JPWO2022026152A5 - - Google Patents
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- Publication number
- JPWO2022026152A5 JPWO2022026152A5 JP2023504653A JP2023504653A JPWO2022026152A5 JP WO2022026152 A5 JPWO2022026152 A5 JP WO2022026152A5 JP 2023504653 A JP2023504653 A JP 2023504653A JP 2023504653 A JP2023504653 A JP 2023504653A JP WO2022026152 A5 JPWO2022026152 A5 JP WO2022026152A5
- Authority
- JP
- Japan
- Prior art keywords
- complex
- seq
- antibody
- conjugate
- oligonucleotide
- 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
Links
- 108091034117 Oligonucleotide Proteins 0.000 claims 19
- 239000002773 nucleotide Substances 0.000 claims 12
- 125000003729 nucleotide group Chemical group 0.000 claims 12
- 125000003275 alpha amino acid group Chemical group 0.000 claims 8
- 102000018658 Myotonin-Protein Kinase Human genes 0.000 claims 6
- 108010052185 Myotonin-Protein Kinase Proteins 0.000 claims 6
- 210000004027 cell Anatomy 0.000 claims 6
- 230000000295 complement effect Effects 0.000 claims 6
- IQFYYKKMVGJFEH-XLPZGREQSA-N Thymidine Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-XLPZGREQSA-N 0.000 claims 4
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 claims 4
- 201000010099 disease Diseases 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 108700028369 Alleles Proteins 0.000 claims 2
- DWRXFEITVBNRMK-UHFFFAOYSA-N Beta-D-1-Arabinofuranosylthymine Natural products O=C1NC(=O)C(C)=CN1C1C(O)C(O)C(CO)O1 DWRXFEITVBNRMK-UHFFFAOYSA-N 0.000 claims 2
- 102000001706 Immunoglobulin Fab Fragments Human genes 0.000 claims 2
- 108010054477 Immunoglobulin Fab Fragments Proteins 0.000 claims 2
- 206010068871 Myotonic dystrophy Diseases 0.000 claims 2
- IQFYYKKMVGJFEH-UHFFFAOYSA-N beta-L-thymidine Natural products O=C1NC(=O)C(C)=CN1C1OC(CO)C(O)C1 IQFYYKKMVGJFEH-UHFFFAOYSA-N 0.000 claims 2
- DRTQHJPVMGBUCF-PSQAKQOGSA-N beta-L-uridine Natural products O[C@H]1[C@@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-PSQAKQOGSA-N 0.000 claims 2
- 230000021615 conjugation Effects 0.000 claims 2
- 239000012634 fragment Substances 0.000 claims 2
- 201000009340 myotonic dystrophy type 1 Diseases 0.000 claims 2
- 239000002777 nucleoside Substances 0.000 claims 2
- 125000003835 nucleoside group Chemical group 0.000 claims 2
- 150000003839 salts Chemical group 0.000 claims 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 2
- 229940104230 thymidine Drugs 0.000 claims 2
- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 claims 2
- 229940045145 uridine Drugs 0.000 claims 2
- ODHCTXKNWHHXJC-VKHMYHEASA-N 5-oxo-L-proline Chemical group OC(=O)[C@@H]1CCC(=O)N1 ODHCTXKNWHHXJC-VKHMYHEASA-N 0.000 claims 1
- DGAQECJNVWCQMB-PUAWFVPOSA-M Ilexoside XXIX Chemical group C[C@@H]1CC[C@@]2(CC[C@@]3(C(=CC[C@H]4[C@]3(CC[C@@H]5[C@@]4(CC[C@@H](C5(C)C)OS(=O)(=O)[O-])C)C)[C@@H]2[C@]1(C)O)C)C(=O)O[C@H]6[C@@H]([C@H]([C@@H]([C@H](O6)CO)O)O)O.[Na+] DGAQECJNVWCQMB-PUAWFVPOSA-M 0.000 claims 1
- ZLMJMSJWJFRBEC-UHFFFAOYSA-N Potassium Chemical compound [K] ZLMJMSJWJFRBEC-UHFFFAOYSA-N 0.000 claims 1
- ODHCTXKNWHHXJC-GSVOUGTGSA-N Pyroglutamic acid Natural products OC(=O)[C@H]1CCC(=O)N1 ODHCTXKNWHHXJC-GSVOUGTGSA-N 0.000 claims 1
- 208000037140 Steinert myotonic dystrophy Diseases 0.000 claims 1
- ODHCTXKNWHHXJC-UHFFFAOYSA-N acide pyroglutamique Natural products OC(=O)C1CCC(=O)N1 ODHCTXKNWHHXJC-UHFFFAOYSA-N 0.000 claims 1
- 230000001668 ameliorated effect Effects 0.000 claims 1
- 125000000151 cysteine group Chemical group N[C@@H](CS)C(=O)* 0.000 claims 1
- 239000005549 deoxyribonucleoside Substances 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 125000003588 lysine group Chemical group [H]N([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])(N([H])[H])C(*)=O 0.000 claims 1
- 159000000003 magnesium salts Chemical class 0.000 claims 1
- 210000000663 muscle cell Anatomy 0.000 claims 1
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 1
- 239000011591 potassium Substances 0.000 claims 1
- 229910052700 potassium Inorganic materials 0.000 claims 1
- 239000011734 sodium Substances 0.000 claims 1
- 229910052708 sodium Inorganic materials 0.000 claims 1
- 125000000547 substituted alkyl group Chemical group 0.000 claims 1
- 239000012581 transferrin Substances 0.000 claims 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063055749P | 2020-07-23 | 2020-07-23 | |
| US63/055,749 | 2020-07-23 | ||
| US202063069075P | 2020-08-23 | 2020-08-23 | |
| US63/069,075 | 2020-08-23 | ||
| US202163143827P | 2021-01-30 | 2021-01-30 | |
| US63/143,827 | 2021-01-30 | ||
| PCT/US2021/040986 WO2022026152A2 (en) | 2020-07-23 | 2021-07-09 | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023535078A JP2023535078A (ja) | 2023-08-15 |
