JP2024516376A - Dna依存性プロテインキナーゼの阻害剤ならびにその組成物及び使用 - Google Patents

Dna依存性プロテインキナーゼの阻害剤ならびにその組成物及び使用 Download PDF

Info

Publication number
JP2024516376A
JP2024516376A JP2023563189A JP2023563189A JP2024516376A JP 2024516376 A JP2024516376 A JP 2024516376A JP 2023563189 A JP2023563189 A JP 2023563189A JP 2023563189 A JP2023563189 A JP 2023563189A JP 2024516376 A JP2024516376 A JP 2024516376A
Authority
JP
Japan
Prior art keywords
dna
composition
cells
cell
pki
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.)
Pending
Application number
JP2023563189A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024516376A5 (enExample
Inventor
マエタニ,マイカ
ジェニー シー,シン
フォーゲット,アンソニー
プロデウス,アーロン
ヤジンスキー,ステファニー
パーマー,ルビナ,ギアレ
Original Assignee
インテリア セラピューティクス,インコーポレーテッド
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 インテリア セラピューティクス,インコーポレーテッド filed Critical インテリア セラピューティクス,インコーポレーテッド
Publication of JP2024516376A publication Critical patent/JP2024516376A/ja
Publication of JP2024516376A5 publication Critical patent/JP2024516376A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D473/00—Heterocyclic compounds containing purine ring systems
    • C07D473/26—Heterocyclic compounds containing purine ring systems with an oxygen, sulphur, or nitrogen atom directly attached in position 2 or 6, but not in both
    • C07D473/32—Nitrogen atom
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/437—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A61K31/52—Purines, e.g. adenine
    • A61K31/522—Purines, e.g. adenine having oxo groups directly attached to the heterocyclic ring, e.g. hypoxanthine, guanine, acyclovir
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/32—T-cell receptors [TCR]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4242—Transcription factors, e.g. SOX or c-MYC
    • A61K40/4243—Wilms tumor 1 [WT1]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/04—Ortho-condensed systems
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0635—B lymphocytes
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0636—T lymphocytes
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/067—Hepatocytes
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • C12N2510/00—Genetically modified cells

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Mycology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Medicinal Preparation (AREA)
JP2023563189A 2021-04-17 2022-04-15 Dna依存性プロテインキナーゼの阻害剤ならびにその組成物及び使用 Pending JP2024516376A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163176225P 2021-04-17 2021-04-17
US63/176,225 2021-04-17
PCT/US2022/025075 WO2022221696A1 (en) 2021-04-17 2022-04-15 Inhibitors of dna-dependent protein kinase and compositions and uses thereof

Publications (2)

Publication Number Publication Date
JP2024516376A true JP2024516376A (ja) 2024-04-15
JP2024516376A5 JP2024516376A5 (enExample) 2025-04-22

Family

ID=81581217

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023563189A Pending JP2024516376A (ja) 2021-04-17 2022-04-15 Dna依存性プロテインキナーゼの阻害剤ならびにその組成物及び使用

Country Status (15)

Country Link
US (1) US20240300977A1 (enExample)
EP (1) EP4323360A1 (enExample)
JP (1) JP2024516376A (enExample)
KR (1) KR20240017791A (enExample)
CN (1) CN117940426A (enExample)
AU (1) AU2022258733A1 (enExample)
BR (1) BR112023021429A2 (enExample)
CA (1) CA3216875A1 (enExample)
CL (1) CL2023003079A1 (enExample)
CO (1) CO2023015478A2 (enExample)
CR (1) CR20230535A (enExample)
IL (1) IL307740A (enExample)
MX (1) MX2023012236A (enExample)
TW (1) TW202309034A (enExample)
WO (1) WO2022221696A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL299612A (en) 2020-07-02 2023-03-01 Incyte Corp Tricyclic urea compounds as JAK2 V617F inhibitors
WO2023220418A2 (en) * 2022-05-13 2023-11-16 The Board Of Trustees Of The Leland Stanford Junior University Enhancing gene targeting efficiency in human cells with dna-pk inhibitor treatment
WO2024191996A1 (en) * 2023-03-13 2024-09-19 Incyte Corporation Bicyclic ureas as kinase inhibitors
TW202515994A (zh) 2023-08-14 2025-04-16 美商英特利亞醫療公司 用於對cd70進行基因修飾之組合物及方法
WO2025049481A1 (en) * 2023-08-28 2025-03-06 Intellia Therapeutics, Inc. Methods of editing an hla-a gene in vitro
WO2025049432A1 (en) 2023-08-28 2025-03-06 Intellia Therapeutics, Inc. Methods for editing hla-a in cells pre-screened for the absence of one or both alleles of hla-h*01
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
WO2025137301A1 (en) 2023-12-20 2025-06-26 Intellia Therapeutics, Inc. Methods for rapid engineering of cells

