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Extract from the Register of European Patents

EP About this file: EP1986661

EP1986661 - GENE THERAPY FOR NIEMANN-PICK DISEASE TYPE A [Right-click to bookmark this link]
StatusNo opposition filed within time limit
Status updated on  05.07.2019
Database last updated on 08.04.2026
FormerThe patent has been granted
Status updated on  27.07.2018
FormerGrant of patent is intended
Status updated on  12.03.2018
FormerExamination is in progress
Status updated on  30.11.2016
Most recent event   Tooltip14.08.2020Lapse of the patent in a contracting state
New state(s): PL		published on 16.09.2020  [2020/38]
Applicant(s)For all designated states
Genzyme Corporation
50 Binney Street
Cambridge, MA 02142 / US
[2018/29]
Former [2008/45]For all designated states
GENZYME CORPORATION
500 Kendall Street
Cambridge, MA 02142 / US
Inventor(s)01 / PASSINI, Marco, A.
14 Nightingale Drive
Shrewsbury, MA 01545 / US
02 / ZIEGLER, Robin, J.
72 Forrest Road
Westford, MA 01886 / US
03 / DODGE, James
1 Gates Circle
Worcester, MA 01603 / US
04 / SHIHABUDDIN, Lamya
1933 Commonwealth Avenue
Brighton, MA 02135 / US
05 / CHENG, Seng, H.
50 Wall Street
Wellesley, MA 02481 / US
 [2008/45]
Representative(s)Mathys & Squire
32 London Bridge Street The Shard
London SE1 9SG / GB
[N/P]
Former [2018/35]Mathys & Squire LLP
The Shard
32 London Bridge Street
London SE1 9SG / GB
Former [2008/45]Mathys & Squire LLP
120 Holborn London
EC1N 2SQ / GB
Application number, filing date07763560.508.02.2007
[2008/45]
WO2007US03388
Priority number, dateUS20060771628P08.02.2006         Original published format: US 771628 P
US20060772360P09.02.2006         Original published format: US 772360 P
[2008/45]
Filing languageEN
Procedural languageEN
PublicationType: A2 Application without search report
No.:WO2007092563
Date:16.08.2007
Language:EN
[2007/33]
Type: A2 Application without search report 
No.:EP1986661
Date:05.11.2008
Language:EN
The application published by WIPO in one of the EPO official languages on 16.08.2007 takes the place of the publication of the European patent application.
[2008/45]
Type: B1 Patent specification 
No.:EP1986661
Date:29.08.2018
Language:EN
[2018/35]
Search report(s)International search report - published on:US22.11.2007
(Supplementary) European search report - dispatched on:EP22.03.2012
ClassificationIPC:A61K31/70, A61K48/00, C12N15/864, C12N9/16, C12N15/85
[2012/16]
CPC:
C12N9/16 (EP,US); A61K48/0083 (EP,US); A61P1/00 (EP);
A61P25/00 (EP); A61P25/28 (EP); A61P3/00 (EP);
A61P43/00 (EP); C12N15/8509 (EP,US); C12N15/86 (EP,US);
A01K2207/00 (EP,US); A01K2227/105 (EP,US); A01K2267/0306 (EP,US);
C12N2750/14143 (EP,US); C12N2840/007 (EP,US); G01N2500/00 (EP,US) (-)
Former IPC [2008/45]A61K31/70
Designated contracting statesAT,   BE,   BG,   CH,   CY,   CZ,   DE,   DK,   EE,   ES,   FI,   FR,   GB,   GR,   HU,   IE,   IS,   IT,   LI,   LT,   LU,   LV,   MC,   NL,   PL,   PT,   RO,   SE,   SI,   SK,   TR [2018/35]
Former [2008/45]AT,  BE,  BG,  CH,  CY,  CZ,  DE,  DK,  EE,  ES,  FI,  FR,  GB,  GR,  HU,  IE,  IS,  IT,  LI,  LT,  LU,  LV,  MC,  NL,  PL,  PT,  RO,  SE,  SI,  SK,  TR 
Extension statesALNot yet paid
BANot yet paid
HRNot yet paid
MKNot yet paid
RSNot yet paid
TitleGerman:GENTHERAPIE FÜR DIE NIEMANN-PICK-KRANKHEIT VOM TYP A[2008/45]
English:GENE THERAPY FOR NIEMANN-PICK DISEASE TYPE A[2008/45]
French:THERAPIE GENIQUE DESTINEE A LA MALADIE DE NIEMANN-PICK DE TYPE A[2008/45]
Entry into regional phase08.09.2008National basic fee paid 
08.09.2008Search fee paid 
08.09.2008Designation fee(s) paid 
08.09.2008Examination fee paid 
Examination procedure08.09.2008Examination requested  [2008/45]
22.10.2012Amendment by applicant (claims and/or description)
23.01.2013Despatch of a communication from the examining division (Time limit: M06)
04.06.2013Reply to a communication from the examining division
08.12.2014Despatch of a communication from the examining division (Time limit: M06)
15.07.2015Despatch of communication that the application is deemed to be withdrawn, reason: reply to the communication from the examining division not received in time
