CA2711179A1 - Rna interference for the treatment of heart failure - Google Patents

Rna interference for the treatment of heart failure Download PDF

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Publication number
CA2711179A1
CA2711179A1 CA2711179A CA2711179A CA2711179A1 CA 2711179 A1 CA2711179 A1 CA 2711179A1 CA 2711179 A CA2711179 A CA 2711179A CA 2711179 A CA2711179 A CA 2711179A CA 2711179 A1 CA2711179 A1 CA 2711179A1
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CA
Canada
Prior art keywords
rnai
vector
aav
expression
plb
Prior art date
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Abandoned
Application number
CA2711179A
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English (en)
French (fr)
Inventor
Roger J. Hajjar
Wolfgang Ch. Poller
Henry Fechner
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Nanocor Therapeutics Inc
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Individual
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Filing date
Publication date
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Publication of CA2711179A1 publication Critical patent/CA2711179A1/en
Abandoned legal-status Critical Current

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    • 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
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/06—Antiarrhythmics
    • 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
    • C12N15/86—Viral vectors
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Cardiology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Virology (AREA)
  • Hospice & Palliative Care (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
CA2711179A 2007-12-31 2008-12-23 Rna interference for the treatment of heart failure Abandoned CA2711179A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US1832607P 2007-12-31 2007-12-31
US61/018,326 2007-12-31
PCT/US2008/088248 WO2009088786A1 (en) 2007-12-31 2008-12-23 Rna interference for the treatment of heart failure

Publications (1)

Publication Number Publication Date
CA2711179A1 true CA2711179A1 (en) 2009-07-16

Family

ID=40853375

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2711179A Abandoned CA2711179A1 (en) 2007-12-31 2008-12-23 Rna interference for the treatment of heart failure

Country Status (8)

Country Link
US (2) US8404658B2 (https=)
EP (1) EP2225002A4 (https=)
JP (1) JP2011512326A (https=)
CN (1) CN101970051A (https=)
AU (1) AU2008346801A1 (https=)
CA (1) CA2711179A1 (https=)
NZ (1) NZ587060A (https=)
WO (1) WO2009088786A1 (https=)

