EA201791939A1 - Композиции и способы интравитреальной доставки полинуклеотидов в колбочки сетчатки - Google Patents

Композиции и способы интравитреальной доставки полинуклеотидов в колбочки сетчатки

Info

Publication number
EA201791939A1
EA201791939A1 EA201791939A EA201791939A EA201791939A1 EA 201791939 A1 EA201791939 A1 EA 201791939A1 EA 201791939 A EA201791939 A EA 201791939A EA 201791939 A EA201791939 A EA 201791939A EA 201791939 A1 EA201791939 A1 EA 201791939A1
Authority
EA
Eurasian Patent Office
Prior art keywords
methods
compositions
retchembals
polynucleotides
intravitreal delivery
Prior art date
Application number
EA201791939A
Other languages
English (en)
Russian (ru)
Inventor
Томас В. Чалберг
Джей НЭЙТЗ
Морин НЭЙТЗ
Original Assignee
Адверум Байотекнолоджиз, Инк.
Юниверсити Оф Вашингтон
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Адверум Байотекнолоджиз, Инк., Юниверсити Оф Вашингтон filed Critical Адверум Байотекнолоджиз, Инк.
Publication of EA201791939A1 publication Critical patent/EA201791939A1/ru

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/861—Adenoviral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
EA201791939A 2015-03-02 2016-03-02 Композиции и способы интравитреальной доставки полинуклеотидов в колбочки сетчатки EA201791939A1 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562127194P 2015-03-02 2015-03-02
US201562134466P 2015-03-17 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

Publications (1)

Publication Number Publication Date
EA201791939A1 true EA201791939A1 (ru) 2018-01-31

Family

ID=56848956

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201791939A EA201791939A1 (ru) 2015-03-02 2016-03-02 Композиции и способы интравитреальной доставки полинуклеотидов в колбочки сетчатки

Country Status (15)

Country Link
US (2) US11021519B2 (enExample)
EP (1) EP3265178A4 (enExample)
JP (3) JP7095994B2 (enExample)
KR (1) KR20170137730A (enExample)
CN (2) CN107405507B (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 (47)

