BR112016028023A2 - Composições e métodos de administração de tratamentos para infecções virais latentes - Google Patents

Composições e métodos de administração de tratamentos para infecções virais latentes

Info

Publication number
BR112016028023A2
BR112016028023A2 BR112016028023A BR112016028023A BR112016028023A2 BR 112016028023 A2 BR112016028023 A2 BR 112016028023A2 BR 112016028023 A BR112016028023 A BR 112016028023A BR 112016028023 A BR112016028023 A BR 112016028023A BR 112016028023 A2 BR112016028023 A2 BR 112016028023A2
Authority
BR
Brazil
Prior art keywords
methods
compositions
administration
treatments
therapeutic agents
Prior art date
Application number
BR112016028023A
Other languages
English (en)
Inventor
R Quake Stephen
Wang Jianbin
Original Assignee
Univ Leland Stanford Junior
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Univ Leland Stanford Junior filed Critical Univ Leland Stanford Junior
Publication of BR112016028023A2 publication Critical patent/BR112016028023A2/pt

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1761Apoptosis related proteins, e.g. Apoptotic protease-activating factor-1 (APAF-1), Bax, Bax-inhibitory protein(s)(BI; bax-I), Myeloid cell leukemia associated protein (MCL-1), Inhibitor of apoptosis [IAP] or Bcl-2
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6901Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/18Antivirals for RNA viruses for HIV
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
    • A61P31/22Antivirals for DNA viruses for herpes viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1131Non-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 viruses
    • C12N15/1133Non-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 viruses against herpetoviridae, e.g. HSV
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • 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
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases RNAses, DNAses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2820/00Vectors comprising a special origin of replication system
    • C12N2820/60Vectors comprising a special origin of replication system from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Abstract

COMPOSIÇÕES E MÉTODOS DE ADMINISTRAÇÃO DE TRATAMENTOS PARA INFECÇÕES VIRAIS LATENTES. A presente invenção refere-se a métodos de administração e composições para agentes terapêuticos antivirais. São providos métodos e composições para administração direcionada de agentes terapêuticos antivirais a células de interesse usando, por exemplo, vetores virais tais como adenovírus, AAV e HSV incompetente em replicação. Estes e outros sistemas de administração podem ser usados como veículos para administrar vetores de DNA codificando uma nuclease ou um gene assassino de célula. Estes métodos de administração podem ser também usados para administrar DNA ou RNA nu, produtos de proteína, plasmídeos contendo um promotor que é ativo apenas em um estado viral latente que aciona um gene assassino de célula, ou outros agentes terapêuticos.
BR112016028023A 2014-05-30 2015-05-29 Composições e métodos de administração de tratamentos para infecções virais latentes BR112016028023A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462005395P 2014-05-30 2014-05-30
US201462029072P 2014-07-25 2014-07-25
PCT/US2015/033186 WO2015184262A1 (en) 2014-05-30 2015-05-29 Compositions and methods of delivering treatments for latent viral infections

Publications (1)

Publication Number Publication Date
BR112016028023A2 true BR112016028023A2 (pt) 2017-08-22

Family

ID=53366336

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112016028023A BR112016028023A2 (pt) 2014-05-30 2015-05-29 Composições e métodos de administração de tratamentos para infecções virais latentes

Country Status (10)

Country Link
US (7) US10066241B2 (pt)
EP (3) EP3149169A1 (pt)
JP (3) JP2017518075A (pt)
KR (1) KR20170005494A (pt)
CN (2) CN107002094A (pt)
AU (3) AU2015266776A1 (pt)
BR (1) BR112016028023A2 (pt)
CA (2) CA2949713A1 (pt)
SG (1) SG11201610028TA (pt)
WO (3) WO2015184268A1 (pt)

