SG11201806758VA - Vcn enhancer compositions and methods of using the same - Google Patents

Vcn enhancer compositions and methods of using the same

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Publication number
SG11201806758VA
SG11201806758VA SG11201806758VA SG11201806758VA SG11201806758VA SG 11201806758V A SG11201806758V A SG 11201806758VA SG 11201806758V A SG11201806758V A SG 11201806758VA SG 11201806758V A SG11201806758V A SG 11201806758VA SG 11201806758V A SG11201806758V A SG 11201806758VA
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SG
Singapore
Prior art keywords
international
pge2
massachusetts
cambridge
pct
Prior art date
Application number
SG11201806758VA
Inventor
Melissa Bonner
Olivier Negre
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Bluebird Bio Inc
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Publication of SG11201806758VA publication Critical patent/SG11201806758VA/en

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    • C12N5/0647Haematopoietic stem cells; Uncommitted or multipotent progenitors
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/715Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
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    • 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
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    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector
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    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076L-Iduronidase (3.2.1.76)
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    • C12Y304/14Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
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    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004Adenosine deaminase (3.5.4.4)

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Abstract

w90 80 70 60 50 40- 30 20 10 0 PGE2 z 0 • 6- ! V • 4- o 2- 0 LL 4,414.4. W O 20 17 / 13957 6 Al (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (10) International Publication Number (43) International Publication Date WO 2017/139576 Al 17 August 2017 (17.08.2017) WIPO I PCT 111111111111110111011111111111010111110 011101111111100111111111111111111110111111 • PS • PS + PGE2 F108 100Oug/mL n F108 1000ug/mL + PGE2 • F108 62.5ug/mL n F108 62.5ug/mL + PGE2 (51) International Patent Classification: C12N 5/078 (2010.01) Cl 2N 5/074 (2010.01) (21) International Application Number: PCT/US2017/017372 (22) International Filing Date: 10 February 2017 (10.02.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/294,615 12 February 2016 (12.02.2016) US 62/313,571 25 March 2016 (25.03.2016) US 62/417,085 3 November 2016 (03.11.2016) US 62/429,514 2 December 2016 (02.12.2016) US (71) Applicant: BLUEBIRD BIO, INC. [US/US]; 60 Binney Street, Cambridge, Massachusetts 02141 (US). (72) Inventors: BONNER, Melissa; c/o bluebird bio, Inc., 60 Binney Street, Cambridge, Massachusetts 02141 (US). NEGRE, Olivier; c/o bluebird bio, Inc., 60 Binney Street, Cambridge, Massachusetts 02141 (US). (74) Agents: MASHRUWALA, Mary Anne et al.; Cooley LLP, 1299 Pennsylvania Avenue, NW, Suite 700, Wash- ington, 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, 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, [Continued on next page] (54) Title: VCN ENHANCER COMPOSITIONS AND METHODS OF USING THE SAME (57) : The invention provides improved gene ther- apy methods and compositions. FIG. 5A *p<0.05 compared to 1 (PS - standard transduction) 8 ug/mL 82.5 ug/mL 1000 ug/mL PS F108 F108 FIG. 5B WO 2017/139576 Al MIDEDIMOHNIIMME30111001HIMMERVOIMIE TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, Published: TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, with international search report (Art. 21(3)) 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).
SG11201806758VA 2016-02-12 2017-02-10 Vcn enhancer compositions and methods of using the same SG11201806758VA (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662294615P 2016-02-12 2016-02-12
US201662313571P 2016-03-25 2016-03-25
US201662417085P 2016-11-03 2016-11-03
US201662429514P 2016-12-02 2016-12-02
PCT/US2017/017372 WO2017139576A1 (en) 2016-02-12 2017-02-10 Vcn enhancer compositions and methods of using the same

Publications (1)

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SG11201806758VA true SG11201806758VA (en) 2018-09-27

