PE20241346A1 - Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones que los comprenden - Google Patents
Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones que los comprendenInfo
- Publication number
- PE20241346A1 PE20241346A1 PE2023002034A PE2023002034A PE20241346A1 PE 20241346 A1 PE20241346 A1 PE 20241346A1 PE 2023002034 A PE2023002034 A PE 2023002034A PE 2023002034 A PE2023002034 A PE 2023002034A PE 20241346 A1 PE20241346 A1 PE 20241346A1
- Authority
- PE
- Peru
- Prior art keywords
- adeno
- factor viii
- formulations
- fviii
- sodium phosphate
- Prior art date
Links
- 241000702421 Dependoparvovirus Species 0.000 title abstract 2
- 229960000301 factor viii Drugs 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 title abstract 2
- 239000013598 vector Substances 0.000 title abstract 2
- 239000002245 particle Substances 0.000 title 1
- 230000003612 virological effect Effects 0.000 title 1
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 abstract 4
- 102100026735 Coagulation factor VIII Human genes 0.000 abstract 2
- FBPFZTCFMRRESA-KVTDHHQDSA-N D-Mannitol Chemical compound OC[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-KVTDHHQDSA-N 0.000 abstract 2
- CTKXFMQHOOWWEB-UHFFFAOYSA-N Ethylene oxide/propylene oxide copolymer Chemical compound CCCOC(C)COCCO CTKXFMQHOOWWEB-UHFFFAOYSA-N 0.000 abstract 2
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 abstract 2
- 229930195725 Mannitol Natural products 0.000 abstract 2
- 239000000594 mannitol Substances 0.000 abstract 2
- 235000010355 mannitol Nutrition 0.000 abstract 2
- 229920001993 poloxamer 188 Polymers 0.000 abstract 2
- 229940044519 poloxamer 188 Drugs 0.000 abstract 2
- 239000011780 sodium chloride Substances 0.000 abstract 2
- 108091026890 Coding region Proteins 0.000 abstract 1
- 108010054218 Factor VIII Proteins 0.000 abstract 1
- 102000001690 Factor VIII Human genes 0.000 abstract 1
- 108700026244 Open Reading Frames Proteins 0.000 abstract 1
- 241000700605 Viruses Species 0.000 abstract 1
- 239000006172 buffering agent Substances 0.000 abstract 1
- 239000004067 bulking agent Substances 0.000 abstract 1
- 239000003795 chemical substances by application Substances 0.000 abstract 1
- 229940061607 dibasic sodium phosphate Drugs 0.000 abstract 1
- BNIILDVGGAEEIG-UHFFFAOYSA-L disodium hydrogen phosphate Chemical compound [Na+].[Na+].OP([O-])([O-])=O BNIILDVGGAEEIG-UHFFFAOYSA-L 0.000 abstract 1
- 238000009472 formulation Methods 0.000 abstract 1
- 210000004185 liver Anatomy 0.000 abstract 1
- 229940045641 monobasic sodium phosphate Drugs 0.000 abstract 1
- 229910000403 monosodium phosphate Inorganic materials 0.000 abstract 1
- 235000019799 monosodium phosphate Nutrition 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 230000001105 regulatory effect Effects 0.000 abstract 1
- AJPJDKMHJJGVTQ-UHFFFAOYSA-M sodium dihydrogen phosphate Chemical compound [Na+].OP(O)([O-])=O AJPJDKMHJJGVTQ-UHFFFAOYSA-M 0.000 abstract 1
- 229910000162 sodium phosphate Inorganic materials 0.000 abstract 1
- 239000001488 sodium phosphate Substances 0.000 abstract 1
- 235000011008 sodium phosphates Nutrition 0.000 abstract 1
- 239000004094 surface-active agent Substances 0.000 abstract 1
- 230000002103 transcriptional effect Effects 0.000 abstract 1
- RYFMWSXOAZQYPI-UHFFFAOYSA-K trisodium phosphate Chemical compound [Na+].[Na+].[Na+].[O-]P([O-])([O-])=O RYFMWSXOAZQYPI-UHFFFAOYSA-K 0.000 abstract 1
Classifications
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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
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/36—Blood coagulation or fibrinolysis factors
- A61K38/37—Factors VIII
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/12—Viral antigens
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
-
- 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
-
- 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
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
-
- 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
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
-
- 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
- C07K14/745—Blood coagulation or fibrinolysis factors
- C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
