CA2999922A1 - Delivery methods and compositions - Google Patents
Delivery methods and compositions Download PDFInfo
- Publication number
- CA2999922A1 CA2999922A1 CA2999922A CA2999922A CA2999922A1 CA 2999922 A1 CA2999922 A1 CA 2999922A1 CA 2999922 A CA2999922 A CA 2999922A CA 2999922 A CA2999922 A CA 2999922A CA 2999922 A1 CA2999922 A1 CA 2999922A1
- Authority
- CA
- Canada
- Prior art keywords
- kit
- nucleic acid
- tissue
- target
- cas9
- 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
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- 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
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- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/20—Antivirals for DNA viruses
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- 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)
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- A—HUMAN NECESSITIES
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- 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
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- 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
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- A—HUMAN NECESSITIES
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- 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
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- 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)
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- Veterinary Medicine (AREA)
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- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- General Chemical & Material Sciences (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
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- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Wood Science & Technology (AREA)
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- Communicable Diseases (AREA)
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- 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)
- Heart & Thoracic Surgery (AREA)
- Hematology (AREA)
- Anesthesiology (AREA)
- Medical Informatics (AREA)
- Apparatus Associated With Microorganisms And Enzymes (AREA)
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| US201562234340P | 2015-09-29 | 2015-09-29 | |
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| PCT/US2016/053960 WO2017058793A1 (en) | 2015-09-29 | 2016-09-27 | Delivery methods and compositions |
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| EP2734621B1 (en) | 2011-07-22 | 2019-09-04 | President and Fellows of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| 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 |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| SG10202104041PA (en) | 2015-10-23 | 2021-06-29 | Harvard College | Nucleobase editors and uses thereof |
| US12403305B2 (en) | 2016-06-27 | 2025-09-02 | Galvanize Therapeutics, Inc. | Immunostimulation in the treatment of viral infection |
| CN114098949B (zh) | 2016-06-27 | 2023-12-19 | 盖能适治疗股份有限公司 | 发生器和具有电极的导管 |
| KR102827276B1 (ko) | 2016-08-03 | 2025-07-01 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| WO2018031683A1 (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 |
| CN110214180A (zh) | 2016-10-14 | 2019-09-06 | 哈佛大学的校长及成员们 | 核碱基编辑器的aav递送 |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| 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 |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| GB2575930A (en) | 2017-03-23 | 2020-01-29 | 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 |
| US11732274B2 (en) | 2017-07-28 | 2023-08-22 | President And Fellows Of Harvard College | Methods and compositions for evolving base 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 |
| CA3082251A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | Uses of adenosine base editors |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| CN111683613B (zh) | 2017-12-26 | 2024-05-28 | 盖能适治疗股份有限公司 | 用于各种应用的能量输送的优化 |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors 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 |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| WO2020102532A1 (en) * | 2018-11-15 | 2020-05-22 | 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 |
| DE112020001306T5 (de) | 2019-03-19 | 2022-01-27 | Massachusetts Institute Of Technology | Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen |
| CN114072527B (zh) * | 2019-03-25 | 2025-03-04 | 香港中文大学 | 确定循环核酸的线性和环状形式 |
| IL285920B2 (en) * | 2019-04-08 | 2025-03-01 | Velanidi Tech 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 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| AU2021267940A1 (en) | 2020-05-08 | 2022-12-08 | President And Fellows Of Harvard College | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| AU2021281307A1 (en) * | 2020-05-29 | 2022-12-15 | 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 |
| 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 |
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| US6713291B2 (en) * | 1999-01-28 | 2004-03-30 | Alan D. King | Electrodes coated with treating agent and uses thereof |
| JP2004535813A (ja) * | 2001-07-17 | 2004-12-02 | ミルナー，アン，ジョセフィーヌ | 遺伝子発現のサイレンシング |
| AU2011213242B2 (en) * | 2010-02-08 | 2015-01-29 | Sangamo Therapeutics, Inc. | Engineered cleavage half-domains |
| US10537725B2 (en) * | 2010-03-05 | 2020-01-21 | Cornell University | Ultrasound-assisted convection enhanced delivery of compounds in vivo with a transducer cannula assembly |
| WO2015089465A1 (en) * | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders |
| 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 |
| US20160060655A1 (en) * | 2014-05-30 | 2016-03-03 | The Board Of Trustees Of The Leland Stanford Junior University | Compositions and methods to treat latent viral infections |
| JP2018516984A (ja) * | 2015-05-29 | 2018-06-28 | アジェノビア コーポレーション | 細胞を標的にしたｈｐｖ処置のための組成物および方法 |
| EP3420077A4 (en) * | 2016-02-25 | 2019-12-25 | Agenovir Corporation | VIRAL AND ONCOVIRAL NUCLEASE TREATMENT |
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- 2016-09-27 US US15/277,595 patent/US20170087224A1/en not_active Abandoned
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| EP3355954A1 (en) | 2018-08-08 |
| HK1258900A1 (zh) | 2019-11-22 |
| US20170087224A1 (en) | 2017-03-30 |
| WO2017058793A1 (en) | 2017-04-06 |
| JP2018532403A (ja) | 2018-11-08 |
| EP3355954A4 (en) | 2020-01-08 |
| CN108601883A (zh) | 2018-09-28 |
| AU2016332704A1 (en) | 2018-04-19 |
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