PL3492593T3 - Regulacja ekspresji genów, w której pośredniczą nukleazy - Google Patents

Regulacja ekspresji genów, w której pośredniczą nukleazy

Info

Publication number
PL3492593T3
PL3492593T3 PL18214711T PL18214711T PL3492593T3 PL 3492593 T3 PL3492593 T3 PL 3492593T3 PL 18214711 T PL18214711 T PL 18214711T PL 18214711 T PL18214711 T PL 18214711T PL 3492593 T3 PL3492593 T3 PL 3492593T3
Authority
PL
Poland
Prior art keywords
nuclease
gene expression
mediated regulation
mediated
regulation
Prior art date
Application number
PL18214711T
Other languages
English (en)
Inventor
Stuart H. Orkin
Andreas Reik
Fyodor Urnov
Original Assignee
Children's Medical Center Corporation
Sangamo Therapeutics, Inc.
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 Children's Medical Center Corporation, Sangamo Therapeutics, Inc. filed Critical Children's Medical Center Corporation
Publication of PL3492593T3 publication Critical patent/PL3492593T3/pl

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
    • 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
    • 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
    • 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
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating 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
    • C12N15/11—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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • 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
    • 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]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Developmental Biology & Embryology (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
PL18214711T 2013-11-13 2014-11-13 Regulacja ekspresji genów, w której pośredniczą nukleazy PL3492593T3 (pl)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361903823P 2013-11-13 2013-11-13
US201462042075P 2014-08-26 2014-08-26
EP14861930.7A EP3068881B1 (en) 2013-11-13 2014-11-13 Nuclease-mediated regulation of gene expression
PCT/US2014/065473 WO2015073683A2 (en) 2013-11-13 2014-11-13 Nuclease-mediated regulation of gene expression
EP18214711.6A EP3492593B1 (en) 2013-11-13 2014-11-13 Nuclease-mediated regulation of gene expression

Publications (1)

Publication Number Publication Date
PL3492593T3 true PL3492593T3 (pl) 2022-04-19

Family

ID=53043976

Family Applications (2)

Application Number Title Priority Date Filing Date
PL18214711T PL3492593T3 (pl) 2013-11-13 2014-11-13 Regulacja ekspresji genów, w której pośredniczą nukleazy
PL14861930T PL3068881T3 (pl) 2013-11-13 2014-11-13 Regulacja ekspresji genów, w której pośredniczą nukleazy

Family Applications After (1)

Application Number Title Priority Date Filing Date
PL14861930T PL3068881T3 (pl) 2013-11-13 2014-11-13 Regulacja ekspresji genów, w której pośredniczą nukleazy

Country Status (13)

Country Link
US (2) US11021696B2 (pl)
EP (3) EP3068881B1 (pl)
CY (1) CY1121630T1 (pl)
DK (2) DK3492593T3 (pl)
ES (2) ES2721936T3 (pl)
HR (2) HRP20190825T1 (pl)
HU (2) HUE056436T2 (pl)
LT (2) LT3492593T (pl)
PL (2) PL3492593T3 (pl)
PT (2) PT3492593T (pl)
RS (2) RS58671B1 (pl)
SI (2) SI3492593T1 (pl)
WO (1) WO2015073683A2 (pl)

