PL3137497T4 - Wektory AAV do terapii genowej siatkówki i OUN - Google Patents
Wektory AAV do terapii genowej siatkówki i OUNInfo
- Publication number
- PL3137497T4 PL3137497T4 PL15723386.7T PL15723386T PL3137497T4 PL 3137497 T4 PL3137497 T4 PL 3137497T4 PL 15723386 T PL15723386 T PL 15723386T PL 3137497 T4 PL3137497 T4 PL 3137497T4
- Authority
- PL
- Poland
- Prior art keywords
- retinal
- gene therapy
- aav vectors
- cns gene
- cns
- Prior art date
Links
- 238000001415 gene therapy Methods 0.000 title 1
- 230000002207 retinal effect Effects 0.000 title 1
- 239000013598 vector Substances 0.000 title 1
Classifications
-
- 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
-
- 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
-
- 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
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/08—Antiepileptics; Anticonvulsants
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
-
- 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
- A61P27/06—Antiglaucoma agents or miotics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- 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
-
- 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
- C07K14/01—DNA viruses
- C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
- C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- 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
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2750/14022—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
- C12N2750/14023—Virus like particles [VLP]
-
- 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
- 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
- C12N2750/14145—Special targeting system for viral vectors
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Ophthalmology & Optometry (AREA)
- Epidemiology (AREA)
- Cell Biology (AREA)
- Toxicology (AREA)
- Psychology (AREA)
- Pain & Pain Management (AREA)
- Psychiatry (AREA)
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201461988131P | 2014-05-02 | 2014-05-02 | |
US201562114575P | 2015-02-10 | 2015-02-10 | |
PCT/US2015/028966 WO2015168666A2 (en) | 2014-05-02 | 2015-05-02 | Aav vectors for retinal and cns gene therapy |
EP15723386.7A EP3137497B1 (en) | 2014-05-02 | 2015-05-02 | Aav vectors for retinal and cns gene therapy |
Publications (2)
Publication Number | Publication Date |
---|---|
PL3137497T3 PL3137497T3 (pl) | 2021-10-25 |
PL3137497T4 true PL3137497T4 (pl) | 2023-04-11 |
Family
ID=53189202
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PL15723386.7T PL3137497T4 (pl) | 2014-05-02 | 2015-05-02 | Wektory AAV do terapii genowej siatkówki i OUN |
Country Status (32)
Country | Link |
---|---|
US (2) | US10982228B2 (pl) |
EP (2) | EP3913061A1 (pl) |
JP (3) | JP6741591B2 (pl) |
KR (2) | KR20160147974A (pl) |
CN (2) | CN114231563A (pl) |
AU (3) | AU2015252797C1 (pl) |
BR (1) | BR112016025263A2 (pl) |
CA (1) | CA2946593A1 (pl) |
CL (2) | CL2016002713A1 (pl) |
CR (1) | CR20160555A (pl) |
DK (1) | DK3137497T3 (pl) |
DO (1) | DOP2016000280A (pl) |
EA (1) | EA034355B1 (pl) |
ES (1) | ES2879636T3 (pl) |
GT (1) | GT201600227A (pl) |
HR (1) | HRP20211024T1 (pl) |
HU (1) | HUE054768T2 (pl) |
IL (2) | IL292951B1 (pl) |
LT (1) | LT3137497T (pl) |
MX (2) | MX2016014220A (pl) |
MY (1) | MY188987A (pl) |
PE (1) | PE20170260A1 (pl) |
PH (1) | PH12016502046A1 (pl) |
PL (1) | PL3137497T4 (pl) |
PT (1) | PT3137497T (pl) |
RS (1) | RS62078B1 (pl) |
SG (3) | SG11201607991WA (pl) |
SI (1) | SI3137497T1 (pl) |
TW (1) | TWI686476B (pl) |
UY (1) | UY36106A (pl) |
