AU2016332704A1 - Delivery methods and compositions - Google Patents

Delivery methods and compositions Download PDF

Info

Publication number
AU2016332704A1
AU2016332704A1 AU2016332704A AU2016332704A AU2016332704A1 AU 2016332704 A1 AU2016332704 A1 AU 2016332704A1 AU 2016332704 A AU2016332704 A AU 2016332704A AU 2016332704 A AU2016332704 A AU 2016332704A AU 2016332704 A1 AU2016332704 A1 AU 2016332704A1
Authority
AU
Australia
Prior art keywords
pct
kit
cas9
nucleic acid
tissue
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.)
Abandoned
Application number
AU2016332704A
Other languages
English (en)
Inventor
Stephen R. Quake
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Agenovir Corp
Original Assignee
Agenovir Corp
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 Agenovir Corp filed Critical Agenovir Corp
Publication of AU2016332704A1 publication Critical patent/AU2016332704A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0002—Galenical forms characterised by the drug release technique; Application systems commanded by energy
    • A61K9/0009—Galenical forms characterised by the drug release technique; Application systems commanded by energy involving or responsive to electricity, magnetism or acoustic waves; Galenical aspects of sonophoresis, iontophoresis, electroporation or electroosmosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61N—ELECTROTHERAPY; MAGNETOTHERAPY; RADIATION THERAPY; ULTRASOUND THERAPY
    • A61N1/00—Electrotherapy; Circuits therefor
    • A61N1/18—Applying electric currents by contact electrodes
    • A61N1/32—Applying electric currents by contact electrodes alternating or intermittent currents
    • A61N1/327—Applying electric currents by contact electrodes alternating or intermittent currents for enhancing the absorption properties of tissue, e.g. by electroporation
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M37/00—Other apparatus for introducing media into the body; Percutany, i.e. introducing medicines into the body by diffusion through the skin
    • A61M2037/0007—Other apparatus for introducing media into the body; Percutany, i.e. introducing medicines into the body by diffusion through the skin having means for enhancing the permeation of substances through the epidermis, e.g. using suction or depression, electric or magnetic fields, sound waves or chemical agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M37/00—Other apparatus for introducing media into the body; Percutany, i.e. introducing medicines into the body by diffusion through the skin
    • A61M37/0015—Other apparatus for introducing media into the body; Percutany, i.e. introducing medicines into the body by diffusion through the skin by using microneedles
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M37/00—Other apparatus for introducing media into the body; Percutany, i.e. introducing medicines into the body by diffusion through the skin
    • A61M37/0092—Other apparatus for introducing media into the body; Percutany, i.e. introducing medicines into the body by diffusion through the skin using ultrasonic, sonic or infrasonic vibrations, e.g. phonophoresis

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Radiology & Medical Imaging (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Dermatology (AREA)
  • Medical Informatics (AREA)
  • Anesthesiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hematology (AREA)
  • Medicinal Preparation (AREA)
AU2016332704A 2015-09-29 2016-09-27 Delivery methods and compositions Abandoned AU2016332704A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562234340P 2015-09-29 2015-09-29
US62/234,340 2015-09-29
PCT/US2016/053960 WO2017058793A1 (en) 2015-09-29 2016-09-27 Delivery methods and compositions

Publications (1)

Publication Number Publication Date
AU2016332704A1 true AU2016332704A1 (en) 2018-04-19

Family

ID=58408949

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2016332704A Abandoned AU2016332704A1 (en) 2015-09-29 2016-09-27 Delivery methods and compositions

Country Status (8)

Country Link
US (1) US20170087224A1 (https=)
EP (1) EP3355954A4 (https=)
JP (1) JP2018532403A (https=)
CN (1) CN108601883A (https=)
AU (1) AU2016332704A1 (https=)
CA (1) CA2999922A1 (https=)
HK (1) HK1258900A1 (https=)
WO (1) WO2017058793A1 (https=)

Families Citing this family (44)

