SG11201811603WA - Novel adeno-associated virus capsid proteins - Google Patents
Novel adeno-associated virus capsid proteinsInfo
- Publication number
- SG11201811603WA SG11201811603WA SG11201811603WA SG11201811603WA SG11201811603WA SG 11201811603W A SG11201811603W A SG 11201811603WA SG 11201811603W A SG11201811603W A SG 11201811603WA SG 11201811603W A SG11201811603W A SG 11201811603WA SG 11201811603W A SG11201811603W A SG 11201811603WA
- Authority
- SG
- Singapore
- Prior art keywords
- international
- capsid proteins
- novato
- novel
- digital drive
- Prior art date
Links
- 108090000565 Capsid Proteins Proteins 0.000 title abstract 6
- 102100023321 Ceruloplasmin Human genes 0.000 title abstract 5
- 241000702421 Dependoparvovirus Species 0.000 title abstract 3
- 239000013607 AAV vector Substances 0.000 abstract 2
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 210000000234 capsid Anatomy 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 1
- 239000002245 particle Substances 0.000 abstract 1
- 102000040430 polynucleotide Human genes 0.000 abstract 1
- 108091033319 polynucleotide Proteins 0.000 abstract 1
- 239000002157 polynucleotide Substances 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
Classifications
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- 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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
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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
- A61K38/00—Medicinal preparations containing peptides
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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
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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
- C12N2750/14122—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
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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
- C12N2750/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral 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
- 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
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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
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
Abstract
INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property MD 1101111 0 DOI HIM 011101 0 01111111111111111111111111111111111111111111111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\" WO 2018/022608 A3 01 February 2018 (01.02.2018) W I P0 I P C T (51) International Patent Classification: C07K 14/005 (2006.01) A61K 48/00 (2006.01) Published: C12N 15/86 (2006.01) — with international search report (Art 21(3)) — before the expiration of the time limit for amending the (21) International Application Number: claims and to be republished in the event of receipt of PCT/US2017/043703 amendments (Rule 48.2(h)) (22) International Filing Date: — with sequence listing part of description (Rule 5.2(a)) 25 July 2017 (25.07.2017) (88) Date of publication of the international search report: 01 March 2018 (01.03.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/366,838 26 July 2016 (26.07.2016) US (71) Applicant: BIOMARIN PHARMACEUTICAL INC. [US/US]; 105 Digital Drive, Novato, CA 94949 (US). (72) Inventors: COLOSI, Peter, Cameron; 105 Digital Drive, Novato, CA 94949 (US). LOCHRIE, Michael; 105 Digital Drive, Novato, CA 94949 (US). NG, Robert; 105 Digital Drive, Novato, CA 94949 (US). Agent: SINTICH, Sharon, M.; Marshall, Gerstein & = (74) Borun LLP, 233 S. Wacker Drive, 6300 Willis Tower, Chicago, IL 60606-6357 (US). — Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, (81) = = = _ KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, = = MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, = = TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. _ Designated States (unless otherwise indicated, for every — (84) _ _ kind of regional protection available): ARIPO (BW, GH, = GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, = UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, = TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, = EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, — MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). = M GC © ei ei © (54) Title: NOVEL ADENO-ASSOCIATED VIRUS CAPSID PROTEINS --.... ,-1 (57) : The invention relates to novel adeno-associated virus (AAV) capsid proteins, AAV particles comprising a novel capsid 0 protein, polynucleotides encoding these capsid proteins and AAV vectors expressing these capsid proteins. The invention also relates to N methods of making the herein described AAV vectors expressing the novel capsid proteins of the invention and associated therapeutic 0 uses of thereof.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662366838P | 2016-07-26 | 2016-07-26 | |
PCT/US2017/043703 WO2018022608A2 (en) | 2016-07-26 | 2017-07-25 | Novel adeno-associated virus capsid proteins |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11201811603WA true SG11201811603WA (en) | 2019-02-27 |
Family
ID=59558468
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11201811603WA SG11201811603WA (en) | 2016-07-26 | 2017-07-25 | Novel adeno-associated virus capsid proteins |
Country Status (15)
Country | Link |
---|---|
US (2) | US11584780B2 (en) |
EP (1) | EP3491008A2 (en) |
JP (4) | JP6994018B2 (en) |
KR (2) | KR20230161535A (en) |
CN (1) | CN109715650B (en) |
AU (2) | AU2017301600B2 (en) |
BR (1) | BR112019001532A2 (en) |
CA (1) | CA3031026A1 (en) |
CL (3) | CL2019000167A1 (en) |
IL (2) | IL263801B2 (en) |
MX (1) | MX2019000962A (en) |
PE (1) | PE20190401A1 (en) |
PH (1) | PH12019500068A1 (en) |
SG (1) | SG11201811603WA (en) |
WO (1) | WO2018022608A2 (en) |
Families Citing this family (39)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
SG10202107733QA (en) | 2015-09-28 | 2021-09-29 | Univ North Carolina Chapel Hill | Methods and compositions for antibody-evading virus vectors |
US20190185864A1 (en) | 2016-08-23 | 2019-06-20 | Akouos, Inc. | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
BR112020017278A2 (en) * | 2018-02-27 | 2020-12-22 | The Trustees Of The University Of Pennsylvania | ADENOSOCIATED VIRUS VECTORS (AAV), AAV VECTORS HAVING REDUCED CAPSIDE DAMMATION AND USES FOR THE SAME |
BR112020020223A2 (en) * | 2018-04-05 | 2021-01-19 | Genethon | RECOMBINANT HYBRID ADENO-ASSOCIATED VIRUS SOROTYPE BETWEEN AAV9 AND AAVRH74 WITH TROPISM BY REDUCED LIVER |
KR20210008501A (en) | 2018-05-09 | 2021-01-22 | 바이오마린 파머수티컬 인크. | How to treat phenylketonuria |
EP3790567A4 (en) * | 2018-05-11 | 2021-12-01 | Massachusetts Eye and Ear Infirmary | Liver-specific tropism of adeno-associated viruses |
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 |
US11821009B2 (en) | 2018-05-15 | 2023-11-21 | Cornell University | Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery |
US20210317478A1 (en) | 2018-08-21 | 2021-10-14 | Massachusetts Eye And Ear Infirmary | Compositions and methods for modulating transduction efficiency of adeno-associated viruses |
CN113423434A (en) * | 2018-12-05 | 2021-09-21 | 阿贝奥纳治疗有限公司 | Recombinant adeno-associated virus vectors for gene delivery |
WO2020154535A1 (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 |
WO2020198737A1 (en) * | 2019-03-28 | 2020-10-01 | The General Hospital Corporation | Engineered adeno-associated (aav) vectors for transgene expression |
WO2020232044A1 (en) | 2019-05-14 | 2020-11-19 | Biomarin Pharmaceutical Inc. | Methods of redosing gene therapy vectors |
US20220403414A1 (en) * | 2019-10-16 | 2022-12-22 | Wuxi Apptec (Shanghai) Co., Ltd. | Novel aav variant |
AU2020367532A1 (en) | 2019-10-17 | 2022-05-12 | Ginkgo Bioworks, Inc. | Adeno-associated viral vectors for treatment of Niemann-Pick disease type C |
TW202128733A (en) | 2019-11-14 | 2021-08-01 | 美商拜奧馬林製藥公司 | Treatment of hereditary angioedema with liver-specific gene therapy vectors |
BR112022016596A2 (en) | 2020-02-21 | 2022-11-16 | Akouos Inc | COMPOSITIONS AND METHODS FOR THE TREATMENT OF NON-AGE-ASSOCIATED HEARING IMPAIRMENT IN A HUMAN INDIVIDUAL |
CA3169063A1 (en) * | 2020-02-25 | 2021-09-02 | Leszek Lisowski | Adeno-associated virus capsid polypeptides and vectors |
