AR127158A1 - Oligonucléotido antisentido para la edición de arn asociado a enfermedades de poliglutamina - Google Patents
Oligonucléotido antisentido para la edición de arn asociado a enfermedades de poliglutaminaInfo
- Publication number
- AR127158A1 AR127158A1 ARP220102593A ARP220102593A AR127158A1 AR 127158 A1 AR127158 A1 AR 127158A1 AR P220102593 A ARP220102593 A AR P220102593A AR P220102593 A ARP220102593 A AR P220102593A AR 127158 A1 AR127158 A1 AR 127158A1
- Authority
- AR
- Argentina
- Prior art keywords
- antisense oligonucleotide
- rna associated
- polyglutamine diseases
- editing rna
- polyq
- Prior art date
Links
- 108010040003 polyglutamine Proteins 0.000 title abstract 4
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 3
- 239000000074 antisense oligonucleotide Substances 0.000 title abstract 3
- 238000012230 antisense oligonucleotides Methods 0.000 title abstract 3
- 201000010099 disease Diseases 0.000 title abstract 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title abstract 3
- 229920000155 polyglutamine Polymers 0.000 title abstract 2
- 108020004999 messenger RNA Proteins 0.000 abstract 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 1
- 102000040650 (ribonucleotides)n+m Human genes 0.000 abstract 1
- 101710169336 5'-deoxyadenosine deaminase Proteins 0.000 abstract 1
- 102000055025 Adenosine deaminases Human genes 0.000 abstract 1
- 230000000692 anti-sense effect Effects 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 230000002265 prevention Effects 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
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
- C12N15/11—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
- 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
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
-
- 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
-
- 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
- C12N2320/33—Alteration of splicing
-
- 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
- C12N2320/34—Allele or polymorphism specific uses
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP21199761 | 2021-09-29 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR127158A1 true AR127158A1 (es) | 2023-12-27 |
Family
ID=78179173
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP220102593A AR127158A1 (es) | 2021-09-29 | 2022-09-27 | Oligonucléotido antisentido para la edición de arn asociado a enfermedades de poliglutamina |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20240336919A1 (https=) |
| EP (1) | EP4408999A1 (https=) |
| JP (1) | JP2024536132A (https=) |
| CN (1) | CN118019848A (https=) |
| AR (1) | AR127158A1 (https=) |
| TW (1) | TW202329983A (https=) |
| WO (1) | WO2023052317A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025132708A1 (en) | 2023-12-20 | 2025-06-26 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of huntington's disease |
| GB202405143D0 (en) * | 2024-04-11 | 2024-05-29 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of poly-q disease |
Family Cites Families (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| JP4236812B2 (ja) | 1997-09-12 | 2009-03-11 | エクシコン エ／エス | オリゴヌクレオチド類似体 |
| ES2234563T5 (es) | 1999-02-12 | 2018-01-17 | Daiichi Sankyo Company, Limited | Nuevos análogos de nucleósidos y oligonucleótidos |
| US7053207B2 (en) | 1999-05-04 | 2006-05-30 | Exiqon A/S | L-ribo-LNA analogues |
| WO2004046160A2 (en) | 2002-11-18 | 2004-06-03 | Santaris Pharma A/S | Amino-lna, thio-lna and alpha-l-oxy-ln |
| WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
| WO2007090071A2 (en) | 2006-01-27 | 2007-08-09 | Isis Pharmaceuticals, Inc. | 6-modified bicyclic nucleic acid analogs |
| US7666854B2 (en) | 2006-05-11 | 2010-02-23 | Isis Pharmaceuticals, Inc. | Bis-modified bicyclic nucleic acid analogs |
| AU2007249349B2 (en) | 2006-05-11 | 2012-03-08 | Isis Pharmaceuticals, Inc. | 5'-Modified bicyclic nucleic acid analogs |
| CA2688321A1 (en) | 2007-05-30 | 2008-12-11 | Isis Pharmaceuticals, Inc. | N-substituted-aminomethylene bridged bicyclic nucleic acid analogs |
| DK2173760T4 (en) | 2007-06-08 | 2016-02-08 | Isis Pharmaceuticals Inc | Carbocyclic bicyclic nukleinsyreanaloge |
| AU2008272918B2 (en) | 2007-07-05 | 2012-09-13 | Isis Pharmaceuticals, Inc. | 6-disubstituted bicyclic nucleic acid analogs |
| WO2009067647A1 (en) | 2007-11-21 | 2009-05-28 | Isis Pharmaceuticals, Inc. | Carbocyclic alpha-l-bicyclic nucleic acid analogs |
| WO2009099326A1 (en) * | 2008-02-08 | 2009-08-13 | Prosensa Holding Bv | Methods and means for treating dna repeat instability associated genetic disorders |
| DK2356129T3 (da) | 2008-09-24 | 2013-05-13 | Isis Pharmaceuticals Inc | Substituerede alpha-L-bicykliske nukleosider |
| US9012421B2 (en) | 2009-08-06 | 2015-04-21 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| EP2580228B1 (en) | 2010-06-08 | 2016-03-23 | Ionis Pharmaceuticals, Inc. | Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom |
| US10202599B2 (en) | 2011-08-11 | 2019-02-12 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| WO2013033223A1 (en) * | 2011-08-29 | 2013-03-07 | Isis Pharmaceuticals, Inc. | Methods and compounds useful in conditions related to repeat expansion |
