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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Prior art keywords
seq
viral
gene
delivery system
expression
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CA3025247A
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English (en)
French (fr)
Inventor
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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    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/08—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from viruses
    • C07K16/10—RNA viruses
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    • 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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
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    • A61K48/0008—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 '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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    • 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
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    • C07K14/43504—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from invertebrates
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    • C07K14/49—Platelet-derived growth factor [PDGF]
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    • C07K14/475—Growth factors; Growth regulators
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    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
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    • C07K16/2863—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against receptors for growth factors, growth regulators
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    • C07K16/32—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against translation products of oncogenes
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    • C12N15/113—Non-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
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    • C12N15/09—Recombinant DNA-technology
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    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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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)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010045659A1 (en) 2008-10-17 2010-04-22 American Gene Technologies International Inc. Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
US11980663B2 (en) 2015-07-08 2024-05-14 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
IL297238B2 (en) 2016-01-15 2024-08-01 American Gene Tech Int Inc Methods and preparations for gamma-delta T-cell activation
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
WO2017213697A1 (en) * 2016-06-08 2017-12-14 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
CA3028982A1 (en) 2016-07-08 2018-01-11 American Gene Technologies International Inc. Hiv pre-immunization and immunotherapy
WO2018017882A1 (en) 2016-07-21 2018-01-25 American Gene Technologies International Inc. Viral vectors for treating parkinson's disease
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
US12559769B2 (en) 2017-06-16 2026-02-24 American Gene Technologies International Inc. Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells
AU2021232603A1 (en) * 2020-03-03 2022-10-20 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

Family Cites Families (139)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5668255A (en) 1984-06-07 1997-09-16 Seragen, Inc. Hybrid molecules having translocation region and cell-binding region
AU6014094A (en) 1992-12-02 1994-06-22 Baylor College Of Medicine Episomal vectors for gene therapy
CA2144040A1 (fr) 1993-07-13 1995-01-26 Michel Perricaudet Vecteurs adenoviraux defectifs et utilisation en therapie genique
CA2300957A1 (en) * 1997-08-15 1999-02-25 Rubicon Laboratory, Inc. Retrovirus and viral vectors
AU1124499A (en) * 1997-10-28 1999-05-17 Maxygen, Inc. Human papillomavirus vectors
ATE286138T1 (de) 1998-03-20 2005-01-15 Univ Pennsylvania Zusammensetzungen und verfahren zur helfer-freien herstellung von rekombinante adeno-assoziierten viren
