PE20211859A1 - Composiciones y metodos para aumentar o mejorar la transduccion de vectores de terapia genetica y para eliminar o reducir las inmunoglobulinas - Google Patents

Composiciones y metodos para aumentar o mejorar la transduccion de vectores de terapia genetica y para eliminar o reducir las inmunoglobulinas

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Publication number
PE20211859A1
PE20211859A1 PE2021000066A PE2021000066A PE20211859A1 PE 20211859 A1 PE20211859 A1 PE 20211859A1 PE 2021000066 A PE2021000066 A PE 2021000066A PE 2021000066 A PE2021000066 A PE 2021000066A PE 20211859 A1 PE20211859 A1 PE 20211859A1
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PE
Peru
Prior art keywords
vector
enzymes
recombinant aav
aav vector
immunoglobulins
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PE2021000066A
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English (en)
Inventor
Sebastien Lacroix-Desmazes
Federico Mingozzi
Jordan Dimitrov
Christian Leborgne
Sean Armour
Original Assignee
Inst Nat Sante Rech Med
Genethon
Univ Sorbonne
Univ Paris
Spark Therapeutics Inc
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Application filed by Inst Nat Sante Rech Med, Genethon, Univ Sorbonne, Univ Paris, Spark Therapeutics Inc filed Critical Inst Nat Sante Rech Med
Publication of PE20211859A1 publication Critical patent/PE20211859A1/es

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    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A—HUMAN NECESSITIES
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    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4873—Cysteine endopeptidases (3.4.22), e.g. stem bromelain, papain, ficin, cathepsin H
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K16/06—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies from serum
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
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    • C12N9/6472—Cysteine endopeptidases (3.4.22)
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Abstract

La presente invencion se refiere a enzimas polipeptidicas que degradan inmunoglobulina G, las cuales comprenden las secuencias de cualquiera de las SEQ ID NO: 3-18, 23 o 48; dichas enzimas son utiles en la prevencion o el tratamiento de una respuesta inmune humoral causada por la administracion de un vector de virus adenoasociado (AAV) recombinante, en donde la respuesta inmune humoral es contra el vector de AAV recombinante y/o un polinucleotido heterologo o una proteina o peptido codificado por un polinucleotido heterologo encapsidado por el vector de AAV recombinante. Tambien se refiere a secuencias de acido nucleico, vector de expresion y un kit que comprende (a) el vector AAV recombinante y (b) la enzima polipeptidica que degrada inmunoglobulina G. Las enzimas de la invencion se aplican en el tratamiento de pacientes que pueden desarrollar o que ya tienen anticuerpos neutralizantes preexistentes de terapia genica de enfermedades tratadas por vector AAV recombinante.
PE2021000066A 2018-07-17 2019-07-17 Composiciones y metodos para aumentar o mejorar la transduccion de vectores de terapia genetica y para eliminar o reducir las inmunoglobulinas PE20211859A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP18305971 2018-07-17
US201862768731P 2018-11-16 2018-11-16
PCT/EP2019/069280 WO2020016318A1 (en) 2018-07-17 2019-07-17 Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins

Publications (1)

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PE20211859A1 true PE20211859A1 (es) 2021-09-21

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ID=67303476

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PE2021000066A PE20211859A1 (es) 2018-07-17 2019-07-17 Composiciones y metodos para aumentar o mejorar la transduccion de vectores de terapia genetica y para eliminar o reducir las inmunoglobulinas

Country Status (26)

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US (3) US20210228738A1 (es)
EP (2) EP4154904A1 (es)
JP (2) JP2021529821A (es)
KR (1) KR20210042308A (es)
CN (1) CN112689515A (es)
AU (1) AU2019306121A1 (es)
BR (1) BR112021000807A8 (es)
CA (1) CA3106368A1 (es)
CL (1) CL2021000112A1 (es)
CO (1) CO2021000326A2 (es)
DK (1) DK3768304T3 (es)
ES (1) ES2935970T3 (es)
FI (1) FI3768304T3 (es)
HR (1) HRP20221542T1 (es)
HU (1) HUE060720T2 (es)
IL (1) IL280099A (es)
LT (1) LT3768304T (es)
MX (1) MX2021000580A (es)
PE (1) PE20211859A1 (es)
PH (1) PH12021550115A1 (es)
PL (1) PL3768304T3 (es)
RS (1) RS63878B1 (es)
SG (1) SG11202100306VA (es)
SI (1) SI3768304T1 (es)
WO (1) WO2020016318A1 (es)
ZA (1) ZA202100202B (es)

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