* 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
US20150044192A1 (en) 2013-08-09 2015-02-12 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
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
JP7109784B2 (ja) 2015-10-23 2022-08-01 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 遺伝子編集のための進化したＣａｓ９蛋白質
JP7287888B2 (ja) 2016-06-27 2023-06-06 ギャラリー，インコーポレイテッド ジェネレータ、電極付きのカテーテル、及び肺通路の治療方法
US12403305B2 (en) 2016-06-27 2025-09-02 Galvanize Therapeutics, Inc. Immunostimulation in the treatment of viral infection
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510038A (ja) 2017-03-09 2020-04-02 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 がんワクチン
JP2020510439A (ja) 2017-03-10 2020-04-09 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 シトシンからグアニンへの塩基編集因子
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
DK4218641T3 (da) 2017-12-26 2025-08-18 Galvanize Therapeutics Inc Optimering af energilevering til forskellige anvendelser
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020015279A1 (zh) * 2018-07-17 2020-01-23 杭州观梓健康科技有限公司 一种在干细胞中进行基因定向敲入的方法
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US20220001027A1 (en) * 2018-11-15 2022-01-06 President And Fellows Of Harvard College Genetically Engineered Skin Cells for the Systemic In Vivo Treatment of Deficient Enzymes, Factors or Proteins
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
JP7657726B2 (ja) 2019-03-19 2025-04-07 ザ ブロード インスティテュート，インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
WO2020192680A1 (en) * 2019-03-25 2020-10-01 The Chinese University Of Hong Kong Determining linear and circular forms of circulating nucleic acids
US11879892B2 (en) * 2019-04-08 2024-01-23 Velanidi Technologies LLC System, apparatus and method for sensing, detecting and effecting in a micro-to-nano environment
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
JP7551648B2 (ja) 2019-04-18 2024-09-17 ガルヴァナイズ セラピューティクス，インコーポレイテッド 異常組織を治療するための器械、及びシステム、
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
WO2021243270A1 (en) * 2020-05-29 2021-12-02 Georgia Tech Research Corporation Devices, systems, and methods for facilitating tissue delivery of drug
CN116829691A (zh) * 2020-08-04 2023-09-29 盖能适治疗股份有限公司 分子到体内细胞的脉冲电场转移
CA3188372A1 (en) * 2020-08-04 2022-02-10 Galvanize Therapeutics, Inc. Pulsed electric field transfer of molecules to cells while in the body
CN112807273B (zh) * 2021-02-23 2022-06-17 浙江大学 一种治疗炎症性皮肤病的基因编辑微针及其应用
JP2023094496A (ja) * 2021-12-23 2023-07-05 委子 陌間 抗菌・ウイルス治療薬の製造方法

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6713291B2 (en) * 1999-01-28 2004-03-30 Alan D. King Electrodes coated with treating agent and uses thereof
CA2452653A1 (en) * 2001-07-17 2003-01-30 Anne Josephine Milner Silencing of gene expression by sirna
EP3594333B1 (en) * 2010-02-08 2023-11-01 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
WO2011109735A2 (en) * 2010-03-05 2011-09-09 Cornell University Ultrasound-assisted convection enhanced delivery of compounds in vivo with a transducer cannula assembly
BR112016013207A2 (pt) * 2013-12-12 2017-09-26 Massachusetts Inst Technology administração, uso e aplicações terapêuticas dos sistemas crispr-cas e composições para o hbv e distúrbios e doenças virais
JP2017506893A (ja) * 2014-02-18 2017-03-16 デューク ユニバーシティ ウイルス複製不活化組成物並びにその製造方法及び使用
EP3149171A1 (en) * 2014-05-30 2017-04-05 The Board of Trustees of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
US20160346360A1 (en) * 2015-05-29 2016-12-01 Agenovir Corporation Compositions and methods for cell targeted hpv treatment
CA3015353A1 (en) * 2016-02-25 2017-08-31 Agenovir Corporation Viral and oncoviral nuclease treatment

Also Published As

Publication number Publication date
JP2018532403A (ja) 2018-11-08
HK1258900A1 (zh) 2019-11-22
CA2999922A1 (en) 2017-04-06
US20170087224A1 (en) 2017-03-30
WO2017058793A1 (en) 2017-04-06
CN108601883A (zh) 2018-09-28
EP3355954A1 (en) 2018-08-08
EP3355954A4 (en) 2020-01-08

Similar Documents

Publication Publication Date Title
US20170087224A1 (en) Delivery methods and compositions
Parhi et al. Enhancement of skin permeability with thermal ablation techniques: concept to commercial products
Vaseem et al. Transdermal drug delivery systems: a focused review of the physical methods of permeation enhancement
US20170246261A1 (en) Antiviral treatment with low immunogenicity
Escobar‐Chávez et al. Electroporation as an efficient physical enhancer for skin drug delivery
Bolhassani et al. Electroporation–Advantages and drawbacks for delivery of drug, gene and vaccine
Escoffre et al. Gene transfer: how can the biological barriers be overcome?
A Charoo et al. Electroporation: an avenue for transdermal drug delivery
Gao et al. How physical techniques improve the transdermal permeation of therapeutics: A review
Zhang et al. Depth-targeted efficient gene delivery and expression in the skin by pulsed electric fields: an approach to gene therapy of skin aging and other diseases
Cervia et al. Current progress in electrotransfection as a nonviral method for gene delivery
JP4664450B2 (ja) 多細胞化真核生物細胞に核酸を導入する改良法およびその組合せ
Alsaggar et al. Physical methods for gene transfer
JP2017518075A (ja) 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
Hui Overview of drug delivery and alternative methods to electroporation
Li et al. Single micelle vectors based on lipid/block copolymer compositions as mRNA formulations for efficient cancer immunogene therapy
JP2002517519A (ja) エレクトロポレーション用製剤
Jakutavičiūtė et al. Physical methods for drug and gene delivery through the cell plasma membrane
Aldawsari et al. Progress in topical siRNA delivery approaches for skin disorders
Elsabahy et al. Needle-free gene delivery through the skin: an overview of recent strategies
Wu et al. Microneedle‐enabled breakthroughs in nucleic acid therapeutics
Atkinson et al. Targets in dermal and transdermal delivery and classification of penetration enhancement methods
Esmaeili et al. Gene delivery
Preat et al. Topical delivery of nucleic acids in the skin
Jain Transdermal drug delivery

Legal Events

Date Code Title Description
MK1 Application lapsed section 142(2)(a) - no request for examination in relevant period