JPWO2020028841A5 - - Google Patents
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- Publication number
- JPWO2020028841A5 JPWO2020028841A5 JP2021529257A JP2021529257A JPWO2020028841A5 JP WO2020028841 A5 JPWO2020028841 A5 JP WO2020028841A5 JP 2021529257 A JP2021529257 A JP 2021529257A JP 2021529257 A JP2021529257 A JP 2021529257A JP WO2020028841 A5 JPWO2020028841 A5 JP WO2020028841A5
- Authority
- JP
- Japan
- Prior art keywords
- oligonucleotide
- transferrin receptor
- conjugate
- receptor antibody
- complex
- 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 15
- 108020003175 receptors Proteins 0.000 claims 9
- 102000005962 receptors Human genes 0.000 claims 9
- 239000012581 transferrin Substances 0.000 claims 9
- 102100039262 Glycogen [starch] synthase, muscle Human genes 0.000 claims 5
- 101710141660 Glycogen synthase 1 Proteins 0.000 claims 5
- 239000002773 nucleotide Substances 0.000 claims 5
- 125000003729 nucleotide group Chemical group 0.000 claims 5
- 238000000034 method Methods 0.000 claims 4
- 239000000203 mixture Substances 0.000 claims 3
- 210000000663 muscle cell Anatomy 0.000 claims 3
- 125000003835 nucleoside group Chemical group 0.000 claims 3
- 101000835093 Homo sapiens Transferrin receptor protein 1 Proteins 0.000 claims 2
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims 2
- 102000014257 Transferrin receptor protein 1 Human genes 0.000 claims 2
- 108050003222 Transferrin receptor protein 1 Proteins 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 239000012634 fragment Substances 0.000 claims 2
- 239000002777 nucleoside Substances 0.000 claims 2
- 108700028369 Alleles Proteins 0.000 claims 1
- 108010001017 CD71 antigen Proteins 0.000 claims 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 1
- 102000001706 Immunoglobulin Fab Fragments Human genes 0.000 claims 1
- 108010054477 Immunoglobulin Fab Fragments 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
- 102100026144 Transferrin receptor protein 1 Human genes 0.000 claims 1
- 239000002253 acid Substances 0.000 claims 1
- 108010028144 alpha-Glucosidases Proteins 0.000 claims 1
- 230000001668 ameliorated effect Effects 0.000 claims 1
- 239000000074 antisense oligonucleotide Substances 0.000 claims 1
- 238000012230 antisense oligonucleotides Methods 0.000 claims 1
- 239000002131 composite material Substances 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
- 208000035475 disorder Diseases 0.000 claims 1
- 201000004502 glycogen storage disease II Diseases 0.000 claims 1
- 230000001404 mediated effect Effects 0.000 claims 1
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025006544A JP2025071093A (ja) | 2018-08-02 | 2025-01-17 | ポンペ病を処置するための筋標的化複合体およびそれらの使用 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862713959P | 2018-08-02 | 2018-08-02 | |
| US62/713,959 | 2018-08-02 | ||
| PCT/US2019/044960 WO2020028841A1 (en) | 2018-08-02 | 2019-08-02 | Muscle targeting complexes and uses thereof for treating pompe disease |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025006544A Division JP2025071093A (ja) | 2018-08-02 | 2025-01-17 | ポンペ病を処置するための筋標的化複合体およびそれらの使用 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021533197A JP2021533197A (ja) | 2021-12-02 |
| JPWO2020028841A5 true JPWO2020028841A5 (enExample) | 2022-08-10 |
Family
ID=69230844
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021529257A Pending JP2021533197A (ja) | 2018-08-02 | 2019-08-02 | ポンペ病を処置するための筋標的化複合体およびそれらの使用 |
