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Extract from the Register of European Patents

EP About this file: EP3980543

EP3980543 - COMPOSITIONS AND METHODS FOR THE TREATMENT OF DBA USING GATA1 GENE THERAPY [Right-click to bookmark this link]
StatusRequest for examination was made
Status updated on  11.03.2022
Database last updated on 03.10.2024
FormerThe international publication has been made
Status updated on  18.12.2020
Most recent event   Tooltip28.06.2024New entry: Renewal fee paid 
Applicant(s)For all designated states
The Children's Medical Center Corporation
55 Shattuck Street
Boston, Massachusetts 02115 / US
[2022/15]
Inventor(s)01 / SANKARAN, Vijay G.
101 Sycamore Street
Roslindale, Massachusetts 02131 / US
02 / VOIT, Richard A.
39 Quirk Street
Watertown, Massachusetts 02472 / US
03 / LUDWIG, Leif S.
884-888 Massachusetts Avenue Apt. 310
Cambridge, Massachusetts 02139 / US
 [2022/15]
Representative(s)Griffin, Philippa Jane, et al
Mathys & Squire The Shard
32 London Bridge Street
London SE1 9SG / GB
[2022/15]
Application number, filing date20821689.508.06.2020
[2022/15]
WO2020US36600
Priority number, dateUS201962859369P10.06.2019         Original published format: US 201962859369 P
[2022/15]
Filing languageEN
Procedural languageEN
PublicationType: A1 Application with search report
No.:WO2020251887
Date:17.12.2020
Language:EN
[2020/51]
Type: A1 Application with search report 
No.:EP3980543
Date:13.04.2022
Language:EN
The application published by WIPO in one of the EPO official languages on 17.12.2020 takes the place of the publication of the European patent application.
[2022/15]
Search report(s)International search report - published on:US17.12.2020
(Supplementary) European search report - dispatched on:EP11.10.2023
ClassificationIPC:C12N15/52, A61P7/00, A61K35/12, A61P7/06, C12N15/63
[2022/15]
CPC:
A61K31/7105 (EP); A61K48/0066 (US); A61K38/1709 (US);
A61K48/005 (EP); A61K48/0058 (EP); A61P7/06 (EP,US);
C07K14/4702 (EP); C12N15/86 (EP); C12N2310/141 (EP);
C12N2740/16043 (EP); C12N2830/008 (EP); C12N2830/48 (EP);
C12N2840/203 (EP) (-)
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 [2022/15]
TitleGerman:ZUSAMMENSETZUNGEN UND VERFAHREN ZUR BEHANDLUNG VON DBA MITTELS GATA1-GENTHERAPIE[2022/15]
English:COMPOSITIONS AND METHODS FOR THE TREATMENT OF DBA USING GATA1 GENE THERAPY[2022/15]
French:COMPOSITIONS ET MÉTHODES POUR LE TRAITEMENT DE LA DBA AU MOYEN D'UNE THÉRAPIE GÉNIQUE AVEC GATA1[2022/15]
Entry into regional phase13.12.2021National basic fee paid 
13.12.2021Search fee paid 
13.12.2021Designation fee(s) paid 
13.12.2021Examination fee paid 
Examination procedure13.12.2021Examination requested  [2022/15]
09.04.2024Amendment by applicant (claims and/or description)
Fees paidRenewal fee
27.06.2022Renewal fee patent year 03
27.06.2023Renewal fee patent year 04
27.06.2024Renewal fee patent year 05
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Documents cited:Search[XY]  - WHYATT D ET AL, "The level of the tissue-specific factor GATA-1 affects the cell-cycle machinery", GENES AND FUNCTION, GB, vol. 1, no. 1, doi:10.1046/j.1365-4624.1997.00003.x, ISSN 1360-7413, (19970201), pages 11 - 24, URL: https://onlinelibrary.wiley.com/doi/pdf/10.1046/j.1365-4624.1997.00003.x, XP093058004 [X] 1,2,5,6,9,12,14 * abstract, results, discussion, experimental procedures, figure 1; * [Y] 1-15

