SG11201908844YA - Vectors and compositions for treating hemoglobinopathies - Google Patents

Vectors and compositions for treating hemoglobinopathies

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Publication number
SG11201908844YA
SG11201908844YA SG11201908844YA SG11201908844YA SG 11201908844Y A SG11201908844Y A SG 11201908844YA SG 11201908844Y A SG11201908844Y A SG 11201908844YA SG 11201908844Y A SG11201908844Y A SG 11201908844YA
Authority
SG
Singapore
Prior art keywords
international
massachusetts
rule
viii
pct
Prior art date
Application number
Inventor
Gabor Veres
David A Williams
Original Assignee
Bluebird Bio Inc
Childrens Medical Center
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 Bluebird Bio Inc, Childrens Medical Center filed Critical Bluebird Bio Inc
Publication of SG11201908844YA publication Critical patent/SG11201908844YA/en

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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • 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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15045—Special targeting system for viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15071—Demonstrated in vivo effect
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16045—Special targeting system for 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16071—Demonstrated in vivo effect
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Abstract

RU9a9 RRE cPPT CoIEI Kanamycin r— f1 Ori bGH poly A -miR223 downsaeam miRNA223 upstream Venus cDNA B-Globin promoter' A-'C HS2 sin 18 R US HS3 Synthetic pA miR223 downstream miRNA223 upstream Exon1 betaGp HS2 W O 2018/ 18369 2 A8 (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) CORRECTED VERSION (19) World Intellectual Property Organization International Bureau (43) International Publication Date 11111111111110111111111111111111111110 1 1 111 11111 11111 11111 11111 1111 1 11 111111 1111 111 1111 (10) International Publication Number WO 2018/183692 A8 04 October 2018 (04.10.2018) WIP0 I PCT (51) International Patent Classification: A 61K 48/00 (2006.01) Cl 2N 15/867 (2006.01) A 61K 31/7088 (2006.01) Cl 2N 5/10 (2006.01) A61K 35/28 (2015.01) C07H 21/04 (2006.01) C12N 15/86 (2006.01) (21) International Application Number: PCT/US2018/025165 (22) International Filing Date: 29 March 2018 (29.03.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/478,375 29 March 2017 (29.03.2017) US 62/489,149 24 April 2017 (24.04.2017) US (71) Applicants: BLUEBIRD BIO, INC. [US/US]; 60 Bin- ney Street, Cambridge, Massachusetts 02142 (US). CHILDREN'S MEDICAL CENTER CORPORATION [IJS/US]; 55 Shattuck St., Boston, Massachusetts 02115 (US). (72) Inventors: VERES, Gabor; c/o bluebird bio. Inc., 60 Binney St., Cambridge, Massachusetts 02142 (US). WIL- LIAMS, David A.; 168 Centre St., Dover, Massachusetts 02030 (US). (74) Agent: ROGEL, Mark E. et al.; Cooley LLP, 1299 Penn- sylvania Avenue, NW, Suite 700, Washington, District of Columbia 20004 (US). (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, JO, 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 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, (54) Title: VECTORS AND COMPOSITIONS FOR TREATING HEMOGLOBINOPATHIES (57) : The invention provides improved gene therapy vectors, compo- FIGURE 1 sitions, and methods. RSV [Continued on next page] WO 2018/183692 A8 I VIII 111111110111111 VIII 11111 OM MI 1 II 111 OM Mil Dill VIII 111110111111101111 I1II 1111 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 4.17(i1)) 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)) (48) Date of publication of this corrected version: 02 May 2019 (02.05.2019) (15) Information about Correction: see Notice of 02 May 2019 (02.05.2019)
SG11201908844Y 2017-03-29 2018-03-29 Vectors and compositions for treating hemoglobinopathies SG11201908844YA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762478375P 2017-03-29 2017-03-29
US201762489149P 2017-04-24 2017-04-24
PCT/US2018/025165 WO2018183692A1 (en) 2017-03-29 2018-03-29 Vectors and compositions for treating hemoglobinopathies

Publications (1)

Publication Number Publication Date
SG11201908844YA true SG11201908844YA (en) 2019-10-30

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ID=63677097

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201908844Y SG11201908844YA (en) 2017-03-29 2018-03-29 Vectors and compositions for treating hemoglobinopathies

Country Status (10)

Country Link
US (1) US20220259594A1 (en)
EP (1) EP3600448A4 (en)
JP (2) JP7228523B2 (en)
CN (1) CN110582305A (en)
AU (1) AU2018243293A1 (en)
BR (1) BR112019020322A2 (en)
CA (1) CA3057862A1 (en)
IL (1) IL269551A (en)
SG (1) SG11201908844YA (en)
WO (1) WO2018183692A1 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3980549A4 (en) * 2019-06-05 2023-07-26 Orchard Therapeutics (Europe) Limited Compositions and methods for modifying eukaryotic cells
EP4038182A1 (en) * 2019-09-30 2022-08-10 Bioverativ Therapeutics Inc. Lentiviral vector formulations
CN117384967A (en) * 2020-04-24 2024-01-12 康霖生物科技（杭州）有限公司 Nucleic acid construct

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102011532B1 (en) * 2011-09-30 2019-08-16 블루버드 바이오, 인코포레이티드. Compounds for improved viral transduction
US10106816B2 (en) * 2012-12-14 2018-10-23 Case Western Reserve University Genomic RNA packaging enhancer element
IL296691B2 (en) * 2014-04-25 2023-11-01 2Seventy Bio Inc Mnd promoter chimeric antigen receptors
BR112016024565A2 (en) * 2014-04-25 2018-01-23 Children's Medical Center Corporation compositions and methods of treating hemoglobinopathies
WO2015164739A1 (en) * 2014-04-25 2015-10-29 Bluebird Bio, Inc. Kappa/lambda chimeric antigen receptors
CN107208093B (en) * 2014-09-04 2022-03-01 纪念斯隆-凯特琳癌症中心 Globulin gene therapy for the treatment of hemoglobinopathies
KR102584938B1 (en) * 2014-12-12 2023-10-05 2세븐티 바이오, 인코포레이티드 Bcma chimeric antigen receptors
AU2016239324B2 (en) * 2015-03-31 2022-04-21 Glycotope Gmbh Eukaryotic expression vectors comprising regulatory elements of the globin gene clusters
KR20180037294A (en) * 2015-08-31 2018-04-11 블루버드 바이오, 인코포레이티드. Anti-sialyl Tn chimeric antigen receptor

Also Published As

Publication number Publication date
WO2018183692A1 (en) 2018-10-04
EP3600448A4 (en) 2021-01-27
CA3057862A1 (en) 2018-10-04
JP2023033551A (en) 2023-03-10
JP2020511991A (en) 2020-04-23
CN110582305A (en) 2019-12-17
AU2018243293A1 (en) 2019-10-31
WO2018183692A8 (en) 2019-05-02
EP3600448A1 (en) 2020-02-05
US20220259594A1 (en) 2022-08-18
IL269551A (en) 2019-11-28
BR112019020322A2 (en) 2020-04-28
JP7228523B2 (en) 2023-02-24

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