WO2021183895A1 (en) | 2020-03-13 | 2021-09-16 | Biomarin Pharmaceutical Inc. | Treatment of fabry disease with aav gene therapy vectors |
TW202144575A (en) | 2020-04-03 | 2021-12-01 | 美商拜奧馬林製藥公司 | Treatment of phenylketonuria with aav and therapeutic formulations |
EP4143216A1 (en) | 2020-04-29 | 2023-03-08 | Bristol-Myers Squibb Company | Miniaturized dystrophins having spectrin fusion domains and uses thereof |
WO2021236908A2 (en) | 2020-05-20 | 2021-11-25 | Biomarin Pharmaceutical Inc. | Use of regulatory proteins for the production of adeno-associated virus |
US20230242905A1 (en) * | 2020-07-03 | 2023-08-03 | Genethon | Method for Engineering Novel Hybrid AAV Capsids Through Hyper Variable Regions Swapping |
JP2023544683A (en) * | 2020-09-10 | 2023-10-25 | ジェネトン | Peptide modified AAV capsid |
AU2021359874A1 (en) * | 2020-10-18 | 2023-05-25 | The Trustees Of The University Of Pennsylvania | Improved adeno-associated virus (aav) vector and uses therefor |
CA3196778A1 (en) | 2020-11-02 | 2022-05-05 | Biomarin Pharmaceutical, Inc. | Process for enriching adeno-associated virus |
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 |
WO2023034996A1 (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 |
WO2023034997A1 (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 |
AR127217A1 (en) | 2021-10-01 | 2023-12-27 | Biomarin Pharm Inc | TREATMENT OF HEREDITARY ANGIOEDEMA WITH AAV GENOTHERAPY VECTORS AND THERAPEUTIC FORMULATIONS |
CN114276419B (en) * | 2021-12-30 | 2023-11-17 | 上海勉亦生物科技有限公司 | Novel adeno-associated virus capsid protein with high affinity for muscle and application thereof |
WO2023183623A1 (en) * | 2022-03-25 | 2023-09-28 | Regenxbio Inc. | Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy |
WO2024064856A1 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of cardiomyopathy with aav gene therapy vectors |
WO2024064863A2 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors |
CN115925819B (en) * | 2022-12-30 | 2023-10-13 | 广州派真生物技术有限公司 | Adeno-associated virus mutant and application thereof |
Family Cites Families (71)
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 |
IE58011B1 (en) | 1983-05-27 | 1993-06-16 | Texas A & M Univ Sys | Method for producing a recombinant baculovirus expression vector |
DK518384A (en) | 1984-01-31 | 1985-07-01 | Idaho Res Found | VECTOR FOR THE MANUFACTURE OF A GENE PRODUCT IN INSECT CELLS, PROCEDURE FOR ITS MANUFACTURING AND ITS USE |
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 |
EP0933997A4 (en) | 1996-09-06 | 2000-08-16 | Chiron Corp | Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors |
JP2001500015A (en) | 1996-09-06 | 2001-01-09 | 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 | Method for producing inducible recombinant adeno-associated virus using T7 polymerase |
WO1999003496A1 (en) | 1997-07-21 | 1999-01-28 | The University Of North Carolina At Chapel Hill | Factor ix antihemophilic factor with increased clotting activity |
DK1071752T3 (en) | 1998-04-21 | 2003-10-20 | Micromet Ag | CD19xCD3-specific polypeptides and their use |
AU2001255575B2 (en) | 2000-04-28 | 2006-08-31 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
DE60239103D1 (en) | 2001-10-16 | 2011-03-17 | Nat Inst Of Advanced Ind Scien | NEW N-ACETYLGLUCOSAMINE TRANSFERASE, CODING NUCLEIC ACIDS, SPECIFIC ANTIBODIES, AND USE TO CANCER 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 |
SG168422A1 (en) | 2001-11-13 | 2011-02-28 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
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 |
AU2003237159A1 (en) | 2002-04-30 | 2003-11-17 | University Of Florida | Treatment for phenylketonuria |
CN102174574B (en) | 2003-09-30 | 2016-08-03 | 宾夕法尼亚大学托管会 | Adeno associated virus (AAV) is evolved, sequence, carrier containing these sequences and their application |
WO2005049614A2 (en) | 2003-11-17 | 2005-06-02 | Biomarin Pharmaceutical Inc. | Processes for preparing tetrahydrobiopterin, and analogs of tetrahydrobiopterin |
EP1771571A2 (en) | 2004-07-30 | 2007-04-11 | Targeted Genetics Corporation | Recombinant aav based vaccine methods |
