RU2020126034A - Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции - Google Patents
Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции Download PDFInfo
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- 102100026735 Coagulation factor VIII Human genes 0.000 title claims 5
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 title claims 5
- 241000700605 Viruses Species 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 239000002245 particle Substances 0.000 title 1
- 230000001225 therapeutic effect Effects 0.000 title 1
- 239000013598 vector Substances 0.000 title 1
- 230000003612 virological effect Effects 0.000 title 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 17
- 239000003550 marker Substances 0.000 claims 12
- 102100036475 Alanine aminotransferase 1 Human genes 0.000 claims 10
- 108010082126 Alanine transaminase Proteins 0.000 claims 10
- 239000003246 corticosteroid Substances 0.000 claims 10
- 206010019851 Hepatotoxicity Diseases 0.000 claims 9
- 230000007686 hepatotoxicity Effects 0.000 claims 9
- 231100000304 hepatotoxicity Toxicity 0.000 claims 9
- 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 claims 2
- CTKXFMQHOOWWEB-UHFFFAOYSA-N Ethylene oxide/propylene oxide copolymer Chemical compound CCCOC(C)COCCO CTKXFMQHOOWWEB-UHFFFAOYSA-N 0.000 claims 2
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 2
- 208000009292 Hemophilia A Diseases 0.000 claims 2
- 229930195725 Mannitol Natural products 0.000 claims 2
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims 2
- BNIILDVGGAEEIG-UHFFFAOYSA-L disodium hydrogen phosphate Chemical compound [Na+].[Na+].OP([O-])([O-])=O BNIILDVGGAEEIG-UHFFFAOYSA-L 0.000 claims 2
- 229910000397 disodium phosphate Inorganic materials 0.000 claims 2
- 235000019800 disodium phosphate Nutrition 0.000 claims 2
- 239000000594 mannitol Substances 0.000 claims 2
- 235000010355 mannitol Nutrition 0.000 claims 2
- 229920001993 poloxamer 188 Polymers 0.000 claims 2
- 229940044519 poloxamer 188 Drugs 0.000 claims 2
- AJPJDKMHJJGVTQ-UHFFFAOYSA-M sodium dihydrogen phosphate Chemical compound [Na+].OP(O)([O-])=O AJPJDKMHJJGVTQ-UHFFFAOYSA-M 0.000 claims 2
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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
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- A61K39/00—Medicinal preparations containing antigens or antibodies
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- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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- 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
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- 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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- 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
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- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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- C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
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- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- 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
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Claims (20)
1. Способ лечения субъекта, страдающего от гемофилии A, включающий введение фармацевтической композиции и кортикостероида,
где фармацевтическая композиция содержит FVIII ААВ-вирус, двухосновный фосфат натрия в концентрации от приблизительно 0,1 мг/мл до приблизительно 3 мг/мл, одноосновный фосфат натрия в концентрации от приблизительно 0,1 мг/мл до приблизительно 3 мг/мл, хлорид натрия в концентрации от приблизительно 1 мг/мл до приблизительно 20 мг/мл, маннит в концентрации от приблизительно 5 мг/мл до приблизительно 40 мг/мл и полоксамер 188 в концентрации от приблизительно 0,1 мг/мл до приблизительно 4 мг/мл.
2. Способ по п. 1, где кортикостероид вводят в концентрации в диапазоне от 5 мг/день до 60 мг/день.
3. Способ по п. 1, где кортикостероид вводят в концентрации в диапазоне от 5 мг/день до 60 мг/день в течение длительного периода времени, равного по меньшей мере 3, 4, 5, 6, 7, 8, 9 или 10 или более недель.
4. Способ по любому из пп. 1-3, дополнительно включающий стадию измерения маркера гепатотоксичности у субъекта после введения фармацевтической композиции и сравнения уровня маркера после введения фармацевтической композиции с уровнем маркера до введения фармацевтической композиции.
5. Способ по п. 4, где маркер гепатотоксичности представляет собой аланин-аминотрансферазу (АЛТ).
6. Способ по п. 5, где субъект имеет уровень АЛТ после введения фармацевтической композиции приблизительно в 1,5 раза выше, чем уровень АЛТ до введения фармацевтической композиции.
