PL3360890T3 - Terapia genowa - Google Patents

Terapia genowa

Info

Publication number
PL3360890T3
PL3360890T3 PL17210269T PL17210269T PL3360890T3 PL 3360890 T3 PL3360890 T3 PL 3360890T3 PL 17210269 T PL17210269 T PL 17210269T PL 17210269 T PL17210269 T PL 17210269T PL 3360890 T3 PL3360890 T3 PL 3360890T3
Authority
PL
Poland
Prior art keywords
gene therapy
therapy
gene
Prior art date
Application number
PL17210269T
Other languages
English (en)
Inventor
Christian Groendahl
Tim FUNNELL
Chris HOLLOWOOD
Original Assignee
Syncona IP Holdco Limited
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=55130363&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PL3360890(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Syncona IP Holdco Limited filed Critical Syncona IP Holdco Limited
Publication of PL3360890T3 publication Critical patent/PL3360890T3/pl

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Classifications

    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1725Complement proteins, e.g. anaphylatoxin, C3a or C5a
    • 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
    • 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
    • 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
    • A61K48/0066Manipulation 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
    • 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
    • A61K48/0075Medicinal 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 delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/472Complement proteins, e.g. anaphylatoxin, C3a, C5a
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424Serine endopeptidases (3.4.21)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/51Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/525Virus
    • A61K2039/5258Virus-like particles
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/60Medicinal preparations containing antigens or antibodies characteristics by the carrier linked to the antigen
    • A61K2039/6031Proteins
    • A61K2039/6075Viral proteins
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector 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)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Ophthalmology & Optometry (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Endocrinology (AREA)
PL17210269T 2015-10-28 2016-10-27 Terapia genowa PL3360890T3 (pl)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
GBGB1519086.1A GB201519086D0 (en) 2015-10-28 2015-10-28 Gene Therapy
EP16788764.5A EP3262066B2 (en) 2015-10-28 2016-10-27 Gene therapy
PCT/GB2016/053343 WO2017072515A1 (en) 2015-10-28 2016-10-27 Gene therapy
EP17210269.1A EP3360890B1 (en) 2015-10-28 2016-10-27 Gene therapy

Publications (1)

Publication Number Publication Date
PL3360890T3 true PL3360890T3 (pl) 2020-12-28

Family

ID=55130363

Family Applications (2)

Application Number Title Priority Date Filing Date
PL17210269T PL3360890T3 (pl) 2015-10-28 2016-10-27 Terapia genowa
PL16788764.5T PL3262066T5 (pl) 2015-10-28 2016-10-27 Terapia genowa

Family Applications After (1)

Application Number Title Priority Date Filing Date
PL16788764.5T PL3262066T5 (pl) 2015-10-28 2016-10-27 Terapia genowa

Country Status (19)

Country Link
US (3) US20190255193A1 (pl)
EP (3) EP3262066B2 (pl)
JP (3) JP7126944B2 (pl)
KR (1) KR102922606B1 (pl)
CN (4) CN116271104A (pl)
AU (3) AU2016344523B2 (pl)
CA (1) CA3002125A1 (pl)
DK (2) DK3262066T4 (pl)
ES (2) ES2828050T3 (pl)
GB (1) GB201519086D0 (pl)
HK (1) HK1248723B (pl)
IL (3) IL284577B2 (pl)
MX (1) MX366343B (pl)
NO (1) NO3262066T3 (pl)
PL (2) PL3360890T3 (pl)
RU (1) RU2740038C2 (pl)
SG (1) SG11201802934WA (pl)
WO (1) WO2017072515A1 (pl)
ZA (1) ZA201802462B (pl)

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EP3359663A4 (en) 2015-09-24 2019-08-14 The Trustees of The University of Pennsylvania COMPOSITION AND METHOD FOR THE TREATMENT OF A COMPLEMENT-MEDIATED DISEASE
GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
WO2017180936A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
GB201608046D0 (en) * 2016-05-09 2016-06-22 Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The Treatment of complement-mediated disorders
CN120989074A (zh) * 2017-07-31 2025-11-21 映像生物有限公司 眼部疾病的细胞模型及用于眼部疾病的疗法
WO2019079718A1 (en) * 2017-10-20 2019-04-25 Gemini Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING AGE-RELATED MACULAR DEGENERATION
US12558434B2 (en) 2018-02-20 2026-02-24 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
JP2022512667A (ja) * 2018-10-10 2022-02-07 ウィスコンシン アラムニ リサーチ ファンデーション Kir7.1遺伝子療法ベクターおよびその使用方法
EP3870711A1 (en) * 2018-10-23 2021-09-01 Gemini Therapeutics Inc. Compositions and methods for treating age-related macular degeneration and other diseases
GB201821089D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Codon-optimised complement factor I
GB201821082D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Combination of complement factors i and h, and vector encoding thereof
MX2021011889A (es) * 2019-04-03 2021-10-26 Regenxbio Inc Tratamiento genico para patologias oculares.
MX2022002652A (es) * 2019-09-09 2022-04-06 Univ Pittsburgh Commonwealth Sys Higher Education Metodos para restaurar la funcion lisosomal de celulas epiteliales de pigmento retiniano mediante activacion de tfeb.
IL299048A (en) 2020-06-14 2023-02-01 Vertex Pharma Variants of complement factor - 1 fusion structures and preparations containing them and their uses
GB202009741D0 (en) 2020-06-25 2020-08-12 Freeline Therapeutics Ltd Polynucleotide
JP2023541058A (ja) * 2020-09-09 2023-09-27 ホモロジー・メディシンズ・インコーポレイテッド ベクター化抗体およびその使用
WO2022092294A1 (ja) 2020-10-30 2022-05-05 学校法人慶應義塾 サルコペニア関連疾患等の新規治療および予防
GB202018320D0 (en) * 2020-11-20 2021-01-06 Univ Newcastle Methods of producing recombinant complement proteins
TW202323270A (zh) 2021-11-04 2023-06-16 英商自由行療法有限公司 多肽
CN118973613A (zh) 2022-01-25 2024-11-15 早野元词 抑制包括肌肉减少症的与衰老相关的异常的新型治疗与预防

