SG11202011145UA - Liver targeting adeno-associated viral vectors - Google Patents
Liver targeting adeno-associated viral vectorsInfo
- Publication number
- SG11202011145UA SG11202011145UA SG11202011145UA SG11202011145UA SG11202011145UA SG 11202011145U A SG11202011145U A SG 11202011145UA SG 11202011145U A SG11202011145U A SG 11202011145UA SG 11202011145U A SG11202011145U A SG 11202011145UA SG 11202011145U A SG11202011145U A SG 11202011145UA
- Authority
- SG
- Singapore
- Prior art keywords
- viral vectors
- associated viral
- liver targeting
- targeting adeno
- adeno
- Prior art date
Links
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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
-
- 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
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
-
- 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
-
- 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
-
- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862671265P | 2018-05-14 | 2018-05-14 | |
PCT/US2019/032097 WO2019222136A2 (en) | 2018-05-14 | 2019-05-14 | Novel liver targeting adeno-associated viral vectors |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11202011145UA true SG11202011145UA (en) | 2020-12-30 |
Family
ID=66821373
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11202011145UA SG11202011145UA (en) | 2018-05-14 | 2019-05-14 | Liver targeting adeno-associated viral vectors |
Country Status (16)
Country | Link |
---|---|
US (1) | US11821008B2 (en) |
EP (1) | EP3794016A2 (en) |
JP (1) | JP2021522842A (en) |
KR (1) | KR20210010496A (en) |
CN (1) | CN112424217A (en) |
AR (1) | AR115097A1 (en) |
AU (1) | AU2019268302A1 (en) |
BR (1) | BR112020023292A2 (en) |
CA (1) | CA3100001A1 (en) |
CL (2) | CL2020002960A1 (en) |
MX (1) | MX2020012170A (en) |
PE (1) | PE20210113A1 (en) |
PH (1) | PH12020551938A1 (en) |
SG (1) | SG11202011145UA (en) |
TW (1) | TW202005978A (en) |
WO (1) | WO2019222136A2 (en) |
Families Citing this family (15)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JP6994018B2 (en) | 2016-07-26 | 2022-01-14 | バイオマリン ファーマシューティカル インコーポレイテッド | New adeno-associated virus capsid protein |
SG11202010830WA (en) | 2018-05-09 | 2020-11-27 | Biomarin Pharm Inc | Methods of treating phenylketonuria |
US20200362368A1 (en) | 2019-05-14 | 2020-11-19 | Biomarin Pharmaceutical Inc. | Methods of redosing gene therapy vectors |
WO2021236908A2 (en) | 2020-05-20 | 2021-11-25 | Biomarin Pharmaceutical Inc. | Use of regulatory proteins for the production of adeno-associated virus |
CN116121274A (en) * | 2020-07-29 | 2023-05-16 | 北京三诺佳邑生物技术有限责任公司 | Acquisition and application of liver targeting novel adeno-associated virus |
AU2021372262A1 (en) | 2020-11-02 | 2023-06-01 | Biomarin Pharmaceutical Inc. | Process for enriching adeno-associated virus |
WO2023034990A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034980A1 (en) | 2021-09-03 | 2023-03-09 | Bomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034997A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034996A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034994A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034989A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023139496A1 (en) * | 2022-01-21 | 2023-07-27 | Pfizer Inc. | Gene therapy for gaucher disease |
WO2024064863A2 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors |
WO2024064856A1 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of cardiomyopathy with aav gene therapy vectors |
Family Cites Families (86)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
EP0127839B1 (en) | 1983-05-27 | 1992-07-15 | THE TEXAS A&M UNIVERSITY SYSTEM | Method for producing a recombinant baculovirus expression vector |
ZA848495B (en) | 1984-01-31 | 1985-09-25 | Idaho Res Found | Production of polypeptides in insect cells |
US4994371A (en) | 1987-08-28 | 1991-02-19 | Davie Earl W | DNA preparation of Christmas factor and use of DNA sequences |
US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
US6309853B1 (en) | 1994-08-17 | 2001-10-30 | The Rockfeller University | Modulators of body weight, corresponding nucleic acids and proteins, and diagnostic and therapeutic uses thereof |