DE69940521D1 (de) 1998-10-01 2009-04-16 Univ Southern California Retrovirales genübertragungssystem und entsprechendes verwendungverfahren
US6156514A (en) 1998-12-03 2000-12-05 Sunol Molecular Corporation Methods for making recombinant cells
US6410013B1 (en) 1999-01-25 2002-06-25 Musc Foundation For Research Development Viral vectors for use in monitoring HIV drug resistance
WO2000072886A1 (en) 1999-05-26 2000-12-07 Dana-Farber Cancer Institute, Inc. Episomally replicating lentiviral vectors
AU2001257611A1 (en) 2000-04-28 2001-11-12 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
CA2408830A1 (en) 2000-05-12 2001-11-22 The Regents Of The University Of California Treatment of human papillomavirus (hpv)-infected cells
WO2001091802A1 (en) 2000-05-30 2001-12-06 Baylor College Of Medicine Chimeric viral vectors for gene therapy
NO314588B1 (no) 2000-09-04 2003-04-14 Bionor Immuno As HIV-peptider, antigener, vaksinesammensetning, immunoassay- testsett og en fremgangsmåte for å påvise antistoffer indusert av HIV
US7122181B2 (en) 2000-12-19 2006-10-17 Research Development Foundation Lentiviral vector-mediated gene transfer and uses thereof
US20030138444A1 (en) 2001-08-18 2003-07-24 Myriad Genetics, Incorporated Composition and method for treating HIV infection
US20030119770A1 (en) 2001-08-02 2003-06-26 Zhennan Lai Intercellular delivery of a herpes simplex virus VP22 fusion protein from cells infected with lentiviral vectors
US7737124B2 (en) 2001-09-13 2010-06-15 California Institute Of Technology Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
US20070203333A1 (en) 2001-11-30 2007-08-30 Mcswiggen James RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
WO2003079757A2 (en) 2002-03-20 2003-10-02 Massachusetts Institute Of Technology Hiv therapeutic
WO2003093436A2 (en) 2002-04-30 2003-11-13 University Of Florida Treatment for phenylketonuria
US20040161412A1 (en) * 2002-08-22 2004-08-19 The Cleveland Clinic Foundation Cell-based VEGF delivery
US8034334B2 (en) 2002-09-06 2011-10-11 The United States Of America As Represented By The Secretary, Department Of Health And Human Services Immunotherapy with in vitro-selected antigen-specific lymphocytes after non-myeloablative lymphodepleting chemotherapy
AU2003290586B2 (en) 2002-11-04 2009-07-02 University Of Massachusetts Allele-specific RNA interference
WO2004053137A2 (en) 2002-12-11 2004-06-24 Cytos Biotechnology Ag Method for protein production
EP1631669A2 (en) 2003-04-09 2006-03-08 Biodelivery Sciences International, Inc. Cochleate compositions directed against expression of proteins
US20060246520A1 (en) 2003-05-23 2006-11-02 Institut National De La Sante Et De La Recherche Medicale Gamma delta t cell-mediated therapy
EP1644508A1 (en) 2003-07-11 2006-04-12 Cytos Biotechnology AG Gene expression system
US20050019927A1 (en) 2003-07-13 2005-01-27 Markus Hildinger DECREASING GENE EXPRESSION IN A MAMMALIAN SUBJECT IN VIVO VIA AAV-MEDIATED RNAi EXPRESSION CASSETTE TRANSFER
US20050138677A1 (en) 2003-09-16 2005-06-23 Pfister Herbert J. Transgenic animal model for the treatment of skin tumors
EP1678292A4 (en) 2003-09-18 2008-05-07 Univ Emory IMPROVED MVA VACCINES
WO2005033282A2 (en) * 2003-10-01 2005-04-14 Pharmacia & Upjohn Company Llc Polyamide compositions and therapeutic methods for treatment of human papilloma virus
US20080039413A1 (en) 2003-10-21 2008-02-14 Morris David W Novel compositions and methods in cancer
EP1753777B1 (en) 2004-02-25 2014-05-07 Dana-Farber Cancer Institute, Inc. METHODS AND COMPOSITIONS FOR THE TREATMENT AND PREVENTION OF HIV INFECTION USING TRIM5a
WO2005085443A2 (en) 2004-03-01 2005-09-15 Massachusetts Institute Of Technology Rnai-based therapeutics for allergic rhinitis and asthma
TWI439284B (zh) 2004-04-09 2014-06-01 艾伯維生物技術有限責任公司 用於治療TNFα相關失調症之多重可變劑量療法
WO2005118802A2 (en) 2004-06-03 2005-12-15 The Regents Of The University Of California Targeting pseudotyped retroviral vectors
US20080131940A1 (en) 2004-06-25 2008-06-05 Robert Chiu Target Cell-Specific Short Interfering Rna and Methods of Use Thereof
