EA202192636A1 - Композиции и способы, содержащие гидовую рнк ttr и полинуклеотид, кодирующий днк-связывающий агент, гидируемый рнк - Google Patents

Композиции и способы, содержащие гидовую рнк ttr и полинуклеотид, кодирующий днк-связывающий агент, гидируемый рнк

Info

Publication number
EA202192636A1
EA202192636A1 EA202192636A EA202192636A EA202192636A1 EA 202192636 A1 EA202192636 A1 EA 202192636A1 EA 202192636 A EA202192636 A EA 202192636A EA 202192636 A EA202192636 A EA 202192636A EA 202192636 A1 EA202192636 A1 EA 202192636A1
Authority
EA
Eurasian Patent Office
Prior art keywords
rna
compositions
ttr
polynucleotoide
gidated
Prior art date
Application number
EA202192636A
Other languages
English (en)
Russian (ru)
Inventor
Юн Чан
Сет С. Александер
Кристи М. Вуд
Арти Махендра Пракаш Канджолия
Сёбу Одате
Джессика Линн Сайцер
Рейнальд Майкл Лескарбо
Уолтер Стрэппс
Original Assignee
Интеллиа Терапьютикс, Инк.
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 Интеллиа Терапьютикс, Инк. filed Critical Интеллиа Терапьютикс, Инк.
Publication of EA202192636A1 publication Critical patent/EA202192636A1/ru

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
EA202192636A 2019-03-28 2020-03-27 Композиции и способы, содержащие гидовую рнк ttr и полинуклеотид, кодирующий днк-связывающий агент, гидируемый рнк EA202192636A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962825637P 2019-03-28 2019-03-28
PCT/US2020/025513 WO2020198697A1 (en) 2019-03-28 2020-03-27 Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent

Publications (1)

Publication Number Publication Date
EA202192636A1 true EA202192636A1 (ru) 2022-03-17

Family

ID=70296147

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202192636A EA202192636A1 (ru) 2019-03-28 2020-03-27 Композиции и способы, содержащие гидовую рнк ttr и полинуклеотид, кодирующий днк-связывающий агент, гидируемый рнк

Country Status (15)

Country Link
US (2) US20230044994A1 (https=)
EP (1) EP3946598A1 (https=)
JP (2) JP7631215B2 (https=)
KR (1) KR20220004648A (https=)
CN (1) CN113874076A (https=)
AU (1) AU2020244887A1 (https=)
BR (1) BR112021019196A2 (https=)
CA (1) CA3134271A1 (https=)
CO (1) CO2021014559A2 (https=)
EA (1) EA202192636A1 (https=)
IL (1) IL286524A (https=)
MX (1) MX2021011565A (https=)
PH (1) PH12021552301A1 (https=)
SG (1) SG11202110434PA (https=)
WO (1) WO2020198697A1 (https=)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3018046A1 (en) * 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
CA3116576A1 (en) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
WO2022115645A1 (en) 2020-11-25 2022-06-02 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids, and related methods of use
WO2022246266A1 (en) * 2021-05-21 2022-11-24 Beam Therapeutics Inc. Base editing of transthyretin gene
WO2022256619A2 (en) * 2021-06-04 2022-12-08 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting transthyretin (ttr) and uses thereof
KR20240038705A (ko) * 2021-06-22 2024-03-25 인텔리아 테라퓨틱스, 인크. 간 유전자의 생체 내 편집 방법
WO2023185697A2 (en) 2022-03-29 2023-10-05 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of transthyretin amyloidosis
CA3256897A1 (en) 2022-05-25 2023-11-30 Akagera Medicines, Inc. Lipid nanoparticles for the administration of nucleic acids and their methods of use
WO2024003805A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Methods and compositions for ttr gene editing and therapy using crispr system
WO2024061296A2 (en) 2022-09-22 2024-03-28 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of hypercholesterolemia and/or cardiovascular disease
EP4627083A1 (en) * 2022-11-30 2025-10-08 Arbor Biotechnologies, Inc. Reverse transcriptase-mediated genetic editing of transthyretin (ttr) and uses thereof
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2026003582A2 (en) * 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
WO2026061971A1 (en) 2024-09-17 2026-03-26 Lonza Ltd Nucleic acid untranslated regions

