JP2021500071A5 - - Google Patents
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- JP2021500071A5 JP2021500071A5 JP2020542539A JP2020542539A JP2021500071A5 JP 2021500071 A5 JP2021500071 A5 JP 2021500071A5 JP 2020542539 A JP2020542539 A JP 2020542539A JP 2020542539 A JP2020542539 A JP 2020542539A JP 2021500071 A5 JP2021500071 A5 JP 2021500071A5
- Authority
- JP
- Japan
- Prior art keywords
- amino acid
- cells
- seq
- vegf
- trap
- 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
- 108010081667 aflibercept Proteins 0.000 claims description 45
- 210000004027 cell Anatomy 0.000 claims description 43
- 238000000034 method Methods 0.000 claims description 34
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 33
- 210000000234 capsid Anatomy 0.000 claims description 19
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 16
- 239000008194 pharmaceutical composition Substances 0.000 claims description 16
- 230000002207 retinal effect Effects 0.000 claims description 16
- 230000003612 virological effect Effects 0.000 claims description 16
- 241000702421 Dependoparvovirus Species 0.000 claims description 14
- 210000005229 liver cell Anatomy 0.000 claims description 14
- 206010028980 Neoplasm Diseases 0.000 claims description 13
- 201000011510 cancer Diseases 0.000 claims description 13
- 108700019146 Transgenes Proteins 0.000 claims description 12
- 239000002773 nucleotide Substances 0.000 claims description 12
- 125000003729 nucleotide group Chemical group 0.000 claims description 12
- 239000013604 expression vector Substances 0.000 claims description 11
- 230000001105 regulatory effect Effects 0.000 claims description 11
- 208000004644 retinal vein occlusion Diseases 0.000 claims description 11
- 206010052358 Colorectal cancer metastatic Diseases 0.000 claims description 10
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims description 10
- 239000013607 AAV vector Substances 0.000 claims description 9
- 108090000623 proteins and genes Proteins 0.000 claims description 9
- 239000013603 viral vector Substances 0.000 claims description 9
- 206010012689 Diabetic retinopathy Diseases 0.000 claims description 8
- 108090000565 Capsid Proteins Proteins 0.000 claims description 7
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 7
- 208000037062 Polyps Diseases 0.000 claims description 7
- 206010064930 age-related macular degeneration Diseases 0.000 claims description 7
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 7
- 210000005166 vasculature Anatomy 0.000 claims description 7
- 206010012688 Diabetic retinal oedema Diseases 0.000 claims description 6
- 208000001309 degenerative myopia Diseases 0.000 claims description 6
- 230000004340 degenerative myopia Effects 0.000 claims description 6
- 239000013598 vector Substances 0.000 claims description 5
- 238000012258 culturing Methods 0.000 claims description 4
- 210000001525 retina Anatomy 0.000 claims description 4
- 210000003994 retinal ganglion cell Anatomy 0.000 claims description 4
- FDJKUWYYUZCUJX-KVNVFURPSA-N N-glycolylneuraminic acid Chemical compound OC[C@H](O)[C@H](O)[C@@H]1O[C@](O)(C(O)=O)C[C@H](O)[C@H]1NC(=O)CO FDJKUWYYUZCUJX-KVNVFURPSA-N 0.000 claims description 3