25.09.2015Reply to a communication from the examining division
25.07.2016Despatch of a communication from the examining division (Time limit: M04)
29.11.2016Reply to a communication from the examining division
26.09.2017Despatch of a communication from the examining division (Time limit: M04)
26.01.2018Reply to a communication from the examining division
13.03.2018Communication of intention to grant the patent
23.07.2018Fee for grant paid
23.07.2018Fee for publishing/printing paid
23.07.2018Receipt of the translation of the claim(s)
Divisional application(s)EP18190420.2  / EP3456331
The date of the Examining Division's first communication in respect of the earliest application for which a communication has been issued is  23.01.2013
Opposition(s)31.05.2019No opposition filed within time limit [2019/32]
Request for further processing for:The application is deemed to be withdrawn due to failure to reply to the examination report
25.09.2015Request for further processing filed
25.09.2015Full payment received (date of receipt of payment)
Request granted
08.10.2015Decision despatched
Fees paidRenewal fee
26.02.2009Renewal fee patent year 03
24.02.2010Renewal fee patent year 04
23.02.2011Renewal fee patent year 05
15.02.2012Renewal fee patent year 06
12.02.2013Renewal fee patent year 07
12.02.2014Renewal fee patent year 08
11.02.2015Renewal fee patent year 09
10.02.2016Renewal fee patent year 10
10.02.2017Renewal fee patent year 11
14.02.2018Renewal fee patent year 12
Opt-out from the exclusive  Tooltip
competence of the Unified
Patent Court		 See the Register of the Unified Patent Court for opt-out data
Responsibility for the accuracy, completeness or quality of the data displayed under the link provided lies entirely with the Unified Patent Court.
Lapses during opposition  TooltipMC29.08.2018
RO29.08.2018
IS29.12.2018
AT08.02.2019
CY08.02.2019
FI08.02.2019
IE08.02.2019
LT08.02.2019
LU08.02.2019
LV08.02.2019
PL08.02.2019
SK08.02.2019
HU09.02.2019
SE09.02.2019
SI09.02.2019
BE28.02.2019
CH28.02.2019
EE28.02.2019
LI28.02.2019
[2020/38]
Former [2020/23]MC29.08.2018
RO29.08.2018
IS29.12.2018
AT08.02.2019
CY08.02.2019
FI08.02.2019
IE08.02.2019
LT08.02.2019
LU08.02.2019
LV08.02.2019
SK08.02.2019
HU09.02.2019
SE09.02.2019
SI09.02.2019
BE28.02.2019
CH28.02.2019
EE28.02.2019
LI28.02.2019
Former [2020/12]MC29.08.2018
RO29.08.2018
IS29.12.2018
CY08.02.2019
FI08.02.2019
IE08.02.2019
LT08.02.2019
LU08.02.2019
LV08.02.2019
SK08.02.2019
HU09.02.2019
SE09.02.2019
SI09.02.2019
BE28.02.2019
CH28.02.2019
EE28.02.2019
LI28.02.2019
Former [2020/09]MC29.08.2018
RO29.08.2018
IS29.12.2018
CY08.02.2019
FI08.02.2019
IE08.02.2019
LT08.02.2019
LU08.02.2019
LV08.02.2019
SK08.02.2019
HU09.02.2019
SE09.02.2019
SI09.02.2019
CH28.02.2019
EE28.02.2019
LI28.02.2019
Former [2020/04]MC29.08.2018
RO29.08.2018
IS29.12.2018
CY08.02.2019
FI08.02.2019
LT08.02.2019
LU08.02.2019
LV08.02.2019
SK08.02.2019
HU09.02.2019
SE09.02.2019
SI09.02.2019
CH28.02.2019
EE28.02.2019
LI28.02.2019
Former [2019/52]MC29.08.2018
RO29.08.2018
IS29.12.2018
CY08.02.2019
FI08.02.2019
LT08.02.2019
LU08.02.2019
LV08.02.2019
SK08.02.2019
HU09.02.2019
SE09.02.2019
SI09.02.2019
EE28.02.2019
Former [2019/50]MC29.08.2018
RO29.08.2018
IS29.12.2018
CY08.02.2019
FI08.02.2019
LT08.02.2019
LU08.02.2019
LV08.02.2019
SK08.02.2019
HU09.02.2019
SE09.02.2019
SI09.02.2019
EE28.02.2019
Former [2019/49]MC29.08.2018
RO29.08.2018
IS29.12.2018
CY08.02.2019
FI08.02.2019
LT08.02.2019
LU08.02.2019
SK08.02.2019
SE09.02.2019
SI09.02.2019
EE28.02.2019
Former [2019/48]MC29.08.2018
IS29.12.2018
CY08.02.2019
FI08.02.2019
LT08.02.2019
LU08.02.2019
SE09.02.2019
Former [2019/47]MC29.08.2018
IS29.12.2018
FI08.02.2019
LT08.02.2019
LU08.02.2019
Former [2019/45]MC29.08.2018
IS29.12.2018
Former [2019/08]IS29.12.2018
Documents cited:Search[YD]   DODGE JAMES C ET AL: "Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, vol. 102, no. 49, December 2005 (2005-12-01), pages 17822 - 17827, XP002671093, ISSN: 0027-8424 [YD] 14-19 * abstract * * page 17823 * * page 17822 *