RU2273645C9 (en) | 1994-08-17 | 2006-11-27 | Дзе Рокефеллер Юниверсити | Obesity polypeptide (ob) (variants), its analogue (variants), and fused protein (variants), nucleic acid isolated molecule, dna molecule, cloning recombinant vector, expression recombinant vector, pharmaceutical composition, monoclonal and polyclonal antibody |
WO1998009524A1 (en) | 1996-09-06 | 1998-03-12 | Chiron Corporation | Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors |
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 |
US6531298B2 (en) | 1997-07-21 | 2003-03-11 | The University Of North Carolina At Chapel Hill | Factor IX antihemophilic factor with increased clotting activity |
JP4169478B2 (en) | 1998-04-21 | 2008-10-22 | マイクロメット アーゲー | CD19xCD3-specific polypeptide and uses thereof |
JP4060531B2 (en) | 1998-05-28 | 2008-03-12 | アメリカ合衆国 | AAV5 vectors and uses thereof |
US6221349B1 (en) | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
US7056502B2 (en) | 2000-04-28 | 2006-06-06 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids |
WO2003033710A1 (en) | 2001-10-16 | 2003-04-24 | National Institute Of Advanced Industrial Science And Technology | Novel n-acetylglucosamine transferase, nucleic acid encoding the same, antibody against the same and use thereof for diagnosing cancer or tumor |
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 |
NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
JP4769417B2 (en) | 2001-12-17 | 2011-09-07 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Adeno-associated virus (AAV) serotype 9 sequences, vectors containing the same and uses thereof |
AU2003212708A1 (en) | 2002-03-05 | 2003-09-16 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
US20030198620A1 (en) | 2002-04-16 | 2003-10-23 | Keiya Ozawa | Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions |
US20040142416A1 (en) | 2002-04-30 | 2004-07-22 | Laipis Philip J. | Treatment for phenylketonuria |
ES2648241T3 (en) | 2003-09-30 | 2017-12-29 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus clades (AAV), sequences, vectors containing the same, and uses thereof |
JP5184783B2 (en) | 2003-11-17 | 2013-04-17 | バイオマリン ファーマシューティカル インコーポレイテッド | Tetrahydrobiopterin and method for producing tetrahydrobiopterin analog |
WO2006073496A2 (en) | 2004-07-30 | 2006-07-13 | Targeted Genetics Corporation | Recombinant aav based vaccine methods |
BRPI0517088A (en) | 2004-11-17 | 2008-09-30 | Biomarin Pharm Inc | stable tablet formulation |
CA2591544A1 (en) | 2004-12-15 | 2006-06-22 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
ES2525067T3 (en) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an AAV vector |
WO2006119432A2 (en) * | 2005-04-29 | 2006-11-09 | The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services | Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes |
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 |
US7531341B1 (en) | 2006-06-12 | 2009-05-12 | Biomarin Pharmaceutical Inc. | Compositions of prokaryotic phenylalanine ammonia-lyase and methods of using compositions thereof |
US7534595B2 (en) | 2006-06-12 | 2009-05-19 | Biomarin Pharmaceutical Inc. | Compositions of prokaryotic phenylalanine ammonia-lyase and methods of using compositions thereof |
WO2008088895A2 (en) | 2007-01-18 | 2008-07-24 | University Of Missouri-Columbia | Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma |
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 |
AR066354A1 (en) | 2007-05-01 | 2009-08-12 | Genzyme Corp | SYSTEMATIC THERAPY WITH INSULIN TYPE GROWTH FACTOR-1 REDUCES DIABETIC PERIPHERAL NEUROPATHY AND IMPROVES RENAL FUNCTION IN DIABETIC NEPHROPATIA |
US7560263B2 (en) | 2007-08-17 | 2009-07-14 | Biomarin Pharmaceutical Inc. | Compositions of prokaryotic phenylalanine ammonia-lyase and methods of treating cancer using compositions thereof |
WO2009088979A1 (en) | 2008-01-07 | 2009-07-16 | Biomarin Pharmaceutical Inc. | Method of synthesizing tetrahydrobiopterin |
KR20100113112A (en) | 2008-01-15 | 2010-10-20 | 아보트 러보러터리즈 | Improved mammalian expression vectors and uses thereof |
US7943379B2 (en) | 2008-04-30 | 2011-05-17 | Nationwide Children's Hospital, Inc. | Production of rAAV in vero cells using particular adenovirus helpers |
WO2010014225A2 (en) | 2008-07-30 | 2010-02-04 | Biomarin Pharmaceutical Inc. | Assays for detection of phenylalanine ammonia-lyase and antibodies to phenylalanine ammonia-lyase |
EP4219547A3 (en) | 2008-09-15 | 2023-10-18 | uniQure biopharma B.V. | Factor ix polypeptide mutant, its uses and a method for its production |
