HRP20231063T1 - Postupci i pripravci namijenjeni inhibiranju eksprimiranja ldha - Google Patents
Postupci i pripravci namijenjeni inhibiranju eksprimiranja ldha Download PDFInfo
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- HRP20231063T1 HRP20231063T1 HRP20231063TT HRP20231063T HRP20231063T1 HR P20231063 T1 HRP20231063 T1 HR P20231063T1 HR P20231063T T HRP20231063T T HR P20231063TT HR P20231063 T HRP20231063 T HR P20231063T HR P20231063 T1 HRP20231063 T1 HR P20231063T1
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- 238000000034 method Methods 0.000 title claims 2
- 230000002401 inhibitory effect Effects 0.000 title 1
- 101150041530 ldha gene Proteins 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 11
- 238000002360 preparation method Methods 0.000 claims 6
- 102100034671 L-lactate dehydrogenase A chain Human genes 0.000 claims 4
- 108010088350 Lactate Dehydrogenase 5 Proteins 0.000 claims 4
- 230000000692 anti-sense effect Effects 0.000 claims 4
- 208000004777 Primary Hyperoxaluria Diseases 0.000 claims 2
- 208000008852 Hyperoxaluria Diseases 0.000 claims 1
- 108091081021 Sense strand Proteins 0.000 claims 1
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 claims 1
- 229910052731 fluorine Inorganic materials 0.000 claims 1
- 239000011737 fluorine Substances 0.000 claims 1
- 210000003494 hepatocyte Anatomy 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 239000000825 pharmaceutical preparation Substances 0.000 claims 1
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- 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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- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A61K9/00—Medicinal preparations characterised by special physical form
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- C12Y101/01—Oxidoreductases acting on the CH-OH group of donors (1.1) with NAD+ or NADP+ as acceptor (1.1.1)
- C12Y101/01027—L-Lactate dehydrogenase (1.1.1.27)
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Claims (6)
1. Oligonukleotid, naznačen time što služi za smanjivanje eksprimiranja laktat-dehidrogenaze A (LDHA), gdje oligonukleotid sadrži protusmisleni lanac koji ima slijed iznijet kao UCAGAUAAAAAGGACAACAUGG (SEQ ID NO: 1) i smisleni lanac koji ima slijed iznijet kao AUGUUGUCCUUUUUAUCUGAGCAGCCGAAAGGCUGC (SEQ ID NO: 2),
gdje su svi od položaja 1, 2, 4, 6, 7, 12, 14, 16, 18-26, te 31-36 u smislenom lancu i položaja 1, 6, 8, 11-13, 15, 17, te 19-22 u protusmislenom lancu modificirani 2′-O-metilom, a svi od položaja 3, 5, 8-11, 13, 15 i 17 u smislenom lancu i položaja 2-5, 7, 9, 10, 14, 16, te 18 u protusmislenom lancu su modificirani 2′-fluorom;
gdje oligonukleotid ima fosforotioatnu spojnicu između svakog od:
položaja 1 i 2 u smislenom lancu, položaja 1 i 2 u protusmislenom lancu, položaja 2 i 3 u protusmislenom lancu, položaja 3 i 4 u protusmislenom lancu, položaja 20 i 21 u protusmislenom lancu, te položaja 21 i 22 u protusmislenom lancu;
gdje oligonukleotid ima sljedeću strukturu na položaju 1 u protusmislenom lancu:
[image]
,
gdje je svaki od nukleotida u slijedu -GAAA- na smislenom lancu konjugiran s jednovalentnim dijelom GalNac kako bi imao strukturu:
[image]
.
2. Farmaceutski pripravak, naznačen time što sadrži oligonukleotid u skladu s patentnim zahtjevom 1, koji služi za smanjivanje eksprimiranja LDHA.
3. Pripravak, naznačen time što sadrži oligonukleotid u skladu s patentnim zahtjevom 1, te što dodatno sadrži Na+ kao protuione.
