CY1123760T1 - Ανασυνδυασμενοι φορεις aav που εκφραζουν οστεοπροστατευτικα γονιδια, που περιλαμβανουν has2 και λουμπρικινη, χρησιμοι στην θεραπευτικη αγωγη οστεοαρθριτιδας και συναφων παθησεων των αρθρωσεων στα θηλαστικα - Google Patents

Ανασυνδυασμενοι φορεις aav που εκφραζουν οστεοπροστατευτικα γονιδια, που περιλαμβανουν has2 και λουμπρικινη, χρησιμοι στην θεραπευτικη αγωγη οστεοαρθριτιδας και συναφων παθησεων των αρθρωσεων στα θηλαστικα

Info

Publication number
CY1123760T1
CY1123760T1 CY20201101100T CY201101100T CY1123760T1 CY 1123760 T1 CY1123760 T1 CY 1123760T1 CY 20201101100 T CY20201101100 T CY 20201101100T CY 201101100 T CY201101100 T CY 201101100T CY 1123760 T1 CY1123760 T1 CY 1123760T1
Authority
CY
Cyprus
Prior art keywords
osteoprotective
has2
osteoarthritis
lubricin
mammals
Prior art date
Application number
CY20201101100T
Other languages
English (en)
Inventor
Monica DIAS FIGUEIREDO
Sirkka RM KYOSTIO-MOORE
Patricia Berthelette
Original Assignee
Genzyme Corporation
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 Genzyme Corporation filed Critical Genzyme Corporation
Publication of CY1123760T1 publication Critical patent/CY1123760T1/el

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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • 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
    • C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1048—Glycosyltransferases (2.4)
    • C12N9/1051—Hexosyltransferases (2.4.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01212—Hyaluronan synthase (2.4.1.212)
    • 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
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)

Abstract

Η παρούσα εφεύρεση αναφέρεται σε ανασυνδυασμένους ιϊκούς φορείς, σε φαρμακευτικές συνθέσεις που αποτελούνται από τέτοιους ανασυνδυασμένους φορείς, και σε μεθόδους για την πρόληψη και θεραπευτική αγωγή οστεοαρθρίτιδας στα θηλαστικά. Ειδικότερα, αυτή η αποκάλυψη παρέχει φορείς αδενο-σχετιζόμενού ιού (AAV) ικανούς να εκφράζουν, σε έναν ξενιστή, οστεοπροστατευτικές/ χονδροπροστατευτικές βιοδραστικές πρωτεΐνες, που περιλαμβάνουν συνθάση 2 υαλουρονάνης 2 (HAS2) και λουμπρικίνη (PRG4). Μέθοδοι παραγωγής αυτών των AAV παρέχονται, καθώς είναι μέθοδοι αντιμετώπισης της οστεοαρθρίτιδας στις αρθρώσεις θηλαστικών, μέσω της μακροπρόθεσμης γονιδιακής έκφρασης των οστεοπροστατευτικών /χονδροπροστατευτικών πρωτεϊνών, που περιλαμβάνουν HAS2 και PRG4, τόσο σε αρθρικά κύτταρα όσο και σε χονδροκυτταρικά κύτταρα.
CY20201101100T 2016-01-13 2020-11-19 Ανασυνδυασμενοι φορεις aav που εκφραζουν οστεοπροστατευτικα γονιδια, που περιλαμβανουν has2 και λουμπρικινη, χρησιμοι στην θεραπευτικη αγωγη οστεοαρθριτιδας και συναφων παθησεων των αρθρωσεων στα θηλαστικα CY1123760T1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662278243P 2016-01-13 2016-01-13
PCT/US2017/013419 WO2017123934A1 (en) 2016-01-13 2017-01-13 Recombinant aav vectors expressing osteoprotective genes, including has2 and lubricin, useful in the treatment of osteoarthritis and related joint conditions in mammals

Publications (1)