Families Citing this family (69)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6261500B2 (ja) 2011-07-22 2018-01-17 プレジデント アンド フェローズ オブ ハーバード カレッジ ヌクレアーゼ切断特異性の評価および改善
AU2014207618A1 (en) 2013-01-16 2015-08-06 Emory University Cas9-nucleic acid complexes and uses related thereto
PT3456831T (pt) 2013-04-16 2021-09-10 Regeneron Pharma Modificação alvejada do genoma de rato
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
CA2922428A1 (en) 2013-08-29 2015-03-05 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
KR102170502B1 (ko) 2013-12-11 2020-10-28 리제너론 파마슈티칼스 인코포레이티드 게놈의 표적화된 변형을 위한 방법 및 조성물
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
JP2017518075A (ja) 2014-05-30 2017-07-06 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 潜伏性ウイルス感染を処置するための組成物および方法
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
PT3221457T (pt) 2014-11-21 2019-06-27 Regeneron Pharma Métodos e composições para modificação genética visada através da utilização de arn guia emparelhados
WO2016089433A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
US10059940B2 (en) * 2015-01-27 2018-08-28 Minghong Zhong Chemically ligated RNAs for CRISPR/Cas9-lgRNA complexes as antiviral therapeutic agents
AU2016246450B2 (en) 2015-04-06 2022-03-17 Agilent Technologies, Inc. Chemically modified guide RNAs for CRISPR/Cas-mediated gene regulation
US20160346362A1 (en) * 2015-05-29 2016-12-01 Agenovir Corporation Methods and compositions for treating cytomegalovirus infections
EP3324999A1 (en) * 2015-05-29 2018-05-30 Agenovir Corporation Compositions and methods for cell targeted hpv treatment
CA3000182A1 (en) * 2015-05-29 2016-12-08 Agenovir Corporation Methods and compositions for treating cells for transplant
US10117911B2 (en) 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
WO2017058793A1 (en) * 2015-09-29 2017-04-06 Agenovir Corporation Delivery methods and compositions
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
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
CA3006305A1 (en) 2015-12-09 2017-06-15 Excision Biotherapeutics, Inc. Gene editing methods and compositions for eliminating risk of jc virus activation and pml (progressive multifocal leukoencephalopathy) during immunosuppressive therapy
JP2019512458A (ja) * 2016-01-25 2019-05-16 エクシジョン バイオセラピューティクス インコーポレイテッド Rnaによって誘導された、ヒトjcウイルス及び他のポリオーマウイルスの根絶
WO2017132112A1 (en) * 2016-01-25 2017-08-03 Excision Biotherapeutics Methods and compositions for rna-guided treatment of hiv infection
CA3015353A1 (en) * 2016-02-25 2017-08-31 Agenovir Corporation Viral and oncoviral nuclease treatment
US20170246260A1 (en) * 2016-02-25 2017-08-31 Agenovir Corporation Modified antiviral nuclease
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
WO2017192172A1 (en) * 2016-05-05 2017-11-09 Temple University - Of The Commonwealth System Of Higher Education Rna guided eradication of varicella zoster virus
EP3455347A4 (en) * 2016-05-10 2019-10-02 United States Government as Represented by The Department of Veterans Affairs LENTIVIRAL DELIVERY OF CRISPR / CAS CONSTRUCTS COLUMNS ESSENTIAL GENES FOR HIV-1 INFECTION AND REPLICATION
