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Extract from the Register of European Patents

EP About this file: EP3598984

EP3598984 - COMPOSITIONS AND METHODS TO TREATING HEMOGLOBINOPATHIES [Right-click to bookmark this link]
StatusThe patent has been granted
Status updated on  08.03.2024
Database last updated on 17.07.2024
FormerGrant of patent is intended
Status updated on  01.11.2023
FormerExamination is in progress
Status updated on  05.03.2021
FormerRequest for examination was made
Status updated on  31.07.2020
FormerThe application has been published
Status updated on  27.12.2019
Most recent event   Tooltip08.03.2024(Expected) grantpublished on 10.04.2024  [2024/15]
Applicant(s)For all designated states
The Children's Medical Center Corporation
300 Longwood Avenue
Boston, MA 02115 / US
[2024/11]
Former [2020/05]For all designated states
Children's Medical Center Corporation
55 Shattuck Street
Boston, MA 02115 / US
Inventor(s)01 / MILSOM, Michael
Schubertstrasse 16
Bammental 69245 / DE
02 / WILLIAMS, David A.
168 Centre Street
Dover, MA Massachusetts 02030 / US
03 / GREGORY, Richard
140 Pleasant Street
Brookline, MA Massachusetts 02446 / US
 [2020/05]
Representative(s)Crease, Devanand John
Keltie LLP
No.1 London Bridge
London SE1 9BA / GB
[2020/05]
Application number, filing date19179502.024.04.2015
[2020/05]
Priority number, dateUS201461984247P25.04.2014         Original published format: US 201461984247 P
US201462066783P21.10.2014         Original published format: US 201462066783 P
[2020/05]
Filing languageEN
Procedural languageEN
PublicationType: A1 Application with search report 
No.:EP3598984
Date:29.01.2020
Language:EN
[2020/05]
Type: B1 Patent specification 
No.:EP3598984
Date:10.04.2024
Language:EN
[2024/15]
Search report(s)(Supplementary) European search report - dispatched on:EP07.01.2020
ClassificationIPC:A61K48/00, C12N15/113
[2020/05]
CPC:
A61P7/00 (EP); C12N15/113 (EP,IL,KR,US); C12Q1/6806 (RU);
C12N15/1135 (US); A61K35/28 (US); A61K48/00 (KR);
A61P43/00 (EP); A61P7/06 (EP); C12N7/00 (KR,US);
C12N2310/14 (IL); C12N2310/141 (EP,IL,KR,US); C12N2310/3519 (EP,IL,KR,US);
C12N2310/531 (IL,KR,US); C12N2740/10032 (US); C12N2740/10043 (US);
C12N2740/15032 (KR); C12N2740/16032 (KR) (-)
C-Set:
C12N2310/14, C12N2310/531 (EP,US)
Designated contracting statesAL,   AT,   BE,   BG,   CH,   CY,   CZ,   DE,   DK,   EE,   ES,   FI,   FR,   GB,   GR,   HR,   HU,   IE,   IS,   IT,   LI,   LT,   LU,   LV,   MC,   MK,   MT,   NL,   NO,   PL,   PT,   RO,   RS,   SE,   SI,   SK,   SM,   TR [2020/36]
Former [2020/05]AL,  AT,  BE,  BG,  CH,  CY,  CZ,  DE,  DK,  EE,  ES,  FI,  FR,  GB,  GR,  HR,  HU,  IE,  IS,  IT,  LI,  LT,  LU,  LV,  MC,  MK,  MT,  NL,  NO,  PL,  PT,  RO,  RS,  SE,  SI,  SK,  SM,  TR 
TitleGerman:ZUSAMMENSETZUNGEN UND VERFAHREN ZUR BEHANDLUNG VON HÄMOGLOBINOPATHIEN[2020/05]
English:COMPOSITIONS AND METHODS TO TREATING HEMOGLOBINOPATHIES[2020/05]
French:COMPOSITIONS ET PROCÉDÉS POUR LE TRAITEMENT D'HÉMOGLOBINOPATHIES[2020/05]
Examination procedure28.07.2020Examination requested  [2020/36]
28.07.2020Date on which the examining division has become responsible
05.03.2021Despatch of a communication from the examining division (Time limit: M06)
31.08.2021Reply to a communication from the examining division
02.11.2023Communication of intention to grant the patent
04.03.2024Fee for grant paid
04.03.2024Fee for publishing/printing paid
04.03.2024Receipt of the translation of the claim(s)
Parent application(s)   TooltipEP15783357.5  / EP3134130
Fees paidRenewal fee
10.07.2019Renewal fee patent year 03
10.07.2019Renewal fee patent year 04
10.07.2019Renewal fee patent year 05
27.04.2020Renewal fee patent year 06
27.04.2021Renewal fee patent year 07
27.04.2022Renewal fee patent year 08
27.04.2023Renewal fee patent year 09
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Responsibility for the accuracy, completeness or quality of the data displayed under the link provided lies entirely with the Unified Patent Court.
Documents cited:Search[A]US2011182867  (ORKIN STUART H [US], et al)[A] 1-15;
 [A]US2014018410  (NOVOBRANTSEVA TATIANA [US], et al) [A] 1-15;