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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
-
- 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
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6854—Immunoglobulins
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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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16041—Use of virus, viral particle or viral elements as a vector
- C12N2710/16044—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
- C12N2750/14121—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
- C12N2750/14141—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
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/005—Assays involving biological materials from specific organisms or of a specific nature from viruses
- G01N2333/01—DNA viruses
- G01N2333/015—Parvoviridae, e.g. feline panleukopenia virus, human Parvovirus
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Immunology (AREA)
- Molecular Biology (AREA)
- Genetics & Genomics (AREA)
- Hematology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Biomedical Technology (AREA)
- Gastroenterology & Hepatology (AREA)
- Wood Science & Technology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Urology & Nephrology (AREA)
- Mycology (AREA)
- Toxicology (AREA)
- Biophysics (AREA)
- Inorganic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Oncology (AREA)
- Communicable Diseases (AREA)
- Food Science & Technology (AREA)
- Physics & Mathematics (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562232242P | 2015-09-24 | 2015-09-24 | |
| US201662323182P | 2016-04-15 | 2016-04-15 | |
| US201662365544P | 2016-07-22 | 2016-07-22 | |
| PCT/US2016/053269 WO2017053677A1 (en) | 2015-09-24 | 2016-09-23 | Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PE20241346A1 true PE20241346A1 (es) | 2024-07-03 |
Family
ID=57113749
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2023002034A PE20241346A1 (es) | 2015-09-24 | 2016-09-23 | Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones que los comprenden |
| PE2018000419A PE20181338A1 (es) | 2015-09-24 | 2016-09-23 | Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2018000419A PE20181338A1 (es) | 2015-09-24 | 2016-09-23 | Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden |
Country Status (18)
| Country | Link |
|---|---|
| US (3) | US10512675B2 (enExample) |
| EP (1) | EP3352787A1 (enExample) |
| JP (3) | JP2018535929A (enExample) |
| KR (1) | KR102805910B1 (enExample) |
| CN (1) | CN108778323A (enExample) |
| AU (2) | AU2016326602A1 (enExample) |
| BR (1) | BR112018006074A2 (enExample) |
| CA (1) | CA2999297A1 (enExample) |
| CL (1) | CL2018000740A1 (enExample) |
| IL (2) | IL257939B2 (enExample) |
| MA (1) | MA42934A (enExample) |
| MX (1) | MX2018003702A (enExample) |
| PE (2) | PE20241346A1 (enExample) |
| PH (1) | PH12018500594A1 (enExample) |
| RU (2) | RU2020126034A (enExample) |
| TW (3) | TWI841908B (enExample) |
| WO (1) | WO2017053677A1 (enExample) |
| ZA (1) | ZA201801657B (enExample) |
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11201703148TA (en) | 2014-11-05 | 2017-05-30 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| RU2020126034A (ru) * | 2015-09-24 | 2020-09-02 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
| EP3368664A4 (en) | 2015-10-28 | 2019-08-28 | Sangamo Therapeutics, Inc. | LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF |
| US11584780B2 (en) | 2016-07-26 | 2023-02-21 | Biomarin Pharmaceutical Inc. | Adeno-associated virus capsid proteins |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| PL3652326T3 (pl) | 2017-07-10 | 2025-02-24 | Uniqure Ip B.V. | Środki i sposoby terapii genowej aav u ludzi |
| MY208862A (en) * | 2017-08-01 | 2025-06-04 | Spark Therapeutics Inc | Factor viii (fviii) gene therapy methods |
| US12296026B2 (en) * | 2018-02-05 | 2025-05-13 | Astellas Gene Therapies, Inc. | Transcription regulatory elements and uses thereof |
| EP3810647A4 (en) * | 2018-04-26 | 2022-08-17 | The University of North Carolina at Chapel Hill | METHODS AND COMPOSITIONS FOR THE TREATMENT OF HEMOPHILIA |
