SG11202010830WA - Methods of treating phenylketonuria - Google Patents
Methods of treating phenylketonuriaInfo
- Publication number
- SG11202010830WA SG11202010830WA SG11202010830WA SG11202010830WA SG11202010830WA SG 11202010830W A SG11202010830W A SG 11202010830WA SG 11202010830W A SG11202010830W A SG 11202010830WA SG 11202010830W A SG11202010830W A SG 11202010830WA SG 11202010830W A SG11202010830W A SG 11202010830WA
- Authority
- SG
- Singapore
- Prior art keywords
- methods
- treating phenylketonuria
- phenylketonuria
- treating
- Prior art date
Links
Classifications
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- 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
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- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
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- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
- C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
- C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/94—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving narcotics or drugs or pharmaceuticals, neurotransmitters or associated receptors
- G01N33/9406—Neurotransmitters
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- 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
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- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862669292P | 2018-05-09 | 2018-05-09 | |
US201862755207P | 2018-11-02 | 2018-11-02 | |
US201962802608P | 2019-02-07 | 2019-02-07 | |
US201962819414P | 2019-03-15 | 2019-03-15 | |
PCT/US2019/031252 WO2019217513A2 (en) | 2018-05-09 | 2019-05-08 | Methods of treating phenylketonuria |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11202010830WA true SG11202010830WA (en) | 2020-11-27 |
Family
ID=66794083
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11202010830WA SG11202010830WA (en) | 2018-05-09 | 2019-05-08 | Methods of treating phenylketonuria |
Country Status (15)
Country | Link |
---|---|
US (1) | US11739345B2 (en) |
EP (1) | EP3790627A2 (en) |
JP (1) | JP2021522811A (en) |
KR (1) | KR20210008501A (en) |
CN (1) | CN113164762A (en) |
AU (1) | AU2019265560A1 (en) |
BR (1) | BR112020022722A8 (en) |
CA (1) | CA3099704A1 (en) |
CL (1) | CL2020002892A1 (en) |
MX (1) | MX2020011937A (en) |
PE (1) | PE20210112A1 (en) |
PH (1) | PH12020551863A1 (en) |
SG (1) | SG11202010830WA (en) |
TW (1) | TW202016314A (en) |
WO (1) | WO2019217513A2 (en) |
Families Citing this family (21)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
WO2019070674A2 (en) * | 2017-10-02 | 2019-04-11 | American Gene Technologies International Inc. | Vectors with promoter and enhancer combinations for treating phenylketonuria |
JP7423521B2 (en) | 2017-11-22 | 2024-01-29 | モダーナティエックス・インコーポレイテッド | Polynucleotide encoding phenylalanine hydroxylase for the treatment of phenylketonuria |
TW202005978A (en) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | Novel liver targeting adeno-associated viral vectors |
JP2023510392A (en) * | 2020-01-16 | 2023-03-13 | 武田薬品工業株式会社 | Adeno-associated virus-based gene therapy for phenylketonuria |
AR122409A1 (en) | 2020-04-03 | 2022-09-07 | Biomarin Pharm Inc | TREATMENT OF PHENYLKETONURIA WITH AAV AND THERAPEUTIC FORMULATIONS |
WO2021207415A1 (en) * | 2020-04-07 | 2021-10-14 | The Curators Of The University Of Missouri | CpG-FREE ITRs FOR AAV GENE THERAPY |
WO2021252952A1 (en) * | 2020-06-11 | 2021-12-16 | Sangamo Therapeutics, Inc. | Methods and compositions for expressing phenylalanine hydroxylase |
KR20230078805A (en) | 2020-11-02 | 2023-06-02 | 바이오마린 파머수티컬 인크. | Enrichment process of adeno-associated virus |
US20220160728A1 (en) * | 2020-11-05 | 2022-05-26 | Homology Medicines, Inc. | Gene therapy methods |
WO2023034989A1 (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 |
WO2023034994A1 (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 |
CN115772503B (en) * | 2022-08-16 | 2023-10-17 | 广东省南山医药创新研究院 | Gene modified cell medicine for expressing PAH and preparation method and application thereof |
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 |