Publication Number Publication Date
CY1123760T1 true CY1123760T1 (el) 2022-03-24

Family

ID=58054500

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20201101100T CY1123760T1 (el) 2016-01-13 2020-11-19 Ανασυνδυασμενοι φορεις aav που εκφραζουν οστεοπροστατευτικα γονιδια, που περιλαμβανουν has2 και λουμπρικινη, χρησιμοι στην θεραπευτικη αγωγη οστεοαρθριτιδας και συναφων παθησεων των αρθρωσεων στα θηλαστικα

Country Status (16)

Country Link
US (3) US20170198263A1 (el)
EP (2) EP3769788A1 (el)
JP (2) JP7058603B2 (el)
KR (1) KR20180095720A (el)
CN (2) CN118048321A (el)
AR (1) AR108683A1 (el)
AU (2) AU2017207917B2 (el)
CA (1) CA3011459A1 (el)
CY (1) CY1123760T1 (el)
DK (1) DK3402530T3 (el)
ES (1) ES2835031T3 (el)
HU (1) HUE051774T2 (el)
MX (2) MX2018008651A (el)
RU (1) RU2771490C2 (el)
WO (1) WO2017123934A1 (el)
ZA (1) ZA201804731B (el)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN109055426B (zh) * 2018-08-06 2021-06-25 智享生物(苏州)有限公司 一种在中华仓鼠卵巢细胞中表达生产类人润滑素的方法
WO2020150390A1 (en) * 2019-01-15 2020-07-23 Cornell University Recombinant mucins, and compositions and methods for using the same
WO2020150633A1 (en) * 2019-01-18 2020-07-23 Orthobio Therapeutics, Inc. Gene editing to improve joint function
IL293141A (en) * 2019-11-21 2022-07-01 Remedium Bio Inc growth factor recovery
KR102584136B1 (ko) * 2020-01-17 2023-10-04 주식회사 아바테라퓨틱스 조직 재생용 조성물
US20240207441A1 (en) * 2021-04-26 2024-06-27 University Of Massachusetts Capsid variants and uses thereof
CN114457086B (zh) * 2022-03-02 2022-11-15 上海勉亦生物科技有限公司 白介素1受体拮抗蛋白的表达盒以及基于aav的基因递送系统
KR20230152503A (ko) * 2022-04-27 2023-11-03 주식회사 헬릭스미스 척수강 내 투여에 최적화 된 간세포 성장인자 유전자가 도입된 aav 벡터
CN117721152B (zh) * 2023-12-29 2024-08-30 广州市第一人民医院（广州消化疾病中心、广州医科大学附属市一人民医院、华南理工大学附属第二医院） Ttp在制备骨关节炎药物中的应用

Family Cites Families (36)

