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1. WO2020115106 - ANTISENSE OLIGONUCLEOTIDES RESCUE ABERRANT SPLICING OF ABCA4


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INTERNATIONAL SEARCH REPORT (ISR)
Part 1:  1  2  3  4  5  6          Part 2:  A  B  C  D  E 
International application No. Applicant's or agent's file reference
PCT/EP2019/083618 P6078940PCT
International filing date (day/month/year) (Earliest) Priority Date (day/month/year)
04 December 2019 04 December 2018
Applicant
STICHTING KATHOLIEKE UNIVERSITEIT
FOR FURTHER ACTION: See Form PCT/ISA/220 as well as, where applicable, item 5 below.
This international search report has been prepared by this International Searching Authority and is transmitted to the applicant according to Article 18. A copy is being transmitted to the International Bureau.
It is also accompanied by a copy of each prior art document cited in this report.
1. Basis of the report
a. With regard to the language, the international search was carried out on the basis of:
the international application in the language in which it was filed.
a translation of the international application into                                          which is the language of a translation furnished for the purposes of international search (Rules 12.3(a) and 23.1(b)).
b.
This international search report has been established taking into account the rectification of an obvious mistake authorized by or notified to this Authority under Rule 91 (Rule 43.6bis(a)).
c.
1. With regard to any nucleotide and/or amino acid sequence disclosed in the international application, the international search was carried out on the basis of a sequence listing:
a. forming part of the international application as filed:
in the form of an Annex C/ST.25 text file.
on paper or in the form of an image file.
b. furnished together with the international application under PCT Rule 13ter.1(a) for the purposes of international search only in the form of an Annex C/ST.25 text file.
c. furnished subsequent to the international filing date for the purposes of international search only:
in the form of an Annex C/ST.25 text file (Rule 13ter.1(a)).
on paper or in the form of an image file (Rule 13ter.1(b) and Administrative Instructions, Section 713).
2.
In addition, in the case that more than one version or copy of a sequence listing has been filed or furnished, the required statements that the information in the subsequent or additional copies is identical to that forming part of the application as filed or does not go beyond the application as filed, as appropriate, were furnished.
3.
Additional comments:
2. Certain claims were found unsearchable
3. Unity of invention is lacking
4. Title of the invention
The text is approved as submitted by the applicant.
The text has been established by this Authority to read as follows:
5. Abstract
The text is approved as submitted by the applicant.
The text has been established, according to Rule 38.2, by this Authority as it appears in Box No. IV. The applicant may, within one month from the date of mailing of this international search report, submit comments to this Authority.
6. Drawings
a.
The figure of the drawings to be published with the abstract is Figure No. ____________
as suggested by the applicant.
as selected by this Authority, because the applicant failed to suggest a figure.
as selected by this Authority, because this figure better characterizes the invention.
b.
none of the figures is to be published with the abstract.

A. CLASSIFICATION OF SUBJECT MATTER

     C12N 15/113 (2010.01)i; A61K 31/7088 (2006.01)i
According to International Patent Classification (IPC) or to both national classification and IPC

B. FIELDS SEARCHED

Minimum documentation searched (classification system followed by classification symbols):
     C12N; A61K
Documentation searched other than minimum documentation to the extent that such documents are included in the fields searched:
Electronic data base consulted during the international search (name of data base and, where practicable, search terms used):
EPO-Internal, WPI Data