DOI:   http://dx.doi.org/10.1046/j.1365-4624.1997.00003.x
 [XY]  - LI Q ET AL, "Binary transgenic mouse model for studying the trans control of globin gene switching: Evidence that GATA-1 is an in vivo repressor of human [epsilon]?gene?expression", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, (19970318), vol. 94, no. 6, doi:10.1073/pnas.94.6.2444, ISSN 0027-8424, pages 2444 - 2448, XP093058003 [X] 1,2,5,6,9,10,12,14 * abstract, materials and methods, results, figure 1; * [Y] 1-15

DOI:   http://dx.doi.org/10.1073/pnas.94.6.2444
 [Y]  - ROMANO O ET AL, "Transcriptional, epigenetic and retroviral signatures identify regulatory regions involved in hematopoietic lineage commitment", SCIENTIFIC REPORTS, vol. 6, no. 1, doi:10.1038/srep24724, (20160420), URL: https://www.nature.com/articles/srep24724, XP093057703 [Y] 1-15 * abstract, results, discussion, methods, supplementary information; *

DOI:   http://dx.doi.org/10.1038/srep24724
 [Y]  - Romano O ET AL, "SUPPLEMENTARY INFORMATION Transcriptional, epigenetic and retroviral signatures identify regulatory regions involved in hematopoietic lineage commitment", (20160420), URL: https://static-content.springer.com/esm/art%3A10.1038%2Fsrep24724/MediaObjects/41598_2016_BFsrep24724_MOESM25_ESM.pdf, (20230626), XP093057706 [Y] 1-15 * the whole document *
 [Y]  - TOSCANO M G ET AL, "Physiological and tissue-specific vectors for treatment of inherited diseases", GENE THERAPY, (20110201), vol. 18, no. 2, doi:10.1038/gt.2010.138, ISSN 0969-7128, pages 117 - 127, XP055088392 [Y] 1-15 * the whole document *

DOI:   http://dx.doi.org/10.1038/gt.2010.138
 [Y]  - SHIMIZU R ET AL, "Gene expression regulation and domain function of hematopoietic GATA factors", SEMINARS IN CELL AND DEVELOPMENTAL BIOLOGY, ACADEMIC PRESS, GB, (20050201), vol. 16, no. 1, doi:10.1016/J.SEMCDB.2004.11.001, ISSN 1084-9521, pages 129 - 136, XP004711663 [Y] 1-15 * the whole document *

DOI:   http://dx.doi.org/10.1016/j.semcdb.2004.11.001
 [A]  - DEBNATH S ET AL, "Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia", MOLECULAR THERAPY, US, vol. 25, no. 8, doi:10.1016/j.ymthe.2017.04.002, ISSN 1525-0016, (20170801), pages 1805 - 1814, URL: https://www.cell.com/molecular-therapy-family/molecular-therapy/pdfExtended/S1525-0016(17)30161-2, XP055879911 [A] 1-15 * abstract, results, discussion, materials and methods; *

DOI:   http://dx.doi.org/10.1016/j.ymthe.2017.04.002
International search[Y]US2018223360  (HATCHWELL ELI [GB], et al) [Y] 3* Abstract; Claim 16; para [0268]; para [0414]; SEQ ID NO:167 *;
 [Y]  - LUDWIG et al., "Altered translation of GATA1 in Diamond-Blackfan anemia", Nat Med., (20140700), vol. 20, no. 7, doi:10.1038/nm.3557, pages 748 - 753, XP055586991 [Y] 1-4 * . Abstract; p2, Fig.1 legend; p7, col 2, para 2 *

DOI:   http://dx.doi.org/10.1038/nm.3557
 [Y]  - SURINYA et al., "Identification and Characterization of a Conserved Erythroid-specific Enhancer Located in Intron 8 of the Human 5-Aminolevulinate Synthase 2 Gene", J. Biol. Chem., (19980703), vol. 273, no. 27, doi:10.1074/jbc.273.27.16798, pages 16798 - 16809, XP002126819 [Y] 1-4 * . Abstract; p16799, col 2, para 2 *

DOI:   http://dx.doi.org/10.1074/jbc.273.27.16798
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