Families Citing this family (77)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
ES2708948T3 (es) 2012-11-27 2019-04-12 Childrens Medical Ct Corp Elementos reguladores distales de bcl11a como dianas para la reinducción de la hemoglobina fetal
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
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
CN111218447B (zh) 2013-11-07 2024-10-11 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
ES2721936T3 (es) * 2013-11-13 2019-08-06 Childrens Medical Center Regulación de la expresión genética mediada por las nucleasas
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
CN106794260B (zh) 2014-04-25 2021-02-23 儿童医疗中心有限公司 治疗血红蛋白病的组合物和方法
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3763814A1 (en) 2015-05-08 2021-01-13 The Children's Medical Center Corporation Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction
SG10202112057QA (en) * 2015-05-12 2021-12-30 Sangamo Therapeutics Inc Nuclease-mediated regulation of gene expression
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
WO2017093969A1 (en) 2015-12-04 2017-06-08 Novartis Ag Compositions and methods for immunooncology
US11352631B2 (en) 2015-12-18 2022-06-07 Sangamo Therapeutics, Inc. Targeted disruption of the T cell receptor
AU2016374253B2 (en) 2015-12-18 2021-10-21 Sangamo Therapeutics, Inc. Targeted disruption of the MHC cell receptor
MX2018007987A (es) * 2015-12-28 2019-01-10 Novartis Ag Composiciones y metodos para el tratamiento de hemoglobinopatias.
WO2017123609A1 (en) * 2016-01-12 2017-07-20 The Regents Of The University Of California Compositions and methods for enhanced genome editing
WO2017143071A1 (en) * 2016-02-18 2017-08-24 The Regents Of The University Of California Methods and compositions for gene editing in stem cells
WO2018005873A1 (en) * 2016-06-29 2018-01-04 The Broad Institute Inc. Crispr-cas systems having destabilization domain
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
IL264792B2 (en) 2016-08-24 2023-10-01 Sangamo Therapeutics Inc Engineered target-specific zinc-finger nucleases
WO2018039440A1 (en) * 2016-08-24 2018-03-01 Sangamo Therapeutics, Inc. Regulation of gene expression using engineered nucleases
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
JP7256739B2 (ja) * 2016-09-07 2023-04-12 サンガモ セラピューティクス， インコーポレイテッド 肝臓遺伝子のモジュレーション
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
WO2018073393A2 (en) 2016-10-19 2018-04-26 Cellectis Tal-effector nuclease (talen) -modified allogenic cells suitable for 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
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
KR20190130608A (ko) 2017-03-22 2019-11-22 노파르티스 아게 면역종양학을 위한 조성물 및 방법
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
SG11201908844YA (en) 2017-03-29 2019-10-30 Bluebird Bio Inc Vectors and compositions for treating hemoglobinopathies
US11261441B2 (en) 2017-03-29 2022-03-01 Bluebird Bio, Inc. Vectors and compositions for treating hemoglobinopathies
JP2020518245A (ja) 2017-05-03 2020-06-25 サンガモ セラピューティクス， インコーポレイテッド 嚢胞性線維症膜貫通コンダクタンス制御因子（ｃｆｔｒ）遺伝子の改変のための方法および組成物
EP3622070A2 (en) * 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
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
US11788087B2 (en) 2017-05-25 2023-10-17 The Children's Medical Center Corporation BCL11A guide delivery
US11512287B2 (en) 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors
WO2018237291A2 (en) * 2017-06-22 2018-12-27 The Children's Medical Center Corporation CENTERS OF ERYTHROID DIFFERENTIATION SIGNALING
US20200140896A1 (en) 2017-06-30 2020-05-07 Novartis Ag Methods for the treatment of disease with gene editing systems
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
JP2020530997A (ja) 2017-08-08 2020-11-05 サンガモ セラピューティクス， インコーポレイテッド キメラ抗原受容体媒介性細胞標的化
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
MA50849A (fr) 2017-10-26 2020-09-02 Vertex Pharma Substances et procédés pour le traitement d'hémoglobinopathies
CN109722414A (zh) * 2017-10-27 2019-05-07 博雅辑因（北京）生物科技有限公司 一种高效制备成熟红细胞的方法以及用于制备成熟红细胞的培养基
WO2019094725A2 (en) * 2017-11-09 2019-05-16 Sangamo Therapeutics, Inc. Genetic modification of cytokine inducible sh2-containing protein (cish) gene
JP2021505587A (ja) 2017-12-05 2021-02-18 バーテックス ファーマシューティカルズ インコーポレイテッドＶｅｒｔｅｘ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｉｎｃｏｒｐｏｒａｔｅｄ Ｃｒｉｓｐｒ−ｃａｓ９改変ｃｄ３４＋ヒト造血幹・前駆細胞及びその使用
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
MA51787A (fr) * 2018-02-05 2020-12-16 Vertex Pharma Substances et méthodes de traitement d'hémoglobinopathies
IL277234B2 (en) 2018-03-14 2026-03-01 Editas Medicine Inc Systems and methods for treating hemoglobin diseases
US12522811B2 (en) 2018-05-01 2026-01-13 The Children's Medical Center Corporation Enhanced BCL11A RNP / CRISPR delivery and editing using a 3XNLS-CAS9