- 101000718529 Saccharolobus solfataricus (strain ATCC 35092 / DSM 1617 / JCM 11322 / P2) Alpha-galactosidase Proteins 0.000 claims description 3
- NNISLDGFPWIBDF-MPRBLYSKSA-N alpha-D-Gal-(1->3)-beta-D-Gal-(1->4)-D-GlcNAc Chemical compound O[C@@H]1[C@@H](NC(=O)C)C(O)O[C@H](CO)[C@H]1O[C@H]1[C@H](O)[C@@H](O[C@@H]2[C@@H]([C@@H](O)[C@@H](O)[C@@H](CO)O2)O)[C@@H](O)[C@@H](CO)O1 NNISLDGFPWIBDF-MPRBLYSKSA-N 0.000 claims description 3
- 201000005667 central retinal vein occlusion Diseases 0.000 claims description 3
- 238000001990 intravenous administration Methods 0.000 claims description 3
- 210000004185 liver Anatomy 0.000 claims description 3
- 238000004519 manufacturing process Methods 0.000 claims description 3
- 208000003098 Ganglion Cysts Diseases 0.000 claims description 2
- 108091081062 Repeated sequence (DNA) Proteins 0.000 claims description 2
- 208000005400 Synovial Cyst Diseases 0.000 claims description 2
- 238000004113 cell culture Methods 0.000 claims description 2
- 150000004676 glycans Chemical class 0.000 claims description 2
- 210000002287 horizontal cell Anatomy 0.000 claims description 2
- 210000000608 photoreceptor cell Anatomy 0.000 claims description 2
- 102000004169 proteins and genes Human genes 0.000 claims description 2
- 210000002763 pyramidal cell Anatomy 0.000 claims description 2
- 230000010076 replication Effects 0.000 claims description 2
- 210000000844 retinal pigment epithelial cell Anatomy 0.000 claims description 2
- 241000701161 unidentified adenovirus Species 0.000 claims description 2
- 125000000539 amino acid group Chemical group 0.000 claims 9
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 6
- 201000010099 disease Diseases 0.000 claims 6
- 201000006165 Kuhnt-Junius degeneration Diseases 0.000 claims 5
- 208000000208 Wet Macular Degeneration Diseases 0.000 claims 5
- 238000006467 substitution reaction Methods 0.000 claims 5
- 201000011190 diabetic macular edema Diseases 0.000 claims 4
- 235000004279 alanine Nutrition 0.000 claims 3
- 239000000203 mixture Substances 0.000 claims 3
- QNAYBMKLOCPYGJ-REOHCLBHSA-N L-alanine Chemical group C[C@H](N)C(O)=O QNAYBMKLOCPYGJ-REOHCLBHSA-N 0.000 claims 2
- CIQHWLTYGMYQQR-QMMMGPOBSA-N O(4')-sulfo-L-tyrosine Chemical compound OC(=O)[C@@H](N)CC1=CC=C(OS(O)(=O)=O)C=C1 CIQHWLTYGMYQQR-QMMMGPOBSA-N 0.000 claims 2
- 241000700605 Viruses Species 0.000 claims 2
- 235000001014 amino acid Nutrition 0.000 claims 2
- 230000002491 angiogenic effect Effects 0.000 claims 2
- 210000001072 colon Anatomy 0.000 claims 2
- ZDXPYRJPNDTMRX-UHFFFAOYSA-N glutamine Chemical group OC(=O)C(N)CCC(N)=O ZDXPYRJPNDTMRX-UHFFFAOYSA-N 0.000 claims 2
- 125000000404 glutamine group Chemical group N[C@@H](CCC(N)=O)C(=O)* 0.000 claims 2
- 230000001394 metastastic effect Effects 0.000 claims 2
- 206010061289 metastatic neoplasm Diseases 0.000 claims 2
- 101100381481 Caenorhabditis elegans baz-2 gene Proteins 0.000 claims 1
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 1
- 206010021143 Hypoxia Diseases 0.000 claims 1
- 102100026120 IgG receptor FcRn large subunit p51 Human genes 0.000 claims 1
- 101710177940 IgG receptor FcRn large subunit p51 Proteins 0.000 claims 1
- 241000283973 Oryctolagus cuniculus Species 0.000 claims 1
- 101100372762 Rattus norvegicus Flt1 gene Proteins 0.000 claims 1
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 claims 1