DOI:   http://dx.doi.org/10.1073/PNAS.0509062102
 [YD]   PASSINI M A ET AL: "AAV Vector-Mediated Correction of Brain Pathology in a Mouse Model of Niemann-Pick A Disease", MOLECULAR THERAPY, ACADEMIC PRESS, SAN DIEGO, CA, US, vol. 11, no. 5, 1 May 2005 (2005-05-01), pages 754 - 762, XP004863183, ISSN: 1525-0016, DOI: 10.1016/J.YMTHE.2005.01.011 [YD] 14-19 * abstract * * page 761 * * page 754 *

DOI:   http://dx.doi.org/10.1016/j.ymthe.2005.01.011
 [Y]   BARBON ET AL: "AAV8-Mediated Hepatic Expression of Acid Sphingomyelinase Corrects the Metabolic Defect in the Visceral Organs of a Mouse Model of Niemann-Pick Disease", MOLECULAR THERAPY, ACADEMIC PRESS, SAN DIEGO, CA, US, vol. 12, no. 3, 1 September 2005 (2005-09-01), pages 431 - 440, XP005472377, ISSN: 1525-0016, DOI: 10.1016/J.YMTHE.2005.03.011 [Y] 14-19 * abstract * * page 438 - page 439 *

DOI:   http://dx.doi.org/10.1016/j.ymthe.2005.03.011
 [A]   "PARTICULARITIES OF LYSOSOMAL STORAGE DISEASES GENE THERAPY", GENE THERAPY, MACMILLAN PRESS LTD., BASINGSTOKE, GB, vol. 3, no. 12, 1 December 1996 (1996-12-01), pages 1039 - 1041, XP001007386, ISSN: 0969-7128 [A] 1-24 * page 1040 ff. *
 [A]   JIN H-K ET AL: "Ex vivo gene therapy using bone marrow-derived cells: combined effects of intracerebral and intravenous transplantation in a mouse model of Niemann-Pick disease", MOLECULAR THERAPY, ACADEMIC PRESS, SAN DIEGO, CA, US, vol. 8, no. 6, 1 December 2003 (2003-12-01), pages 876 - 885, XP003002614, ISSN: 1525-0016 [A] 1-24 * abstract *
 [T]   PASSINI MARCO A ET AL: "Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA, NATIONAL ACADEMY OF SCIENCE, WASHINGTON, DC; US, vol. 104, no. 22, 29 May 2007 (2007-05-29), pages 9505 - 9510, XP002455719, ISSN: 0027-8424, DOI: 10.1073/PNAS.0703509104 [T] * the whole document *