 [A]  - RENELLA RAFFAELE ET AL, "Hematopoietic SIN Lentiviral Micro RNA-Mediated Silencing of BCL11A: Pre-Clinical Evidence for a Sickle Cell Disease Gene-Therapy Trial (Abstract 753)", & 54TH ANNUAL MEETING AND EXPOSITION OF THE AMERICAN-SOCIETY-OF-HEMATOLOGY (ASH); ATLANTA, GA, USA; DECEMBER 08 -11, 2012, vol. 120, no. 21, ISSN 0006-4971, (201211), Blood, URL: http://www.bloodjournal.org/content/120/21/753?sso-checked=true, (20171110), XP002775471 [A] 1-15 * the whole document *
by applicantUS5681599
 WO9318759
 US5460964
 US5635387
 US5677136
 US5716827
 US5750397
 US5759793
 US6365150
 US8383604
    - KAUF et al., American Journal of Hematology, (20090000), vol. 84, pages 323 - 327
    - AMENDAH et al., American Journal of Preventive Medicine, (20100000), vol. 38, pages 550 - 556
    - PLATT et al., New England Journal of Medicine, (19940000), vol. 330, pages 1639 - 1644
    - PAULING et al., Science, (19490000), vol. 110, pages 543 - 548
    - HSIEH et al., New England Journal of Medicine, (20090000), vol. 361, pages 2309 - 2317
    - J. H. JANDL, Blood: Textbook of Hematology, Little, Brown and Company, (19960000), pages 544 - 545
    - ATWEH, Semin. Hematol., (20010000), vol. 38, no. 4, pages 367 - 73
    - PEMBREY et al., Br. J. Haematol., (19780000), vol. 40, pages 415 - 429
    - JANE et al., Br. J. Haematol., (19980000), vol. 102, pages 415 - 422
    - BUNN, N. Engl. J. Med., (19930000), vol. 328, pages 129 - 131
    - AMENDOLA, M. et al., "Regulated and multiple miRNA and siRNA delivery into primary cells by a lentiviral platform", Mol Ther, (20090000), vol. 17, pages 1039 - 1052
    - PAWLIUK et al., Science, (20010000), vol. 294, page 2368
    - IMREN et al., PNAS, (20020000), vol. 99, page 14380
    - COBURN, G.CULLEN, B., J. Virology, (20020000), vol. 76, no. 18, page 9225
    - STEWART et al., RNA, (20030400), vol. 9, no. 4, pages 493 - 501
    - JACKSON et al., Nature Biotechnology, (20030000), vol. 6, pages 635 - 637
    - BRAASCH et al., Biochemistry, (20030000), vol. 42, pages 7967 - 7975
    - MASTERS, J. R. et al., Proc. Natl. Acad. Sci., (20010000), vol. 100, pages 8012 - 8017
    - XIAO-FENG QIN et al., Proc. Natl. Acad. Sci. U.S.A., vol. 100, pages 183 - 188
    - ELBASHIR, S. M. et al., Nature, (20010000), vol. 411, pages 494 - 498
    - ELBASHIR, S. M.W. LENDECKELT. TUSCHL, Genes & Development, (20010000), vol. 15, pages 188 - 200
    - HARBORTH, J. et al., J. Cell Science, (20010000), vol. 114, pages 4557 - 4565
    - TUSCHL, T. et al., Genes & Development, (19990000), vol. 13, pages 3191 - 3197
    - PADDISON, P. J. et al., Genes Dev., (20020000), vol. 16, pages 948 - 958
    - MCMANUS, M. T. et al., RNA, (20020000), vol. 8, pages 842 - 850
    - MIYAGISHI, M. et al., Nat. Biotechnol., (20020000), vol. 20, pages 500 - 505
    - YU, J. Y. et al., Proc. Natl. Acad. Sci., (20020000), vol. 99, pages 6047 - 6052
    - BRUMMELKAMP, T. et al., Cancer Cell, (20020000), vol. 2, page 243
    - ZENG, Y. et al., Mol. Cell, (20020000), vol. 9, pages 1327 - 1333
    - RUBINSON, D. A. et al., Nat. Genet., (20030000), vol. 33, pages 401 - 406
    - STEWART, S. A. et al., RNA, (20030000), vol. 9, pages 493 - 501
    - MILLER, A. D., Blood, (19900000), vol. 76, page 271
    - ALT, M., J. Hepatol., (19950000), vol. 23, pages 746 - 633
    - N. R. LANDAU et al., J. Virol., (19920000), vol. 66, pages 5110 - 5113
    - KAY, M. A., Chest, (19970000), vol. 111, no. 6, pages 1385 - 1425
    - FERRY, N.HEARD, J. M., Hum. Gene Ther., (19980000), vol. 9, pages 1975 - 81
    - SHIRATORY, Y. et al., Liver, (19990000), vol. 19, pages 265 - 74
    - OKA, K. et al., Curr. Opin. Lipidol., (20000000), vol. 11, pages 179 - 86
    - THULE, P. M.LIU, J. M., Gene Ther., (20000000), vol. 7, pages 1744 - 52
    - YANG, N. S., Crit. Rev. Biotechnol., (19920000), vol. 12, pages 335 - 56
    - BRODY, S. L.CRYSTAL, R. G., Ann. N.Y. Acad. Sci., (19940000), vol. 716, pages 90 - 101
    - STRAYER, D. S., Expert Opin. Investig. Drugs, (19990000), vol. 8, pages 2159 - 2172
    - SMITH-ARICA, J. R.BARTLETT, J. S., Curr. Cardiol. Rep., (20010000), vol. 3, pages 43 - 49
    - LEE, H. C. et al., Nature, (20000000), vol. 408, pages 483 - 8
    - XIA, H. et al., Nat Biotechnol, (20020000), vol. 20, no. 10, page 1006
    - LEE, Y. et al., "The nuclear RNase III Drosha initiates microRNA processing", Nature, (20030000), vol. 425, doi:doi:10.1038/nature01957, pages 415 - 419, XP002974889