Families Citing this family (169)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3075867B1 (en) * 2008-09-03 2019-03-13 Quantumdx Group Limited Sensing strategies and methods for nucleic acid detection using biosensors
KR20140004646A (ko) 2010-12-15 2014-01-13 미라젠 세러퓨틱스 잠금 뉴클레오티드를 포함하는 마이크로rna 억제제
MX374963B (es) 2011-04-29 2025-03-06 Selecta Biosciences Inc Nanoportadores sintéticos tolerogénicos para reducir las respuestas de anticuerpos.
KR20140091688A (ko) 2011-10-06 2014-07-22 미라젠 세러퓨틱스 인코포레이티드 마이크로rna 조절에 의한 전신 에너지 항상성의 제어
CA2864100A1 (en) * 2012-02-14 2013-08-22 The Regents Of The University Of California Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions
CN107236735B (zh) 2012-06-21 2019-11-08 米拉根医疗股份有限公司 包含锁核酸基序的基于寡核苷酸的抑制剂
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
US20140186843A1 (en) 2012-12-12 2014-07-03 Massachusetts Institute Of Technology Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
CN113355357B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
EP3031921B1 (en) 2012-12-12 2025-03-12 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
BR122020023215B1 (pt) 2013-05-03 2022-11-22 Selecta Biosciences, Inc Composição e kit de nanocarreadores sintéticos tolerogênicos para reduzir ou prevenir anafilaxia em resposta a um antígeno não alergênico
MX374532B (es) 2013-06-17 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos.
WO2014204724A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
RU2716420C2 (ru) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени
CN105683379A (zh) 2013-06-17 2016-06-15 布罗德研究所有限公司 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
WO2015077717A1 (en) 2013-11-25 2015-05-28 The Broad Institute Inc. Compositions and methods for diagnosing, evaluating and treating cancer by means of the dna methylation status
WO2015085147A1 (en) 2013-12-05 2015-06-11 The Broad Institute Inc. Polymorphic gene typing and somatic change detection using sequencing data
JP6712948B2 (ja) 2013-12-12 2020-06-24 ザ・ブロード・インスティテュート・インコーポレイテッド 組成物、及びヌクレオチドリピート障害におけるｃｒｉｓｐｒ−ｃａｓ系の使用方法
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
SG10201804975PA (en) 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for HBV and Viral Diseases and Disorders
JP7103750B2 (ja) 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用
EP3470089A1 (en) 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
KR20230076867A (ko) 2013-12-20 2023-05-31 더 브로드 인스티튜트, 인코퍼레이티드 신생항원 백신과의 병용 요법
WO2016028682A1 (en) 2014-08-17 2016-02-25 The Broad Institute Inc. Genome editing using cas9 nickases
JP7242180B2 (ja) * 2014-09-07 2023-03-20 セレクタ バイオサイエンシーズ インコーポレーテッド 抗ウイルス導入ベクター免疫応答を減弱化するための方法および組成物
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049251A1 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
EP3212788A2 (en) 2014-10-27 2017-09-06 The Broad Institute, Inc. Compositions, methods and use of synthetic lethal screening
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
US10975442B2 (en) 2014-12-19 2021-04-13 Massachusetts Institute Of Technology Molecular biomarkers for cancer immunotherapy
EP3234130B1 (en) 2014-12-19 2020-11-25 The Broad Institute, Inc. Methods for profiling the t-cell- receptor repertoire
EP3234192B1 (en) 2014-12-19 2021-07-14 The Broad Institute, Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology Crispr having or associated with destabilization domains
EP3247716A4 (en) 2015-01-20 2018-10-17 Miragen Therapeutics, Inc. Mir-92 inhibitors and uses thereof
EP3268044A2 (en) 2015-03-11 2018-01-17 The Broad Institute Inc. Prmt5 inhibitors for the treatment of cancer with reduced mtap activty
IL294183B2 (en) 2015-05-20 2023-10-01 Dana Farber Cancer Inst Inc shared neoantigens
TW202241500A (zh) 2015-06-09 2022-11-01 美商博德研究所有限公司 用於贅瘤疫苗之調配物及其製備方法
EP3822291A1 (en) 2015-06-10 2021-05-19 The Broad Institute Inc. Antibodies, compounds and screens for identifying and treating cachexia or pre-cachexia
WO2016205745A2 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
TWI906646B (zh) 2015-06-18 2025-12-01 美商博得學院股份有限公司 降低脫靶效應的ｃｒｉｓｐｒ酶突變
AU2016279077A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
WO2017031370A1 (en) 2015-08-18 2017-02-23 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
US12241053B2 (en) 2015-10-09 2025-03-04 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
CN116814590A (zh) 2015-10-22 2023-09-29 布罗德研究所有限公司 Vi-b型crispr酶和系统
EP3368687B1 (en) 2015-10-27 2021-09-29 The Broad Institute, Inc. Compositions and methods for targeting cancer-specific sequence variations
WO2017075451A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1
WO2017075465A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
WO2017075478A2 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by use of immune cell gene signatures
JP6978774B2 (ja) * 2015-11-06 2021-12-08 国立大学法人 熊本大学 心不全の予防又は治療のための医薬組成物
ES2934848T3 (es) 2015-12-11 2023-02-27 Univ Pennsylvania Método de purificación escalable para AAV8
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
US11028372B2 (en) 2015-12-11 2021-06-08 The Trustees Of The University Of Pennsylvania Scalable purification method for AAVRH10
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
AU2017254477A1 (en) 2016-04-18 2018-11-01 Jennifer G. ABELIN Improved HLA epitope prediction
EP3445853A1 (en) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
AU2017257274B2 (en) 2016-04-19 2023-07-13 Massachusetts Institute Of Technology Novel CRISPR enzymes and systems
KR20260004568A (ko) 2016-04-19 2026-01-08 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
CN109642231A (zh) 2016-06-17 2019-04-16 博德研究所 Vi型crispr直向同源物和系统
US12595478B2 (en) 2016-06-29 2026-04-07 The Broad Institute, Inc. Crispr-Cas systems having destabilization domain