WO2006023491A2 (en) 2004-08-16 2006-03-02 The Cbr Institute For Biomedical Research, Inc. Method of delivering rna interference and uses thereof
WO2006039721A2 (en) 2004-10-08 2006-04-13 The Board Of Trustees Of The University Of Illinois Bisphosphonate compounds and methods for bone resorption diseases, cancer, bone pain, immune disorders, and infectious diseases
EP1647595A1 (en) 2004-10-15 2006-04-19 Academisch Medisch Centrum bij de Universiteit van Amsterdam Nucleic acids against viruses, in particular HIV
CA2585523A1 (en) 2004-11-02 2006-05-11 Istituto Di Ricerche Di Biologia Molecolare P. Angeletti S.P.A. Adenoviral amplicon and producer cells for the production of replication-defective adenoviral vectors, methods of preparation and use thereof
US7790446B2 (en) * 2005-02-11 2010-09-07 Kosagen Cell Factory Oü Vectors, cell lines and their use in obtaining extended episomal maintenance replication of hybrid plasmids and expression of gene products
EP2573185A3 (en) 2005-02-16 2013-06-05 Lentigen Corporation Lentiviral vectors and their use
DK2002003T3 (en) 2005-05-27 2016-03-21 Ospedale San Raffaele Srl Gene vector comprising miRNA
US20070032443A1 (en) 2005-08-02 2007-02-08 Jaeseob Kim Therapy for Alzheimer's disease
WO2007015122A1 (en) 2005-08-02 2007-02-08 Genexel, Inc. Therapy for alzheimer’s disease
US20070105835A1 (en) 2005-11-07 2007-05-10 Kazantsev Aleksey G Compositions and methods for modulating poly(ADP-ribose) polymerase activity
GB0526211D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Viral vectors
WO2007133674A2 (en) 2006-05-12 2007-11-22 Lentigen Corporation Lentiviral vector compositions, methods and applications
WO2007149476A2 (en) 2006-06-19 2007-12-27 Trustees Of Columbia University In The City Of New York Assays for non-apoptotic cell death and uses thereof
US20080003225A1 (en) 2006-06-29 2008-01-03 Henri Vie Method for enhancing the antibody-dependent cellular cytotoxicity (ADCC) and uses of T cells expressing CD16 receptors
US8124752B2 (en) 2006-07-10 2012-02-28 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the MYC gene
EP1878440A1 (en) 2006-07-13 2008-01-16 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and compositions for increasing the efficiency of therapeutic antibodies using gamma delta cell activator compounds
RU2009106214A (ru) 2006-07-26 2010-09-10 Новартис АГ (CH) Ингибиторы ундекапренилпирофосфатсинтазы
US20080199961A1 (en) 2006-08-25 2008-08-21 Avi Biopharma, Inc. ANTISENSE COMPOSITION AND METHOD FOR INHIBITION OF miRNA BIOGENESIS
EP2415783B1 (en) 2006-10-16 2016-12-14 Genelux Corporation Modified vaccinia virus strains for use in a diagnostic and therapeutic method
WO2008090185A1 (en) 2007-01-23 2008-07-31 Virco Bvba Method for designing a drug regime for hiv-infected patients
JP2010523709A (ja) 2007-04-12 2010-07-15 ザ ボード オブ トラスティーズ オブ ザ ユニヴァーシティー オブ イリノイ Fpps、ggpps及びdppsを含む複数の標的に対して高い効能を有するビスホスホネート化合物及び方法
US20080293142A1 (en) 2007-04-19 2008-11-27 The Board Of Regents For Oklahoma State University Multiple shRNA Expression Vectors and Methods of Construction
EP2008656A1 (en) 2007-06-28 2008-12-31 Bergen Teknologioverforing AS Compositions for the treatment of hyperphenylalaninemia
WO2009026328A2 (en) 2007-08-21 2009-02-26 Immune Disease Institute, Inc. Methods of delivery of agents to leukocytes and endothelial cells
EP2090659A1 (en) 2008-02-14 2009-08-19 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Infectious particle, process for its preparation and use thereof
US8288094B2 (en) * 2008-04-09 2012-10-16 Institut Pasteur APOBEC3 mediated DNA editing
GB0810209D0 (en) 2008-06-04 2008-07-09 Cambridge Entpr Ltd Pluripotency associated epigenetic factor
US8629334B2 (en) 2008-07-16 2014-01-14 University Of Florida Research Foundation, Inc. Viral-based transient-expression vector system for trees
US20110207226A1 (en) 2008-08-20 2011-08-25 Virxsys Corporation Non-integrating lenti/adeno-associated virus hybrid vector system
EP2342321B1 (en) 2008-09-17 2018-04-11 Isogenis, Inc. Construction of fully-deleted adenovirus-based gene delivery vectors and uses thereof