CN115896135B (en) * | 2022-11-02 | 2024-03-01 | 苏州诺洁贝生物技术有限公司 | Optimized PAH gene and expression cassette and use thereof |
CN116656740A (en) * | 2023-05-30 | 2023-08-29 | 浙江大学 | Adeno-associated virus gene therapy vector and application thereof in preparation of medicament for treating phenylketonuria |
Family Cites Families (69)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US955734A (en) | 1909-04-26 | 1910-04-19 | Joseph Wolkerstorfer | Display-rack. |
US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
ATE78293T1 (en) | 1983-05-27 | 1992-08-15 | Texas A & M Univ Sys | METHOD OF GENERATING 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 |
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 |
US6521225B1 (en) | 1996-09-06 | 2003-02-18 | Chiron Corporation | AAV vectors |
WO1999003496A1 (en) | 1997-07-21 | 1999-01-28 | The University Of North Carolina At Chapel Hill | Factor ix antihemophilic factor with increased clotting activity |
TR200003087T2 (en) | 1998-04-21 | 2001-02-21 | Micromet Ag | New CD19 X CD3 Specific polypeptides and their use |
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 |
JPWO2003033710A1 (en) | 2001-10-16 | 2005-02-03 | 独立行政法人産業技術総合研究所 | Novel N-acetylglucosamine transferase, nucleic acid encoding the same, antibody thereto, and use for cancer or tumor diagnosis |
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 |
CN105671005B (en) | 2001-11-13 | 2020-09-15 | 宾夕法尼亚大学托管会 | Methods for detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified |
EP3339430A1 (en) | 2001-12-17 | 2018-06-27 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) serotype 9 sequences, vectors containing 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 |
DK2292779T3 (en) | 2003-09-30 | 2017-02-27 | Univ Pennsylvania | ADENOASS-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME AND APPLICATIONS 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 |
EP1771571A2 (en) | 2004-07-30 | 2007-04-11 | Targeted Genetics Corporation | Recombinant aav based vaccine methods |
JP2008520574A (en) | 2004-11-17 | 2008-06-19 | 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 stable tablet formulation |
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 |
US7534595B2 (en) | 2006-06-12 | 2009-05-19 | Biomarin Pharmaceutical Inc. | Compositions of prokaryotic phenylalanine ammonia-lyase and methods of using compositions thereof |
US7531341B1 (en) | 2006-06-12 | 2009-05-12 | Biomarin Pharmaceutical Inc. | Compositions of prokaryotic phenylalanine ammonia-lyase and methods of using compositions thereof |
CN101711164B (en) | 2007-01-18 | 2014-06-04 | 密苏里-哥伦比亚大学 | Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma |
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 |
US7537923B2 (en) | 2007-08-17 | 2009-05-26 | Biomarin Pharmaceutical Inc. | Compositions of prokaryotic phenylalanine ammonia-lyase and methods of treating cancer using compositions thereof |
JP2011508781A (en) | 2008-01-07 | 2011-03-17 | ビオマリン プハルマセウトイカル インコーポレイテッド | Method for the synthesis of tetrahydrobiopterin |
ES2579231T3 (en) | 2008-01-15 | 2016-08-08 | Abbvie Inc | Enhanced 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 |
US20110201022A1 (en) | 2008-07-30 | 2011-08-18 | Biomarin Pharmaceutical Inc. | Assays for detection of phenylalanine ammonia-lyase and antibodies to phenylalanine ammonia-lyase |
PL2337849T3 (en) | 2008-09-15 | 2018-11-30 | Uniqure Biopharma B.V. | Factor ix polypeptide mutant, its uses and a method for its production |
JP6174318B2 (en) | 2009-04-30 | 2017-08-02 | 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 | Composition for targeting conducting airway cells comprising an adeno-associated virus construct |
GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
EP3284821B1 (en) | 2010-03-23 | 2022-01-05 | Precigen, Inc. | Vectors conditionally expressing therapeutic proteins, host cells comprising the vectors, and uses thereof |
CN102869779A (en) | 2010-03-29 | 2013-01-09 | 宾夕法尼亚大学托管会 | 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 |
