RU2015144234A - Композиции и способы лечения mps1 - Google Patents
Композиции и способы лечения mps1 Download PDFInfo
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- RU2015144234A RU2015144234A RU2015144234A RU2015144234A RU2015144234A RU 2015144234 A RU2015144234 A RU 2015144234A RU 2015144234 A RU2015144234 A RU 2015144234A RU 2015144234 A RU2015144234 A RU 2015144234A RU 2015144234 A RU2015144234 A RU 2015144234A
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- 239000000203 mixture Substances 0.000 title claims 5
- 238000000034 method Methods 0.000 title claims 2
- 239000003623 enhancer Substances 0.000 claims 14
- 241000702421 Dependoparvovirus Species 0.000 claims 4
- 101001019502 Homo sapiens Alpha-L-iduronidase Proteins 0.000 claims 4
- 108090000623 proteins and genes Proteins 0.000 claims 4
- 239000003937 drug carrier Substances 0.000 claims 3
- 239000002245 particle Substances 0.000 claims 3
- 230000001105 regulatory effect Effects 0.000 claims 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 108010003381 Iduronidase Proteins 0.000 claims 2
- 102000004627 Iduronidase Human genes 0.000 claims 2
- 208000002678 Mucopolysaccharidoses Diseases 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 150000001413 amino acids Chemical class 0.000 claims 2
- 210000000234 capsid Anatomy 0.000 claims 2
- 206010028093 mucopolysaccharidosis Diseases 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 102100035028 Alpha-L-iduronidase Human genes 0.000 claims 1
- 108090000790 Enzymes Proteins 0.000 claims 1
- 102000004190 Enzymes Human genes 0.000 claims 1
- 108010044091 Globulins Proteins 0.000 claims 1
- 102000006395 Globulins Human genes 0.000 claims 1
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 claims 1
- 208000028781 Mucopolysaccharidosis type 1 Diseases 0.000 claims 1
- 241000283973 Oryctolagus cuniculus Species 0.000 claims 1
- 102000056929 human IDUA Human genes 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
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- 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
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- A61K38/00—Medicinal preparations containing peptides
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- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
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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
- 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
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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- 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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- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01076—L-Iduronidase (3.2.1.76)
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- Plant Pathology (AREA)
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- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
Claims (32)
1. Рекомбинантный вектор, несущий кассету экспрессии, содержащую ген альфа-L-идуронидазы человека (hIDUA), имеющий нуклеотидную последовательность SEQ ID NO: 1 или последовательность, по меньшей мере приблизительно на 80% идентичную SEQ ID NO: 1, которая кодирует функциональную альфа-L-идуронидазу человека, где упомянутая кассета экспрессии дополнительно содержит регуляторные контролирующие последовательности, которые управляют экспрессией альфа-L-идуронидазы человека, при этом упомянутые регуляторные контролирующие последовательности содержат печеночно-специфический промотор.
2. Рекомбинантный вектор по п. 1, где модифицированный ген hIDUA по меньшей мере от приблизительно на 85% до приблизительно на 90% идентичен SEQ ID NO: 1.
3. Рекомбинантный вектор по п. 2, где модифицированный ген hIDUA по меньшей мере от приблизительно на 95% до приблизительно на 99% идентичен SEQ ID NO: 1.
4. Рекомбинантный вектор по п. 1, где функциональная альфа-L-идуронидаза человека выбрана из группы, состоящей из:
(a) от приблизительно 1 до приблизительно 653 аминокислоты SEQ ID NO: 2;
(b) синтетического фермента человека, содержащего гетерологическую лидерную последовательность, слитую с аминокислотами от приблизительно 27 до приблизительно 653 из SEQ ID NO: 2; и
(c) варианта аминокислотной последовательности SEQ ID N0:2, имеющей одну или несколько модификаций, содержащих: восстановление H до Q в аминокислотном положении 82 (SEQ ID NO: 8); замену R на Q в положении 105 (SEQ ID NO: 9); замену G на R в положении 116 (SEQ ID NO: 10); замену V на А в положении 279 (SEQ ID NO: 11); замену L на R в положении 346 (SEQ ID NO: 12); замену А на Τ в положении 361 (SEQ ID NO: 13); замену Η на N в положении 449 (SEQ ID NO: 14); замену V на I в положении 454 (SEQ ID NO: 15); замену А на Τ в положении 591 (SEQ ID NO: 16) и замену А на Τ в положении 622 (SEQ ID NO: 17).