| US20150315595A1 (en) | 2012-03-12 | 2015-11-05 | Santaris Pharma A/S | Compositions and Methods for Modulation of ATXN3 Expression |
| EP2850092B1 (en) | 2012-04-09 | 2017-03-01 | Ionis Pharmaceuticals, Inc. | Tricyclic nucleic acid analogs |
| LT2841578T (lt) * | 2012-04-23 | 2017-09-25 | Biomarin Technologies B.V. | Rnr moduliuojantys oligonukleotidai su pagerintomis savybėmis, skirti neuromuskulinių susirgimų gydymui |
| US10435430B2 (en) | 2013-07-31 | 2019-10-08 | Ionis Pharmaceuticals, Inc. | Methods and compounds useful in conditions related to repeat expansion |
| EP3234134B1 (en) | 2014-12-17 | 2020-05-27 | ProQR Therapeutics II B.V. | Targeted rna editing |
| WO2017220751A1 (en) | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
| CN110352244B (zh) | 2016-09-01 | 2023-03-21 | ProQR治疗上市公司Ⅱ | 化学修饰的编辑rna的单链寡核苷酸 |
| JOP20190104A1 (ar) | 2016-11-10 | 2019-05-07 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن atxn3 |
| EP3571300A1 (en) | 2017-01-19 | 2019-11-27 | ProQR Therapeutics II B.V. | Oligonucleotide complexes for use in rna editing |
| WO2019158475A1 (en) | 2018-02-14 | 2019-08-22 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for rna editing |
| US20200385713A1 (en) * | 2019-01-22 | 2020-12-10 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
-
2022
- 2022-09-27 JP JP2024519267A patent/JP2024536132A/ja active Pending
- 2022-09-27 CN CN202280064982.3A patent/CN118019848A/zh active Pending
- 2022-09-27 EP EP22797687.5A patent/EP4408999A1/en active Pending
- 2022-09-27 WO PCT/EP2022/076748 patent/WO2023052317A1/en not_active Ceased
- 2022-09-27 AR ARP220102593A patent/AR127158A1/es unknown
- 2022-09-28 TW TW111136781A patent/TW202329983A/zh unknown
-
2024
- 2024-03-27 US US18/618,604 patent/US20240336919A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4408999A1 (en) | 2024-08-07 |
| WO2023052317A1 (en) | 2023-04-06 |
| US20240336919A1 (en) | 2024-10-10 |
| CN118019848A (zh) | 2024-05-10 |
| TW202329983A (zh) | 2023-08-01 |
| JP2024536132A (ja) | 2024-10-04 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| AR127158A1 (es) | Oligonucléotido antisentido para la edición de arn asociado a enfermedades de poliglutamina | |
| MX2023008469A (es) | Oligonucleotidos de doble hebra modificados. | |
| BRPI0911273B8 (pt) | composição farmacêutica compreendendo um inibidor de dpp-iv, bem como processo para sua preparação | |
| MX2022013606A (es) | Composiciones de ácido ribonucleico interferente (arni) del factor b de complemento (cfb) y métodos de uso de las mismas. | |
| MX338338B (es) | Composiciones y metodos utiles para el tratamiento de enfermedades respiratorias. | |
| BRPI0412909A (pt) | uso de rnai inibindo atividade de parp para a fabricação de um medicamento para o tratamento de cáncer | |
| CL2024001319A1 (es) | Composiciones de arni del factor b del complemento (cfb) y métodos de uso | |
| CL2007001724A1 (es) | Uso de un estrogeno para el tratamiento de enfermedades, condiciones o sintomas asociados con niveles deficientes de estrogenos en una mujer; composicion farmaceutica que comprende unidades de dosificacion diarias de hemidrato de estradiol > 0,75 y 1 | |
| BRPI0414428A (pt) | composições farmacêuticas | |
| AR054139A1 (es) | Formulacion modificada y de liberacion inmediata de esferas de memantina | |
| ECSP077999A (es) | Formulaciones de una sal de bupropion de liberación modificada | |
| CO6351742A2 (es) | Compuestos organicos para la cicatrizacion de heridas | |
| BRPI0407306A (pt) | 1,2,4- triazinas de inibição de hiv | |
| MX2022004388A (es) | Composiciones de agentes de arni de huntingtina (htt) y metodos de uso de las mismas. | |
| CO2024013531A2 (es) | Formas sólidas, sales y procesos de preparación de un inhibidor de cdk2 | |
| BR112012013164A2 (pt) | composições farmacêuticas para o estímulo de células-tronco | |
| CL2007002875A1 (es) | Composicion farmaceutica recubierta por una pelicula que comprende capecitabina y al menos un disgregante, util en el tratamiento del cancer para pacientes que tienen dificultad para tragar las formas de dosificacion solidas orales. | |
| CU20070094A7 (es) | Formas de dosificación oral sólidas que contienen una dosis baja de estradiol | |
| CL2024000086A1 (es) | Formas cristalinas de trofinetida | |
| CO2026004373A2 (es) | Composiciones de oligonucleótidos de cadena doble relacionadas con la subunidad beta e de la inhibina y métodos relacionados con estas | |
| CY1113404T1 (el) | Μεθοδος αντιμετωπισης ή προληψης της πολυ- οργανικης ανεπαρκειας | |
| BR112022023408A2 (pt) | Oligonucleotídeo de fita dupla e composição para tratar covid-19 contendo o mesmo | |
| BR0315836A (pt) | Derivados de [6,7-dihidro-5h-imidazol[1,2-alfa]imadazol-3-sulfonila mino]-propionamida, composição farmacêutica, uso do mesmo, bem como processo para sua preparação | |
| AR058173A1 (es) | Uso de sdf-1 para el tratamiento y/o prevencion de enfermedades neurologicas | |
| BRPI0513938A (pt) | composição farmacêutica contendo drospirenona e etinilestradiol |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FB | Suspension of granting procedure |