JPWO2022020106A5 - - Google Patents
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- Publication number
- JPWO2022020106A5 JPWO2022020106A5 JP2023504622A JP2023504622A JPWO2022020106A5 JP WO2022020106 A5 JPWO2022020106 A5 JP WO2022020106A5 JP 2023504622 A JP2023504622 A JP 2023504622A JP 2023504622 A JP2023504622 A JP 2023504622A JP WO2022020106 A5 JPWO2022020106 A5 JP WO2022020106A5
- Authority
- JP
- Japan
- Prior art keywords
- antibody
- complex
- conjugate
- oligonucleotide
- complex according
- 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 11
- 125000003275 alpha amino acid group Chemical group 0.000 claims 8
- 102100021158 Double homeobox protein 4 Human genes 0.000 claims 6
- 101000968549 Homo sapiens Double homeobox protein 4 Proteins 0.000 claims 6
- 230000000692 anti-sense effect Effects 0.000 claims 5
- 210000004027 cell Anatomy 0.000 claims 5
- 125000003835 nucleoside group Chemical group 0.000 claims 5
- 102000001706 Immunoglobulin Fab Fragments Human genes 0.000 claims 4
- 108010054477 Immunoglobulin Fab Fragments Proteins 0.000 claims 4
- 238000000034 method Methods 0.000 claims 4
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 3
- 230000000694 effects Effects 0.000 claims 3
- 239000002773 nucleotide Substances 0.000 claims 3
- 125000003729 nucleotide group Chemical group 0.000 claims 3
- 108091081021 Sense strand Proteins 0.000 claims 2
- 239000012634 fragment Substances 0.000 claims 2
- 150000003839 salts Chemical group 0.000 claims 2
- 108020005345 3' Untranslated Regions Proteins 0.000 claims 1
- ODHCTXKNWHHXJC-VKHMYHEASA-N 5-oxo-L-proline Chemical group OC(=O)[C@@H]1CCC(=O)N1 ODHCTXKNWHHXJC-VKHMYHEASA-N 0.000 claims 1
- 208000037149 Facioscapulohumeral dystrophy Diseases 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
- 108091030071 RNAI Proteins 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 claims 1
- ODHCTXKNWHHXJC-UHFFFAOYSA-N acide pyroglutamique Natural products OC(=O)C1CCC(=O)N1 ODHCTXKNWHHXJC-UHFFFAOYSA-N 0.000 claims 1
- 210000000349 chromosome Anatomy 0.000 claims 1
- 125000000151 cysteine group Chemical group N[C@@H](CS)C(=O)* 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 208000008570 facioscapulohumeral muscular dystrophy Diseases 0.000 claims 1
- 230000009368 gene silencing by RNA 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
- 238000012986 modification Methods 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 210000000663 muscle cell Anatomy 0.000 claims 1
- 239000002777 nucleoside Substances 0.000 claims 1
- 239000011591 potassium Substances 0.000 claims 1
- 229910052700 potassium Inorganic materials 0.000 claims 1
- 235000018102 proteins Nutrition 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 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
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
- 239000012581 transferrin Substances 0.000 claims 1
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063055768P | 2020-07-23 | 2020-07-23 | |
| US63/055,768 | 2020-07-23 | ||
| US202063061839P | 2020-08-06 | 2020-08-06 | |
| US63/061,839 | 2020-08-06 | ||
| US202163143828P | 2021-01-30 | 2021-01-30 | |
| US63/143,828 | 2021-01-30 | ||
| US202163181456P | 2021-04-29 | 2021-04-29 | |
| US63/181,456 | 2021-04-29 | ||
| PCT/US2021/040987 WO2022020106A1 (en) | 2020-07-23 | 2021-07-09 | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023535444A JP2023535444A (ja) | 2023-08-17 |
| JPWO2022020106A5 true JPWO2022020106A5 (enExample) | 2024-07-11 |
Family
ID=79729849
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023504622A Pending JP2023535444A (ja) | 2020-07-23 | 2021-07-09 | 顔面肩甲上腕型筋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れらの使用 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230272065A1 (enExample) |
| EP (1) | EP4185314A4 (enExample) |
| JP (1) | JP2023535444A (enExample) |
| KR (1) | KR20230046297A (enExample) |
| CN (1) | CN116348139A (enExample) |
