CN118910166A - 调节性多核苷酸 - Google Patents
调节性多核苷酸 Download PDFInfo
- Publication number
- CN118910166A CN118910166A CN202411018854.2A CN202411018854A CN118910166A CN 118910166 A CN118910166 A CN 118910166A CN 202411018854 A CN202411018854 A CN 202411018854A CN 118910166 A CN118910166 A CN 118910166A
- Authority
- CN
- China
- Prior art keywords
- seq
- aav
- sequence
- region
- viral genome
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
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Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/20—Vector systems having a special element relevant for transcription transcription of more than one cistron
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Immunology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurosurgery (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (8)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762501787P | 2017-05-05 | 2017-05-05 | |
| US62/501,787 | 2017-05-05 | ||
| US201762507923P | 2017-05-18 | 2017-05-18 | |
| US62/507,923 | 2017-05-18 | ||
| US201762520093P | 2017-06-15 | 2017-06-15 | |
| US62/520,093 | 2017-06-15 | ||
| CN201880042534.7A CN110914427B (zh) | 2017-05-05 | 2018-05-04 | 调节性多核苷酸 |
| PCT/US2018/031108 WO2018204797A1 (en) | 2017-05-05 | 2018-05-04 | Modulatory polynucleotides |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201880042534.7A Division CN110914427B (zh) | 2017-05-05 | 2018-05-04 | 调节性多核苷酸 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN118910166A true CN118910166A (zh) | 2024-11-08 |
Family
ID=64016722
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202411018854.2A Pending CN118910166A (zh) | 2017-05-05 | 2018-05-04 | 调节性多核苷酸 |
| CN201880042534.7A Active CN110914427B (zh) | 2017-05-05 | 2018-05-04 | 调节性多核苷酸 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201880042534.7A Active CN110914427B (zh) | 2017-05-05 | 2018-05-04 | 调节性多核苷酸 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US20200270635A1 (enExample) |
| EP (1) | EP3619310A4 (enExample) |
| JP (2) | JP2020518266A (enExample) |
| CN (2) | CN118910166A (enExample) |
| AU (1) | AU2018260998B2 (enExample) |
| CA (1) | CA3061365A1 (enExample) |
| SG (1) | SG11201909777YA (enExample) |
| TW (1) | TWI897844B (enExample) |
| WO (1) | WO2018204797A1 (enExample) |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9585971B2 (en) | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| KR102584655B1 (ko) | 2014-11-14 | 2023-10-06 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| AU2016366549B2 (en) | 2015-12-11 | 2022-11-10 | California Institute Of Technology | Targeting peptides for directing adeno-associated viruses (AAVs) |
| RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
| AU2017268382B2 (en) | 2016-05-18 | 2023-09-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| EP3510161A4 (en) | 2016-08-23 | 2020-04-22 | Akouos, Inc. | COMPOSITIONS AND METHODS FOR TREATING NON-AGE-ASSOCIATED HEARING DEFICIENCY IN A HUMAN SUBJECT |
| AU2018261790B2 (en) | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| WO2019241486A1 (en) | 2018-06-13 | 2019-12-19 | Voyager Therapeutics, Inc. | Engineered 5' untranslated regions (5' utr) for aav production |
| AU2019299861A1 (en) * | 2018-07-02 | 2021-01-14 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
| WO2020023612A1 (en) | 2018-07-24 | 2020-01-30 | Voyager Therapeutics, Inc. | Systems and methods for producing gene therapy formulations |
| EP3861113A1 (en) | 2018-10-04 | 2021-08-11 | Voyager Therapeutics, Inc. | Methods for measuring the titer and potency of viral vector particles |
| EP3861107A1 (en) | 2018-10-05 | 2021-08-11 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding aav production proteins |
| JP2022505106A (ja) | 2018-10-15 | 2022-01-14 | ボイジャー セラピューティクス インコーポレイテッド | バキュロウイルス／Ｓｆ９システムにおけるｒＡＡＶの大規模産生のための発現ベクター |
| CN113631225A (zh) | 2019-01-18 | 2021-11-09 | 沃雅戈治疗公司 | 用于生产aav颗粒的方法和系统 |
| US20220243225A1 (en) | 2019-04-29 | 2022-08-04 | Voyager Therapeutics, Inc. | SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS |
| WO2020223280A1 (en) * | 2019-04-29 | 2020-11-05 | Voyager Therapeutics, Inc. | Aav variants with enhanced tropism |
| WO2021030125A1 (en) | 2019-08-09 | 2021-02-18 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| EP4022070A1 (en) | 2019-08-26 | 2022-07-06 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| WO2021168362A1 (en) | 2020-02-21 | 2021-08-26 | Akouos, Inc. | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| CN111450083A (zh) * | 2020-04-28 | 2020-07-28 | 天津大学 | 一种肝癌靶向治疗纳米颗粒的合成方法 |
| GB202010981D0 (en) * | 2020-07-16 | 2020-09-02 | Ucl Business Ltd | Gene therapy for neuromuscular and neuromotor disorders |
| WO2022023284A1 (en) | 2020-07-27 | 2022-02-03 | Anjarium Biosciences Ag | Compositions of dna molecules, methods of making therefor, and methods of use thereof |
