CA3209883A1 - Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues - Google Patents
Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissuesInfo
- Publication number
- CA3209883A1 CA3209883A1 CA3209883A CA3209883A CA3209883A1 CA 3209883 A1 CA3209883 A1 CA 3209883A1 CA 3209883 A CA3209883 A CA 3209883A CA 3209883 A CA3209883 A CA 3209883A CA 3209883 A1 CA3209883 A1 CA 3209883A1
- Authority
- CA
- Canada
- Prior art keywords
- aav
- sequence
- seq
- rhm15
- protein
- 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.)
- Pending
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| US9255154B2 (en) | 2012-05-08 | 2016-02-09 | Alderbio Holdings, Llc | Anti-PCSK9 antibodies and use thereof |
| MX2016001044A (es) * | 2013-07-22 | 2016-09-09 | The Children's Hospital Of Philadelphia | Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos. |
| CN114395559A (zh) | 2014-04-25 | 2022-04-26 | 吉尼松公司 | 高胆红素血症的治疗 |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| SG11201703148TA (en) | 2014-11-05 | 2017-05-30 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| MX2017006217A (es) | 2014-11-14 | 2018-05-02 | Voyager Therapeutics Inc | Polinucleotidos moduladores. |
| US10597660B2 (en) | 2014-11-14 | 2020-03-24 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| HK1245326A1 (zh) | 2014-12-12 | 2018-08-24 | Voyager Therapeutics, Inc. | 用於生产scaav的组合物和方法 |
| US10081659B2 (en) | 2015-04-06 | 2018-09-25 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Adeno-associated vectors for enhanced transduction and reduced immunogenicity |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| JP6929230B2 (ja) * | 2015-05-14 | 2021-09-01 | セント ジュード チルドレンズ リサーチ ホスピタル インコーポレイテッド | スペーサーを含む核酸分子およびその使用の方法 |
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| PE20231949A1 (es) * | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| KR20210005994A (ko) | 2015-11-05 | 2021-01-15 | 뱀부 테라퓨틱스 인코포레이티드 | 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터 |
| RU2766583C2 (ru) * | 2015-12-01 | 2022-03-15 | Спарк Терапьютикс, Инк. | Масштабируемые способы получения рекомбинантного вектора на основе аденоассоциированного вируса (aav) в системе бессывороточной суспензионной культуры клеток, подходящего для клинического применения |
| EP3384034B1 (en) | 2015-12-02 | 2020-07-08 | The Board of Trustees of the Leland Stanford Junior University | Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism |
| WO2017096162A1 (en) | 2015-12-02 | 2017-06-08 | Voyager Therapeutics, Inc. | Assays for the detection of aav neutralizing antibodies |
| US10406244B2 (en) | 2015-12-02 | 2019-09-10 | The Board Of Trustees Of The Leland Stanford Junior University | AAV vectors with expanded packaging capacity |
| US20180363000A1 (en) * | 2015-12-14 | 2018-12-20 | The Trustees Of The University Of Pennsylvania | Aav-anti pcsk9 antibody constructs and uses thereof |
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