AU2017273713A1 (en) * 2016-06-01 2018-10-25 Excision Biotherapeutics, Inc. Compositions and methods of treatment for lytic and lysogenic viruses
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
CA3033327A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US11801374B2 (en) * 2016-09-02 2023-10-31 The General Hospital Corporation Methods and systems for non-contact construction of an internal structure
GB2573062A (en) 2016-10-14 2019-10-23 Harvard College AAV delivery of nucleobase editors
KR102305215B1 (ko) 2016-10-14 2021-09-28 프리시젼 바이오사이언시스 인코포레이티드 B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제
US11584931B2 (en) 2016-12-14 2023-02-21 The J. David Gladstone Institutes Methods and compositions for generating a deletion library and for identifying a defective interfering particle (DIP)
CN106729753A (zh) * 2016-12-16 2017-05-31 谭旭 抗乙型肝炎病毒的递送系统和生物制剂
WO2018118586A1 (en) * 2016-12-22 2018-06-28 Agenovir Corporation Antiviral/endogenous combination therapy
WO2018118587A1 (en) * 2016-12-22 2018-06-28 Agenovir Corporation Modified polynucleotides for antiviral therapy
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US20180201921A1 (en) * 2017-01-18 2018-07-19 Excision Biotherapeutics, Inc. CRISPRs
US20190071673A1 (en) * 2017-01-18 2019-03-07 Thomas Malcolm CRISPRs WITH IMPROVED SPECIFICITY
CA3047350A1 (en) * 2017-01-26 2018-08-02 Thomas MALCOLM Lentivirus and non-integrating lentivirus as viral vector to deliver crispr therapeutic
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
KR20190130613A (ko) 2017-03-23 2019-11-22 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
US20220090127A1 (en) * 2017-06-02 2022-03-24 Institut National De La Sante Et De La Recherche Medicale (Inserm Viral vector combining gene therapy and genome editing approaches for gene therapy of genetic disorders
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
JP2020533968A (ja) * 2017-08-25 2020-11-26 オービッド・セラピューティクス・インコーポレイテッドOvid Therapeutics Inc. 組換えアデノ随伴ベクター
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
WO2019079347A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. USES OF BASIC EDITORS ADENOSINE
US11142750B2 (en) 2018-04-12 2021-10-12 Precision Biosciences, Inc. Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome
WO2019204210A1 (en) * 2018-04-16 2019-10-24 Georgia Tech Research Corporation Mrna driven expression of rna editors for treatment of pathologies
CN109943563A (zh) * 2019-03-08 2019-06-28 内蒙古大学 CRISPR-Cas9系统介导的狂犬病病毒基因组敲除的方法
KR20210143230A (ko) 2019-03-19 2021-11-26 더 브로드 인스티튜트, 인코퍼레이티드 뉴클레오티드 서열을 편집하기 위한 방법 및 조성물
EP3981002A4 (en) * 2019-06-06 2023-08-09 Tata Consultancy Services Limited METHOD AND SYSTEM FOR IDENTIFYING TARGET SITES IN PROTEIN ENCODING REGIONS TO CONTROL PATHOGENS
CN112168808B (zh) * 2019-07-02 2023-01-24 深圳市第二人民医院 一种基于crispr的细胞核靶向药物递送系统
CN116096873A (zh) 2020-05-08 2023-05-09 布罗德研究所股份有限公司 同时编辑靶标双链核苷酸序列的两条链的方法和组合物
CN112795696A (zh) * 2020-12-31 2021-05-14 北京思尔成生物技术有限公司 乙型肝炎病毒pgRNA芯片数字PCR绝对定量检测试剂盒及方法
WO2023039586A1 (en) 2021-09-10 2023-03-16 Agilent Technologies, Inc. Guide rnas with chemical modification for prime editing
CN114196556B (zh) * 2021-12-31 2023-08-01 重庆大学 一种高毒力蝗绿僵菌菌株及其构建方法
CN117065052A (zh) * 2022-05-16 2023-11-17 上海行深生物科技有限公司 递送自复制rna分子的方法

