TWI702955B - 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) - Google Patents

使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) Download PDF

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TWI702955B
TWI702955B TW102116146A TW102116146A TWI702955B TW I702955 B TWI702955 B TW I702955B TW 102116146 A TW102116146 A TW 102116146A TW 102116146 A TW102116146 A TW 102116146A TW I702955 B TWI702955 B TW I702955B
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sflt
aspects
sequence
vegf
raav
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TW201408307A (zh
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愛恩J 康斯坦堡
伊莉薩貝斯 瑞可西,P
裘伊 梅 賴
湯瑪士W 二世 洽爾貝格
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澳大利亞商艾佛蘭屈澳洲私營有限公司
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TW102116146A 2012-05-15 2013-05-06 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) TWI702955B (zh)

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Application Number Priority Date Filing Date Title
US201261647461P 2012-05-15 2012-05-15
US61/647,461 2012-05-15
US201261670535P 2012-07-11 2012-07-11
US61/670,535 2012-07-11
US201261678555P 2012-08-01 2012-08-01
US61/678,555 2012-08-01
US201261691660P 2012-08-21 2012-08-21
US61/691,660 2012-08-21
US201361775440P 2013-03-08 2013-03-08
US61/775,440 2013-03-08

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TWI702955B true TWI702955B (zh) 2020-09-01

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TW109119818A TWI775096B (zh) 2012-05-15 2013-05-06 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
TW107100972A TWI698240B (zh) 2012-05-15 2013-05-06 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
TW102116146A TWI702955B (zh) 2012-05-15 2013-05-06 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)

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TW107100972A TWI698240B (zh) 2012-05-15 2013-05-06 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)

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Publication number Priority date Publication date Assignee Title
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9730790B2 (en) 2009-09-29 2017-08-15 Edwards Lifesciences Cardiaq Llc Replacement valve and method
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
LT3254703T (lt) 2011-04-22 2020-05-25 The Regents Of The University Of California Adenoasocijuoto viruso virionai su variantine kapside ir jų panaudojimo būdai
TWI775096B (zh) 2012-05-15 2022-08-21 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
US9681951B2 (en) 2013-03-14 2017-06-20 Edwards Lifesciences Cardiaq Llc Prosthesis with outer skirt and anchors
WO2014194132A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
KR101591823B1 (ko) 2013-12-27 2016-02-04 재단법인 목암생명공학연구소 증가된 유전자 발현능을 갖는 발현벡터
RU2703145C2 (ru) * 2014-02-06 2019-10-15 Джензим Корпорейшн Композиции и способы лечения и предотвращения дегенерации желтого пятна
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
CA2942776C (en) 2014-03-17 2023-01-24 Adverum Biotechnologies, Inc. Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter
RS58337B1 (sr) * 2014-03-24 2019-03-29 Translate Bio Inc Irnk terapija za lečenje očnih oboljenja
WO2015153889A2 (en) * 2014-04-02 2015-10-08 University Of Florida Research Foundation, Incorporated Materials and methods for the treatment of latent viral infection
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
US9840553B2 (en) 2014-06-28 2017-12-12 Kodiak Sciences Inc. Dual PDGF/VEGF antagonists
KR20170096998A (ko) 2014-11-05 2017-08-25 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 aadc 폴리뉴클레오티드
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2016077689A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
GB201502137D0 (en) 2015-02-09 2015-03-25 Ucl Business Plc Treatment
KR20170137730A (ko) 2015-03-02 2017-12-13 애드베룸 바이오테크놀로지스, 인코포레이티드 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법
