CL2021001488A1 - Transposasa de piggybac mutada - Google Patents

Transposasa de piggybac mutada

Info

Publication number
CL2021001488A1
CL2021001488A1 CL2021001488A CL2021001488A CL2021001488A1 CL 2021001488 A1 CL2021001488 A1 CL 2021001488A1 CL 2021001488 A CL2021001488 A CL 2021001488A CL 2021001488 A CL2021001488 A CL 2021001488A CL 2021001488 A1 CL2021001488 A1 CL 2021001488A1
Authority
CL
Chile
Prior art keywords
rnai constructs
chemically modified
mutated
vivo
target gene
Prior art date
Application number
CL2021001488A
Other languages
English (en)
Spanish (es)
Inventor
Kristine M Daris
Neeraj J Agrawal
Jennitte L Stevens
Huong Thi Ngoc Le
Noelia Blanco Talavan
Original Assignee
Amgen 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 Amgen Inc filed Critical Amgen Inc
Publication of CL2021001488A1 publication Critical patent/CL2021001488A1/es

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
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
    • 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
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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
    • 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
    • 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
    • C12N5/0681—Cells of the genital tract; Non-germinal cells from gonads
    • C12N5/0682—Cells of the female genital tract, e.g. endometrium; Non-germinal cells from ovaries, e.g. ovarian follicle cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C12P21/02—Preparation of peptides or proteins having a known sequence of two or more amino acids, e.g. glutathione
    • 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)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/90—Vectors containing a transposable element

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Mycology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Reproductive Health (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Hematology (AREA)
  • Developmental Biology & Embryology (AREA)
CL2021001488A 2018-12-10 2021-06-08 Transposasa de piggybac mutada CL2021001488A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862777325P 2018-12-10 2018-12-10
US201962925516P 2019-10-24 2019-10-24

Publications (1)

Publication Number Publication Date
CL2021001488A1 true CL2021001488A1 (es) 2021-11-26

Family

ID=69143664

Family Applications (3)

Application Number Title Priority Date Filing Date
CL2021001488A CL2021001488A1 (es) 2018-12-10 2021-06-08 Transposasa de piggybac mutada
CL2023003339A CL2023003339A1 (es) 2018-12-10 2023-11-09 Transposasa de piggybac mutada
CL2025004022A CL2025004022A1 (es) 2018-12-10 2025-12-19 Transposasa de piggybac mutada.

Family Applications After (2)

Application Number Title Priority Date Filing Date
CL2023003339A CL2023003339A1 (es) 2018-12-10 2023-11-09 Transposasa de piggybac mutada
CL2025004022A CL2025004022A1 (es) 2018-12-10 2025-12-19 Transposasa de piggybac mutada.

Country Status (22)

Country Link
US (1) US12570966B2 (enExample)
EP (1) EP3894549B1 (enExample)
JP (1) JP7441840B2 (enExample)
KR (1) KR102999228B1 (enExample)
CN (2) CN113195712B (enExample)
AU (2) AU2019398101B2 (enExample)
BR (1) BR112021011063A2 (enExample)
CA (1) CA3121268A1 (enExample)
CL (3) CL2021001488A1 (enExample)
DK (1) DK3894549T3 (enExample)
ES (1) ES2991895T3 (enExample)
FI (1) FI3894549T3 (enExample)
HU (1) HUE068630T2 (enExample)
IL (1) IL283314A (enExample)
LT (1) LT3894549T (enExample)
MX (1) MX2021006783A (enExample)
PL (1) PL3894549T3 (enExample)
PT (1) PT3894549T (enExample)
SG (1) SG11202106049RA (enExample)
SI (1) SI3894549T1 (enExample)
TW (1) TWI862531B (enExample)
WO (1) WO2020123327A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR3124522B1 (fr) * 2021-06-25 2025-02-28 Cherbonneau Francois Composition et méthode permettant l’édition génomique
WO2023122716A1 (en) * 2021-12-22 2023-06-29 Vanderbilt University Next generation transpososomes
KR20250018168A (ko) 2022-04-28 2025-02-04 이매틱스 유에스 인코포레이티드 막-결합된 il-15, cd8 폴리펩티드, 세포, 조성물, 및 이의 사용 방법
US20230348561A1 (en) 2022-04-28 2023-11-02 Immatics US, Inc. Dominant negative tgfbeta receptor polypeptides, cd8 polypeptides, cells, compositions, and methods of using thereof
WO2023212655A1 (en) 2022-04-28 2023-11-02 Immatics US, Inc. Il-12 polypeptides, il-15 polypeptides, il-18 polypeptides, cd8 polypeptides, compositions, and methods of using thereof
CN121127601A (zh) * 2023-05-12 2025-12-12 隆萨生物制剂股份有限公司 使用转座酶进行混合瞬时转染以用于高产率产物生产
AU2024300966A1 (en) 2023-07-21 2026-01-08 Amgen Inc. Vectors incorporating a combination of promoters driving selectable marker expression
US20250134931A1 (en) 2023-11-01 2025-05-01 Immatics US, Inc. Membrane-bound il-15, cd8 polypeptides, cells, compositions, and methods of using thereof
CN120737218B (zh) * 2025-08-15 2026-01-06 云南彤宁生物制药技术有限公司 融合蛋白、调控系统及利用该系统构建转基因细胞的方法