Family Cites Families (78)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1003131A (en) * 1910-08-17 1911-09-12 Leonard F Bellingrath Valve operating and indicating mechanism.
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5208036A (en) 1985-01-07 1993-05-04 Syntex (U.S.A.) Inc. N-(ω, (ω-1)-dialkyloxy)- and N-(ω, (ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5466468A (en) 1990-04-03 1995-11-14 Ciba-Geigy Corporation Parenterally administrable liposome formulation comprising synthetic lipids
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5580571A (en) 1991-10-15 1996-12-03 Hostetler; Karl Y. Nucleoside analogues
SE9200951D0 (sv) 1992-03-27 1992-03-27 Kabi Pharmacia Ab Pharmaceutical composition containing a defined lipid system
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
WO1993023569A1 (en) * 1992-05-11 1993-11-25 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting viral replication
EP0702516A4 (en) 1993-06-01 1998-04-22 Life Technologies Inc GENETIC IMMUNIZATION WITH CATIONIC LIPIDS
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
AU698152B2 (en) 1994-08-20 1998-10-22 Gendaq Limited Improvements in or relating to binding proteins for recognition of DNA
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
US6383481B1 (en) 1998-03-30 2002-05-07 Japan Immunoresearch Laboratories Co., Ltd. Method for transplantation of hemopoietic stem cells
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6642008B1 (en) * 1999-11-24 2003-11-04 Oklahoma Medical Research Foundation Assays and therapies for latent viral infection
WO2004027036A2 (en) * 2002-09-19 2004-04-01 Johns Hopkins University School Of Medicine Cancer immunotherapy with a viral antigen-defined, immunomodulator-secreting cell vaccine
US7888121B2 (en) * 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
CN101144817B (zh) * 2003-08-20 2011-10-05 香港神农有限公司 一种eb病毒蛋白酶联免疫吸附诊断试剂盒及其制备方法
EP1678292A4 (en) 2003-09-18 2008-05-07 Univ Emory IMPROVED MVA VACCINES
US20080131962A1 (en) 2006-05-25 2008-06-05 Sangamo Biosciences, Inc. Engineered cleavage half-domains
EP2410043A3 (en) 2004-12-29 2013-01-23 Hadasit Medical Research Services And Development Ltd. Stem cells culture systems
DK2650365T3 (en) * 2005-10-18 2016-12-05 Prec Biosciences RATIONAL MEGANUCLEASES constructed with altered sequence specificity and DNA binding affinity
CA2630218A1 (en) * 2005-11-18 2007-08-30 The Ohio State University Research Foundation Viral gene products and methods for vaccination to prevent viral associated diseases
GB0526211D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Viral vectors
US8481506B2 (en) * 2006-12-05 2013-07-09 Rosetta Genomics, Ltd. Nucleic acids involved in viral infection
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
US20110023144A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in amyotrophyic lateral sclerosis disease
CN101880649B (zh) 2009-05-05 2012-09-05 中国福利会国际和平妇幼保健院 一种干细胞培养方法
US10238734B2 (en) * 2010-03-23 2019-03-26 The Regents Of The University Of California Compositions and methods for self-adjuvanting vaccines against microbes and tumors
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
CN103361328B (zh) * 2012-03-30 2017-08-18 复旦大学 介导整合的hiv‑1前病毒基因敲除的锌指核酸内切酶及其制法和应用
JP6343605B2 (ja) 2012-05-25 2018-06-13 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Rna依存性標的dna修飾およびrna依存性転写調節のための方法および組成物
WO2013188037A2 (en) 2012-06-11 2013-12-19 Agilent Technologies, Inc Method of adaptor-dimer subtraction using a crispr cas6 protein
US20150315576A1 (en) 2012-11-01 2015-11-05 Massachusetts Institute Of Technology Genetic device for the controlled destruction of dna
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
US20140186843A1 (en) 2012-12-12 2014-07-03 Massachusetts Institute Of Technology Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
RU2701850C2 (ru) 2012-12-12 2019-10-01 Те Брод Инститьют, Инк. Конструирование систем, способы и оптимизированные направляющие композиции для манипуляции с последовательностями
ES2576128T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones para la manipulación de secuencias con dominios funcionales
AU2013359199C1 (en) * 2012-12-12 2021-06-17 Massachusetts Institute Of Technology Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
SG11201504519TA (en) 2012-12-12 2015-07-30 Broad Inst Inc Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
EP3553174A1 (en) 2012-12-17 2019-10-16 President and Fellows of Harvard College Rna-guided human genome engineering
AU2014207618A1 (en) 2013-01-16 2015-08-06 Emory University Cas9-nucleic acid complexes and uses related thereto
US10660943B2 (en) 2013-02-07 2020-05-26 The Rockefeller University Sequence specific antimicrobials
US10612043B2 (en) 2013-03-09 2020-04-07 Agilent Technologies, Inc. Methods of in vivo engineering of large sequences using multiple CRISPR/cas selections of recombineering events
CA2905432C (en) 2013-03-14 2021-09-14 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
EP3865586A1 (en) 2013-03-15 2021-08-18 The General Hospital Corporation Increasing specificity for rna-guided genome editing
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
CA2908253C (en) 2013-04-04 2024-01-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
US20160186208A1 (en) 2013-04-16 2016-06-30 Whitehead Institute For Biomedical Research Methods of Mutating, Modifying or Modulating Nucleic Acid in a Cell or Nonhuman Mammal
CA2910489A1 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
US9873907B2 (en) 2013-05-29 2018-01-23 Agilent Technologies, Inc. Method for fragmenting genomic DNA using CAS9
IL303973A (en) 2013-07-09 2023-08-01 Harvard College Multiplexed RNA-guided genome engineering
CN105392885B (zh) 2013-07-19 2020-11-03 赖瑞克斯生物科技公司 用于产生双等位基因敲除的方法和组合物
CA2922428A1 (en) 2013-08-29 2015-03-05 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
WO2015034872A2 (en) 2013-09-05 2015-03-12 Massachusetts Institute Of Technology Tuning microbial populations with programmable nucleases
AU2014346559B2 (en) 2013-11-07 2020-07-09 Editas Medicine,Inc. CRISPR-related methods and compositions with governing gRNAs
CA2932479A1 (en) * 2013-12-12 2015-06-18 The Rockefeller University Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders
AU2014361826A1 (en) 2013-12-12 2016-06-23 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for targeting disorders and diseases using particle delivery components
EP3083958B1 (en) 2013-12-19 2019-04-17 Amyris, Inc. Methods for genomic integration
CN106232823A (zh) 2014-02-18 2016-12-14 杜克大学 灭活病毒复制的组合物及其制备和使用方法
WO2015153889A2 (en) 2014-04-02 2015-10-08 University Of Florida Research Foundation, Incorporated Materials and methods for the treatment of latent viral infection
CN103911376B (zh) * 2014-04-03 2017-02-15 黄行许 CRISPR‑Cas9靶向敲除乙肝病毒cccDNA及其特异性sgRNA
JP2017518075A (ja) 2014-05-30 2017-07-06 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 潜伏性ウイルス感染を処置するための組成物および方法

