PL3233129T3 - Konstrukty kwasu nukleinowego i wektory terapii genowej do zastosowania w leczeniu choroby Wilsona i innych schorzeń - Google Patents

Konstrukty kwasu nukleinowego i wektory terapii genowej do zastosowania w leczeniu choroby Wilsona i innych schorzeń

Info

Publication number
PL3233129T3
PL3233129T3 PL15817239T PL15817239T PL3233129T3 PL 3233129 T3 PL3233129 T3 PL 3233129T3 PL 15817239 T PL15817239 T PL 15817239T PL 15817239 T PL15817239 T PL 15817239T PL 3233129 T3 PL3233129 T3 PL 3233129T3
Authority
PL
Poland
Prior art keywords
wilson
disease
treatment
conditions
nucleic acid
Prior art date
Application number
PL15817239T
Other languages
English (en)
Inventor
Oihana MURILLO SAUCA
Gloria GONZÁLEZ ASEGUINOLAZA
Rubén HERNÁNDEZ ALCOCEBA
Original Assignee
Fundacion Para La Investigacion Medica Aplicada
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 Fundacion Para La Investigacion Medica Aplicada filed Critical Fundacion Para La Investigacion Medica Aplicada
Publication of PL3233129T3 publication Critical patent/PL3233129T3/pl

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    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/03—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
    • C12Y306/03004—Cu2+-exporting ATPase (3.6.3.4)
    • 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
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • 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
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Psychiatry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
PL15817239T 2014-12-17 2015-12-17 Konstrukty kwasu nukleinowego i wektory terapii genowej do zastosowania w leczeniu choroby Wilsona i innych schorzeń PL3233129T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP14382530 2014-12-17
PCT/EP2015/080356 WO2016097218A1 (en) 2014-12-17 2015-12-17 Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson's disease and other conditions
EP15817239.5A EP3233129B1 (en) 2014-12-17 2015-12-17 Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson's disease and other conditions

Publications (1)

Publication Number Publication Date
PL3233129T3 true PL3233129T3 (pl) 2020-11-02

Family

ID=52278488

Family Applications (1)

Application Number Title Priority Date Filing Date
PL15817239T PL3233129T3 (pl) 2014-12-17 2015-12-17 Konstrukty kwasu nukleinowego i wektory terapii genowej do zastosowania w leczeniu choroby Wilsona i innych schorzeń

Country Status (10)

Country Link
US (1) US11118238B2 (pl)
EP (2) EP3233129B1 (pl)
JP (1) JP6956635B2 (pl)
DK (1) DK3233129T3 (pl)
ES (1) ES2784264T3 (pl)
HU (1) HUE048551T2 (pl)
PL (1) PL3233129T3 (pl)
PT (1) PT3233129T (pl)
SI (1) SI3233129T1 (pl)
WO (1) WO2016097218A1 (pl)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE048551T2 (hu) 2014-12-17 2020-08-28 Fundacion Para La Investig Medica Aplicada Nukleinsav-szerkezetek és génterápiás vektorok Wilson-kór és egyéb állapotok kezelésében történõ alkalmazásra
IL252917B2 (en) 2014-12-17 2023-10-01 Fundacion Para La Investig Medica Aplicada Vectors and vectors for gene therapy for use in the treatment of Wilson's disease
GB201522416D0 (en) 2015-12-18 2016-02-03 Ucl Business Plc Wilson's disease gene therapy
CA3048044A1 (en) * 2016-12-30 2018-07-05 The Trustees Of The University Of Pennsylvania Gene therapy for treating wilson's disease
EP3621637A1 (en) 2017-05-09 2020-03-18 Fundacion para la Investigacion Medica Aplicada Human porphobilinogen deaminase derived proteins and polynucleotides and uses thereof
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
WO2020005341A1 (en) 2018-06-29 2020-01-02 Icahn School Of Medicine At Mount Sinai Anc80 encoding sphingolipid-metabolizing proteins
TW202039854A (zh) * 2018-11-16 2020-11-01 美商編碼製藥公司 治療威爾遜氏病的組合物和方法
WO2021050064A1 (en) * 2019-09-11 2021-03-18 Icahn School Of Medicine At Mount Sinai Anc80 encoding sphingolipid-metabolizing proteins for mitigating disease-induced tissue damage
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
MX2023005218A (es) 2020-11-03 2023-05-16 Pfizer Metodos de purificacion de vectores de aav mediante cromatografia de intercambio anionico.
JP2024508324A (ja) 2021-03-04 2024-02-26 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Ｄｍｄエクソンの重複を修正するためのｃｒｉｓｐｒ－ｃａｓ９を使用したジストロフィンベースのミオパチーの治療のための生成物及び方法
WO2022208342A1 (en) 2021-04-01 2022-10-06 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
CN117925624B (zh) * 2024-03-22 2024-05-31 上海凌医生物科技有限公司 一种金属响应调控元件