| WO2019217513A2 (en) | 2018-05-09 | 2019-11-14 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| KR20210008491A (ko) * | 2018-05-14 | 2021-01-22 | 바이오마린 파머수티컬 인크. | 청소년 대상체에서 aav 벡터의 안정한 발현 |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| US12091675B2 (en) * | 2018-07-16 | 2024-09-17 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia a using viral vectors encoding recombinant FVIII variants with increased expression |
| MX2021001375A (es) * | 2018-08-03 | 2021-04-19 | Sangamo Therapeutics Inc | Parametros clinicos mejorados por la expresion del factor viii. |
| WO2020041773A1 (en) * | 2018-08-24 | 2020-02-27 | Spark Therapeutics, Inc. | Optimized promoter sequences, intron-free expression constructs and methods of use |
| AU2019346655B2 (en) | 2018-09-28 | 2025-03-13 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| MA54951A (fr) | 2019-02-15 | 2021-12-22 | Bayer Healthcare Llc | Édition de gène pour l'hémophilie a avec une expression de facteur viii améliorée |
| MA55313A (fr) | 2019-03-13 | 2022-01-19 | Generation Bio Co | Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents thérapeutiques du facteur viii |
| JP2022534798A (ja) * | 2019-06-05 | 2022-08-03 | オーチャード セラピューティクス（ヨーロッパ）リミテッド | 真核細胞を修飾するための組成物及び方法 |
| US20200405883A1 (en) * | 2019-06-20 | 2020-12-31 | Baxalta Incorporated | Method of treatment with viral-based gene therapy |
| WO2021084277A2 (en) | 2019-11-01 | 2021-05-06 | Freeline Therapeutics Limited | Transcription regulatory elements |
| US20220403005A1 (en) * | 2019-12-06 | 2022-12-22 | The Children's Hospital Of Philadelphia | Compositions and methods for modulating factor viii function |
| EP4073106A2 (en) * | 2019-12-12 | 2022-10-19 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| KR102167829B1 (ko) * | 2020-02-10 | 2020-10-20 | 주식회사 이노테라피 | 아데노연관바이러스용 안정화제 및 이를 이용한 아데노연관바이러스의 안정화 방법 |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| MX2023005041A (es) | 2020-11-02 | 2023-05-17 | Biomarin Pharm Inc | Proceso para enriquecer virus adenoasociado. |
| CN115554418B (zh) * | 2022-11-22 | 2023-04-14 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
| WO2025045082A1 (zh) * | 2023-09-01 | 2025-03-06 | 康霖生物科技（杭州）有限公司 | 一种缓冲系统及其相应液体制剂及其用途 |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0127839B1 (en) | 1983-05-27 | 1992-07-15 | THE TEXAS A&M UNIVERSITY SYSTEM | Method for producing a recombinant baculovirus expression vector |
| US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
| DK518384A (da) | 1984-01-31 | 1985-07-01 | Idaho Res Found | Vektor til fremstilling af et gen-produkt i insektceller, fremgangsmaade til dens fremstilling samt dens anvendelse |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US6521225B1 (en) | 1996-09-06 | 2003-02-18 | Chiron Corporation | AAV vectors |
| AU4185699A (en) | 1998-05-27 | 1999-12-13 | Avigen, Inc. | Adeno-associated viral vector-mediated expression of factor viii activity |
| US6221349B1 (en) | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| 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 |
| WO2003074714A1 (en) | 2002-03-05 | 2003-09-12 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
| ES2436741T3 (es) | 2004-09-22 | 2014-01-07 | St. Jude Children's Research Hospital | Expresión mejorada de Factor IX en vectores de terapia génica |
| US7943374B2 (en) | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| KR20100113478A (ko) | 2007-11-01 | 2010-10-21 | 유니버시티 오브 로체스터 | 증가된 안정성을 갖는 재조합 인자 ⅴⅰⅰⅰ |
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| DK2911687T3 (da) * | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
| SI3044231T1 (sl) * | 2013-09-12 | 2020-12-31 | Biomarin Pharmaceutical Inc. | Vektorji AAV, ki vsebujejo gen, ki kodira faktor VIII |
| SG11201706445SA (en) | 2015-02-10 | 2017-09-28 | Genzyme Corp | Enhanced delivery of viral particles to the striatum and cortex |
| TWI707951B (zh) | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| RU2020126034A (ru) * | 2015-09-24 | 2020-09-02 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
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2016
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- 2016-09-23 WO PCT/US2016/053269 patent/WO2017053677A1/en not_active Ceased
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