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GB9803351D0 (en) 1998-02-17 1998-04-15 Oxford Biomedica Ltd Anti-viral vectors
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
GB0009760D0 (en) 2000-04-19 2000-06-07 Oxford Biomedica Ltd Method
ATE423134T1 (de) * 2003-06-10 2009-03-15 Nsgene As Verbesserte sezernierung von neublastin
US8088579B2 (en) 2005-02-14 2012-01-03 University Of Iowa Research Foundation Complement factor H for diagnosis of age-related macular degeneration
MX2008004693A (es) * 2005-10-21 2008-09-03 Catalyst Biosciences Inc Proteasas modificadas que inhiben la activacion del complemento.
PT2044111E (pt) 2006-06-21 2014-11-12 Univ Colorado Regents Abordagem seletiva do fator h do complemento para o tratamento de doenças
CN101657097A (zh) * 2007-03-01 2010-02-24 先进视觉疗法公司 以炎症为特征的疾病的治疗
NZ578873A (en) * 2007-03-01 2012-01-12 Wellstat Ophthalmics Corp Complement factor B analogs and uses for the treatment of complement mediated disease such as inflammation
HUE030719T2 (en) 2007-04-09 2017-05-29 Univ Florida Raav vector compositions having tyrosine-modified capsid proteins and methods for use
GB0816702D0 (en) * 2008-09-12 2008-10-22 Trinity College Dublin Complement proteins
GB0904427D0 (en) 2009-03-13 2009-04-29 Lachmann Peter Treatment of diseases related to hyperactivity of the complement system
TWI775096B (zh) * 2012-05-15 2022-08-21 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
CA2899034A1 (en) * 2013-01-23 2014-07-31 Musc Foundation For Research Development Targeting constructs based on natural antibodies and uses thereof
US20140234275A1 (en) * 2013-02-15 2014-08-21 Jason Williams Method for treating als via the increased production of factor h
CN105682674A (zh) * 2013-04-17 2016-06-15 建新公司 用于治疗和预防黄斑变性的组合物和方法
US9597415B2 (en) * 2013-06-28 2017-03-21 Inserm (Institute National De La Sante Et De La Recherche Medicale) Methods for expressing a polynucleotide of interest in the retina of a subject
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
GB201608046D0 (en) * 2016-05-09 2016-06-22 Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The Treatment of complement-mediated disorders

Also Published As

Publication number Publication date
MX2018005145A (es) 2018-08-09
ZA201802462B (en) 2021-01-27
WO2017072515A1 (en) 2017-05-04
AU2016344523B2 (en) 2022-03-17
US20250319210A1 (en) 2025-10-16
ES2666204T5 (es) 2023-11-03
GB201519086D0 (en) 2015-12-09
DK3360890T3 (da) 2020-11-02
IL258806B (en) 2021-07-29
CN117343960A (zh) 2024-01-05
MX366343B (es) 2019-07-05
JP2018537122A (ja) 2018-12-20
IL304084B2 (en) 2026-03-01
RU2018118954A3 (pl) 2020-03-13
SG11201802934WA (en) 2018-05-30
CN108431030A (zh) 2018-08-21
EP3360890B1 (en) 2020-10-07
EP3262066B2 (en) 2023-06-07
BR112018008731A8 (pt) 2020-03-31
EP3360890A1 (en) 2018-08-15
CN116370655A (zh) 2023-07-04
US20230277689A1 (en) 2023-09-07
IL284577B2 (en) 2023-12-01
US20190255193A1 (en) 2019-08-22
AU2025200979A1 (en) 2025-03-06
JP2024156706A (ja) 2024-11-06
CN108431030B (zh) 2023-10-20
RU2740038C2 (ru) 2020-12-30
CN116271104A (zh) 2023-06-23
JP2022141670A (ja) 2022-09-29
CA3002125A1 (en) 2017-05-04
ES2828050T3 (es) 2021-05-25
PL3262066T5 (pl) 2023-10-30
IL304084A (en) 2023-08-01
EP3786176A1 (en) 2021-03-03
IL284577A (en) 2021-08-31
IL258806A (en) 2018-06-28
IL284577B1 (en) 2023-08-01
BR112018008731A2 (en) 2018-11-06
AU2022203675A1 (en) 2022-06-16
EP3262066A1 (en) 2018-01-03
NO3262066T3 (pl) 2018-07-07
HK1248723B (en) 2019-11-22
DK3262066T3 (en) 2018-05-22
EP3262066B1 (en) 2018-02-07
ES2666204T3 (es) 2018-05-03
DK3262066T4 (da) 2023-08-21
AU2016344523A1 (en) 2018-06-14
KR20180066247A (ko) 2018-06-18
JP7126944B2 (ja) 2022-08-29
IL304084B1 (en) 2025-11-01
RU2018118954A (ru) 2019-12-02
KR102922606B1 (ko) 2026-02-03
PL3262066T3 (pl) 2018-07-31
AU2022203675B2 (en) 2025-03-06

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