[go: up one dir, main page]

MX2023006444A - Composiciones novedosas con motivos dirigidos a tejido específico y composiciones que los contienen. - Google Patents

Composiciones novedosas con motivos dirigidos a tejido específico y composiciones que los contienen.

Info

Publication number
MX2023006444A
MX2023006444A MX2023006444A MX2023006444A MX2023006444A MX 2023006444 A MX2023006444 A MX 2023006444A MX 2023006444 A MX2023006444 A MX 2023006444A MX 2023006444 A MX2023006444 A MX 2023006444A MX 2023006444 A MX2023006444 A MX 2023006444A
Authority
MX
Mexico
Prior art keywords
compositions
tissue
specific targeting
containing same
targeting motifs
Prior art date
Application number
MX2023006444A
Other languages
English (en)
Inventor
James M Wilson
Joshua Joyner Sims
Yuan Yuan
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 MX2023006444A publication Critical patent/MX2023006444A/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
    • 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
    • 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
    • A61K48/0025—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 wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A61K39/215—Coronaviridae, e.g. avian infectious bronchitis virus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/42—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum viral
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/525—Virus
    • A61K2039/5256—Virus expressing foreign proteins
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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
    • C12N2750/14145—Special targeting system for 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/40—Vectors comprising a peptide as targeting moiety, e.g. a synthetic peptide, from undefined source

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Communicable Diseases (AREA)
  • Mycology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pulmonology (AREA)
  • Oncology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Se proporcionan en la presente composiciones que incluyen péptidos de direccionamiento ligados a estas o insertados en una proteína de direccionamiento de un vector recombinante que tiene al menos un péptido exógeno que comprende una secuencia de aminoácidos de N- x- (T/I/V/A)- (K/R) (SEQ ID NO: 47). Se proporcionan composiciones que proporcionan tales conjugados, péptidos de direccionamiento o vectores recombinantes que tienen una cápside mutante o proteína de envoltura, así como usos de estos.
MX2023006444A 2020-12-01 2021-12-01 Composiciones novedosas con motivos dirigidos a tejido específico y composiciones que los contienen. MX2023006444A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063119863P 2020-12-01 2020-12-01
PCT/US2021/061312 WO2022119871A2 (en) 2020-12-01 2021-12-01 Novel compositions with tissue-specific targeting motifs and compositions containing same

Publications (1)

Publication Number Publication Date
MX2023006444A true MX2023006444A (es) 2023-08-10

Family

ID=80050581

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023006444A MX2023006444A (es) 2020-12-01 2021-12-01 Composiciones novedosas con motivos dirigidos a tejido específico y composiciones que los contienen.

Country Status (12)

Country Link
US (1) US20240024507A1 (es)
EP (1) EP4256065A2 (es)
JP (1) JP2023551903A (es)
KR (1) KR20230117157A (es)
CN (1) CN116670152A (es)
AR (1) AR124216A1 (es)
AU (1) AU2021390480A1 (es)
CA (1) CA3200014A1 (es)
IL (1) IL303236A (es)
MX (1) MX2023006444A (es)
TW (1) TW202237850A (es)
WO (1) WO2022119871A2 (es)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2020253435A1 (en) 2019-04-01 2021-11-18 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
CA3234664A1 (en) 2021-10-08 2023-04-13 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2024016003A2 (en) * 2022-07-14 2024-01-18 The Broad Institute, Inc. Aav capsids that enable cns-wide gene delivery through interactions with the transferrin receptor
WO2024207061A1 (en) * 2023-04-04 2024-10-10 Children's Medical Research Institute Aav capsids and vectors for transduction of cells of the peripheral nervous system
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
WO2025137290A1 (en) * 2023-12-19 2025-06-26 The Broad Institute, Inc. Methods and systems directed to therapeutic delivery of the human gba1 gene