HRP20201225T1 (hr) * 2015-03-06 2020-11-13 Massachusetts Eye & Ear Infirmary Terapije poboljšavanja gena kod nasljedne degeneracije mrežnice uzrokovanu mutacijama u genu prpf31
WO2016154344A1 (en) 2015-03-24 2016-09-29 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
US11104917B2 (en) * 2015-05-08 2021-08-31 Children's Medical Research Institute Promoters for expression of heterologous genes
US11103597B2 (en) 2015-05-14 2021-08-31 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers outside ITR
US10016514B2 (en) * 2015-05-15 2018-07-10 New Hope Research Foundation Polynucleotides, vectors and methods for insertion and expression of transgenes
WO2017066579A1 (en) 2015-10-14 2017-04-20 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
EP3368054A4 (en) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. REGULATORY EXPRESSION USING THE ADENO-ASSOCIATED VIRUS (AAV)
GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
ES2761328T3 (es) * 2015-12-03 2020-05-19 Friedrich Miescher Institute For Biomedical Res SynP161, un promotor para la expresión específica de genes en fotorreceptores de bastón
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
KR20250057128A (ko) 2015-12-30 2025-04-28 코디악 사이언시스 인코포레이티드 항체 및 이의 접합체
KR20190003556A (ko) * 2016-04-15 2019-01-09 리젠엑스바이오 인크. 완전히-인간형의 번역후 변형된 항-VEGF Fab를 이용한 눈 질환의 치료
WO2017180936A1 (en) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
MX2018013352A (es) * 2016-05-03 2019-08-01 Univ Wayne State Metodo para mejorar suministro de genes mediado por virus en el ojo usando inhibidores de proteosoma.
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics Inc composições e métodos de tratamento da doença de huntington
DK3472317T3 (da) * 2016-06-16 2022-05-23 Adverum Biotechnologies Inc Sammensætninger og fremgangsmåder til reduktion af okulær vaskularisation
PT3795181T (pt) 2016-06-16 2025-11-05 Adverum Biotechnologies Inc Tratamento de dmi com utilização da variante aav2 com aflibercept
KR20230039779A (ko) 2016-07-29 2023-03-21 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법
JP2019531787A (ja) 2016-08-30 2019-11-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
EP3528785A4 (en) 2016-10-19 2020-12-02 Adverum Biotechnologies, Inc. MODIFIED AAV CASPIDS AND USES THEREOF
CN107058315B (zh) * 2016-12-08 2019-11-08 上海优卡迪生物医药科技有限公司 敲减人PD-1的siRNA、重组表达CAR-T载体及其构建方法和应用
MX2019007876A (es) * 2016-12-30 2019-10-15 Univ Pennsylvania Terapia genetica para tratar fenilcetonuria.
AU2018234933B2 (en) * 2017-03-16 2024-03-21 Lineage Cell Therapeutics, Inc. Methods for measuring therapeutic effects of retinal disease therapies
KR102616820B1 (ko) 2017-03-17 2023-12-21 애드베룸 바이오테크놀로지스, 인코포레이티드 향상된 유전자 발현을 위한 조성물 및 방법
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN110461368A (zh) 2017-06-30 2019-11-15 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
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
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3676373A4 (en) 2017-08-28 2021-06-09 The Regents of The University of California Adeno-associated virus capsid variants and methods of use thereof
WO2019067540A1 (en) * 2017-09-27 2019-04-04 Regenxbio Inc. TREATMENT OF OCULAR DISEASES WITH A TOTALLY HUMAN POST-TRANSLATIONAL MODIFICATION ANTI-VEGF FAB
CA3075656A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
KR102205830B1 (ko) * 2017-10-26 2021-01-21 주식회사 큐로진생명과학 솔루블 VEGFR-1 변이체 cDNA를 함유하는 rAAV를 포함하는 황반변성 치료용 조성물
MX2020006435A (es) * 2017-12-19 2021-02-09 Akouos Inc Administracion de anticuerpos terapeuticos mediada por vector de virus adenoasociado (aav) en el oido interno.
US10610606B2 (en) * 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
US20210093734A1 (en) * 2018-02-20 2021-04-01 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-realted macular degeneration
MX2020009152A (es) 2018-03-02 2020-11-09 Kodiak Sciences Inc Anticuerpos de il-6 y constructos de fusion y conjugados de los mismos.