4. Oligonukleotid u skladu s patentnim zahtjevom 1 ili pripravak u skladu s patentnim zahtjevom 2 ili patentnim zahtjevom 3, namijenjen upotrebi u liječenju subjekta koji ima ili je u opasnosti od toga da ima primarnu hiperoksaluriju, koju se bira između PH1, PH2, PH3, i/ili idiopatsku hiperoksaluriju, naznačen time što se postupak sastoji u smanjivanje eksprimiranja proteina LDHA u hepatocitima kod subjekta primjenom oligonukleotida ili pripravka.
5. Oligonukleotid ili pripravak, namijenjeni upotrebi u skladu s patentnim zahtjevom 4, naznačeni time što navedeni subjekt ima ili je u opasnosti od toga da ima primarnu hiperoksaluriju.
6. Oligonukleotid ili pripravak, namijenjeni upotrebi u skladu s patentnim zahtjevom 4 ili patentnim zahtjevom 5, naznačeni time što se oligonukleotid ili pripravak primijenjuje na subjektu intravenski ili supkutano.
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762572398P | 2017-10-13 | 2017-10-13 | |
| US201762572403P | 2017-10-13 | 2017-10-13 | |
| US201862726950P | 2018-09-04 | 2018-09-04 | |
| EP18867097.0A EP3679141B1 (en) | 2017-10-13 | 2018-10-12 | Methods and compositions for inhibiting expression of ldha |
| PCT/US2018/055735 WO2019075419A1 (en) | 2017-10-13 | 2018-10-12 | METHODS AND COMPOSITIONS FOR INHIBITING LDHA EXPRESSION |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20231063T1 true HRP20231063T1 (hr) | 2023-12-22 |
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20231063TT HRP20231063T1 (hr) | 2017-10-13 | 2018-10-12 | Postupci i pripravci namijenjeni inhibiranju eksprimiranja ldha |
Country Status (15)
| Country | Link |
|---|---|
| US (3) | US11286488B2 (hr) |
| EP (2) | EP3679141B1 (hr) |
| JP (2) | JP7308213B2 (hr) |
| KR (2) | KR102609396B1 (hr) |
| CN (1) | CN111448319B (hr) |
| AU (1) | AU2018346971A1 (hr) |
| CA (1) | CA3078933A1 (hr) |
| ES (1) | ES2955045T3 (hr) |
| HR (1) | HRP20231063T1 (hr) |
| HU (1) | HUE063026T2 (hr) |
| IL (1) | IL273875A (hr) |
| MX (1) | MX2020003836A (hr) |
| PL (1) | PL3679141T3 (hr) |
| RS (1) | RS64483B1 (hr) |
| WO (1) | WO2019075419A1 (hr) |
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3204497B1 (en) | 2014-10-10 | 2020-03-11 | Dicerna Pharmaceuticals, Inc. | Therapeutic inhibition of lactate dehydrogenase and agents therefor |
| WO2019075419A1 (en) | 2017-10-13 | 2019-04-18 | Dicerna Pharmaceuticals, Inc. | METHODS AND COMPOSITIONS FOR INHIBITING LDHA EXPRESSION |
| PE20220561A1 (es) * | 2017-10-20 | 2022-04-13 | Dicerna Pharmaceuticals Inc | Metodos para el tratamiento de infeccion de hepatitis b |
| US11661603B2 (en) | 2018-01-16 | 2023-05-30 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting ALDH2 expression |
| WO2021041247A1 (en) * | 2019-08-23 | 2021-03-04 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| CN114761557A (zh) * | 2019-10-02 | 2022-07-15 | 迪克纳制药公司 | 具有最小氟含量的小干扰rna的化学修饰 |
| EP4121534A1 (en) | 2020-03-18 | 2023-01-25 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant |
| EP4153586A1 (en) * | 2020-05-18 | 2023-03-29 | Chinook Therapeutics Canada, Inc. | Substituted pyrazolyl compounds and methods of use thereof |
| CN116745420A (zh) * | 2020-11-13 | 2023-09-12 | 迪克纳制药公司 | 用于抑制aldh2表达的化学修饰 |
| CA3207897A1 (en) | 2021-03-19 | 2022-09-22 | Ralf Eisenhuth | Improved oligonucleotide synthesis |
| JP2024513470A (ja) | 2021-04-09 | 2024-03-25 | バッヘン・ホールディング・アクチエンゲゼルシャフト | オリゴヌクレオチドおよびオリゴヌクレオチドコンジュゲートを合成するための擬似固相保護基および方法 |
| US11649260B2 (en) | 2021-06-18 | 2023-05-16 | Hongene Biotech Corporation | Functionalized N-acetylgalactosamine nucleosides |
| WO2023003805A1 (en) | 2021-07-19 | 2023-01-26 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder |
| AR124636A1 (es) * | 2021-08-25 | 2023-04-19 | Dicerna Pharmaceuticals Inc | COMPOSICIONES Y MÉTODOS PARA INHIBIR LA EXPRESIÓN DE a-1 ANTITRIPSINA |
| US11692001B2 (en) | 2021-08-30 | 2023-07-04 | Hongene Biotech Corporation | Functionalized n-acetylgalactosamine analogs |
| TW202334414A (zh) * | 2021-10-05 | 2023-09-01 | 美商黛瑟納製藥公司 | 用於抑制黑皮質素２受體和細胞色素ｐ４５０ １１ｂ１表現之組成物及方法 |
| US11993626B2 (en) | 2021-12-15 | 2024-05-28 | Hongene Biotech Corporation | Functionalized N-acetylgalactosamine analogs |
| WO2024061842A1 (en) | 2022-09-19 | 2024-03-28 | Bachem Holding Ag | Improved oligonucleotide synthesis |
| WO2024061157A1 (en) * | 2022-09-19 | 2024-03-28 | Kylonova (Xiamen) Biopharma Co., Ltd. | Carbohydrate-oligonucleotide conjugates, pharmaceutical compositions, and therapeutic applications |
| WO2024083746A1 (en) | 2022-10-17 | 2024-04-25 | Bachem Holding Ag | Method and composition for oligonucleotide synthesis |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2006006948A2 (en) | 2002-11-14 | 2006-01-19 | Dharmacon, Inc. | METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY |
| US10920226B2 (en) | 2002-11-14 | 2021-02-16 | Thermo Fisher Scientific Inc. | siRNA targeting LDHA |
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| WO2019075419A1 (en) | 2019-04-18 |
| EP3679141A1 (en) | 2020-07-15 |
| MX2020003836A (es) | 2020-11-06 |
| EP3679141A4 (en) | 2021-06-16 |
| PL3679141T3 (pl) | 2023-10-02 |
| ES2955045T3 (es) | 2023-11-28 |
| KR20200078530A (ko) | 2020-07-01 |
| EP3679141B1 (en) | 2023-06-07 |
| US11286488B2 (en) | 2022-03-29 |
| RS64483B1 (sr) | 2023-09-29 |
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| JP2020536587A (ja) | 2020-12-17 |
| EP4265261A2 (en) | 2023-10-25 |
| US20210062199A1 (en) | 2021-03-04 |
| HUE063026T2 (hu) | 2023-12-28 |
| CA3078933A1 (en) | 2019-04-18 |
| KR20230169413A (ko) | 2023-12-15 |
| JP2023139014A (ja) | 2023-10-03 |
| EP4265261A3 (en) | 2024-01-24 |
| KR102609396B1 (ko) | 2023-12-01 |
| CN111448319B (zh) | 2024-05-17 |
| IL273875A (en) | 2020-05-31 |
| US20200283775A1 (en) | 2020-09-10 |
| US11661604B2 (en) | 2023-05-30 |
| EP3679141C0 (en) | 2023-06-07 |
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