Family Cites Families (96)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
NZ261277A (en) 1993-02-05 1997-04-24 Laporte Group Australia Ltd Slag defoaming composition comprising a carbon source; an exothermic material (eg aluminium) and a dense inert granular material (eg calcium silicate)
US6140120A (en) 1993-02-12 2000-10-31 Board Of Trustees Of Leland Stanford Jr. University Regulated transcription of targeted genes and other biological events
US5834266A (en) 1993-02-12 1998-11-10 President & Fellows Of Harvard College Regulated apoptosis
US20020173474A1 (en) 1993-02-12 2002-11-21 President And Fellows Of Harvard College Methods & materials involving dimerization-mediated regulation of biological events
US6972193B1 (en) 1993-02-12 2005-12-06 Board Of Trustees Of Leland Stanford Junior University Regulated transcription of targeted genes and other biological events
PL310327A1 (en) 1993-02-12 1995-12-11 Univ Leland Stanford Junior Adjustable transcription of target genes and other biological processes
US5830462A (en) 1993-02-12 1998-11-03 President & Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
US5869337A (en) 1993-02-12 1999-02-09 President And Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
US6150137A (en) 1994-05-27 2000-11-21 Ariad Pharmaceuticals, Inc. Immunosuppressant target proteins
US6476200B1 (en) 1994-06-27 2002-11-05 The Johns Hopkins University Mammalian proteins that bind to FKBP12 in a rapamycin-dependent fashion
US6492106B1 (en) 1994-06-27 2002-12-10 The Johns Hopkins University Mammalian proteins that bind to FKBP12 in a rapamycin-dependent fashion
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
CA2197793C (en) 1994-08-18 2007-04-24 Dennis A. Holt New multimerizing agents
US6133456A (en) 1994-08-18 2000-10-17 Ariad Gene Therapeutics, Inc. Synthetic multimerizing agents
US6150527A (en) 1994-08-18 2000-11-21 Ariad Pharmaceuticals, Inc. Synthetic multimerizing agents
US6326166B1 (en) 1995-12-29 2001-12-04 Massachusetts Institute Of Technology Chimeric DNA-binding proteins
WO1996020951A1 (en) 1994-12-29 1996-07-11 Massachusetts Institute Of Technology Chimeric dna-binding proteins
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
US5811524A (en) 1995-06-07 1998-09-22 Idec Pharmaceuticals Corporation Neutralizing high affinity human monoclonal antibodies specific to RSV F-protein and methods for their manufacture and therapeutic use thereof
WO1996041865A1 (en) 1995-06-07 1996-12-27 Ariad Gene Therapeutics, Inc. Rapamcycin-based regulation of biological events
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
AU731826B2 (en) 1996-02-28 2001-04-05 Ariad Pharmaceuticals, Inc. Synthetic Multimerizing Agents
US6723531B2 (en) 1996-04-05 2004-04-20 The Salk Institute For Biological Studies Method for modulating expression of exogenous genes in mammalian systems, and products related thereto
EP0937082A2 (en) 1996-07-12 1999-08-25 Ariad Pharmaceuticals, Inc. Materials and method for treating or preventing pathogenic fungal infection
AU728220B2 (en) 1997-04-14 2001-01-04 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
WO1999010510A2 (en) 1997-08-26 1999-03-04 Ariad Gene Therapeutics, Inc. Fusion proteins comprising a dimerization, trimerization or tetramerization domain and an additional heterologous transcription activation, transcription repression, dna binding or ligand binding domain
US6015709A (en) 1997-08-26 2000-01-18 Ariad Pharmaceuticals, Inc. Transcriptional activators, and compositions and uses related thereto
US6479653B1 (en) 1997-08-26 2002-11-12 Ariad Gene Therapeutics, Inc. Compositions and method for regulation of transcription
IL134643A0 (en) 1997-08-27 2001-04-30 Ariad Gene Therapeutics Inc Chimeric transcriptional activators and compositions and uses related thereto
EP1045915A2 (en) 1998-01-15 2000-10-25 Ariad Gene Therapeutics, Inc. Regulation of biological events using multimeric chimeric proteins
WO1999041258A1 (en) 1998-02-13 1999-08-19 President And Fellows Of Harvard College Novel dimerizing agents, their production and use
US6984635B1 (en) 1998-02-13 2006-01-10 Board Of Trustees Of The Leland Stanford Jr. University Dimerizing agents, their production and use
US6333318B1 (en) 1998-05-14 2001-12-25 The Salk Institute For Biological Studies Formulations useful for modulating expression of exogenous genes in mammalian systems, and products related thereto
WO1999061643A1 (en) 1998-05-27 1999-12-02 University Of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient
US6258603B1 (en) 1998-06-17 2001-07-10 Rohm And Haas Company Ligands for modulating the expression of exogenous genes via an ecdysone receptor complex
US7109317B1 (en) 1998-11-06 2006-09-19 President And Fellows Of Harvard College FK506-based regulation of biological events
US6491907B1 (en) 1998-11-10 2002-12-10 The University Of North Carolina At Chapel Hill Recombinant parvovirus vectors and method of making
US6596535B1 (en) 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
US7067526B1 (en) 1999-08-24 2006-06-27 Ariad Gene Therapeutics, Inc. 28-epirapalogs
EP1212331B1 (en) 1999-08-24 2004-04-21 Ariad Gene Therapeutics, Inc. 28-epirapalogs
MXPA02009157A (es) 2000-03-22 2004-04-05 Rohm & Haas Nuevo sistema de expresion genetica inducible a base del receptor de ecdisona.
US20040033600A1 (en) 2001-03-21 2004-02-19 Palli Subba Reddy Ecdysone receptor-based inducible gene expression system
ATE438414T1 (de) 2000-06-01 2009-08-15 Univ North Carolina Verfahren und zusammensetzungen zur kontrollierter abgabe von rekombinant parvovirus vektoren