| AU (1) | AU2021313057A1 (enExample) |
| BR (1) | BR112023001003A2 (enExample) |
| CA (1) | CA3186746A1 (enExample) |
| IL (1) | IL299666A (enExample) |
| MX (1) | MX2023000961A (enExample) |
| WO (1) | WO2022020106A1 (enExample) |
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20210081324A (ko) | 2018-08-02 | 2021-07-01 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
| 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 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| SG11202100934PA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| 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 |
| BR112022019889A2 (pt) | 2020-04-02 | 2023-03-07 | Mirecule Inc | Inibição direcionada com o uso de oligonucleotídeos modificados geneticamente |
| WO2022020109A1 (en) * | 2020-07-23 | 2022-01-27 | Dyne Therapeutics, Inc. | Muscle-targeting complexes and uses thereof in treating muscle atrophy |
| 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 |
| US11648318B2 (en) | 2021-07-09 | 2023-05-16 | Dyne Therapeutics, Inc. | Anti-transferrin receptor (TFR) antibody and uses thereof |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11633498B2 (en) | 2021-07-09 | 2023-04-25 | 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 |
| AU2022309028A1 (en) * | 2021-07-09 | 2024-01-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating dystrophinopathies |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| IL316143A (en) | 2022-04-15 | 2024-12-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of myotonic dystrophy |
| IL317868A (en) * | 2022-07-06 | 2025-02-01 | Dyne Therapeutics Inc | CNS targeting complexes and their uses |
| CA3261325A1 (en) * | 2022-07-06 | 2024-01-11 | Dyne Therapeutics, Inc. | MUSCLE TARGETTING COMPLEXES AND THEIR USES TO TREAT FACIO-SCAPULO-HUMERAL MYOPATHY |
| 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 |
|---|---|---|---|---|
| KR20250073514A (ko) * | 2015-06-24 | 2025-05-27 | 제이씨알 파마 가부시키가이샤 | 혈액뇌관문을 통과하는 항인간 트랜스페린 수용체 항체 |
| FR3049951A1 (fr) * | 2016-04-12 | 2017-10-13 | Univ Rabelais Francois | Nouvelles constructions peptidiques et leur utilisation dans le traitement de la toxoplasmose |
| US20190240346A1 (en) * | 2016-06-20 | 2019-08-08 | Genahead Bio, Inc. | Antibody-drug conjugate |
| MX2019008199A (es) * | 2017-01-06 | 2019-11-25 | Avidity Biosciences Llc | Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon. |
| TWI869346B (zh) * | 2018-05-30 | 2025-01-11 | 瑞士商諾華公司 | Ｅｎｔｐｄ２抗體、組合療法、及使用該等抗體和組合療法之方法 |
| EP3829594A4 (en) * | 2018-08-02 | 2022-05-11 | Dyne Therapeutics, Inc. | MUSCLE TARGETING COMPLEXES AND THEIR USES FOR THE TREATMENT OF HYPERTROPHIC CARDIOMYOPATHY |
| EP3829635A4 (en) * | 2018-08-02 | 2022-05-11 | Dyne Therapeutics, Inc. | MUSCLE TARGETING COMPLEXES AND THEIR USES FOR THE TREATMENT OF MUSCLE ATROPHY |
| KR20210081324A (ko) * | 2018-08-02 | 2021-07-01 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
| JP7705793B2 (ja) * | 2018-08-02 | 2025-07-10 | ダイン セラピューティクス，インコーポレーテッド | 筋強直症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れらの使用 |
| IL319265A (en) * | 2018-12-21 | 2025-04-01 | Avidity Biosciences Inc | Anti-transferrin receptor antibodies and their uses |
-
2021
- 2021-07-09 IL IL299666A patent/IL299666A/en unknown
- 2021-07-09 WO PCT/US2021/040987 patent/WO2022020106A1/en not_active Ceased
- 2021-07-09 JP JP2023504622A patent/JP2023535444A/ja active Pending
- 2021-07-09 KR KR1020237005852A patent/KR20230046297A/ko active Pending
- 2021-07-09 CN CN202180065042.1A patent/CN116348139A/zh active Pending
- 2021-07-09 AU AU2021313057A patent/AU2021313057A1/en active Pending
- 2021-07-09 EP EP21846007.9A patent/EP4185314A4/en active Pending
- 2021-07-09 CA CA3186746A patent/CA3186746A1/en active Pending
- 2021-07-09 BR BR112023001003A patent/BR112023001003A2/pt unknown
- 2021-07-09 MX MX2023000961A patent/MX2023000961A/es unknown
- 2021-07-09 US US18/017,173 patent/US20230272065A1/en active Pending