WO2018035387A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
EP4485466A3 (en) 2016-08-17 2025-04-02 The Broad Institute Inc. Novel crispr enzymes and systems
WO2018049025A2 (en) 2016-09-07 2018-03-15 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses
US12499971B2 (en) 2016-09-28 2025-12-16 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
US12447213B2 (en) 2016-10-07 2025-10-21 The Broad Institute, Inc. Modulation of novel immune checkpoint targets
US11549149B2 (en) 2017-01-24 2023-01-10 The Broad Institute, Inc. Compositions and methods for detecting a mutant variant of a polynucleotide
ES3036482T3 (en) 2017-03-11 2025-09-19 Cartesian Therapeutics Inc Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
JP2020511141A (ja) 2017-03-15 2020-04-16 ザ・ブロード・インスティテュート・インコーポレイテッド 新規Ｃａｓ１３ｂオルソログＣＲＩＳＰＲ酵素及び系
AU2018251801B2 (en) 2017-04-12 2024-11-07 Massachusetts Institute Of Technology Novel type VI CRISPR orthologs and systems
WO2018191750A2 (en) 2017-04-14 2018-10-18 The Broad Institute Inc. Novel delivery of large payloads
US11591601B2 (en) 2017-05-05 2023-02-28 The Broad Institute, Inc. Methods for identification and modification of lncRNA associated with target genotypes and phenotypes
WO2018208910A1 (en) 2017-05-09 2018-11-15 The Broad Institute Inc. Gut microbiome function predicts response to anti-integrin biologic therapy in inflammatory bowel diseases
WO2018213726A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
KR102761791B1 (ko) 2017-06-26 2025-02-05 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 crispr/cas-아데닌 데아미나아제 기반 조성물, 시스템 및 방법
AU2018338318B2 (en) 2017-09-21 2022-12-22 Massachusetts Institute Of Technology Systems, methods, and compositions for targeted nucleic acid editing
WO2019071054A1 (en) 2017-10-04 2019-04-11 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR MODIFYING THE FUNCTION AND STRUCTURE OF BUCKLES AND / OR CHROMATIN DOMAINS
WO2019135816A2 (en) 2017-10-23 2019-07-11 The Broad Institute, Inc. Novel nucleic acid modifiers
WO2019089803A1 (en) 2017-10-31 2019-05-09 The Broad Institute, Inc. Methods and compositions for studying cell evolution
US12221720B2 (en) 2017-11-13 2025-02-11 The Broad Institute, Inc. Methods for determining spatial and temporal gene expression dynamics during adult neurogenesis in single cells
EP3710039A4 (en) 2017-11-13 2021-08-04 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR TREATMENT OF CANCER BY TARGETING THE CLEC2D-KLRB1 PATH
WO2019126709A1 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
CN108192922A (zh) * 2018-01-03 2018-06-22 李江 受磷蛋白基因反义rna重组aav载体、病毒、构建方法及应用
US10968257B2 (en) 2018-04-03 2021-04-06 The Broad Institute, Inc. Target recognition motifs and uses thereof
KR20250133482A (ko) 2018-05-11 2025-09-05 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일 뉴클레오티드 다형성을 편집하는 방법
CA3108281A1 (en) 2018-08-03 2020-02-06 Beam Therapeutics Inc. Multi-effector nucleobase editors and methods of using same to modify a nucleic acid target sequence
JP2021532815A (ja) 2018-08-07 2021-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 新規Ｃａｓ１２ｂ酵素およびシステム
US12421507B2 (en) 2018-08-20 2025-09-23 The Broad Institute, Inc. Methods and compositions for optochemical control of CRISPR-CAS9
WO2020072700A1 (en) 2018-10-02 2020-04-09 Dana-Farber Cancer Institute, Inc. Hla single allele lines
US20220062394A1 (en) 2018-12-17 2022-03-03 The Broad Institute, Inc. Methods for identifying neoantigens
CN113544266A (zh) 2018-12-17 2021-10-22 博德研究所 Crispr相关转座酶系统和其使用方法
WO2020127990A1 (en) 2018-12-20 2020-06-25 Biomedrex Ab System for production of crispr-based pharmaceutical compositions
MX2021009750A (es) 2019-02-12 2021-09-08 Univ Degli Studi Di Trento Moleculas del acido ribonucleico guia de cas12a y usos de las mismas.
US20220119787A1 (en) 2019-02-20 2022-04-21 Biomedrex Ab Virus therapy
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
US12419915B2 (en) 2019-03-13 2025-09-23 The Broad Institute, Inc. Microglial progenitors for regeneration of functional microglia in the central nervous system and therapeutics uses thereof
US20220152278A1 (en) * 2019-03-15 2022-05-19 University Of Washington Improved survival of human cells differentiated in vitro by prpf31 gene expression knockdown
US12534714B2 (en) 2019-03-18 2026-01-27 The Broad Institute, Inc. Type VII CRISPR proteins and systems
WO2020232271A1 (en) 2019-05-14 2020-11-19 The Broad Institute, Inc. Compositions and methods for targeting multinucleated cells
US20220235340A1 (en) 2019-05-20 2022-07-28 The Broad Institute, Inc. Novel crispr-cas systems and uses thereof
AU2020287627A1 (en) 2019-06-04 2021-11-18 Selecta Biosciences, Inc. Formulations and doses of PEGylated uricase
JP7759311B2 (ja) 2019-07-19 2025-10-23 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー リコンビナーゼ組成物及び使用方法
WO2021041922A1 (en) 2019-08-30 2021-03-04 The Broad Institute, Inc. Crispr-associated mu transposase systems
US20230279373A1 (en) 2019-09-09 2023-09-07 Beam Therapeutics Inc. Novel crispr enzymes, methods, systems and uses thereof
EP4028064A1 (en) 2019-09-12 2022-07-20 The Broad Institute, Inc. Engineered adeno-associated virus capsids
US12297426B2 (en) 2019-10-01 2025-05-13 The Broad Institute, Inc. DNA damage response signature guided rational design of CRISPR-based systems and therapies
US12394502B2 (en) 2019-10-02 2025-08-19 The General Hospital Corporation Method for predicting HLA-binding peptides using protein structural features
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
US20230055682A1 (en) 2019-12-03 2023-02-23 Beam Therapeutics lnc. Synthetic guide rna, compositions, methods, and uses thereof
