JP2018508519A5 - - Google Patents

Download PDF

Info

Publication number
JP2018508519A5
JP2018508519A5 JP2017546102A JP2017546102A JP2018508519A5 JP 2018508519 A5 JP2018508519 A5 JP 2018508519A5 JP 2017546102 A JP2017546102 A JP 2017546102A JP 2017546102 A JP2017546102 A JP 2017546102A JP 2018508519 A5 JP2018508519 A5 JP 2018508519A5
Authority
JP
Japan
Prior art keywords
cone
composition
amino acids
seq
item
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.)
Granted
Application number
JP2017546102A
Other languages
English (en)
Japanese (ja)
Other versions
JP7095994B2 (ja
JP2018508519A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2016/020482 external-priority patent/WO2016141078A1/en
Publication of JP2018508519A publication Critical patent/JP2018508519A/ja
Publication of JP2018508519A5 publication Critical patent/JP2018508519A5/ja
Priority to JP2022082310A priority Critical patent/JP2022107042A/ja
Application granted granted Critical
Publication of JP7095994B2 publication Critical patent/JP7095994B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2017546102A 2015-03-02 2016-03-02 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法 Active JP7095994B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022082310A JP2022107042A (ja) 2015-03-02 2022-05-19 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562127194P 2015-03-02 2015-03-02
US62/127,194 2015-03-02
US201562134466P 2015-03-17 2015-03-17
US62/134,466 2015-03-17
PCT/US2016/020482 WO2016141078A1 (en) 2015-03-02 2016-03-02 Compositions and methods for intravitreal delivery of polynucleotides to retinal cones

Related Child Applications (2)

Application Number Title Priority Date Filing Date
JP2020081106A Division JP2020122008A (ja) 2015-03-02 2020-05-01 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法
JP2022082310A Division JP2022107042A (ja) 2015-03-02 2022-05-19 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法

Publications (3)

Publication Number Publication Date
JP2018508519A JP2018508519A (ja) 2018-03-29
JP2018508519A5 true JP2018508519A5 (enExample) 2019-04-11
JP7095994B2 JP7095994B2 (ja) 2022-07-05

Family

ID=56848956

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2017546102A Active JP7095994B2 (ja) 2015-03-02 2016-03-02 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法
JP2020081106A Pending JP2020122008A (ja) 2015-03-02 2020-05-01 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法
JP2022082310A Withdrawn JP2022107042A (ja) 2015-03-02 2022-05-19 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2020081106A Pending JP2020122008A (ja) 2015-03-02 2020-05-01 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法
JP2022082310A Withdrawn JP2022107042A (ja) 2015-03-02 2022-05-19 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法

Country Status (15)

Country Link
US (2) US11021519B2 (enExample)
EP (1) EP3265178B1 (enExample)
JP (3) JP7095994B2 (enExample)
KR (1) KR20170137730A (enExample)
CN (2) CN114480502A (enExample)
AU (2) AU2016226289B2 (enExample)
BR (1) BR112017018846A2 (enExample)
CA (1) CA2978068C (enExample)
EA (1) EA201791939A1 (enExample)
HK (1) HK1249067A1 (enExample)
IL (1) IL254148B (enExample)
MX (1) MX2017011041A (enExample)
MY (1) MY187898A (enExample)
SG (1) SG11201707063TA (enExample)
WO (1) WO2016141078A1 (enExample)