WO (1) | WO2015168666A2 (pl) |
ZA (1) | ZA201606763B (pl) |
Families Citing this family (149)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
MX356669B (es) | 2009-05-02 | 2018-06-08 | Genzyme Corp | Terapia genica para trastornos neurodegenerativos. |
WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
JP2013533847A (ja) | 2010-04-23 | 2013-08-29 | ユニバーシティ オブ マサチューセッツ | コレステロール関連障害のaavベースの治療 |
CA3049237C (en) | 2010-04-23 | 2024-06-11 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
US9546369B2 (en) | 2010-04-23 | 2017-01-17 | University Of Massachusetts | Multicistronic expression constructs |
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 |
WO2012145624A2 (en) | 2011-04-21 | 2012-10-26 | University Of Massachusetts | Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
EP2699270B1 (en) | 2011-04-22 | 2017-06-21 | The Regents of The University of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
KR20240093939A (ko) * | 2013-05-15 | 2024-06-24 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달 |
US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
EP3564379A1 (en) | 2013-09-13 | 2019-11-06 | California Institute of Technology | Selective recovery |
WO2015127128A2 (en) | 2014-02-19 | 2015-08-27 | University Of Massachusetts | Recombinant aavs having useful transcytosis properties |
AU2015231439B2 (en) | 2014-03-17 | 2019-11-14 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
JP6756700B2 (ja) | 2014-03-18 | 2020-09-16 | ユニバーシティ オブ マサチューセッツ | 筋萎縮性側索硬化症を処置するためのrAAVベースの組成物および方法 |
WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
JP6634073B2 (ja) * | 2014-05-14 | 2020-01-22 | エステベ ファーマシューティカルズ, ソシエダッド アノニマEsteve Pharmaceuticals, S.A. | リソソーム蓄積症処置用のアデノ随伴ウイルスベクター |
US10689653B2 (en) | 2014-06-03 | 2020-06-23 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
JP6842410B2 (ja) | 2014-10-03 | 2021-03-17 | ユニバーシティ オブ マサチューセッツ | 新規の高効率ライブラリーにより同定されるaavベクター |
WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
RU2020140209A (ru) | 2014-10-21 | 2021-01-25 | Юниверсити Оф Массачусетс | Варианты рекомбинантных aav и их применения |
EP3256588A2 (en) | 2015-02-10 | 2017-12-20 | Genzyme Corporation | VARIANT RNAi |
CN107530447A (zh) * | 2015-02-10 | 2018-01-02 | 建新公司 | 病毒颗粒至纹状体和皮质的增强递送 |
US10584321B2 (en) | 2015-02-13 | 2020-03-10 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
KR20170137730A (ko) | 2015-03-02 | 2017-12-13 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법 |
CA2979229A1 (en) | 2015-03-11 | 2016-09-15 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Rp2 vectors for treating x-linked retinitis pigmentosa |
WO2016154344A1 (en) | 2015-03-24 | 2016-09-29 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
TWI707951B (zh) | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
EP3285780A4 (en) | 2015-04-24 | 2018-12-19 | University of Massachusetts | Modified aav constructions and uses thereof |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
ES2865487T3 (es) | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
US11426469B2 (en) | 2015-10-22 | 2022-08-30 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
CA3002982A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance in neurodegenerative disease |
WO2017075335A1 (en) | 2015-10-28 | 2017-05-04 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
EP3875593A1 (en) | 2015-12-11 | 2021-09-08 | California Institute of Technology | Targeting peptides for directing adeno-associated viruses (aavs) |
SG11201804994QA (en) * | 2015-12-15 | 2018-07-30 | Genzyme Corp | Adeno-associated viral vectors for treating mucolipidosis type ii |