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
JP3131222B2 (ja) 1991-12-24 2001-01-31 アイシス・ファーマシューティカルス・インコーポレーテッド ギャップを有する２′修飾オリゴヌクレオチド
JPH10500310A (ja) 1994-05-19 1998-01-13 ダコ アクティーゼルスカブ 淋菌及びトラコーマ クラミジアの検出のためのｐｎａ プローブ
US20050244869A1 (en) * 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
US20060051405A1 (en) 2004-07-19 2006-03-09 Protiva Biotherapeutics, Inc. Compositions for the delivery of therapeutic agents and uses thereof
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
DK2931891T3 (da) 2012-12-17 2019-08-19 Harvard College Rna-styret modificering af menneskelige genomer
CN107879960B (zh) 2013-03-08 2021-06-22 诺华股份有限公司 用于传递活性成分的脂质和脂质组合物
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3083556B1 (en) 2013-12-19 2019-12-25 Novartis AG Lipids and lipid compositions for the delivery of active agents
CN106794141B (zh) 2014-07-16 2021-05-28 诺华股份有限公司 将核酸包封在脂质纳米粒主体中的方法
KR20230156800A (ko) 2015-03-03 2023-11-14 더 제너럴 하스피탈 코포레이션 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제
WO2017019867A1 (en) * 2015-07-28 2017-02-02 Danisco Us Inc Genome editing systems and methods of use
CN120842296A (zh) 2015-09-21 2025-10-28 垂林克生物技术有限公司 用于合成5’-加帽rna的组合物和方法
LT3436077T (lt) 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
WO2018007871A1 (en) 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
WO2018067447A1 (en) 2016-10-03 2018-04-12 Itellia Therapeutics, Inc. Improved methods for identifying double strand break sites
WO2019067910A1 (en) * 2017-09-29 2019-04-04 Intellia Therapeutics, Inc. POLYNUCLEOTIDES, COMPOSITIONS AND METHODS FOR GENOMIC EDITION
US20230140670A1 (en) 2017-09-29 2023-05-04 Intellia Therapeutics, Inc. Formulations
EP3688161A1 (en) * 2017-09-29 2020-08-05 Intellia Therapeutics, Inc. Compositions and methods for ttr gene editing and treating attr amyloidosis
AU2019282824C1 (en) * 2018-06-08 2026-04-23 Intellia Therapeutics, Inc. Modified guide RNAS for gene editing
TW202028460A (zh) * 2018-09-28 2020-08-01 美商英特利亞醫療公司 用於乳酸脫氫酶(ldha)基因編輯之組合物及方法

Also Published As

Publication number Publication date
JP2025072483A (ja) 2025-05-09
CO2021014559A2 (es) 2021-11-19
AU2020244887A1 (en) 2021-11-11
WO2020198697A1 (en) 2020-10-01
IL286524A (en) 2021-10-31
KR20220004648A (ko) 2022-01-11
EP3946598A1 (en) 2022-02-09
PH12021552301A1 (en) 2022-07-04
US20240124897A1 (en) 2024-04-18
CA3134271A1 (en) 2020-10-01
JP2022525428A (ja) 2022-05-13
SG11202110434PA (en) 2021-10-28
JP7631215B2 (ja) 2025-02-18
MX2021011565A (es) 2021-11-12
US20230044994A1 (en) 2023-02-09
CN113874076A (zh) 2021-12-31
BR112021019196A2 (pt) 2022-01-18

Similar Documents

Publication Publication Date Title
EA202192636A1 (ru) Композиции и способы, содержащие гидовую рнк ttr и полинуклеотид, кодирующий днк-связывающий агент, гидируемый рнк
MX2025014378A (es) Composiciones utiles para la edicion del gen ttr y el tratamiento de la amiloidosis attr
WO2020198509A3 (en) Modified oligonucleotides with increased stability
MX2021003187A (es) Inhibición de la peptidasa específica de ubiquitina 9x.
MX2018014152A (es) Composiciones y métodos para tratar la enfermedad de huntington.
WO2016106401A3 (en) Rna agents for p21 gene modulation
EA202092086A1 (ru) Ингибиторы аргиназы
SG142330A1 (en) Anti-cd38 human antibodies and uses therefor
EP4431607A3 (en) High-throughput precision genome editing
MX2021010025A (es) Tratamiento de lesión renal.
WO2007081680A3 (en) Microrna expression abnormalities in pancreatic endocrine and acinar tumors
DE602004015811D1 (de) Verfahren zur inhibierung der proliferation von tumorzellen, bei dem platinkomplexe eingesetzt werden
BR112022008821A2 (pt) Ligantes de pseudoquinase tyk2
ATE557088T1 (de) Pankreasinsel-mikro-rna und verfahren zu ihrer unterdrückung
PH12022552009A1 (en) Compositions and methods for kallikrein ( klkb1) gene editing
BR112021015616A2 (pt) Ligantes de pseudoquinase tyk2
MX2021014334A (es) Agentes afines a aav9.
MX2022015966A (es) Anticuerpos y metodos para tratar enfermedades asociadas a claudina.
JOP20220315A1 (ar) جزيئات ربط لعلاج السرطان
MX2021005980A (es) Adenovirus y metodos para usar adenovirus.
WO2023086938A3 (en) Type v nucleases
MX2019007110A (es) Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y uso de estos.
PH12022550277A1 (en) Biopharmacuetical compositions and related methods
MX2025012761A (es) Moleculas y metodos de uso de estas
DE602005015329D1 (de) Rutheniumkomplexe zur krebsbehandlung