| US20230295656A1 (en) | 2020-08-06 | 2023-09-21 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| WO2022072324A1 (en) * | 2020-09-29 | 2022-04-07 | NeuExcell Therapeutics Inc. | Isl1 and lhx3 vector |
| US20240269322A1 (en) * | 2020-10-21 | 2024-08-15 | Chan Zuckerberg Biohub, Inc. | Adeno-associated virus virions and methods of use thereof |
| WO2022187473A2 (en) | 2021-03-03 | 2022-09-09 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| US20240141378A1 (en) | 2021-03-03 | 2024-05-02 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| MX2024004217A (es) | 2021-10-08 | 2024-06-26 | Dyno Therapeutics Inc | Variantes de cápside y métodos de uso de estas. |
| WO2024054983A1 (en) | 2022-09-08 | 2024-03-14 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| CA3272944A1 (en) | 2022-12-29 | 2024-07-04 | Voyager Therapeutics Inc | MAPPING COMPOSITIONS AND REGULATION METHODS |
| WO2024226790A1 (en) * | 2023-04-26 | 2024-10-31 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| TW202449167A (zh) | 2023-04-26 | 2024-12-16 | 美商航海家醫療公司 | 用於治療肌肉萎縮性脊髓側索硬化症之組成物及方法 |
| WO2025122644A1 (en) | 2023-12-05 | 2025-06-12 | Voyager Therapeutics, Inc. | Compositions and methods for regulating mapt |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0755454B1 (en) * | 1994-04-13 | 2008-02-13 | The Rockefeller University | Aav-mediated delivery of dna to cells of the nervous system |
| US20080318210A1 (en) * | 2003-08-27 | 2008-12-25 | Rosetta Genomics | Bioinformatically detectable group of novel regulatory viral and viral associated oligonucleotides and uses thereof |
| WO2005017127A2 (en) * | 2003-02-21 | 2005-02-24 | The Penn State Research Foundation | Rna interference compositions and methods |
| AU2004239114B2 (en) * | 2003-05-14 | 2008-03-13 | Japan Science And Technology Agency | Inhibition of the expression of huntingtin gene |
| US20050064489A1 (en) * | 2003-09-24 | 2005-03-24 | Zhang Fang Liang | Engineered U6 and H1 promoters |
| AU2005307737C1 (en) * | 2004-11-18 | 2013-08-29 | The Board Of Trustees Of The University Of Illinois | Multicistronic siRNA constructs to inhibit tumors |
| WO2008134646A2 (en) * | 2007-04-26 | 2008-11-06 | University Of Iowa Research Foundation | Rna interference suppression of neurodegenerative diseases and methods of use thereof |
| WO2010078536A1 (en) * | 2009-01-05 | 2010-07-08 | Rxi Pharmaceuticals Corporation | Inhibition of pcsk9 through rnai |
| WO2010140862A2 (ko) * | 2009-06-05 | 2010-12-09 | Seol Dai-Wu | 단일 또는 멀티 표적 유전자를 억제하는 멀티-시스트로닉 ｓｈＲＮＡ 발현 카세트 |
| DK3119797T3 (da) * | 2014-03-18 | 2021-03-15 | Univ Massachusetts | Raav-baserede sammensætninger og fremgangsmåder til behandling af amyotrofisk lateralsklerose |
| MX2017006216A (es) * | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| KR102584655B1 (ko) * | 2014-11-14 | 2023-10-06 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| US20190224339A1 (en) * | 2016-04-29 | 2019-07-25 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
-
2018
- 2018-05-04 EP EP18795140.5A patent/EP3619310A4/en active Pending
- 2018-05-04 JP JP2019560388A patent/JP2020518266A/ja active Pending
- 2018-05-04 US US16/611,046 patent/US20200270635A1/en not_active Abandoned
- 2018-05-04 CN CN202411018854.2A patent/CN118910166A/zh active Pending
- 2018-05-04 WO PCT/US2018/031108 patent/WO2018204797A1/en not_active Ceased
- 2018-05-04 TW TW107115348A patent/TWI897844B/zh active
- 2018-05-04 CN CN201880042534.7A patent/CN110914427B/zh active Active
- 2018-05-04 SG SG11201909777Y patent/SG11201909777YA/en unknown
- 2018-05-04 CA CA3061365A patent/CA3061365A1/en active Pending
- 2018-05-04 AU AU2018260998A patent/AU2018260998B2/en active Active
-
2021
- 2021-12-23 US US17/561,252 patent/US20220333131A1/en active Pending
-
2023
- 2023-05-01 JP JP2023075746A patent/JP7768928B2/ja active Active
Also Published As
| Publication number | Publication date |
|---|---|
| CN110914427A (zh) | 2020-03-24 |
| AU2018260998A1 (en) | 2019-11-28 |
| JP7768928B2 (ja) | 2025-11-12 |
| AU2018260998A2 (en) | 2020-01-16 |
| EP3619310A1 (en) | 2020-03-11 |
| CA3061365A1 (en) | 2018-11-08 |
| TWI897844B (zh) | 2025-09-21 |
| AU2018260998B2 (en) | 2025-09-11 |
| TW201905200A (zh) | 2019-02-01 |
| EP3619310A4 (en) | 2021-01-27 |
| US20200270635A1 (en) | 2020-08-27 |
| SG11201909777YA (en) | 2019-11-28 |
| JP2020518266A (ja) | 2020-06-25 |
| US20220333131A1 (en) | 2022-10-20 |
| WO2018204797A1 (en) | 2018-11-08 |
| JP2023087019A (ja) | 2023-06-22 |
| CN110914427B (zh) | 2024-08-09 |
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| SE01 | Entry into force of request for substantive examination | ||
| SE01 | Entry into force of request for substantive examination |