US12350284B2 (en) 2018-05-02 2025-07-08 The Children's Medical Center Corporation BCL11A microRNAs for treating hemoglobinopathies
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
CN110678553B (zh) * 2018-08-21 2023-08-11 中国医学科学院血液病医院(中国医学科学院血液学研究所) 在哺乳动物干细胞中进行基因组编辑的方法
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020205838A1 (en) 2019-04-02 2020-10-08 Sangamo Therapeutics, Inc. Methods for the treatment of beta-thalassemia
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
JP2022533713A (ja) 2019-05-21 2022-07-25 サンガモ セラピューティクス， インコーポレイテッド 調節性ｔ細胞における制御された導入遺伝子発現
EP4010004A4 (en) * 2019-08-07 2023-09-13 Altius Institute For Biomedical Sciences Compositions and methods for modulation of gene expression
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
WO2022167421A1 (en) * 2021-02-02 2022-08-11 Limagrain Europe Linkage of a distal promoter to a gene of interest by gene editing to modify gene expression
CN114015674B (zh) 2021-11-02 2024-08-30 辉大(上海)生物科技有限公司 一种CRISPR-Cas12i系统
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
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (114)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
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
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
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
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US5422251A (en) 1986-11-26 1995-06-06 Princeton University Triple-stranded nucleic acids
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5176996A (en) 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
AU7979491A (en) 1990-05-03 1991-11-27 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
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
JP4012243B2 (ja) 1994-01-18 2007-11-21 ザ スクリップス リサーチ インスティチュート 亜鉛フィンガータンパク質誘導体およびそのための方法
CA2186118C (en) 1994-03-23 2010-10-19 Richard W. Hanson Compacted nucleic acids and their delivery to cells
JPH10501686A (ja) 1994-04-13 1998-02-17 ザ ロックフェラー ユニヴァーシティ 神経系の細胞へのｄｎａのａａｖ仲介送達
US5585245A (en) 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
CA2196419C (en) 1994-08-20 2007-08-21 Yen Choo 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
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
US6342345B1 (en) 1997-04-02 2002-01-29 The Board Of Trustees Of The Leland Stanford Junior University Detection of molecular interactions by reporter subunit complementation
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6240461B1 (en) 1997-09-25 2001-05-29 Cisco Technology, Inc. Methods and apparatus for caching network data traffic
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
DE69942334D1 (de) 1998-03-02 2010-06-17 Massachusetts Inst Technology Poly-zinkfinger-proteine mit verbesserten linkern
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
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
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6503717B2 (en) 1999-12-06 2003-01-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
AU2982900A (en) 1999-02-03 2000-08-25 Children's Medical Center Corporation Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site
US20030104526A1 (en) * 1999-03-24 2003-06-05 Qiang Liu Position dependent recognition of GNN nucleotide triplets by zinc fingers
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
AU2001250774B2 (en) 2000-02-08 2005-01-27 Sangamo Biosciences, Inc. Cells expressing zinc finger proteins for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
AU2001263155A1 (en) 2000-05-16 2001-11-26 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
WO2002077277A2 (en) 2001-03-26 2002-10-03 Council Of Scientific And Industrial Research Dna markers for assessing seed purity and a method of using dna sequences for assessing seed purity
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F Zinc finger binding domains for cnn
US6998118B2 (en) 2001-12-21 2006-02-14 The Salk Institute For Biological Studies Targeted retrograde gene delivery for neuronal protection
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
ATE347593T1 (de) 2002-01-23 2006-12-15 Univ Utah Res Found Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen
EP1504092B2 (en) 2002-03-21 2014-06-25 Sangamo BioSciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
US9447434B2 (en) 2002-09-05 2016-09-20 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
EP2927318B1 (en) * 2003-08-08 2020-05-20 Sangamo Therapeutics, Inc. Methods and compositions for targeted cleavage and recombination
CN1897977A (zh) 2003-10-20 2007-01-17 Ns基因公司 帕金森氏病的体内基因治疗
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
ATE418346T1 (de) 2004-04-08 2009-01-15 Sangamo Biosciences Inc Zusammensetzungen zur behandlung von neuropathischen und neurodegenerativen erkrankungen