Families Citing this family (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN107012171B (zh) 2011-04-22 2021-07-13 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
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)
CN105247044B (zh) 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
EP3800191B1 (en) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
JP7095994B2 (ja) 2015-03-02 2022-07-05 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 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法
ES2860712T3 (es) 2015-03-24 2021-10-05 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
SI3471780T1 (sl) * 2016-06-16 2021-03-31 Adverum Biotechnologies, Inc. Zdravljenje SDM-ja z uporabo različice AAV2 z afliberceptom
EP4700118A3 (en) 2016-07-29 2026-04-22 The Regents of the University of California Adeno-associated virus virions with varint capsid and methods of use thereof
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
CA3046668A1 (en) * 2017-01-03 2018-07-12 Vitrean, Inc. Methods and devices for treating a retinal detachment
EP3570895A1 (en) * 2017-01-17 2019-11-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors
EP4653536A3 (en) 2017-03-17 2026-02-25 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
EP3645052A4 (en) * 2017-06-30 2021-07-28 Regents of the University of California ADENO-ASSOCIATED VIRUS VIRIONS WITH ASSOCIATED CAPSIDE VARIANTS AND METHODS OF USE
KR20200039617A (ko) 2017-08-28 2020-04-16 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 아데노-연관 바이러스 캡시드 변이체 및 이의 사용 방법
PE20201264A1 (es) * 2017-09-20 2020-11-19 4D Molecular Therapeutics Inc Variantes de capsides de virus adenoasociados y metodos de uso de estas
US12043848B2 (en) 2017-10-16 2024-07-23 Vigeneron Gmbh AAV vectors
KR102763312B1 (ko) * 2017-10-20 2025-02-07 인쎄름 (엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔) 치료적으로 유효한 양의 재조합 aav9- 유도 벡터의 망막하 전달을 포함하는 대상체의 원추 광수용체 내 관심의 폴리뉴클레오티드를 발현하는 방법
GB201800546D0 (en) 2018-01-12 2018-02-28 Ucl Business Plc Treatment
WO2019195423A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
EP3823567B1 (en) * 2018-07-20 2025-06-18 The USA, as represented by The Secretary, Department of Health and Human Services Intraocular injection of gene therapy expression vectors
CN111118016B (zh) 2018-10-30 2022-04-15 上海朗昇生物科技有限公司 治疗视网膜色素变性疾病的基因治疗载体
CN113825521A (zh) * 2018-12-21 2021-12-21 美利坚合众国由健康及人类服务部部长代表 腺相关病毒及其用于内耳疗法的用途
WO2020168111A1 (en) 2019-02-15 2020-08-20 Exhaura, Ltd. Dual leucine zipper kinase inhibitors for gene therapy
EP3934698A1 (en) * 2019-03-04 2022-01-12 Adverum Biotechnologies, Inc. Sequential intravitreal administration of aav gene therapy to contralateral eyes
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
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
KR20220083708A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 Aav 전달 카세트
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
EP4051694B1 (en) 2019-10-31 2025-07-02 Universität Bern Aav vector variants for ocular gene delivery
CN115515613A (zh) 2020-04-27 2022-12-23 4D分子治疗有限公司 密码子优化的gla基因及其用途
CA3179597A1 (en) * 2020-05-29 2021-12-02 John G. Flannery Adeno-associated virus virions with variant capsids and methods of use thereof
MX2023002016A (es) 2020-08-19 2023-06-26 Sarepta Therapeutics Inc Vectores de virus adenoasociados para el tratamiento del sindrome de rett.
US12385064B2 (en) * 2021-03-26 2025-08-12 Adverum Biotechnologies, Inc. Intravitreal dosing for delivery of polynucleotides to retinal cones
WO2022247917A1 (zh) * 2021-05-28 2022-12-01 上海瑞宏迪医药有限公司 衣壳变异的重组腺相关病毒及其应用
WO2022271984A1 (en) * 2021-06-24 2022-12-29 Applied Genetic Technologies Corporation Methods for the treatment of achromatopsia and other cngb3 associated diseases
EP4413147A4 (en) 2021-10-08 2025-07-30 Dyno Therapeutics Inc CAPSID VARIANTS AND METHODS OF USE THEREOF
CN116444626A (zh) * 2021-12-28 2023-07-18 成都弘基生物科技有限公司 经过修饰的aav衣壳蛋白及其用途
CN119630688A (zh) * 2022-04-15 2025-03-14 迪诺治疗股份有限公司 衣壳变体及其使用方法
CN116970648A (zh) * 2022-04-24 2023-10-31 上海朗昇生物科技有限公司 新型aav衣壳改造株及其用途
WO2024059667A2 (en) * 2022-09-13 2024-03-21 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2024110770A1 (en) * 2022-11-22 2024-05-30 INSERM (Institut National de la Santé et de la Recherche Médicale) A new promoter for retinal pigment epithelium (rpe) targeted gene therapy
CN119350454B (zh) * 2024-12-27 2025-03-25 上海朗昇生物科技有限公司 衣壳重排型腺相关病毒载体及其用途