WO2021152181A1 (en) 2020-01-30 2021-08-05 Biomedrex Ab Virus therapy
CN116209756A (zh) 2020-03-04 2023-06-02 旗舰先锋创新Vi有限责任公司 调控基因组的方法和组合物
EP4149618A4 (en) 2020-05-12 2024-07-03 Duke University COMPOSITIONS AND METHODS FOR CONTROLLING BLOOD PRESSURE
WO2021242793A2 (en) 2020-05-26 2021-12-02 The Broad Institute, Inc. Nucleic acid artificial mini-proteome libraries
US12419948B2 (en) 2020-08-31 2025-09-23 The Broad Institute, Inc. Immunogenic compositions and use thereof
CA3211495A1 (en) 2021-03-23 2022-09-29 Bernd ZETSCHE Novel crispr enzymes, methods, systems and uses thereof
KR20240007651A (ko) 2021-04-16 2024-01-16 빔 테라퓨틱스, 인크. 간세포의 유전적 변형
AU2022285744A1 (en) 2021-06-02 2023-12-14 Beam Therapeutics Inc. Circular guide rnas for crispr/cas editing systems
US20240325568A1 (en) 2021-07-20 2024-10-03 The Broad Institute, Inc. Engineered targeting compositions for endothelial cells of the central nervous system vasculature and methods of use thereof
KR20240037299A (ko) 2021-07-23 2024-03-21 빔 테라퓨틱스, 인크. Crispr/cas 편집 시스템용 가이드 rna
AU2022343268A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
EP4426828A1 (en) 2021-11-01 2024-09-11 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
EP4426829A1 (en) 2021-11-01 2024-09-11 Dana-Farber Cancer Institute, Inc. Biologically selected nucleic acid artificial mini-proteome libraries
WO2023091988A1 (en) 2021-11-18 2023-05-25 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Expression of the spike s glycoprotein of sars-cov-2 from avian paramyxovirus type 3 (apmv3)
WO2023093862A1 (en) 2021-11-26 2023-06-01 Epigenic Therapeutics Inc. Method of modulating pcsk9 and uses thereof
CA3240917A1 (en) 2021-12-17 2023-06-22 Bernd ZETSCHE Crispr enzymes, methzods, systems and uses thereof
AU2022420615A1 (en) 2021-12-22 2024-07-04 Tome Biosciences, Inc. Co-delivery of a gene editor construct and a donor template
WO2023196772A1 (en) 2022-04-04 2023-10-12 Beam Therapeutics Inc. Novel rna base editing compositions, systems, methods and uses thereof
WO2023205744A1 (en) 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
WO2023215831A1 (en) 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
EP4526324A1 (en) 2022-05-18 2025-03-26 The Broad Institute, Inc. Engineered viral capsids with increased stability and methods of use thereof
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
WO2024015892A1 (en) 2022-07-13 2024-01-18 The Broad Institute, Inc. Hla-ii immunopeptidome methods and systems for antigen discovery
AU2023307223A1 (en) 2022-07-14 2025-02-20 The Broad Institute, Inc. Aav capsids that enable cns-wide gene delivery through interactions with the transferrin receptor
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
WO2024059791A1 (en) 2022-09-16 2024-03-21 Beam Therapeutics Inc. Large serine recombinases, systems and uses thereof
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
WO2024163842A2 (en) 2023-02-03 2024-08-08 The Broad Institute, Inc. Delivering genes to the brain endothelium to treat lysosomal storage disorder-derived neuropathology
WO2024168253A1 (en) 2023-02-10 2024-08-15 Possible Medicines Llc Delivery of an rna guided recombination system
WO2024168265A1 (en) 2023-02-10 2024-08-15 Possible Medicines Llc Aav delivery of rna guided recombination system
CN116286820B (zh) * 2023-02-17 2024-11-12 华中科技大学同济医学院附属同济医院 一种shRNA、其重组表达载体、转化体、抗心衰药物及制备方法和制药用途
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
US12531162B1 (en) * 2023-05-31 2026-01-20 Northeastern University Multi-dimensional phenotypic space for genotype to phenotype mapping and intelligent design of cancer drug therapies using a deep learning net
WO2025022367A2 (en) 2023-07-27 2025-01-30 Life Edit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
WO2025059533A1 (en) 2023-09-13 2025-03-20 The Broad Institute, Inc. Crispr enzymes and systems
WO2025083619A1 (en) 2023-10-18 2025-04-24 Life Edit Therapeutics, Inc. Rna-guided nucleases and acive fragments and variants thereof and methods of use
WO2025097055A2 (en) 2023-11-02 2025-05-08 The Broad Institute, Inc. Compositions and methods of use of t cells in immunotherapy
US12562256B2 (en) * 2023-11-07 2026-02-24 New York University Systems, methods and computer-accessible medium for identifying target pairs for CAR-T therapy
WO2025106663A1 (en) 2023-11-15 2025-05-22 Slate Bio, Inc. Interleukin-33 variants, compositions and uses thereof
WO2025106685A1 (en) 2023-11-15 2025-05-22 Slate Bio, Inc. Interleukin-2 superkine and interleukin-33, compositions and uses thereof
WO2025106677A1 (en) 2023-11-15 2025-05-22 Slate Bio, Inc. Bifunctional il-2 and il-33 proteins for treating autoimmune disease
WO2025117544A1 (en) 2023-11-29 2025-06-05 The Broad Institute, Inc. Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof
WO2025155923A1 (en) 2024-01-17 2025-07-24 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with the transferrin receptor
WO2025160155A1 (en) 2024-01-22 2025-07-31 The Broad Institute, Inc. Epigenetic targeting of prion diseases
WO2025174908A1 (en) 2024-02-12 2025-08-21 Life Edit Therapeutics, Inc. Novel rna-guided nucleases and proteins for polymerase editing
WO2025188687A1 (en) 2024-03-04 2025-09-12 Beam Therapeutics Inc. Stable rna compositions with stem loop, and methods thereof
WO2025217163A2 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Novel aav capsids binding to human cd59
WO2025217174A1 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with carbonic anhydrase iv
EP4677108A1 (en) 2024-04-22 2026-01-14 Basecamp Research Ltd Method and compositions for detecting off-target editing
WO2026003754A1 (en) 2024-06-25 2026-01-02 Life Edit Therapeutics, Inc. Novel reverse transcriptases and uses thereof