5. Рекомбинантный вектор по п. 1, где промотором является промотор TBG.
6. Рекомбинантный вектор по п. 1, где вектор дополнительно содержит один или несколько энхансеров.
7. Рекомбинантный вектор по п. 6, где энхансеры являются одинаковыми или разными.
8. Рекомбинантный вектор по п. 6, где энхансеры выбраны из группы, состоящей из интрона, энхансера CMV и энхансера Alpha mic/bik.
9. Рекомбинантный вектор по п. 6, где в векторе присутствуют несколько копий выбранного энхансера.
10. Рекомбинантный вектор по п. 9, где несколько копий энхансера расположены в тандеме.
11. Рекомбинантный вектор по п. 1, где кассета экспрессии дополнительно содержит поли-А, выбранную из поли-А глобулина кролика.
12. Рекомбинантный вектор по п. 1, где плазмидой является pENN.TBG.hIDUA.nRBG.
13. Частица рекомбинантного аденоассоциированного вируса (rAAV), имеющая AAV-капсид и имеющая упакованный в него 5' инвертированный концевой повтор (ITR), ген альфа-L-идуронидазы человека (hIDUA) под контролем регуляторных последовательностей, которые контролируют его экспрессию, и 3' ITR AAV, где упомянутый ген hIDUA имеет последовательность, показанную в SEQ ID NO: 1 (ФИГ. 1), или последовательность, по меньшей мере приблизительно на 95% идентичную ей, которая кодирует функциональную альфа-L-идуронидазу человека.
14. rAAV по п. 13, где модифицированный ген hIDUA экспрессируется под контролем печеночно-специфического промотора.
15. rAAV по п. 14, где промотором является промотор TBG.
16. rAAV по п. 13, где вектор дополнительно содержит один или несколько энхансеров.
17. rAAV по п. 16, где энхансеры являются одинаковыми или разными.
18. rAAV по п. 16, где энхансеры выбраны из группы, состоящей из интрона, энхансера CMV и энхансера Alpha mic/bik.
19. rAAV по п. 18, где в векторе присутствуют несколько копий выбранного энхансера.
20. rAAV по п. 19, где несколько копий энхансера расположены в тандеме.
21. rAAV по п. 13, где частица rAAV содержит AAV-капсид, выбранный из AAV8 и AAV9.
22. rAAV по п. 21, где rAAV является псевдотипированным.
23. rAAV по п. 22, где ITR происходят из AAV2.
24. Частица рекомбинантного аденоассоциированного вируса AAV2/8.TBG.hIDUA.со.
25. Композиция, пригодная для лечения мукополисахаридоза I типа (MPS I), содержащая rAAV по любому из пп. 13-24 и фармацевтически приемлемый носитель.
26. Способ лечения мукополисахаридоза I типа, включающий доставку эффективного количества композиции, содержащей фармацевтически приемлемый носитель и rAAV по любому из пп. 13-24.
27. Композиция по п. 25, где упомянутый rAAV доставляют внутривенно.
28. Рекомбинантный вектор по п. 1, где субъекту доставляют от приблизительно 3×109 до приблизительно 3×1012 геномных копий rAAV.
29. Применение композиции, содержащей rAAV по любому из пп. 13-24 и фармацевтически приемлемый носитель, для применения при лечении мукополисахаридоза I типа (MPS I).