Family Cites Families (193)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US46016A (en) * 1865-01-24 Improvement in coats with inner sleeves
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
WO1992008796A1 (en) 1990-11-13 1992-05-29 Immunex Corporation Bifunctional selectable fusion genes
US5383917A (en) 1991-07-05 1995-01-24 Jawahar M. Desai Device and method for multi-phase radio-frequency ablation
GB9206016D0 (en) 1992-03-19 1992-04-29 Sandoz Ltd Improvements in or relating to organic compounds
WO1994021679A1 (en) 1993-03-25 1994-09-29 Merck & Co., Inc. Inhibitor of vascular endothelial cell growth factor
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
EP0745134A1 (en) 1994-02-22 1996-12-04 Danafarber 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
US6440425B1 (en) 1995-05-01 2002-08-27 Aventis Pasteur Limited High molecular weight major outer membrane protein of moraxella
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
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
US6096548A (en) 1996-03-25 2000-08-01 Maxygen, Inc. Method for directing evolution of a virus
AU2678097A (en) 1996-04-16 1997-11-07 Immusol Incorporated Targeted viral vectors
CA2260992C (en) 1996-08-20 2004-03-09 The Regents Of The University Of California Eye treatments using synthetic thyroid hormone compositions
ATE343400T1 (de) 1996-09-24 2006-11-15 Merck & Co Inc Verbindungen zur hemmung der angiogenese durch gentherapie
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
WO1998051323A1 (en) 1997-05-13 1998-11-19 The Regents Of The University Of California Novel antiangiogenic peptide agents and their therapeutic and diagnostic use
US6710036B2 (en) 1997-07-25 2004-03-23 Avigen, Inc. Induction of immune response to antigens expressed by recombinant adeno-associated virus
US6458157B1 (en) 1997-08-04 2002-10-01 Suaning Gregg Joergen Retinal stimulator
CA2303768C (en) 1997-09-19 2009-11-24 The Trustees Of The University Of Pennsylvania Methods and vector constructs useful for production of recombinant aav
JPH11100327A (ja) 1997-09-26 1999-04-13 Toray Ind Inc 網膜変性症治療剤
EP1019515A1 (en) 1997-10-01 2000-07-19 G.D. SEARLE & CO. Fusion proteins comprising an angiostatin moiety and their use in anti-tumour treatment
EP1030861A4 (en) 1997-10-31 2001-09-05 Maxygen Inc MODIFICATION OF VIRUSTROPISMIUS AND THE ECONOMIC SPECTRUM BY VIRAL GENOM MIXING
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
CA2318575A1 (en) 1998-01-16 1999-07-22 Chiron Corporation Feline immunodeficiency virus gene therapy vectors
US6551795B1 (en) 1998-02-18 2003-04-22 Genome Therapeutics Corporation Nucleic acid and amino acid sequences relating to pseudomonas aeruginosa for diagnostics and therapeutics
WO1999045952A2 (en) 1998-03-12 1999-09-16 Genentech, Inc. Method of preventing the death of retinal neurons and treating ocular diseases
DE19827457C1 (de) 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
US6593133B1 (en) 1998-07-06 2003-07-15 Nsgene A/S Neurotrophic factors
CA2340276A1 (en) 1998-08-28 2000-03-09 Duke University Adenoviruses deleted in the iva2, 100k and/or preterminal protein sequences
JP2002525064A (ja) 1998-09-11 2002-08-13 ザ レジェンツ オブ ザ ユニヴァースティ オブ カリフォルニア 心臓で組織特異的に発現する新規組換えアデノウイルス
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
ATE454445T1 (de) 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
US6943153B1 (en) 1999-03-15 2005-09-13 The Regents Of The University Of California Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
WO2000054813A2 (en) 1999-03-15 2000-09-21 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
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
US7314912B1 (en) 1999-06-21 2008-01-01 Medigene Aktiengesellschaft AAv scleroprotein, production and use thereof
DE19933288A1 (de) 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
DE19933719A1 (de) 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
US8232081B2 (en) 1999-09-21 2012-07-31 Basf Se Methods and microorganisms for production of panto-compounds
EP1229839A4 (en) 1999-10-25 2005-12-07 Therus Corp USING FOCUSED ULTRASOUND FOR VASCULAR SEALING
US20070026394A1 (en) 2000-02-11 2007-02-01 Lawrence Blatt Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
NZ521952A (en) 2000-04-17 2004-03-26 Stuart Graham Method and apparatus for electrophysiological assessment of visual function using a head-mounted stereo display
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
US8034803B2 (en) 2001-02-06 2011-10-11 Qlt Inc. Photodynamic therapy of occult 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
WO2002082904A2 (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
WO2003012054A2 (en) 2001-08-02 2003-02-13 Institut Clayton De La Recherche Methods and compositions relating to improved lentiviral vector production systems
US20030129203A1 (en) 2001-08-27 2003-07-10 Nautilus Biotech S.A. Mutant recombinant adeno-associated viruses
