JPWO2022076556A5 - - Google Patents
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- JPWO2022076556A5 JPWO2022076556A5 JP2023521337A JP2023521337A JPWO2022076556A5 JP WO2022076556 A5 JPWO2022076556 A5 JP WO2022076556A5 JP 2023521337 A JP2023521337 A JP 2023521337A JP 2023521337 A JP2023521337 A JP 2023521337A JP WO2022076556 A5 JPWO2022076556 A5 JP WO2022076556A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- seq
- subject
- composition
- aav vector
- 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
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- 150000007523 nucleic acids Chemical class 0.000 claims description 110
- 108020004707 nucleic acids Proteins 0.000 claims description 106
- 102000039446 nucleic acids Human genes 0.000 claims description 106
- 239000013607 AAV vector Substances 0.000 claims description 74
- 108091029430 CpG site Proteins 0.000 claims description 48
- 101710087595 Ribitol 5-phosphate transferase FKRP Proteins 0.000 claims description 39
- 102100031774 Ribitol 5-phosphate transferase FKRP Human genes 0.000 claims description 39
- 239000008194 pharmaceutical composition Substances 0.000 claims description 37
- 239000013598 vector Substances 0.000 claims description 31
- 210000003205 muscle Anatomy 0.000 claims description 30
- 108091026890 Coding region Proteins 0.000 claims description 27
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 25
- 208000017081 Qualitative or quantitative defects of alpha-dystroglycan Diseases 0.000 claims description 20
- 208000035475 disorder Diseases 0.000 claims description 20
- 102400000310 Alpha-dystroglycan Human genes 0.000 claims description 16
- 102000004420 Creatine Kinase Human genes 0.000 claims description 15
- 108010042126 Creatine kinase Proteins 0.000 claims description 15
- 108010076504 Protein Sorting Signals Proteins 0.000 claims description 14
- 108010071885 Dystroglycans Proteins 0.000 claims description 10
- 101000846198 Homo sapiens Ribitol 5-phosphate transferase FKRP Proteins 0.000 claims description 10
- 230000013595 glycosylation Effects 0.000 claims description 10
- 238000006206 glycosylation reaction Methods 0.000 claims description 10
- 102000056880 human FKRP Human genes 0.000 claims description 10
- 210000002027 skeletal muscle Anatomy 0.000 claims description 9
- 241000701161 unidentified adenovirus Species 0.000 claims description 8
- 210000002966 serum Anatomy 0.000 claims description 7
- 101800000379 Alpha-dystroglycan Proteins 0.000 claims description 6
- 101150117564 ITR2 gene Proteins 0.000 claims description 6
- 239000003623 enhancer Substances 0.000 claims description 6
- 102000008186 Collagen Human genes 0.000 claims description 5
- 108010035532 Collagen Proteins 0.000 claims description 5
- 201000009342 Limb-girdle muscular dystrophy Diseases 0.000 claims description 5
- 229920001436 collagen Polymers 0.000 claims description 5
- 230000007170 pathology Effects 0.000 claims description 5
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims description 4
- 230000008021 deposition Effects 0.000 claims description 4
- 201000010099 disease Diseases 0.000 claims description 4
- 238000001802 infusion Methods 0.000 claims description 4
- 238000001990 intravenous administration Methods 0.000 claims description 4
- 210000004165 myocardium Anatomy 0.000 claims description 4
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 2
- 208000014644 Brain disease Diseases 0.000 claims description 2
- 108090000565 Capsid Proteins Proteins 0.000 claims description 2
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 208000032274 Encephalopathy Diseases 0.000 claims description 2
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims description 2
- 101001023021 Homo sapiens LIM domain-binding protein 3 Proteins 0.000 claims description 2
- 102100035112 LIM domain-binding protein 3 Human genes 0.000 claims description 2
- 102100022745 Laminin subunit alpha-2 Human genes 0.000 claims description 2
- 108010059343 MM Form Creatine Kinase Proteins 0.000 claims description 2
- 241001465754 Metazoa Species 0.000 claims description 2
- 102000016349 Myosin Light Chains Human genes 0.000 claims description 2
- 108010067385 Myosin Light Chains Proteins 0.000 claims description 2
- 208000020584 Polyploidy Diseases 0.000 claims description 2
- 201000006793 Walker-Warburg syndrome Diseases 0.000 claims description 2
- 230000005856 abnormality Effects 0.000 claims description 2
- 201000006815 congenital muscular dystrophy Diseases 0.000 claims description 2
- 230000002950 deficient Effects 0.000 claims description 2
- 238000012217 deletion Methods 0.000 claims description 2
- 230000037430 deletion Effects 0.000 claims description 2
- 238000000338 in vitro Methods 0.000 claims description 2
- 238000003780 insertion Methods 0.000 claims description 2
- 230000037431 insertion Effects 0.000 claims description 2
- 230000035772 mutation Effects 0.000 claims description 2