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018114999A1 (en) * 2016-12-20 2018-06-28 Astrazeneca Ab Amino-triazolopyridine compounds and their use in treating cancer
WO2019238929A1 (en) * 2018-06-15 2019-12-19 Astrazeneca Ab Purinone compounds and their use in treating cancer
US20210008161A1 (en) * 2019-06-17 2021-01-14 Crispr Therapeutics Ag Methods and compositions for improved homology directed repair
WO2021041546A1 (en) * 2019-08-27 2021-03-04 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of disorders associated with repetitive dna
JP2023504021A (ja) * 2019-11-25 2023-02-01 メッドシャイン ディスカバリー インコーポレイテッド Dna-pk阻害剤としてのピリミドイミダゾル系化合物
JP2023509011A (ja) * 2019-12-31 2023-03-06 成都百裕制薬股▲ふん▼有限公司 プリン誘導体および医薬におけるその使用
JP2023542548A (ja) * 2020-09-24 2023-10-10 オークランド ユニサービシズ リミテッド 新規アミノピリジン及びその癌治療への使用

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1021549A2 (en) 1997-09-19 2000-07-26 Sequitur, Inc. SENSE mRNA THERAPY
GB0119865D0 (en) 2001-08-14 2001-10-10 Cancer Res Campaign Tech DNA-PK inhibitors
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
WO2011076807A2 (en) 2009-12-23 2011-06-30 Novartis Ag Lipids, lipid compositions, and methods of using them
EP2526199A4 (en) 2010-01-22 2013-08-07 Scripps Research Inst PROCESS FOR PRODUCING ZINC FINGERUCLEASES WITH CHANGED EFFECT
CN103668470B (zh) 2012-09-12 2015-07-29 上海斯丹赛生物技术有限公司 一种dna文库及构建转录激活子样效应因子核酸酶质粒的方法
PT2931898E (pt) 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
IL308158A (en) 2012-12-17 2023-12-01 Harvard College Rna-guided human genome engineering
ES2887254T3 (es) 2013-03-08 2021-12-22 Novartis Ag Lípidos y composiciones lipídicas para el suministro de agentes activos
RS62649B1 (sr) 2013-03-12 2021-12-31 Vertex Pharma Inhibitori dnk-pk
DE102013008118A1 (de) 2013-05-11 2014-11-13 Merck Patent Gmbh Arylchinazoline
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
US10059655B2 (en) 2013-12-19 2018-08-28 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
HRP20220156T1 (hr) 2015-09-17 2022-04-15 Modernatx, Inc. Spojevi i pripravci za unutarstaničnu isporuku terapeutskih sredstava
ES3030658T3 (en) 2016-03-30 2025-07-01 Intellia Therapeutics Inc Lipid nanoparticle formulations for crispr/cas components
WO2018073393A2 (en) 2016-10-19 2018-04-26 Cellectis Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy
EP3688162B1 (en) 2017-09-29 2024-03-06 Intellia Therapeutics, Inc. Formulations
WO2019147805A2 (en) 2018-01-26 2019-08-01 The Board Of Trustees Of The Leland Stanford Junior University Regulatory t cells targeted with chimeric antigen receptors
MX2021003455A (es) 2018-10-02 2021-09-21 Intellia Therapeutics Inc Lípidos con amina ionizables.
CN119285480A (zh) 2018-12-05 2025-01-10 英特利亚治疗股份有限公司 改性的胺脂质
WO2020219876A1 (en) 2019-04-25 2020-10-29 Intellia Therapeutics, Inc. Ionizable amine lipids and lipid nanoparticles