US7647184B2 (en) 2001-08-27 2010-01-12 Hanall Pharmaceuticals, Co. Ltd High throughput directed evolution by rational mutagenesis
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
SG10202108118RA (en) 2001-11-13 2021-08-30 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2467156T3 (es) 2001-12-21 2014-06-12 Medigene Ag Una biblioteca de genes estructurales modificados o de partículas modificadas de cápside útiles para la identificación de clones virales con tropismo celular deseado
AU2003225910A1 (en) 2002-03-20 2003-10-08 Johns Hopkins University Raav vector compositions and methods for the treatment of choroidal neovascularization
US20060292117A1 (en) 2002-04-17 2006-12-28 Loiler Scott A Improved rAAv vectors
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
US20040142416A1 (en) 2002-04-30 2004-07-22 Laipis Philip J. Treatment for phenylketonuria
US7254489B2 (en) 2002-05-31 2007-08-07 Microsoft Corporation Systems, methods and apparatus for reconstructing phylogentic trees
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
JP2006519026A (ja) 2003-03-04 2006-08-24 独立行政法人産業技術総合研究所 標的物質の細胞導入効率を上昇させるための組成物および方法
US20070172460A1 (en) 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
WO2004108922A2 (en) 2003-04-25 2004-12-16 The Trustees Of The University Of Pennsylvania Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
PL2357189T3 (pl) 2003-06-19 2017-08-31 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
CA2530582C (en) 2003-06-23 2014-09-09 A&G Pharmaceutical, Inc. Compositions and methods for restoring sensitivity of tumor cells to antitumor therapy and inducing apoptosis
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
US20050148069A1 (en) 2003-09-24 2005-07-07 Gage Fred H. IGF-1 instructs multipotent adult CNS neural stem cells to an oligodendroglial lineage
HUE054805T2 (hu) 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
US7427396B2 (en) 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
EP1802334B1 (en) 2004-10-21 2012-08-29 Genentech, Inc. Method for treating intraocular neovascular diseases
CN101090968B (zh) 2004-11-08 2011-10-26 科罗迈吉尼科斯公司 高水平表达蛋白质的宿主细胞的选择
AU2005316476A1 (en) 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
WO2006101634A1 (en) 2005-02-17 2006-09-28 The Regents Of The University Of California Müller cell specific gene therapy
US7922670B2 (en) 2005-02-24 2011-04-12 Warren Jones System and method for quantifying and mapping visual salience
EP3085389A1 (en) 2005-04-07 2016-10-26 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
AU2006304997B2 (en) 2005-10-20 2012-03-01 Uniqure Ip B.V. Improved AAV vectors produced in insect cells
EP1981548A4 (en) 2006-01-20 2010-03-24 Univ North Carolina REINFORCED PRODUCTION OF INFECTIOUS PARVOVIRUS VECTORS IN INSECT CELLS
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
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
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
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
WO2007128526A2 (en) 2006-05-04 2007-11-15 Fovea Pharmaceuticals Sa Combination comprising a vegf inhibitor and a serine protease for treating neovascular diseases
WO2007131180A2 (en) 2006-05-04 2007-11-15 Wayne State University Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
JP2009540011A (ja) 2006-06-12 2009-11-19 エクセジェニックス、インク.ディー/ビー/エー オプコ ヘルス、インク. 血管新生のsiRNA阻害のための組成物及び方法
GB0612096D0 (en) 2006-06-19 2006-07-26 Greater Glasgow Nhs Board Functional imaging of the retina
ES2785223T3 (es) 2006-06-21 2020-10-06 Uniqure Ip Bv Células de insecto para la producción de vectores de AAV
JPWO2008001543A1 (ja) 2006-06-27 2009-11-26 株式会社アドバンテスト 半導体試験装置および半導体メモリの試験方法
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
CN1966082B (zh) 2006-11-03 2010-06-30 许瑞安 一种防治结直肠癌的基因药物及其制备方法和用途
US8075137B2 (en) 2007-02-16 2011-12-13 Objectivision Limited Stimulus method for multifocal visual evoked potential
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
EP2481797A1 (en) 2007-04-13 2012-08-01 Catalyst Biosciences, Inc. Modified factor VII polypeptides and uses thereof
EP1985708B1 (en) 2007-04-27 2015-04-15 Universität Rostock Selective targeting of viruses to neural precursor cells
EP1995309A1 (en) 2007-05-21 2008-11-26 Vivalis Recombinant protein production in avian EBx® cells
US10696983B2 (en) 2007-05-30 2020-06-30 The Trustees Of The University Of Pennsylvania Method for transducing cells with primary cilia
WO2009023805A1 (en) 2007-08-16 2009-02-19 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
JP2011505147A (ja) 2007-11-30 2011-02-24 スカラブ ゲノミクス, エルエルシー lac発現システム
ES2644880T3 (es) 2008-02-19 2017-11-30 Uniqure Ip B.V. Optimización de expresión de proteínas Rep y Cap parvovirales en las células de insecto
JP2011512417A (ja) 2008-02-20 2011-04-21 ジェンザイム・コーポレーション 血管形成の阻害
WO2009105690A2 (en) 2008-02-21 2009-08-27 Targeted Genetics Corporation 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
