JPWO2020028840A5 - - Google Patents
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- Publication number
- JPWO2020028840A5 JPWO2020028840A5 JP2021529256A JP2021529256A JPWO2020028840A5 JP WO2020028840 A5 JPWO2020028840 A5 JP WO2020028840A5 JP 2021529256 A JP2021529256 A JP 2021529256A JP 2021529256 A JP2021529256 A JP 2021529256A JP WO2020028840 A5 JPWO2020028840 A5 JP WO2020028840A5
- Authority
- JP
- Japan
- Prior art keywords
- conjugate
- oligonucleotide
- transferrin receptor
- receptor antibody
- fxn
- 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 13
- 108020003175 receptors Proteins 0.000 claims 8
- 102000005962 receptors Human genes 0.000 claims 8
- 239000012581 transferrin Substances 0.000 claims 8
- 101150103820 Fxn gene Proteins 0.000 claims 5
- 238000000034 method Methods 0.000 claims 4
- 210000000663 muscle cell Anatomy 0.000 claims 4
- 239000002131 composite material Substances 0.000 claims 3
- 125000003835 nucleoside group Chemical group 0.000 claims 3
- 239000002773 nucleotide Substances 0.000 claims 3
- 125000003729 nucleotide group Chemical group 0.000 claims 3
- 102000001706 Immunoglobulin Fab Fragments Human genes 0.000 claims 2
- 108010054477 Immunoglobulin Fab Fragments Proteins 0.000 claims 2
- 239000012634 fragment Substances 0.000 claims 2
- 239000002777 nucleoside Substances 0.000 claims 2
- 108010001017 CD71 antigen Proteins 0.000 claims 1
- 208000024412 Friedreich ataxia Diseases 0.000 claims 1
- 101000835093 Homo sapiens Transferrin receptor protein 1 Proteins 0.000 claims 1
- 102000017727 Immunoglobulin Variable Region Human genes 0.000 claims 1
- 108010067060 Immunoglobulin Variable Region Proteins 0.000 claims 1
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 claims 1
- 239000004472 Lysine Substances 0.000 claims 1
- 108091005804 Peptidases Proteins 0.000 claims 1
- 239000004365 Protease Substances 0.000 claims 1
- 102100037486 Reverse transcriptase/ribonuclease H Human genes 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 claims 1
- 108010033576 Transferrin Receptors Proteins 0.000 claims 1
- 102000014257 Transferrin receptor protein 1 Human genes 0.000 claims 1
- 102100026144 Transferrin receptor protein 1 Human genes 0.000 claims 1
- 108050003222 Transferrin receptor protein 1 Proteins 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 230000001668 ameliorated effect Effects 0.000 claims 1
- 230000000692 anti-sense effect Effects 0.000 claims 1
- 239000000074 antisense oligonucleotide Substances 0.000 claims 1
- 238000012230 antisense oligonucleotides Methods 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 230000021615 conjugation Effects 0.000 claims 1
- 125000000151 cysteine group Chemical group N[C@@H](CS)C(=O)* 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 230000001404 mediated effect Effects 0.000 claims 1
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862714035P | 2018-08-02 | 2018-08-02 | |
| US62/714,035 | 2018-08-02 | ||
| PCT/US2019/044959 WO2020028840A1 (en) | 2018-08-02 | 2019-08-02 | Muscle targeting complexes and uses thereof for treating friedreich's ataxia |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021532832A JP2021532832A (ja) | 2021-12-02 |
| JPWO2020028840A5 true JPWO2020028840A5 (enExample) | 2022-08-10 |
Family
ID=69232074
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021529256A Pending JP2021532832A (ja) | 2018-08-02 | 2019-08-02 | フリートライヒ運動失調症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 (9)
| Country | Link |
|---|---|
| US (1) | US20210308274A1 (enExample) |
| EP (1) | EP3830131A4 (enExample) |
| JP (1) | JP2021532832A (enExample) |
| KR (1) | KR20210086601A (enExample) |
| CN (1) | CN113166240A (enExample) |
| CA (1) | CA3108386A1 (enExample) |
| EA (1) | EA202190419A1 (enExample) |
| IL (1) | IL280535A (enExample) |
| WO (1) | WO2020028840A1 (enExample) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11202100934PA (en) | 2018-08-02 | 2021-02-25 | 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 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | 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 |
| 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 |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| CN118251238A (zh) * | 2021-06-21 | 2024-06-25 | 达因疗法公司 | 肌肉靶向复合物及其用于治疗弗里德赖希共济失调的用途 |
| 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 |
| 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 |
| MX2024000489A (es) | 2021-07-09 | 2024-04-09 | Dyne Therapeutics Inc | Complejos dirigidos al musculo y formulaciones para el tratamiento de las distrofinopatías. |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| KR20240107202A (ko) | 2021-09-01 | 2024-07-08 | 바이오젠 엠에이 인코포레이티드 | 항-트랜스페린 수용체 항체 및 이의 용도 |
| CA3255934A1 (en) | 2022-04-15 | 2023-10-19 | Dyne Therapeutics, Inc. | MUSCLE TARGETTING COMPLEXES AND FORMULATIONS FOR THE TREATMENT OF MYOTONIC DYSTROPHY |
| US20240182561A1 (en) * | 2022-11-04 | 2024-06-06 | Regeneron Pharmaceuticals, Inc. | Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015023939A1 (en) * | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Compositions and methods for modulating expression of frataxin |
| CA2921457A1 (en) * | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Heterochromatin forming non-coding rnas |
| JP6779876B2 (ja) * | 2014-11-19 | 2020-11-04 | ジェネンテック， インコーポレイテッド | 抗トランスフェリン受容体抗体及びその使用方法 |
| WO2016094374A1 (en) * | 2014-12-09 | 2016-06-16 | The Board Of Regents Of The University Of Texas System | Compositions and mentods for treatment of friedreich's ataxia |
| KR20210005994A (ko) * | 2015-11-05 | 2021-01-15 | 뱀부 테라퓨틱스 인코포레이티드 | 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터 |
| JP6823269B2 (ja) * | 2016-06-20 | 2021-02-03 | 株式会社ＧｅｎＡｈｅａｄ Ｂｉｏ | 抗体−薬物コンジュゲート |
| EP3829649A4 (en) * | 2018-08-02 | 2022-05-04 | Dyne Therapeutics, Inc. | ANTI-MUSCLE COMPLEXES AND THEIR USES |
-
2019
- 2019-08-02 WO PCT/US2019/044959 patent/WO2020028840A1/en not_active Ceased
- 2019-08-02 US US17/265,019 patent/US20210308274A1/en not_active Abandoned
- 2019-08-02 JP JP2021529256A patent/JP2021532832A/ja active Pending
- 2019-08-02 CA CA3108386A patent/CA3108386A1/en active Pending
- 2019-08-02 KR KR1020217006029A patent/KR20210086601A/ko not_active Withdrawn
- 2019-08-02 EA EA202190419A patent/EA202190419A1/ru unknown
- 2019-08-02 CN CN201980064588.8A patent/CN113166240A/zh active Pending
- 2019-08-02 EP EP19845433.2A patent/EP3830131A4/en active Pending
-
2021
- 2021-01-31 IL IL280535A patent/IL280535A/en unknown
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