SG11202107969SA - Compositions and methods for delivering cftr polypeptides - Google Patents

Compositions and methods for delivering cftr polypeptides

Info

Publication number
SG11202107969SA
SG11202107969SA SG11202107969SA SG11202107969SA SG11202107969SA SG 11202107969S A SG11202107969S A SG 11202107969SA SG 11202107969S A SG11202107969S A SG 11202107969SA SG 11202107969S A SG11202107969S A SG 11202107969SA SG 11202107969S A SG11202107969S A SG 11202107969SA
Authority
SG
Singapore
Prior art keywords
compositions
methods
cftr polypeptides
delivering
delivering cftr
Prior art date
Application number
SG11202107969SA
Inventor
Trevor Parry
Suma Krishnan
Pooja Agarwal
Original Assignee
Krystal Biotech 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 Krystal Biotech Inc filed Critical Krystal Biotech Inc
Publication of SG11202107969SA publication Critical patent/SG11202107969SA/en

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
    • 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
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712—Cystic fibrosis
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0075—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a dry powder inhaler [DPI], e.g. comprising micronized drug mixed with lactose carrier particles
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0078—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16041—Use of virus, viral particle or viral elements as a 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/16043—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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16071—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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16111—Cytomegalovirus, e.g. human herpesvirus 5
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16511—Roseolovirus, e.g. human herpesvirus 6, 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16621—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16632—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16641—Use of virus, viral particle or viral elements as a vector
    • C12N2710/16643—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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16671—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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16711—Varicellovirus, e.g. human herpesvirus 3, Varicella Zoster, pseudorabies

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)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Pulmonology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Otolaryngology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
SG11202107969SA 2019-02-08 2020-02-07 Compositions and methods for delivering cftr polypeptides SG11202107969SA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962802871P 2019-02-08 2019-02-08
PCT/US2020/017191 WO2020163703A1 (en) 2019-02-08 2020-02-07 Compositions and methods for delivering cftr polypeptides

Publications (1)

Publication Number Publication Date
SG11202107969SA true SG11202107969SA (en) 2021-08-30

Family

ID=69740894

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202107969SA SG11202107969SA (en) 2019-02-08 2020-02-07 Compositions and methods for delivering cftr polypeptides

Country Status (14)

Country Link
US (3) US10829529B2 (en)
EP (1) EP3921334A1 (en)
JP (1) JP2022519734A (en)
KR (1) KR20210124277A (en)
CN (1) CN113454105A (en)
AU (1) AU2020219343A1 (en)
BR (1) BR112021014400A2 (en)
CA (1) CA3127133A1 (en)
CL (1) CL2021002078A1 (en)
CO (1) CO2021009575A2 (en)
MX (1) MX2021008832A (en)
SG (1) SG11202107969SA (en)
WO (1) WO2020163703A1 (en)
ZA (1) ZA202205420B (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3921334A1 (en) * 2019-02-08 2021-12-15 Krystal Biotech, Inc. Compositions and methods for delivering cftr polypeptides
AU2022246917A1 (en) 2021-04-02 2023-10-12 Krystal Biotech, Inc. Viral vectors for cancer therapy
WO2024052734A2 (en) * 2022-07-13 2024-03-14 Intergalactic Therapeutics, Inc. Respiratory vectors and uses thereof

