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Extract from the Register of European Patents

EP About this file: EP3863677

EP3863677 - POLY(Beta-AMINO ESTER) NANOPARTICLES FOR THE NON-VIRAL DELIVERY OF PLASMID DNA FOR GENE EDITING AND RETINAL GENE THERAPY [Right-click to bookmark this link]
StatusRequest for examination was made
Status updated on  16.07.2021
Database last updated on 13.09.2024
FormerThe international publication has been made
Status updated on  18.04.2020
Most recent event   Tooltip28.10.2023New entry: Renewal fee paid 
Applicant(s)For all designated states
The Johns Hopkins University
3400 North Charles Street
Baltimore, Maryland 21218 / US
[2021/33]
Inventor(s)01 / GREEN, Jordan J.
c/o The Johns Hopkins University 3400 North
Charles Street
Baltimore, Maryland 21218 / US
02 / WILSON, David
c/o The Johns Hopkins University 3400 North
Charles Street
Baltimore, Maryland 21218 / US
03 / RUI, Yuan
c/o The Johns Hopkins University 3400 North
Charles Street
Baltimore, Maryland 21218 / US
04 / ZACK, Donald J.
c/o The Johns Hopkins University 3400 North
Charles Street
Baltimore, Maryland 21218 / US
05 / MISHRA, Bibhudatta
c/o The Johns Hopkins University 3400 North
Charles Street
Baltimore, Maryland 21218 / US
06 / SRIPATHI, Srinivasa Rao
c/o The Johns Hopkins University 3400 North
Charles Street
Baltimore, Maryland 21218 / US
07 / BERLINICKE, Cynthia A.
c/o The Johns Hopkins University 3400 North
Charles Street
Baltimore, Maryland 21218 / US
 [2021/33]
Representative(s)Murgitroyd & Company
165-169 Scotland Street
Glasgow G5 8PL / GB
[N/P]
Former [2021/33]Murgitroyd & Company
Murgitroyd House
165-169 Scotland Street
Glasgow G5 8PL / GB
Application number, filing date19870203.710.10.2019
[2021/33]
WO2019US55741
Priority number, dateUS201862743883P10.10.2018         Original published format: US 201862743883 P
[2021/33]
Filing languageEN
Procedural languageEN
PublicationType: A1 Application with search report
No.:WO2020077159
Date:16.04.2020
Language:EN
[2020/16]
Type: A1 Application with search report 
No.:EP3863677
Date:18.08.2021
Language:EN
The application published by WIPO in one of the EPO official languages on 16.04.2020 takes the place of the publication of the European patent application.
[2021/33]
Search report(s)International search report - published on:US16.04.2020
(Supplementary) European search report - dispatched on:EP28.11.2022
ClassificationIPC:A61K47/34, A61K9/51, A61K47/14, A61K48/00, C08G69/26
[2021/33]
CPC:
C12N15/88 (EP,US); C08G69/26 (EP,US); C12N15/111 (EP,US);
C12N15/85 (EP); C12N15/907 (EP); A61K48/00 (EP);
A61K9/51 (EP); C12N2310/20 (EP); C12N2320/32 (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 [2021/33]
TitleGerman:POLY(BETA-AMINO-ESTER)-NANOPARTIKEL ZUR NICHT-VIRALEN ABGABE VON PLASMID-DNA FÜR DIE GENBEARBEITUNG UND DIE RETINALE GENTHERAPIE[2021/33]
English:POLY(Beta-AMINO ESTER) NANOPARTICLES FOR THE NON-VIRAL DELIVERY OF PLASMID DNA FOR GENE EDITING AND RETINAL GENE THERAPY[2021/33]
French:NANOPARTICULES DE POLY (Beta-AMINO ESTER) POUR L'ADMINISTRATION NON VIRALE D'ADN DE PLASMIDE POUR L'ÉDITION GÉNIQUE ET LA THÉRAPIE GÉNIQUE DE RÉTINE[2021/33]
Entry into regional phase13.04.2021National basic fee paid 
13.04.2021Search fee paid 
13.04.2021Designation fee(s) paid 
13.04.2021Examination fee paid 
Examination procedure13.04.2021Examination requested  [2021/33]
18.07.2023Despatch of communication that the application is deemed to be withdrawn, reason: reply to the Extended European Search Report/Written Opinion of the International Searching Authority/International Preliminary Examination Report/Supplementary international search report not received in time
27.09.2023Amendment by applicant (claims and/or description)
Request for further processing for:The application is deemed to be withdrawn due to failure to reply to the Extended European Search Report/Written Opinion of the International Searching Authority/International Preliminary Examination Report/Supplementary international search report/Supplementary European search report
27.09.2023Request for further processing filed
27.09.2023Full payment received (date of receipt of payment)
Request granted
06.10.2023Decision despatched
Fees paidRenewal fee
27.10.2021Renewal fee patent year 03
27.10.2022Renewal fee patent year 04
27.10.2023Renewal fee patent year 05
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Documents cited:Search[XI]  - SUNSHINE J C ET AL, "Poly(beta-Amino Ester)-Nanoparticle Mediated Transfection of Retinal Pigment Epithelial Cells In Vitro and In Vivo", PLOS ONE, PUBLIC LIBRARY OF SCIENCE, US, vol. 7, no. 5, doi:10.1371/JOURNAL.PONE.0037543, ISSN 1932-6203, (20120521), pages E37543 - 1, (20120521), XP002746169 [X] 1,2,5,7-15 * Title; Abstract; Fig. 1; Fig. 10 * [I] 1-5,7-15