PL2425000T3 (en) | 2009-04-30 | 2019-08-30 | The Trustees Of The University Of Pennsylvania | Compositions for targeting conducting airway cells comprising adeno-associated virus constructs |
GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
CN103038343A (en) | 2010-03-23 | 2013-04-10 | 英特瑞克斯顿股份有限公司 | Vectors conditionally expressing therapeutic proteins, host cells comprising the vectors, and uses thereof. |
CA2793633A1 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
FR2977562B1 (en) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | SEALED AND THERMALLY INSULATING TANK INTEGRATED IN A CARRIER STRUCTURE |
CA2864879C (en) | 2012-02-17 | 2021-07-20 | The Children's Hospital Of Philadelphia | Aav vector compositions and methods for gene transfer to cells, organs and tissues |
GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
JP6591956B2 (en) | 2013-03-15 | 2019-10-16 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Compositions and methods for treating MPS1 |
CA2907799A1 (en) | 2013-05-31 | 2014-12-04 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
WO2015013313A2 (en) | 2013-07-22 | 2015-01-29 | The Children's Hospital Of Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
PL3044231T3 (en) | 2013-09-12 | 2021-01-11 | Biomarin Pharmaceutical Inc. | Aav vectors comprising a gene encoding factor viii |
HUE052676T2 (en) | 2013-10-11 | 2021-05-28 | Massachusetts Eye & Ear Infirmary | Methods of predicting ancestral virus sequences and uses thereof |
EP3060258A1 (en) | 2013-10-22 | 2016-08-31 | Shire Human Genetic Therapies, Inc. | Mrna therapy for phenylketonuria |
SI3116900T1 (en) | 2014-03-09 | 2021-02-26 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency |
EP2960336A1 (en) | 2014-06-27 | 2015-12-30 | Genethon | Efficient systemic treatment of dystrophic muscle pathologies |
CN114344275A (en) | 2014-07-02 | 2022-04-15 | 川斯勒佰尔公司 | Encapsulation of messenger RNA |
EP3194600B1 (en) | 2014-07-26 | 2019-08-28 | Consiglio Nazionale Delle Ricerche | Compositions and methods for treatment of muscular dystrophy |
ES2856090T3 (en) | 2014-09-24 | 2021-09-27 | Hope City | Adeno-associated virus vector variants for high-efficiency genomic editing and their methods |
EP3245220B1 (en) * | 2015-01-14 | 2023-09-20 | The University of North Carolina at Chapel Hill | Methods and compositions for targeted gene transfer |
AU2016235163B2 (en) * | 2015-03-24 | 2022-03-24 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
GB201507842D0 (en) | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
EP3872085B1 (en) | 2015-07-30 | 2023-03-01 | Massachusetts Eye & Ear Infirmary | Ancestral aav sequences and uses thereof |
BR112018006074A2 (en) | 2015-09-24 | 2018-10-09 | Biomarin Pharm Inc | adeno-associated virus factor viii vectors, associated viral particles, and therapeutic formulations comprising the same |
WO2017066764A2 (en) | 2015-10-16 | 2017-04-20 | William Marsh Rice University | Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses |
BR112018008519A2 (en) | 2015-10-28 | 2018-11-06 | Sangamo Therapeutics Inc | liver-specific constructs, factor viii expression cassettes and methods of use thereof |
PE20231949A1 (en) | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTS OF FACTOR VIII REDUCED WITH CpG, COMPOSITIONS AND METHODS AND USES FOR THE TREATMENT OF HEMOSTASY DISORDERS |
SG11201808812RA (en) | 2016-04-15 | 2018-11-29 | Univ Pennsylvania | Novel aav8 mutant capsids and compositions containing same |
JP6994018B2 (en) | 2016-07-26 | 2022-01-14 | バイオマリン ファーマシューティカル インコーポレイテッド | New adeno-associated virus capsid protein |
EP3500676A4 (en) | 2016-08-16 | 2020-07-01 | The University of North Carolina at Chapel Hill | Methods and compositions for targeted gene transfer |
CA3042689A1 (en) | 2016-11-04 | 2018-07-12 | Baxalta Incorporated | Adeno-associated virus formulations |
BR112019013576A2 (en) | 2016-12-30 | 2020-02-04 | Univ Pennsylvania | gene therapy for the treatment of phenylketonuria |
US20210228738A1 (en) | 2017-07-17 | 2021-07-29 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins |
US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
AU2019215150A1 (en) | 2018-02-01 | 2020-09-24 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
SG11202010830WA (en) | 2018-05-09 | 2020-11-27 | Biomarin Pharm Inc | Methods of treating phenylketonuria |
