MX2019012452A - Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9. - Google Patents

Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9.

Info

Publication number
MX2019012452A
MX2019012452A MX2019012452A MX2019012452A MX2019012452A MX 2019012452 A MX2019012452 A MX 2019012452A MX 2019012452 A MX2019012452 A MX 2019012452A MX 2019012452 A MX2019012452 A MX 2019012452A MX 2019012452 A MX2019012452 A MX 2019012452A
Authority
MX
Mexico
Prior art keywords
engineered meganucleases
pcsk9 gene
recognition sequences
engineered
hypercholesterolemia
Prior art date
Application number
MX2019012452A
Other languages
English (en)
Inventor
Derek Jantz
Victor Bartsevich
James Jefferson Smith
Janel Lape
Original Assignee
Prec Biosciences 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 Prec Biosciences Inc filed Critical Prec Biosciences Inc
Publication of MX2019012452A publication Critical patent/MX2019012452A/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
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • 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
    • 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
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • 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
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Abstract

La presente invención abarca meganucleasas diseñadas que reconocen y cortan una secuencia de reconocimiento dentro del gen humano PCSK9. La presente invención también abarca métodos para utilizar dichas meganucleasas diseñadas en una composición farmacéutica y en métodos para tratar o reducir los síntomas de trastornos relacionados con el colesterol, tales como hipercolesterolemia Además, la invención abarca composiciones farmacéuticas que comprenden proteínas de meganucleasas diseñadas, ácidos nucleicos que codifican meganucleasas diseñadas y el uso de dichas composiciones para el tratamiento de trastornos relacionados con el colesterol, tales como hipercolesterolemia.
MX2019012452A 2017-04-21 2018-04-20 Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9. MX2019012452A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762488403P 2017-04-21 2017-04-21
US201762516966P 2017-06-08 2017-06-08
PCT/US2018/028607 WO2018195449A1 (en) 2017-04-21 2018-04-20 Engineered meganucleases specific for recognition sequences in the pcsk9 gene

Publications (1)

Publication Number Publication Date
MX2019012452A true MX2019012452A (es) 2020-08-03

Family

ID=62117064

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2019012452A MX2019012452A (es) 2017-04-21 2018-04-20 Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9.
MX2023001877A MX2023001877A (es) 2017-04-21 2019-10-17 Meganucleasas dise?adas especificas para secuencias de reconocimiento en el gen pcsk9.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2023001877A MX2023001877A (es) 2017-04-21 2019-10-17 Meganucleasas dise?adas especificas para secuencias de reconocimiento en el gen pcsk9.

Country Status (11)

