N2760/18811—Sendai virus
    • C12N2760/18841—Use of virus, viral particle or viral elements as a vector
    • C12N2760/18845—Special targeting system for 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6072—Vectors comprising as targeting moiety peptide derived from defined protein from viruses negative strand RNA viruses
    • 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
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hematology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pulmonology (AREA)
  • Toxicology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
GB1409089.8A 2014-05-21 2014-05-21 Lentiviral vectors Withdrawn GB2526339A (en)

Priority Applications (21)

Application Number Priority Date Filing Date Title
GB1409089.8A GB2526339A (en) 2014-05-21 2014-05-21 Lentiviral vectors
ES15720762T ES2805045T3 (es) 2014-05-21 2015-04-23 Vectores lentivirales
PCT/GB2015/051201 WO2015177501A1 (en) 2014-05-21 2015-04-23 Lentiviral vectors
SM20200360T SMT202000360T1 (it) 2014-05-21 2015-04-23 Vettori lentivirali
HRP20200930TT HRP20200930T1 (hr) 2014-05-21 2015-04-23 Lentivirusni vektori
JP2016567082A JP6637444B2 (ja) 2014-05-21 2015-04-23 レンチウイルスベクター
PT157207622T PT3145949T (pt) 2014-05-21 2015-04-23 Vetores lentivirais
RS20200724A RS60586B1 (sr) 2014-05-21 2015-04-23 Lentivirusni vektori
AU2015263150A AU2015263150B2 (en) 2014-05-21 2015-04-23 Lentiviral vectors
EP15720762.2A EP3145949B1 (en) 2014-05-21 2015-04-23 Lentiviral vectors
MEP-2020-114A ME03789B (me) 2014-05-21 2015-04-23 Lentivirusni vektori
HUE15720762A HUE051269T2 (hu) 2014-05-21 2015-04-23 Lentivírus vektorok
DK15720762.2T DK3145949T3 (da) 2014-05-21 2015-04-23 Lentivirale vektorer
EA201692361A EA038966B1 (ru) 2014-05-21 2015-04-23 Лентивирусный вектор для генной терапии, способ его получения и его применение
LTEP15720762.2T LT3145949T (lt) 2014-05-21 2015-04-23 Lentivirusiniai vektoriai
SI201531275T SI3145949T1 (sl) 2014-05-21 2015-04-23 Lentiviralni vektorji
PL15720762T PL3145949T3 (pl) 2014-05-21 2015-04-23 Wektory lentiwirusowe
UAA201612986A UA122127C2 (uk) 2014-05-21 2015-04-23 Лентивірусний вектор
US15/312,026 US10704061B2 (en) 2014-05-21 2015-04-23 Lentiviral vectors
CA2947125A CA2947125C (en) 2014-05-21 2015-04-23 Lentiviral vectors
CY20201100617T CY1123413T1 (el) 2014-05-21 2020-07-03 Φακοϊικοι φορεις

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
GB1409089.8A GB2526339A (en) 2014-05-21 2014-05-21 Lentiviral vectors

Publications (2)

Publication Number Publication Date
GB201409089D0 GB201409089D0 (en) 2014-07-02
GB2526339A true GB2526339A (en) 2015-11-25

Family

ID=51135270

Family Applications (1)

Application Number Title Priority Date Filing Date
GB1409089.8A Withdrawn GB2526339A (en) 2014-05-21 2014-05-21 Lentiviral vectors

Country Status (21)

Country Link
US (1) US10704061B2 (cg-RX-API-DMAC7.html)
EP (1) EP3145949B1 (cg-RX-API-DMAC7.html)
JP (1) JP6637444B2 (cg-RX-API-DMAC7.html)
AU (1) AU2015263150B2 (cg-RX-API-DMAC7.html)
CA (1) CA2947125C (cg-RX-API-DMAC7.html)
CY (1) CY1123413T1 (cg-RX-API-DMAC7.html)
DK (1) DK3145949T3 (cg-RX-API-DMAC7.html)
EA (1) EA038966B1 (cg-RX-API-DMAC7.html)
ES (1) ES2805045T3 (cg-RX-API-DMAC7.html)
GB (1) GB2526339A (cg-RX-API-DMAC7.html)
HR (1) HRP20200930T1 (cg-RX-API-DMAC7.html)
HU (1) HUE051269T2 (cg-RX-API-DMAC7.html)
LT (1) LT3145949T (cg-RX-API-DMAC7.html)
ME (1) ME03789B (cg-RX-API-DMAC7.html)
PL (1) PL3145949T3 (cg-RX-API-DMAC7.html)
PT (1) PT3145949T (cg-RX-API-DMAC7.html)
RS (1) RS60586B1 (cg-RX-API-DMAC7.html)
SI (1) SI3145949T1 (cg-RX-API-DMAC7.html)
SM (1) SMT202000360T1 (cg-RX-API-DMAC7.html)
UA (1) UA122127C2 (cg-RX-API-DMAC7.html)
WO (1) WO2015177501A1 (cg-RX-API-DMAC7.html)

Families Citing this family (41)