DOI:   http://dx.doi.org/10.1371/journal.pone.0037543
 [XDI]  - DEZHONG ZHOU ET AL, "Poly(beta-Amino Ester)-Nanoparticle Mediated Transfection of Retinal Pigment Epithelial Cells In Vitro and In Vivo", SCIENCE ADVANCES, (20160601), vol. 2, no. 6, doi:10.1126/sciadv.1600102, page e1600102, XP055539263 [XD] 1,5,6,8-10,15 * Title; Abstract; Fig. 1, Fig. 2 * [I] 1-15

DOI:   http://dx.doi.org/10.1126/sciadv.1600102
 [XDI]  - ZHU DA ET AL, "Nanoparticles Based on Poly ([beta]-Amino Ester) and HPV16-Targeting CRISPR/shRNA as Potential Drugs for HPV16-Related Cervical Malignancy", MOLECULAR THERAPY, US, vol. 26, no. 10, doi:10.1016/j.ymthe.2018.07.019, ISSN 1525-0016, (20181001), pages 2443 - 2455, URL: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6171328/pdf/main.pdf, XP055907040 [XD] 1-3,5,8-12,15 * Title; Abstract * [I] 1-5,7-15

DOI:   http://dx.doi.org/10.1016/j.ymthe.2018.07.019
 [XI]  - James Cliff Kaczmarek, "Development of Polymer -Lipid Nanoparticles for Potent mRNA Delivery to the Lung", (20180930), URL: https://dspace.mit.edu/bitstream/handle/1721.1/121895/1103314257-MIT.pdf?sequence=1&isAllowed=y, (20200402), XP055682159 [X] 1-3,5,8-12 * Title; p. 82; Chapters 3, 4, 5, 6; p. 95; p. 172 * [I] 1-5,7-15
 [XP]  - RUI YUAN ET AL, "Reducible Branched Ester-Amine Quadpolymers (rBEAQs) Codelivering Plasmid DNA and RNA Oligonucleotides Enable CRISPR/Cas9 Genome Editing", APPLIED MATERIALS & INTERFACES, US, (20190222), vol. 11, no. 11, doi:10.1021/acsami.8b20206, ISSN 1944-8244, pages 10472 - 10480, XP055925498 [XP] 1-5,7-15 * the whole document *

DOI:   http://dx.doi.org/10.1021/acsami.8b20206
 [XP]  - WILSON DAVID R. ET AL, "Differentially Branched Ester Amine Quadpolymers with Amphiphilic and pH-Sensitive Properties for Efficient Plasmid DNA Delivery", MOLECULAR PHARMACEUTICS, US, vol. 16, no. 2, doi:10.1021/acs.molpharmaceut.8b00963, ISSN 1543-8384, (20190204), pages 655 - 668, URL: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7297465/pdf/nihms-1581884.pdf, XP055840765 [XP] 1-5,7-15 * the whole document *

DOI:   http://dx.doi.org/10.1021/acs.molpharmaceut.8b00963
 [XI]  - KOZIELSKI KRISTEN L. ET AL, "Bioreducible Cationic Polymer-Based Nanoparticles for Efficient and Environmentally Triggered Cytoplasmic siRNA Delivery to Primary Human Brain Cancer Cells", ACS NANO, US, (20140327), vol. 8, no. 4, doi:10.1021/nn500704t, ISSN 1936-0851, pages 3232 - 3241, XP055958014 [X] 1,5,8-10,15 * Title; Abstract; Scheme 1 * [I] 1-5,7-15

DOI:   http://dx.doi.org/10.1021/nn500704t
 [XI]  - KRISTEN L. KOZIELSKI ET AL, "A bioreducible linear poly(β-amino ester) for siRNA delivery", CHEMICAL COMMUNICATIONS, UK, (20130101), vol. 49, no. 46, doi:10.1039/c3cc40718g, ISSN 1359-7345, page 5319, XP055341016 [X] 1,5,8-10,15 * Title; Abstract; Scheme 1 * [I] 1-5,7-15

DOI:   http://dx.doi.org/10.1039/c3cc40718g
 [XI]  - CUTLAR LARA ET AL, "Highly Branched Poly([beta]-Amino Esters): Synthesis and Application in Gene Delivery", BIOMACROMOLECULES, US, vol. 16, no. 9, doi:10.1021/acs.biomac.5b00966, ISSN 1525-7797, (20150825), pages 2609 - 2617, URL: https://pubs.acs.org/doi/pdf/10.1021/acs.biomac.5b00966, XP055921486 [X] 1,5,6,8-10,15 * Title; Abstract; Fig .1 * [I] 1-6,8-15