WO2010045659A1 (en) 2008-10-17 2010-04-22 American Gene Technologies International Inc. Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
WO2010051521A1 (en) 2008-10-31 2010-05-06 Lentigen Corporation Cell therapy product for the treatment of hiv infection
JP2012507299A (ja) 2008-10-31 2012-03-29 バイオジェン・アイデック・エムエイ・インコーポレイテッド Light標的分子およびその使用
JP2012508591A (ja) 2008-11-14 2012-04-12 ライフ テクノロジーズ コーポレーション 細胞を操作するための組成物および方法
EP2191834A1 (en) 2008-11-26 2010-06-02 Centre National De La Recherche Scientifique (Cnrs) Compositions and methods for treating retrovirus infections
CN102438631A (zh) 2009-04-09 2012-05-02 史坦赛特股份有限公司 抗hiv干细胞及其用途
JP5727458B2 (ja) 2009-04-13 2015-06-03 アプセト ゲゼルシャフト ミット ベシュレンクテル ハフツング ウント コンパニー コマンディトゲゼルシャフトApceth Gmbh & Co. Kg 改変間葉系幹細胞及びそれを使用した腫瘍の治療方法
EP2425001A4 (en) 2009-04-30 2012-11-14 Univ California ANTI-HIV COMBINATION VECTORS, TARGETING VECTORS AND METHOD OF USE
US20120201794A1 (en) 2009-07-15 2012-08-09 Calimmune Inc. Dual vector for inhibition of human immunodeficiency virus
US20120027725A1 (en) 2009-11-30 2012-02-02 Galvin Jeffrey A Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules to treat liver cancer
CN101805750B (zh) 2009-12-29 2011-11-30 浙江大学 法呢基焦磷酸合成酶rna干扰重组慢病毒载体构建及用途
AU2011217955A1 (en) 2010-02-18 2012-08-16 Emory University Vectors expressing HIV antigens and GM-CSF and related methods for generating an immune response
WO2011119942A1 (en) 2010-03-25 2011-09-29 Vistagen Therapeutics, Inc. Induction of ips cells using transient episomal vectors
US20130090371A1 (en) 2010-04-20 2013-04-11 President And Fellows Of Harvard College Methods and compositions for inhibition of beta2-adrenergic receptor degradation
PT2561078T (pt) 2010-04-23 2018-12-03 Cold Spring Harbor Laboratory Sharn com uma conceção estrutural inovadora
CN102946907A (zh) 2010-05-28 2013-02-27 牛津生物医学(英国)有限公司 慢病毒载体向脑的递送
JP5805089B2 (ja) 2010-08-10 2015-11-04 タカラバイオ株式会社 細胞集団の製造方法
EP2625294A4 (en) 2010-10-07 2014-03-26 Univ Columbia PROCESS FOR TREATING TUMORS WITH A P53 MUTATION
WO2012061075A2 (en) 2010-10-25 2012-05-10 The Regents Of The University Of California Hiv resistant and functional hematopoietic stem/progenitor cells and macrophages from induced pluripotent stem cells
JP6091435B2 (ja) 2011-02-22 2017-03-08 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス(aav)ベクターを用いたタンパク質の送達
WO2012145624A2 (en) 2011-04-21 2012-10-26 University Of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
EP2782596A4 (en) 2011-11-22 2015-07-29 Philadelphia Children Hospital VIRUS VECTORS FOR HIGHLY EFFICIENT TRANSGENIC ADMINISTRATION
US9745631B2 (en) 2011-12-20 2017-08-29 Dana-Farber Cancer Institute, Inc. Methods for diagnosing and treating oncogenic kras-associated cancer
SG10201604810UA (en) 2012-02-07 2016-08-30 Global Bio Therapeutics Inc Compartmentalized method of nucleic acid delivery and compositions and uses thereof
WO2013174404A1 (en) 2012-05-23 2013-11-28 Ganymed Pharmaceuticals Ag Combination therapy involving antibodies against claudin 18.2 for treatment of cancer
US20150174235A1 (en) 2012-06-06 2015-06-25 Bionor Immuno As Vaccine
US9556433B2 (en) * 2012-07-17 2017-01-31 Université De Genève Nucleic acids for down-regulation of gene expression
WO2014052855A1 (en) 2012-09-27 2014-04-03 Population Diagnostics, Inc. Methods and compositions for screening and treating developmental disorders
EP3415627A1 (en) 2012-11-13 2018-12-19 Codiak BioSciences, Inc. Delivery of therapeutic agent
CA2892448A1 (en) 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
US9642921B2 (en) 2012-12-20 2017-05-09 Tocagen Inc. Cancer combination therapy and recombinant vectors
WO2014117050A2 (en) 2013-01-26 2014-07-31 Mirimus, Inc. Modified mirna as a scaffold for shrna
CN103184224A (zh) 2013-04-03 2013-07-03 衡阳师范学院 一种抗艾滋病病毒感染的三联miRNA及构建方法
US10501803B2 (en) 2013-05-21 2019-12-10 Max-Planck Gesellschaft zur Förderung der Wissenschaften e.V. Isoforms of GATA6 and NKX2-1 as markers for diagnosis and therapy of cancer and as targets for anti-cancer therapy
EP3027755B1 (en) 2013-08-02 2019-10-09 The Regents of The University of California Engineering antiviral t cell immunity through stem cells and chimeric antigen receptors