| JP2025006544A Pending JP2025071093A (ja) | 2018-08-02 | 2025-01-17 | ポンペ病を処置するための筋標的化複合体およびそれらの使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025006544A Pending JP2025071093A (ja) | 2018-08-02 | 2025-01-17 | ポンペ病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) | US20210317226A1 (enExample) |
| EP (1) | EP3830127A4 (enExample) |
| JP (2) | JP2021533197A (enExample) |
| KR (1) | KR20210054513A (enExample) |
| CN (2) | CN112912399A (enExample) |
| CA (1) | CA3108285A1 (enExample) |
| EA (1) | EA202190417A1 (enExample) |
| IL (1) | IL280483A (enExample) |
| WO (1) | WO2020028841A1 (enExample) |
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3635009A1 (en) | 2017-06-07 | 2020-04-15 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
| IL322464A (en) | 2018-02-07 | 2025-09-01 | Regeneron Pharma | Methods and compositions for administering therapeutic protein |
| EP3793591A1 (en) | 2018-05-17 | 2021-03-24 | Regeneron Pharmaceuticals, Inc. | Anti-cd63 antibodies, conjugates, and uses thereof |
| SG11202100934PA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| 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 |
| 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 |
| KR20210081324A (ko) | 2018-08-02 | 2021-07-01 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
| US20230330246A1 (en) * | 2020-05-05 | 2023-10-19 | Avidity Biosciences, Inc. | Compositions and methods of treating pompe disease |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11633498B2 (en) | 2021-07-09 | 2023-04-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11648318B2 (en) | 2021-07-09 | 2023-05-16 | Dyne Therapeutics, Inc. | Anti-transferrin receptor (TFR) antibody and uses thereof |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | 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 |
| CR20240139A (es) | 2021-09-01 | 2024-06-14 | Biogen Ma Inc | Anticuerpos del receptor antitransferrina y usos de los mismos |
| IL316143A (en) | 2022-04-15 | 2024-12-01 | Dyne Therapeutics Inc | Muscle targeting complexes and formulations for the treatment of myotonic dystrophy |
| IL318627A (en) * | 2022-07-29 | 2025-03-01 | Regeneron Pharma | Anti-TFR charge fusions and methods of using them |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2004094647A2 (en) * | 2003-04-18 | 2004-11-04 | Cytovia, Inc. | Methods of treating diseases responsive to induction of apoptosis and screening assays |
| TWI787678B (zh) * | 2014-05-23 | 2022-12-21 | 美商健臻公司 | 藉由使用反義寡核苷酸創造提前終止密碼子抑制或下調肝醣合成酶 |
| EA201891277A1 (ru) * | 2015-12-08 | 2018-11-30 | Ридженерон Фармасьютикалз, Инк. | Композиции и способы интернализации ферментов |
| US11060089B2 (en) * | 2016-04-18 | 2021-07-13 | Sarepta Therapeutics, Inc. | Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene |
| US20190240346A1 (en) * | 2016-06-20 | 2019-08-08 | Genahead Bio, Inc. | Antibody-drug conjugate |
-
2019
- 2019-08-02 CA CA3108285A patent/CA3108285A1/en active Pending
- 2019-08-02 WO PCT/US2019/044960 patent/WO2020028841A1/en not_active Ceased
- 2019-08-02 EP EP19844183.4A patent/EP3830127A4/en active Pending
- 2019-08-02 US US17/265,016 patent/US20210317226A1/en not_active Abandoned
- 2019-08-02 EA EA202190417A patent/EA202190417A1/ru unknown
- 2019-08-02 KR KR1020217005994A patent/KR20210054513A/ko active Pending
- 2019-08-02 CN CN201980064590.5A patent/CN112912399A/zh active Pending
- 2019-08-02 JP JP2021529257A patent/JP2021533197A/ja active Pending
- 2019-08-02 CN CN202410663435.8A patent/CN118662653A/zh active Pending
-
2021
- 2021-01-28 IL IL280483A patent/IL280483A/en unknown
-
2025
- 2025-01-17 JP JP2025006544A patent/JP2025071093A/ja active Pending
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