Also Published As

Publication number Publication date
WO2015184259A1 (en) 2015-12-03
US20150376583A1 (en) 2015-12-31
JP2017518075A (ja) 2017-07-06
US20190032090A1 (en) 2019-01-31
SG11201610028TA (en) 2016-12-29
EP3149170A1 (en) 2017-04-05
CN107002094A (zh) 2017-08-01
JP2017518372A (ja) 2017-07-06
AU2015266767A1 (en) 2016-12-08
US20190010518A1 (en) 2019-01-10
EP3149169A1 (en) 2017-04-05
WO2015184262A1 (en) 2015-12-03
US20160060655A1 (en) 2016-03-03
US9487802B2 (en) 2016-11-08
AU2015266770A1 (en) 2016-12-08
JP2020043862A (ja) 2020-03-26
AU2015266776A1 (en) 2016-12-08
US20170182190A1 (en) 2017-06-29
WO2015184268A1 (en) 2015-12-03
CN106574256A (zh) 2017-04-19
CA2949713A1 (en) 2015-12-03
EP3149171A1 (en) 2017-04-05
US10066241B2 (en) 2018-09-04
US20150368670A1 (en) 2015-12-24
US20190185882A1 (en) 2019-06-20
KR20170005494A (ko) 2017-01-13
CA2949710A1 (en) 2015-12-03