Family Cites Families (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4965195A (en) 1987-10-26 1990-10-23 Immunex Corp. Interleukin-7
US4968607A (en) 1987-11-25 1990-11-06 Immunex Corporation Interleukin-1 receptors
WO1990005183A1 (en) 1988-10-31 1990-05-17 Immunex Corporation Interleukin-4 receptors
WO1991018982A1 (en) 1990-06-05 1991-12-12 Immunex Corporation Type ii interleukin-1 receptors
US6319494B1 (en) 1990-12-14 2001-11-20 Cell Genesys, Inc. Chimeric chains for receptor-associated signal transduction pathways
IL104570A0 (en) 1992-03-18 1993-05-13 Yeda Res & Dev Chimeric genes and cells transformed therewith
US5827642A (en) 1994-08-31 1998-10-27 Fred Hutchinson Cancer Research Center Rapid expansion method ("REM") for in vitro propagation of T lymphocytes
NZ330926A (en) 1996-01-11 1999-11-29 Immunex Corp Expression augmenting sequence elements (ease) for eukaryotic expression systems
US6218185B1 (en) 1996-04-19 2001-04-17 The United States Of America As Represented By The Secretary Of Agriculture Piggybac transposon-based genetic transformation system for insects
CA2196496A1 (en) 1997-01-31 1998-07-31 Stephen William Watson Michnick Protein fragment complementation assay for the detection of protein-protein interactions
US6867041B2 (en) 2000-02-24 2005-03-15 Xcyte Therapies, Inc. Simultaneous stimulation and concentration of cells
HK1052372A1 (zh) 2000-02-24 2003-09-11 Xcyte Therapies, Inc. 细胞的同步刺激和富集
US6797514B2 (en) 2000-02-24 2004-09-28 Xcyte Therapies, Inc. Simultaneous stimulation and concentration of cells
US7105343B1 (en) 2000-10-31 2006-09-12 University Of Notre Dame Du Lac Methods and compositions for transposition using minimal segments of the eukaryotic transformation vector Piggybac
AU2003268022B2 (en) 2002-07-24 2009-02-05 Manoa Biosciences Inc. Transposon-based vectors and methods of nucleic acid integration
US7932088B1 (en) 2005-04-25 2011-04-26 University Of Notre Dame Du Lac High efficiency transformation of Plasmodium falciparum by the lepidopteran transposon, piggyBac
WO2007100821A2 (en) 2006-02-28 2007-09-07 University Of Notre Dame Piggybac constructs in vertebrates
US20090042297A1 (en) 2007-06-01 2009-02-12 George Jr Alfred L Piggybac transposon-based vectors and methods of nucleic acid integration
WO2010085699A2 (en) 2009-01-23 2010-07-29 The Johns Hopkins University Mammalian piggybac transposon and methods of use
CA2750027C (en) 2009-01-23 2020-11-10 Nsgene A/S Improved cell lines and their use in encapsulated cell biodelivery
US20110311506A1 (en) 2009-02-25 2011-12-22 The Johns Hopkins University Piggybac transposon variants and methods of use
EP4733396A2 (en) 2009-02-26 2026-04-29 Poseida Therapeutics, Inc. Hyperactive piggybac transposases
US20100311116A1 (en) 2009-06-04 2010-12-09 Excellgene Sa Fast generation of high expression stable cell lines expressing recombinant proteins under minimal and short-term selective pressure