Family Cites Families (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE253633T1 (en) * 1989-08-22 2003-11-15 Hsc Res Dev Corp CYSTIC FIBROSIS GEN
US5658724A (en) 1992-07-31 1997-08-19 University Of Pittsburgh Of The Commonwealth System Of Higher Education Herpes simplex virus strains deficient for the essential immediate early genes ICP4 and ICP27 and methods for their production, growth and use
AU2283895A (en) * 1994-04-11 1995-10-30 Sloan-Kettering Institute For Cancer Research Defective herpes and defective adeno-associated virus vectors with p53 for the treatment of cancer
KR100201352B1 (en) 1995-03-16 1999-06-15 성재갑 Single shot vaccine formulation
GB9626029D0 (en) 1996-12-14 1997-01-29 Univ Leeds EVH-1 vectors
WO1998044938A1 (en) 1997-04-10 1998-10-15 University Of Southern California Modified proteins which bind extracellular matrix components
US5998174A (en) 1997-05-12 1999-12-07 University Of Pittsburgh Of The Commonwealth System Of Higher Education Multigene vectors
GB9715085D0 (en) 1997-07-18 1997-09-24 Jahoda Amanda J Gene therapy vehicle
US6106826A (en) 1997-12-17 2000-08-22 Wisconsin Alumni Research Foundation Replication competent, avirulent Herpes simplex virus as a vector for neural and ocular gene therapy
GB9801930D0 (en) 1998-01-29 1998-03-25 Univ London Mutant herpes simplex viruses and uses thereof
WO1999064094A1 (en) 1998-06-12 1999-12-16 Aradigm Corporation Methods of delivering aerosolized polynucleotides to the respiratory tract
US7244714B1 (en) * 1998-06-12 2007-07-17 Aradigm Corporation Methods of delivering aerosolized polynucleotides to the respiratory tract
JP2003535565A (en) 1998-12-31 2003-12-02 アーチ ディベロプメント コーポレイション Recombinant herpes simplex virus useful for the treatment of neoplastic diseases
GB9930418D0 (en) 1999-12-22 2000-02-16 Neurovex Ltd Replication incompetent herpes virus vectors
WO2002045431A2 (en) 2000-11-28 2002-06-06 The University Of Chicago Genetically engineered herpes virus for the treatment of cardiovascular disease
US7081483B2 (en) 2001-07-03 2006-07-25 The University Of Chicago Polyfunctional dispersants for controlling viscosity of phyllosilicates
WO2004064751A2 (en) 2003-01-16 2004-08-05 St. Johns University New York Nanoparticle based stabilization of ir fluorescent dyes
WO2006052285A2 (en) 2004-05-13 2006-05-18 The Trustees Of Columbia University In The City Of New York Polymeric nanoparticles and nanogels for extraction and release of compounds
US7731952B2 (en) * 2004-06-24 2010-06-08 New York University Avirulent oncolytic herpes simplex virus strains engineered to counter the innate host response
US7342048B2 (en) 2005-04-28 2008-03-11 Nipro Corporation Bioabsorbable pharmaceutical formulation
US20070092575A1 (en) 2005-05-10 2007-04-26 Naomi Balaban Compositions for administering RNAIII-inhibiting peptides
CA2615690A1 (en) * 2005-06-03 2006-12-21 University Of Rochester Herpes virus-based compositions and methods of use in the prenatal and perinatal periods
US20080066739A1 (en) * 2006-09-20 2008-03-20 Lemahieu Edward Methods and systems of delivering medication via inhalation
US8703703B2 (en) 2006-12-20 2014-04-22 University Of Central Flordia Research Foundation, Inc. MCPIP as wound therapy
WO2008106646A2 (en) 2007-03-01 2008-09-04 Introgen Therapeutics, Inc Methods and formulations for topical gene therapy
WO2009148488A2 (en) * 2008-05-29 2009-12-10 The General Hospital Corporation Use of oncolytic herpes viruses for killing cancer stem cells
WO2012109363A2 (en) * 2011-02-08 2012-08-16 The Johns Hopkins University Mucus penetrating gene carriers
CN102212559B (en) 2011-04-14 2014-04-09 郑州威瑞生物技术有限公司 Recombinant HSV (Herpes Simplex Virus) amplicon vector and application thereof
EP3279324B1 (en) 2011-10-11 2021-02-17 Institut National De La Sante Et De La Recherche Medicale (Inserm) Exon skipping therapy for dystrophic epidermolysis bullosa
GB201202561D0 (en) 2012-02-15 2012-03-28 Univ Dundee Treatment of skin disorders
EP2849774A4 (en) 2012-05-18 2015-11-25 Otago Innovation Ltd Combination treatments and compositions for wound healing
US9393257B2 (en) 2013-03-01 2016-07-19 Regents Of The University Of Minnesota TALEN-based gene correction
LT2968586T (en) * 2013-03-14 2018-11-26 Translate Bio, Inc. Cftr mrna compositions and related methods and uses
KR102649583B1 (en) 2013-07-17 2024-03-20 유니버시티 오브 피츠버그-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Non-toxic HSV vectors for efficient gene delivery applications and complementing cells for their production
MX2016009771A (en) 2014-01-31 2016-11-14 Factor Bioscience Inc Methods and products for nucleic acid production and delivery.
US9840709B2 (en) 2015-02-20 2017-12-12 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting genes associated with cystic fibrosis
EP3432912B1 (en) 2016-03-25 2022-12-21 PeriphaGen, Inc. High-transducing hsv vectors
MX2018012135A (en) 2016-04-08 2019-03-28 Krystal Biotech Inc Compositions and methods for the treatment of wounds, disorders, and diseases of the skin.
JP2019514840A (en) 2016-04-26 2019-06-06 アッヴィ・エス・ア・エール・エル Modulator of cystic fibrosis transmembrane conductance regulator protein
EP3458108A4 (en) 2016-05-18 2020-04-22 ModernaTX, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
WO2019200163A1 (en) 2018-04-12 2019-10-17 Krystal Biotech, Inc. Compositions and methods for the treatment of autosomal recessive congenital ichthyosis
WO2019210219A1 (en) 2018-04-27 2019-10-31 Krystal Biotech, Inc. Recombinant nucleic acids encoding cosmetic protein(s) for aesthetic applications
CA3103374A1 (en) 2018-06-29 2020-01-02 Krystal Biotech, Inc. Compositions and methods for antibody delivery
EP3856761A1 (en) 2018-09-24 2021-08-04 Krystal Biotech, Inc. Compositions and methods for the treatment of netherton syndrome
EP3921334A1 (en) * 2019-02-08 2021-12-15 Krystal Biotech, Inc. Compositions and methods for delivering cftr polypeptides