US11826433B2 (en) | 2016-02-02 | 2023-11-28 | University Of Massachusetts | Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system |
EP4094780A3 (en) | 2016-02-12 | 2023-02-08 | University of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
WO2017147477A1 (en) * | 2016-02-26 | 2017-08-31 | University Of Florida Research Foundation, Inc. | Aav heparin mutants that display significantly improved eye and brain transduction |
WO2017176929A1 (en) | 2016-04-05 | 2017-10-12 | University Of Massachusetts | Compositions and methods for selective inhibition of grainyhead-like protein expression |
CN107287238B (zh) * | 2016-04-11 | 2020-10-16 | 厦门继景生物技术有限责任公司 | 一种基因载体及其用于治疗雷柏氏先天性黑矇2型疾病的基因治疗药物 |
US11413356B2 (en) | 2016-04-15 | 2022-08-16 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
GB201608046D0 (en) | 2016-05-09 | 2016-06-22 | Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The | Treatment of complement-mediated disorders |
SG11201809643UA (en) * | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
US11882815B2 (en) | 2016-06-15 | 2024-01-30 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
IL297095A (en) * | 2016-07-08 | 2022-12-01 | Univ Pennsylvania | Methods and preparations for the treatment of disorders and diseases involving rdh12 |
EP3485005A1 (en) * | 2016-07-12 | 2019-05-22 | The University Of Manchester | Gene therapy |
KR102508820B1 (ko) | 2016-07-29 | 2023-03-13 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법 |
AU2017313064B2 (en) * | 2016-08-16 | 2024-05-02 | The University Of North Carolina At Chapel Hill | Methods and compositions for targeted gene transfer |
JP2019524162A (ja) * | 2016-08-18 | 2019-09-05 | 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 | モジュラーAAV送達システムによるCRISPR−Casゲノム編集 |
US20210283222A1 (en) * | 2016-08-19 | 2021-09-16 | Calimmune, Inc. | Methods and compositions for treating canine conditions using recombinant self-complementary adeno-associated virus |
CA3073137A1 (en) * | 2016-08-19 | 2018-02-22 | Jeffrey S. Bartlett | Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus |
US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
WO2018064098A1 (en) | 2016-09-28 | 2018-04-05 | Cohbar, Inc. | Therapeutic mots-c related peptides |
EP3518985A4 (en) * | 2016-09-29 | 2020-08-05 | University of Florida Research Foundation, Incorporated | AAVRH.10 VARIANTS WITH HOST ANTIBODY EXHAUST CAPACITIES AND MODIFIED TISSUE TARGETING PROPERTIES |
KR20190075964A (ko) | 2016-10-13 | 2019-07-01 | 유니버시티 오브 매사추세츠 | Aav 캡시드 설계 |
CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
EP3551650A4 (en) | 2016-12-07 | 2020-07-08 | The University of Florida Research Foundation Incorporated | IL-1RA-CDNAS |
WO2018145009A1 (en) * | 2017-02-06 | 2018-08-09 | University Of Tennessee Research Foundation | Dna-zyme based methods & compositions for treating huntington's disease |
US11117930B2 (en) | 2017-02-23 | 2021-09-14 | Adrx, Inc. | Peptide inhibitors of transcription factor aggregation |
JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
CA3059891A1 (en) | 2017-04-14 | 2018-10-18 | National Taiwan University | Gene therapy for aadc deficiency |
WO2018200419A1 (en) * | 2017-04-23 | 2018-11-01 | The Trustees Of The University Of Pennsylvania | Viral vectors comprising engineered aav capsids and compositions containing the same |
MX2019013172A (es) | 2017-05-05 | 2020-09-07 | Voyager Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de huntington. |