US7253273B2 (en) 2004-04-08 2007-08-07 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
KR20070060115A (ko) 2004-09-16 2007-06-12 상가모 바이오사이언스 인코포레이티드 단백질 생산을 위한 조성물 및 방법
WO2006121866A2 (en) 2005-05-05 2006-11-16 The Arizona Board Of Regents On Behalf Of The University Of Arizona Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
EP1913149A4 (en) 2005-07-26 2009-08-05 Sangamo Biosciences Inc TARGETED INTEGRATION AND EXPRESSION OF EXOGENOUS NUCLEIC ACID SEQUENCES
DK3246403T3 (da) 2005-10-18 2020-10-26 Prec Biosciences Rationelt designede meganucleaser med ændret sekvens-specificitet og dna-bindende affinitet
EP2027262B1 (en) 2006-05-25 2010-03-31 Sangamo Biosciences Inc. Variant foki cleavage half-domains
US7951925B2 (en) 2006-05-25 2011-05-31 Sangamo Biosciences, Inc. Methods and compositions for gene inactivation
US7837668B2 (en) 2006-10-10 2010-11-23 Ceregene, Inc. Needle assembly for use in delivering precise dosages of proteinaceous pharmaceutical compositions and methods for use of same
CA2669746C (en) * 2006-12-14 2017-05-16 Dow Agrosciences Llc Optimized non-canonical zinc finger proteins
US8110379B2 (en) 2007-04-26 2012-02-07 Sangamo Biosciences, Inc. Targeted integration into the PPP1R12C locus
US8585526B2 (en) 2007-06-18 2013-11-19 American Axle & Manufacturing, Inc. Vehicle driveline component having heat sink for increased heat rejection capabilities
US8153773B2 (en) 2007-06-19 2012-04-10 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse phosphorothioate analogs of the messenger RNA cap
JP5763341B2 (ja) 2007-09-27 2015-08-12 サンガモ バイオサイエンシーズ， インコーポレイテッド 生物学的活性のあるヌクレアーゼの迅速なインビボ同定法
EP2597155B1 (en) 2007-10-25 2016-11-23 Sangamo BioSciences, Inc. Methods and compositions for targeted integration
DE102007056956B4 (de) 2007-11-27 2009-10-29 Moosbauer, Peter, Dipl.-Ing.(FH) Schaltung zur Regelung der Stromversorgung eines Verbrauchers und Verfahren zum Betrieb einer Schaltung
CA2725773C (en) 2008-05-28 2017-12-05 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
JP5908725B2 (ja) 2008-08-22 2016-04-26 サンガモ バイオサイエンシーズ， インコーポレイテッド 標的一本鎖開裂および標的組込みのための方法、並びに組成物
ES2738980T3 (es) 2008-09-15 2020-01-28 Childrens Medical Ct Corp Modulación de BCL11A para el tratamiento de hemoglobinopatías
EP3156494B8 (en) 2008-12-04 2018-09-19 Sangamo Therapeutics, Inc. Genome editing in rats using zinc-finger nucleases
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
AU2010235161B2 (en) 2009-04-09 2015-01-22 Sangamo Therapeutics, Inc. Targeted integration into stem cells
WO2010147621A1 (en) * 2009-06-16 2010-12-23 The Trustees Of Columbia University In The City Of New York Methods for ameliorating adverse effects associated with transfusion of aged red blood cells
EA022786B1 (ru) 2009-07-31 2016-03-31 Этрис Гмбх Рнк с комбинацией из немодифицированных и модифицированных нуклеотидов для экспрессии белков
JP6137596B2 (ja) 2010-02-08 2017-05-31 サンガモ セラピューティクス， インコーポレイテッド 遺伝子操作された切断ハーフドメイン
US9255259B2 (en) 2010-02-09 2016-02-09 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US9567573B2 (en) 2010-04-26 2017-02-14 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using nucleases
EP2566972B1 (en) 2010-05-03 2020-01-15 Sangamo Therapeutics, Inc. Compositions for linking zinc finger modules
AU2011256838B2 (en) 2010-05-17 2014-10-09 Sangamo Therapeutics, Inc. Novel DNA-binding proteins and uses thereof
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
MX363013B (es) * 2011-02-25 2019-03-04 Recombinetics Inc Animales genéticamente modificados y métodos para su obtención.
EP2758529B1 (en) 2011-09-21 2019-03-20 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression
EP2766041B1 (en) * 2011-10-12 2018-12-05 Children's Medical Center Corporation Combinatorial compositions and methods of treating hemoglobinopathies
US9222105B2 (en) * 2011-10-27 2015-12-29 Sangamo Biosciences, Inc. Methods and compositions for modification of the HPRT locus
EP2836226B1 (en) * 2012-02-24 2017-06-28 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
CA2871524C (en) 2012-05-07 2021-07-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
EP2890780B8 (en) 2012-08-29 2020-08-19 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
ES2708948T3 (es) * 2012-11-27 2019-04-12 Childrens Medical Ct Corp Elementos reguladores distales de bcl11a como dianas para la reinducción de la hemoglobina fetal
EP2997146A4 (en) 2013-05-15 2017-04-26 Sangamo BioSciences, Inc. Methods and compositions for treatment of a genetic condition
CA2920899C (en) 2013-08-28 2023-02-28 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
ES2721936T3 (es) * 2013-11-13 2019-08-06 Childrens Medical Center Regulación de la expresión genética mediada por las nucleasas
SG10202112057QA (en) 2015-05-12 2021-12-30 Sangamo Therapeutics Inc Nuclease-mediated regulation of gene expression