* 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
BR102015012334A2 (pt) 2015-05-27 2016-11-29 Fundação Hemoct De Ribeirão Preto Fundherp processo de produção do fator vii de coagulação sanguínea e fator vii de coagulação sanguínea
ES2796488T3 (es) 2016-04-08 2020-11-27 Krystal Biotech Inc Composiciones para el uso en métodos para el tratamiento de heridas, trastornos y enfermedades de la piel
JP2018174874A (ja) * 2017-04-19 2018-11-15 国立大学法人 東京医科歯科大学 上皮細胞の水分泌機能測定方法
US12134790B2 (en) 2017-09-27 2024-11-05 Inozyme Pharma, Inc. Methods of improving cardiovascular function and treating cardiovascular disease using a recombinant ectonucleotide pyrophosphatase phosphodiesterase (NPP1)
GB201814590D0 (en) * 2018-09-07 2018-10-24 Oxford Biomedica Ltd Viral vector production system
EP3775236A1 (en) 2018-04-12 2021-02-17 Krystal Biotech, LLC Compositions and methods for the treatment of autosomal recessive congenital ichthyosis
MX2020011257A (es) 2018-04-27 2021-01-15 Krystal Biotech Inc Ácidos nucleicos recombinantes que codifican una o más proteínas cosméticas para usos estéticos.
WO2020009444A1 (ko) * 2018-07-02 2020-01-09 서울대학교산학협력단 세포융합 기술을 이용한 유전자 및 세포 치료제 및 이의 용도
CN112384247B (zh) * 2018-07-02 2024-09-27 库拉米斯公司 利用细胞融合技术的基因及细胞治疗剂以及其的用途
AU2019346549B2 (en) 2018-09-24 2024-09-12 Krystal Biotech, Inc. Compositions and methods for the treatment of Netherton Syndrome
EP3856914A1 (en) 2018-09-26 2021-08-04 Krystal Biotech, Inc. Compositions and methods for the treatment of skin diseases
CN113454105A (zh) 2019-02-08 2021-09-28 克里斯托生物技术股份有限公司 用于递送cftr多肽的组合物和方法
KR20210151785A (ko) * 2019-03-13 2021-12-14 제너레이션 바이오 컴퍼니 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
CN112575034B (zh) * 2019-09-29 2023-04-25 济南赛尔生物科技股份有限公司 一种治疗a型血友病的产品及应用
EP4077688A1 (en) * 2019-12-20 2022-10-26 Krystal Biotech, Inc. Compositions and methods for gene delivery to the airways and/or lungs
EP4143326A1 (en) * 2020-04-27 2023-03-08 University of Iowa Research Foundation Compositions and methods for the treatment of cystic fibrosis
US20230190871A1 (en) 2020-05-20 2023-06-22 Sana Biotechnology, Inc. Methods and compositions for treatment of viral infections
EP4162036A1 (en) 2020-06-09 2023-04-12 Inozyme Pharma, Inc. Soluble enpp1 or enpp3 proteins and uses thereof
JP2023537565A (ja) * 2020-06-24 2023-09-04 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 タンパク質を発現するように改変されたレンチウイルスベクターの調製物から遊離第ｖｉｉｉ因子を除去する方法
WO2022047316A1 (en) 2020-08-28 2022-03-03 Sana Biotechnology, Inc. Modified anti-viral binding agents
CA3191743A1 (en) * 2020-09-16 2022-03-24 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing fviii therapeutics
GB202100688D0 (en) * 2021-01-19 2021-03-03 Autolus Ltd Process
GB202102832D0 (en) * 2021-02-26 2021-04-14 Ip2Ipo Innovations Ltd Retroviral vectors
IL307350A (en) 2021-04-02 2023-11-01 Krystal Biotech Inc Viral vectors for cancer treatment
GB202105278D0 (en) 2021-04-13 2021-05-26 Imperial College Innovations Ltd Cell therapy
GB202105276D0 (en) * 2021-04-13 2021-05-26 Imperial College Innovations Ltd Delivery of gene therapy vectors
GB202105277D0 (en) * 2021-04-13 2021-05-26 Imperial College Innovations Ltd Signal peptides
EP4373951A1 (en) * 2021-07-19 2024-05-29 2seventy bio, Inc. Vector manufacturing processes
WO2023114949A1 (en) 2021-12-16 2023-06-22 Sana Biotechnology, Inc. Methods and systems of particle production
US20250082777A1 (en) 2022-01-10 2025-03-13 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023150647A1 (en) 2022-02-02 2023-08-10 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
AU2023253826A1 (en) 2022-04-11 2024-11-21 Boehringer Ingelheim International Gmbh Combination treatment
US20260027145A1 (en) 2022-07-27 2026-01-29 Sana Biotechnology, Inc. Methods of transduction using a viral vector and inhibitors of antiviral restriction factors
WO2024044655A1 (en) 2022-08-24 2024-02-29 Sana Biotechnology, Inc. Delivery of heterologous proteins
GB202212472D0 (en) * 2022-08-26 2022-10-12 Ip2Ipo Innovations Ltd Retroviral vectors
WO2024057315A1 (en) 2022-09-15 2024-03-21 Yeda Research And Development Co. Ltd. Compositions and methods for virotherapy
WO2024119157A1 (en) 2022-12-02 2024-06-06 Sana Biotechnology, Inc. Lipid particles with cofusogens and methods of producing and using the same
WO2024220597A2 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Digital droplet based assay for detecting replication competent lentiviral vector
AU2024274792A1 (en) 2023-05-23 2025-10-23 Sana Biotechnology, Inc. Tandem fusogens and related lipid particles
WO2025184529A1 (en) 2024-03-01 2025-09-04 Sana Biotechnology, Inc. Viral particles with fusogen display and related compositions and methods