AU2018264996A1 (en) | 2017-05-09 | 2019-12-05 | University Of Massachusetts | Methods of treating Amyotrophic Lateral Sclerosis (ALS) |
CA3061968A1 (en) * | 2017-05-10 | 2018-11-15 | Massachusetts Eye And Ear Infirmary | Methods and compositions for modifying assembly-activating protein (aap)-dependence of viruses |
CN110997693A (zh) | 2017-06-07 | 2020-04-10 | 阿德克斯公司 | τ聚集抑制剂 |
KR20200022372A (ko) * | 2017-06-30 | 2020-03-03 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법 |
EP3662060A2 (en) | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
CA3059995A1 (en) | 2017-08-28 | 2019-03-07 | The Regents Of The University Of California | Adeno-associated virus capsid variants and methods of use thereof |
CN111448321A (zh) | 2017-09-22 | 2020-07-24 | 马萨诸塞大学 | Sod1双表达载体及其用途 |
CA3076191A1 (en) | 2017-09-22 | 2019-03-28 | Genzyme Corporation | Variant rnai |
US11723988B2 (en) | 2017-10-20 | 2023-08-15 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector |
US11464803B2 (en) | 2017-11-14 | 2022-10-11 | Arcellx, Inc. | D-domain containing polypeptides and uses thereof |
WO2019099440A1 (en) | 2017-11-14 | 2019-05-23 | Arcellx, Inc. | Multifunctional immune cell therapies |
CN117752822A (zh) * | 2017-11-15 | 2024-03-26 | 密歇根大学董事会 | 包括rdh12编码区的病毒载体和治疗视网膜营养不良的方法 |
CN111726985B (zh) * | 2017-11-15 | 2022-06-10 | 弗里德里克·米谢尔生物医学研究所 | 灵长类动物视网膜色素上皮细胞特异性启动子 |
AU2018372763A1 (en) | 2017-11-21 | 2020-05-28 | Bayer Healthcare Llc | Materials and methods for treatment of autosomal dominant Retinitis Pigmentosa |
CN108085316B (zh) * | 2017-12-25 | 2021-02-09 | 中国人民解放军第四军医大学 | 一种通过抑制微小核糖核酸来促进小鼠视网膜前体细胞体外扩增的方法 |
CN112272672A (zh) | 2018-04-03 | 2021-01-26 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
US20210230229A1 (en) * | 2018-05-15 | 2021-07-29 | President And Fellows Of Harvard College | Viral vectors exhibiting improved gene delivery properties |
US20210230632A1 (en) | 2018-05-15 | 2021-07-29 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
MA52631A (fr) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics Inc | Compositions et méthodes pour le traitement de la maladie de parkinson |
EP3793615A2 (en) | 2018-05-16 | 2021-03-24 | Voyager Therapeutics, Inc. | Directed evolution of aav to improve tropism for cns |
WO2019232517A1 (en) * | 2018-06-01 | 2019-12-05 | University Of Florida Research Foundation, Incorporated | Compositions and methods for treatment of dominant retinitis pigmentosa |
BR112021001980A2 (pt) | 2018-08-03 | 2021-05-04 | Genzyme Corporation | rnai variante contra alfa-sinucleína |
CN109022487A (zh) * | 2018-08-15 | 2018-12-18 | 上海市第人民医院 | 一种表达p65的基因载体及其用于治疗视网膜神经节细胞变性的基因治疗药物 |
WO2020068990A1 (en) | 2018-09-26 | 2020-04-02 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
AU2019346655A1 (en) | 2018-09-28 | 2021-05-06 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
WO2020077250A1 (en) * | 2018-10-12 | 2020-04-16 | Genzyme Corporation | Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy |
WO2020077165A1 (en) | 2018-10-12 | 2020-04-16 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
UY38407A (es) | 2018-10-15 | 2020-05-29 | Novartis Ag | Anticuerpos estabilizadores de trem2 |
US20230061069A1 (en) * | 2018-11-13 | 2023-03-02 | Jayandharan Giridhara Rao | A process for producing a plurality of sumoylation target-site modified aav vector and product thereof |
SG11202106855YA (en) * | 2019-01-04 | 2021-07-29 | Ultragenyx Pharmaceutical Inc | Gene therapy constructs for treating wilson disease |
AU2020208467A1 (en) | 2019-01-18 | 2021-08-05 | Voyager Therapeutics, Inc. | Methods and systems for producing AAV particles |
