AR124227A1 - Oligonucleótidos antisentido que actúan sobre atxn3 - Google Patents
Oligonucleótidos antisentido que actúan sobre atxn3Info
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- AR124227A1 AR124227A1 ARP210103342A ARP210103342A AR124227A1 AR 124227 A1 AR124227 A1 AR 124227A1 AR P210103342 A ARP210103342 A AR P210103342A AR P210103342 A ARP210103342 A AR P210103342A AR 124227 A1 AR124227 A1 AR 124227A1
- Authority
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- Prior art keywords
- compound
- seq
- atxn3
- nucleoside
- lna
- Prior art date
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- 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
- C12N15/1137—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 against enzymes
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- 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
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- 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
- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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- 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
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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- 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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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- 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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Organic Chemistry (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
La presente invención se refiere a oligonucleótidos antisentido de LNA (oligómeros) complementarios de secuencias de pre-mARN de ATXN3, que son capaces de inhibir la expresión de proteína de ATNX3. La inhibición de la expresión de ATXN3 es beneficiosa para el tratamiento de la ataxia espinocerebelosa. Reivindicación 1: Un oligonucleótido antisentido seleccionado del grupo que consiste en compuestos ID Nº 1605_2, 1605_4, 1605_3, 1605_5, 1605_23, 1809_8, 1810_39, 1812_4, 1813_4, 1813_15 y 1813_16; o una sal de este aceptable desde el punto de vista farmacéutico. Reivindicación 2: Un oligonucleótido antisentido de acuerdo con la siguiente anotación química: a) [LR]T[ₛP]×[LR][⁵ᵐᵉ]C[ₛP]×[ᵈR]T[ₛP]×[LR]T[ₛP]×[ᵈR]C[ₛP]×[LR]A[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]C[ₛP]×[ᵈR]C[ₛP]×[ᵈR]A[ₛP]×[ᵈR]T[ₛP]×[LR][⁵ᵐᵉ]C[ₛP]×[LR]A[ₛP]×[LR]A (SEQ ID Nº 1605) (compuesto ID Nº 1605_2); b) [LR]T[ₛP]×[LR][⁵ᵐᵉ]C[ₛP]×[ᵈR]T[ₛP]×[LR]T[ₛP]×[ᵈR]C[ₛP]×[ᵈR]A[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]C[ₛP]×[ᵈR]C[ₛP]×[LR]A[ₛP]×[ᵈR]T[ₛP]×[LR][⁵ᵐᵉ]C[ₛP]×[LR]A[ₛP]×[LR]A (SEQ ID Nº 1605) (compuesto ID Nº 1605_4); c) [LR]T[ₛP]×[LR][⁵ᵐᵉ]C[ₛP]×[ᵈR]T[ₛP]×[LR]T[ₛP]×[ᵈR]C[ₛP]×[ᵈR]A[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]C[ₛP]×[ᵈR]C[ₛP]×[ᵈR]A[ₛP]×[ᵈR]T[ₛP]×[LR][⁵ᵐᵉ]C[ₛP]×[LR]A[ₛP]×[LR]A (SEQ ID Nº 1605) (compuesto ID Nº 1605_3); d) [LR]T[ₛP]×[LR][⁵ᵐᵉ]C[ₛP]×[LR]T[ₛP]×[ᵈR]T[ₛP]×[LR][⁵ᵐᵉ]C[ₛP]×[ᵈR]A[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]C[ₛP]×[ᵈR]C[ₛP]×[ᵈR]A[ₛP]×[LR]T[ₛP]×[LR][⁵ᵐᵉ]C[ₛP]×[LR]A[ₛP]×[LR]A (SEQ ID Nº 1605) (compuesto ID Nº 1605_5); e) [LR]T[ₛP]×[LR][⁵ᵐᵉ]C[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[LR][⁵ᵐᵉ]C[ₛP]×[ᵈR]A[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]C[ₛP]×[ᵈR]C[ₛP]×[LR]A[ₛP]×[LR]T[ₛP]×[ᵈR]C[ₛP]×[LR]A[ₛP]×[LR]A (SEQ ID Nº 1605) (compuesto ID Nº 1605_23); f) [LR]G[ₛP]×[LR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]C[ₛP]×[LR]A[ₛP]×[ᵈR]C[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]C[ₛP]×[LR]A[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]C[ₛP]×[LR][⁵ᵐᵉ]C[ₛP]×[LR][⁵ᵐᵉ]C (SEQ ID Nº 1809) (compuesto ID Nº 1809_8); g) [LR]T[ₛP]×[LR]A[ₛP]×[ᵈR]C[ₛP]×[LR]A[ₛP]×[ᵈR]C[ₛP]×[LR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]C[ₛP]×[ᵈR]A[ₛP]×[ᵈR]T[ₛP]×[LR]T[ₛP]×[ᵈR]C[ₛP]×[LR][⁵ᵐᵉ]C[ₛP]×[LR][⁵ᵐᵉ]C (SEQ ID Nº 1810) (compuesto ID Nº 1810_39); h) [LR]T[ₛP]×[LR]G[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]C[ₛP]×[ᵈR]A[ₛP]×[ᵈR]C[ₛP]×[LR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]C[ₛP]×[ᵈR]A[ₛP]×[ᵈR]T[ₛP]×[LR]T[ₛP]×[LR][⁵ᵐᵉ]C[ₛP]×[LR][⁵ᵐᵉ]C (SEQ ID Nº 1812) (compuesto ID Nº 1812_4); i) [LR][⁵ᵐᵉ]C[ₛP]×[LR]T[ₛP]×[LR]G[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]C[ₛP]×[LR]A[ₛP]×[ᵈR]C[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]C[ₛP]×[ᵈR]A[ₛP]×[LR]T[ₛP]×[LR]T[ₛP]×[LR][⁵ᵐᵉ]C (SEQ ID Nº 1813) (compuesto ID Nº 1813_4); j) [LR][⁵ᵐᵉ]C[ₛP]×[LR]T[ₛP]×[LR]G[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵐR]C[ₛP]×[ᵐR]A[ₛP]×[ᵈR]C[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]C[ₛP]×[ᵈR]A[ₛP]×[LR]T[ₛP]×[LR]T[ₛP]×[LR][⁵ᵐᵉ]C (SEQ ID Nº1813) (compuesto ID Nº 1813_15); o k) [LR][⁵ᵐᵉ]C[ₛP]×[LR]T[ₛP]×[LR]G[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵐR]C[ₛP]×[ᵈR]A[ₛP]×[ᵐR]C[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]T[ₛP]×[ᵈR]A[ₛP]×[ᵈR]C[ₛP]×[ᵈR]A[ₛP]×[LR]T[ₛP]×[LR]T[ₛP]×[LR][⁵ᵐᵉ]C (SEQ ID Nº 1813) (compuesto ID Nº 1813_16); o una sal de este aceptable desde el punto de vista farmacéutico, en donde [LR] es un nucleósido de b-D-oxi-LNA, [LR][5me]C es un nucleósido de b-D-oxi-LNA 5-metil citosina, [dR] es un nucleósido de ADN, [sP] es un enlace internucleosídico fosforotioato (estereoindefinido), y [mR] es un nucleósido de 2-O-metilo.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP20211623 | 2020-12-03 |
Publications (1)
Publication Number | Publication Date |
---|---|
AR124227A1 true AR124227A1 (es) | 2023-03-01 |
Family
ID=73698681
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ARP210103342A AR124227A1 (es) | 2020-12-03 | 2021-12-02 | Oligonucleótidos antisentido que actúan sobre atxn3 |
Country Status (4)
Country | Link |
---|---|
US (1) | US20230060373A1 (es) |
AR (1) | AR124227A1 (es) |
TW (1) | TW202237843A (es) |
WO (1) | WO2022117747A2 (es) |
Family Cites Families (31)
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JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
CN1273476C (zh) | 1997-09-12 | 2006-09-06 | 埃克西康有限公司 | 寡核苷酸类似物 |
ES2234563T5 (es) | 1999-02-12 | 2018-01-17 | Daiichi Sankyo Company, Limited | Nuevos análogos de nucleósidos y oligonucleótidos |
ATE356824T1 (de) | 1999-05-04 | 2007-04-15 | Santaris Pharma As | L-ribo-lna analoge |
US6617442B1 (en) | 1999-09-30 | 2003-09-09 | Isis Pharmaceuticals, Inc. | Human Rnase H1 and oligonucleotide compositions thereof |
DK3222722T3 (da) | 2002-11-18 | 2019-06-17 | Roche Innovation Ct Copenhagen As | Antisense-design |
WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
CN102908630B (zh) | 2006-01-27 | 2014-11-19 | Isis制药公司 | 6-修饰的双环核酸类似物 |