| JPWO2022026152A5 true JPWO2022026152A5 (enExample) | 2024-07-18 |
Family
ID=80038121
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023504653A Pending JP2023535078A (ja) | 2020-07-23 | 2021-07-09 | 筋緊張性ジストロフィーを処置するための筋標的化複合体およびその使用 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20240016952A1 (enExample) |
| EP (1) | EP4185383A4 (enExample) |
| JP (1) | JP2023535078A (enExample) |
| KR (1) | KR20230042713A (enExample) |
| CN (1) | CN116438304A (enExample) |
| AU (1) | AU2021318851A1 (enExample) |
| BR (1) | BR112023001126A8 (enExample) |
| CA (1) | CA3186742A1 (enExample) |
| IL (1) | IL299663A (enExample) |
| MX (1) | MX2023000958A (enExample) |
| WO (1) | WO2022026152A2 (enExample) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US12370264B1 (en) | 2018-08-02 | 2025-07-29 | Dyne Therapeutics, Inc. | Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| WO2020028832A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| CN116194470B (zh) * | 2020-07-23 | 2025-10-03 | 达因疗法公司 | 肌肉靶向复合物及其用途 |
| EP4208173A4 (en) * | 2020-09-03 | 2025-11-26 | Dyne Therapeutics Inc | PROCESSES FOR PREPARING PROTEIN-OLIGONUCLEOTIC COMPLEXES |
| IL304048A (en) * | 2020-12-31 | 2023-08-01 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| EP4366784A1 (en) | 2021-07-09 | 2024-05-15 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating dystrophinopathies |
| US11672872B2 (en) | 2021-07-09 | 2023-06-13 | Dyne Therapeutics, Inc. | Anti-transferrin receptor antibody and uses thereof |
| US11633498B2 (en) | 2021-07-09 | 2023-04-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| AU2023254846A1 (en) | 2022-04-15 | 2024-10-10 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating myotonic dystrophy |
| IL315944A (en) * | 2022-04-15 | 2024-11-01 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| AU2023252927A1 (en) * | 2022-04-15 | 2024-10-10 | Dyne Therapeutics, Inc. | Dosing of muscle targeting complexes for treating myotonic dystrophy |
| WO2023220719A2 (en) * | 2022-05-13 | 2023-11-16 | University Of Washington | Method for treatment of myotonic dystrophy combining protein expression and rna interference vector delivery with tissue detargeting |
| WO2025006955A2 (en) * | 2023-06-30 | 2025-01-02 | Ionis Pharmaceuticals, Inc. | Cd29 targeted oligonucleotides and uses thereof |
| WO2025024334A1 (en) | 2023-07-21 | 2025-01-30 | Marrow Therapeutics, Inc. | Hematopoietic cell targeting conjugates and related methods |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NZ610976A (en) * | 2010-12-20 | 2015-07-31 | Genentech Inc | Anti-mesothelin antibodies and immunoconjugates |
| TWI769982B (zh) * | 2015-06-24 | 2022-07-11 | 日商Ｊｃｒ製藥股份有限公司 | 通過血腦障壁之抗人類運鐵蛋白受體抗體 |
| FR3049951A1 (fr) * | 2016-04-12 | 2017-10-13 | Univ Rabelais Francois | Nouvelles constructions peptidiques et leur utilisation dans le traitement de la toxoplasmose |
| CA3025345A1 (en) * | 2016-05-27 | 2017-11-30 | Abbvie Biotherapeutics Inc. | Bispecific binding proteins binding an immunomodulatory protein and a tumor antigen |
| KR20250148679A (ko) * | 2017-12-06 | 2025-10-14 | 어비디티 바이오사이언시스 인크. | 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법 |
| WO2020028842A1 (en) * | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating hypertrophic cardiomyopathy |
| IL320597A (en) * | 2018-08-02 | 2025-07-01 | Dyne Therapeutics Inc | Transferrin receptor-targeting conjugates and their uses |
| EP3829596A4 (en) * | 2018-08-02 | 2022-05-11 | Dyne Therapeutics, Inc. | MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF MYOTONIC DYSTROPHY |
| WO2020028836A1 (en) * | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle-targeting complexes and uses thereof in treating muscle atrophy |
| IL319265A (en) * | 2018-12-21 | 2025-04-01 | Avidity Biosciences Inc | Anti-transferrin receptor antibodies and their uses |
-
2021
- 2021-07-09 US US18/017,170 patent/US20240016952A1/en active Pending
- 2021-07-09 BR BR112023001126A patent/BR112023001126A8/pt unknown
- 2021-07-09 JP JP2023504653A patent/JP2023535078A/ja active Pending
- 2021-07-09 IL IL299663A patent/IL299663A/en unknown
- 2021-07-09 CA CA3186742A patent/CA3186742A1/en active Pending
- 2021-07-09 EP EP21850418.1A patent/EP4185383A4/en active Pending
- 2021-07-09 CN CN202180064935.4A patent/CN116438304A/zh active Pending
- 2021-07-09 AU AU2021318851A patent/AU2021318851A1/en active Pending
- 2021-07-09 MX MX2023000958A patent/MX2023000958A/es unknown
- 2021-07-09 WO PCT/US2021/040986 patent/WO2022026152A2/en not_active Ceased
- 2021-07-09 KR KR1020237005830A patent/KR20230042713A/ko active Pending