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000039302A2 (en) * 1998-12-31 2000-07-06 Chiron Corporation Improved expression of hiv polypeptides and production of virus-like particles
EP1291419A1 (en) * 2000-06-01 2003-03-12 Dnavec Research Inc. Pseudo-type retrovirus vector containing membrane protein having hemagglutinin activity
WO2003029412A2 (en) * 2001-10-02 2003-04-10 Institut Clayton De La Recherche Restricted expression lentivial vectors
WO2008124724A1 (en) * 2007-04-09 2008-10-16 University Of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
US20090162320A1 (en) * 2005-10-28 2009-06-25 Dnavec Corporation Gene transfer into airway epithelial stem cell by using lentiviral vector pseudotyped with rna virus or dna virus spike protein

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050048030A1 (en) * 2001-09-28 2005-03-03 Raymond Pickles Paramyxoviruses as gene transfer vectors to lung cells
GB0606190D0 (en) * 2006-03-28 2006-05-10 Isis Innovation Construct
EP1950303A1 (de) 2007-01-26 2008-07-30 Bayer CropScience AG Genetisch modifizierte Pflanzen, die eine Stärke mit geringem Amylosegehalt und erhöhtem Quellvermögen synthetisieren

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000039302A2 (en) * 1998-12-31 2000-07-06 Chiron Corporation Improved expression of hiv polypeptides and production of virus-like particles
EP1291419A1 (en) * 2000-06-01 2003-03-12 Dnavec Research Inc. Pseudo-type retrovirus vector containing membrane protein having hemagglutinin activity
WO2003029412A2 (en) * 2001-10-02 2003-04-10 Institut Clayton De La Recherche Restricted expression lentivial vectors
US20090162320A1 (en) * 2005-10-28 2009-06-25 Dnavec Corporation Gene transfer into airway epithelial stem cell by using lentiviral vector pseudotyped with rna virus or dna virus spike protein
WO2008124724A1 (en) * 2007-04-09 2008-10-16 University Of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Molecular Therapy, Vol 16, 2008, D Zychlinski et al, "Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors" 718-725 *

Also Published As

Publication number Publication date
LT3145949T (lt) 2020-08-25
JP2017521049A (ja) 2017-08-03
CA2947125C (en) 2023-02-28
GB201409089D0 (en) 2014-07-02
US20170096684A1 (en) 2017-04-06
SMT202000360T1 (it) 2020-09-10
EP3145949A1 (en) 2017-03-29
PT3145949T (pt) 2020-07-13
CA2947125A1 (en) 2015-11-26
DK3145949T3 (da) 2020-06-29
EP3145949B1 (en) 2020-04-08
JP6637444B2 (ja) 2020-01-29
CY1123413T1 (el) 2021-12-31
ES2805045T3 (es) 2021-02-10
PL3145949T3 (pl) 2021-01-25
AU2015263150A1 (en) 2016-11-10
HUE051269T2 (hu) 2021-03-01
AU2015263150B2 (en) 2020-04-16
SI3145949T1 (sl) 2020-08-31
WO2015177501A1 (en) 2015-11-26
EA201692361A1 (ru) 2017-05-31
HRP20200930T1 (hr) 2020-09-18
EA038966B1 (ru) 2021-11-16
US10704061B2 (en) 2020-07-07
ME03789B (me) 2021-04-20
RS60586B1 (sr) 2020-08-31
UA122127C2 (uk) 2020-09-25

Similar Documents

Publication Publication Date Title
CA2947125C (en) Lentiviral vectors
CN110944674A (zh) 高活性调控元件
CN114555809A (zh) 组合癌症免疫疗法
CA3249548A1 (en) NEW VECTOR COMPOSITIONS OF THE ANELLOVIRIDAE FAMILY AND PROCESSES
JP2020500562A (ja) ムコ多糖症ｉｉ型のための遺伝子治療
KR102803305B1 (ko) 트랜스-스플라이싱 아데노-연관 바이러스 벡터를 포함하는 프라임에디팅용 조성물
CN109970861B (zh) 一种靶向线粒体的nd4融合蛋白及其制备方法和应用
EP4593886A1 (en) Gene therapy
US20240082327A1 (en) Retroviral vectors
US20250177574A1 (en) Retroviral vectors
EP4590696A1 (en) Retroviral vector comprising rre inserted within an intron
CN116761812A (zh) Neurod1和dlx2载体
CN116710566A (zh) Neurod1载体

Legal Events

Date Code Title Description
WAP Application withdrawn, taken to be withdrawn or refused ** after publication under section 16(1)