- 125000003295 alanine group Chemical group N[C@@H](C)C(=O)* 0.000 claims 1
- 210000004899 c-terminal region Anatomy 0.000 claims 1
- 125000000151 cysteine group Chemical group N[C@@H](CS)C(=O)* 0.000 claims 1
- 230000007850 degeneration Effects 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 230000000938 luteal effect Effects 0.000 claims 1
- 238000004806 packaging method and process Methods 0.000 claims 1
- 235000018102 proteins Nutrition 0.000 claims 1
- 125000003607 serino group Chemical group [H]N([H])[C@]([H])(C(=O)[*])C(O[H])([H])[H] 0.000 claims 1
- 230000006107 tyrosine sulfation Effects 0.000 description 4
- 208000030533 eye disease Diseases 0.000 description 2
- 208000002780 macular degeneration Diseases 0.000 description 2
- 230000004048 modification Effects 0.000 description 2
- 238000012986 modification Methods 0.000 description 2
- 208000001491 myopia Diseases 0.000 description 2
- 230000004379 myopia Effects 0.000 description 2
- 230000001717 pathogenic effect Effects 0.000 description 2
- 238000011084 recovery Methods 0.000 description 2
- 210000005156 Müller Glia Anatomy 0.000 description 1
- 201000007737 Retinal degeneration Diseases 0.000 description 1
- 210000000411 amacrine cell Anatomy 0.000 description 1
- 208000035475 disorder Diseases 0.000 description 1
- 238000002955 isolation Methods 0.000 description 1
- 108020004707 nucleic acids Proteins 0.000 description 1
- 102000039446 nucleic acids Human genes 0.000 description 1
- 150000007523 nucleic acids Chemical class 0.000 description 1
- 230000004258 retinal degeneration Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762574038P | 2017-10-18 | 2017-10-18 | |
| US62/574,038 | 2017-10-18 | ||
| PCT/US2018/056343 WO2019079494A1 (en) | 2017-10-18 | 2018-10-17 | TREATMENT OF OCULAR DISEASES AND METASTATIC COLON CANCER WITH A VEGF TRAP WITH HUMAN POST-TRANSLATIONAL MODIFICATION |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021500071A JP2021500071A (ja) | 2021-01-07 |
| JP2021500071A5 true JP2021500071A5 (https=) | 2021-12-16 |
Family
ID=64267910
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020542539A Pending JP2021500071A (ja) | 2017-10-18 | 2018-10-17 | ヒト翻訳後修飾ｖｅｇｆ−ｔｒａｐによる眼疾患および転移性大腸がんの処置 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20210010025A1 (https=) |
| EP (1) | EP3697449A1 (https=) |
| JP (1) | JP2021500071A (https=) |
| AU (1) | AU2018350990A1 (https=) |
| CA (1) | CA3079565A1 (https=) |
| MX (1) | MX2020003945A (https=) |
| WO (1) | WO2019079494A1 (https=) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK3717636T5 (da) * | 2017-11-27 | 2024-08-26 | 4D Molecular Therapeutics Inc | Adeno-associeret-virus-variantcapsider og anvendelse til inhibering af angiogenese |
| JP2022533645A (ja) * | 2019-05-17 | 2022-07-25 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | グリコシドヒドロラーゼ酵素を使用する網膜細胞への遺伝子治療ベクターの送達の改善 |
| CN112342228B (zh) * | 2019-08-09 | 2023-07-21 | 上海朗昇生物科技有限公司 | 表达抗vegf融合蛋白的aav病毒载体及其应用 |
| WO2021041373A1 (en) * | 2019-08-26 | 2021-03-04 | Regenxbio Inc. | Treatment of diabetic retinopathy with fully-human post-translationally modified anti-vegf fab |
| CA3152239A1 (en) * | 2019-09-23 | 2021-04-01 | Lars Ittner | Treatment of tauopathies |