EP2066684B1 (en) | 2006-05-11 | 2012-07-18 | Isis Pharmaceuticals, Inc. | 5'-modified bicyclic nucleic acid analogs |
US7666854B2 (en) | 2006-05-11 | 2010-02-23 | Isis Pharmaceuticals, Inc. | Bis-modified bicyclic nucleic acid analogs |
EP2410054B2 (en) | 2006-10-18 | 2019-12-11 | Ionis Pharmaceuticals, Inc. | Antisense compounds |
US8278425B2 (en) | 2007-05-30 | 2012-10-02 | Isis Pharmaceuticals, Inc. | N-substituted-aminomethylene bridged bicyclic nucleic acid analogs |
WO2008154401A2 (en) | 2007-06-08 | 2008-12-18 | Isis Pharmaceuticals, Inc. | Carbocyclic bicyclic nucleic acid analogs |
DK2176280T4 (en) | 2007-07-05 | 2015-07-20 | Isis Pharmaceuticals Inc | 6-Disubstituerede bicykliske nukleinsyreanaloge |
WO2009067647A1 (en) | 2007-11-21 | 2009-05-28 | Isis Pharmaceuticals, Inc. | Carbocyclic alpha-l-bicyclic nucleic acid analogs |
WO2010036698A1 (en) | 2008-09-24 | 2010-04-01 | Isis Pharmaceuticals, Inc. | Substituted alpha-l-bicyclic nucleosides |
EP2462153B1 (en) | 2009-08-06 | 2015-07-29 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
US8846637B2 (en) | 2010-06-08 | 2014-09-30 | Isis Pharmaceuticals, Inc. | Substituted 2′-amino and 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom |
EP3067421B1 (en) | 2011-02-08 | 2018-10-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
EP3922722B1 (en) | 2011-08-11 | 2023-06-28 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
US20150315595A1 (en) | 2012-03-12 | 2015-11-05 | Santaris Pharma A/S | Compositions and Methods for Modulation of ATXN3 Expression |
WO2013154798A1 (en) | 2012-04-09 | 2013-10-17 | Isis Pharmaceuticals, Inc. | Tricyclic nucleic acid analogs |
AU2013346767B2 (en) | 2012-11-15 | 2019-04-11 | Roche Innovation Center Copenhagen A/S | Oligonucleotide conjugates |
US10435430B2 (en) | 2013-07-31 | 2019-10-08 | Ionis Pharmaceuticals, Inc. | Methods and compounds useful in conditions related to repeat expansion |
KR102287532B1 (ko) | 2014-01-30 | 2021-08-11 | 에프. 호프만-라 로슈 아게 | 생분해성 컨쥬게이트를 갖는 폴리 올리고머 화합물 |
WO2016127002A1 (en) * | 2015-02-04 | 2016-08-11 | Bristol-Myers Squibb Company | Lna oligonucleotides with alternating flanks |
EP3653712B1 (en) | 2015-02-04 | 2022-11-30 | F. Hoffmann-La Roche AG | Tau antisense oligomers and uses thereof |
JOP20190104A1 (ar) | 2016-11-10 | 2019-05-07 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن atxn3 |
EP3799602A4 (en) | 2018-05-09 | 2022-02-16 | Ionis Pharmaceuticals, Inc. | COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION |
WO2020172559A1 (en) | 2019-02-22 | 2020-08-27 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing atxn3 expression |
WO2020245233A1 (en) * | 2019-06-06 | 2020-12-10 | F. Hoffmann-La Roche Ag | Antisense oligonucleotides targeting atxn3 |
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2021
- 2021-12-02 AR ARP210103342A patent/AR124227A1/es unknown
- 2021-12-02 US US17/540,534 patent/US20230060373A1/en not_active Abandoned
- 2021-12-02 WO PCT/EP2021/084018 patent/WO2022117747A2/en active Application Filing
- 2021-12-02 TW TW110145028A patent/TW202237843A/zh unknown
Also Published As
Publication number | Publication date |
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TW202237843A (zh) | 2022-10-01 |
US20230060373A1 (en) | 2023-03-02 |
WO2022117747A3 (en) | 2022-07-28 |
WO2022117747A2 (en) | 2022-06-09 |
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