CA3125294A1 (en) * | 2019-01-23 | 2020-07-30 | University Of Florida Research Foundation, Incorporated | Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design |
MX2021008979A (es) | 2019-01-28 | 2021-11-12 | Cohbar Inc | Peptidos terapeuticos. |
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 |
WO2020210713A1 (en) * | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
US20240124889A1 (en) | 2019-05-07 | 2024-04-18 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
WO2020232005A1 (en) * | 2019-05-13 | 2020-11-19 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Polymer-based implant for retinal therapy and methods of making and using the same |
US20220226507A1 (en) * | 2019-05-17 | 2022-07-21 | Research Institute At Nationwide Children's Hospital | Optimized gene therapy targeting retinal cells |
CA3142932A1 (en) * | 2019-06-10 | 2020-12-17 | Homology Medicines, Inc. | Adeno-associated virus compositions for arsa gene transfer and methods of use thereof |
MX2021016014A (es) * | 2019-06-27 | 2022-04-06 | Univ Florida | Mejora de la transducción mediada por aav en los tejidos oculares con ácido hialurónico. |
US11446084B2 (en) | 2019-07-12 | 2022-09-20 | Neuralink Corp. | Laser drilling of pia mater |
MX2022000551A (es) * | 2019-07-15 | 2022-05-18 | Meiragtx Uk Ii Ltd | Proteínas de la cápside de aav modificadas para el tratamiento de una enfermedad artrítica. |
CN110295151A (zh) * | 2019-07-17 | 2019-10-01 | 博鑫仪器(天津)有限公司 | 一类腺依赖病毒病毒重组质粒、重组病毒及构建方法 |
EP4017873A4 (en) * | 2019-08-23 | 2023-12-20 | Duke University | COMPOSITIONS AND METHODS FOR THE TREATMENT OF PATHOLOGICAL PAIN AND ITCH |
WO2021046155A1 (en) | 2019-09-03 | 2021-03-11 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
US20220324921A1 (en) * | 2019-09-03 | 2022-10-13 | University Of Cincinnati | Methods and compositions for the treatment of als |
IL292297A (en) | 2019-10-17 | 2022-06-01 | Stridebio Inc | Adeno-associated viral vectors for the treatment of Niemann-Pick type c disease |
US20220389457A1 (en) * | 2019-10-23 | 2022-12-08 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
CN113025618B (zh) * | 2019-12-24 | 2024-02-06 | 朗信启昇(苏州)生物制药有限公司 | 一种x连锁遗传性视网膜劈裂症的基因治疗方案与应用 |
TW202142552A (zh) | 2020-01-29 | 2021-11-16 | 美商健臻公司 | 用於眼部基因療法的經修飾的腺相關病毒衣殼蛋白及其使用方法 |
KR20220150906A (ko) * | 2020-02-07 | 2022-11-11 | 더 트러스티스 오브 컬럼비아 유니버시티 인 더 시티 오브 뉴욕 | 신경변성의 발병 지연 또는 치료를 위한 대사체의 재프로그래밍 |
CN115210248A (zh) * | 2020-02-18 | 2022-10-18 | 北卡罗来纳大学查佩尔希尔分校 | Aav衣壳-启动子相互作用和细胞选择性基因表达 |
EP4110913A4 (en) * | 2020-02-28 | 2024-07-24 | Univ Massachusetts | OLIGONUCLEOTIDES FOR PRNP MODULATION |
CN115379834A (zh) * | 2020-03-11 | 2022-11-22 | 马萨诸塞眼科耳科诊所 | 用于nmnat1相关视网膜变性的基因疗法 |
KR20230113689A (ko) * | 2020-03-31 | 2023-08-01 | 유니버시티 오브 매사추세츠 | Aav 캡시드 변이체 및 그의 용도 |
IL297200A (en) | 2020-04-15 | 2022-12-01 | Voyager Therapeutics Inc | tau binding compounds |
BR112022023106A2 (pt) | 2020-05-13 | 2023-01-17 | Voyager Therapeutics Inc | Redirecionamento de tropismo de capsídeos de aav |
WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
WO2021252989A2 (en) * | 2020-06-13 | 2021-12-16 | Oculogenex Inc. | Aav-mediated gene transfer for retinopathy |
CN113952472A (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 |
WO2022017630A1 (en) | 2020-07-23 | 2022-01-27 | Ucl Business Ltd | GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF |
BR112023001456A2 (pt) | 2020-07-27 | 2023-04-11 | Voyager Therapeutics Inc | Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase |
WO2022026410A2 (en) | 2020-07-27 | 2022-02-03 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