* 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
RS66184B1 (sr) 2011-04-22 2024-12-31 Univ California Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu
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)
ES2897508T3 (es) 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
KR102288849B1 (ko) 2014-03-17 2021-08-12 애드베룸 바이오테크놀로지스, 인코포레이티드 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
KR20170137730A (ko) 2015-03-02 2017-12-13 애드베룸 바이오테크놀로지스, 인코포레이티드 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법
EP3277819B1 (en) 2015-03-24 2021-03-03 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
EP3831842A1 (en) 2015-09-28 2021-06-09 The University of North Carolina at Chapel Hill 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
HUE051953T2 (hu) * 2016-06-16 2021-04-28 Adverum Biotechnologies Inc Idõskori makula degeneráció kezelése AAV2 variáns és aflibercept alkalmazásával
JP7071332B2 (ja) 2016-07-29 2022-05-18 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 変異カプシドを有するアデノ随伴ウイルスビリオン及びその使用方法
US11192925B2 (en) 2016-10-19 2021-12-07 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
WO2018134168A1 (en) * 2017-01-17 2018-07-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors
EP4653536A2 (en) 2017-03-17 2025-11-26 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
US20200121746A1 (en) 2017-06-30 2020-04-23 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
US11680249B2 (en) 2017-08-28 2023-06-20 The Regents Of The University Of California Adeno-associated virus capsid variants and methods of use thereof
FI3684423T3 (fi) * 2017-09-20 2023-06-15 4D Molecular Therapeutics Inc Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä
CN111601884A (zh) * 2017-10-16 2020-08-28 维格内罗有限责任公司 Aav载体
WO2019077159A1 (en) * 2017-10-20 2019-04-25 INSERM (Institut National de la Santé et de la Recherche Médicale) METHODS OF EXPRESSING POLYNUCLEOTIDE OF INTEREST IN CONE PHOTORECCEPTORS OF A SUBJECT COMPRISING SUB-RETINAL ADMINISTRATION OF A THERAPEUTICALLY EFFECTIVE AMOUNT OF A RECOMBINANT AAV9 DERIVATIVE VECTOR
GB201800546D0 (en) 2018-01-12 2018-02-28 Ucl Business Plc Treatment
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
WO2019195449A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
AU2019307911B2 (en) * 2018-07-20 2022-06-09 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Intraocular delivery 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
US20220096658A1 (en) * 2018-12-21 2022-03-31 The United States Of America, As Represented By The Secretary, Department Of Health And Human Svcs. Adeno-associated viruses and their uses for inner ear therapy
WO2020168111A1 (en) 2019-02-15 2020-08-20 Exhaura, Ltd. Dual leucine zipper kinase inhibitors for gene therapy
US20200297869A1 (en) * 2019-03-04 2020-09-24 Adverum Biotechnologies, Inc. Sequential intravitreal administration of aav gene therapy to contralateral eyes
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
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
TW202128736A (zh) 2019-10-17 2021-08-01 美商史崔德生物公司 用於治療ｃ型尼曼—匹克病之腺相關病毒載體
WO2021084133A1 (en) * 2019-10-31 2021-05-06 Universität Bern Aav vector variants for ocular gene delivery
MX2022013411A (es) 2020-04-27 2022-11-14 4D Molecular Therapeutics Inc Genes gla con codones optimizados y usos de los mismos.
US12031147B2 (en) * 2020-05-29 2024-07-09 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
US12385064B2 (en) * 2021-03-26 2025-08-12 Adverum Biotechnologies, Inc. Intravitreal dosing for delivery of polynucleotides to retinal cones
MX2023014041A (es) * 2021-05-28 2023-12-15 Shanghai Regenelead Therapies Co Ltd Virus adenoasociado recombinante que tiene una capsida variante y su aplicacion.
WO2022271984A1 (en) * 2021-06-24 2022-12-29 Applied Genetic Technologies Corporation Methods for the treatment of achromatopsia and other cngb3 associated diseases
MX2024004217A (es) * 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
CA3244412A1 (en) * 2021-12-28 2025-06-13 Chengdu Origen Biotechnology Co Ltd MODIFIED VAA CAPSID PROTEIN AND RELATED USES
EP4508064A2 (en) * 2022-04-15 2025-02-19 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
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
EP0557459B1 (en) 1990-11-13 1997-10-22 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
WO1994028143A1 (en) 1993-05-21 1994-12-08 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
EP0927044A4 (en) 1996-04-16 1999-09-08 Immusol Inc DEFINED TARGET VIRAL VECTORS
US6054485A (en) 1996-08-20 2000-04-25 Regents Of The University Of California Eye treatments using synthetic thyroid hormone compositions
ES2274549T3 (es) 1996-09-24 2007-05-16 MERCK & CO., INC. Compuestos para la inhibicion de la angiogenesis por terapia de genes .
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
DE69824859T2 (de) 1997-04-14 2005-08-04 Cell Genesys, Inc., Foster City Methoden zur erhöhung der effizienz rekombinanter aav produkte
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