US20210010028A1 (en) 2018-03-06 2021-01-14 Voyager Therapeutics, Inc. Insect cell manufactured partial self-complementary aav genomes
CA3098871A1 (en) * 2018-05-08 2019-11-14 Rutgers, The State University Of New Jersey Aav-compatible laminin-linker polymerization proteins
KR20210021299A (ko) 2018-05-10 2021-02-25 리제너론 파아마슈티컬스, 인크. 고농도 vegf 수용체 융합 단백질 함유 제제
JP2021523914A (ja) 2018-05-15 2021-09-09 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. パーキンソン病を治療するための組成物および方法
EP3793686A1 (en) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Aav serotypes for brain specific payload delivery
US20210214749A1 (en) 2018-05-16 2021-07-15 Voyager Therapeutics, Inc. Directed evolution
US20210189424A1 (en) * 2018-06-11 2021-06-24 Gensight Biologics Sa Recombinant aav vectors and methods of using the same
JP7565218B2 (ja) 2018-07-02 2024-10-10 ボイジャー セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症および脊髄に関連する障害の治療
US20210355454A1 (en) 2018-07-24 2021-11-18 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
JP7528066B2 (ja) 2018-09-28 2024-08-05 ボイジャー セラピューティクス インコーポレイテッド 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
EP3886809A4 (en) 2018-11-28 2022-03-30 Genascence Corporation METHODS AND COMPOSITIONS FOR THE TREATMENT OF OSTEOARTHRITIS
WO2020136384A1 (en) * 2018-12-28 2020-07-02 Constable Ian Jeffery Methods of retinal administration
US20220064671A1 (en) 2019-01-18 2022-03-03 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
JP7597380B2 (ja) 2019-03-04 2024-12-10 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 対側眼へのaav遺伝子治療の逐次的硝子体内投与
TW202532428A (zh) 2019-04-26 2025-08-16 美商愛德維仁生物科技公司 用於玻璃體內遞送之變異體aav蛋白殼
EP3962536A1 (en) 2019-04-29 2022-03-09 Voyager Therapeutics, Inc. Systems and methods for producing baculoviral infected insect cells (biics) in bioreactors
WO2021030125A1 (en) 2019-08-09 2021-02-18 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
TW202122582A (zh) 2019-08-26 2021-06-16 美商航海家醫療公司 病毒蛋白之控制表現
CN114555814A (zh) * 2019-09-13 2022-05-27 罗特格斯新泽西州立大学 Aav相容的层粘连蛋白-连接子聚合蛋白
CA3151700A1 (en) * 2019-09-20 2021-03-25 Girish Chitnis Injection systems and methods of their use
CN112552410A (zh) * 2019-09-26 2021-03-26 三生国健药业(上海)股份有限公司 一种抗体融合蛋白及其制法和在抗肿瘤中的应用
CN114786731A (zh) 2019-10-10 2022-07-22 科达制药股份有限公司 治疗眼部病症的方法
AU2020396699B2 (en) * 2019-12-04 2024-07-18 Cdmogen Co., Ltd Composition for treating diabetic retinopathy, comprising rAAV containing soluble VEGFR-1 variant cDNA
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
CN113952473A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
WO2022032153A1 (en) 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
KR102775781B1 (ko) * 2020-10-13 2025-03-06 재단법인 아산사회복지재단 Acta2 억제제를 유효성분으로 포함하는 망막 또는 맥락막 질환 치료용 조성물
CR20230228A (es) 2020-12-01 2023-07-18 Akouos Inc Construcciones de anticuerpos anti-vegf y métodos relacionados para el tratamiento de los síntomas asociados al schwannoma vestibular
US20220267795A1 (en) * 2021-02-24 2022-08-25 Kinase Pharma Inc. Compositions and methods for regulating production of an angiogensis inhibitor