US8105825B2 (en) 2000-10-03 2012-01-31 Intrexon Corporation Multiple inducible gene regulation system
MXPA03007493A (es) 2001-02-20 2004-10-15 Rheogene Holdings Inc Nuevo sistema de expresion genetica inducible a base de receptor de ecdisona/receptor x retinoide de invertebrado.
MXPA03007496A (es) 2001-02-20 2004-10-15 Rheogene Inc Nuevos receptores mutantes de sustitucion y su uso en un sistema de expresion genetica inducible a base de receptores nucleares.
EP1534738B1 (en) 2001-02-20 2012-06-20 Intrexon Corporation Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system
ES2392508T3 (es) 2001-02-20 2012-12-11 Intrexon Corporation Receptores X retinoides quiméricos y su uso en un sistema inducible de expresión génica basado en receptores de ecdisona novedoso
HU230406B1 (hu) 2001-11-13 2016-04-28 The Trustees Of The University Of Pennsylvania Eljárás adeno-asszociált vírus(AAV)szekvenciák detektálására és/vagy azonosítására és az azzal azonosított új szekvenciák izolálására
AU2002360291A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
HUE054805T2 (hu) 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik
US20060246079A1 (en) 2003-11-14 2006-11-02 Morrow Phillip R Neutralizing human antibodies to anthrax toxin
US7935510B2 (en) 2004-04-30 2011-05-03 Intrexon Corporation Mutant receptors and their use in a nuclear receptor-based inducible gene expression system
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
JP4495210B2 (ja) 2005-06-09 2010-06-30 パナソニック株式会社 振幅誤差補償装置及び直交度誤差補償装置
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
WO2008156763A2 (en) 2007-06-15 2008-12-24 The Board Of Trustees Of The Leland Stanford Junior University Human neutralizing monoclonal antibodies to h5n1 influenza a virus
ITTO20080204A1 (it) 2008-03-17 2009-09-18 Pomona Biotechnologies Llc Anticorpi monoclonali atti a reagire con una pluralita di sottotipi del virus influenzale a
ITTO20080398A1 (it) 2008-05-27 2009-11-28 Pomona Biotechnologies Llc Anticorpi monoclonali aventi proprieta' di cross-neutralizzazione omosubtipica per virus influenzali di tipo a sottotipo h1
WO2010010466A2 (en) 2008-07-25 2010-01-28 Institute For Research In Biomedicine Neutralizing anti-influenza a virus antibodies and uses thereof
GB0813784D0 (en) 2008-07-28 2008-09-03 Ct Integrated Photonics Ltd Optical intergration system
MX353900B (es) 2008-11-07 2018-02-01 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
CN105418757B (zh) 2009-05-11 2019-07-09 扬森疫苗与预防公司 能中和流感病毒h3n2的人结合分子及其应用
EP2772542B1 (en) * 2009-05-28 2016-12-28 Deutsches Krebsforschungszentrum Modified AAV capsid polypeptides
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
IT1395961B1 (it) 2009-06-01 2012-11-02 Pomona Biotechnologies Llc Anticorpi monoclonali come medicamento per il trattamento terapeutico e/o profilattico delle infezioni da virus influenzale a (h1n1) di origine suina (s-oiv)
SG10201502270TA (en) 2010-03-29 2015-05-28 Univ Pennsylvania Pharmacologically induced transgene ablation system
EP2558074B1 (en) 2010-04-08 2018-06-06 The Trustees of Princeton University Preparation of lipid nanoparticles
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
WO2012145572A1 (en) 2011-04-20 2012-10-26 The Trustees Of The University Of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
LT2714752T (lt) 2011-05-27 2018-03-12 Amicus Therapeutics, Inc. Nukreipiančių peptidų kopuliavimo ant rekombinantinių lizosominių fermentų būdai, skirti lizosominio kaupimo ligų gydymo pagerinimui
EP3586861B1 (en) 2011-06-08 2022-02-09 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mrna delivery
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
EA037448B1 (ru) 2012-06-08 2021-03-30 Этрис Гмбх Способ доставки в легкие терапевтической мрнк с pei и фармацевтическая композиция
EP2929035A1 (en) 2012-12-07 2015-10-14 Shire Human Genetic Therapies, Inc. Lipidic nanoparticles for mrna delivering
SG11201505936QA (en) 2013-03-15 2015-09-29 Amicus Therapeutics Inc Chemical crosslinkers
CN110003066B (zh) 2013-11-18 2021-09-03 阿克丘勒斯治疗公司 用于rna递送的可电离的阳离子脂质
WO2015164778A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennysylvania Ldlr variants and their use in compositions for reducing cholesterol levels
SG10201913259QA (en) 2014-09-24 2020-02-27 Hope City Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
US10640748B2 (en) * 2014-12-24 2020-05-05 Neuorphan Pty Ltd Oligodendroglial cell culturing methods and in methods for treating neurodegenerative disorders by using thyroid hormones or analogues
EP3307310A2 (en) 2015-05-13 2018-04-18 The Trustees Of The University Of Pennsylvania Aav-mediated expression of anti-influenza antibodies and methods of use thereof
MX2018006840A (es) 2015-12-11 2019-03-28 California Inst Of Techn Focalizacion de peptidos para dirigir virus adenoasociados.
AU2018227440B2 (en) 2017-02-28 2024-06-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clade f vector and uses therefor
EP3589649A1 (en) 2017-02-28 2020-01-08 The Trustees Of The University Of Pennsylvania Influenza vaccines based on aav vectors
EP3609542B1 (en) * 2017-04-11 2023-02-22 Ruprecht-Karls-Universität Heidelberg Adeno-associated virus library
SG11202008182TA (en) 2018-02-27 2020-09-29 Univ Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
US11867060B2 (en) 2019-02-04 2024-01-09 Technische Universität Wien Reinforced concrete tubbing segment
CN113853209B (zh) 2019-04-29 2024-12-13 宾夕法尼亚州大学信托人 新型aav衣壳和含有其的组合物

