CA2946392A1 - Ldlr variants and their use in compositions for reducing cholesterol levels - Google Patents
Ldlr variants and their use in compositions for reducing cholesterol levels Download PDFInfo
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- A61P3/06—Antihyperlipidemics
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- 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
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- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- 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
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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| CN110769845A (zh) * | 2017-04-21 | 2020-02-07 | 精密生物科学公司 | 对pcsk9基因中的识别序列具有特异性的工程化大范围核酸酶 |
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| BR112016024379A2 (pt) | 2014-04-25 | 2017-10-10 | Univ Pennsylvania | variantes ldlr e seu uso em composições para reduzir os níveis de colesterol |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| SG10202007103TA (en) | 2014-11-05 | 2020-09-29 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| IL284949B2 (en) | 2014-11-14 | 2024-06-01 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| WO2016164762A1 (en) * | 2015-04-08 | 2016-10-13 | Moderna Therapeutics, Inc. | Polynucleotides encoding low density lipoprotein receptor egf-a and intracellular domain mutants and methods of using the same |
| EP3307310A2 (en) | 2015-05-13 | 2018-04-18 | The Trustees Of The University Of Pennsylvania | Aav-mediated expression of anti-influenza antibodies and methods of use thereof |
| EP3400304B1 (en) * | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| JP7046828B2 (ja) * | 2016-04-15 | 2022-04-04 | 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 | 滲出型加齢性黄斑変性の治療のための組成物 |
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| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| AU2017268382B2 (en) | 2016-05-18 | 2023-09-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| EP3458588B1 (en) | 2016-05-18 | 2025-10-29 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| US11883470B2 (en) | 2016-07-25 | 2024-01-30 | The Trustees Of The University Of Pennsylvania | Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof |
| US11298041B2 (en) | 2016-08-30 | 2022-04-12 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| WO2018126112A1 (en) * | 2016-12-30 | 2018-07-05 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating phenylketonuria |
| US11554147B2 (en) | 2017-02-20 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| CN110573523A (zh) | 2017-02-28 | 2019-12-13 | 宾夕法尼亚州大学信托人 | 基于aav载体的流感疫苗 |
| EP3589730B1 (en) | 2017-02-28 | 2023-12-27 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clade f vector and uses therefor |
| CN111108198A (zh) | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
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| TWI832036B (zh) | 2017-08-03 | 2024-02-11 | 美商航海家醫療公司 | 用於aav之遞送之組合物及方法 |
| EP3687582A4 (en) | 2017-09-29 | 2021-07-14 | Voyager Therapeutics, Inc. | RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| CN111479924B (zh) | 2017-10-16 | 2024-06-14 | 沃雅戈治疗公司 | 肌萎缩性侧索硬化症(als)的治疗 |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
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| WO2020132155A1 (en) * | 2018-12-20 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| JP2023518415A (ja) * | 2020-03-19 | 2023-05-01 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | 組換えａａｖにおいてｃａｐおよびｒｅｐ配列の逆パッケージングを低減するための組成物および方法 |
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| CN103717615A (zh) * | 2011-06-20 | 2014-04-09 | 霍夫曼-拉罗奇有限公司 | 结合pcsk9的多肽及使用方法 |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| EP2748185A1 (en) | 2011-08-24 | 2014-07-02 | The Board of Trustees of The Leland Stanford Junior University | New aav capsid proteins for nucleic acid transfer |
| MY170089A (en) | 2011-09-16 | 2019-07-04 | Regeneron Pharma | Methods for reducing lipoproten(a) levels by administering an inhibitor of proprotein convertase subtilisin kexin-9 (pcsk9) |
| BR112014019431A8 (pt) * | 2012-02-07 | 2017-07-11 | Global Bio Therapeutics Usa Inc | Método compartimentalizado de distribuição de ácido nucleico e composições e usos desses |
| KR102057540B1 (ko) | 2012-02-17 | 2019-12-19 | 더 칠드런스 호스피탈 오브 필라델피아 | 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법 |
| EP2929017A4 (en) * | 2012-12-05 | 2016-09-28 | Sangamo Biosciences Inc | METHOD AND COMPOSITIONS FOR REGULATING METABOLISM DISEASES |
| US9567376B2 (en) | 2013-02-08 | 2017-02-14 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| US9873893B2 (en) | 2013-02-15 | 2018-01-23 | The United States Of America As Represented By The Secretary, Dept. Of Health And Human Services | Methods and compositions for treating genetically linked diseases of the eye |
| US9254332B2 (en) | 2013-03-15 | 2016-02-09 | Arecor Limited | Stable aqueous formulations of adenovirus vectors |
| PL2984166T3 (pl) * | 2013-03-15 | 2021-01-11 | The Trustees Of The University Of Pennsylvania | Kompozycje do leczenia mpsi |
| 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 |
| US20150065556A1 (en) * | 2013-08-05 | 2015-03-05 | Whitehead Institute For Biomedical Research | Therapeutic targets for mitochondrial disorders |
| JP2016538829A (ja) * | 2013-10-03 | 2016-12-15 | モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 低密度リポタンパク質受容体をコードするポリヌクレオチド |
| BR112016024379A2 (pt) | 2014-04-25 | 2017-10-10 | Univ Pennsylvania | variantes ldlr e seu uso em composições para reduzir os níveis de colesterol |
| EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| EP3400304B1 (en) | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| US11554147B2 (en) | 2017-02-20 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
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2015
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- 2015-04-24 CA CA2946392A patent/CA2946392A1/en active Pending
- 2015-04-24 JP JP2016564200A patent/JP6741590B2/ja active Active
- 2015-04-24 WO PCT/US2015/027572 patent/WO2015164778A1/en not_active Ceased
- 2015-04-24 EP EP21166865.2A patent/EP3919508A1/en not_active Withdrawn
- 2015-04-24 EP EP15721097.2A patent/EP3134431B1/en active Active
- 2015-04-24 ES ES15721097T patent/ES2876409T3/es active Active
- 2015-04-24 US US15/306,419 patent/US11555059B2/en active Active
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2020
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- 2023-05-12 JP JP2023078988A patent/JP2023100960A/ja active Pending
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN110769845A (zh) * | 2017-04-21 | 2020-02-07 | 精密生物科学公司 | 对pcsk9基因中的识别序列具有特异性的工程化大范围核酸酶 |
| CN110769845B (zh) * | 2017-04-21 | 2023-08-15 | 精密生物科学公司 | 对pcsk9基因中的识别序列具有特异性的工程化大范围核酸酶 |
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| Publication number | Publication date |
|---|---|
| EP3919508A1 (en) | 2021-12-08 |
| WO2015164778A1 (en) | 2015-10-29 |
| JP2017513504A (ja) | 2017-06-01 |
| JP6741590B2 (ja) | 2020-08-19 |
| US11555059B2 (en) | 2023-01-17 |
| EP3134431A1 (en) | 2017-03-01 |
| ES2876409T3 (es) | 2021-11-12 |
| US20230272032A1 (en) | 2023-08-31 |
| JP7280853B2 (ja) | 2023-05-24 |
| EP3134431B1 (en) | 2021-04-07 |
| US20170101458A1 (en) | 2017-04-13 |
| JP2023100960A (ja) | 2023-07-19 |
| JP2020120673A (ja) | 2020-08-13 |
| BR112016024379A2 (pt) | 2017-10-10 |
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