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US20240141377A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
AU2022281825A1 (en) 2021-05-28 2024-01-18 Shanghai Regenelead Therapies Co., Ltd. Recombinant adeno-associated virus having variant capsid, and application thereof
WO2023206134A1 (en) * 2022-04-27 2023-11-02 Beijing Sightnovo Medical Technology Co., Ltd Compositions and methods for eye diseases
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2025019458A2 (en) * 2023-07-14 2025-01-23 The Trustees Of Columbia University In The City Of New York Specific blood retinal barrier defect in glaucoma directs new treatments and clinical tests
CN117925664B (zh) 2023-11-29 2025-01-21 上海科锐克医药科技有限公司 用于递送人vegf受体融合蛋白的基因递送载体及其应用
WO2025188755A1 (en) * 2024-03-04 2025-09-12 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
CN118879705A (zh) * 2024-06-05 2024-11-01 上海凌医生物科技有限公司 一种诱导型启动子

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999014354A1 (en) * 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
US20020194630A1 (en) * 1999-03-15 2002-12-19 Manning William C. Use of recombinant gene delivery vectors for treating or preventing diseases of the eye

Family Cites Families (119)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
US4874237A (en) 1987-05-07 1989-10-17 Lions Eye Inst. Of Western Australia Electroretinogram apparatus
CA2002839C (en) 1988-11-14 2000-10-10 Neal S. Young Parvovirus capsids
FR2643252B1 (fr) 1989-02-21 1991-06-07 Technomed Int Sa Appareil de destruction selective de cellules incluant les tissus mous et les os a l'interieur du corps d'un etre vivant par implosion de bulles de gaz
US5112946A (en) 1989-07-06 1992-05-12 Repligen Corporation Modified pf4 compositions and methods of use
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6379885B1 (en) 1989-09-14 2002-04-30 Rijksuniversiteit Te Leiden Human parvovirus B19 proteins and virus-like particles, their production and their use in diagnostic assays and vaccines
CA2096222C (en) 1990-11-13 1998-12-29 Stephen D. Lupton Bifunctional selectable fusion genes
US5383917A (en) 1991-07-05 1995-01-24 Jawahar M. Desai Device and method for multi-phase radio-frequency ablation
ATE281469T1 (de) 1993-03-25 2004-11-15 Merck & Co Inc Inhibitor des wachstumsfaktors für gefäss- endothelzellen
AU6953394A (en) 1993-05-21 1994-12-20 Targeted Genetics Corporation Bifunctional selectable fusion genes based on the cytosine deaminase (cd) gene
US5814618A (en) 1993-06-14 1998-09-29 Basf Aktiengesellschaft Methods for regulating gene expression
WO1995022618A1 (en) 1994-02-22 1995-08-24 Dana-Farber Cancer Institute Nucleic acid delivery system, method of synthesis and uses thereof
US6551618B2 (en) 1994-03-15 2003-04-22 University Of Birmingham Compositions and methods for delivery of agents for neuronal regeneration and survival
FR2718150B1 (fr) 1994-03-29 1996-04-26 Rhone Poulenc Rorer Sa Virus recombinants, préparation et utilisation en thérapie génique.
