SA518400233B1 - علاج جيني لعلاج نزف الدم الوراثي a - Google Patents
علاج جيني لعلاج نزف الدم الوراثي aInfo
- Publication number
- SA518400233B1 SA518400233B1 SA518400233A SA518400233A SA518400233B1 SA 518400233 B1 SA518400233 B1 SA 518400233B1 SA 518400233 A SA518400233 A SA 518400233A SA 518400233 A SA518400233 A SA 518400233A SA 518400233 B1 SA518400233 B1 SA 518400233B1
- Authority
- SA
- Saudi Arabia
- Prior art keywords
- treating hemophilia
- gene therapy
- compositions
- hemophilia
- treating
- Prior art date
Links
- 208000009292 Hemophilia A Diseases 0.000 title abstract 2
- 208000031220 Hemophilia Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 abstract 2
- 239000000203 mixture Substances 0.000 abstract 2
- 102100026735 Coagulation factor VIII Human genes 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 201000003542 Factor VIII deficiency Diseases 0.000 abstract 1
- 108010071690 Prealbumin Proteins 0.000 abstract 1
- 102000009190 Transthyretin Human genes 0.000 abstract 1
- 239000003623 enhancer Substances 0.000 abstract 1
- 102000057593 human F8 Human genes 0.000 abstract 1
- 229960000900 human factor viii Drugs 0.000 abstract 1
Classifications
-
- 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
-
- 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
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
-
- 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
- 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
-
- 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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Hematology (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Diabetes (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Immunology (AREA)
- Dermatology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Dispersion Chemistry (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662323336P | 2016-04-15 | 2016-04-15 | |
| US201662331807P | 2016-05-04 | 2016-05-04 | |
| US201662428866P | 2016-12-01 | 2016-12-01 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SA518400233B1 true SA518400233B1 (ar) | 2022-08-23 |
Family
ID=59253992
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SA518400233A SA518400233B1 (ar) | 2016-04-15 | 2018-10-14 | علاج جيني لعلاج نزف الدم الوراثي a |
Country Status (17)
| Country | Link |
|---|---|
| US (3) | US11779656B2 (enExample) |
| EP (1) | EP3452102B9 (enExample) |
| JP (2) | JP7347933B2 (enExample) |
| KR (1) | KR102450833B1 (enExample) |
| CN (1) | CN109562191A (enExample) |
| AU (1) | AU2017248659B2 (enExample) |
| BR (1) | BR112018071200A2 (enExample) |
| CA (1) | CA3019425A1 (enExample) |
| IL (1) | IL262215B2 (enExample) |
| MX (1) | MX2018012537A (enExample) |
| PE (2) | PE20250736A1 (enExample) |
| RU (1) | RU2762257C2 (enExample) |
| SA (1) | SA518400233B1 (enExample) |
| SG (2) | SG11201808422QA (enExample) |
| TW (1) | TWI791433B (enExample) |
| WO (1) | WO2017180857A1 (enExample) |
| ZA (1) | ZA201806477B (enExample) |
Families Citing this family (28)
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| US9249405B2 (en) | 2008-09-15 | 2016-02-02 | Paolo Simioni | Factor IX polypeptide mutant, its uses and a method for its production |
| HK1257955A1 (zh) | 2015-11-16 | 2019-11-01 | The Research Institute At Nationwide Children's Hospital | 用於治疗肌联蛋白类肌病和其他肌联蛋白病变的材料和方法 |
| KR102450833B1 (ko) * | 2016-04-15 | 2022-10-05 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 혈우병 a 치료용 유전자 요법 |
| WO2017180861A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsulvania | Gene therapy for treating hemophilia b |
| KR102824021B1 (ko) | 2018-01-31 | 2025-06-24 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 2c형 지대근이영양증에 대한 유전자 치료 |
| MX2020014119A (es) | 2018-06-29 | 2021-06-18 | Res Inst Nationwide Childrens Hospital | Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a. |
| JOP20210054A1 (ar) * | 2018-09-21 | 2020-03-21 | Nightstarx Ltd | تركيبات وطرق لعلاج التهاب الشبكية الصباغي |
| MX2021004213A (es) * | 2018-10-12 | 2021-07-16 | Genzyme Corp | Generacion de pah humana mejorada para el tratamiento de pku grave mediante terapia de sustitucion de genes dirigida al higado. |
| WO2020081415A1 (en) * | 2018-10-15 | 2020-04-23 | Regenxbio Inc. | Method for measuring the infectivity of replication defective viral vectors and viruses |
| BR112021007229A2 (pt) | 2018-10-16 | 2021-08-10 | Blueallele, Llc | métodos para inserção dirigida de dna em genes |
| GB201818816D0 (en) * | 2018-11-19 | 2019-01-02 | Synpromics Ltd | Regulatory nucleic acid sequences |
| TWI851647B (zh) * | 2019-01-16 | 2024-08-11 | 日商武田藥品工業股份有限公司 | 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體 |
| MX2021010356A (es) * | 2019-02-26 | 2022-03-04 | Res Inst Nationwide Childrens Hospital | SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR. |