Also Published As

Publication number Publication date
TW202237850A (zh) 2022-10-01
IL303236A (en) 2023-07-01
JP2023551903A (ja) 2023-12-13
EP4256065A2 (en) 2023-10-11
KR20230117157A (ko) 2023-08-07
CN116670152A (zh) 2023-08-29
CA3200014A1 (en) 2022-06-09
US20240024507A1 (en) 2024-01-25
WO2022119871A2 (en) 2022-06-09
WO2022119871A3 (en) 2022-07-14
AR124216A1 (es) 2023-03-01
AU2021390480A1 (en) 2023-06-22
AU2021390480A9 (en) 2024-02-08

Similar Documents

Publication Publication Date Title
MX2023006444A (es) Composiciones novedosas con motivos dirigidos a tejido específico y composiciones que los contienen.
MX2023012513A (es) Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen.
IL152625A (en) Synthetic but not amyloidogenic immunogenic peptides equivalent to amyloid b to elicit an immune response to amyloid b and amyloid deposits
NZ333999A (en) GnRH-leukotoxin chimeric proteins for use in vaccines
AU5376201A (en) Methods and compositions for impairing multiplication of hiv-1
AU5848090A (en) Vector-mediated genomic insertion and expression of dna in bcg
JP7246323B2 (ja) Ｓ２又はｓ２’ポケット中の突然変異を有するサブチリシンバリアント
Shinkai et al. Amino acid sequence of cytochrome c 3 from Desulfovibrio vulgaris, Miyazaki
MX2023011194A (es) Peptido y composicion que contiene peptido.
Endermann et al. Major proteins of the Escherichia coli outer cell envelope membrane
WO2021239980A3 (en) A peptide cocktail
AU5776799A (en) Modified hcv peptide vaccines
ZA961349B (en) Amplifying sequences vectors comprising these sequences and their uses in compositions for the expression of nucleotide sequences in transfected cells therapeutic and vaccine applications
WO2024130067A3 (en) Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
MX2022011642A (es) Conjugados de ngr y usos de los mismos.
WO2021150713A3 (en) Human immunogenic epitopes of h, k, and e human endogenous retroviruses (hervs)
WO2002002148A3 (en) Dual recombinant gene therapy compositions and methods of use
WO2022235923A3 (en) Peptide-lipid conjugates
Yoshida et al. Primary structure of a regulating factor, 15 kDa protein, of ATP synthase in yeast mitochondria
PT1962889E (pt) Polipéptido imunogénico formado por péptidos crípticos optimizados provenientes de antigénios tumorais, e suas utilizações
NZ603379A (en) Ect2 peptides and vaccines including the same
SG170092A1 (en) Peptides of regulatory or accessory proteins of hiv, compositions and the utilization thereof
WO2009118710A3 (en) Cd4-related polypeptides and methods of use
WO2024130070A3 (en) Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
WO2023196871A3 (en) Secretion-optimized de novo designed protein nanoparticles for eukaryotic expression and genetic delivery