US5639725A (en) 1994-04-26 1997-06-17 Children's Hospital Medical Center Corp. Angiostatin protein
US5527533A (en) * 1994-10-27 1996-06-18 Board Of Trustees Of The University Of Illinois Method of retarding and ameliorating central nervous system and eye damage
US20020168342A1 (en) 1994-11-03 2002-11-14 Cell Genesys, Inc. Novel adenoviral vectors, packaging cell lines, recombinant adenoviruses and methods
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US5641749A (en) 1995-11-29 1997-06-24 Amgen Inc. Method for treating retinal ganglion cell injury using glial cell line-derived neurothrophic factor (GDNF) protein product
WO1998007435A1 (en) * 1996-08-20 1998-02-26 The Regents Of The University Of California Eye treatments using synthetic thyroid hormone compositions
CA2266419A1 (en) 1996-09-24 1998-04-02 Merck & Co., Inc. Gene therapy for inhibition of angiogenesis
FR2756297B1 (fr) 1996-11-22 1999-01-08 Centre Nat Rech Scient Procede de production de virus recombinants
US6153436A (en) 1997-01-10 2000-11-28 The Board Of Trustees Of The University Of Arkansas Method of gene delivery using wildtype adeno associated viral (AAV) vectors with insertions
AU728220B2 (en) 1997-04-14 2001-01-04 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
AU7383298A (en) 1997-05-13 1998-12-08 Regents Of The University Of California, The Novel antiangiogenic peptide agents and their therapeutic and diagnostic use
US6458157B1 (en) * 1997-08-04 2002-10-01 Suaning Gregg Joergen Retinal stimulator
JPH11100327A (ja) 1997-09-26 1999-04-13 Toray Ind Inc 網膜変性症治療剤
AU9673998A (en) 1997-10-01 1999-04-23 G.D. Searle & Co. Fusion proteins comprising an angiostatin moiety and their use in anti-tumor treatment
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
EP1045921A2 (en) 1998-01-16 2000-10-25 Chiron Corporation Feline immunodeficiency virus gene therapy vectors
EP1061943B1 (en) 1998-03-12 2002-10-23 Genentech, Inc. Use of fgf-5 polypeptides for preventing the death of retinal neurons and treating ocular diseases
US6593133B1 (en) 1998-07-06 2003-07-15 Nsgene A/S Neurotrophic factors
JP2002528056A (ja) * 1998-08-28 2002-09-03 デューク・ユニバーシティー IVa2、100K及び/又は末端前タンパク質配列における欠失型アデノウイルス
NZ511037A (en) 1998-09-17 2005-02-25 Univ Florida Methods for treatment of degenerative retinal diseases
US20040234505A1 (en) 1998-09-23 2004-11-25 Stuart Naylor Polynucleotide constructs and uses thereof
WO2000054813A2 (en) * 1999-03-15 2000-09-21 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
US7070959B1 (en) * 1999-06-08 2006-07-04 Regeneron Pharmaceuticals, Inc. Modified chimeric polypeptides with improved pharmacokinetic properties
US7306799B2 (en) * 1999-06-08 2007-12-11 Regeneron Pharmaceuticals, Inc. Use of VEGF inhibitors for treatment of eye disorders
US7087411B2 (en) 1999-06-08 2006-08-08 Regeneron Pharmaceuticals, Inc. Fusion protein capable of binding VEGF
CA2387127A1 (en) 1999-10-25 2001-05-17 Therus Corporation Use of focused ultrasound for vascular sealing
WO2001078586A1 (en) 2000-04-17 2001-10-25 Stuart Graham Method and apparatus for objective electrophysiological assessment of visual function
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
AU2002236988A1 (en) * 2001-02-06 2002-08-19 Novartis Ag Photodynamic therapy of occult choroidal neovascularization linked to age-related macular degeneration
US20040102765A1 (en) 2001-03-27 2004-05-27 Karsten Koenig Method for the minimal-to non-invase optical treatment of tissues of the eye and for diagnosis thereof and device for carrying out said method
CA2442670A1 (en) 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
JP2002363107A (ja) 2001-06-04 2002-12-18 Noriyuki Azuma 色覚不全動物の色覚復元方法
EP1412493B1 (en) 2001-08-02 2011-10-05 Institut Clayton De La Recherche Methods and compositions relating to improved lentiviral vector production systems
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
EP1490113A4 (en) 2002-03-20 2007-05-02 Univ Florida RAAV VECTOR COMPOSITIONS AND METHOD FOR TREATING CHOROIDAL NEOVASCULARIZATION