Country Link
US (3) US11680254B2 (es)
EP (2) EP3612205B9 (es)
JP (3) JP2020517255A (es)
KR (2) KR20230170125A (es)
CN (2) CN117264929A (es)
AU (2) AU2018254576B2 (es)
CA (1) CA3060112A1 (es)
ES (1) ES2953153T3 (es)
IL (2) IL301059A (es)
MX (2) MX2019012452A (es)
WO (1) WO2018195449A1 (es)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020214724A1 (en) * 2019-04-15 2020-10-22 The Trustees Of The University Of Pennsylvania Compositions for regulating and self-inactivating enzyme expression and methods for modulating off-target activity of enzymes
EP3966322A1 (en) 2019-05-07 2022-03-16 Precision BioSciences, Inc. Optimization of engineered meganucleases for recognition sequences
EP4143308A1 (en) * 2020-04-27 2023-03-08 The Trustees of The University of Pennsylvania Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity
WO2021231259A1 (en) 2020-05-11 2021-11-18 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
EP4162059A1 (en) 2020-05-12 2023-04-12 The Trustees of The University of Pennsylvania Compositions for drg-specific reduction of transgene expression
US20230407333A1 (en) 2020-10-29 2023-12-21 The Trustees Of The University Of Pennsylvania Aav capsids and compositions containing same
AR125467A1 (es) 2021-04-27 2023-07-19 Univ Pennsylvania Cápsides de virus adenoasociados derivados de porcinos y usos de los estos
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2024015966A2 (en) 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4873192A (en) 1987-02-17 1989-10-10 The United States Of America As Represented By The Department Of Health And Human Services Process for site specific mutagenesis without phenotypic selection
US5652224A (en) * 1995-02-24 1997-07-29 The Trustees Of The University Of Pennsylvania Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism
US6015832A (en) 1997-12-31 2000-01-18 The Regents Of The University Of Michigan Methods of inactivating bacteria including bacterial spores
US6559189B2 (en) 1999-04-28 2003-05-06 Regents Of The University Of Michigan Non-toxic antimicrobial compositions and methods of use
US6506803B1 (en) 1999-04-28 2003-01-14 Regents Of The University Of Michigan Methods of preventing and treating microbial infections
US7767216B2 (en) 1999-04-28 2010-08-03 The Regents Of The University Of Michigan Antimicrobial compositions and methods of use
US6635676B2 (en) 1999-04-28 2003-10-21 Regents Of The University Of Michigan Non-toxic antimicrobial compositions and methods of use
US6555674B2 (en) 2000-08-09 2003-04-29 Nsgene A/S JeT promoter
EP2359869B1 (en) 2001-12-17 2018-12-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor
JP2006518372A (ja) * 2003-01-28 2006-08-10 セレクティス 脊椎動物の体組織においてエクスビボかつイントトで相同的組換えを誘発するためのメガヌクレアーゼの使用およびその応用
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
EP3795682A3 (en) 2005-10-18 2021-06-16 Precision Biosciences Rationally designed meganucleases with altered sequence specificity and dna-binding affinity
ES2366974T3 (es) * 2006-05-05 2011-10-27 Isis Pharmaceuticals, Inc. Compuestos y procedimientos para modular la expresión de sglt2.
AU2013201688B2 (en) * 2006-11-07 2016-03-03 Merck Sharp & Dohme Llc Antagonists of PCSK9
AU2013203689B2 (en) * 2007-08-23 2016-08-11 Amgen Inc. Antigen Binding Proteins to Proprotein Convertase Subtilisin Kexin Type 9 (PCSK9)
CA2603615A1 (en) 2007-09-21 2009-03-21 Patrick Labonte Methods of reducing viral infection and kits therefore
ES2422291T3 (es) 2007-10-31 2013-09-10 Prec Biosciences Inc Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas
US7928189B2 (en) * 2008-05-05 2011-04-19 Ottawa Health Research Institute PCSK9 polypeptide fragment
EP4001408A3 (en) * 2008-07-14 2022-06-08 Precision Biosciences, Inc. Recognition sequences for i-crei-derived meganucleases and uses thereof
EP2180058A1 (en) * 2008-10-23 2010-04-28 Cellectis Meganuclease recombination system
AU2013273674B2 (en) * 2009-09-03 2015-11-05 Pfizer Vaccines Llc PCSK9 vaccine
ES2773891T3 (es) 2011-06-01 2020-07-15 Prec Biosciences Inc Procedimientos para la producción de líneas celulares de mamífero modificadas con transgenes amplificados
EP2929035A1 (en) * 2012-12-07 2015-10-14 Shire Human Genetic Therapies, Inc. Lipidic nanoparticles for mrna delivering
US10329574B2 (en) * 2013-03-12 2019-06-25 E I Du Pont De Nemours And Company Methods for the identification of variant recognition sites for rare-cutting engineered double-strand-break-inducing agents and compositions and uses thereof
BR112015031608A2 (pt) * 2013-06-17 2017-08-22 Massachusetts Inst Technology Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos
CA2938857A1 (en) * 2014-02-11 2015-08-20 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
ES2876409T3 (es) * 2014-04-25 2021-11-12 Univ Pennsylvania Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol
WO2015175642A2 (en) * 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease
JP6775584B2 (ja) * 2015-09-08 2020-10-28 プレシジョン バイオサイエンシズ，インク． 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療
EP4108255A1 (en) * 2015-10-05 2022-12-28 Precision Biosciences, Inc. Genetically-modified cells comprising a modified human t cell receptor alpha constant region gene

