MX2023012747A - Composiciones y métodos para el direccionamiento genético in vivo mediado por nucleasas para el tratamiento de trastornos genéticos. - Google Patents

Composiciones y métodos para el direccionamiento genético in vivo mediado por nucleasas para el tratamiento de trastornos genéticos.

Info

Publication number
MX2023012747A
MX2023012747A MX2023012747A MX2023012747A MX2023012747A MX 2023012747 A MX2023012747 A MX 2023012747A MX 2023012747 A MX2023012747 A MX 2023012747A MX 2023012747 A MX2023012747 A MX 2023012747A MX 2023012747 A MX2023012747 A MX 2023012747A
Authority
MX
Mexico
Prior art keywords
pcsk9
nuclease
treatment
nucleic acid
acid sequence
Prior art date
Application number
MX2023012747A
Other languages
English (en)
Spanish (es)
Inventor
Lili Wang
Anna Tretiakova
James M Wilson
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of MX2023012747A publication Critical patent/MX2023012747A/es

Links

Classifications

    • 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
    • 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
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C12N2800/40—Systems of functionally co-operating 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
MX2023012747A 2021-04-27 2022-04-27 Composiciones y métodos para el direccionamiento genético in vivo mediado por nucleasas para el tratamiento de trastornos genéticos. MX2023012747A (es)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202163180603P 2021-04-27 2021-04-27
US202163242474P 2021-09-09 2021-09-09
US202163244205P 2021-09-14 2021-09-14
US202263301933P 2022-01-21 2022-01-21
US202263331385P 2022-04-15 2022-04-15
PCT/US2022/026483 WO2022232232A1 (en) 2021-04-27 2022-04-27 Compositions and methods for in vivo nuclease-mediated gene targeting for the treatment of genetic disorders

Publications (1)

Publication Number Publication Date
MX2023012747A true MX2023012747A (es) 2024-01-05

Family

ID=83847293

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023012747A MX2023012747A (es) 2021-04-27 2022-04-27 Composiciones y métodos para el direccionamiento genético in vivo mediado por nucleasas para el tratamiento de trastornos genéticos.

Country Status (12)

Country Link
US (1) US20240197916A1 (https=)
EP (1) EP4330412A4 (https=)
JP (1) JP2024519469A (https=)
KR (1) KR20240001708A (https=)
AU (1) AU2022266662A1 (https=)
BR (1) BR112023021129A2 (https=)
CA (1) CA3216285A1 (https=)
CO (1) CO2023016031A2 (https=)
IL (1) IL307958A (https=)
MX (1) MX2023012747A (https=)
TW (1) TW202304528A (https=)
WO (1) WO2022232232A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023140971A1 (en) * 2022-01-21 2023-07-27 The Trustees Of The University Ofpennsylvania Methods for treatment of ornithine transcarbamylase (otc) deficiency
GB2634837A (en) 2022-06-07 2025-04-23 Scribe Therapeutics Inc Compositions and methods for the targeting of PCSK9
KR20260027890A (ko) * 2023-04-28 2026-03-03 오스페달레 산 라파엘 에스.알.엘. 벡터 생산
WO2025006782A2 (en) * 2023-06-30 2025-01-02 Chroma Medicine, Inc. Guide rna compositions
CN116790604B (zh) * 2023-08-18 2023-10-27 成都中科奥格生物科技有限公司 一种sgRNA、CRISPR/Cas9载体及其构建方法和用途
WO2025217184A1 (en) * 2024-04-08 2025-10-16 The Trustees Of The University Of Pennsylvania Compositions and methods for in vivo nuclease-mediated treatment of phenylketonuria (pku)
WO2025217186A1 (en) * 2024-04-08 2025-10-16 The Trustees Of The University Of Pennsylvania Compositions and methods for in vivo nuclease-mediated treatment of citrullinemia type 1 (ctln1)