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018114999A1 (en) * 2016-12-20 2018-06-28 Astrazeneca Ab Amino-triazolopyridine compounds and their use in treating cancer
WO2019238929A1 (en) * 2018-06-15 2019-12-19 Astrazeneca Ab Purinone compounds and their use in treating cancer
US20210008161A1 (en) * 2019-06-17 2021-01-14 Crispr Therapeutics Ag Methods and compositions for improved homology directed repair
WO2021041546A1 (en) * 2019-08-27 2021-03-04 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of disorders associated with repetitive dna
JP2023504021A (ja) * 2019-11-25 2023-02-01 メッドシャイン ディスカバリー インコーポレイテッド Dna-pk阻害剤としてのピリミドイミダゾル系化合物
JP2023509011A (ja) * 2019-12-31 2023-03-06 成都百裕制薬股▲ふん▼有限公司 プリン誘導体および医薬におけるその使用
JP2023542548A (ja) * 2020-09-24 2023-10-10 オークランド ユニサービシズ リミテッド 新規アミノピリジン及びその癌治療への使用

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
FREDERICK W. GOLDBERG ET AL.: ""The Discovery of 7-Methyl-2-[(7-methyl[1,2,4]triazolo[1,5-a]pyridin-6-yl)amino]-9-(tetrahydro-2H-p", JOURNAL OF MEDICINAL CHEMISTRY, vol. 63, no. 7, JPN6026023751, 18 December 2019 (2019-12-18), pages 3461 - 3471, ISSN: 0005880815 *
MIN QIU ET AL.: ""Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific i", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, vol. 118, no. 10, JPN6026023753, 1 March 2021 (2021-03-01), pages 2020401118, ISSN: 0005880817 *
NURIA BOSCH ET AL.: ""Enhancing CRISPR deletion via pharmacological delay of DNA-PK"", BIORXIV, JPN6026023752, 27 May 2020 (2020-05-27), ISSN: 0005880816 *

Also Published As

Publication number Publication date
KR20240017791A (ko) 2024-02-08
AU2022258733A1 (en) 2023-11-30
US20240300977A1 (en) 2024-09-12
CN117940426A (zh) 2024-04-26
CR20230535A (es) 2024-02-16
CA3216875A1 (en) 2022-10-20
EP4323360A1 (en) 2024-02-21
BR112023021429A2 (pt) 2024-01-23
MX2023012236A (es) 2024-01-23
IL307740A (en) 2023-12-01
WO2022221696A1 (en) 2022-10-20
TW202309034A (zh) 2023-03-01
CL2023003079A1 (es) 2024-04-12
CO2023015478A2 (es) 2025-07-07

Similar Documents

Publication Publication Date Title
US20230183753A1 (en) Methods of in Vitro Cell Delivery
US20240300977A1 (en) Inhibitors of dna-dependent protein kinase and compositions and uses thereof
US20250090471A1 (en) Lipid nanoparticle compositions
US20240200106A1 (en) Lipid nanoparticles compositions
US20260132376A1 (en) Genetically-modified cells comprising a modified human t cell receptor alpha constant region gene
AU2021410751A9 (en) Compositions and methods for genetically modifying ciita in a cell
US20250276090A1 (en) Methods and Compositions for Genetically Modifying a Cell
CN117835968A (zh) 脂质纳米颗粒组合物
CN117479926A (zh) 脂质纳米颗粒组合物
WO2025049481A1 (en) Methods of editing an hla-a gene in vitro
HK40085839A (en) Methods of in vitro cell delivery

Legal Events

Date Code Title Description
RD01 Notification of change of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7426

Effective date: 20240222

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20240222

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250411

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20250411

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20260609