AU9319198A (en) 1997-09-19 1999-04-05 Trustees Of The University Of Pennsylvania, The 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
WO1999016889A1 (en) 1997-10-01 1999-04-08 G.D. Searle & Co. Fusion proteins comprising an angiostatin moiety and their use in anti-tumor treatment
AU1449499A (en) 1997-10-31 1999-05-24 Maxygen, Incorporated Modification of virus tropism and host range by viral genome shuffling
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
WO1999036511A2 (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
IL138033A0 (en) 1998-03-12 2001-10-31 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
IL141403A0 (en) 1998-08-28 2002-03-10 Univ Duke Deleted adenoviruses and methods of making and administering the same
US6451594B1 (en) 1998-09-11 2002-09-17 The Regents Of The University Of California Recombinant adenovirus for tissue specific expression in heart
WO2000015822A1 (en) 1998-09-17 2000-03-23 University Of Florida Methods for treatment of degenerative retinal diseases
US20040234505A1 (en) 1998-09-23 2004-11-25 Stuart Naylor Polynucleotide constructs and uses thereof
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
CA2367375A1 (en) 1999-03-15 2000-09-21 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
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
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
US7087411B2 (en) 1999-06-08 2006-08-08 Regeneron Pharmaceuticals, Inc. Fusion protein capable of binding VEGF
US7306799B2 (en) 1999-06-08 2007-12-11 Regeneron Pharmaceuticals, Inc. Use of VEGF inhibitors for treatment of eye disorders
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
US6656136B1 (en) 1999-10-25 2003-12-02 Therus Corporation Use of 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
EP1274339B1 (en) 2000-04-17 2011-06-08 The University of Sydney Method for objective electrophysiological assessment of visual function
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
CA2437563C (en) 2001-02-06 2010-03-23 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
EP1381276A4 (en) * 2001-04-13 2005-02-02 Univ Pennsylvania METHOD FOR TREATMENT OR DEVELOPMENT SLUDGE DEGRADATION
JP2002363107A (ja) 2001-06-04 2002-12-18 Noriyuki Azuma 色覚不全動物の色覚復元方法
DK1412493T3 (da) 2001-08-02 2012-01-09 Inst Clayton De La Rech Fremgangsmåder og sammensætninger der angår forbedrede lentiviral-vektor.-produktionsystemer
US7647184B2 (en) 2001-08-27 2010-01-12 Hanall Pharmaceuticals, Co. Ltd High throughput directed evolution by rational mutagenesis
US20030129203A1 (en) 2001-08-27 2003-07-10 Nautilus Biotech S.A. Mutant recombinant adeno-associated viruses
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
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
JP2005512569A (ja) 2001-12-21 2005-05-12 メディジーン・アクチェンゲゼルシャフト 目的とする細胞指向性をもつウイルスクローンの同定に有用な修飾した構造遺伝子またはキャプシド修飾した粒子のライブラリー
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
US7858367B2 (en) 2002-04-30 2010-12-28 Duke University Viral vectors and methods for producing and using the same
AU2003237159A1 (en) 2002-04-30 2003-11-17 University Of Florida 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
PL3235827T3 (pl) 2003-06-19 2021-07-05 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowania
US7368428B2 (en) 2003-06-23 2008-05-06 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
AU2004276316A1 (en) 2003-09-24 2005-04-07 The Board Of Trustees Of The Leland Stanford Junior University IGF-1 instructs multipotent adult CNS neural stem cells to an oligodendroglial lineage
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
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
EP2311433A3 (en) 2004-10-21 2011-08-10 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
CA2591544A1 (en) 2004-12-15 2006-06-22 The University Of North Carolina At Chapel Hill Chimeric vectors
US20100172871A1 (en) 2005-02-17 2010-07-08 Flannery John G Muller Cell Specific Gene Therapy
US7922670B2 (en) 2005-02-24 2011-04-12 Warren Jones System and method for quantifying and mapping visual salience
EP2357010B1 (en) 2005-04-07 2013-06-12 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
WO2007046703A2 (en) 2005-10-20 2007-04-26 Amsterdam Molecular Therapeutics B.V. Improved aav vectors produced in insect cells
US20090191597A1 (en) 2006-01-20 2009-07-30 Asklepios Biopharmaceutical, Inc. Enhanced 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
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University 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
US8470790B2 (en) 2006-05-04 2013-06-25 Wayne State University Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
EP2012826B1 (en) 2006-05-04 2016-04-13 Fovea Pharmaceuticals Combination comprising a vegf inhibitor and a serine protease for treating neovascular diseases
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
US8512981B2 (en) 2006-06-21 2013-08-20 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of AAV-Rep78 useful for production of AAV
