KR20210060429A - 적응 면역의 조절을 위한 조성물 및 방법 - Google Patents

적응 면역의 조절을 위한 조성물 및 방법 Download PDF

Info

Publication number
KR20210060429A
KR20210060429A KR1020217000507A KR20217000507A KR20210060429A KR 20210060429 A KR20210060429 A KR 20210060429A KR 1020217000507 A KR1020217000507 A KR 1020217000507A KR 20217000507 A KR20217000507 A KR 20217000507A KR 20210060429 A KR20210060429 A KR 20210060429A
Authority
KR
South Korea
Prior art keywords
sequence
rna
composition
protein
present disclosure
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.)
Ceased
Application number
KR1020217000507A
Other languages
English (en)
Korean (ko)
Inventor
데이비드 에이 넬스
란잔 바트라
유진 예오
Original Assignee
로카나바이오 인크.
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 로카나바이오 인크. filed Critical 로카나바이오 인크.
Publication of KR20210060429A publication Critical patent/KR20210060429A/ko
Ceased legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1136Non-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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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 against receptors or cell surface proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Endocrinology (AREA)
  • Mycology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020217000507A 2018-06-08 2019-06-07 적응 면역의 조절을 위한 조성물 및 방법 Ceased KR20210060429A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862682276P 2018-06-08 2018-06-08
US62/682,276 2018-06-08
PCT/US2019/036050 WO2019236998A1 (en) 2018-06-08 2019-06-07 Compositions and methods for the modulation of adaptive immunity

Publications (1)

Publication Number Publication Date
KR20210060429A true KR20210060429A (ko) 2021-05-26

Family

ID=68769461

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217000507A Ceased KR20210060429A (ko) 2018-06-08 2019-06-07 적응 면역의 조절을 위한 조성물 및 방법

Country Status (9)

Country Link
US (2) US20190382759A1 (https=)
EP (1) EP3801641A4 (https=)
JP (1) JP2021526860A (https=)
KR (1) KR20210060429A (https=)
CN (1) CN113286619A (https=)
AU (1) AU2019281006A1 (https=)
CA (1) CA3102783A1 (https=)
SG (1) SG11202012015YA (https=)
WO (1) WO2019236998A1 (https=)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9499805B2 (en) 2010-06-18 2016-11-22 The University Of North Carolina At Chapel Hill Methods and compositions for synthetic RNA endonucleases
US20190284553A1 (en) 2018-03-15 2019-09-19 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
JP2021526858A (ja) 2018-06-08 2021-10-11 ロックアネイビオ, インコーポレイテッド Rna標的化融合タンパク質組成物および使用方法
AU2020208346B2 (en) 2019-01-14 2026-01-29 University Of Rochester Targeted nuclear RNA cleavage and polyadenylation with CRISPR-cas
WO2021011504A1 (en) * 2019-07-12 2021-01-21 Duke University Nanoparticle systems for targeted delivery of crispr/cas13 and methods of using same
US11661459B2 (en) 2020-12-03 2023-05-30 Century Therapeutics, Inc. Artificial cell death polypeptide for chimeric antigen receptor and uses thereof
AU2021392032A1 (en) 2020-12-03 2023-06-22 Century Therapeutics, Inc. Genetically engineered cells and uses thereof
CN113136376B (zh) * 2021-05-26 2022-10-21 武汉大学 一种Cas12a变体及其在基因编辑中的应用
WO2023150131A1 (en) * 2022-02-01 2023-08-10 The Regents Of The University Of California Method of regulating alternative polyadenylation in rna
CN114848808B (zh) * 2022-03-24 2023-04-25 四川大学 基于阳离子脂多肽及细胞因子的免疫增强剂及制法、应用
CN114720699A (zh) * 2022-04-22 2022-07-08 浙江大学 一种结直肠癌早期预警血清指标及其检测方法和应用
CN116949011A (zh) * 2022-04-26 2023-10-27 中国科学院动物研究所 经分离的Cas13蛋白、基于它的基因编辑系统及其用途
CN115068632A (zh) * 2022-06-22 2022-09-20 华中科技大学同济医学院附属同济医院 Ago2在制备治疗心衰的药物方面的用途及其蛋白、基因、转化体、药物与制备方法
WO2024264035A2 (en) * 2023-06-22 2024-12-26 The General Hospital Corporation Compositions and methods of generating endogenous tdr molecules

