CL2021002078A1 - Composiciones y métodos para la administración de polipéptidos cftr. - Google Patents

Composiciones y métodos para la administración de polipéptidos cftr.

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Publication number
CL2021002078A1
CL2021002078A1 CL2021002078A CL2021002078A CL2021002078A1 CL 2021002078 A1 CL2021002078 A1 CL 2021002078A1 CL 2021002078 A CL2021002078 A CL 2021002078A CL 2021002078 A CL2021002078 A CL 2021002078A CL 2021002078 A1 CL2021002078 A1 CL 2021002078A1
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CL
Chile
Prior art keywords
compositions
methods
nucleic acids
cftr
administration
Prior art date
Application number
CL2021002078A
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English (en)
Inventor
Suma Krishnan
Pooja Agarwal
Trevor Parry
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Krystal Biotech Inc
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Publication date
Application filed by Krystal Biotech Inc filed Critical Krystal Biotech Inc
Publication of CL2021002078A1 publication Critical patent/CL2021002078A1/es

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    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712Cystic fibrosis
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    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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Abstract

La presente divulgación proporciona ácidos nucleicos recombinantes que comprenden uno o más polinucleótidos que codifican un polipéptido del regulador de conductancia transmembrana de fibrosis quística (CFTR) (por ejemplo, un polinucleótido CFTR humano); virus que comprenden los ácidos nucleicos recombinantes; composiciones y formulaciones que comprenden los virus y/o ácidos nucleicos recombinantes; métodos para utilizarlos (por ejemplo, para el tratamiento de una enfermedad pulmonar crónica, tal como fibrosis quística); y artículos de elaboración o kits de estos.
CL2021002078A 2019-02-08 2021-08-05 Composiciones y métodos para la administración de polipéptidos cftr. CL2021002078A1 (es)

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

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2020219343A1 (en) * 2019-02-08 2021-08-19 Krystal Biotech, Inc. Compositions and methods for delivering CFTR polypeptides
BR112023020209A2 (pt) 2021-04-02 2023-12-19 Krystal Biotech Inc Genoma do vírus do herpes recombinante, vírus do herpes, composição farmacêutica, uso do vírus do herpes ou da composição farmacêutica, método para expressar, realçar, aumentar, ampliar e/ou suplementar os níveis de um polipeptídeo imunomodulatório, método para prover alívio profilático, paliativo ou terapêutico, e, método para tratar câncer
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
CA2066204C (en) * 1989-08-22 2009-03-31 Lap-Chee Tsui Cystic fibrosis gene
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 (ko) 1995-03-16 1999-06-15 성재갑 단일주사 백신 제형
GB9626029D0 (en) * 1996-12-14 1997-01-29 Univ Leeds EVH-1 vectors
ATE388720T1 (de) 1997-04-10 2008-03-15 Univ Southern California Sich an extrazelluläre matrixbestandteile anbindende modifizierte proteine
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
US7244714B1 (en) * 1998-06-12 2007-07-17 Aradigm Corporation Methods of delivering aerosolized polynucleotides to the respiratory tract
EP1083951A4 (en) 1998-06-12 2004-07-07 Aradigm Corp METHOD FOR DISCHARGING POLYNUCLEOTIDS IN THE FORM OF AN AEROSOL IN THE RESPIRATION WAY
CA2356937A1 (en) 1998-12-31 2000-07-13 Richard J. Whitley Recombinant herpes simplex virus useful for treating neoplastic disease
GB9930418D0 (en) 1999-12-22 2000-02-16 Neurovex Ltd Replication incompetent herpes virus vectors
CA2430341A1 (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
EP1583473A2 (en) 2003-01-16 2005-10-12 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
WO2006002394A2 (en) * 2004-06-24 2006-01-05 New York University Avirulent oncolytic herpes simplex virus strains engineered to counter the innate host response
DE602006000381T2 (de) 2005-04-28 2008-12-18 Nipro Corp., Osaka Bioabsorbierbare pharmazeutische Zusammensetzung enthaltend einen PLGA-Copolymer
JP2008540544A (ja) 2005-05-10 2008-11-20 バラバン,ナオミ Rnaiii阻害ペプチドを投与するための組成物
AU2006258099A1 (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
US8703120B2 (en) * 2008-05-29 2014-04-22 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 (zh) 2011-04-14 2014-04-09 郑州威瑞生物技术有限公司 一种重组的hsv扩增子载体及其用途
US9340783B2 (en) 2011-10-11 2016-05-17 Inserm (Institut National De La Sante Et De La Recherche Medicale Exon skipping therapy for dystrophic epidermolysis bullosa
GB201202561D0 (en) 2012-02-15 2012-03-28 Univ Dundee Treatment of skin disorders
CA2873881A1 (en) 2012-05-18 2013-11-21 Stephen Bruce Fleming Combination treatments and compositions for wound healing
JP6480874B2 (ja) 2013-03-01 2019-03-13 リージェンツ・オブ・ザ・ユニバーシティ・オブ・ミネソタRegents Of The University Of Minnesota Talenに基づく遺伝子修正
PL2968586T3 (pl) * 2013-03-14 2019-01-31 Translate Bio, Inc. Kompozycje mrna cftr i związne z nimi sposoby i zastosowania
US10174341B2 (en) 2013-07-17 2019-01-08 University of Pittsburgh—of the Commonwealth System of Higher Education Non-toxic HSV vectors for efficient gene delivery applications and complementing cells for their production
KR20220100078A (ko) 2014-01-31 2022-07-14 팩터 바이오사이언스 인크. 핵산 제조 및 송달을 위한 방법 및 산물
WO2016134021A1 (en) 2015-02-20 2016-08-25 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting genes associated with cystic fibrosis
DK3432912T3 (da) 2016-03-25 2023-03-13 Periphagen Inc Hsv-vektorer med høj transduktion
PL3377637T3 (pl) 2016-04-08 2020-09-07 Krystal Biotech, Inc. Kompozycje do stosowania w sposobach leczenia ran, zaburzeń i chorób skóry
EP3448842A1 (en) 2016-04-26 2019-03-06 AbbVie S.À.R.L. Modulators of cystic fibrosis transmembrane conductance regulator protein
WO2017201347A1 (en) 2016-05-18 2017-11-23 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
KR20210005141A (ko) 2018-04-27 2021-01-13 크리스탈 바이오테크, 인크. 미적 적용을 위한 미용 단백질(들)을 인코딩하는 재조합 핵산
AU2019292580A1 (en) 2018-06-29 2021-02-04 Krystal Biotech, Inc. Compositions and methods for antibody delivery
WO2020068862A1 (en) 2018-09-24 2020-04-02 Krystal Biotech, Inc. Compositions and methods for the treatment of netherton syndrome
AU2020219343A1 (en) * 2019-02-08 2021-08-19 Krystal Biotech, Inc. Compositions and methods for delivering CFTR polypeptides

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