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 |
|---|---|---|---|---|
| CN111770999A (zh) * | 2017-11-27 | 2020-10-13 | 4D分子治疗有限公司 | 腺相关病毒变体衣壳和用于抑制血管生成的应用 |
| US20220233655A1 (en) * | 2019-05-17 | 2022-07-28 | Research Institute At Nationwide Children's Hospital | Improved delivery of gene therapy vectors to retinal cells using a glycoside hydrolase enzyme |
| 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 |
| CN114502197A (zh) * | 2019-08-26 | 2022-05-13 | 再生生物股份有限公司 | 用全人经翻译后修饰的抗VEGF Fab治疗糖尿病性视网膜病变 |
| WO2021056064A1 (en) * | 2019-09-23 | 2021-04-01 | Macquarie University | Treatment of tauopathies |
| IL293286A (en) * | 2019-12-06 | 2022-07-01 | Regeneron Pharma | vegf mini-traps and methods of using them |
| SG11202110968VA (en) | 2019-12-06 | 2021-10-28 | Regeneron Pharma | Anti-vegf protein compositions and methods for producing the same |
| AU2021268026A1 (en) * | 2020-05-08 | 2023-01-19 | Regeneron Pharmaceuticals, Inc. | VEGF traps and mini-traps and methods for treating ocular disorders and cancer |
| 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 |
| CA3192736A1 (en) * | 2020-09-03 | 2022-03-10 | University Of Massachusetts | Adeno-associated virus for delivery of kh902 (conbercept) and uses thereof |
| CR20230202A (es) * | 2020-10-16 | 2023-06-13 | Gyroscope Therapeutics Ltd | Ácido nucleico que codifica una entidad anti-vegf y un regulador del complemento negativo y sus usos para el tratamiento de la degeneración macular relacionada con la edad |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| AU2022251523A1 (en) * | 2021-03-31 | 2023-09-28 | Hangzhou Exegenesis Bio Ltd. | Fusion molecules targeting vegf and angiopoietin and uses thereof |
| BR112023020490A2 (pt) * | 2021-04-09 | 2023-11-21 | Avirmax Biopharma Inc | Composições e métodos para expressão de transgenes oculares |
| US20250137011A1 (en) * | 2022-02-02 | 2025-05-01 | Adverum Biotechnologies, Inc. | Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept |
| US20260034243A1 (en) * | 2022-04-06 | 2026-02-05 | Regenxbio Inc. | Pharmaceutical composition comprising a recombinant adeno-associated virus vector with an expression cassette encoding a transgene for suprachoroidal 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 |
| CN121772934A (zh) * | 2023-08-01 | 2026-03-31 | 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 |
|---|---|---|---|---|
| EP0804561B1 (en) | 1993-02-12 | 2009-12-30 | The Board Of Trustees Of The Leland Stanford Junior University | Regulated transcription of targeted genes and other biological events |
| AU719001B2 (en) | 1994-12-29 | 2000-05-04 | Massachusetts Institute Of Technology | Chimeric DNA-binding proteins |
| WO1996041865A1 (en) | 1995-06-07 | 1996-12-27 | Ariad Gene Therapeutics, Inc. | Rapamcycin-based regulation of biological events |
| JP2003524368A (ja) | 1997-08-26 | 2003-08-19 | アリアド ジーン セラピューティクス インコーポレイテッド | ニ量化ドメイン、三量化ドメインまたは四量化ドメインおよび補足的非相同転写活性化ドメイン、転写抑制ドメイン、ｄｎａ結合ドメインまたはリガンド結合ドメインを含む融合蛋白 |
| JP2001514007A (ja) | 1997-08-27 | 2001-09-11 | アリアド ジーン セラピューティクス インコーポレイテッド | キメラ転写アクチベーター、ならびにそれに関連する組成物および使用 |
| JP2002508971A (ja) | 1998-01-15 | 2002-03-26 | アリアド・ジーン・セラピューティクス・インコーポレーテッド | 多量体キメラ蛋白質を使用する生物学的イベントの調節 |
| JP2002503667A (ja) | 1998-02-13 | 2002-02-05 | プレジデント・アンド・フェローズ・オブ・ハーバード・カレッジ | 新規な二量体化剤、その製造および使用 |
| US7087411B2 (en) * | 1999-06-08 | 2006-08-08 | Regeneron Pharmaceuticals, Inc. | Fusion protein capable of binding VEGF |
| CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs |
| US7067526B1 (en) | 1999-08-24 | 2006-06-27 | Ariad Gene Therapeutics, Inc. | 28-epirapalogs |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| JP4495210B2 (ja) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | 振幅誤差補償装置及び直交度誤差補償装置 |
| EP2233500A1 (en) * | 2009-03-20 | 2010-09-29 | LFB Biotechnologies | Optimized Fc variants |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| 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 |
| WO2012057363A1 (ja) | 2010-10-27 | 2012-05-03 | 学校法人自治医科大学 | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| 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 |
| CN105755044A (zh) | 2011-04-22 | 2016-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| 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 |
| CA2905952A1 (en) | 2013-03-13 | 2014-10-02 | The Children's Hospital Of Philadelphia | Adeno-associated virus vectors and methods of use thereof |
| ES2739288T3 (es) | 2013-09-13 | 2020-01-30 | California Inst Of Techn | Recuperación selectiva |
| CN106232618A (zh) | 2013-10-11 | 2016-12-14 | 马萨诸塞眼科耳科诊所 | 预测祖先病毒序列的方法及其用途 |
| KR102281881B1 (ko) * | 2013-12-06 | 2021-07-27 | 엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔 (인쎄름) | 대상체의 망막 색소 상피에서 목적 폴리뉴클레오티드를 발현시키기 위한 방법 및 약학적 조성물 |
| 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 |
| IL294183B2 (en) * | 2015-05-20 | 2023-10-01 | Dana Farber Cancer Inst Inc | shared neoantigens |
| WO2017001990A1 (en) * | 2015-06-28 | 2017-01-05 | Allgenesis Biotherapeutics Inc. | Fusion proteins for inhibiting angiogenesis |
| SG11201808426XA (en) * | 2016-04-15 | 2018-10-30 | Univ Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| ES2840059T3 (es) * | 2016-06-16 | 2021-07-06 | Adverum Biotechnologies Inc | Tratamiento de la DMRE mediante el uso de la variante AAV2 con aflibercept |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| JP2020510424A (ja) * | 2017-02-28 | 2020-04-09 | 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 | 改変ａａｖカプシドおよびその使用 |
-
2018
- 2018-10-17 AU AU2018350990A patent/AU2018350990A1/en not_active Abandoned
- 2018-10-17 WO PCT/US2018/056343 patent/WO2019079494A1/en not_active Ceased
- 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 MX MX2020003945A patent/MX2020003945A/es unknown
- 2018-10-17 JP JP2020542539A patent/JP2021500071A/ja active Pending
-
2020
- 2020-03-05 US US16/810,422 patent/US20210010025A1/en not_active Abandoned
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