Family Cites Families (21)

* 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
WO1992001070A1 (en) 1990-07-09 1992-01-23 The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce High efficiency packaging of mutant adeno-associated virus using amber suppressions
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
CA2115742A1 (en) 1991-08-20 1993-03-04 Ronald G. Crystal Adenovirus mediated transfer of genes to the gastrointestinal tract
JPH10146191A (ja) 1996-11-15 1998-06-02 Sumitomo Pharmaceut Co Ltd ウィルソン病治療用の組換えアデノウイルスベクター
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
EP1103610A1 (en) 1999-11-26 2001-05-30 Introgene B.V. Production of vaccines from immortalised mammalian cell lines
FR2808803B1 (fr) 2000-05-11 2004-12-10 Agronomique Inst Nat Rech Cellules es modifiees et gene specifique de cellules es
DE10156121A1 (de) * 2001-11-16 2003-05-28 Wolfgang Stremmel Somatische Gentherapie mit Vektoren, die einen verkürzten metallsensitiven Promotor besitzen, zur Behandlung des Morbus Wilson sowie anderer Erkrankungen, die mit Veränderungen im Metallstoffwechsel einhergehen, wie z.B. Alzheimer-Erkrankung
FR2836924B1 (fr) 2002-03-08 2005-01-14 Vivalis Lignees de cellules aviaires utiles pour la production de substances d'interet
EP1528101A1 (en) 2003-11-03 2005-05-04 ProBioGen AG Immortalized avian cell lines for virus production
FR2884255B1 (fr) 2005-04-11 2010-11-05 Vivalis Utilisation de lignees de cellules souches aviaires ebx pour la production de vaccin contre la grippe
EP2208786B1 (en) 2005-12-13 2018-08-01 Kyoto University Nuclear reprogramming factor
CN101743306A (zh) 2007-03-23 2010-06-16 威斯康星校友研究基金会 体细胞重编程
EP1985305A1 (en) 2007-04-24 2008-10-29 Vivalis Duck embryonic derived stem cell lines for the production of viral vaccines
EP1995309A1 (en) 2007-05-21 2008-11-26 Vivalis Recombinant protein production in avian EBx® cells
ES2337973B8 (es) 2008-05-16 2011-07-21 Proyecto De Biomedicina Cima, S.L. Adenovirus auxiliares auto-inactivantes para la produccion de adenovirus recombinantes de alta capacidad.
EP2394667A1 (en) * 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
WO2013151666A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of biologics and proteins associated with human disease
AU2014227766B2 (en) 2013-03-15 2018-10-04 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV vectors
HUE048551T2 (hu) 2014-12-17 2020-08-28 Fundacion Para La Investig Medica Aplicada Nukleinsav-szerkezetek és génterápiás vektorok Wilson-kór és egyéb állapotok kezelésében történõ alkalmazásra

Also Published As

Publication number Publication date
EP3744351A1 (en) 2020-12-02
SI3233129T1 (sl) 2020-07-31
HUE048551T2 (hu) 2020-08-28
US11118238B2 (en) 2021-09-14
JP6956635B2 (ja) 2021-11-02
EP3233129B1 (en) 2020-01-15
DK3233129T3 (da) 2020-04-06
JP2018505663A (ja) 2018-03-01
WO2016097218A1 (en) 2016-06-23
ES2784264T3 (es) 2020-09-23
US20170356060A1 (en) 2017-12-14
EP3233129A1 (en) 2017-10-25
PT3233129T (pt) 2020-04-15

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