C. DOCUMENTS CONSIDERED TO BE RELEVANT

Category* Citation of document, with indication, where appropriate, of the relevant passages Relevant to claim No.
(1)
X
WO 2018109011 A1 (STICHTING KATHOLIEKE UNIV [NL]) 21 June 2018 (2018-06-21)
1-3,5-14
page 3, line 4 - line 32; sequences 85, 88, 91
page 33 - page 35; claims 7-19; sequences 80-84, 86, 87, 89, 80
(2)
X
WO 2016201272 A1 (KING ABDULAZIZ CITY SCI & TECH [US]) 15 December 2016 (2016-12-15)
4
sequence 253350
(3)
A
RICCARDO SANGERMANO ET AL, "ABCA4 midigenes reveal the full splice spectrum of all reported noncanonical splice site variants in Stargardt disease", GENOME RESEARCH,Vol. 28, No. 1, 21 November 2017 (2017-11-21), page 100-110,
XP055538937
1-14
the whole document
(4)
A
ALBERT S ET AL, "ABCA4 mRNA analysis of iPSC-derived photoreceptor cells of Stargardt patients reveals protein-truncating non-canonical and deep-intronic splice site variants", HUMAN GENE THERAPY, LIEBERT, US,Vol. 27, No. 11, 01 November 2016 (2016-11-01), page A119,
XP002770882
1-14
the whole document
(5)
A
SANGERMANO RICCARDO ET AL, "Photoreceptor Progenitor mRNA Analysis Reveals Exon Skipping Resulting from theABCA4c.5461-10T->C Mutation in Stargardt Disease", OPHTHALMOLOGY,Vol. 123, No. 6, 05 June 2016 (2016-06-05), page 1375-1385,
XP029552329
1-14
the whole document
(6)
A
JANA ZERNANT ET AL, "Analysis of the ABCA4 Gene by Next-Generation Sequencing", INVESTIGATIVE OPTHALMOLOGY & VISUAL SCIENCE,Vol. 52, No. 11, 31 October 2011 (2011-10-31), page 8479,
XP055538925
1-14
the whole document
(7)
A
ALBERT SILVIA ET AL, "Identification and Rescue of Splice Defects Caused by Two Neighboring Deep-IntronicABCA4Mutations Underlying Stargardt Disease", AMERICAN JOURNAL OF HUMAN GENETICS, AMERICAN SOCIETY OF HUMAN GENETICS, CHICAGO, IL, US,Vol. 102, No. 4, 08 March 2018 (2018-03-08), page 517-527,
XP085371352
1-14
the whole document
(8)
A
ARTUR V. CIDECIYAN ET AL, "ABCA4 disease progression and a proposed strategy for gene therapy", HUMAN MOLECULAR GENETICS,Vol. 18, No. 5, 12 December 2008 (2008-12-12), page 931-941,
XP055378779
1-14
the whole document
(9)
A
JANA ZERNANT ET AL, "Extremely hypomorphic and severe deep intronic variants in the ABCA4 locus result in varying Stargardt disease phenotypes", MOLECULAR CASE STUDIES,Vol. 4, No. 4, 30 May 2018 (2018-05-30), page a002733,
XP055538929
1-14
the whole document
(10)
A
INGVILD AUKRUST ET AL, "The intronic ABCA4 c.5461-10T>C variant, frequently seen in patients with Stargardt disease, causes splice defects and reduced ABCA4 protein level", ACTA OPHTHALMOLOGICA: THE OPHTHALMOLOGICAL JOURNAL OF THE NORDIC COUNTRIES,Vol. 95, No. 3, 24 October 2016 (2016-10-24), page 240-246,
XP055378943
1-14
the whole document
*
Special categories of cited documents:
"A"
document defining the general state of the art which is not considered to be of particular relevance
"D"
document cited by the applicant in the international application
"E"
earlier application or patent but published on or after the international filing date
"L"
document which may throw doubts on priority claim(s) or which is cited to establish the publication date of another citation or other special reason (as specified)
"O"
document referring to an oral disclosure, use, exhibition or other means
"P"
document published prior to the international filing date but later than the priority date claimed
"T"
later document published after the international filing date or priority date and not in conflict with the application but cited to understand the principle or theory underlying the invention
"X"
document of particular relevance; the claimed invention cannot be considered novel or cannot be considered to involve an inventive step when the document is taken alone
"Y"
document of particular relevance; the claimed invention cannot be considered to involve an inventive step when the document is combined with one or more other such documents, such combination being obvious to a person skilled in the art
"&"
document member of the same patent family

D. INFORMATION ON PATENT FAMILY MEMBERS

Patent document cited in search report Publication date
(day/month/year)
Patent family member(s) Publication date
(day/month/year)
WO 2018109011 A1
21 June 2018
EP 3555291 A1
JP 2020500524 A
US 2019367919 A1
WO 2018109011 A1
23 October 2019
16 January 2020
05 December 2019
21 June 2018
WO 2016201272 A1
15 December 2016
NONE
Name and mailing address of the ISA/:
European Patent Office
P.B. 5818, Patentlaan 2, 2280 HV Rijswijk,
Netherlands
Telephone No. (+31-70)340-2040
Facsimile No. (+31-70)340-3016
Date of the actual completion of the international search:
10 March 2020
Date of mailing of the international search report:
18 March 2020
Authorized officer:
Franz, Cerstin
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