SG11201811603WA - Novel adeno-associated virus capsid proteins - Google Patents

Novel adeno-associated virus capsid proteins

Info

Publication number
SG11201811603WA
SG11201811603WA SG11201811603WA SG11201811603WA SG11201811603WA SG 11201811603W A SG11201811603W A SG 11201811603WA SG 11201811603W A SG11201811603W A SG 11201811603WA SG 11201811603W A SG11201811603W A SG 11201811603WA SG 11201811603W A SG11201811603W A SG 11201811603WA
Authority
SG
Singapore
Prior art keywords
international
capsid proteins
novato
novel
publication
Prior art date
Application number
SG11201811603WA
Inventor
Peter Colosi
Michael Lochrie
Robert Ng
Original Assignee
Biomarin Pharm Inc
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
Priority to US201662366838P priority Critical
Application filed by Biomarin Pharm Inc filed Critical Biomarin Pharm Inc
Priority to PCT/US2017/043703 priority patent/WO2018022608A2/en
Publication of SG11201811603WA publication Critical patent/SG11201811603WA/en

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    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N2750/00011MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA Viruses ssDNA Viruses
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2750/00011MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA Viruses ssDNA Viruses
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA Viruses
    • C12N2750/00011MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA Viruses ssDNA Viruses
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14144Chimeric viral vector comprising heterologous viral elements for production of another viral 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
    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/021Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property MD 1101111 0 DOI HIM 011101 0 01111111111111111111111111111111111111111111111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\" WO 2018/022608 A3 01 February 2018 (01.02.2018) W I P0 I P C T (51) International Patent Classification: C07K 14/005 (2006.01) A61K 48/00 (2006.01) Published: C12N 15/86 (2006.01) — with international search report (Art 21(3)) — before the expiration of the time limit for amending the (21) International Application Number: claims and to be republished in the event of receipt of PCT/US2017/043703 amendments (Rule 48.2(h)) (22) International Filing Date: — with sequence listing part of description (Rule 5.2(a)) 25 July 2017 (25.07.2017) (88) Date of publication of the international search report: 01 March 2018 (01.03.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/366,838 26 July 2016 (26.07.2016) US (71) Applicant: BIOMARIN PHARMACEUTICAL INC. [US/US]; 105 Digital Drive, Novato, CA 94949 (US). (72) Inventors: COLOSI, Peter, Cameron; 105 Digital Drive, Novato, CA 94949 (US). LOCHRIE, Michael; 105 Digital Drive, Novato, CA 94949 (US). NG, Robert; 105 Digital Drive, Novato, CA 94949 (US). Agent: SINTICH, Sharon, M.; Marshall, Gerstein & = (74) Borun LLP, 233 S. Wacker Drive, 6300 Willis Tower, Chicago, IL 60606-6357 (US). — 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, JO, JP, KE, KG, KH, KN, KP, (81) = = = _ 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. _ Designated States (unless otherwise indicated, for every — (84) _ _ kind of regional protection available): ARIPO (BW, GH, = GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, = UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, = TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, = EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, — MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). = M GC © ei ei © (54) Title: NOVEL ADENO-ASSOCIATED VIRUS CAPSID PROTEINS --.... ,-1 (57) : The invention relates to novel adeno-associated virus (AAV) capsid proteins, AAV particles comprising a novel capsid 0 protein, polynucleotides encoding these capsid proteins and AAV vectors expressing these capsid proteins. The invention also relates to N methods of making the herein described AAV vectors expressing the novel capsid proteins of the invention and associated therapeutic 0 uses of thereof.
SG11201811603WA 2016-07-26 2017-07-25 Novel adeno-associated virus capsid proteins SG11201811603WA (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
US201662366838P true 2016-07-26 2016-07-26
PCT/US2017/043703 WO2018022608A2 (en) 2016-07-26 2017-07-25 Novel adeno-associated virus capsid proteins

Publications (1)

Publication Number Publication Date
SG11201811603WA true SG11201811603WA (en) 2019-02-27

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201811603WA SG11201811603WA (en) 2016-07-26 2017-07-25 Novel adeno-associated virus capsid proteins

Country Status (15)

Country Link
EP (1) EP3491008A2 (en)
JP (1) JP2019525758A (en)
KR (1) KR20190032375A (en)
CN (1) CN109715650A (en)
AU (1) AU2017301600A1 (en)
BR (1) BR112019001532A2 (en)
CA (1) CA3031026A1 (en)
CL (2) CL2019000167A1 (en)
IL (1) IL263801D0 (en)
MX (1) MX2019000962A (en)
PE (1) PE20190401A1 (en)
PH (1) PH12019500068A1 (en)
RU (1) RU2019103983A3 (en)
SG (1) SG11201811603WA (en)
WO (1) WO2018022608A2 (en)

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GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
PT3356390T (en) 2015-09-28 2021-04-21 Univ Florida Methods and compositions for antibody-evading virus vectors
WO2019169004A1 (en) * 2018-02-27 2019-09-06 The Trustees Of The University Of Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
CA3093347A1 (en) * 2018-04-05 2019-10-10 Genethon Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism
TW202005978A (en) * 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 Novel liver targeting adeno-associated viral vectors
WO2020198737A1 (en) * 2019-03-28 2020-10-01 The General Hospital Corporation Engineered adeno-associated (aav) vectors for transgene expression
US20200362368A1 (en) 2019-05-14 2020-11-19 Biomarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
WO2021073568A1 (en) * 2019-10-16 2021-04-22 Wuxi Apptec (Shanghai) Co., Ltd. A novel aav variant

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US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
AU5557501A (en) 2000-04-28 2001-11-12 Univ Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
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WO2006119432A2 (en) * 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
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MX2019000962A (en) 2019-08-01
CL2020003176A1 (en) 2021-06-04
CL2019000167A1 (en) 2019-05-24
AU2017301600A1 (en) 2019-01-17
RU2019103983A (en) 2020-08-26
CN109715650A (en) 2019-05-03
CA3031026A1 (en) 2018-02-01
BR112019001532A2 (en) 2019-09-10
JP2019525758A (en) 2019-09-12
PE20190401A1 (en) 2019-03-13
PH12019500068A1 (en) 2019-10-21
WO2018022608A3 (en) 2018-03-01
WO2018022608A2 (en) 2018-02-01
EP3491008A2 (en) 2019-06-05
IL263801D0 (en) 2019-01-31
RU2019103983A3 (en) 2021-03-03
KR20190032375A (en) 2019-03-27

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