KR20230127263A - Clrn1-연관된 청력손실 및/또는 시력손실을 치료하기위한 조성물 및 방법 - Google Patents
Clrn1-연관된 청력손실 및/또는 시력손실을 치료하기위한 조성물 및 방법 Download PDFInfo
- Publication number
- KR20230127263A KR20230127263A KR1020237025236A KR20237025236A KR20230127263A KR 20230127263 A KR20230127263 A KR 20230127263A KR 1020237025236 A KR1020237025236 A KR 1020237025236A KR 20237025236 A KR20237025236 A KR 20237025236A KR 20230127263 A KR20230127263 A KR 20230127263A
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- South Korea
- Prior art keywords
- promoter
- amino acid
- construct
- amino acids
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
-
- 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
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
-
- 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
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
-
- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
- C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Immunology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Toxicology (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Cell Biology (AREA)
- Plant Pathology (AREA)
- Ophthalmology & Optometry (AREA)
- Manufacturing & Machinery (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063131413P | 2020-12-29 | 2020-12-29 | |
| US63/131,413 | 2020-12-29 | ||
| PCT/US2021/064924 WO2022146839A1 (en) | 2020-12-29 | 2021-12-22 | Compositions and methods for treating clrn1-associated hearing loss and/or vision loss |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20230127263A true KR20230127263A (ko) | 2023-08-31 |
Family
ID=80050943
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020237025236A Pending KR20230127263A (ko) | 2020-12-29 | 2021-12-22 | Clrn1-연관된 청력손실 및/또는 시력손실을 치료하기위한 조성물 및 방법 |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20240139344A1 (enExample) |
| EP (1) | EP4271419A1 (enExample) |
| JP (1) | JP2024500786A (enExample) |
| KR (1) | KR20230127263A (enExample) |
| CN (1) | CN117157108A (enExample) |
| AU (1) | AU2021412962A1 (enExample) |
| CA (1) | CA3203373A1 (enExample) |
| CL (1) | CL2023001896A1 (enExample) |
| CO (1) | CO2023007325A2 (enExample) |
| DO (2) | DOP2023000132A (enExample) |
| EC (1) | ECSP23057004A (enExample) |
| IL (1) | IL304006A (enExample) |
| JO (1) | JOP20230151A1 (enExample) |
| MX (1) | MX2023007800A (enExample) |
| PE (1) | PE20240012A1 (enExample) |
| WO (1) | WO2022146839A1 (enExample) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN120285151B (zh) * | 2025-04-14 | 2026-03-31 | 山东第一医科大学(山东省医学科学院) | HuR及其编码基因在制备治疗老年性耳聋药物中的应用 |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| EP0173552B1 (en) | 1984-08-24 | 1991-10-09 | The Upjohn Company | Recombinant dna compounds and the expression of polypeptides such as tpa |
| US5168062A (en) | 1985-01-30 | 1992-12-01 | University Of Iowa Research Foundation | Transfer vectors and microorganisms containing human cytomegalovirus immediate-early promoter-regulatory DNA sequence |
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US6548286B1 (en) | 1997-04-14 | 2003-04-15 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant AAV product |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| JP4608210B2 (ja) | 2001-05-31 | 2011-01-12 | ノバルティス バクシンズ アンド ダイアグノスティックス,インコーポレーテッド | キメラアルファウイルスレプリコン粒子 |
| ES2330202T5 (es) | 2001-09-06 | 2014-01-20 | Alphavax, Inc. | Sistemas vectores basados en replicones de alfavirus |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| CA2553676A1 (en) | 2004-01-28 | 2005-08-11 | Research Development Foundation | Human soluble neuropilin-1 primary polyadenylation signal and uses thereof |