CN102803484A (zh) 2009-06-11 2012-11-28 大学共同利用机关法人情报·系统研究机构 生产蛋白质的方法
JP2013519652A (ja) * 2010-02-12 2013-05-30 ディーエスエム アイピー アセッツ ビー．ブイ． 単一ユニット抗体精製
US9089520B2 (en) 2010-05-21 2015-07-28 Baylor College Of Medicine Methods for inducing selective apoptosis
GB201014169D0 (en) 2010-08-25 2010-10-06 Cambridge Entpr Ltd In vitro hepatic differentiation
WO2012074758A1 (en) 2010-11-16 2012-06-07 Transposagen Bioharmaceuticals, Inc. Hyperactive piggybac transposases
PH12013501201A1 (en) 2010-12-09 2013-07-29 Univ Pennsylvania Use of chimeric antigen receptor-modified t cells to treat cancer
WO2012129514A1 (en) 2011-03-23 2012-09-27 Fred Hutchinson Cancer Research Center Method and compositions for cellular immunotherapy
US9783790B2 (en) * 2011-07-15 2017-10-10 The Johns Hopkins University Trichoplusia ni piggybac transposases with reduced integration activity
WO2013019625A2 (en) 2011-07-29 2013-02-07 The University Of Wyoming Methods of using a bacterial glcnac-6-p 2'-epimerase to promote sialylation of glycoconjugates
US9796974B2 (en) 2011-11-18 2017-10-24 Alnylam Pharmaceuticals, Inc. Modified RNAi agents
DK2951309T3 (en) 2013-02-01 2019-04-23 Selexis Sa INCREASED TRANSGEN EXPRESSION AND PROCESSING
US20160015749A1 (en) * 2013-03-05 2016-01-21 Baylor College Of Medicine Engager cells for immunotherapy
CN103360464A (zh) 2013-07-17 2013-10-23 武汉摩尔生物科技有限公司 多肽、编码该多肽的dna分子、载体、制备方法及应用
US11306328B2 (en) 2013-07-26 2022-04-19 President And Fellows Of Harvard College Genome engineering
GB201316644D0 (en) 2013-09-19 2013-11-06 Kymab Ltd Expression vector production & High-Throughput cell screening
HUE052552T2 (hu) * 2014-04-09 2021-05-28 Dna Twopointo Inc Javított nukleinsav-konstrukciók eukarióta génexpresszióhoz
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
EP3018200A1 (en) 2014-11-07 2016-05-11 Fondazione Matilde Tettamanti e Menotti de Machi Onlus Improved method for the generation of genetically modified cells
CN105154473B (zh) 2015-09-30 2019-03-01 上海细胞治疗研究院 一种高效安全的转座子整合系统及其用途
LT3359671T (lt) 2015-10-08 2021-12-10 Dna Twopointo Inc. Dnr vektoriai, transpozonai ir transpozazės, skirti eukariotinio genomo modifikacijai
CA3004665C (en) 2015-12-14 2021-08-24 GenomeFrontier Therapeutics, Inc. Transposon system, kit comprising the same, and uses thereof
JP2019506177A (ja) 2016-02-26 2019-03-07 ポセイダ セラピューティクス， インコーポレイテッド トランスポゾン系および使用方法
EP3219800A1 (en) 2016-03-15 2017-09-20 Max-Delbrück-Centrum Für Molekulare Medizin A transposon-based transfection system for primary cells
CN110402305B (zh) 2016-11-30 2023-07-21 北京复昇生物科技有限公司 一种crispr文库筛选的方法
US20210115453A1 (en) 2017-08-31 2021-04-22 Poseida Therapeutics, Inc. Transposon system and methods of use
SG11202105900UA (en) 2018-12-10 2021-07-29 Amgen Inc CHEMICALLY-MODIFIED RNAi CONSTRUCTS AND USES THEREOF