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EP (2) EP4151720A1 (en)
JP (1) JP6970106B2 (en)
KR (1) KR20180110112A (en)
CN (1) CN108713059A (en)
AU (1) AU2017217813B2 (en)
BR (1) BR112018016450A2 (en)
CA (1) CA3014078A1 (en)
IL (2) IL310683A (en)
MX (1) MX2018009750A (en)
RU (2) RU2021103425A (en)
SG (1) SG11201806758VA (en)
WO (1) WO2017139576A1 (en)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG10201602423TA (en) * 2011-09-30 2016-05-30 Bluebird Bio Inc Compounds For Improved Viral Transduction
SG10201704008RA (en) 2012-11-27 2017-06-29 The Children's Medical Center Corp Targeting Bcl11a Distal Regulatory Elements for Fetal Hemoglobin Reinduction
CA2946309C (en) 2014-04-25 2021-11-09 Michael MILSOM Synthetic bcl11a micrornas for treating hemoglobinopathies
WO2016182917A1 (en) 2015-05-08 2016-11-17 Children's Medical Center Corporation Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction
US11261441B2 (en) 2017-03-29 2022-03-01 Bluebird Bio, Inc. Vectors and compositions for treating hemoglobinopathies
WO2018218135A1 (en) 2017-05-25 2018-11-29 The Children's Medical Center Corporation Bcl11a guide delivery
MX2021001292A (en) * 2018-07-30 2021-07-15 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Methods for gene modification of hematopoietic cells.
CN109055432B (en) * 2018-08-16 2020-10-13 南京科佰生物科技有限公司 Lentivirus infection reagent and preparation method and application thereof
CN109880852A (en) * 2019-03-25 2019-06-14 和元生物技术(上海)股份有限公司 Poloxamer P338 is improving application and method in efficiency of infection of the virus to cell
US20220163512A1 (en) * 2019-03-27 2022-05-26 Bluebird Bio, Inc. Beta-thalassemia potency assay
US20230002784A1 (en) * 2019-06-05 2023-01-05 Orchard Therapeutics (Europe) Limited Compositions and methods for modifying eukaryotic cells
US11597920B2 (en) * 2019-06-25 2023-03-07 Bio-Rad Laboratories, Inc. Compositions and methods for enhancing reverse transcriptase activity and/or reducing the inhibition of reverse transcriptase
EP3766981A1 (en) 2019-07-18 2021-01-20 OZ Biosciences Cationic poloxamers and their use in transduction
CN114761567A (en) * 2019-10-16 2022-07-15 奥查德疗法(欧洲)有限公司 Compositions and methods for modifying eukaryotic cells
CN113025660A (en) * 2019-12-25 2021-06-25 深圳华大生命科学研究院 Kit and method for transfecting human hematopoietic stem cells in vitro by lentiviruses
CN113621611B (en) * 2021-04-26 2024-04-19 北京美康基免生物科技有限公司 Marrow specific promoter and application thereof
CN114457119B (en) * 2022-04-11 2022-08-12 中吉智药(南京)生物技术有限公司 Application of lentiviral vector in preparation of drug for treating beta-thalassemia
CN114438130B (en) * 2022-04-11 2022-07-22 中吉智药(南京)生物技术有限公司 Beta-globin recombinant lentiviral vector and application thereof