| WO2006012414A2 (en) | 2004-07-20 | 2006-02-02 | Critical Therapeutics, Inc. | Novel polyadenylation signal for use in expression vectors |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| AU2013240248B2 (en) | 2012-03-26 | 2018-12-20 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Delivery of packaged RNA to mammalian cells |
| GB201518979D0 (en) | 2015-10-27 | 2015-12-09 | Univ Leuven Kath | Treatment of hepatic steatosis related oligo-ovulation |
| US10201455B2 (en) | 2016-06-22 | 2019-02-12 | The Charles Stark Draper Laboratory, Inc. | System for inner ear drug delivery via trans-round window membrane injection |
| EP3510161A4 (en) | 2016-08-23 | 2020-04-22 | Akouos, Inc. | COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON |
| WO2019084145A1 (en) | 2017-10-25 | 2019-05-02 | The Charles Stark Draper Laboratory Inc. | APPARATUS AND METHOD FOR DRUG DELIVERY ACROSS THE ROUND WINDOW MEMBRANE |
| IL325122A (en) | 2018-02-22 | 2026-02-01 | Akouos Inc | Compositions and methods for treating non-age-related hearing impairment in humans |
| WO2019200016A1 (en) * | 2018-04-10 | 2019-10-17 | President And Fellows Of Harvard College | Aav vectors encoding clarin-1 or gjb2 and uses thereof |
| US20210277417A1 (en) * | 2018-06-25 | 2021-09-09 | Akouos, Inc. | Methods of treating clrn1-associated hearing loss and/or vision loss |
| EP4063510A1 (en) | 2018-11-07 | 2022-09-28 | Akouos, Inc. | Use of adeno-associated viral vectors to correct gene defects/ express proteins in hair cells and supporting cells in the inner ear |
| AU2021279008A1 (en) | 2020-05-27 | 2022-12-08 | Akouos, Inc. | Devices, systems, and methods for delivering fluid to the inner ear |
-
2021
- 2021-12-22 IL IL304006A patent/IL304006A/en unknown
- 2021-12-22 CA CA3203373A patent/CA3203373A1/en active Pending
- 2021-12-22 EP EP21848394.9A patent/EP4271419A1/en active Pending
- 2021-12-22 US US18/269,307 patent/US20240139344A1/en active Pending
- 2021-12-22 KR KR1020237025236A patent/KR20230127263A/ko active Pending
- 2021-12-22 CN CN202180088219.XA patent/CN117157108A/zh active Pending
- 2021-12-22 JP JP2023537130A patent/JP2024500786A/ja active Pending
- 2021-12-22 MX MX2023007800A patent/MX2023007800A/es unknown
- 2021-12-22 AU AU2021412962A patent/AU2021412962A1/en active Pending
- 2021-12-22 WO PCT/US2021/064924 patent/WO2022146839A1/en not_active Ceased
- 2021-12-22 PE PE2023001959A patent/PE20240012A1/es unknown
-
2023
- 2023-06-02 CO CONC2023/0007325A patent/CO2023007325A2/es unknown
- 2023-06-23 CL CL2023001896A patent/CL2023001896A1/es unknown
- 2023-06-25 JO JOJO/P/2023/0151A patent/JOP20230151A1/ar unknown
- 2023-06-26 DO DO2023000132A patent/DOP2023000132A/es unknown
- 2023-07-27 EC ECSENADI202357004A patent/ECSP23057004A/es unknown
-
2025
- 2025-11-19 DO DO2025000286A patent/DOP2025000286A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP4271419A1 (en) | 2023-11-08 |
| ECSP23057004A (es) | 2023-08-31 |
| CO2023007325A2 (es) | 2023-06-20 |
| WO2022146839A1 (en) | 2022-07-07 |
| AU2021412962A1 (en) | 2023-08-17 |
| DOP2025000286A (es) | 2025-12-30 |
| CN117157108A (zh) | 2023-12-01 |
| DOP2023000132A (es) | 2023-07-31 |
| MX2023007800A (es) | 2023-07-11 |
| US20240139344A1 (en) | 2024-05-02 |
| IL304006A (en) | 2023-08-01 |
| CA3203373A1 (en) | 2022-07-07 |
| JP2024500786A (ja) | 2024-01-10 |
| JOP20230151A1 (ar) | 2023-06-25 |
| PE20240012A1 (es) | 2024-01-04 |
| CL2023001896A1 (es) | 2023-12-15 |
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| PA0105 | International application |
Patent event date: 20230724 Patent event code: PA01051R01D Comment text: International Patent Application |
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| PG1501 | Laying open of application | ||
| A201 | Request for examination | ||
| PA0201 | Request for examination |
Patent event code: PA02012R01D Patent event date: 20241219 Comment text: Request for Examination of Application |