DE60230456D1 - Faktor-viii-polypeptide auf hohem expressionsnivea und verwendungsverfahren - Google Patents
Faktor-viii-polypeptide auf hohem expressionsnivea und verwendungsverfahrenInfo
- Publication number
- DE60230456D1 DE60230456D1 DE60230456T DE60230456T DE60230456D1 DE 60230456 D1 DE60230456 D1 DE 60230456D1 DE 60230456 T DE60230456 T DE 60230456T DE 60230456 T DE60230456 T DE 60230456T DE 60230456 D1 DE60230456 D1 DE 60230456D1
- Authority
- DE
- Germany
- Prior art keywords
- factor viii
- expression level
- high expression
- compositions
- methods
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Expired - Lifetime
Links
- 229960000301 factor viii Drugs 0.000 title abstract 3
- 229920001184 polypeptide Polymers 0.000 title abstract 3
- 102000004196 processed proteins & peptides Human genes 0.000 title abstract 3
- 108090000765 processed proteins & peptides Proteins 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 abstract 3
- 108010054218 Factor VIII Proteins 0.000 abstract 2
- 102000001690 Factor VIII Human genes 0.000 abstract 2
- 201000003542 Factor VIII deficiency Diseases 0.000 abstract 2
- 208000009292 Hemophilia A Diseases 0.000 abstract 2
- 102100026735 Coagulation factor VIII Human genes 0.000 abstract 1
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 abstract 1
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
Classifications
-
- 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)
-
- 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
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Hematology (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Zoology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Diabetes (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Animal Behavior & Ethology (AREA)
- Engineering & Computer Science (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Toxicology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Gastroenterology & Hepatology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US32738801P | 2001-10-05 | 2001-10-05 | |
| PCT/US2002/033403 WO2003031598A2 (en) | 2001-10-05 | 2002-10-07 | Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DE60230456D1 true DE60230456D1 (de) | 2009-01-29 |
Family
ID=23276347
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DE60230456T Expired - Lifetime DE60230456D1 (de) | 2001-10-05 | 2002-10-07 | Faktor-viii-polypeptide auf hohem expressionsnivea und verwendungsverfahren |
Country Status (9)
| Country | Link |
|---|---|
| US (3) | US7635763B2 (https=) |
| EP (1) | EP1572889B1 (https=) |
| JP (2) | JP4634036B2 (https=) |
| AT (1) | ATE417618T1 (https=) |
| AU (1) | AU2002337901B2 (https=) |
| CA (1) | CA2461443C (https=) |
| DE (1) | DE60230456D1 (https=) |
| ES (1) | ES2319745T3 (https=) |
| WO (1) | WO2003031598A2 (https=) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7560107B2 (en) | 1996-06-26 | 2009-07-14 | Emory University | Modified factor VIII |
| WO2003031598A2 (en) * | 2001-10-05 | 2003-04-17 | Emory University | Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use |
| WO2005017149A1 (en) | 2003-06-03 | 2005-02-24 | Cell Genesys, Inc. | Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site |
| US7485291B2 (en) | 2003-06-03 | 2009-02-03 | Cell Genesys, Inc. | Compositions and methods for generating multiple polypeptides from a single vector using a virus derived peptide cleavage site, and uses thereof |
| SI1750733T1 (sl) * | 2004-05-03 | 2014-03-31 | Emory University | POSTOPEK DAJANJA PRAĹ IÄŚJEGA fVIII BREZ DOMENE B |
| KR101468345B1 (ko) | 2004-11-12 | 2014-12-03 | 바이엘 헬스케어 엘엘씨 | Ｆｖｉｉｉ의 부위 지향 변형 |
| CN102112144A (zh) * | 2008-05-16 | 2011-06-29 | 拜耳医药保健有限公司 | 靶向性凝固因子及其使用方法 |
| JP5922141B2 (ja) | 2010-11-05 | 2016-05-24 | バクスター・インターナショナル・インコーポレイテッドＢａｘｔｅｒ Ｉｎｔｅｒｎａｔｉｏｎａｌ Ｉｎｃｏｒｐ０Ｒａｔｅｄ | 増加した比活性を有する抗血友病第ｖｉｉｉ因子の新規変異体 |
| CN103796670A (zh) * | 2011-07-08 | 2014-05-14 | 比奥根艾迪克依蒙菲利亚公司 | 因子viii嵌合和杂合多肽及其使用方法 |
| WO2014008172A2 (en) * | 2012-07-03 | 2014-01-09 | Expression Therapeutics, Llc | High yield suspension cell line, system and method for making same |
| EP3904376B1 (en) | 2013-06-24 | 2024-11-13 | Xiao, Weidong | Mutant factor viii compositions and methods |
| CN115074366A (zh) | 2015-04-16 | 2022-09-20 | 埃默里大学 | 用于肝脏中蛋白质表达的重组启动子和载体及其用途 |
| EP3374387A1 (en) | 2015-11-13 | 2018-09-19 | Baxalta Incorporated | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