WO2015042308A2 (en) 2013-09-18 2015-03-26 City Of Hope Rna-based hiv inhibitors
EP3574923A1 (en) 2013-10-22 2019-12-04 Translate Bio, Inc. Mrna therapy for phenylketonuria
CN111218447B (zh) 2013-11-07 2024-10-11 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
EP2878674A1 (en) 2013-11-28 2015-06-03 Fundación Centro Nacional de Investigaciones Cardiovasculares Carlos III (CNIC) Stable episomes based on non-integrative lentiviral vectors
GB201322091D0 (en) 2013-12-13 2014-01-29 Cambridge Entpr Ltd Modified serpins for the treatment of bleeding disorders
IL292038B2 (en) 2014-04-23 2024-12-01 Juno Therapeutics Inc Methods for enriching or producing immune cell populations for adoptive therapy
ES2744565T3 (es) 2014-04-25 2020-02-25 Genethon Tratamiento de la hiperbilirrubinemia
WO2015164759A2 (en) 2014-04-25 2015-10-29 Bluebird Bio, Inc. Mnd promoter chimeric antigen receptors
WO2016033163A1 (en) 2014-08-29 2016-03-03 Immunomedics, Inc. Identification of cancer genes by in-vivo fusion of human cancer cells and animal cells
WO2016046234A2 (en) 2014-09-22 2016-03-31 Nexttobe Ab Recombinant phe-free proteins for use in the treatment of phenylketonuria
AU2015329696A1 (en) 2014-10-10 2017-04-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Methods to eliminate cancer stem cells by targeting CD47
US10968451B2 (en) 2014-10-14 2021-04-06 Texas Tech University System Multiplexed shRNAs and uses thereof
WO2016069716A1 (en) 2014-10-30 2016-05-06 The Scripps Research Institute Compositions and methods comprising tyrosyl-trna synthetases and resveratrol compounds
GB201509202D0 (en) 2015-05-28 2015-07-15 Ge Healthcare Bio Sciences Ab Semi-static cell culture
WO2016200997A1 (en) * 2015-06-10 2016-12-15 America Gene Technologies International, Inc. Non-integrating viral delivery system and methods of use thereof
US11980663B2 (en) 2015-07-08 2024-05-14 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
CA2994192C (en) 2015-08-13 2020-06-16 Peking University Induced extended pluripotent stem cells, methods of making and using
CN105112370B (zh) 2015-08-25 2019-02-05 杭州优善生物科技有限公司 一种体外刺激外周血γδT细胞高效增殖的方法及其应用
JP7059179B2 (ja) 2015-10-20 2022-04-25 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 遺伝子操作のための方法及び製品
WO2017100551A1 (en) 2015-12-09 2017-06-15 Alexion Pharmaceuticals, Inc. HETEROLOGOUS UTR SEQUENCES FOR ENHANCED mRNA EXPRESSION
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
IL297238B2 (en) 2016-01-15 2024-08-01 American Gene Tech Int Inc Methods and preparations for gamma-delta T-cell activation
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
WO2017173453A1 (en) 2016-04-01 2017-10-05 The Brigham And Women's Hospital, Inc. Stimuli-responsive nanoparticles for biomedical applications
WO2017213697A1 (en) 2016-06-08 2017-12-14 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
CA3028982A1 (en) 2016-07-08 2018-01-11 American Gene Technologies International Inc. Hiv pre-immunization and immunotherapy
WO2018017882A1 (en) 2016-07-21 2018-01-25 American Gene Technologies International Inc. Viral vectors for treating parkinson's disease
EP3562494A4 (en) 2016-12-30 2020-08-19 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR THE TREATMENT OF PHENYLKETONURIS
KR20230093381A (ko) 2017-01-09 2023-06-27 아메리칸 진 테크놀로지스 인터내셔널 인코포레이티드 예비-면역화 단계가 없는 hiv 면역요법
US12023385B2 (en) 2017-02-08 2024-07-02 Dana-Farber Cancer Institute, Inc. Tunable endogenous protein degradation with heterobifunctional compounds
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
US12559769B2 (en) 2017-06-16 2026-02-24 American Gene Technologies International Inc. Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells
KR20200057766A (ko) 2017-10-02 2020-05-26 아메리칸 진 테크놀로지스 인터내셔널 인코포레이티드 페닐케톤뇨증을 치료하기 위한 프로모터 및 인핸서 조합을 포함하는 벡터
EP3876952A4 (en) 2018-11-05 2022-08-24 American Gene Technologies International Inc. VECTOR SYSTEM FOR EXPRESSING REGULATORY RNA
BR112021024124A2 (pt) 2019-05-31 2022-01-11 American Gene Tech Int Inc Expressão otimizada de fenilalanina hidroxilase

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