Family Cites Families (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US568159A (en) 1896-06-26 1896-09-22 Veneering-machine
US4873192A (en) 1987-02-17 1989-10-10 The United States Of America As Represented By The Department Of Health And Human Services Process for site specific mutagenesis without phenotypic selection
US5061620A (en) 1990-03-30 1991-10-29 Systemix, Inc. Human hematopoietic stem cell
US5635387A (en) 1990-04-23 1997-06-03 Cellpro, Inc. Methods and device for culturing human hematopoietic cells and their precursors
US5460964A (en) 1992-04-03 1995-10-24 Regents Of The University Of Minnesota Method for culturing hematopoietic cells
WO1995000657A1 (en) 1993-06-21 1995-01-05 The Uab Research Foundation Anti-sickling hemoglobin
US5409813A (en) 1993-09-30 1995-04-25 Systemix, Inc. Method for mammalian cell separation from a mixture of cell populations
US5869039A (en) 1993-10-15 1999-02-09 Institut National De La Sante Et De La Recherche Medicale X-linked adrenoleukodystrophy gene and corresponding protein
CA2199832C (en) 1994-09-19 2000-08-01 Philippe Leboulch Anti-sickling beta-globin protein, compositions and methods for treating sickle cell disease
US5677136A (en) 1994-11-14 1997-10-14 Systemix, Inc. Methods of obtaining compositions enriched for hematopoietic stem cells, compositions derived therefrom and methods of use thereof
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6057117A (en) 1996-04-04 2000-05-02 Chiron Corporation Identification and use of selective inhibitors of glycogen synthase kinase 3
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
FR2777909B1 (en) 1998-04-24 2002-08-02 Pasteur Institut USE OF TRIPLEX-STRUCTURED DNA SEQUENCES FOR THE TRANSFER OF NUCLEOTID SEQUENCES IN CELLS, RECOMBINANT VECTORS CONTAINING THESE TRIPLEX SEQUENCES
AU2183900A (en) 1998-12-24 2000-07-31 Alcon Laboratories, Inc. Ep4 receptor agonists for treatment of dry eye
US6822112B1 (en) 1999-08-13 2004-11-23 Taisho Pharmaceutical Co., Ltd. Prostaglandin derivatives
AU784195B2 (en) 1999-11-12 2006-02-16 Baxter Biotech Technology S.A.R.L. Reduced side-effect hemoglobin compositions
CN1272328C (en) 1999-12-17 2006-08-30 希龙公司 Pyrazine based inhibitors of glycogen synthase kinase 3
ATE327751T1 (en) 2000-01-31 2006-06-15 Pfizer Prod Inc USE OF PROSTAGLAND RECEPTOR 4 ACTIVATORS FOR THE TREATMENT OF ACUTE OR CHRONIC RENAL INSUFFICIENCY
US20020013294A1 (en) 2000-03-31 2002-01-31 Delong Mitchell Anthony Cosmetic and pharmaceutical compositions and methods using 2-decarboxy-2-phosphinico derivatives
US6627442B1 (en) * 2000-08-31 2003-09-30 Virxsys Corporation Methods for stable transduction of cells with hiv-derived viral vectors
WO2002088346A2 (en) 2001-05-01 2002-11-07 National Research Council Of Canada A system for inducible expression in eukaryotic cells
US7994144B2 (en) * 2001-07-10 2011-08-09 Johnson & Johnson Research Pty, Limited Process for the preparation of a composition of genetically modified hematopoietic progenitor cells
EP1448182A1 (en) * 2001-11-30 2004-08-25 Pfizer Products Inc. Pharmaceutical compositions and methods for administering ep 2 receptor selective agonists
EP1539754A4 (en) 2002-08-23 2009-02-25 Novartis Vaccines & Diagnostic Benzimidazole quinolinones and uses thereof
US7119200B2 (en) 2002-09-04 2006-10-10 Schering Corporation Pyrazolopyrimidines as cyclin dependent kinase inhibitors
DE60333487D1 (en) 2002-12-13 2010-09-02 Genetix Pharmaceuticals Inc THERAPEUTIC RETROVIRUS VECTORS FOR GENE THERAPY
US6747037B1 (en) 2003-06-06 2004-06-08 Allergan, Inc. Piperidinyl prostaglandin E analogs
CA2585367A1 (en) 2004-10-26 2006-05-04 Allergan, Inc. Therapeutic and delivery methods of prostaglandin ep4 agonists
DE102005062741A1 (en) 2005-12-22 2007-06-28 Bayer Schering Pharma Ag Fluorenes and carbazoles as ligands of the EP2 receptor
CN101495623B (en) 2006-03-24 2013-09-11 儿童医疗中心有限公司 Method to modulate hematopoietic stem cell growth
AU2007314299B2 (en) * 2006-11-03 2013-07-25 Aastrom Biosciences, Inc. Mixed cell populations for tissue repair and separation technique for cell processing
WO2008070082A2 (en) * 2006-12-04 2008-06-12 The Johns Hopkins University Stem-progenitor cell specific micro-ribonucleic acids and uses thereof
AU2008261901A1 (en) * 2007-06-08 2008-12-18 University Of Virginia Patent Foundation Topical poloxamer formulations for enhancing microvascular flow: compositions and uses thereof
EP2341911A4 (en) * 2008-10-01 2012-10-24 Univ North Carolina Hematopoietic protection against chemotherapeutic compounds using selective cyclin-dependent kinase 4/6 inhibitors
JP5836262B2 (en) 2009-03-19 2015-12-24 フェイト セラピューティクス, インコーポレイテッド Compositions containing cyclic AMP enhancers and / or EP ligands and methods of preparing and using the same
KR20150029756A (en) 2011-06-10 2015-03-18 블루버드 바이오, 인코포레이티드. Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy
SG10201602423TA (en) * 2011-09-30 2016-05-30 Bluebird Bio Inc Compounds For Improved Viral Transduction
ES2905250T3 (en) * 2012-02-29 2022-04-07 Helmholtz Zentrum Muenchen Deutsches Forschungszentrum Gesundheit & Umwelt Gmbh Retroviral transduction using poloxamers
WO2014026110A2 (en) * 2012-08-10 2014-02-13 Bluebird Bio, Inc. Compounds for improved viral transduction
SG10201704008RA (en) 2012-11-27 2017-06-29 The Children's Medical Center Corp Targeting Bcl11a Distal Regulatory Elements for Fetal Hemoglobin Reinduction
WO2016014794A1 (en) * 2014-07-25 2016-01-28 Sangamo Biosciences, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells

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IL261002A (en) 2018-09-20
JP2019504635A (en) 2019-02-21
IL261002B1 (en) 2024-03-01
EP4151720A1 (en) 2023-03-22
EP3414321B8 (en) 2023-05-03
BR112018016450A2 (en) 2018-12-26
EP3414321A4 (en) 2019-07-10
IL310683A (en) 2024-04-01
CN108713059A (en) 2018-10-26
AU2017217813A1 (en) 2018-09-20
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MX2018009750A (en) 2019-02-07
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AU2017217813B2 (en) 2023-08-03
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