| EA202092049A1 (ru) | 2015-11-13 | 2020-11-10 | Баксалта Инкорпорейтед | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a |
| KR102450833B1 (ko) | 2016-04-15 | 2022-10-05 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 혈우병 a 치료용 유전자 요법 |
| WO2019040747A1 (en) | 2017-08-23 | 2019-02-28 | Wake Forest University Health Sciences | IN UTERO TRANSPLANTATION OF CELLS EXPRESSING FACTOR VIII FOR THE TREATMENT OF HEMOPHILIA |
| US20200360441A1 (en) | 2017-08-23 | 2020-11-19 | Wake Forest University Health Sciences | Post-natal transplantation of factor viii-expressing cells for treatment of hemophilia |
| EP3823985A1 (en) | 2018-07-16 | 2021-05-26 | Baxalta Incorporated | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| 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 |
| US11680109B2 (en) | 2019-05-29 | 2023-06-20 | Duke University | Compositions and methods for detection and measurement of RNA modifications through targeted RNA editing |
| US20230357360A1 (en) * | 2021-01-29 | 2023-11-09 | Expression Therapeutics, Llc | Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5859204A (en) | 1992-04-07 | 1999-01-12 | Emory University | Modified factor VIII |
| US6376463B1 (en) | 1992-04-07 | 2002-04-23 | Emory University | Modified factor VIII |
| US6180371B1 (en) | 1996-06-26 | 2001-01-30 | Emory University | Modified factor VIII |
| US5663060A (en) | 1992-04-07 | 1997-09-02 | Emory University | Hybrid human/animal factor VIII |
| US5888974A (en) | 1992-04-07 | 1999-03-30 | Emory University | Hybrid human/animal factor VIII |
| US5364771A (en) | 1992-04-07 | 1994-11-15 | Emory University | Hybrid human/porcine factor VIII |
| US5744446A (en) | 1992-04-07 | 1998-04-28 | Emory University | Hybrid human/animal factor VIII |
| CA2162497A1 (en) * | 1993-06-10 | 1994-12-22 | Sheila Connelly | Adenoviral vectors for treatment of hemophilia |
| US6818439B1 (en) * | 1994-12-30 | 2004-11-16 | Chiron Corporation | Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders |
| US6458563B1 (en) * | 1996-06-26 | 2002-10-01 | Emory University | Modified factor VIII |
| WO1999029848A1 (en) * | 1997-12-05 | 1999-06-17 | The Immune Response Corporation | Novel vectors and genes exhibiting increased expression |
| US6200560B1 (en) * | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| WO2003031598A2 (en) * | 2001-10-05 | 2003-04-17 | Emory University | Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use |
-
2002
- 2002-10-07 WO PCT/US2002/033403 patent/WO2003031598A2/en not_active Ceased
- 2002-10-07 EP EP02773804A patent/EP1572889B1/en not_active Expired - Lifetime
- 2002-10-07 AU AU2002337901A patent/AU2002337901B2/en not_active Ceased
- 2002-10-07 AT AT02773804T patent/ATE417618T1/de not_active IP Right Cessation
- 2002-10-07 CA CA2461443A patent/CA2461443C/en not_active Expired - Lifetime
- 2002-10-07 JP JP2003534569A patent/JP4634036B2/ja not_active Expired - Lifetime
- 2002-10-07 DE DE60230456T patent/DE60230456D1/de not_active Expired - Lifetime
- 2002-10-07 ES ES02773804T patent/ES2319745T3/es not_active Expired - Lifetime
-
2004
- 2004-03-30 US US10/813,507 patent/US7635763B2/en not_active Expired - Lifetime
-
2009
- 2009-12-11 US US12/636,424 patent/US8188246B2/en not_active Expired - Lifetime
-
2010
- 2010-09-17 JP JP2010210204A patent/JP5162635B2/ja not_active Expired - Lifetime
-
2012
- 2012-05-27 US US13/481,840 patent/US8519111B2/en not_active Expired - Fee Related
Also Published As
| Publication number | Publication date |
|---|---|
| CA2461443C (en) | 2011-07-12 |
| JP2005518783A (ja) | 2005-06-30 |
| JP2011015698A (ja) | 2011-01-27 |
| EP1572889A4 (en) | 2006-09-06 |
| US8519111B2 (en) | 2013-08-27 |
| US20110077203A1 (en) | 2011-03-31 |
| US8188246B2 (en) | 2012-05-29 |
| JP4634036B2 (ja) | 2011-02-16 |
| WO2003031598A3 (en) | 2006-01-05 |
| CA2461443A1 (en) | 2003-04-17 |
| ATE417618T1 (de) | 2009-01-15 |
| US7635763B2 (en) | 2009-12-22 |
| US20050118684A1 (en) | 2005-06-02 |
| AU2002337901B2 (en) | 2008-01-17 |
| EP1572889A2 (en) | 2005-09-14 |
| HK1080517A1 (en) | 2006-04-28 |
| US20120270266A1 (en) | 2012-10-25 |
| ES2319745T3 (es) | 2009-05-12 |
| EP1572889B1 (en) | 2008-12-17 |
| JP5162635B2 (ja) | 2013-03-13 |
| WO2003031598A2 (en) | 2003-04-17 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| 8327 | Change in the person/name/address of the patent owner |
Owner name: EXPRESSION THERAPEUTICS, LLC, TUCKER, GA., US |
|
| 8364 | No opposition during term of opposition |