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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- 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
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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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| EP3134431B1 (en) | 2014-04-25 | 2021-04-07 | The Trustees Of The University Of Pennsylvania | Ldlr variants and their use in compositions for reducing cholesterol levels |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| WO2016073693A2 (en) | 2014-11-05 | 2016-05-12 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| CA2975583A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| CN114717264A (zh) | 2014-11-14 | 2022-07-08 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | 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 |
| US11535665B2 (en) | 2015-05-13 | 2022-12-27 | The Trustees Of The University Of Pennsylvania | AAV-mediated expression of anti-influenza antibodies and methods of use thereof |
| IL259850B2 (en) | 2015-12-11 | 2023-11-01 | Univ Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| US11197937B2 (en) * | 2016-04-15 | 2021-12-14 | The Trustees Of The University Of Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| SG11201809643UA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| IL262784B2 (en) | 2016-05-18 | 2023-10-01 | Voyager Therapeutics Inc | modulatory polynucleotides |
| 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 |
| WO2018044933A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| MX2019007876A (es) * | 2016-12-30 | 2019-10-15 | Univ Pennsylvania | Terapia genetica para tratar fenilcetonuria. |
| AU2018220212A1 (en) * | 2017-02-20 | 2019-08-08 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| PT3589730T (pt) | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
| 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 |
| WO2018204786A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| MX2019013172A (es) | 2017-05-05 | 2020-09-07 | Voyager Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de huntington. |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| JP7229989B2 (ja) | 2017-07-17 | 2023-02-28 | ボイジャー セラピューティクス インコーポレイテッド | 軌道アレイガイドシステム |
| AU2018311069A1 (en) | 2017-08-03 | 2020-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of AAV |
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| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| EP3902918A4 (en) * | 2018-12-20 | 2022-10-12 | The Trustees of the University of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| US20230235353A1 (en) * | 2020-03-19 | 2023-07-27 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav |
| IL297756A (en) * | 2020-04-29 | 2022-12-01 | Saliogen Therapeutics Inc | Compositions and methods for treatment of familial hypercholesterolemia and elevated low-density lipoprotein cholesterol |
| JP2023526310A (ja) | 2020-05-12 | 2023-06-21 | 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 | 導入遺伝子発現のｄｒｇ特異的低減のための組成物 |
| JP2023537625A (ja) | 2020-08-14 | 2023-09-04 | 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 | 新規ａａｖカプシド及びそれを含む組成物 |
| WO2022094180A1 (en) | 2020-10-29 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Aav capsids and compositions containing same |
| TW202237850A (zh) | 2020-12-01 | 2022-10-01 | 賓州大學委員會 | 具有組織特異性靶向基序的新穎構成物及含有其之組成物 |
| EP4334334A1 (en) | 2021-04-23 | 2024-03-13 | The Trustees of The University of Pennsylvania | Novel compositions with brain-specific targeting motifs and compositions containing same |
| AR125467A1 (es) | 2021-04-27 | 2023-07-19 | Univ Pennsylvania | Cápsides de virus adenoasociados derivados de porcinos y usos de los estos |
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| US20240384298A1 (en) | 2021-10-02 | 2024-11-21 | The Trustees Of The University Of Pennsylvania | Novel aav capsids and compositions containing same |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
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| EP3147295B2 (en) | 2011-08-24 | 2023-11-22 | The Board of Trustees of the Leland Stanford Junior University | New avv capsid proteins for nucleic acid transfer |
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| RU2653444C2 (ru) | 2012-02-17 | 2018-05-08 | Дзе Чилдрен'З Хоспитал Оф Филадельфия | Композиции вектора aav и способы переноса генов в клетки, органы и ткани |
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| CA2900231C (en) | 2013-02-15 | 2019-07-30 | Paul Albert Sieving | Aav8 retinoschisin expression vector for treating x-linked retinoschisis |
| MX386059B (es) * | 2013-03-15 | 2025-03-18 | Univ Pennsylvania | COMPOSICIONES Y SUS usos EN EL TRATAMIENTO DE LA MUCOPOLISACARIDOSIS TIPO I. |
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| 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 |
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| EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| AU2018220212A1 (en) | 2017-02-20 | 2019-08-08 | 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 WO PCT/US2015/027572 patent/WO2015164778A1/en not_active Ceased
- 2015-04-24 US US15/306,419 patent/US11555059B2/en active Active
- 2015-04-24 EP EP21166865.2A patent/EP3919508A1/en not_active Withdrawn
- 2015-04-24 BR BR112016024379A patent/BR112016024379A2/pt active Search and Examination
- 2015-04-24 ES ES15721097T patent/ES2876409T3/es active Active
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2020
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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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|---|---|
| US20230272032A1 (en) | 2023-08-31 |
| US11555059B2 (en) | 2023-01-17 |
| WO2015164778A1 (en) | 2015-10-29 |
| EP3919508A1 (en) | 2021-12-08 |
| BR112016024379A2 (pt) | 2017-10-10 |
| US20170101458A1 (en) | 2017-04-13 |
| JP7280853B2 (ja) | 2023-05-24 |
| JP2023100960A (ja) | 2023-07-19 |
| EP3134431A1 (en) | 2017-03-01 |
| ES2876409T3 (es) | 2021-11-12 |
| JP2020120673A (ja) | 2020-08-13 |
| JP2017513504A (ja) | 2017-06-01 |
| JP6741590B2 (ja) | 2020-08-19 |
| EP3134431B1 (en) | 2021-04-07 |
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