SG11202107421YA - Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a - Google Patents

Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Info

Publication number
SG11202107421YA
SG11202107421YA SG11202107421YA SG11202107421YA SG11202107421YA SG 11202107421Y A SG11202107421Y A SG 11202107421YA SG 11202107421Y A SG11202107421Y A SG 11202107421YA SG 11202107421Y A SG11202107421Y A SG 11202107421YA SG 11202107421Y A SG11202107421Y A SG 11202107421YA
Authority
SG
Singapore
Prior art keywords
hemophilia
gene therapy
viral vectors
increased expression
vectors encoding
Prior art date
Application number
SG11202107421YA
Other languages
English (en)
Inventor
Hanspeter Rottensteiner
Friedrich Scheiflinger
Original Assignee
Baxalta Inc
Baxalta GmbH
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 Baxalta Inc, Baxalta GmbH filed Critical Baxalta Inc
Publication of SG11202107421YA publication Critical patent/SG11202107421YA/en

Links

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
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • 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
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (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)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
SG11202107421YA 2019-01-16 2020-01-15 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a SG11202107421YA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962793058P 2019-01-16 2019-01-16
PCT/US2020/013722 WO2020150375A1 (fr) 2019-01-16 2020-01-15 Vecteurs viraux codant pour des variants fviii de recombinaison avec une expression accrue pour la thérapie génique de l'hémophilie a

Publications (1)

Publication Number Publication Date
SG11202107421YA true SG11202107421YA (en) 2021-08-30

Family

ID=69593774

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202107421YA SG11202107421YA (en) 2019-01-16 2020-01-15 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Country Status (18)

Country Link
US (2) US11707536B2 (fr)
EP (1) EP3911672A1 (fr)
JP (1) JP2022517267A (fr)
KR (1) KR20210117291A (fr)
CN (1) CN113614104A (fr)
AR (1) AR117825A1 (fr)
AU (1) AU2020208375A1 (fr)
BR (1) BR112021013874A2 (fr)
CA (1) CA3127065A1 (fr)
CL (1) CL2021001880A1 (fr)
CO (1) CO2021009338A2 (fr)
EA (1) EA202191977A1 (fr)
EC (1) ECSP21060605A (fr)
IL (2) IL284648B1 (fr)
MX (1) MX2021008550A (fr)
PE (1) PE20220429A1 (fr)
SG (1) SG11202107421YA (fr)
WO (1) WO2020150375A1 (fr)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11795207B2 (en) 2021-03-30 2023-10-24 AAVnerGene Inc. Modified plasma clotting factor VIII and method of use thereof
EP4314041A1 (fr) * 2021-03-30 2024-02-07 Aavnergene Inc. Facteur viii de coagulation plasmatique modifié et son procédé d'utilisation
US20240254197A1 (en) * 2021-11-25 2024-08-01 Sichuan Real&Best Biotech Co., Ltd. Engineered Human FVIII with Enhanced Secretion Ability and Clotting Activity
AR128760A1 (es) * 2022-03-11 2024-06-12 Homology Medicines Inc Vectores de expresión con promotores dobles bidireccionales y sus usos
CN116715752A (zh) * 2023-06-16 2023-09-08 苏州诺洁贝生物技术有限公司 凝血因子fviii蛋白变体、表达载体和用途