BR112023001336A2 (pt) | 2020-08-05 | 2023-02-14 | Spacecraft Seven Llc | Terapia gênica de csrp3 (proteína 3 rica em cisteína e glicina) |
EP4192487A1 (en) | 2020-08-07 | 2023-06-14 | Spacecraft Seven, LLC | Plakophilin-2 (pkp2) gene therapy using aav vector |
US20230330267A1 (en) * | 2020-09-04 | 2023-10-19 | Ohio State Innovation Foundation | Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain |
US20230392166A1 (en) * | 2020-10-21 | 2023-12-07 | Wisconsin Alumni Research Foundation | Anti-apoptotic vector and method of using the same |
US20220160825A1 (en) * | 2020-11-25 | 2022-05-26 | The Penn State Research Foundation | Brain repair after traumatic brain injury through neurod1-mediated astrocyte-to-neuron conversion |
JP2024507860A (ja) * | 2021-02-22 | 2024-02-21 | ザ・リージエンツ・オブ・ザ・ユニバーシテイ・オブ・コロラド、ア・ボデイー・コーポレイト | 眼病態に対するウイルスベクターベースの遺伝子療法 |
CN115044614B (zh) * | 2021-03-09 | 2023-10-20 | 上海目镜生物医药科技有限公司 | 一种用于基因靶向与表达的aav-8型血清型的改造型载体及其构建方法及应用 |
CA3216809A1 (en) * | 2021-04-26 | 2022-11-03 | Brendan BEAHM | Compositions and methods for treating ngyl1 deficiency |
CN115997012B (zh) * | 2021-09-06 | 2024-03-08 | 辉大(上海)生物科技有限公司 | Rpe65相关眼睛疾病和障碍的治疗 |
WO2023091949A2 (en) | 2021-11-17 | 2023-05-25 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
AU2022397707A1 (en) | 2021-11-29 | 2024-06-13 | Shanghai Regenelead Therapies Co., Ltd. | Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease |
WO2023183623A1 (en) * | 2022-03-25 | 2023-09-28 | Regenxbio Inc. | Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy |
WO2023211857A1 (en) | 2022-04-25 | 2023-11-02 | Lineage Cell Therapeutics, Inc. | Methods and compositions for treating vision loss |
WO2023212521A2 (en) * | 2022-04-25 | 2023-11-02 | Myrtelle Inc. | Treatments of disorders of myelin |
WO2023214346A1 (en) | 2022-05-06 | 2023-11-09 | Novartis Ag | Novel recombinant aav vp2 fusion polypeptides |
WO2023240220A1 (en) * | 2022-06-09 | 2023-12-14 | The University Of North Carolina At Chapel Hill | Aav-sgsh vectors for treatment of mucopolysaccharidosis iiia |
WO2023240236A1 (en) | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of spinal muscular atrophy related disorders |
WO2024008950A1 (en) | 2022-07-08 | 2024-01-11 | Ospedale San Raffaele S.R.L. | Transgene cassettes |
EP4303226A1 (en) | 2022-07-08 | 2024-01-10 | Ospedale San Raffaele S.r.l. | Transgene cassettes and epigenetic silencers for the treatment of disorders |
WO2024015794A2 (en) * | 2022-07-12 | 2024-01-18 | The Research Institute At Nationwide Children's Hospital | Adeno-associated virus gene therapy products and methods |
Family Cites Families (32)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4692147A (en) | 1980-04-02 | 1987-09-08 | Medtronic, Inc. | Drug administration device |
US5735815A (en) | 1993-07-26 | 1998-04-07 | Sentinel Medical, Inc. | Method of using fluid jet surgical cutting tool |
WO1998010088A1 (en) | 1996-09-06 | 1998-03-12 | Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
CA2287508A1 (en) | 1997-04-21 | 1998-10-29 | The University Of Florida | Materials and methods for ribozyme treatment of retinal diseases |
US6042579A (en) | 1997-04-30 | 2000-03-28 | Medtronic, Inc. | Techniques for treating neurodegenerative disorders by infusion of nerve growth factors into the brain |
US6995006B2 (en) | 1997-09-05 | 2006-02-07 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
ES2324540T3 (es) | 1998-05-27 | 2009-08-10 | Genzyme Corporation | Vectores aav para la fabricacion de medicamentos para la administracion potenciada por conveccion. |
CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs |
JP4860886B2 (ja) | 2000-06-01 | 2012-01-25 | ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 二本鎖パルボウイルスベクター |
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 |
GB2389791B (en) | 2002-04-30 | 2006-12-13 | Steven Gill | Implantable drug delivery pump |
WO2004027019A2 (en) | 2002-05-01 | 2004-04-01 | University Of Florida Research Foundation, Inc. | Improved raav expression systems for genetic modification of specific capsid proteins |
WO2004099423A1 (en) * | 2003-05-01 | 2004-11-18 | University Of Florida Research Foundation, Inc. | Vp2-modified raav vectors and uses thereof |
US7261544B2 (en) | 2003-05-21 | 2007-08-28 | Genzyme Corporation | Methods for producing preparations of recombinant AAV virions substantially free of empty capsids |
EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
ES2629380T3 (es) * | 2003-06-19 | 2017-08-09 | Genzyme Corporation | Viriones de AAV con inmunorreactividad reducida y usos de los mismos |
EP1807009B1 (en) | 2004-10-05 | 2014-11-26 | Genzyme Corporation | Stepped cannula |
ES2525143T3 (es) * | 2005-04-07 | 2014-12-18 | The Trustees Of The University Of Pennsylvania | Cápsides de AAVrh.64 modificado, composiciones que las contienen y usos de las mismas |
WO2007024841A2 (en) | 2005-08-23 | 2007-03-01 | The Regents Of The University Of California | Reflux resistant cannula and system for chronic delivery of therapeutic agents using convection-enhanced delivery |
WO2007089632A2 (en) * | 2006-01-27 | 2007-08-09 | The University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
DK2019683T4 (da) | 2006-04-25 | 2022-08-29 | Univ California | Indgivelse af vækstfaktorer til behandling af CNS-lidelser |
US20070259031A1 (en) | 2006-04-26 | 2007-11-08 | The Regents Of The University Of California | Compositions and methods for convection enhanced delivery of high molecular weight neurotherapeutics |
WO2008027084A2 (en) | 2006-04-28 | 2008-03-06 | The Trustees Of The University Of Pennsylvania | Modified aav vectors having reduced capsid immunogenicity and use thereof |
US10065021B2 (en) | 2007-05-17 | 2018-09-04 | Medgenesis Therapeutix, Inc. | Convection-enhanced delivery catheter with removable stiffening member and method for using same |
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 |
CA2751266A1 (en) | 2009-01-29 | 2010-08-05 | Krystof Bankiewicz | Methods for distributing high levels of therapeutic agent throughout the cortex to treat neurological disorders |
WO2010093784A2 (en) * | 2009-02-11 | 2010-08-19 | The University Of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
KR101812813B1 (ko) | 2009-06-16 | 2017-12-27 | 젠자임 코포레이션 | 재조합 aav 벡터에 대한 개선된 정제 방법 |
CA3049237C (en) | 2010-04-23 | 2024-06-11 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
EP2699270B1 (en) | 2011-04-22 | 2017-06-21 | The Regents of The University of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
TWI702955B (zh) * | 2012-05-15 | 2020-09-01 | 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) |
-
2015
- 2015-05-02 PT PT157233867T patent/PT3137497T/pt unknown
- 2015-05-02 CR CR20160555A patent/CR20160555A/es unknown
- 2015-05-02 SG SG11201607991WA patent/SG11201607991WA/en unknown
- 2015-05-02 JP JP2016564301A patent/JP6741591B2/ja active Active
- 2015-05-02 DK DK15723386.7T patent/DK3137497T3/da active
- 2015-05-02 EA EA201692206A patent/EA034355B1/ru not_active IP Right Cessation
- 2015-05-02 RS RS20210849A patent/RS62078B1/sr unknown
- 2015-05-02 MX MX2016014220A patent/MX2016014220A/es unknown
