SG11201707116QA - Recombinant glut1 adeno-associated viral vector constructs and related methods for restoring glut1 expression - Google Patents

Recombinant glut1 adeno-associated viral vector constructs and related methods for restoring glut1 expression

Info

Publication number
SG11201707116QA
SG11201707116QA SG11201707116QA SG11201707116QA SG11201707116QA SG 11201707116Q A SG11201707116Q A SG 11201707116QA SG 11201707116Q A SG11201707116Q A SG 11201707116QA SG 11201707116Q A SG11201707116Q A SG 11201707116QA SG 11201707116Q A SG11201707116Q A SG 11201707116QA
Authority
SG
Singapore
Prior art keywords
glut1
adeno
viral vector
recombinant
related methods
Prior art date
Application number
SG11201707116QA
Inventor
Vivo Darryl De
Umrao Monani
Guangping Gao
Kristin Engelstad
Original Assignee
Univ Columbia
Univ Of Massachusetts Medical Center
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 Columbia, Univ Of Massachusetts Medical Center filed Critical Univ Columbia
Publication of SG11201707116QA publication Critical patent/SG11201707116QA/en

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Classifications

    • 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
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
SG11201707116QA 2015-03-10 2016-03-10 Recombinant glut1 adeno-associated viral vector constructs and related methods for restoring glut1 expression SG11201707116QA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562130899P 2015-03-10 2015-03-10
PCT/US2016/021810 WO2016145217A1 (en) 2015-03-10 2016-03-10 Recombinant glut1 adeno-associated viral vector constructs and related methods for restoring glut1 expression

Publications (1)

Publication Number Publication Date
SG11201707116QA true SG11201707116QA (en) 2017-09-28

Family

ID=56879055

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201707116QA SG11201707116QA (en) 2015-03-10 2016-03-10 Recombinant glut1 adeno-associated viral vector constructs and related methods for restoring glut1 expression

Country Status (19)

Country Link
US (2) US20180042991A1 (en)
EP (1) EP3268024B1 (en)
JP (3) JP2018509164A (en)
KR (1) KR20180016722A (en)
CN (1) CN107635575A (en)
AU (1) AU2016229000B2 (en)
BR (1) BR112017019294A2 (en)
CA (1) CA2978917A1 (en)
CL (1) CL2017002282A1 (en)
CO (1) CO2017010149A2 (en)
DK (1) DK3268024T3 (en)
EA (1) EA036051B1 (en)
ES (1) ES2836258T3 (en)
HU (1) HUE052577T2 (en)
IL (2) IL282053B2 (en)
MX (2) MX2017011615A (en)
PT (1) PT3268024T (en)
SG (1) SG11201707116QA (en)
WO (1) WO2016145217A1 (en)

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JP6401871B2 (en) 2014-11-05 2018-10-10 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． AADC polynucleotide for the treatment of Parkinson's disease
JP6996728B2 (en) * 2016-01-07 2022-01-17 学校法人自治医科大学 Adeno-associated virus vector for glucose transporter 1 expression
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
SG11202009914SA (en) * 2018-05-08 2020-11-27 Rutgers The State University Of New Jersey Aav-compatible laminin-linker polymerization proteins
AU2019346655A1 (en) * 2018-09-28 2021-05-06 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
KR20220002609A (en) 2019-05-13 2022-01-06 디앤에이 투포인토 인크. Modification of Mammalian Cells Using Artificial Micro-RNAs and Compositions of These Products to Alter Properties of Mammalian Cells
KR20220079857A (en) * 2019-09-13 2022-06-14 루트거스, 더 스테이트 유니버시티 오브 뉴 저지 AAV-compatible laminin-linker polymeric protein
IL297741A (en) * 2020-05-01 2022-12-01 Atlas Biotechnology S A Treatment and/or prevention of a disease or a syndrome related to a virus infection
US20230248824A1 (en) * 2020-07-09 2023-08-10 The Board of Trutees of the Leland Stanford Junior University Immune cells with increased glycolytic flux
EP4192960A1 (en) * 2020-08-05 2023-06-14 Spacecraft Seven, LLC Adeno-associated viral vector for glut1 expression and uses thereof
IL300263A (en) 2020-08-07 2023-03-01 Spacecraft Seven Llc Plakophilin-2 (pkp2) gene therapy using aav vector
US20230313187A1 (en) * 2020-09-04 2023-10-05 Dna Twopointo Inc. Modifications of mammalian cells using artificial micro-rna to alter their properties and the compositions of their products
WO2023154763A2 (en) * 2022-02-08 2023-08-17 Spacecraft Seven, Llc Adeno-associated viral vector for glut1 expression and uses thereof

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US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
IL128780A0 (en) 1996-09-06 2000-01-31 Univ Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
ES2258601T3 (en) 2001-11-13 2006-09-01 The Trustees Of The University Of Pennsylvania A METHOD FOR THE IDENTIFICATION OF THE UNKNOWN SEQUENCES OF ADENO-ASSOCIATED VIRUSES (VAA) AND A KIT FOR THE METHOD.
EP1648926B1 (en) * 2003-05-02 2013-07-03 Centre National De La Recherche Scientifique (Cnrs) Glut-1 as a receptor for htlv envelopes and its uses
CN1856576B (en) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
EP2359865B1 (en) 2005-04-07 2013-10-02 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
WO2008134632A1 (en) * 2007-04-26 2008-11-06 President And Fellows Of Harvard College Wnt ligands involved in blood-brain barrier development and uses therefor
WO2010071832A1 (en) * 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
WO2010121010A2 (en) 2009-04-16 2010-10-21 President And Fellows Of Harvard College Methods for inhibiting starvation of a cell
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EP2394667A1 (en) * 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases

Also Published As

Publication number Publication date
EA201791936A8 (en) 2018-11-30
CN107635575A (en) 2018-01-26
EP3268024A1 (en) 2018-01-17
JP2021097689A (en) 2021-07-01
IL254286B (en) 2021-04-29
JP2018509164A (en) 2018-04-05
PT3268024T (en) 2020-12-09
JP2023144087A (en) 2023-10-06
AU2016229000B2 (en) 2021-11-04
US20180042991A1 (en) 2018-02-15
MX2017011615A (en) 2018-04-11
AU2016229000A1 (en) 2017-09-21
DK3268024T3 (en) 2020-12-07
IL254286A0 (en) 2017-10-31
KR20180016722A (en) 2018-02-19
CA2978917A1 (en) 2016-09-15
CO2017010149A2 (en) 2018-02-28
EP3268024B1 (en) 2020-09-09
US20210069292A1 (en) 2021-03-11
EA201791936A1 (en) 2018-01-31
EP3268024A4 (en) 2018-08-01
EA036051B1 (en) 2020-09-18
ES2836258T3 (en) 2021-06-24
WO2016145217A1 (en) 2016-09-15
CL2017002282A1 (en) 2018-04-13
IL282053A (en) 2021-05-31
HUE052577T2 (en) 2021-05-28
IL282053B (en) 2022-11-01
IL282053B2 (en) 2023-03-01
MX2023001213A (en) 2023-03-03
BR112017019294A2 (en) 2018-05-02

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