DOI:   http://dx.doi.org/10.1021/acs.biomac.5b00966
 [XI]  - LIU SHUAI ET AL, "Biodegradable Highly Branched Poly([beta]-Amino Ester)s for Targeted Cancer Cell Gene Transfection", ACS BIOMATERIALS SCIENCE & ENGINEERING, (20161026), vol. 3, no. 7, doi:10.1021/acsbiomaterials.6b00503, ISSN 2373-9878, pages 1283 - 1286, XP055925529 [X] 1,5,6,8-10,15 * Title; Abstract; Fig. 1 * [I] 1-6,8-15

DOI:   http://dx.doi.org/10.1021/acsbiomaterials.6b00503
 [XI]  - YONGSHENG GAO ET AL, "Highly Branched Poly(?-amino esters) for Non-Viral Gene Delivery: High Transfection Efficiency and Low Toxicity Achieved by Increasing Molecular Weight", BIOMACROMOLECULES, US, (20161003), vol. 17, no. 11, doi:10.1021/acs.biomac.6b01120, ISSN 1525-7797, pages 3640 - 3647, XP055539247 [X] 1,5,6,8-10,15 * Title; Abstract; Scheme 1 * [I] 1-6,8-15

DOI:   http://dx.doi.org/10.1021/acs.biomac.6b01120
 [XI]  - ZHOU DEZHONG ET AL, "Highly branched poly([beta]-amino ester)s for skin gene therapy", JOURNAL OF CONTROLLED RELEASE, ELSEVIER, AMSTERDAM, NL, (20160608), vol. 244, doi:10.1016/J.JCONREL.2016.06.014, ISSN 0168-3659, pages 336 - 346, XP029850289 [X] 1,5,6,8-10,15 * Title; Abstract; Fig. 2 * [I] 1-6,8-15

DOI:   http://dx.doi.org/10.1016/j.jconrel.2016.06.014
 [XI]  - SHUAI LIU ET AL, "Alkylated branched poly(beta-amino esters) demonstrate strong DNA encapsulation, high nanoparticle stability and robust gene transfection efficacy", JOURNAL OF MATERIALS CHEMISTRY. B, GB, (20170101), vol. 5, no. 27, doi:10.1039/C7TB00996H, ISSN 2050-750X, pages 5307 - 5310, XP055539258 [X] 1,5,6,8-10,15 * Title; Abstract; Fig .1 * [I] 1-15

DOI:   http://dx.doi.org/10.1039/C7TB00996H
    [ ] - LIU SHUAI ET AL, "Supplementary information.- Alkylated branched poly([beta]-amino esters) demonstrate strong DNA encapsulation, high nanoparticle stability and robust gene transfection efficacy", JOURNAL OF MATERIALS CHEMISTRY. B, GB, (20170101), vol. 5, no. 27, doi:10.1039/C7TB00996H, ISSN 2050-750X, pages 5307 - 5310, XP055971350 [ ] * Supporting information sections 2.2, 2.5, Fig. S7; p. 8 *

DOI:   http://dx.doi.org/10.1039/C7TB00996H
 [XI]  - JIAN-YUAN HUANG ET AL, "Tailoring highly branched poly(β-amino ester)s: a synthetic platform for epidermal gene therapy", CHEMICAL COMMUNICATIONS, (20150414), vol. 51, no. 40, doi:10.1039/C5CC02193F, ISSN 1359-7345, pages 8473 - 8476, XP055211464 [X] 1,5,6,8-10,15 * Title; abstract; figure 1; supplementary information section 2.2 * [I] 1-6,8-15

DOI:   http://dx.doi.org/10.1039/C5CC02193F
    [ ] - HUANG JIAN-YUAN ET AL, "Supplementary information.- Tailoring highly branched poly([beta]-amino ester)s: a synthetic platform for epidermal gene therapy", CHEMICAL COMMUNICATIONS, UK, (20150101), vol. 51, no. 40, doi:10.1039/C5CC02193F, ISSN 1359-7345, pages 8473 - 8476, XP055971365 [ ] * Supplementary information section 2.2 *

DOI:   http://dx.doi.org/10.1039/C5CC02193F
International search[A]US2014199306  (GHOSH JOY [US], et al) [A] 1-8, 14-16, 21-44 * entire document *;
 [A]US2017216363  (QUINONES-HINOJOSA ALFREDO [US], et al) [A] 1-8, 14-16, 21-44 * entire document *;
 [A]WO2018002719  (CRISPR THERAPEUTICS AG [CH]) [A] 1-8, 14-16, 21-44 * entire document *;
 [PA]WO2019075056  (UNIV JOHNS HOPKINS [US]) [PA] 1-8, 14-16, 21-44* entire document *
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