AU2003223775A1 (en) 2002-04-30 2003-11-17 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
US8304233B2 (en) 2002-06-04 2012-11-06 Poetic Genetics, Llc Methods of unidirectional, site-specific integration into a genome, compositions and kits for practicing the same
US7220577B2 (en) 2002-08-28 2007-05-22 University Of Florida Research Foundation, Inc. Modified AAV
GB0220467D0 (en) 2002-09-03 2002-10-09 Oxford Biomedica Ltd Composition
WO2004079332A2 (en) 2003-03-04 2004-09-16 National Institute Of Advanced Industrial Science And Technology Composition and method for increasing efficiency of introduction of target substance into cell
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
DE602005021811D1 (de) 2004-09-13 2010-07-22 Genzyme Corp Multimere konstrukte
EP2324848A3 (en) * 2004-10-21 2011-09-14 Genentech, Inc. Method for treating intraocular neovascular diseases
SI1809750T1 (sl) 2004-11-08 2012-08-31 Chromagenics Bv Izbira gostiteljskih celic, ki imajo visok nivo izraĹľanja proteina
US7922670B2 (en) 2005-02-24 2011-04-12 Warren Jones System and method for quantifying and mapping visual salience
EP3409296A1 (en) 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
US20060234347A1 (en) * 2005-04-13 2006-10-19 Harding Thomas C Targeting multiple angiogenic pathways for cancer therapy using soluble tyrosine kinase receptors
PL1945779T3 (pl) 2005-10-20 2013-08-30 Uniqure Ip Bv Ulepszone wektory AAV produkowane w komórkach owadzich
WO2007084773A2 (en) 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
US20070190028A1 (en) 2006-02-13 2007-08-16 Jihong Qu Method and apparatus for heat or electromagnetic control of gene expression
US8118752B2 (en) 2006-02-16 2012-02-21 The Board Of Trustees Of The University Of Illinois Apparatus and methods for mapping retinal function
US7384145B2 (en) 2006-02-16 2008-06-10 The Board Of Trustees Of The University Of Illinois Mapping retinal function using corneal electrode array
US8216575B2 (en) 2006-03-31 2012-07-10 Chengdu Kanghong Biotechnologies Co., Ltd. Inhibition of neovascularization with a soluble chimeric protein comprising VEGF FLT-1 and KDR domains
CA2651194A1 (en) * 2006-05-04 2007-11-15 Fondation Ophthalmologique Aldolphe De Rothschild Methods for treating neovascular diseases
EP2029746B1 (en) * 2006-06-12 2012-07-04 Exegenics, Inc., D/b/a Opko Health, Inc. Compositions and methods for sirna inhibition of angiogenesis
GB0612096D0 (en) 2006-06-19 2006-07-26 Greater Glasgow Nhs Board Functional imaging of the retina
CA2655957C (en) 2006-06-21 2016-05-03 Amsterdam Molecular Therapeutics (Amt) B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
CN101313366A (zh) 2006-06-27 2008-11-26 株式会社爱德万测试 半导体试验装置以及半导体存储器的试验方法
EP2061891B1 (en) 2006-08-24 2012-04-11 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
WO2008098310A1 (en) 2007-02-16 2008-08-21 Objectivision Limited Stimulus method for multifocal visual evoked potential
EP1995309A1 (en) 2007-05-21 2008-11-26 Vivalis Recombinant protein production in avian EBx® cells
WO2008150459A1 (en) 2007-05-30 2008-12-11 The Trustees Of The University Of Pennsylvania A method for transducing cells with primary cilia
US8540369B2 (en) 2007-08-16 2013-09-24 The Research Foundation Of State University Of New York Led variable light source
US8518037B2 (en) 2007-10-30 2013-08-27 Boston Scientific Scimed, Inc. Radiofrequency ablation device
WO2009073551A2 (en) 2007-11-30 2009-06-11 Scarab Genomics Llc Lac expression system
CN102007209B (zh) 2008-02-19 2013-11-13 阿姆斯特丹分子治疗(Amt)股份有限公司 细小病毒rep和cap蛋白在昆虫细胞中的表达优化
CN101951925A (zh) 2008-02-20 2011-01-19 建新公司 血管发生抑制
US20100081707A1 (en) * 2008-02-21 2010-04-01 Ali Robin R Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
ITFI20080081A1 (it) 2008-04-18 2009-10-19 Strumenti Oftalmici C S O S R Procedimento e sistema per la registrazione di risposte elettrofunzionali erg, perg e vep multifocali in tempo reale
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
ES2330826B1 (es) 2008-06-04 2010-07-26 Proyecto De Biomedicina Cima, S.L. Sistema para empaquetamiento de adenovirus de alta capacidad.
