MX2018013665A - Tratamiento de trastornos mediados por el complemento. - Google Patents

Tratamiento de trastornos mediados por el complemento.

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Publication number
MX2018013665A
MX2018013665A MX2018013665A MX2018013665A MX2018013665A MX 2018013665 A MX2018013665 A MX 2018013665A MX 2018013665 A MX2018013665 A MX 2018013665A MX 2018013665 A MX2018013665 A MX 2018013665A MX 2018013665 A MX2018013665 A MX 2018013665A
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MX
Mexico
Prior art keywords
complement
treatment
methods
factor
mediated disorders
Prior art date
Application number
MX2018013665A
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English (en)
Inventor
Lachmann Peter
Alexander Ian
Original Assignee
Cambridge Entpr Ltd
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 Cambridge Entpr Ltd filed Critical Cambridge Entpr Ltd
Publication of MX2018013665A publication Critical patent/MX2018013665A/es

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    • 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
    • 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
    • C07K14/472—Complement proteins, e.g. anaphylatoxin, C3a, C5a
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
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    • C07—ORGANIC CHEMISTRY
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    • 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
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    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/86—Viral vectors
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Mycology (AREA)
  • Neurology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Neurosurgery (AREA)

Abstract

Se describen métodos de tratamiento de trastornos mediados por el complemento, en particular trastornos asociados con la hiperactividad del ciclo de retroalimentación de C3b del complemento (por ejemplo, degeneración macular relacionada con la edad (AMD)) usando terapia génica. De acuerdo con los métodos, los niveles del factor I del complemento se elevan con la administración de un vector viral recombinante que codifica el factor I, de tal manera que una cantidad terapéuticamente eficaz del factor I codificado es expresada desde el vector en el sujeto. También se describen vectores virales recombinantes que codifican el factor I, partículas de virus recombinantes que encapsulan los vectores, y su uso en los métodos de tratamiento.
MX2018013665A 2016-05-09 2017-05-03 Tratamiento de trastornos mediados por el complemento. MX2018013665A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB1608046.7A GB201608046D0 (en) 2016-05-09 2016-05-09 Treatment of complement-mediated disorders
PCT/GB2017/051233 WO2017194912A1 (en) 2016-05-09 2017-05-03 Treatment of complement-mediated disorders

Publications (1)

Publication Number Publication Date
MX2018013665A true MX2018013665A (es) 2019-07-18

Family

ID=56297354

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018013665A MX2018013665A (es) 2016-05-09 2017-05-03 Tratamiento de trastornos mediados por el complemento.

Country Status (14)

Country Link
US (1) US20200147240A1 (es)
EP (1) EP3454910A1 (es)
JP (1) JP7178905B2 (es)
KR (2) KR20190019065A (es)
CN (1) CN109328076B (es)
AU (2) AU2017264750B2 (es)
CA (1) CA3022500A1 (es)
GB (1) GB201608046D0 (es)
IL (1) IL262852A (es)
MX (1) MX2018013665A (es)
RU (1) RU2768982C2 (es)
SG (1) SG11201809543QA (es)
WO (1) WO2017194912A1 (es)
ZA (1) ZA201807218B (es)

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GB201519086D0 (en) 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
GB201800620D0 (en) * 2018-01-15 2018-02-28 Univ Manchester C3b Binding Polypeptide
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
AR122286A1 (es) * 2019-07-17 2022-08-31 Gemini Therapeutics Sub Inc Anticuerpos potenciadores del factor h y usos de estos
MX2022004939A (es) * 2019-10-23 2022-08-10 Gemini Therapeutics Sub Inc Metodos para tratar pacientes que tienen mutaciones de cfh con proteinas de cfh recombinantes.
GB202009741D0 (en) * 2020-06-25 2020-08-12 Freeline Therapeutics Ltd Polynucleotide
EP4214242A1 (en) * 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized antibodies for anti-viral therapy
GB202018320D0 (en) * 2020-11-20 2021-01-06 Univ Newcastle Methods of producing recombinant complement proteins

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WO1998010088A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
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EP2438931B1 (en) 2004-09-22 2013-11-13 St. Jude Children's Research Hospital Improved expression of factor IX in gene therapy vectors
BRPI0812767A2 (pt) * 2007-06-07 2014-12-02 Genentech Inc Anticorpos para c3b e métodos para prevenção e tratamento de distúrbios associados ao complemento
JP5797551B2 (ja) * 2008-04-22 2015-10-21 フエー・イー・ベー・フエー・ゼツト・ウエー 肝特異的核酸調節要素ならびにその方法および用途
GB0904427D0 (en) * 2009-03-13 2009-04-29 Lachmann Peter Treatment of diseases related to hyperactivity of the complement system
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Also Published As

Publication number Publication date
CN109328076A (zh) 2019-02-12
CN109328076B (zh) 2024-03-01
AU2017264750A1 (en) 2018-12-13
RU2018143411A3 (es) 2020-09-30
KR20230047420A (ko) 2023-04-07
JP7178905B2 (ja) 2022-11-28
IL262852A (en) 2018-12-31
RU2768982C2 (ru) 2022-03-28
AU2023202099A1 (en) 2023-07-06
SG11201809543QA (en) 2018-11-29
AU2017264750B2 (en) 2023-01-05
WO2017194912A1 (en) 2017-11-16
RU2018143411A (ru) 2020-06-10
JP2019519494A (ja) 2019-07-11
EP3454910A1 (en) 2019-03-20
KR20190019065A (ko) 2019-02-26
CA3022500A1 (en) 2017-11-16
GB201608046D0 (en) 2016-06-22
ZA201807218B (en) 2023-12-20
US20200147240A1 (en) 2020-05-14

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