* 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
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
US5693531A (en) 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6423514B1 (en) 1996-04-22 2002-07-23 Millennium Pharmaceuticals, Inc. Mammalian hyaluronan synthases, nucleic acids and uses thereof
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6743774B1 (en) 1999-04-23 2004-06-01 Rhode Island Hospital Tribonectins
US6315992B1 (en) * 1999-06-30 2001-11-13 Tissuegene Co. Generating cartilage in a mammal using fibroblasts transfected with a vector encoding TGF-β-1
US7338655B1 (en) * 1999-06-30 2008-03-04 Tissuegene, Inc. Gene therapy using TGF-β
ES2308989T3 (es) * 1999-08-09 2008-12-16 Targeted Genetics Corporation Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios.
JP2003164289A (ja) * 2001-11-30 2003-06-10 Seikagaku Kogyo Co Ltd 骨組織導入用遺伝子
EP1593668A4 (en) 2003-02-14 2007-07-11 Wako Pure Chem Ind Ltd METHOD FOR PRODUCING A HYDROXYLAMINE COMPOUND USING A PLATINATE CATALYST IMMOBILIZED ON AN ION EXCHANGE RESIN
WO2005000331A2 (en) 2003-06-04 2005-01-06 Mucosal Therapeutics, Inc. Compositions for the treatment and prevention of degenerative joint disorders
CN102924584A (zh) 2003-08-14 2013-02-13 惠氏公司 重组润滑素分子及其用途
JP4863874B2 (ja) 2003-09-01 2012-01-25 アカデミッシュ メディッシュ セントラム 関節リウマチのインビボ遺伝子治療のためのａａｖベクター
US8529885B2 (en) * 2003-09-01 2013-09-10 Academisch Medisch Centrum AAV vectors for in vivo gene therapy of rheumatoid arthritis
US20090155200A1 (en) 2004-04-20 2009-06-18 Jay Gregory D Methods of promoting cartilage healing or cartilage integration
EP1827478A4 (en) 2004-12-03 2009-08-05 Mucosal Therapeutics Llc METHODS OF TREATING JOINED OR SICK JOINTS
US7771995B2 (en) 2005-11-14 2010-08-10 Merial Limited Plasmid encoding human BMP-7
US8298818B2 (en) * 2006-04-28 2012-10-30 University Of Florida Research Foundation, Inc. Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter
US20080187576A1 (en) * 2006-06-16 2008-08-07 University Of Florida Research Foundation, Inc. Methods for treating articular disease or dysfunction using self-complimentary adeno-associated viral vectors
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
US7846428B2 (en) 2007-10-05 2010-12-07 Merial Limited Articular cartilage gene therapy with recombinant vector encoding BMP-7
KR20100097726A (ko) * 2007-12-04 2010-09-03 프로테오바이오엑티브스 피티와이 엘티디 전구 세포의 보호 및 그의 분화 조절
WO2010115841A1 (en) 2009-04-01 2010-10-14 Galapagos Nv Methods and means for treatment of osteoarthritis
US20120114755A1 (en) 2009-06-22 2012-05-10 Mayo Foundation For Medical Education And Research Methods and materials for tissue repair
EP2531604B1 (en) * 2010-02-05 2017-04-05 The University of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
MX341578B (es) * 2011-02-08 2016-08-25 Abbvie Inc Tratamiento de la osteoartritis y del dolor.
US20130116186A1 (en) 2011-10-04 2013-05-09 Rhode Island Hospital, A Lifespan Partner Lubricin injections to maintain cartilage health
EP2623604B8 (en) * 2012-02-02 2015-04-22 Baylor College of Medicine Adenoviral-based biological delivery and expression system for use in the treatment of osteoarthritis
WO2013151664A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
EP2948553B1 (en) 2013-01-25 2020-04-01 Baylor College Of Medicine A helper-dependent adenoviral gene therapy delivery and expression system
WO2014205237A2 (en) * 2013-06-19 2014-12-24 University Of Rochester Compositions and methods for use of high molecular weight hyaluronic acid for cancer therapy
AU2014317861B2 (en) * 2013-09-09 2019-11-28 Figene, Llc Gene therapy for the regeneration of chondrocytes or cartilage type cells
JP2017531652A (ja) * 2014-10-06 2017-10-26 アルスロジェン ビー．ブイ．Ａｒｔｈｒｏｇｅｎ Ｂ．Ｖ． Ａａｖに基づく遺伝子治療