WO2009137006A2 (en) 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
EP2315833B8 (en) 2008-05-20 2015-05-27 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
ES2330826B1 (es) 2008-06-04 2010-07-26 Proyecto De Biomedicina Cima, S.L. Sistema para empaquetamiento de adenovirus de alta capacidad.
EP2297185A1 (en) 2008-06-17 2011-03-23 Amsterdam Molecular Therapeutics (AMT) B.V. Parvoviral capsid with incorporated gly-ala repeat region
US9192685B2 (en) 2008-10-07 2015-11-24 Bracco Suisse S.A. Targeting construct comprising anti-polymer antibody and contrast/therapeutic agents binding to the same
US8889641B2 (en) 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
CN102341406B (zh) 2009-03-04 2016-06-08 德国癌症研究中心 组装活化蛋白(aap)及其用于制备基本上由vp3组成的细小病毒颗粒的应用
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
CN101532024A (zh) 2009-04-30 2009-09-16 许瑞安 一种用于基因治疗的新型细胞特异性内含microRNA结合序列的基因HAAVmir
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
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
EP2545165B1 (en) 2010-03-11 2020-07-29 uniQure IP B.V. Mutated rep encoding sequences for use in aav production
WO2011117258A2 (en) 2010-03-22 2011-09-29 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
KR20130040844A (ko) 2010-03-29 2013-04-24 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 약학적으로 유발된 전이유전자 제거 시스템
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
EP2563395A4 (en) 2010-04-30 2013-11-27 Lpath Inc TREATMENT WITH ANTI-S1P ANTIBODY OF PATIENTS WITH OCULAR DISEASES
CN201704771U (zh) 2010-04-30 2011-01-12 陈福环 无水箱节水型抽水马桶
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
DK2640743T3 (en) 2010-11-16 2017-01-23 Excelimmune Inc PROCEDURES FOR THE PREPARATION OF RECOMBINANT PROTEINS
WO2012094560A2 (en) 2011-01-07 2012-07-12 Applied Genetic Technologies Corporation Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
CN107012171B (zh) * 2011-04-22 2021-07-13 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
EP3147295B2 (en) 2011-08-24 2023-11-22 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
AU2012327236B2 (en) 2011-10-28 2016-11-10 University Of Florida Research Foundation, Inc. Chimeric promoter for cone photoreceptor targeted gene therapy
WO2013158309A2 (en) 2012-04-18 2013-10-24 The Board Of Trustees Of The Leland Stanford Junior University Non-disruptive gene targeting
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
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
EP2940131B1 (en) 2012-12-25 2019-02-20 Takara Bio Inc. Aav variant
CN112779255A (zh) 2013-05-16 2021-05-11 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
EP3004339B1 (en) 2013-05-29 2021-07-07 Cellectis New compact scaffold of cas9 in the type ii crispr system
CN105247044B (zh) 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
US9597415B2 (en) * 2013-06-28 2017-03-21 Inserm (Institute National De La Sante Et De La Recherche Medicale) Methods for expressing a polynucleotide of interest in the retina of a subject
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
AU2014324717B2 (en) 2013-09-26 2020-10-08 University Of Florida Research Foundation, Inc. Synthetic combinatorial AAV capsid library for targeted gene therapy
NZ718926A (en) 2013-10-11 2021-12-24 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
MX377714B (es) 2013-10-18 2025-03-11 Regeneron Pharma Composiciones que comprenden una combinación de un antagonista de vegf y un anticuerpo anti-ctla-a.
ES2768763T3 (es) 2014-03-04 2020-06-23 Univ Florida Vectores rAAV mejorados y métodos para la transducción de fotorreceptores y células EPR
EP3800191B1 (en) * 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
EP3137497B2 (en) 2014-05-02 2025-03-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
WO2015191693A2 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
JP7095994B2 (ja) 2015-03-02 2022-07-05 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 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法
PT3265568T (pt) 2015-03-06 2020-08-20 Massachusetts Eye & Ear Infirmary Terapias de aumento de genes para a degeneração hereditária da retina causada por mutações no gene prpf31
US20190000940A1 (en) 2015-07-31 2019-01-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
US20190142975A1 (en) 2016-04-29 2019-05-16 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
WO2017197355A2 (en) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
DK3472317T3 (da) 2016-06-16 2022-05-23 Adverum Biotechnologies Inc Sammensætninger og fremgangsmåder til reduktion af okulær vaskularisation
SI3471780T1 (sl) 2016-06-16 2021-03-31 Adverum Biotechnologies, Inc. Zdravljenje SDM-ja z uporabo različice AAV2 z afliberceptom
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
CN110650733A (zh) 2017-02-28 2020-01-03 阿德夫拉姆生物技术股份有限公司 经过修饰的aav衣壳和其用途
EP4653536A3 (en) 2017-03-17 2026-02-25 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression

Similar Documents

Publication Publication Date Title
JP2025039615A (ja) 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法
JP2021503914A5 (enExample)
ES2916344T3 (es) Composiciones y métodos para reducir la neovascularización ocular
JP2022141670A5 (enExample)
ES2840059T3 (es) Tratamiento de la DMRE mediante el uso de la variante AAV2 con aflibercept
JP2020528734A5 (enExample)
JP2022529775A5 (enExample)
JP2019511570A5 (enExample)
JP2015523060A5 (enExample)
JP2023545424A5 (enExample)
US10849991B2 (en) Gene therapy for the treatment of a disease of retinal cone cells
JP2022522776A5 (enExample)
JP2021500922A5 (enExample)
CN117821513A (zh) 治疗退行性眼部疾病的药物组合物及方法
EP4703475A1 (en) Nucleic acid encoding anti-vegf protein, polynucleotide expression cassette and recombinant adeno-associated virus
JP7707081B2 (ja) 網膜細胞を標的とした最適化された遺伝子治療
JP2022533983A5 (enExample)
CA3125294A1 (en) Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design
JP2023502474A5 (enExample)
RU2025132907A (ru) Нуклеиновая кислота, кодирующая белок, специфичный к vegf, полинуклеотидная кассета экспрессии и рекомбинантный аденоассоциированный вирус
WO2025218741A1 (zh) 治疗眼部疾病的药物组合物及方法
AU2024322589A1 (en) Compositions and methods for expressing therapeutics
WO2026080587A2 (en) Compositions and methods for gene therapy
HK40035863B (en) Adeno-associated virus virions with variant capsid and methods of use thereof
HK40035863A (en) Adeno-associated virus virions with variant capsid and methods of use thereof