| US11180540B2 (en) | 2019-12-06 | 2021-11-23 | Regeneron Pharmaceuticals, Inc. | Anti-VEGF protein compositions and methods for producing the same |
| AU2020397053A1 (en) * | 2019-12-06 | 2022-06-23 | Regeneron Pharmaceuticals, Inc. | VEGF mini-traps and methods of use thereof |
| JP2023525034A (ja) * | 2020-05-08 | 2023-06-14 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | Ｖｅｇｆトラップおよびミニトラップならびに眼障害およびがんの治療方法 |
| JP7842082B2 (ja) * | 2020-07-21 | 2026-04-07 | エフティージェン コーポレーション | 眼疾患を処置するための組成物および方法 |
| BR112023003548A2 (pt) * | 2020-09-03 | 2023-04-04 | Univ Massachusetts | Vírus adenoassociado para liberação de kh902 (conbercept) e usos do mesmo |
| MX2023004377A (es) * | 2020-10-16 | 2023-06-28 | Gyroscope Therapeutics Ltd | Acido nucleico que codifica una entidad anti-vegf y un regulador del complemento negativo y sus usos para el tratamiento de la degeneracion macular relacionada con la edad. |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2022206838A1 (en) * | 2021-03-31 | 2022-10-06 | Hangzhou Jiayin Biotech Ltd. | Fusion molecules targeting vegf and angiopoietin and uses thereof |
| CA3210368A1 (en) * | 2021-04-09 | 2022-10-13 | Shengjiang Liu | Compositions and methods for ocular transgene expression |
| JP2025506383A (ja) * | 2022-02-02 | 2025-03-11 | アドヴェラム バイオテクノロジーズ， インコーポレイテッド | アフリベルセプトをコードするａａｖ２バリアントを使用して眼内血管新生疾患を処置する方法 |
| CA3247507A1 (en) * | 2022-04-06 | 2023-10-12 | Regenxbio Inc. | PHARMACEUTICAL COMPOSITION COMPRISING A RECOMMENDED ADENO-ASSOCIATED VIRUS (AAV) VECTOR WITH AN EXPRESSION CASSETTE ENCODED BY A TRANSGENE FOR SUPRACHOROID ADMINISTRATION |
| CN117304307A (zh) * | 2022-06-27 | 2023-12-29 | 上海鼎新基因科技有限公司 | 一种治疗新生血管相关眼底疾病的aav药物 |
| WO2025026182A1 (zh) * | 2023-08-01 | 2025-02-06 | 北京因诺惟康医药科技有限公司 | 融合多肽、包含其编码基因的表达盒、基因递送载体、药物组合物及用途 |
Family Cites Families (38)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CZ206195A3 (en) | 1993-02-12 | 1996-04-17 | Univ Leland Stanford Junior | Controlled transcription of target genes and other biological materials |
| ATE544776T1 (de) | 1994-12-29 | 2012-02-15 | Massachusetts Inst Technology | Chimäre dna-bindeproteine |
| KR19990022651A (ko) | 1995-06-07 | 1999-03-25 | 데이비드 엘. 버스테인 | 생물학적 사건에 대한 라파마이신 기재 조절방법 |
| US6117680A (en) | 1997-08-26 | 2000-09-12 | Ariad Gene Therapeutics, Inc. | Compositions and methods for regulation of transcription |
| CA2303482A1 (en) | 1997-08-27 | 1999-03-04 | Ariad Gene Therapeutics, Inc. | Chimeric transcriptional activators and compositions and uses related thereto |
| CA2318402A1 (en) | 1998-01-15 | 1999-07-22 | Ariad Gene Therapeutics, Inc. | Regulation of biological events using multimeric chimeric proteins |
| AU766513B2 (en) | 1998-02-13 | 2003-10-16 | Board Of Trustees Of The Leland Stanford Junior University | Novel dimerizing agents, their production and use |
| US7087411B2 (en) * | 1999-06-08 | 2006-08-08 | Regeneron Pharmaceuticals, Inc. | Fusion protein capable of binding VEGF |
| PT1916258E (pt) | 1999-08-09 | 2014-07-29 | Genzyme Corp | Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia |
| US7067526B1 (en) | 1999-08-24 | 2006-06-27 | Ariad Gene Therapeutics, Inc. | 28-epirapalogs |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| EP1777906A1 (en) | 2005-06-09 | 2007-04-25 | Matsushita Electric Industrial Co., Ltd. | Amplitude error compensating apparatus and orthogonality error compensating apparatus |