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2573170T3 (en) * 2001-12-17 2018-04-09 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them, and their use
CN1498964A (zh) * 2002-11-07 2004-05-26 本元正阳基因技术股份有限公司 可诱导RNAi途径的用于基因治疗的系列重组腺相关病毒
US20050019927A1 (en) * 2003-07-13 2005-01-27 Markus Hildinger DECREASING GENE EXPRESSION IN A MAMMALIAN SUBJECT IN VIVO VIA AAV-MEDIATED RNAi EXPRESSION CASSETTE TRANSFER
US20060148742A1 (en) * 2004-02-26 2006-07-06 Kaye David M Polynucleotide delivery to cardiac tissue
EP1688356A3 (en) * 2005-02-04 2006-08-16 The Procter and Gamble Company Injection stretch blow-molded container
US20060198825A1 (en) * 2005-03-03 2006-09-07 Kaemmerer William F Reagents, methods and systems to suppress phospholamban expression
GB0518027D0 (en) * 2005-09-05 2005-10-12 Birkeland Innovasjon As Compounds
EP1939289A1 (en) 2006-12-29 2008-07-02 Charité-Universitätsmedizin Berlin Polynucleotide sequence for the inhibition of phospholamban synthesis

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US20110098338A1 (en) 2011-04-28
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