7. Способ по любому из пп. 1-3, где субъект имеет гепатотоксичность после введения фармацевтической композиции.
8. Способ по любому из пп. 1-3, где субъект имеет уровень АЛТ после введения фармацевтической композиции, который приблизительно в 1,5 раза выше, чем уровень АЛТ до введения фармацевтической композиции.
9. Способ лечения субъекта, страдающего от гемофилии A, включающий введение фармацевтической композиции,
и измерение уровня маркера гепатотоксичности у субъекта после введения фармацевтической композиции,
и сравнение уровня маркера после введения фармацевтической композиции с уровнем маркера до введения фармацевтической композиции,
где фармацевтическая композиция содержит FVIII ААВ-вирус, двухосновный фосфат натрия в концентрации от приблизительно 0,1 мг/мл до приблизительно 3 мг/мл, одноосновный фосфат натрия в концентрации от приблизительно 0,1 мг/мл до приблизительно 3 мг/мл, хлорид натрия в концентрации от приблизительно 1 мг/мл до приблизительно 20 мг/мл, маннит в концентрации от приблизительно 5 мг/мл до приблизительно 40 мг/мл и полоксамер 188 в концентрации от приблизительно 0,1 мг/мл до приблизительно 4 мг/мл.
10. Способ по п. 9, где кортикостероид вводят в концентрации в диапазоне от 5 мг/день до 60 мг/день.
11. Способ по п. 9, где кортикостероид вводят в концентрации в диапазоне от 5 мг/день до 60 мг/день в течение длительного периода времени, равного по меньшей мере 3, 4, 5, 6, 7, 8, 9 или 10 или более недель.
12. Способ по любому из пп. 9-11, где маркер гепатотоксичности представляет собой аланин-аминотрансферазу (АЛТ).
13. Способ по любому из пп. 9-12, дополнительно включающий стадию введения кортикостероида субъекту, где субъект, как определено, имеет гепатотоксичность после введения фармацевтической композиции.
14. Способ по любому из пп. 9-12, дополнительно включающий стадию введения кортикостероида для предотвращения гепатотоксичности после введения фармацевтической композиции.
15. Способ по п. 13 или 14, дополнительно содержащий стадию измерения уровня маркера гепатотоксичности у субъекта после введения кортикостероида и сравнения уровня маркера после введения кортикостероида с уровнем маркера до введения кортикостероида.
16. Способ по п. 15, где маркер гепатотоксичности представляет собой аланин-аминотрансферазу (АЛТ).
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562232242P | 2015-09-24 | 2015-09-24 | |
| US62/232,242 | 2015-09-24 | ||
| US201662323182P | 2016-04-15 | 2016-04-15 | |
| US62/323,182 | 2016-04-15 | ||
| US201662365544P | 2016-07-22 | 2016-07-22 | |
| US62/365,544 | 2016-07-22 |
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| RU2018114888A Division RU2742352C2 (ru) | 2015-09-24 | 2016-09-23 | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
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| RU2020126034A RU2020126034A (ru) | 2015-09-24 | 2016-09-23 | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
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| US (3) | US10512675B2 (ru) |
| EP (1) | EP3352787A1 (ru) |
| JP (3) | JP2018535929A (ru) |
| KR (1) | KR102805910B1 (ru) |
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| AU (2) | AU2016326602A1 (ru) |
| BR (1) | BR112018006074A2 (ru) |
| CA (1) | CA2999297A1 (ru) |
| CL (1) | CL2018000740A1 (ru) |
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| MX (1) | MX2018003702A (ru) |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| MX2017005834A (es) | 2014-11-05 | 2017-11-17 | Voyager Therapeutics Inc | Polinucleotidos aad para el tratamiento de la enfermedad de parkinson. |
| WO2017053677A1 (en) * | 2015-09-24 | 2017-03-30 | Biomarin Pharmaceutical Inc. | Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same |
| MY189674A (en) | 2015-10-28 | 2022-02-24 | Sangamo Therapeutics Inc | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
| KR102604510B1 (ko) | 2016-07-26 | 2023-11-23 | 바이오마린 파머수티컬 인크. | 신규 아데노-관련 바이러스 캡시드 단백질 |
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