CA3025247A1 - Non-integrating viral delivery system and methods related thereto - Google Patents

Non-integrating viral delivery system and methods related thereto Download PDF

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Publication number
CA3025247A1
CA3025247A1 CA3025247A CA3025247A CA3025247A1 CA 3025247 A1 CA3025247 A1 CA 3025247A1 CA 3025247 A CA3025247 A CA 3025247A CA 3025247 A CA3025247 A CA 3025247A CA 3025247 A1 CA3025247 A1 CA 3025247A1
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seq
viral
gene
delivery system
expression
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French (fr)
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Charles David PAUZA
Tyler LAHUSEN
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American Gene Technologies International Inc
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American Gene Technologies International Inc
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    • C07K16/10RNA viruses
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    • A61K38/18Growth factors; Growth regulators
    • A61K38/1858Platelet-derived growth factor [PDGF]
    • A61K38/1866Vascular endothelial growth factor [VEGF]
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    • A61K48/0008Medicinal 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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    • C12N15/1138Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
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CA3025247A 2016-06-08 2017-06-07 Non-integrating viral delivery system and methods related thereto Pending CA3025247A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662347552P 2016-06-08 2016-06-08
US62/347,552 2016-06-08
US201662431760P 2016-12-08 2016-12-08
US62/431,760 2016-12-08
USPCT/US2016/066185 2016-12-12
PCT/US2016/066185 WO2017213697A1 (en) 2016-06-08 2016-12-12 Non-integrating viral delivery system and methods related thereto
PCT/US2017/036433 WO2017214327A2 (en) 2016-06-08 2017-06-07 Non-integrating viral delivery system and methods related thereto

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CA3025247A1 true CA3025247A1 (en) 2017-12-14

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CA3025247A Pending CA3025247A1 (en) 2016-06-08 2017-06-07 Non-integrating viral delivery system and methods related thereto

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US (4) US11976292B2 (enExample)
EP (2) EP3468617A4 (enExample)
JP (5) JP7173548B2 (enExample)
KR (2) KR102762623B1 (enExample)
CN (1) CN109641064A (enExample)
AU (2) AU2017279542A1 (enExample)
BR (1) BR112018075399A2 (enExample)
CA (1) CA3025247A1 (enExample)
IL (1) IL263402A (enExample)
WO (2) WO2017213697A1 (enExample)

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CN111569090A (zh) 2016-01-15 2020-08-25 美国基因技术国际有限公司 用于活化γ-δ T细胞的方法和组合物
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
JP7153332B2 (ja) 2016-02-08 2022-10-14 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド Hivワクチン接種および免疫療法
ES2911448T3 (es) 2016-03-09 2022-05-19 American Gene Tech Int Inc Vectores combinados y métodos para el tratamiento del cáncer
US11976292B2 (en) * 2016-06-08 2024-05-07 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
EP3481418A4 (en) 2016-07-08 2020-03-11 American Gene Technologies International Inc. PRE-IMMUNIZATION AND IMMUNOTHERAPY OF HIV
US11583562B2 (en) 2016-07-21 2023-02-21 American Gene Technologies International Inc. Viral vectors for treating Parkinson's disease
JP2020512815A (ja) 2017-04-03 2020-04-30 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド フェニルケトン尿症を処置するための組成物および方法
WO2018232359A1 (en) 2017-06-16 2018-12-20 American Gene Technologies International Inc. Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells
WO2021178571A1 (en) * 2020-03-03 2021-09-10 American Gene Technologies International Inc. On demand expression of exogenous factors in lymphocytes to treat hiv
CN112029803A (zh) * 2020-09-21 2020-12-04 西人马联合测控(泉州)科技有限公司 一种慢病毒过表达病毒载体及其制备方法、用途
CN113046330B (zh) * 2021-03-23 2023-03-21 中吉智药(南京)生物技术有限公司 携带红系基因编辑系统的慢病毒及药物
CN114395586A (zh) * 2022-01-12 2022-04-26 中国科学院天津工业生物技术研究所 非整合慢病毒载体系统在基因编辑器递送中的应用
CN114317610B (zh) * 2022-01-27 2024-07-02 上海本导基因技术有限公司 一种适用于帕金森疾病基因治疗的慢病毒载体

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