Similar Documents

Publication Publication Date Title
BR112016028023A2 (pt) Composições e métodos de administração de tratamentos para infecções virais latentes
BR112019000107A2 (pt) composições e métodos com base em crispr / cas9 para o tratamento do câncer
BR112019001887A2 (pt) composições e métodos para o tratamento de doença associada a cep290
MX2019011215A (es) Ácidos nucleicos que codifican proteínas asociadas a crispr y usos de los mismos.
MX2015014423A (es) Administración efectiva de genes grandes por vectores aav duales.
BR112016020688A2 (pt) Composições úteis no tratamento da deficiência da ornitina transcarbamilase (otc)
BR112018075479A2 (pt) portadores híbridos para carga de ácido nucleico
CL2016000502A1 (es) Vectores mejorados del factor viii del virus adeno-asociado de menos de 5,0 kb de longitud, que producen un polipéptido del factor viii funcionalmente activo para el tratamiento de la hemofilia a.
BR112018011975A2 (pt) composições úteis no tratamento de atrofia muscular espinhal
PH12017501942A1 (en) Oncolytic adenovirus encoding a b7 protein
BR112015021884A8 (pt) vetores de plasmídeo recombinantes, partícula viral avv ou pluralidade de partículas virais, seus métodos de produção e seus usos, e composição farmacêutica
BR112018008766A2 (pt) variantes, composições e métodos e utilizações do fator viii reduzido cpg para o tratamento de transtornos hemostásicos
MX2023004479A (es) Sistema de vector multiple y sus usos.
BR112015024605A2 (pt) sistemas e métodos para a produção visada de proteína terapêutica dentro de célula alvo
MX2017004039A (es) Fosforamiditas de n-acetilgalactosamina (galnac), conjugados de acido nucleico de las mismas y su uso.
BR112018008783A8 (pt) terapia-alvo de câncer
BR112013030824A8 (pt) Bactéria gram-positiva, ácido necleico recombinante, composição farmacêutica e vetor
CA2863964C (en) Compartmentalized method of nucleic acid delivery and compositions and uses thereof
CL2015003199A1 (es) Peptidos terapeuticos
BR112014025985A2 (pt) composição e métodos para transferência de gene altamente eficaz com o uso de variantes de capsídeo de aav
BR112014012600A2 (pt) sistema de produção de vetor lentiviral escalonável compatível com aplicações farmacêuticas industriais
BR112016027871A2 (pt) composição para melhorar a memória, a função de aprendizado e/ou função cognitiva
AR099837A1 (es) Terapia génica para la retinitis pigmentaria
NZ704275A (en) Aav-mediated gene therapy for rpgr x-linked retinal degeneration
BR112015008612A2 (pt) métodos de tratamento utilizando adenovirus

Legal Events

Date Code Title Description
B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]

Free format text: DE ACORDO COM O ARTIGO 229-C DA LEI NO 10196/2001, QUE MODIFICOU A LEI NO 9279/96, A CONCESSAO DA PATENTE ESTA CONDICIONADA A ANUENCIA PREVIA DA ANVISA. CONSIDERANDO A APROVACAO DOS TERMOS DO PARECER NO 337/PGF/EA/2010, BEM COMO A PORTARIA INTERMINISTERIAL NO 1065 DE 24/05/2012, ENCAMINHA-SE O PRESENTE PEDIDO PARA AS PROVIDENCIAS CABIVEIS.

B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]
B11B Dismissal acc. art. 36, par 1 of ipl - no reply within 90 days to fullfil the necessary requirements