Extract from the Register of European Patents

About this file: EP3469080

EP3469080 - OPTIMIZED CLN1 GENES AND EXPRESSION CASSETTES AND THEIR USE [Right-click to bookmark this link]
StatusRequest for examination was made
Status updated on  15.03.2019
Database last updated on 14.09.2019
FormerThe international publication has been made
Status updated on  28.12.2017
Most recent event   Tooltip23.08.2019Change - inventorpublished on 25.09.2019 [2019/39]
Applicant(s)For all designated states
The University of North Carolina at Chapel Hill
109 Church Street
Chapel Hill, North Carolina 27516 / US
[2019/16]
Inventor(s)01 / GRAY, Steven
518 Timber Lake Drive
Southlake Texas 76092 / US
[2019/39]
Former [2019/16]01 / GRAY, Steven
1403 Birkdale Circle
Mebane North Carolina 27302 / US
Representative(s)Kransell & Wennborg KB
P.O. Box 27834
115 93 Stockholm / SE
[2019/16]
Application number, filing date17813890.513.06.2017
[2019/16]
WO2017US37118
Priority number, dateUS201662349411P13.06.2016         Original published format: US 201662349411 P
[2019/16]
Filing languageEN
Procedural languageEN
PublicationType: A1  Application with search report
No.:WO2017218450
Date:21.12.2017
Language:EN
[2017/51]
Type: A1 Application with search report 
No.:EP3469080
Date:17.04.2019
Language:EN
The application has been published by WIPO in one of the EPO official languages on 21.12.2017
[2019/16]
Search report(s)International search report - published on:RU21.12.2017
ClassificationInternational:C12N15/11, C12N15/55, C12N15/63, C12N15/85, A61K48/00, A61K31/713, C12N15/86, C12N15/33
[2019/16]
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 [2019/16]
TitleGerman:OPTIMIERTE CLN1-GENE UND EXPRESSIONSKASSETTEN SOWIE DEREN VERWENDUNG[2019/16]
English:OPTIMIZED CLN1 GENES AND EXPRESSION CASSETTES AND THEIR USE[2019/16]
French:GÈNES DE CLN1 ET CASSETTES D'EXPRESSION OPTIMISÉS ET LEUR UTILISATION[2019/16]
Entry into regional phase16.11.2018National basic fee paid 
16.11.2018Search fee paid 
16.11.2018Designation fee(s) paid 
16.11.2018Examination fee paid 
Examination proceduredeletedDate on which the examining division has become responsible
16.11.2018Examination requested  [2019/16]
01.08.2019Amendment by applicant (claims and/or description)
Fees paidRenewal fee
13.06.2019Renewal fee patent year 03
Cited inInternational search[XYA]  - GRIFFEY M. et al., "Adeno-associated virus 2-mediated gene therapy decreases autofluorescent storage material and increases brain mass in a murine model of infantile neuronal ceroid lipofuscinosis", Neurobiology of Disease, (20040700), vol. 16, no. 2, pages 360 - 369, XP055449785 [X] 3, 5, 6, 24-26, 31, 33-40 * , especially abstract, p.360-362, 366-368, Figure 1 * [Y] 1, 4, 27-30, 32, 41 [A] 2

DOI:   http://dx.doi.org/10.1016/j.nbd.2004.03.005
 [Y]  - VANCE M. et al., "AAV gene therapy for MPS1-associated corneal blindness", Scientific reports, (20160222), vol. 6, no. 22131, pages 1 - 10, XP055449788 [Y] 1, 4 * , especially abstract, p.2 *

DOI:   http://dx.doi.org/10.1038/srep22131
 [Y]  - MICHELFELDER S. et al., "Peptide ligands incorporated into the threefold spike capsid domain to re-direct gene transduction of AAV8 and AAV9 in vivo", PloS one, (20110800), vol. 6, no. 8, pages 1 - 11, XP055147101 [Y] 27-30 * , p.1-11, especially abstract, p.2, Figures 1c, 3a, 4a *

DOI:   http://dx.doi.org/10.1371/journal.pone.0023101
 [Y]  - SEN D. et al., "Targeted modifications in adeno-associated virus serotype 8 capsid improves its hepatic gene transfer efficiency in vivo", Human Gene Therapy Methods, (20130400), vol. 24, no. 2, pages 104 - 116, XP055086037 [Y] 30, 41 * , especially abstract, Table 4, pp.106, 109 *

DOI:   http://dx.doi.org/10.1089/hgtb.2012.195
 [Y]  - BUNING H. et al., "Recent developments in adeno-associated virus vector technology", The Journal of Gene Medicine, (20080700), vol. 10, no. 7, pages 717 - 733, XP055449844 [Y] 32 * , especially Figure 2, p.728 *

DOI:   http://dx.doi.org/10.1002/jgm.1205
 [A]  - BOSCH A. et al., "Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors", Molecular Therapy, (20000100), vol. 1, no. 1, pages 63 - 70, XP008123629 [A] 1-6, 24-41 * , especially abstract *

DOI:   http://dx.doi.org/10.1006/MTHE.1999.0005
 [A]  - KOLLMANN K. et al., "Cell biology and function of neuronal ceroid lipofuscinosis- related proteins", Biochimica et Biophysica Acta, (20131100), vol. 1832, no. 11, pages 1866 - 1881, XP028691301 [A] 1-6, 24-41 * , especially abstract, p.1867 *

DOI:   http://dx.doi.org/10.1016/j.bbadis.2013.01.019
 [A]  - MAURO V. et al., "A critical analysis of codon optimization in human therapeutics", Trends in molecular medicine, (20141100), vol. 20, no. 11, pages 604 - 613, XP055391316 [A] 1-6, 24-41 * , especially abstract *

DOI:   http://dx.doi.org/10.1016/j.molmed.2014.09.003