Also Published As

Publication number Publication date
US11680254B2 (en) 2023-06-20
IL270020B1 (en) 2023-04-01
EP3612205B1 (en) 2023-06-07
US20240101985A1 (en) 2024-03-28
CN110769845A (zh) 2020-02-07
US20200131489A1 (en) 2020-04-30
EP3612205B9 (en) 2023-10-04
JP7214915B2 (ja) 2023-01-30
AU2018254576A1 (en) 2019-11-07
IL270020B2 (en) 2023-08-01
IL301059A (en) 2023-05-01
CA3060112A1 (en) 2018-10-25
JP2020517255A (ja) 2020-06-18
CN110769845B (zh) 2023-08-15
WO2018195449A1 (en) 2018-10-25
AU2018254576B2 (en) 2022-12-22
CN117264929A (zh) 2023-12-22
EP4269596A2 (en) 2023-11-01
US20240101986A1 (en) 2024-03-28
EP4269596A3 (en) 2024-02-14
KR20190140982A (ko) 2019-12-20
ES2953153T3 (es) 2023-11-08
JP2023058509A (ja) 2023-04-25
IL270020A (es) 2019-12-31
KR102620399B1 (ko) 2024-01-04
JP2022188058A (ja) 2022-12-20
AU2023201658A1 (en) 2023-06-22
EP3612205C0 (en) 2023-06-07
KR20230170125A (ko) 2023-12-18
EP3612205A1 (en) 2020-02-26
MX2023001877A (es) 2023-03-10

Similar Documents

Publication Publication Date Title
MX2023001877A (es) Meganucleasas dise?adas especificas para secuencias de reconocimiento en el gen pcsk9.
PH12018502361A1 (en) Gdf15 fusion proteins and uses thereof
PH12019500709A1 (en) Anti-lag-3 antibodies and methods of use thereof
MX2021006615A (es) Proteinas de union triespecificas y metodos de uso.
AU2017260426A1 (en) Engineered nucleases useful for treatment of hemophilia A
MX2020011939A (es) Nuevas enzimas y sistemas crispr.
SA518400424B1 (ar) جزيئات الجسم المضاد لعلاج السرطان
MX2018016389A (es) Composiciones y metodos para suministro de arn mensajero.
MX2019006045A (es) Proteinas triespecificas dirigidas a psma y metodos de uso.
WO2019006471A3 (en) NOVEL CRISPR RNA TARGETING ENZYMES, SYSTEMS AND USES THEREOF
PH12018501355A1 (en) Rsv f protein mutants
WO2017191274A3 (en) Rna encoding a therapeutic protein
MX2021014007A (es) Proteinas de union a epcam y metodos de uso.
PH12019500596A1 (en) Recombinant binding proteins and their use
WO2016154127A3 (en) Compositions and methods for treating hypertriglyceridemia
EA201171220A1 (ru) Мутанты fgf21 и их применение
MX2022007122A (es) Dominios de repetición de anquirina diseñados con residuos de superficie alterados.
EP3936141A3 (en) Anti-senescence compounds and uses thereof
MX2019004859A (es) Tratamientos de combinacion que comprenden la administracion de imidazopirazinonas.
WO2020030984A3 (en) Compositions and methods for genome engineering with cas12a proteins
JOP20200323A1 (ar) طرق لتصنيع تركيبات الأحماض الأمينية
WO2019014360A8 (en) POLYPEPTIDES BINDING COMPONENT C5 OR SERUM ALBUMIN COMPONENT AND FUSION PROTEINS THEREOF
EP4095152A3 (en) Engineered ribosomal promoters for protein production in microorganisms
MX2019006495A (es) Tratamiento de enfermedades neurologicas.
MX2019013124A (es) Proteinas de fusion de inhibidor de tripsina urinaria (uti).