US20200069819A1 (en) | 2018-05-14 | 2020-03-05 | Biomarin Pharmaceutical Inc. | Stable expression of aav vectors in juvenile subjects |
US20210355454A1 (en) | 2018-07-24 | 2021-11-18 | Voyager Therapeutics, Inc. | Systems and methods for producing gene therapy formulations |
JP2022529002A (en) | 2019-04-19 | 2022-06-16 | レジェンクスバイオ インコーポレーテッド | Adeno-associated virus vector preparation and method |
US20200362368A1 (en) | 2019-05-14 | 2020-11-19 | Biomarin Pharmaceutical Inc. | Methods of redosing gene therapy vectors |
AR122409A1 (en) | 2020-04-03 | 2022-09-07 | Biomarin Pharm Inc | TREATMENT OF PHENYLKETONURIA WITH AAV AND THERAPEUTIC FORMULATIONS |
WO2023034994A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034996A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034990A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034997A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034980A1 (en) | 2021-09-03 | 2023-03-09 | Bomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034989A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
-
2019
- 2019-05-13 TW TW108116478A patent/TW202005978A/en unknown
- 2019-05-14 WO PCT/US2019/032097 patent/WO2019222136A2/en unknown
- 2019-05-14 CN CN201980047255.4A patent/CN112424217A/en active Pending
- 2019-05-14 AR ARP190101289A patent/AR115097A1/en unknown
- 2019-05-14 JP JP2020564217A patent/JP2021522842A/en active Pending
- 2019-05-14 EP EP19729947.2A patent/EP3794016A2/en not_active Withdrawn
- 2019-05-14 MX MX2020012170A patent/MX2020012170A/en unknown
- 2019-05-14 SG SG11202011145UA patent/SG11202011145UA/en unknown
- 2019-05-14 CA CA3100001A patent/CA3100001A1/en active Pending
- 2019-05-14 PE PE2020001846A patent/PE20210113A1/en unknown
- 2019-05-14 BR BR112020023292-0A patent/BR112020023292A2/en unknown
- 2019-05-14 US US16/411,848 patent/US11821008B2/en active Active
- 2019-05-14 AU AU2019268302A patent/AU2019268302A1/en active Pending
- 2019-05-14 KR KR1020207035559A patent/KR20210010496A/en not_active Application Discontinuation
-
2020
- 2020-11-11 PH PH12020551938A patent/PH12020551938A1/en unknown
- 2020-11-13 CL CL2020002960A patent/CL2020002960A1/en unknown
-
2023
- 2023-01-26 CL CL2023000264A patent/CL2023000264A1/en unknown
Also Published As
Publication number | Publication date |
---|---|
CL2023000264A1 (en) | 2023-09-01 |
EP3794016A2 (en) | 2021-03-24 |
US11821008B2 (en) | 2023-11-21 |
CL2020002960A1 (en) | 2021-04-30 |
PE20210113A1 (en) | 2021-01-19 |
US20200024579A1 (en) | 2020-01-23 |
JP2021522842A (en) | 2021-09-02 |
TW202005978A (en) | 2020-02-01 |
AR115097A1 (en) | 2020-11-25 |
KR20210010496A (en) | 2021-01-27 |
CA3100001A1 (en) | 2019-11-21 |
WO2019222136A9 (en) | 2019-12-26 |
AU2019268302A1 (en) | 2020-12-03 |
WO2019222136A2 (en) | 2019-11-21 |
CN112424217A (en) | 2021-02-26 |
PH12020551938A1 (en) | 2021-06-21 |
WO2019222136A3 (en) | 2020-02-20 |
BR112020023292A2 (en) | 2021-02-23 |
MX2020012170A (en) | 2021-01-29 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
SG11202011145UA (en) | Liver targeting adeno-associated viral vectors | |
EP3645021A4 (en) | Adeno-associated viral vectors for gene therapy | |
HK1257563A1 (en) | Targeting peptides for directing adeno-associated viruses (aavs) | |
EP3558393A4 (en) | Adeno associated viral vectors | |
IL277666A (en) | Antibody-evading virus vectors | |
IL266264A (en) | Adeno-associated virus formulations | |
IL282885A (en) | Aav viral vectors and uses thereof | |
EP3364996A4 (en) | Prostate-targeting adeno-associated virus serotype vectors | |
IL286464A (en) | Recombinant adeno-associated virus vectors | |
GB201401707D0 (en) | Adeno-associated viral vectors | |
GB201800903D0 (en) | Vectors | |
EP4061427A4 (en) | Adeno-associated viral vector variants | |
IL275833A (en) | Modified vaccinia vectors | |
HUE064133T2 (en) | Canine adenovirus vectors | |
IL268291A (en) | Multiple transgene recombinant adenovirus | |
IL277664A (en) | Antibody-evading virus vectors | |
IL273933A (en) | Aav vectors | |
IL284188A (en) | Modified orthopoxvirus vectors | |
IL275813A (en) | Modified orthopoxvirus vectors | |
IL272655A (en) | Recombinant adeno-associated vectors | |
ZA202108996B (en) | Viral vector | |
IL285639A (en) | Adeno-associated virus vectors for the delivery of therapeutics | |
GB201706945D0 (en) | Self-inactivating viral vector | |
NO345114B1 (en) | Ski assembly | |
AT521543A3 (en) |