MX367100B (en) | 2012-02-17 | 2019-08-05 | The Children´S Hospital Of Philadelphia | Aav vector compositions and methods for gene transfer to cells, organs and tissues. |
CN108949772A (en) | 2012-04-02 | 2018-12-07 | 现代泰克斯公司 | For generating the modification polynucleotides of biological agent relevant to human diseases and protein |
GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
CA2901328C (en) | 2013-03-15 | 2023-08-01 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating mps1 |
PE20160769A1 (en) | 2013-09-12 | 2016-08-12 | Biomarin Pharm Inc | VECTORS OF ADENO-ASSOCIATED VIRUS FACTOR VIII |
CA3182790A1 (en) | 2013-10-11 | 2015-04-16 | Massachusetts Eye & Ear Infirmary | Ancestral adeno-associated virus sequences and uses thereof |
EA201992208A1 (en) * | 2013-10-22 | 2020-07-31 | Транслейт Био, Инк. | TREATMENT OF PHENYLKETONURIA USING mRNA |
DK3116900T3 (en) | 2014-03-09 | 2020-09-28 | Univ Pennsylvania | COMPOSITIONS THAT CAN BE USED TO TREAT ORNITHIN TRANSCARBAMYLASE (OTC) DEFECT |
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 |
PT3198018T (en) | 2014-09-24 | 2021-02-24 | Hope City | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
GB201507842D0 (en) | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
PL3872085T3 (en) | 2015-07-30 | 2023-06-05 | Massachusetts Eye & Ear Infirmary | Ancestral aav sequences and uses thereof |
AU2016326602A1 (en) | 2015-09-24 | 2018-03-29 | Biomarin Pharmaceutical 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 |
IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
JP2019537578A (en) | 2016-11-04 | 2019-12-26 | バクスアルタ インコーポレイテッド | Adeno-associated virus preparation |
EP3562494A4 (en) * | 2016-12-30 | 2020-08-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating phenylketonuria |
EA202091844A1 (en) | 2018-02-01 | 2021-02-03 | Хомолоджи Медисинс, Инк. | COMPOSITIONS OF ADENO-ASSOCIATED VIRUSES FOR PAH GENE TRANSFER AND METHODS OF THEIR APPLICATION |
US10610606B2 (en) * | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
BR112020023159A2 (en) | 2018-05-14 | 2021-02-02 | Biomarin Pharmaceutical Inc. | method for improving symptoms of a genetic disorder in a young individual, use of a therapeutic aav virus, composition, method for reducing bleeding time, use of a therapeutically effective amount of a therapeutic aav virus and method for increasing protein expression |
TW202005978A (en) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | Novel liver targeting adeno-associated viral vectors |
CA3107462A1 (en) | 2018-07-24 | 2020-01-30 | Voyager Therapeutics, Inc. | Systems and methods for producing gene therapy formulations |
AR118734A1 (en) | 2019-04-19 | 2021-10-27 | Regenxbio Inc | FORMULATIONS AND METHODS OF ADENO-ASSOCIATED VIRUS VECTORS |
WO2020232044A1 (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 |
-
2019
- 2019-05-08 CA CA3099704A patent/CA3099704A1/en active Pending
- 2019-05-08 CN CN201980046511.8A patent/CN113164762A/en active Pending
- 2019-05-08 PE PE2020001818A patent/PE20210112A1/en unknown
- 2019-05-08 MX MX2020011937A patent/MX2020011937A/en unknown
- 2019-05-08 TW TW108115952A patent/TW202016314A/en unknown
- 2019-05-08 KR KR1020207035184A patent/KR20210008501A/en active Search and Examination
- 2019-05-08 EP EP19729398.8A patent/EP3790627A2/en active Pending
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- 2019-05-08 SG SG11202010830WA patent/SG11202010830WA/en unknown
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CN113164762A (en) | 2021-07-23 |
US11739345B2 (en) | 2023-08-29 |
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PH12020551863A1 (en) | 2021-07-19 |
CL2020002892A1 (en) | 2021-05-07 |
KR20210008501A (en) | 2021-01-22 |
BR112020022722A2 (en) | 2021-02-02 |
US20190376081A1 (en) | 2019-12-12 |
WO2019217513A3 (en) | 2019-12-19 |
EP3790627A2 (en) | 2021-03-17 |
PE20210112A1 (en) | 2021-01-19 |
AU2019265560A1 (en) | 2020-11-26 |
BR112020022722A8 (en) | 2022-01-18 |
WO2019217513A2 (en) | 2019-11-14 |
JP2021522811A (en) | 2021-09-02 |
CA3099704A1 (en) | 2019-11-14 |
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