DOI:   http://dx.doi.org/10.1038/nature01957
    - HA, M.KIM, V. N., "Regulation of microRNA biogenesis. Nature reviews", Molecular cell biology, (20140000), vol. 15, pages 509 - 524
    - WINTER, J. et al., "Many roads to maturity: microRNA biogenesis pathways and their regulation", Nature cell biology, (20090000), vol. 11, pages 228 - 234
    - KHVOROVA, A. et al., "Functional siRNAs and miRNAs exhibit strand bias", Cell, (20030000), vol. 115, doi:doi:10.1016/S0092-8674(03)00801-8, pages 209 - 216, XP002609792

DOI:   http://dx.doi.org/10.1016/S0092-8674(03)00801-8
    - SCHWARZ, D. S. et al., "Asymmetry in the assembly of the RNAi enzyme complex", Cell, (20030000), vol. 115, doi:doi:10.1016/S0092-8674(03)00759-1, pages 199 - 208, XP002521543

DOI:   http://dx.doi.org/10.1016/S0092-8674(03)00759-1
    - LAI, E. C., "Micro RNAs are complementary to 3' UTR sequence motifs that mediate negative post-transcriptional regulation", Nature genetics, (20020000), vol. 30, pages 363 - 364
    - LEWIS, B. P. et al., "Prediction of mammalian microRNA targets", Cell, (20030000), vol. 115, doi:doi:10.1016/S0092-8674(03)01018-3, pages 787 - 798, XP002295226

DOI:   http://dx.doi.org/10.1016/S0092-8674(03)01018-3
    - PARK, J. E. et al., "Dicer recognizes the 5' end of RNA for efficient and accurate processing", Nature, (20110000), vol. 475, pages 201 - 205
    - GRIMM, D. et al., "Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways", Nature, (20060000), vol. 441, doi:doi:10.1038/NATURE04791, pages 537 - 541, XP003027602

DOI:   http://dx.doi.org/10.1038/NATURE04791
    - MCBRIDE, J. L. et al., "Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi", Proc Natl Acad Sci U S A, (20080000), vol. 105, pages 5868 - 5873
    - KHAN, A. A. et al., "Transfection of small RNAs globally perturbs gene regulation by endogenous microRNAs", Nat Biotechnol, (20090000), vol. 27, pages 549 - 555
    - YI, R. et al., "Overexpression of exportin 5 enhances RNA interference mediated by short hairpin RNAs and microRNAs", RNA, (20050000), vol. 11, doi:doi:10.1261/rna.7233305, pages 220 - 226, XP002488883