Also Published As

Publication number Publication date
SG11202106049RA (en) 2021-07-29
AU2026201887A1 (en) 2026-04-02
CL2025004022A1 (es) 2026-04-10
CA3121268A1 (en) 2020-06-18
CL2023003339A1 (es) 2024-06-07
DK3894549T3 (da) 2024-10-14
SI3894549T1 (sl) 2024-10-30
BR112021011063A2 (pt) 2021-08-31
JP2022512332A (ja) 2022-02-03
MX2021006783A (es) 2021-07-15
ES2991895T3 (es) 2024-12-05
EP3894549A1 (en) 2021-10-20
PT3894549T (pt) 2024-10-16
KR20210101270A (ko) 2021-08-18
US20220064610A1 (en) 2022-03-03
CN113195712B (zh) 2024-09-13
IL283314A (en) 2021-07-29
FI3894549T3 (fi) 2024-10-23
JP7441840B2 (ja) 2024-03-01
LT3894549T (lt) 2024-09-25
KR102999228B1 (ko) 2026-07-31
AU2019398101A1 (en) 2021-06-10
HUE068630T2 (hu) 2025-01-28
AU2019398101B2 (en) 2025-12-18
EP3894549B1 (en) 2024-07-31
PL3894549T3 (pl) 2024-11-25
TWI862531B (zh) 2024-11-21
TW202035687A (zh) 2020-10-01
WO2020123327A1 (en) 2020-06-18
CN113195712A (zh) 2021-07-30
CN118995660A (zh) 2024-11-22
US12570966B2 (en) 2026-03-10

Similar Documents

Publication Publication Date Title
CL2021001490A1 (es) Constructos de iarn modificados químicamente y usos de estos
CL2019000732A1 (es) Tratamiento avv de la enfermedad de huntington.
PH12019550076A1 (en) SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
MX2018000981A (es) Composiciones de acido ribonucleico interferente (arni) de transtiretina (ttr) y metodos para su uso para el tratamiento o prevencion de enfermedades asociadas con transtiretina (ttr).
JOP20160211B1 (ar) تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
UY38003A (es) Construcciones de arni para inhibir la expresión de pnpla3 y métodos de uso de las mismas
MX381069B (es) Profármacos de carbidopa y l-dopa y métodos de uso.
MX382225B (es) Composiciones y metodos para inhibir la expresion del gen de hidroxiacido-oxidasa 1 (glicolato-oxidasa) (hao1).
BR112017013597A2 (pt) molécula de ácido nucleico, composição farmacêutica, vetor ou célula, e, método para tratamento de uma doença.
CL2021001489A1 (es) Constructos de arni para inhibir la expresión de pnpla3 y métodos de uso de estos
BR112018007453A2 (pt) genes modificados de ataxia de friedreich e vetores para a terapia gênica
MX378277B (es) Metodo para tratar cancer usando una combinacion de inhibidores de chk1 y relacionado con atm y rad3 (atr).
MX2020007060A (es) Compuestos, composiciones y metodos para tratar enfermedades que involucren tejidos con enfermedades acidas o hipoxicas.
UY40039A (es) COMPOSICIONES DE ARNi DEL COMPONENTE DEL COMPLEMENTO C5 Y MÉTODOS PARA SU USO
EA201491953A1 (ru) СОЕДИНЕНИЯ НА ОСНОВЕ МИКРОРНК И СПОСОБЫ МОДУЛИРОВАНИЯ АКТИВНОСТИ miR-21
MX351028B (es) Determinadadas entidades, composiciones y metodos quimicos.
SG10201804472YA (en) SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
NI201700056A (es) 2-amino-3,5-difluoro-6-metil-6-fenil-3,4,5,6-tetrahidropiridinas en calidad de inhibidores de bace1 para el tratamiento de la enfermedad de alzhéimer
EA201792103A1 (ru) Композиции и способы для ингибирования экспрессии генов hif2альфа
MX2017011298A (es) Métodos para tratar cáncer que albergan la pérdida hemicigota de tp53.
MX2022004388A (es) Composiciones de agentes de arni de huntingtina (htt) y metodos de uso de las mismas.
WO2019217397A3 (en) Compositions and methods for improving strand biased
CL2017000643A1 (es) Inhibidores de histona desmetilasa
CL2021003169A1 (es) Construcciones de arni para inhibir la expresión de scap y métodos de uso de las mismas
BR112021021686A2 (pt) Moléculas inibidoras de ácido nucleico de fita dupla com fitas senso curtas