US20100008170A1 (en) 2006-06-27 2010-01-14 Shinya Sato Semiconductor tester and testing method of semiconductor memory
DK2061891T3 (da) 2006-08-24 2012-07-23 Virovek Inc Ekspression i insektceller af gener med overlappende åbne læserammer, fremgangsmåder og sammensætninger hertil
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
NZ579985A (en) 2007-04-13 2012-02-24 Catalyst Biosciences Inc Modified factor vii polypetides 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
WO2009104964A1 (en) 2008-02-19 2009-08-27 Amsterdam Molecular Therapeutics B.V. Optimisation of expression of parvoviral rep and cap proteins in insect cells
WO2009105669A2 (en) 2008-02-20 2009-08-27 Genzyme Corporation Angiogenesis inhibition
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
MX2010012592A (es) 2008-05-20 2011-05-05 Eos Neuroscience Inc Vectores para la administracion de proteinas sensibles a la luz y metodos de uso de las mismas.
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
JP5491511B2 (ja) 2008-10-07 2014-05-14 ブラッコ・シュイス・ソシエテ・アノニム 抗ポリマー抗体およびそれと結合するリポソームまたは微小胞を含むターゲッティング構築物
ES2634118T3 (es) 2009-02-11 2017-09-26 The University Of North Carolina At Chapel Hill Vectores de virus modificados y métodos para fabricar y utilizar los mismos
MX2011009193A (es) 2009-03-04 2011-12-16 Deutsches Krebsforsch Proteina activadora del ensamblaje (aap) y su uso para la fabricacion de particulas de parvovirus que consisten esencialmente de 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
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
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts 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
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
WO2011112089A2 (en) 2010-03-11 2011-09-15 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Mutated rep encoding sequences for use in aav production
EP2550021A2 (en) 2010-03-22 2013-01-30 Association Institut de Myologie Methods of increasing efficiency of vector penetration of target tissue
SG10201502270TA (en) 2010-03-29 2015-05-28 Univ 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
EP2563395A4 (en) 2010-04-30 2013-11-27 Lpath Inc TREATMENT WITH ANTI-S1P ANTIBODY OF PATIENTS WITH OCULAR DISEASES
WO2011140450A1 (en) 2010-05-07 2011-11-10 Medicinelodge, Inc Dba Imds Co-Innovation Surgical rasp with radiofrequency ablation
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
CA2817751C (en) 2010-11-16 2019-04-30 Excelimmune, Inc. Methods for producing recombinant proteins
EP2661494B1 (en) 2011-01-07 2019-06-12 Applied Genetic Technologies Corporation Promoters, expression cassettes and vectors for use treating achromatopsia and other diseases
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
US9375491B2 (en) 2011-10-28 2016-06-28 University Of Florida Research Foundation, Inc. Chimeric promoter for cone photoreceptor targeted gene therapy
ES2828663T3 (es) 2012-04-18 2021-05-27 Univ Leland Stanford Junior Dirección genética no disruptiva
EP2847337A4 (en) 2012-05-09 2016-04-27 Univ Oregon Health & Science 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
JP6363958B2 (ja) 2012-12-25 2018-07-25 タカラバイオ株式会社 Ａａｖ変異体
EP3517135B1 (en) * 2013-05-16 2025-11-19 Beacon Therapeutics (USA), Inc. Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
US11685935B2 (en) 2013-05-29 2023-06-27 Cellectis Compact scaffold of Cas9 in the type II CRISPR system
ES2897508T3 (es) 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
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
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
JP6985795B2 (ja) 2013-09-26 2021-12-22 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 標的遺伝子治療のための合成コンビナトリアルａａｖカプシドライブラリー
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
JP6607850B2 (ja) 2013-10-18 2019-11-20 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｖｅｇｆアンタゴニスト及び抗ｃｔｌａ−４抗体の組み合わせを含む方法及び組成物
CA2941640A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
KR102288849B1 (ko) * 2014-03-17 2021-08-12 애드베룸 바이오테크놀로지스, 인코포레이티드 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
EP4600255A3 (en) 2014-05-02 2025-10-22 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
KR20170137730A (ko) 2015-03-02 2017-12-13 애드베룸 바이오테크놀로지스, 인코포레이티드 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법
EP3748007B1 (en) 2015-03-06 2024-06-26 Massachusetts Eye & Ear Infirmary Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene
WO2017023724A1 (en) 2015-07-31 2017-02-09 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
WO2017190125A1 (en) 2016-04-29 2017-11-02 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
CN109476707B (zh) 2016-05-13 2022-12-02 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
AU2017286680B2 (en) 2016-06-16 2023-01-19 Adverum Biotechnologies, Inc. Compositions and methods for reducing ocular neovascularization
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
EP4653536A2 (en) 2017-03-17 2025-11-26 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression

Also Published As

Publication number Publication date
EP3265178A1 (en) 2018-01-10
JP2022107042A (ja) 2022-07-20
CN107405507A (zh) 2017-11-28
KR20170137730A (ko) 2017-12-13
MY187898A (en) 2021-10-27
AU2021209206A1 (en) 2021-08-19
CN107405507B (zh) 2022-05-03
US11021519B2 (en) 2021-06-01
CA2978068C (en) 2024-05-28
AU2016226289A1 (en) 2017-09-21
JP2020122008A (ja) 2020-08-13
US20210388030A1 (en) 2021-12-16
EP3265178A4 (en) 2018-09-05
JP7095994B2 (ja) 2022-07-05
CA2978068A1 (en) 2016-09-09
HK1249067A1 (zh) 2018-10-26
US20180066022A1 (en) 2018-03-08
SG11201707063TA (en) 2017-09-28
MX2017011041A (es) 2018-03-02
AU2016226289B2 (en) 2021-04-29
WO2016141078A1 (en) 2016-09-09
JP2018508519A (ja) 2018-03-29
IL254148A0 (en) 2017-10-31
CN114480502A (zh) 2022-05-13
BR112017018846A2 (pt) 2018-07-31
IL254148B (en) 2022-09-01

Similar Documents

Publication Publication Date Title
EA201791939A1 (ru) Композиции и способы интравитреальной доставки полинуклеотидов в колбочки сетчатки
ZA202004557B (en) Modulatory polynucleotides
PH12018500714A1 (en) Anti-tigit antigen-binding proteins and methods of use thereof
EA201692109A1 (ru) Варианты антител к фактору d и их применение
EA201890725A1 (ru) Агонисты фарнезоидного х-рецептора и их применение
EA201791168A1 (ru) Способы и составы для лечения сосудистых заболеваний глаз
EA201891186A1 (ru) Антитела, нейтрализующие вирус иммунодефицита человека
EA201692529A1 (ru) Способы и композиции для лечения расстройств с использованием фоллистатиновых полипептидов
MX2022001105A (es) Anticuerpos anti tigit.
EA201692177A1 (ru) Соединения и композиции в качестве агонистов toll-подобного рецептора 7
PH12017501522B1 (en) Antibodies to tau and uses thereof
EA201791005A1 (ru) Улучшенные антитела против il-6
MX388562B (es) Composiciones y métodos para el tratamiento de trastornos de almacenamiento lisosomal y trastornos caracterizados por disfunción lisosomal.
EA202091865A1 (ru) Композиции и способы для снижения экспрессии tau
WO2015132675A3 (en) Methods and compositions for modifying the immune response
CL2018000565A1 (es) Compuestos derivados de piridinona dicarboxamida, inhibidores de bromodominio; composicion farmaceutica; combinacion farmaceutica; utiles en el tratamiento de enfermedades autoinmunitarias, inflamatorias, virales y cancer, entre otras.
EA201792399A1 (ru) Пептидные композиции и способы использования
EP3500278C0 (en) COMPOSITIONS FOR THE TREATMENT OF CONDITIONS USING A SELF-COMPLEMENTARY RECOMBINANT ADENO-ASSOCIATED VIRUS
EA201792571A1 (ru) Терапевтическое применение l-4-хлорокинуренина
EP3229793A4 (en) Compositions and methods relating to the treatment of cancer, autoimmune disease, and neurodegenerative disease
EA202090270A1 (ru) Новые замещенные производные ксантина
EA201791249A1 (ru) Получаемые из cd44v6 циклические пептиды для лечения злокачественных опухолей и связанных с ангиогенезом заболеваний
EA201790893A1 (ru) Способы лечения офтальмологических расстройств
EA202091567A1 (ru) Химерные белки мти
GB201518456D0 (en) Novel compounds and pharmaceutical compositions thereof for the treatment of inflammatory, autoimmune and/or proliferative diseases