DOI:   http://dx.doi.org/10.1073/pnas.0703509104
International search[Y] US2004204379  (CHENG SENG H et al.)
 [Y] US2004258666  (PASSINI MARCO A et al.)
 [Y] US6180613  (KAPLITT MICHAEL G et al.)
 [Y] US2004180440  (ZOLOTUKHIN SERGEI et al.)
 [Y] WO0160414  (GENZYME CORP et al.)
 [X]   ELLIGER ET AL., MOLECULAR THERAPY, vol. 5, no. 5, 2002, pages 617 - 626, XP008090917
 [Y]   OHASHI T. ET AL., PNAS, vol. 94, no. 4, 1997, pages 1287 - 1292, XP002122348

DOI:   http://dx.doi.org/10.1073/pnas.94.4.1287
 [A]   GONZALEZ-GOMEZ ET AL., AM. J. PATHOLOGY, vol. 153, no. 4, 1998, pages 1293 - 1300, XP008090668
 [A]   AGUDO ET AL., HUMAN GENE THERAPY, vol. 13, no. 5, 20 March 2002 (2002-03-20), pages 665 - 674, XP003017729

DOI:   http://dx.doi.org/10.1089/10430340252837251
 [A]   VOLGER ET AL., J. INHER. METAB. DIS., vol. 21, 1998, pages 575 - 586, XP008091995

DOI:   http://dx.doi.org/10.1023/A:1005423222927
by applicantUS6066626
 US5773278
 US6541218
 WO0136620
 US4603102
 US3817837
 US3850752
 US3939350
 US3996345
 US4277437
 US4275149
 US4366241
   "Inherited Disease", MCGRAW-HILL, pages: 3589 - 3610
   HORINOUCHI ET AL., NAT. GENET., vol. 10, 1995, pages 288 - 293
   SAMBROOK; FRITSCH; MANIATIS: "Molecular Cloning: A Laboratory Manual", 1989
   "Current Protocols In Molecular Biology", 1987
   "Methods In Enzymology", 1995, ACADEMIC PRESS, INC., article "Per 2: A Practical Approach"
   "ANTIBODIES, A LABORATORY MANUAL And ANIMAL CELL CULTURE", 1988
   CLARK ET AL., HUM. GENE THER., vol. 10, 1999, pages 1031 - 1039
   VELDWIJK ET AL., MOL. THER., vol. 6, 2002, pages 272 - 278
   MCLAUGHLIN ET AL., J. VIROL., vol. 62, 1988, pages 1963 - 1973
   XIAO ET AL., EXP. NEUROBIOL., vol. 144, 1997, pages 113 - 124
   FISHER ET AL., J. VIROL., vol. 70, 1996, pages 520 - 532
   DAVIDSON ET AL., NATURE REV., vol. 4, 2003, pages 353 - 364
   KAPLITT ET AL., NAT. GENET., vol. 8, 1994, pages 148 - 154
   BARTLETT ET AL., HUM. GENE THER., vol. 9, 1998, pages 1181 - 1186
   PASSINI ET AL., J. NEUROSCI., vol. 22, 2002, pages 6437 - 6446
   WATSON ET AL., METHODS MOL. MED., vol. 76, 2001, pages 383 - 403
   JEYAKUMAR ET AL., NEUROPATH. APPL. NEUROBIOL., vol. 28, 2002, pages 343 - 357
   "Metabolic and Molecular Bases of Inherited Disease", 2001, MCGRAW-HILL
   JANSON ET AL., HUM. GENE THER., vol. 13, 2002, pages 1391 - 1412
   VELCULESCU, V. ET AL., SCIENCE, vol. 270, 1995, pages 484 - 487
   CLARK, HUM. GENE THER., vol. 10, 1999, pages 1031 - 1039
The EPO accepts no responsibility for the accuracy of data originating from other authorities; in particular, it does not guarantee that it is complete, up to date or fit for specific purposes.