| EP2233500A1 (en) * | 2009-03-20 | 2010-09-29 | LFB Biotechnologies | Optimized Fc variants |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| US8628966B2 (en) | 2010-04-30 | 2014-01-14 | City Of Hope | CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| EP2634253B1 (en) | 2010-10-27 | 2016-05-11 | Jichi Medical University | Adeno-associated virus virions for transferring genes into neural cells |
| WO2012109570A1 (en) | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| HRP20171334T1 (hr) | 2011-04-22 | 2017-11-17 | The Regents Of The University Of California | Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe |
| ES2857773T5 (es) | 2011-08-24 | 2024-06-04 | Univ Leland Stanford Junior | Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos |
| CN103304668B (zh) * | 2012-03-12 | 2015-10-28 | 江苏健德生物药业有限公司 | Ultra-VEGF-trap免疫融合蛋白、其制备方法及其应用 |
| WO2013170078A1 (en) | 2012-05-09 | 2013-11-14 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| EP2970946A4 (en) | 2013-03-13 | 2016-09-07 | Philadelphia Children Hospital | ADENOASSOZED VIRUS VECTORS AND METHOD FOR USE THEREOF |
| US9585971B2 (en) | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| HUE052676T2 (hu) | 2013-10-11 | 2021-05-28 | Massachusetts Eye & Ear Infirmary | Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik |
| AU2014359136B2 (en) * | 2013-12-06 | 2020-06-25 | Centre Hospitalier Universitaire De Montpellier | Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject |
| WO2015164757A1 (en) | 2014-04-25 | 2015-10-29 | Oregon Health & Science University | Methods of viral neutralizing antibody epitope mapping |
| JP6668345B2 (ja) * | 2014-11-21 | 2020-03-18 | ブリストル−マイヤーズ スクイブ カンパニーＢｒｉｓｔｏｌ−Ｍｙｅｒｓ Ｓｑｕｉｂｂ Ｃｏｍｐａｎｙ | 修飾された重鎖定常領域を含む抗体 |
| EA201792500A1 (ru) * | 2015-05-13 | 2018-04-30 | Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания | Aav-опосредованная экспрессия антител против гриппа и способы их использования |
| TW202523682A (zh) * | 2015-05-20 | 2025-06-16 | 美商博德研究所有限公司 | 共有之gata3相關之腫瘤特異性新抗原 |
| ES2797901T3 (es) * | 2015-06-28 | 2020-12-04 | Allgenesis Biotherapeutics Inc | Proteínas de fusión para la inhibición de angiogénesis |
| JP7046828B2 (ja) * | 2016-04-15 | 2022-04-04 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 滲出型加齢性黄斑変性の治療のための組成物 |
| DK3471780T3 (da) * | 2016-06-16 | 2020-11-23 | Adverum Biotechnologies Inc | Behandling af amd med aav2-variant med aflibercept |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| AU2018227483A1 (en) * | 2017-02-28 | 2019-09-12 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
-
2018
- 2018-10-17 AU AU2018350990A patent/AU2018350990A1/en not_active Abandoned
- 2018-10-17 MX MX2020003945A patent/MX2020003945A/es unknown
- 2018-10-17 CA CA3079565A patent/CA3079565A1/en active Pending
- 2018-10-17 EP EP18800376.8A patent/EP3697449A1/en not_active Withdrawn
- 2018-10-17 JP JP2020542539A patent/JP2021500071A/ja active Pending
- 2018-10-17 WO PCT/US2018/056343 patent/WO2019079494A1/en not_active Ceased
-
2020
- 2020-03-05 US US16/810,422 patent/US20210010025A1/en not_active Abandoned
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