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 (enExample) | 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 (enExample) |
| EP (1) | EP3697449A1 (enExample) |
| JP (1) | JP2021500071A (enExample) |
| AU (1) | AU2018350990A1 (enExample) |
| CA (1) | CA3079565A1 (enExample) |
| MX (1) | MX2020003945A (enExample) |
| WO (1) | WO2019079494A1 (enExample) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2018372235B9 (en) * | 2017-11-27 | 2022-03-10 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and use for inhibiting angiogenesis |
| KR20220009427A (ko) * | 2019-05-17 | 2022-01-24 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 글리코시드 가수분해효소를 사용하는 망막 세포에 대한 유전자 치료 벡터의 개선된 전달 |
| 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 |
| AU2020353719A1 (en) * | 2019-09-23 | 2022-03-10 | Celosia Therapeutics Pty Ltd | Treatment of tauopathies |
| WO2021113591A1 (en) * | 2019-12-06 | 2021-06-10 | Regeneron Pharmaceuticals, Inc. | Vegf mini-traps and methods of use thereof |
| JP2022548197A (ja) | 2019-12-06 | 2022-11-17 | 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 | 抗ｖｅｇｆタンパク質組成物及びその製造方法 |
| CN115803009A (zh) * | 2020-05-08 | 2023-03-14 | 瑞泽恩制药公司 | 用于治疗眼病和癌症的vegf阱和微阱及方法 |
| AU2021313839A1 (en) * | 2020-07-21 | 2023-03-23 | FTGEN Corp | Composition and method for treating eye diseases |
| MX2023002695A (es) * | 2020-09-03 | 2023-05-19 | Univ Massachusetts | Virus adeno-asociado para la administración de kh902 (conbercept) y usos del mismo. |
| AU2021362770A1 (en) * | 2020-10-16 | 2022-12-08 | Gyroscope Therapeutics Limited | Nucleic acid encoding an anti-VEGF entity and a negative complement regulator and uses thereof for the treatment of age-related macular degeneration |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| KR20230163462A (ko) * | 2021-03-31 | 2023-11-30 | 항저우 엑제제네시스 바이오 엘티디. | Vegf 및 안지오포이에틴을 표적화하는 융합 분자 및 이의 용도 |
| JP2024516128A (ja) * | 2021-04-09 | 2024-04-12 | アビラマックス・バイオファーマ・インコーポレイテッド | 眼での導入遺伝子発現のための組成物および方法 |
| CA3243256A1 (en) * | 2022-02-02 | 2023-08-10 | Adverum Biotechnologies Inc | Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept |
| AU2023250660A1 (en) * | 2022-04-06 | 2024-10-24 | Regenxbio Inc. | Pharmaceutical composition comprising a recombinant adenoassociated virus vector with an expression cassette encoding a transgene for suprachoroidal administration |
| 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 |
|---|---|---|---|---|
| PL310327A1 (en) | 1993-02-12 | 1995-12-11 | Univ Leland Stanford Junior | Adjustable transcription of target genes and other biological processes |
| CA2209183A1 (en) | 1994-12-29 | 1996-07-11 | Joel L. Pomerantz | Chimeric dna-binding proteins |
| CA2219080A1 (en) | 1995-06-07 | 1996-12-27 | Ariad Gene Therapeutics, Inc. | Rapamycin-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 |
| AU752129B2 (en) | 1997-08-27 | 2002-09-05 | Ariad Gene Therapeutics, Inc. | Chimeric transcriptional activators and compositions and uses related thereto |
| AU755784B2 (en) | 1998-01-15 | 2002-12-19 | Ariad Pharmaceuticals, Inc. | Regulation of biological events using multimeric chimeric proteins |
| CA2319492A1 (en) | 1998-02-13 | 1999-08-19 | President And Fellows Of Harvard College | Novel dimerizing agents, their production and use |
| US7087411B2 (en) * | 1999-06-08 | 2006-08-08 | Regeneron Pharmaceuticals, Inc. | Fusion protein capable of binding VEGF |
| DE60039766D1 (de) | 1999-08-09 | 2008-09-18 | Targeted Genetics Corp | Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| US7067526B1 (en) | 1999-08-24 | 2006-06-27 | Ariad Gene Therapeutics, Inc. | 28-epirapalogs |
| CA2915124C (en) | 2001-11-13 | 2018-08-14 | The Trustees Of The University Of Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2602352T3 (es) | 2001-12-17 | 2017-02-20 | The Trustees Of The University Of Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| EP2357010B1 (en) | 2005-04-07 | 2013-06-12 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| 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 |
| CN107828820B (zh) | 2010-10-27 | 2022-06-07 | 学校法人自治医科大学 | 用于向神经系统细胞导入基因的腺相关病毒粒子 |
| EP4234571A3 (en) | 2011-02-10 | 2023-09-27 | The University of North Carolina at Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| RS66184B1 (sr) | 2011-04-22 | 2024-12-31 | Univ California | Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu |
| 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 |
| EP2847337A4 (en) | 2012-05-09 | 2016-04-27 | Univ Oregon Health & Science | 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 |
| JP6552511B2 (ja) | 2013-10-11 | 2019-07-31 | マサチューセッツ アイ アンド イヤー インファーマリー | 祖先ウイルス配列を予測する方法およびその使用 |
| WO2015082690A1 (en) * | 2013-12-06 | 2015-06-11 | INSERM (Institut National de la Santé et de la Recherche Médicale) | 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 |
| HRP20201756T8 (hr) * | 2014-11-21 | 2021-08-20 | Bristol-Myers Squibb Company | Antitijela koja sadrže modificirane regije teškog lanca |
| CN108135994B (zh) * | 2015-05-13 | 2022-01-25 | 宾夕法尼亚州大学信托人 | Aav-介导的抗-流感抗体的表达及其使用方法 |
| CR20200476A (es) * | 2015-05-20 | 2020-12-02 | Dana Farber Cancer Inst Inc | ANTÍGENOS COMPARTIDOS (Divisional 2017-0584) |
| WO2017001990A1 (en) * | 2015-06-28 | 2017-01-05 | Allgenesis Biotherapeutics Inc. | Fusion proteins for inhibiting angiogenesis |
| MA44873A (fr) * | 2016-04-15 | 2019-03-13 | Univ Pennsylvania | Composition pour le traitement de la dégénérescence maculaire liée a l'âge exsudative |
| HUE051953T2 (hu) * | 2016-06-16 | 2021-04-28 | Adverum Biotechnologies Inc | Idõskori makula degeneráció kezelése AAV2 variáns és aflibercept alkalmazásával |
| US11192925B2 (en) | 2016-10-19 | 2021-12-07 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
| US20210130413A1 (en) * | 2017-02-28 | 2021-05-06 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
-
2018
- 2018-10-17 AU AU2018350990A patent/AU2018350990A1/en not_active Abandoned
- 2018-10-17 JP JP2020542539A patent/JP2021500071A/ja active Pending
- 2018-10-17 CA CA3079565A patent/CA3079565A1/en active Pending
- 2018-10-17 MX MX2020003945A patent/MX2020003945A/es unknown
- 2018-10-17 WO PCT/US2018/056343 patent/WO2019079494A1/en not_active Ceased
- 2018-10-17 EP EP18800376.8A patent/EP3697449A1/en not_active Withdrawn
-
2020
- 2020-03-05 US US16/810,422 patent/US20210010025A1/en not_active Abandoned
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