SG11201809543QA - Treatment of complement-mediated disorders - Google Patents

Treatment of complement-mediated disorders

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Publication number
SG11201809543QA
SG11201809543QA SG11201809543QA SG11201809543QA SG11201809543QA SG 11201809543Q A SG11201809543Q A SG 11201809543QA SG 11201809543Q A SG11201809543Q A SG 11201809543QA SG 11201809543Q A SG11201809543Q A SG 11201809543QA SG 11201809543Q A SG11201809543Q A SG 11201809543QA
Authority
SG
Singapore
Prior art keywords
international
complement
treatment
factor
westmead
Prior art date
Application number
SG11201809543QA
Inventor
Peter Lachmann
Ian Alexander
Original Assignee
Cambridge Entpr Ltd
The Sydney Childrens Hospitals Network Randwick And Westmead
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, The Sydney Childrens Hospitals Network Randwick And Westmead filed Critical Cambridge Entpr Ltd
Publication of SG11201809543QA publication Critical patent/SG11201809543QA/en

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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
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • C07K14/75—Fibrinogen
    • 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
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    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
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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
    • C12N2750/14123—Virus like particles [VLP]
    • 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
    • C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • 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
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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

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 1111111011110111111111111110101110 III 0111110111111111110111011111110 III 0111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\" WO 2017/194912 Al 16 November 2017 (16.11.2017) WIPO I PCT (51) International Patent Classification: MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, A61K 48/00 (2006.01) CO7K 14/47 (2006.01) TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). (21) International Application Number: PCT/GB2017/051233 Published: (22) International Filing Date: — with international search report (Art. 21(3)) 03 May 2017 (03.05.2017) — with sequence listing part of description (Rule 5.2(a)) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 1608046.7 09 May 2016 (09.05.2016) GB (71) Applicants: CAMBRIDGE ENTERPRISE LIMIT- ED [GB/GB]; The Old Schools, Trinity Lane, Cam- bridge Cambridgeshire CB2 1TN (GB). THE SYD- NEY CHILDREN'S HOSPITALS NETWORK (RAND- WICK AND WESTMEAD) [AU/AU]; (incorporating The Royal Alexandra Hospital For Children), Cnr Hawkesbury Road and Hainsworth Street, Westmead, New South Wales (AU). = (72) Inventors: LACHMANN, Peter; Department of Veteri- _ nary Medicine, University of Cambridge, Madingley Road, = Cambridge Cambridgeshire CB3 OES (GB). ALEXAN- DER, Ian; Children's Medical Research Institute, 214 Hawkesbury Road, Westmead, Sydney, New South Wales = 2145 (AU). (74) Agent: O'BRIEN Simon; D Young & Co LLP, 120 Hol- born, London, EC1N 2DY (GB). (81) Designated States (unless otherwise indicated, for every — kind of national protection available): AE, AG, AL, AM, = AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, = CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, = = KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, = PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, = SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, = — TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. = — (84) Designated States (unless otherwise indicated, for every = kind ARIPO GH, of regional protection available): (BW, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, — UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, 1-1 TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, .4 EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, ei ,-1 C:N (54) Title: TREATMENT OF COMPLEMENT-MEDIATED DISORDERS 71' C:N (57) : Methods of treatment of complement-mediated disorders, in particular disorders associated with over-activity of the 1-1 ---- complement C3b feedback cycle (for example, age-related macular degeneration (AMD)), using gene therapy is described. According IN to the methods, levels of complement Factor I are elevated by administration of a recombinant viral vector encoding Factor I such that a © therapeutically effective amount of the encoded Factor I is expressed from the vector in the subject. Recombinant viral vectors encoding N Factor I, recombinant virus particles encapsidating the vectors, and their use in the methods of treatment, is also described. O
SG11201809543QA 2016-05-09 2017-05-03 Treatment of complement-mediated disorders SG11201809543QA (en)

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
SG11201809543QA true SG11201809543QA (en) 2018-11-29

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SG11201809543QA SG11201809543QA (en) 2016-05-09 2017-05-03 Treatment of complement-mediated disorders

Country Status (14)

Country Link
US (1) US20200147240A1 (en)
EP (1) EP3454910A1 (en)
JP (1) JP7178905B2 (en)
KR (2) KR20230047420A (en)
CN (1) CN109328076B (en)
AU (2) AU2017264750B2 (en)
CA (1) CA3022500A1 (en)
GB (1) GB201608046D0 (en)
IL (1) IL262852A (en)
MX (1) MX2018013665A (en)
RU (1) RU2768982C2 (en)
SG (1) SG11201809543QA (en)
WO (1) WO2017194912A1 (en)
ZA (1) ZA201807218B (en)

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GB201800620D0 (en) * 2018-01-15 2018-02-28 Univ Manchester C3b Binding Polypeptide
WO2020086735A1 (en) * 2018-10-23 2020-04-30 Gemini Therapeutics Inc. Compositions and methods for treating age-related macular degeneration and other diseases
GB201821082D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Combination of complement factors i and h, and vector encoding thereof
GB201821089D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Codon-optimised complement factor I
JP2022541275A (en) * 2019-07-17 2022-09-22 ジェミニ・セラピューティクス・サブ・インコーポレイテッド Factor H-enhancing antibodies and uses thereof
MX2022004939A (en) * 2019-10-23 2022-08-10 Gemini Therapeutics Sub Inc Methods for treating patients having cfh mutations with recombinant cfh proteins.
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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Publication number Publication date
AU2017264750A1 (en) 2018-12-13
RU2018143411A (en) 2020-06-10
RU2018143411A3 (en) 2020-09-30
RU2768982C2 (en) 2022-03-28
AU2017264750B2 (en) 2023-01-05
CN109328076A (en) 2019-02-12
US20200147240A1 (en) 2020-05-14
GB201608046D0 (en) 2016-06-22
IL262852A (en) 2018-12-31
EP3454910A1 (en) 2019-03-20
JP7178905B2 (en) 2022-11-28
CN109328076B (en) 2024-03-01
WO2017194912A1 (en) 2017-11-16
AU2023202099A1 (en) 2023-07-06
KR20190019065A (en) 2019-02-26
JP2019519494A (en) 2019-07-11
ZA201807218B (en) 2023-12-20
CA3022500A1 (en) 2017-11-16
KR20230047420A (en) 2023-04-07
MX2018013665A (en) 2019-07-18

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