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3288594B1 (en) * 2015-04-27 2022-06-29 The Trustees of The University of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
EP3416689B1 (en) * 2016-02-18 2023-01-18 CRISPR Therapeutics AG Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
EP3429632B1 (en) * 2016-03-16 2023-01-04 CRISPR Therapeutics AG Materials and methods for treatment of hereditary haemochromatosis
US20180127786A1 (en) * 2016-09-23 2018-05-10 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing
IL301059B2 (en) * 2017-04-21 2026-04-01 Prec Biosciences Inc Engineered nucleosides specific for recognition sequences in the PCSK9 gene
CA3079428A1 (en) * 2017-11-22 2019-05-31 Modernatx, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders

Also Published As

Publication number Publication date
US20240197916A1 (en) 2024-06-20
CO2023016031A2 (es) 2023-12-11
WO2022232232A1 (en) 2022-11-03
JP2024519469A (ja) 2024-05-14
EP4330412A4 (en) 2026-01-28
CA3216285A1 (en) 2022-11-03
AU2022266662A1 (en) 2023-11-02
AU2022266662A9 (en) 2023-11-16
IL307958A (en) 2023-12-01
EP4330412A1 (en) 2024-03-06
TW202304528A (zh) 2023-02-01
KR20240001708A (ko) 2024-01-03
BR112023021129A2 (pt) 2023-12-12

Similar Documents

Publication Publication Date Title
CO2023016031A2 (es) Composiciones y métodos para el direccionamiento genético in vivo mediado por nucleasas para el tratamiento de trastornos genéticos
Sui et al. Epigenetic regulation of mesenchymal stem cell homeostasis
Tatapudy et al. Cell fate decisions: emerging roles for metabolic signals and cell morphology
Jin et al. Circular RNA hsa_circ_0000523 regulates the proliferation and apoptosis of colorectal cancer cells as miRNA sponge
BR112021019512A2 (pt) Métodos para inserir uma sequência de codificação de proteína de ligação ao antígeno e para tratar ou efetuar a profilaxia de uma doença em um animal, animal, célula, genoma, ácido nucleico doador exógeno, gene de porto seguro, e, agente de nuclease ou um ou mais ácidos nucleicos
Huang et al. Synthetic chemically modified mrna-based delivery of cytoprotective factor promotes early cardiomyocyte survival post-acute myocardial infarction
EP4529958A3 (en) Controllable transcription
PH12022551594A1 (en) Method for treating usher syndrome and composition thereof
Boyraz et al. Posttranscriptional manipulation of TERC reverses molecular hallmarks of telomere disease
WO2020198509A3 (en) Modified oligonucleotides with increased stability
MX2021003457A (es) Composiciones y métodos para la edición génica de lactato deshidrogenasa (ldha).
Cleys et al. Identification of microRNAs in exosomes isolated from serum and umbilical cord blood, as well as placentomes of gestational day 90 pregnant sheep
MX2017011298A (es) Métodos para tratar cáncer que albergan la pérdida hemicigota de tp53.
Stimpfel et al. New challenge: mitochondrial epigenetics?
WO2006135436A3 (en) Inhibition of gene expression and therapeutic uses thereof
Iuso et al. Exogenous expression of human protamine 1 (hPrm1) remodels fibroblast nuclei into spermatid-like structures
Patel et al. Non-coding RNA as mediators in microenvironment–breast cancer cell communication
WO2018231018A3 (ko) 간에서 목적하는 단백질 발현하기 위한 플랫폼
Yang et al. The role of CDR1as in proliferation and differentiation of human umbilical cord‐derived mesenchymal stem cells
Huang et al. MicroRNA-133b negatively regulates zebrafish single mauthner-cell axon regeneration through targeting tppp3 in vivo
Hartwig et al. Telomeres and tissue engineering: the potential roles of TERT in VEGF-mediated angiogenesis
MX2025010451A (es) Composiciones y métodos para la inhibición de la expresión de genes de la subunidad beta e de la inhibina (inhbe)
CO2024005281A2 (es) Composiciones y métodos para expresar el factor ix para terapia para la hemofilia b
Zhao et al. A mesenchymal stem cell aging framework, from mechanisms to strategies
MX2022001984A (es) Terapia combinada transgénica y de miarn derivado de intrones para el tratamiento de ataxia espinocerebelosa 1 (sca1).