Also Published As

Publication number Publication date
WO2017123934A1 (en) 2017-07-20
RU2018129159A3 (el) 2020-03-27
AU2017207917A1 (en) 2018-08-16
EP3769788A1 (en) 2021-01-27
HUE051774T2 (hu) 2021-03-29
ZA201804731B (en) 2019-05-29
MX2021004397A (es) 2021-06-08
ES2835031T3 (es) 2021-06-21
AR108683A1 (es) 2018-09-19
CN108697813A (zh) 2018-10-23
CN108697813B (zh) 2024-01-16
AU2017207917B2 (en) 2024-03-07
US20170198263A1 (en) 2017-07-13
RU2771490C2 (ru) 2022-05-05
JP2022058602A (ja) 2022-04-12
RU2018129159A (ru) 2020-02-13
EP3402530A1 (en) 2018-11-21
JP7058603B2 (ja) 2022-04-22
KR20180095720A (ko) 2018-08-27
US20240254454A1 (en) 2024-08-01
CA3011459A1 (en) 2017-07-20
JP2019506859A (ja) 2019-03-14
DK3402530T3 (da) 2020-11-23
CN118048321A (zh) 2024-05-17
US20200231940A1 (en) 2020-07-23
MX2018008651A (es) 2019-03-14
AU2024203920A1 (en) 2024-07-11
EP3402530B1 (en) 2020-08-19
US11905531B2 (en) 2024-02-20

Similar Documents

Publication Publication Date Title
CY1123760T1 (el) Ανασυνδυασμενοι φορεις aav που εκφραζουν οστεοπροστατευτικα γονιδια, που περιλαμβανουν has2 και λουμπρικινη, χρησιμοι στην θεραπευτικη αγωγη οστεοαρθριτιδας και συναφων παθησεων των αρθρωσεων στα θηλαστικα
BR112021005769A2 (pt) proteínas de ligação a dll3 e métodos de uso
EA202090817A1 (ru) Триспецифические белки и способы их применения
BR112018068189A2 (pt) proteínas de ligação induzíveis e métodos de uso
MX2020009514A (es) Anticuerpos anti-claudina 18.2 (cldn18.2).
CY1121132T1 (el) ΠΛΗΡΩΣ-ΑΝΘΡΩΠΙΝΑ ΜΟΝΟΚΛΩΝΙΚΑ ΑΝΤΙΣΩΜΑΤΑ ANTI-vap-1
BR112021022874A2 (pt) Proteínas de ligação epcam e métodos de uso
MX2021006615A (es) Proteinas de union triespecificas y metodos de uso.
BR112019000015A2 (pt) distribuição por vírus oncolítico pseudotipado de polipeptídeos terapêuticos
CL2018001522A1 (es) Focalización de péptidos para dirigir virus adenoasociados (aavs)
CY1124055T1 (el) Ετεροκυκλικες ενωσεις ως ανοσοδιαμορφωτες
BR112019023856A2 (pt) proteínas triespecíficas que visam msln e métodos de utilização
BR112018011975A2 (pt) composições úteis no tratamento de atrofia muscular espinhal
BR112018006074A2 (pt) vetores de fator viii de vírus adeno-associado, partículas virais associadas e formulações terapêuticas compreendendo as mesmas
BR112017007765A2 (pt) composições de adenosina deaminase-2 (ada2), variantes do mesmo e métodos de usar o mesmo
PH12020551096A1 (en) A modified raav capsid protein for gene therapy
EA201892561A1 (ru) Гибридные белки gdf15 и их применение
EA201992882A1 (ru) САМОРЕГУЛИРУЕМЫЕ ВЕКТОРЫ НА ОСНОВЕ AAV ДЛЯ БЕЗОПАСНОЙ ЭКСПРЕССИИ MeCP2 ПРИ СИНДРОМЕ PETTA
CY1123485T1 (el) Συνθεσεις και μεθοδοι για τη θεραπεια νοσων
BR112018074978A2 (pt) anticorpos para alfa-sinucleína e usos dos mesmos
BR112016016658A2 (pt) proteína de fusão de um anticorpo, polinucleotídeo, vetor, célula hospedeira, composição farmacêutica, métodos para produzir uma proteína de fusão e de tratamento de uma doença em um indivíduo e uso da proteína de fusão
BR112016018288A2 (pt) proteínas de fusão de interleucina 2 e uso das mesmas
BR112018071200A2 (pt) terapia genética para o tratamento da hemofilia a
BR112022016550A2 (pt) Proteínas de ligação a flt3 e métodos de uso
PH12019500571A1 (en) Anti-pd-1 antibodies