EP2403867B1 (en) 2009-03-04 2019-05-22 Deutsches Krebsforschungszentrum Assembly activating protein (aap) and its use for the manufacture of parvovirus particles essential consisting of vp3
WO2010114948A2 (en) 2009-04-02 2010-10-07 University Of Florida Research Foundation, Inc. An inducible system for highly efficient production of recombinant adeno-associated virus (raav) vectors
TWI466158B (zh) 2009-07-03 2014-12-21 Univ Lunghwa Sci & Technology 電漿測量裝置、電漿系統及測量電漿特性之方法
EP2287323A1 (en) * 2009-07-31 2011-02-23 Association Institut de Myologie Widespread gene delivery to the retina using systemic administration of AAV vectors
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
US20120172419A1 (en) 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
US20110270256A1 (en) 2009-09-24 2011-11-03 Medicinelodge, Inc. Dba Imds Co-Innovation Surgical rasp with radiofrequency ablation
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
US9228174B2 (en) 2010-03-11 2016-01-05 Uniqure Ip B.V. Mutated rep encoding sequences for use in AAV production
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
CN201704771U (zh) 2010-04-30 2011-01-12 陈福环 无水箱节水型抽水马桶
WO2011137344A2 (en) * 2010-04-30 2011-11-03 Lpath, Inc. Anti-s1p antibody treatment of patients with ocular disease
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US20110052678A1 (en) * 2010-11-05 2011-03-03 Shantha Totada R Method for treating age related macular degeneration
JP6092782B2 (ja) 2010-11-16 2017-03-08 エクセリミューン, インコーポレイテッド 組換えタンパク質の製造方法
CN103608455B (zh) 2011-01-07 2019-01-15 应用遗传科技公司 用于治疗色盲和其它疾病的启动子、表达盒、载体、药盒和方法
LT3254703T (lt) 2011-04-22 2020-05-25 The Regents Of The University Of California Adenoasocijuoto viruso virionai su variantine kapside ir jų panaudojimo būdai
AU2012327236B2 (en) 2011-10-28 2016-11-10 University Of Florida Research Foundation, Inc. Chimeric promoter for cone photoreceptor targeted gene therapy
TWI775096B (zh) * 2012-05-15 2022-08-21 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
WO2013188316A1 (en) 2012-06-11 2013-12-19 Avalanche Biotechnologies, Inc. Optical regulation of gene expression in the retina
ES2716615T3 (es) 2013-06-28 2019-06-13 Inst Nat Sante Rech Med Métodos para expresar un polinucleótido de interés en la retina de un sujeto
US20150025939A1 (en) 2013-07-18 2015-01-22 Somnath Chatterjee Company Centric Social Media Platfonn for Content Sharing aud Tracking
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
EP3633041A3 (en) 2013-09-26 2020-07-29 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
EP3113787B1 (en) 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
CA2942776C (en) 2014-03-17 2023-01-24 Adverum Biotechnologies, Inc. Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter
KR20170137730A (ko) 2015-03-02 2017-12-13 애드베룸 바이오테크놀로지스, 인코포레이티드 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999014354A1 (en) * 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
US20020194630A1 (en) * 1999-03-15 2002-12-19 Manning William C. Use of recombinant gene delivery vectors for treating or preventing diseases of the eye

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Gray and Zolotukhin,Design and construction of functional AAV vectors. Methods in Molecular Biology(2011),807,pp.25-46. *
Timothy K MacLachlan et al,Preclinical Safety Evaluation of AAV2-sFLT01—A Gene Therapy for Age-related Macular Degeneration,The American Society of Gene & Cell Therapy,vol. 19 no. 2, 326–334 feb. 2011 *

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