SG11201804064WA - Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a - Google Patents

Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

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Publication number
SG11201804064WA
SG11201804064WA SG11201804064WA SG11201804064WA SG11201804064WA SG 11201804064W A SG11201804064W A SG 11201804064WA SG 11201804064W A SG11201804064W A SG 11201804064WA SG 11201804064W A SG11201804064W A SG 11201804064WA SG 11201804064W A SG11201804064W A SG 11201804064WA
Authority
SG
Singapore
Prior art keywords
international
rule
gene therapy
hemophilia
vienna
Prior art date
Application number
SG11201804064WA
Inventor
Falko-Günter Falkner
Franziska Horling
Johannes Lengler
Hanspeter Rottensteiner
Friedrich Scheiflinger
Original Assignee
Baxalta Inc
Baxalta GmbH
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 Baxalta Inc, Baxalta GmbH filed Critical Baxalta Inc
Publication of SG11201804064WA publication Critical patent/SG11201804064WA/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
    • 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
    • A61K48/0008—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 '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
    • 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
    • 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
    • A61K48/0066—Manipulation 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • 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
    • 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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • 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 Organization International Bureau IIIIII Hit II EH DID IIIII DEEM II III 111111111111111111111111 RID IIII Human wild-type FVIII MOB rre Refacto-type BDD-FVIII (10) International Publication Number WO 2017/083764 Al WIPO I PCT (43) International Publication Date 18 May 2017 (18.05.2017) 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, 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 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). Declarations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.170) as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) Published: with international search report (Art 21(3)) with sequence listing part of description (Rule 5.2(a)) (51) International Patent Classification: C07K 14/755 (2006.01) C12N 15/86 (2006.01) A61K 48/00 (2006.01) (21) International Application Number: PCT/US2016/061688 (22) International Filing Date: 11 November 2016 (11.11.2016) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/255,323 13 November 2015 (13.11.2015) US (71) Applicants: BAXALTA INCORPORATED [US/US]; 1200 Lakeside Drive, Bannockburn, IL 60015 (US). BAX- ALTA GMBH [CH/CH]; Thurgauerstrasse 130, 8152 Glattpark (Opfikon) (CH). (72) Inventors: FALKNER, Falko-Giinter; Neusiedlzeile 76A, 2304 Orth/Donau (AT). HORLING, Franziska; Bauemfeldgasse 3/9, 2230 Gaenserndorf (AT). LEN- GLER, Johannes; Schrotzbergstrabe 2/23, 1020 Vienna (AT). ROTTENSTEINER, Hanspeter; Haidgasse 10/17, 1020 Vienna (AT). SCHEIFLINGER, Friedrich; Michel- beuemgasse 4/17, 1090 Vienna (AT). (74) Agents: ANTCZAK, Andrew J. et al.; Morgan, Lewis & Bockius LLP, One Market, Spear Street Tower, San Fran- cisco, CA 94105 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, (54) Title: VIRAL VECTORS ENCODING RECOMBINANT FVIII VARIANTS WITH INCREASED EXPRESSION FOR GENE THERAPY OF HEMOPHILIA A W O 20 17 /0837 6 4 Al tigtgall 11; tiff Figure 1 (57) : The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and meth- ods for treating hemophilia A.
SG11201804064WA 2015-11-13 2016-11-11 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a SG11201804064WA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562255323P 2015-11-13 2015-11-13
PCT/US2016/061688 WO2017083764A1 (en) 2015-11-13 2016-11-11 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Publications (1)

Publication Number Publication Date
SG11201804064WA true SG11201804064WA (en) 2018-06-28

Family

ID=57539606

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201804064WA SG11201804064WA (en) 2015-11-13 2016-11-11 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Country Status (18)

Country Link
US (1) US10189889B2 (en)
EP (1) EP3374388A1 (en)
JP (1) JP6695426B2 (en)
KR (1) KR102404550B1 (en)
CN (1) CN108602876B (en)
AU (1) AU2016354550B2 (en)
BR (1) BR112018009732A8 (en)
CA (1) CA3005565A1 (en)
CL (2) CL2018001301A1 (en)
CO (1) CO2018005380A2 (en)
EA (2) EA036944B1 (en)
IL (1) IL259295A (en)
MX (1) MX2018005969A (en)
NZ (1) NZ742555A (en)
SG (1) SG11201804064WA (en)
TW (2) TWI823830B (en)
WO (1) WO2017083764A1 (en)
ZA (1) ZA201803442B (en)

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CN109735547B (en) * 2019-03-19 2022-05-31 青岛蔚蓝生物集团有限公司 Promoter for improving pichia pastoris exogenous protein expression quantity and application thereof
JP2022537555A (en) * 2019-06-20 2022-08-26 武田薬品工業株式会社 Virus-based gene therapy treatment methods
CN112575034B (en) * 2019-09-29 2023-04-25 济南赛尔生物科技股份有限公司 Product for treating hemophilia A and application thereof

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Publication number Publication date
CL2020001611A1 (en) 2020-10-30
TW202229555A (en) 2022-08-01
AU2016354550A1 (en) 2018-06-07
JP6695426B2 (en) 2020-05-20
KR20180070700A (en) 2018-06-26
NZ742555A (en) 2023-12-22
US10189889B2 (en) 2019-01-29
KR102404550B1 (en) 2022-05-31
JP2018537089A (en) 2018-12-20
CA3005565A1 (en) 2017-05-18
CN108602876B (en) 2022-07-05
TW201723180A (en) 2017-07-01
EA036944B1 (en) 2021-01-19
AU2016354550B2 (en) 2020-04-16
CN108602876A (en) 2018-09-28
MX2018005969A (en) 2018-11-29
EA201891138A1 (en) 2018-12-28
EA202092049A1 (en) 2020-11-10
EP3374388A1 (en) 2018-09-19
US20170226188A1 (en) 2017-08-10
BR112018009732A2 (en) 2018-11-21
CL2018001301A1 (en) 2018-12-28
IL259295A (en) 2018-07-31
TWI823830B (en) 2023-12-01
BR112018009732A8 (en) 2019-02-26
CO2018005380A2 (en) 2018-05-31
WO2017083764A1 (en) 2017-05-18
ZA201803442B (en) 2019-07-31

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