| AU2020234713A1 (en) | 2019-03-13 | 2021-11-04 | Generation Bio Co. | Non-viral DNA vectors and uses thereof for expressing FVIII therapeutics |
| CN120400170A (zh) | 2019-08-21 | 2025-08-01 | 全国儿童医院研究所 | Α-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗 |
| CN114901686A (zh) * | 2019-11-01 | 2022-08-12 | 自由行疗法有限公司 | 因子viii多肽 |
| CN111218446B (zh) * | 2019-12-25 | 2023-03-28 | 劲帆生物医药科技（武汉）有限公司 | 一种肝脏特异性启动子及其应用 |
| PE20231100A1 (es) * | 2020-01-16 | 2023-07-18 | Takeda Pharmaceuticals Co | Terapia genica basada en virus adenoasociados para la fenilcetonuria |
| CN115803064A (zh) | 2020-05-12 | 2023-03-14 | 宾夕法尼亚州大学信托人 | 用于drg特异性降低转基因表达的组合物 |
| MX2023001137A (es) * | 2020-07-29 | 2023-02-22 | Freeline Therapeutics Ltd | Beta-glucocerebrosidasa mutada con estabilidad mejorada. |
| WO2022165245A1 (en) * | 2021-01-29 | 2022-08-04 | The Trustees Of The University Of Pennsylvania | Compositions and method of use of mutant ace2 decoy variants |
| AU2022214429A1 (en) | 2021-02-01 | 2023-09-14 | Tern Therapeutics, Llc | Gene therapy for neuronal ceroid lipofuscinoses |
| JP2024506860A (ja) * | 2021-02-01 | 2024-02-15 | 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 | ニーマンピック病ａ型を治療するための組成物及び方法 |
| EP4219726A1 (en) | 2021-10-15 | 2023-08-02 | Research Institute at Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| WO2023078220A1 (zh) * | 2021-11-02 | 2023-05-11 | 苏州华毅乐健生物科技有限公司 | 分离的核酸分子及其应用 |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| US20250276093A1 (en) * | 2022-04-20 | 2025-09-04 | AskBio Inc. | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii |
| US20250352626A1 (en) * | 2022-06-10 | 2025-11-20 | University Of Florida Research Foundation, Incorporated | Immune tolerance induction to viral capsids |
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| EP2480678B1 (en) | 2010-09-02 | 2014-02-12 | Molmed SpA | Stable production of lentiviral vectors |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| JP6454643B2 (ja) | 2012-10-26 | 2019-01-16 | フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ | 血友病の肝臓指向性遺伝子治療のためのベクター並びにその方法及び使用 |
| EP3513802B1 (en) * | 2012-10-26 | 2023-11-29 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| CA3178379A1 (en) * | 2013-09-12 | 2015-03-19 | Biomarin Pharmaceutical Inc. | Adeno-associated virus factor viii vectors |
| KR102554850B1 (ko) * | 2015-02-06 | 2023-07-13 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도 |
| US20180110877A1 (en) * | 2015-04-27 | 2018-04-26 | The Trustees Of The University Of Pennsylvania | DUAL AAV VECTOR SYSTEM FOR CRISPR/Cas9 MEDIATED CORRECTION OF HUMAN DISEASE |
| EA202190827A1 (ru) * | 2015-11-13 | 2021-11-30 | Баксалта Инкорпорейтед | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a |
| ES2918998T3 (es) | 2015-12-11 | 2022-07-21 | Univ Pennsylvania | Método de purificación escalable para AAVrh10 |
| KR102450833B1 (ko) * | 2016-04-15 | 2022-10-05 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 혈우병 a 치료용 유전자 요법 |
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2017
- 2017-04-13 KR KR1020187032617A patent/KR102450833B1/ko active Active
- 2017-04-13 MX MX2018012537A patent/MX2018012537A/es unknown
- 2017-04-13 TW TW106112302A patent/TWI791433B/zh not_active IP Right Cessation
- 2017-04-13 BR BR112018071200-0A patent/BR112018071200A2/pt active Search and Examination
- 2017-04-13 RU RU2018136611A patent/RU2762257C2/ru active
- 2017-04-13 SG SG11201808422QA patent/SG11201808422QA/en unknown
- 2017-04-13 SG SG10202009849WA patent/SG10202009849WA/en unknown
- 2017-04-13 PE PE2023002954A patent/PE20250736A1/es unknown
- 2017-04-13 PE PE2018001994A patent/PE20181922A1/es unknown
- 2017-04-13 CN CN201780023787.5A patent/CN109562191A/zh active Pending
- 2017-04-13 CA CA3019425A patent/CA3019425A1/en active Pending
- 2017-04-13 EP EP17734159.1A patent/EP3452102B9/en active Active
- 2017-04-13 AU AU2017248659A patent/AU2017248659B2/en not_active Ceased
- 2017-04-13 US US16/093,798 patent/US11779656B2/en active Active
- 2017-04-13 WO PCT/US2017/027396 patent/WO2017180857A1/en not_active Ceased
- 2017-04-13 JP JP2018554331A patent/JP7347933B2/ja active Active
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2018
- 2018-09-28 ZA ZA2018/06477A patent/ZA201806477B/en unknown
- 2018-10-08 IL IL262215A patent/IL262215B2/en unknown
- 2018-10-14 SA SA518400233A patent/SA518400233B1/ar unknown
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2019
- 2019-11-22 US US16/691,822 patent/US10888628B2/en active Active
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2022
- 2022-01-27 JP JP2022011052A patent/JP2022062121A/ja active Pending
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2023
- 2023-08-08 US US18/446,243 patent/US20240000974A1/en not_active Abandoned
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