- 239000002773 nucleotide Substances 0.000 claims description 2
- 125000003729 nucleotide group Chemical group 0.000 claims description 2
- 229920001184 polypeptide Polymers 0.000 claims description 2
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 2
- 238000006467 substitution reaction Methods 0.000 claims description 2
- 230000009885 systemic effect Effects 0.000 claims description 2
- 238000012360 testing method Methods 0.000 claims description 2
- 230000009261 transgenic effect Effects 0.000 claims description 2
- 208000034373 type A muscular dystrophy-dystroglycanopathy Diseases 0.000 claims description 2
- 239000013603 viral vector Substances 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 28
- 238000000034 method Methods 0.000 description 26
- 241000699670 Mus sp. Species 0.000 description 4
- 208000020446 Cardiac disease Diseases 0.000 description 1
- 206010061818 Disease progression Diseases 0.000 description 1
- 208000019693 Lung disease Diseases 0.000 description 1
- 208000029549 Muscle injury Diseases 0.000 description 1
- 206010071759 Muscle oedema Diseases 0.000 description 1
- 208000027418 Wounds and injury Diseases 0.000 description 1
- 230000001154 acute effect Effects 0.000 description 1
- 239000000090 biomarker Substances 0.000 description 1
- 230000000747 cardiac effect Effects 0.000 description 1
- 230000001684 chronic effect Effects 0.000 description 1
- 230000006378 damage Effects 0.000 description 1
- 210000000188 diaphragm Anatomy 0.000 description 1
- 230000005750 disease progression Effects 0.000 description 1
- 238000011156 evaluation Methods 0.000 description 1
- 239000012634 fragment Substances 0.000 description 1
- 208000019622 heart disease Diseases 0.000 description 1
- 230000004217 heart function Effects 0.000 description 1
- 210000005003 heart tissue Anatomy 0.000 description 1
- 208000014674 injury Diseases 0.000 description 1
- 230000000366 juvenile effect Effects 0.000 description 1
- 210000003141 lower extremity Anatomy 0.000 description 1
- 238000002705 metabolomic analysis Methods 0.000 description 1
- 230000001431 metabolomic effect Effects 0.000 description 1
- 238000001964 muscle biopsy Methods 0.000 description 1
- 238000010606 normalization Methods 0.000 description 1
- 230000004202 respiratory function Effects 0.000 description 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063088757P | 2020-10-07 | 2020-10-07 | |
| US63/088,757 | 2020-10-07 | ||
| US202163214123P | 2021-06-23 | 2021-06-23 | |
| US63/214,123 | 2021-06-23 | ||
| US202163229726P | 2021-08-05 | 2021-08-05 | |
| US63/229,726 | 2021-08-05 | ||
| PCT/US2021/053768 WO2022076556A2 (en) | 2020-10-07 | 2021-10-06 | Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy disorders including limb girdle 2i (lgmd2i) |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023545731A JP2023545731A (ja) | 2023-10-31 |
| JP2023545731A5 JP2023545731A5 (https=) | 2024-10-15 |
| JPWO2022076556A5 true JPWO2022076556A5 (https=) | 2024-10-15 |
Family
ID=81127135
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023521337A Pending JP2023545731A (ja) | 2020-10-07 | 2021-10-06 | 肢帯型２ｉ（ｌｇｍｄ２ｉ）を含むジストログリカノパチー障害を処置するためのフクチン関連タンパク質（ｆｋｒｐ）の治療的アデノ随伴ウイルス送達 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20230374542A1 (https=) |
| EP (1) | EP4225382A4 (https=) |
| JP (1) | JP2023545731A (https=) |
| AU (1) | AU2021358957A1 (https=) |
| CA (1) | CA3195177A1 (https=) |
| IL (1) | IL301955A (https=) |
| WO (1) | WO2022076556A2 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2024524270A (ja) * | 2021-06-23 | 2024-07-05 | アスクバイオ・ユーケー・リミテッド | 調節性核酸配列 |
| US20250312398A1 (en) * | 2022-04-29 | 2025-10-09 | University Of Washington | Method for expressing a muscle-specific gene and cassettes for same |
| GB202215198D0 (en) * | 2022-10-14 | 2022-11-30 | Univ Sheffield | Gene therapy treatment |
| EP4665749A1 (en) * | 2023-02-13 | 2025-12-24 | Astrazeneca Ireland Limited | Nucleic acids encoding bcl2-associated athanogene 3 (bag3) for gene therapy |
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| EP3648783B1 (en) * | 2017-07-07 | 2024-09-04 | Genethon | Novel polynucleotides encoding a human fkrp protein |
| US20210228738A1 (en) | 2017-07-17 | 2021-07-29 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins |
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| CN114521143B (zh) * | 2019-09-19 | 2025-03-14 | 吉尼松公司 | 减轻fkrp心脏毒性的基因治疗表达系统 |
-
2021
- 2021-10-06 AU AU2021358957A patent/AU2021358957A1/en active Pending
- 2021-10-06 JP JP2023521337A patent/JP2023545731A/ja active Pending
- 2021-10-06 US US18/030,730 patent/US20230374542A1/en active Pending
- 2021-10-06 EP EP21878456.9A patent/EP4225382A4/en active Pending
- 2021-10-06 WO PCT/US2021/053768 patent/WO2022076556A2/en not_active Ceased
- 2021-10-06 CA CA3195177A patent/CA3195177A1/en active Pending
- 2021-10-06 IL IL301955A patent/IL301955A/en unknown
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