- 2015-05-02 KR KR1020167033392A patent/KR20160147974A/ko not_active Application Discontinuation
- 2015-05-02 EP EP21159585.5A patent/EP3913061A1/en active Pending
- 2015-05-02 PL PL15723386.7T patent/PL3137497T4/pl unknown
- 2015-05-02 SG SG10201912977RA patent/SG10201912977RA/en unknown
- 2015-05-02 ES ES15723386T patent/ES2879636T3/es active Active
- 2015-05-02 IL IL292951A patent/IL292951B1/en unknown
- 2015-05-02 US US15/308,335 patent/US10982228B2/en active Active
- 2015-05-02 SI SI201531642T patent/SI3137497T1/sl unknown
- 2015-05-02 KR KR1020247006248A patent/KR20240032156A/ko active Application Filing
- 2015-05-02 SG SG10201809739QA patent/SG10201809739QA/en unknown
- 2015-05-02 HU HUE15723386A patent/HUE054768T2/hu unknown
- 2015-05-02 LT LTEP15723386.7T patent/LT3137497T/lt unknown
- 2015-05-02 CN CN202111211678.0A patent/CN114231563A/zh active Pending
- 2015-05-02 EP EP15723386.7A patent/EP3137497B1/en active Active
- 2015-05-02 CN CN201580035996.2A patent/CN106661591B/zh active Active
- 2015-05-02 MY MYPI2016703542A patent/MY188987A/en unknown
- 2015-05-02 WO PCT/US2015/028966 patent/WO2015168666A2/en active Application Filing
- 2015-05-02 IL IL248102A patent/IL248102B/en unknown
- 2015-05-02 AU AU2015252797A patent/AU2015252797C1/en active Active
- 2015-05-02 PE PE2016002164A patent/PE20170260A1/es unknown
- 2015-05-02 BR BR112016025263-2A patent/BR112016025263A2/pt active Search and Examination
- 2015-05-02 CA CA2946593A patent/CA2946593A1/en active Pending
- 2015-05-04 TW TW104114126A patent/TWI686476B/zh active
- 2015-05-04 UY UY0001036106A patent/UY36106A/es not_active Application Discontinuation
-
2016
- 2016-09-30 ZA ZA2016/06763A patent/ZA201606763B/en unknown
- 2016-10-13 PH PH12016502046A patent/PH12016502046A1/en unknown
- 2016-10-19 DO DO2016000280A patent/DOP2016000280A/es unknown
- 2016-10-24 GT GT201600227A patent/GT201600227A/es unknown
- 2016-10-25 CL CL2016002713A patent/CL2016002713A1/es unknown
- 2016-10-28 MX MX2022012490A patent/MX2022012490A/es unknown
-
2018
- 2018-01-19 CL CL2018000170A patent/CL2018000170A1/es unknown
-
2020
- 2020-06-24 JP JP2020108310A patent/JP7174011B2/ja active Active
-
2021
- 2021-01-15 AU AU2021200242A patent/AU2021200242B9/en active Active
- 2021-03-05 US US17/194,009 patent/US20210189430A1/en active Pending
- 2021-06-29 HR HRP20211024TT patent/HRP20211024T1/hr unknown
-
2022
- 2022-11-02 JP JP2022175918A patent/JP2023011812A/ja active Pending
-
2023
- 2023-12-20 AU AU2023285817A patent/AU2023285817A1/en active Pending
Also Published As
Similar Documents
Publication | Publication Date | Title |
---|---|---|
ZA201606763B (en) | Aav vectors for retinal and cns gene therapy | |
IL251468A0 (en) | aav-based gene therapy | |
HK1257801A1 (zh) | 用於基因治療的腺伴隨病毒載體的鞘內給藥 | |
IL284577A (en) | Garden healing | |
SG11201703193XA (en) | Treatment for depression and depressive disorders | |
HK1258518A1 (zh) | 用於眼病的基因療法 | |
GB201407322D0 (en) | Gene therapy | |
RS62619B1 (sr) | Genska terapija | |
LT3133923T (lt) | Aav pagrindo genų terapija, skirta išsėtinei sklerozei | |
GB201707212D0 (en) | Gene therapy for ciliopathies | |
GB201701968D0 (en) | Gene therapy | |
HUE055902T2 (hu) | Vektorok és eljárások regeneratív terápiára | |
IL275613A (en) | Gene therapy for eosinophilic disorders | |
GB201513151D0 (en) | Functional gene replacement therapy | |
IL263085A (en) | Garden healing | |
GB201616821D0 (en) | Gene therapy | |
GB201612105D0 (en) | Gene therapy | |
GB201612104D0 (en) | Gene therapy | |
GB201704634D0 (en) | Gene therapy |