Family Cites Families (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
CA1341174C (fr) 1985-04-12 2001-01-30 John J. Toole Jr. Proteines procoagulantes derivees du facteur viii: c
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5543502A (en) 1986-06-24 1996-08-06 Novo Nordisk A/S Process for producing a coagulation active complex of factor VIII fragments
US6060447A (en) 1987-05-19 2000-05-09 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
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
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
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
CA2162497A1 (fr) 1993-06-10 1994-12-22 Sheila Connelly Vecteurs adenoviraux pour le traitement de l'hemophilie
SE504074C2 (sv) 1993-07-05 1996-11-04 Pharmacia Ab Proteinberedning för subkutan, intramuskulär eller intradermal administrering
US6818439B1 (en) 1994-12-30 2004-11-16 Chiron Corporation Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders
SE9503380D0 (sv) 1995-09-29 1995-09-29 Pharmacia Ab Protein derivatives
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6458563B1 (en) 1996-06-26 2002-10-01 Emory University Modified factor VIII
US6114148C1 (en) 1996-09-20 2012-05-01 Gen Hospital Corp High level expression of proteins
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6924365B1 (en) 1998-09-29 2005-08-02 Transkaryotic Therapies, Inc. Optimized messenger RNA
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US20060099685A1 (en) 1999-04-15 2006-05-11 Yallop Christopher A Recombinant expression of factor VIII in human cells
EP1572889B1 (fr) 2001-10-05 2008-12-17 Expression Therapeutics, LLC Sequences d'acides nucleiques et d'acides amines codant des polypeptides de facteur viii a expresseur a haut rendement et leurs procedes d'utilisation
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
US7041635B2 (en) 2003-01-28 2006-05-09 In2Gen Co., Ltd. Factor VIII polypeptide
US7943374B2 (en) 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
CN104087591A (zh) 2006-06-19 2014-10-08 阿斯克肋匹奥生物制药公司 用于基因治疗的修饰的因子viii和因子ix基因和载体
CA2673459C (fr) 2006-12-22 2016-09-13 Stefan Schulte Facteurs de coagulation modifies presentant une demi-vie in vivo prolongee
KR100959454B1 (ko) 2007-12-10 2010-05-25 주식회사 동부하이텍 반도체 소자 및 그 제조 방법
DK2282764T3 (da) 2008-04-22 2019-10-14 Vib Vzw Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf
CN102482340B (zh) 2009-04-06 2015-05-13 诺沃—诺迪斯克有限公司 因子viii蛋白向血小板的靶向递送
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
NZ605400A (en) 2010-07-09 2015-05-29 Biogen Idec Hemophilia Inc Chimeric clotting factors
US20130017997A1 (en) * 2010-08-19 2013-01-17 Amunix Operating Inc. Factor VIII Compositions and Methods of Making and Using Same
US10656167B2 (en) 2011-07-25 2020-05-19 Bioverativ Therapeutics Inc. Assays to monitor bleeding disorders
SI2802668T1 (sl) 2012-01-12 2019-01-31 Bioverativ Therapeutics Inc. Zmanjšanje imunogeničnosti proti faktorju VIII pri posameznikih, ki se zdravijo zaradi faktorja VIII
WO2013106787A1 (fr) 2012-01-12 2013-07-18 Biogen Idec Ma Inc. Facteur viii chimérique et polypeptides et leurs utilisations
RS63870B1 (sr) 2012-02-15 2023-01-31 Bioverativ Therapeutics Inc Sastavi faktora viii i postupci za pravljenje i upotrebu istih
KR102057540B1 (ko) 2012-02-17 2019-12-19 더 칠드런스 호스피탈 오브 필라델피아 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법
AU2013243949A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
US8986991B2 (en) 2012-07-03 2015-03-24 Expression Therapeutics, Llc High yield suspension cell line, system and method for making same
US10398787B2 (en) 2012-10-26 2019-09-03 Vrije Universiteit Brussel Vectors for liver-directed gene therapy of hemophilia and methods and use thereof
CA2888931C (fr) 2012-10-26 2023-09-05 Vrije Universiteit Brussel Vecteur pour therapie genique ciblee sur le foie de l'hemophilie et procedes associes
ES2959747T3 (es) 2013-02-15 2024-02-28 Bioverativ Therapeutics Inc Gen del factor VIII optimizado
RU2015144076A (ru) 2013-03-15 2017-04-24 БАЙЕР ХелсКер ЛЛСи Композиции рекомбинантного фактора viii
PL3044231T3 (pl) 2013-09-12 2021-01-11 Biomarin Pharmaceutical Inc. Wektory aav zawierające gen kodujący czynnik viii
SG11201701033QA (en) 2014-08-13 2017-03-30 Philadelphia Children Hospital An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders
EP3253786A4 (fr) 2015-02-06 2018-10-17 The University of North Carolina at Chapel Hill Cassettes optimisées d'expression génétique du facteur viii de coagulation humain et leur utilisation
WO2016146757A1 (fr) 2015-03-17 2016-09-22 Vrije Universiteit Brussel Systèmes d'expression spécifiques du foie optimisés pour fviii et fix
KR20230008256A (ko) * 2015-11-13 2023-01-13 다케다 야쿠힌 고교 가부시키가이샤 A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터
JP6695426B2 (ja) * 2015-11-13 2020-05-20 バクスアルタ インコーポレイテッド 組換えｆｖｉｉｉ変異型をコードする血友病ａ遺伝子治療用高発現ウイルスベクター
JP7529286B2 (ja) * 2018-10-26 2024-08-06 フリーイェ・ユニヴェルシテイト・ブリュッセル 肝特異的核酸調節エレメント並びにその方法及び使用