Also Published As

Publication number Publication date
EP3068881B1 (en) 2019-02-27
PT3068881T (pt) 2019-05-31
ES2721936T3 (es) 2019-08-06
HRP20211706T1 (hr) 2022-02-04
DK3068881T3 (en) 2019-04-15
SI3068881T1 (sl) 2019-05-31
HRP20190825T1 (hr) 2019-06-28
LT3068881T (lt) 2019-08-12
EP3492593B1 (en) 2021-08-18
US11021696B2 (en) 2021-06-01
PL3068881T3 (pl) 2019-08-30
US20210348143A1 (en) 2021-11-11
HUE044540T2 (hu) 2019-10-28
RS62559B1 (sr) 2021-12-31
ES2895069T3 (es) 2022-02-17
WO2015073683A3 (en) 2015-07-09
HUE056436T2 (hu) 2022-02-28
EP3068881A4 (en) 2017-04-19
EP3068881A2 (en) 2016-09-21
DK3492593T3 (da) 2021-11-08
RS58671B1 (sr) 2019-06-28
WO2015073683A2 (en) 2015-05-21
LT3492593T (lt) 2021-11-25
SI3492593T1 (sl) 2022-03-31
EP3492593A1 (en) 2019-06-05
CY1121630T1 (el) 2020-07-31
EP3960856A1 (en) 2022-03-02
PT3492593T (pt) 2021-10-18
US20150132269A1 (en) 2015-05-14

Similar Documents

Publication Publication Date Title
IL284707B1 (en) Regulation of nuclease-mediated gene expression
PT3068881T (pt) Regulação da expressão de genes mediada por nucleases
IL244402A0 (en) Biomarkers of gene expression to predict responsiveness to lequinimod
PL2956477T5 (pl) Zoptymalizowany gen czynnika viii
LT3011037T (lt) Rizomanijai atsparus genas
ZA201504442B (en) Regulation of gene expression
GB201316880D0 (en) Controlling disclosure of structured data
ZA201804397B (en) Biosynthetic gene cluster of carrimycin
GB201206455D0 (en) Gene expression
IL236116A0 (en) Methods for controlled gene expression
PL2888228T3 (pl) Inhibitory oddziaływania CD40-TRAF6
SG10201800502VA (en) Clock gene expression level regulator
IL240204A0 (en) Bioreactor rack
GB201309132D0 (en) Regulatable gene expression
IL230063A0 (en) A technique to control selection order
GB201310482D0 (en) Regulation of DNA repair
AU2013903557A0 (en) Modulation of gene expression
GB201304608D0 (en) BenTime of Yisrael operable arrangement