KR20070084270A (en) | 2004-11-17 | 2007-08-24 | 바이오마린 파머수티컬 인크. | Stable tablet formulation |
ES2428218T3 (en) | 2005-04-07 | 2013-11-06 | The Trustees Of The University Of Pennsylvania | AAV rh48 modified capsules, compositions containing them and uses thereof |
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 |
EP2125006B1 (en) | 2007-01-18 | 2013-10-16 | University of Missouri-Columbia | 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 |
PL2173888T3 (en) | 2007-07-26 | 2017-02-28 | Uniqure Ip B.V. | Baculoviral vectors comprising repeated coding sequences with differential codon biases |
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 |
EP2240484A1 (en) | 2008-01-07 | 2010-10-20 | BioMarin Pharmaceutical Inc. | Method of synthesizing tetrahydrobiopterin |
RU2502800C2 (en) | 2008-01-15 | 2013-12-27 | Эббви Инк. | Improved expression vectors of mammals and their use |
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 |
EP3581650B1 (en) | 2008-09-15 | 2022-12-28 | 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 |
US20130195800A1 (en) | 2010-03-23 | 2013-08-01 | Intrexon Corporation | Vectors Conditionally Expressing Therapeutic Proteins, Host Cells Comprising the Vectors, and Uses Thereof |
MX342858B (en) | 2010-03-29 | 2016-10-13 | The Trustees Of The Univ 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 |
EP2814958B1 (en) | 2012-02-17 | 2019-08-07 | 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 |
MX2015012739A (en) | 2013-03-15 | 2016-02-19 | Univ Pennsylvania | Compositions and methods for treating mpsi. |
JP6600624B2 (en) | 2013-05-31 | 2019-10-30 | 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 | Adeno-associated virus mutant and method of use thereof |
US9840719B2 (en) | 2013-07-22 | 2017-12-12 | The Children's Hospital Of Philadelphia | Variant AAV and compositions, methods and uses for gene transfer to cells, organs and tissues |
CA3178379A1 (en) | 2013-09-12 | 2015-03-19 | Biomarin Pharmaceutical Inc. | Adeno-associated virus factor viii vectors |
EP3060575B1 (en) | 2013-10-11 | 2018-12-05 | 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 |
AU2015229750A1 (en) | 2014-03-09 | 2016-09-15 | 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 |
BR112016030852A2 (en) | 2014-07-02 | 2018-01-16 | Shire Human Genetic Therapies | rna messenger encapsulation |
US10301367B2 (en) | 2014-07-26 | 2019-05-28 | Consiglio Nazionale Delle Ricerche | Compositions and methods for treatment of muscular dystrophy |
DK3198018T3 (en) * | 2014-09-24 | 2021-03-01 | Hope City | VECTOR VARIANTS OF ADENO ASSOCIATED VIRUS FOR HIGH-EFFECTIVE GENERATING 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 |
CN108603235A (en) * | 2015-07-30 | 2018-09-28 | 马萨诸塞眼科耳科诊所 | Ancestors' virus sequence and application thereof |
EP3352787A1 (en) | 2015-09-24 | 2018-08-01 | 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 |
US11583563B2 (en) | 2016-11-04 | 2023-02-21 | Takeda Pharmaceutical Company Limited | Adeno-associated virus formulations |
MX2019007876A (en) | 2016-12-30 | 2019-10-15 | Univ Pennsylvania | Gene therapy for treating phenylketonuria. |
US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
MX2020008085A (en) | 2018-02-01 | 2020-12-10 | Homology Medicines Inc | Adeno-associated virus compositions for pah gene transfer and methods of use thereof. |
KR20210008501A (en) | 2018-05-09 | 2021-01-22 | 바이오마린 파머수티컬 인크. | How to treat 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 |
MX2020012167A (en) | 2018-05-14 | 2022-07-13 | Biomarin Pharm Inc | Stable expression of aav vectors in juvenile subjects. |
SG11202100704PA (en) | 2018-07-24 | 2021-02-25 | Voyager Therapeutics Inc | Systems and methods for producing gene therapy formulations |
CA3136939A1 (en) | 2019-04-19 | 2020-10-22 | Regenxbio Inc. | Adeno-associated virus vector formulations and methods |
WO2020232044A1 (en) | 2019-05-14 | 2020-11-19 | Biomarin Pharmaceutical Inc. | Methods of redosing gene therapy vectors |
TW202144575A (en) | 2020-04-03 | 2021-12-01 | 美商拜奧馬林製藥公司 | Treatment of phenylketonuria with aav and therapeutic formulations |
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