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361788724P | 2013-03-15 | 2013-03-15 | |
| US61/788,724 | 2013-03-15 | ||
| PCT/US2014/025509 WO2014151341A1 (en) | 2013-03-15 | 2014-03-13 | Compositions and methods for treating mpsi |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| RU2015144234A true RU2015144234A (ru) | 2017-04-24 |
| RU2708318C2 RU2708318C2 (ru) | 2019-12-05 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2015144234A RU2708318C2 (ru) | 2013-03-15 | 2014-03-13 | Композиции и способы лечения mps1 |
Country Status (22)
| Country | Link |
|---|---|
| US (3) | US10137176B2 (ru) |
| EP (2) | EP3747998A1 (ru) |
| JP (4) | JP6591956B2 (ru) |
| KR (2) | KR102346455B1 (ru) |
| CN (1) | CN105026554B (ru) |
| AU (2) | AU2014235096B2 (ru) |
| BR (1) | BR112015021036A2 (ru) |
| CA (1) | CA2901328C (ru) |
| CY (1) | CY1123169T1 (ru) |
| DK (1) | DK2984166T3 (ru) |
| ES (1) | ES2805355T3 (ru) |
| HK (1) | HK1221735A1 (ru) |
| HR (1) | HRP20201063T1 (ru) |
| HU (1) | HUE051373T2 (ru) |
| LT (1) | LT2984166T (ru) |
| MX (2) | MX2015012739A (ru) |
| PL (1) | PL2984166T3 (ru) |
| PT (1) | PT2984166T (ru) |
| RU (1) | RU2708318C2 (ru) |
| SG (2) | SG10201707319UA (ru) |
| SI (1) | SI2984166T1 (ru) |
| WO (1) | WO2014151341A1 (ru) |
Cited By (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10137176B2 (en) | 2013-03-15 | 2018-11-27 | The Trustee Of The University Of Pennsylvania | Compositions and methods for treating MPSI |
| RU2788484C2 (ru) * | 2018-05-09 | 2023-01-19 | Байомарин Фармасьютикал Инк. | Способы лечения фенилкетонурии |
| US11584780B2 (en) | 2016-07-26 | 2023-02-21 | Biomarin Pharmaceutical Inc. | Adeno-associated virus capsid proteins |
| US11739345B2 (en) | 2018-05-09 | 2023-08-29 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| US11821008B2 (en) | 2018-05-14 | 2023-11-21 | Biomarin Pharmaceutical Inc. | Liver targeting adeno-associated viral vectors |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2946392A1 (en) * | 2014-04-25 | 2015-10-29 | James M. Wilson | Ldlr variants and their use in compositions for reducing cholesterol levels |
| WO2017106345A1 (en) * | 2015-12-14 | 2017-06-22 | The Trustees Of The University Of Pennsylvania | Composition for treatment of crigler-najjar syndrome |
| WO2017136533A1 (en) * | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Methods for treating diagnosing, and monitoring treatment of mucopolysaccharidoses |
| AU2017215211C1 (en) | 2016-02-03 | 2023-11-16 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| JP7231922B2 (ja) * | 2016-02-22 | 2023-03-02 | ザ ユニヴァーシティー オブ ノース カロライナ アト チャペル ヒル | Ｍｐｓ－ｉ関連失明の治療のためのａａｖ－ｉｄｕａベクター |
| WO2017180936A1 (en) * | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| KR20230125339A (ko) | 2016-04-15 | 2023-08-29 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| WO2018022511A1 (en) | 2016-07-25 | 2018-02-01 | The Trustees Of The University Of Pennsylvania | Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof |
| KR20190100318A (ko) | 2016-12-30 | 2019-08-28 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 페닐케톤뇨증을 치료하기 위한 유전자 치료 |
| SG11201912631PA (en) | 2017-07-06 | 2020-01-30 | Univ Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
| BR112020005249A2 (pt) | 2017-09-22 | 2020-09-24 | The Trustees Of The University Of Pennsylvania | terapia de gene para tratar mucopolissacaridose tipo ii |
| CN107699590A (zh) * | 2017-10-13 | 2018-02-16 | 成都中医药大学 | 一种制备重组人α‑L‑艾杜糖醛酸酶的方法 |
| WO2019108856A1 (en) | 2017-11-30 | 2019-06-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for mucopolysaccharidosis iiib |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| JP7244547B2 (ja) | 2018-02-19 | 2023-03-22 | ホモロジー・メディシンズ・インコーポレイテッド | Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法 |
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| US10137176B2 (en) | 2013-03-15 | 2018-11-27 | The Trustee Of The University Of Pennsylvania | Compositions and methods for treating MPSI |
| US11584780B2 (en) | 2016-07-26 | 2023-02-21 | Biomarin Pharmaceutical Inc. | Adeno-associated virus capsid proteins |
| RU2788484C2 (ru) * | 2018-05-09 | 2023-01-19 | Байомарин Фармасьютикал Инк. | Способы лечения фенилкетонурии |
| US11739345B2 (en) | 2018-05-09 | 2023-08-29 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| US11821008B2 (en) | 2018-05-14 | 2023-11-21 | Biomarin Pharmaceutical Inc. | Liver targeting adeno-associated viral vectors |
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