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019500899A (ja) * 2015-11-23 2019-01-17 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア CRISPR/Cas9の核送達を通じた細胞RNAの追跡と操作
EA201891338A1 (ru) * 2015-12-04 2018-12-28 Новартис Аг Композиции и способы для иммуноонкологии
US12180499B2 (en) * 2016-10-31 2024-12-31 University Of Florida Research Foundation, Inc. Compositions and methods for impeding transcription of expanded microsatellite repeats
AU2018234825B2 (en) * 2017-03-15 2020-12-17 Massachusetts Institute Of Technology Novel CAS13B orthologues CRISPR enzymes and systems
US11168322B2 (en) * 2017-06-30 2021-11-09 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof

Also Published As

Publication number Publication date
US20240344060A1 (en) 2024-10-17
EP3801641A1 (en) 2021-04-14
SG11202012015YA (en) 2021-01-28
WO2019236998A1 (en) 2019-12-12
CN113286619A (zh) 2021-08-20
US20190382759A1 (en) 2019-12-19
CA3102783A1 (en) 2019-12-12
JP2021526860A (ja) 2021-10-11
EP3801641A4 (en) 2022-09-28
AU2019281006A1 (en) 2021-01-28

Similar Documents

Publication Publication Date Title
AU2022201296B2 (en) Genetically modified cells, tissues, and organs for treating disease
AU2020270508B2 (en) C/EBP alpha short activating RNA compositions and methods of use
KR20210060429A (ko) 적응 면역의 조절을 위한 조성물 및 방법
KR102907773B1 (ko) 인간 대상체에서의 비-노화-관련 청각 손상의 치료를 위한 조성물 및 방법
AU2017315679B2 (en) Compositions and methods for treating non-age-associated hearing impairment in a human subject
CN114176043B (zh) 用于治疗疾病的遗传修饰的细胞、组织和器官
AU2017235661B2 (en) Oligonucleotide probes and uses thereof
AU2023251441A1 (en) RNA containing composition for treatment of tumor diseases
KR101234281B1 (ko) 염색체 안정화에 관한 유전자를 표적으로 하는 암세포 특이적 아포토시스 유도제
AU2021202758A1 (en) Terminally modified RNA
KR20210049859A (ko) 게놈을 조절하는 방법 및 조성물
AU2016376191A1 (en) Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
AU2016364667A1 (en) Materials and methods for treatment of Alpha-1 antitrypsin deficiency
AU2016335698A1 (en) Multiplexed genome editing
KR20220157944A (ko) 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
CN110088272A (zh) 用于将细胞重编程为树突状细胞或抗原呈递细胞的组合物、其方法和用途
KR20120082906A (ko) 자가포식현상-향상 유전자 생성물의 조절을 통한 자가포식현상의 조절 방법
KR20220054401A (ko) 감염의 숙주 rna 바이오마커의 신속한 조기-검출 및 인간의 covid-19 코로나바이러스 감염의 조기 식별을 위한 시스템, 방법 및 조성물
KR20040065524A (ko) 백혈병을 검사 및 치료하는 방법
CN115151558A (zh) 哺乳动物序列中的靶向整合增强基因表达
KR20160059446A (ko) 암 유전체 돌연변이 검출용 유전자 패널
RU2829170C1 (ru) Адресное встраивание в последовательности млекопитающих, усиливающее экспрессию генов

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20210107

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20220603

Comment text: Request for Examination of Application

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20241220

Patent event code: PE09021S01D

E601 Decision to refuse application
PE0601 Decision on rejection of patent

Patent event date: 20250224

Comment text: Decision to Refuse Application

Patent event code: PE06012S01D