DOI:   http://dx.doi.org/10.1261/rna.7233305
    - BORNER, K. et al., "Robust RNAi enhancement via human Argonaute-2 overexpression from plasmids, viral vectors and cell lines", Nucleic Acids Res, (20130000), vol. 41, doi:doi:10.1093/nar/gkt836, page e199, XP055602586

DOI:   http://dx.doi.org/10.1093/nar/gkt836
    - GRIMM, D. et al., "Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver", The Journal of clinical investigation, (20100000), vol. 120, pages 3106 - 3119
    - DIEDERICHS, S.HABER, D. A., "Dual role for argonautes in microRNA processing and posttranscriptional regulation of microRNA expression", Cell, (20070000), vol. 131, pages 1097 - 1108
    - PERSENGIEV, S. P. et al., "Nonspecific, concentration-dependent stimulation and repression of mammalian gene expression by small interfering RNAs (siRNAs", RNA, (20040000), vol. 10, doi:doi:10.1261/rna5160904, pages 12 - 18, XP002369414

DOI:   http://dx.doi.org/10.1261/rna5160904
    - FISH, R. J.KRUITHOF, E. K., "Short-term cytotoxic effects and long-term instability of RNAi delivered using lentiviral vectors", BMC Mol Biol, (20040000), vol. 5, no. 9
    - JACKSON, A. L.LINSLEY, P. S., "Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application", Nat Rev Drug Discov, (20100000), vol. 9, doi:doi:10.1038/nrd3010, pages 57 - 67, XP055391201

DOI:   http://dx.doi.org/10.1038/nrd3010
    - MARTIN, J. N. et al., "Lethal toxicity caused by expression of shRNA in the mouse striatum: implications for therapeutic design", Gene Ther, (20110000), vol. 18, pages 666 - 673
    - ZENG, Y. et al., "Both natural and designed micro RNAs can inhibit the expression of cognate mRNAs when expressed in human cells", Molecular cell, (20020000), vol. 9, doi:doi:10.1016/S1097-2765(02)00541-5, pages 1327 - 1333, XP002394626

DOI:   http://dx.doi.org/10.1016/S1097-2765(02)00541-5
    - FELLMANN, C. et al., "An optimized microRNA backbone for effective single-copy RNAi", Cell Rep, (20130000), vol. 5, doi:doi:10.1016/j.celrep.2013.11.020, pages 1704 - 1713, XP055140183

DOI:   http://dx.doi.org/10.1016/j.celrep.2013.11.020
    - SANKARAN, V. G. et al., "Human fetal hemoglobin expression is regulated by the developmental stage-specific repressor BCL11A", Science, (20080000), vol. 322, doi:doi:10.1126/science.1165409, pages 1839 - 1842, XP055568754

DOI:   http://dx.doi.org/10.1126/science.1165409
    - XU, J. et al., "Correction of sickle cell disease in adult mice by interference with fetal hemoglobin silencing", Science, (20110000), vol. 334, doi:doi:10.1126/science.1211053, pages 993 - 996, XP055568725

DOI:   http://dx.doi.org/10.1126/science.1211053
    - CULLEN, B. R., "Derivation and function of small interfering RNAs and microRNAs", Virus Res, (20040000), vol. 102, doi:doi:10.1016/j.virusres.2004.01.009, pages 3 - 9, XP002993013

DOI:   http://dx.doi.org/10.1016/j.virusres.2004.01.009
    - MOFFAT, J. et al., "A lentiviral RNAi library for human and mouse genes applied to an arrayed viral high-content screen", Cell, (20060000), vol. 124, doi:doi:10.1016/j.cell.2006.01.040, pages 1283 - 1298, XP055021141

DOI:   http://dx.doi.org/10.1016/j.cell.2006.01.040
    - XU, J. et al., "Transcriptional silencing of {gamma}-globin by BCL11A involves long-range interactions and cooperation with SOX6", Genes Dev, (20100000), vol. 24, doi:doi:10.1101/gad.1897310, pages 783 - 798, XP055255421

DOI:   http://dx.doi.org/10.1101/gad.1897310
    - WEBER, K. et al., "Lentiviral gene ontology (LeGO) vectors equipped with novel drug-selectable fluorescent proteins: new building blocks for cell marking and multi-gene analysis", Gene Ther, (20100000), vol. 17, pages 511 - 520
    - WILBER, A. et al., "Therapeutic levels of fetal hemoglobin in erythroid progeny of beta-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer", Blood, (20110000), vol. 117, doi:doi:10.1182/blood-2010-08-300723, pages 2817 - 2826, XP055216323