Also Published As

Publication number Publication date
MX2021008832A (en) 2021-09-08
KR20210124277A (en) 2021-10-14
US20200254060A1 (en) 2020-08-13
US10829529B2 (en) 2020-11-10
US20210087245A1 (en) 2021-03-25
BR112021014400A2 (en) 2021-09-21
AU2020219343A1 (en) 2021-08-19
CO2021009575A2 (en) 2021-08-09
JP2022519734A (en) 2022-03-24
EP3921334A1 (en) 2021-12-15
CN113454105A (en) 2021-09-28
ZA202205420B (en) 2022-09-28
US20210317174A1 (en) 2021-10-14
WO2020163703A1 (en) 2020-08-13
CA3127133A1 (en) 2020-08-13
CL2021002078A1 (en) 2022-02-04

Similar Documents

Publication Publication Date Title
IL277079A (en) Cartyrin compositions and methods for use
IL273823A (en) Methods and compositions for topical delivery
EP3704219C0 (en) Polypeptides and compositions comprising such polypeptides
IL280134A (en) Anti-cd112r compositions and methods
IL283177A (en) Compositions and methods for compartment-specific cargo delivery
IL278081A (en) Methods and compositions for oilseed materials
IL282238A (en) Compositions and methods for expressing factor ix
EP3866924A4 (en) Compositions and methods for antibody delivery
ZA202205420B (en) Compositions and methods for delivering cftr polypeptides
GB202017929D0 (en) Compositions and methods for gene editing
EP3474857A4 (en) Compositions and methods for the delivery of therapeutics
IL282369A (en) Compositions and methods for delivering transgenes
EP3946547A4 (en) Devices and methods for delivering pharmaceutical compositions
IL287781A (en) Clec12a-binding polypeptides and uses thereof
EP3853204A4 (en) Improved and stable apremilast pharmaceutical compositions
IL287782A (en) Cd33-binding polypeptides and uses thereof
SG11202104448WA (en) Compositions and methods
IL274524A (en) Compositions and methods for aquaculturing
IL288024A (en) Methods and compositions for preventing type
IL290641A (en) Compositions and particles for payload delivery
EP3773654C0 (en) Polypharmaceutical drug compositions and related methods
IL287262A (en) Methods and compositions for transgene expression
GB201817444D0 (en) Methods and compositions
GB2603645B (en) Polypeptides having anti-senescent effects and uses thereof
IL279877A (en) Ivosidenib forms and pharmaceutical compositions