Also Published As

Publication number Publication date
MX2021008550A (es) 2021-08-19
US20200289672A1 (en) 2020-09-17
IL284648A (en) 2021-08-31
CA3127065A1 (fr) 2020-07-23
CN113614104A (zh) 2021-11-05
US20230211017A1 (en) 2023-07-06
IL314658A (en) 2024-09-01
TW202039546A (zh) 2020-11-01
AR117825A1 (es) 2021-08-25
KR20210117291A (ko) 2021-09-28
BR112021013874A2 (pt) 2021-09-21
ECSP21060605A (es) 2022-01-31
AU2020208375A1 (en) 2021-07-29
CO2021009338A2 (es) 2021-07-30
PE20220429A1 (es) 2022-03-29
JP2022517267A (ja) 2022-03-07
CL2021001880A1 (es) 2022-01-14
US11707536B2 (en) 2023-07-25
WO2020150375A1 (fr) 2020-07-23
IL284648B1 (en) 2024-08-01
EP3911672A1 (fr) 2021-11-24
EA202191977A1 (ru) 2021-12-15

Similar Documents

Publication Publication Date Title
IL281038A (en) Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy in hemophilia A
IL284648A (en) Viral vectors encoding recombinant FVIII variants with enhanced expression for hemophilia A gene therapy
IL280637A (en) Viral vectors encoding recombinant FIX with increased expression for gene therapy of hemophilia B
ZA201803442B (en) Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
IL283546A (en) A recombinant adeno-associated virus vector for gene delivery
IL282216A (en) Viral administration of a cellular solution that mimics a type of medicinal polypeptides
GB202108457D0 (en) Recombinant varicella-zoster virus vaccine
IL286464A (en) A virus of vectors related to recombinant DNA
EP3731850A4 (fr) Administration par un virus oncolytique de polypeptides thérapeutiques
IL286475A (en) Methods for the production of recombinant viral vectors
IL289518A (en) Recombinant ad35 vectors and improvements in gene therapy
SG11202110909XA (en) Continuous production of recombinant proteins
EP4182467A4 (fr) Vecteurs viraux adéno-associés recombinés pour la délivrance de gènes multiples
IL288419A (en) Recombinant viral herpes vector
IL299380A (en) An improved adenovirus as a vector for gene therapy
EP3743084A4 (fr) Vaccins antiviraux recombinants
ZA202109011B (en) Stable formulations of recombinant proteins
MX2020001655A (es) Vacuna vph.
IL285144A (en) Medicinal peptides
EP4073087A4 (fr) Vecteur viral adéno-associé recombiné pour la délivrance de gènes
IL291127A (en) Amino-end extension sequence for expression of recombinant medicinal peptides
IL287846A (en) Adenovirus polypeptide 9 increases productivity and infectivity of an adenovirus vector for gene therapy
IL290520A (en) Medicinal peptides
EP4190902A4 (fr) Virus de la vaccine recombiné
IL315248A (en) A viral vector genome encoding an insulin fusion protein