DOI:   http://dx.doi.org/10.1182/blood-2010-08-300723
    - LIU, P. et al., "BCL11A is essential for normal lymphoid development", Nat Immunol, (20030000), vol. 4, doi:doi:10.1038/ni925, pages 525 - 532, XP055017589

DOI:   http://dx.doi.org/10.1038/ni925
    - YU, Y. et al., "BCL11A is essential for lymphoid development and negatively regulates p53", J Exp Med, (20120000), vol. 209, pages 2467 - 2483
    - MICCIO, A. et al., "In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of beta-thalassemia", Proceedings of the National Academy of Sciences of the United States of America, (20080000), vol. 105, doi:doi:10.1073/pnas.0711666105, pages 10547 - 10552, XP055330623

DOI:   http://dx.doi.org/10.1073/pnas.0711666105
    - DEMAISON, C. et al., "High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency [correction of imunodeficiency] virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter", Hum Gene Ther, (20020000), vol. 13, pages 803 - 813
    - HANSBURY, E. N. et al., "Bone marrow transplant options and preferences in a sickle cell anemia cohort on chronic transfusions", Pediatr Blood Cancer, (20120000), vol. 58, pages 611 - 615
    - ANDREANI, M. et al., "Quantitatively different red cell/nucleated cell chimerism in patients with long-term, persistent hematopoietic mixed chimerism after bone marrow transplantation for thalassemia major or sickle cell disease", Haematologica, (20110000), vol. 96, pages 128 - 133
    - CARTIER, N. et al., "Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy", Science, (20090000), vol. 326, doi:doi:10.1126/science.1171242, pages 818 - 823, XP055268270

DOI:   http://dx.doi.org/10.1126/science.1171242
    - AIUTI, A. et al., "Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome", Science, (20130000), vol. 341, page 1233151
    - CAVAZZANA-CALVO, M. et al., "Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia", Nature, (20100000), vol. 467, doi:doi:10.1038/nature09328, pages 318 - 322, XP055258772

DOI:   http://dx.doi.org/10.1038/nature09328
    - WU, X. et al., "BCL11A controls Flt3 expression in early hematopoietic progenitors and is required for pDC development in vivo", PLoS One, (20130000), vol. 8, page e64800
    - IPPOLITO, G. C. et al., "Dendritic cell fate is determined by BCL11A", Proc Natl Acad Sci U S A, (20140000), vol. 111, pages E998 - 1006
    - ROSELLI, E. A. et al., "Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients", EMBO Mol Med, (20100000), vol. 2, doi:doi:10.1002/emmm.201000083, pages 315 - 328, XP055416239

DOI:   http://dx.doi.org/10.1002/emmm.201000083
    - ZYCHLINSKI, D. et al., "Physiological promoters reduce the genotoxic risk of integrating gene vectors", Mol Ther, (20080000), vol. 16, doi:doi:10.1038/mt.2008.5, pages 718 - 725, XP055199418

DOI:   http://dx.doi.org/10.1038/mt.2008.5
    - ZHANG, H. et al., "Single processing center models for human Dicer and bacterial RNase III", Cell, (20040000), vol. 118, pages 57 - 68
    - VERMEULEN, A. et al., "The contributions of dsRNA structure to Dicer specificity and efficiency", RNA, (20050000), vol. 11, doi:doi:10.1261/rna.7272305, pages 674 - 682, XP002403258

DOI:   http://dx.doi.org/10.1261/rna.7272305
    - CHIANG, H. R. et al., "Mammalian microRNAs: experimental evaluation of novel and previously annotated genes", Genes Dev, (20100000), vol. 24, doi:doi:10.1101/gad.1884710, pages 992 - 1009, XP055019380

DOI:   http://dx.doi.org/10.1101/gad.1884710
    - FRANK, F. et al., "Structural basis for 5'-nucleotide base-specific recognition of guide RNA by human AG02", Nature, (20100000), vol. 465, doi:doi:10.1038/nature09039, pages 818 - 822, XP055223099

DOI:   http://dx.doi.org/10.1038/nature09039
    - GIARRATANA, M. C. et al., "Proof of principle for transfusion of in vitro-generated red blood cells", Blood, (20110000), vol. 118, doi:doi:10.1182/blood-2011-06-362038, pages 5071 - 5079, XP055258936

DOI:   http://dx.doi.org/10.1182/blood-2011-06-362038
    - GRIMSON, A. et al., "Early origins and evolution of microRNAs and Piwi-interacting RNAs in animals", Nature, (20080000), vol. 455, pages 1193 - 1197
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