MX2022002132A - Administracion de vectores de virus adenoasociado de alfa-sarcoglicano y el tratamiento de la distrofia muscular. - Google Patents

Administracion de vectores de virus adenoasociado de alfa-sarcoglicano y el tratamiento de la distrofia muscular.

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Publication number
MX2022002132A
MX2022002132A MX2022002132A MX2022002132A MX2022002132A MX 2022002132 A MX2022002132 A MX 2022002132A MX 2022002132 A MX2022002132 A MX 2022002132A MX 2022002132 A MX2022002132 A MX 2022002132A MX 2022002132 A MX2022002132 A MX 2022002132A
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MX
Mexico
Prior art keywords
sarcoglycan
alpha
sup
muscular dystrophy
adeno
Prior art date
Application number
MX2022002132A
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English (en)
Inventor
Jerry R Mendell
Louise Rodino-Klapac
Danielle Griffin
Original Assignee
Res Inst Nationwide Childrens Hospital
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Filing date
Publication date
Application filed by Res Inst Nationwide Childrens Hospital filed Critical Res Inst Nationwide Childrens Hospital
Publication of MX2022002132A publication Critical patent/MX2022002132A/es

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    • 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
    • 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
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

En la presente se describen métodos para tratar la distrofia muscular en un sujeto, que comprenden la administración de un vector de AAV recombinante AAVrh74.tMCK.hSCGA mediante una vía de administración sistémica y a una dosis de alrededor de 1,0 x 1012 vg/kg a alrededor de 5,0 x 1015 vg/kg; se divulgan además métodos para expresar el gen de alfa-sarcoglicano en una célula o en un sujeto que lo necesite, disminuir el nivel de CK en suero y aumentar las fibras positivas en alfasarcoglicano en el tejido muscular de un sujeto.
MX2022002132A 2019-08-21 2020-08-21 Administracion de vectores de virus adenoasociado de alfa-sarcoglicano y el tratamiento de la distrofia muscular. MX2022002132A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962889749P 2019-08-21 2019-08-21
US202063014934P 2020-04-24 2020-04-24
US202063022843P 2020-05-11 2020-05-11
PCT/US2020/047339 WO2021035120A1 (en) 2019-08-21 2020-08-21 Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy

Publications (1)

Publication Number Publication Date
MX2022002132A true MX2022002132A (es) 2022-05-18

Family

ID=72340432

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022002132A MX2022002132A (es) 2019-08-21 2020-08-21 Administracion de vectores de virus adenoasociado de alfa-sarcoglicano y el tratamiento de la distrofia muscular.

Country Status (18)

Country Link
US (1) US20220290180A1 (es)
EP (2) EP4374880A2 (es)
JP (1) JP2022544627A (es)
KR (1) KR20220092489A (es)
CN (1) CN114945583A (es)
AU (1) AU2020331987A1 (es)
BR (1) BR112022003142A2 (es)
CA (1) CA3150964A1 (es)
CO (1) CO2022002864A2 (es)
DK (1) DK4017871T3 (es)
FI (1) FI4017871T3 (es)
IL (1) IL290734A (es)
LT (1) LT4017871T (es)
MX (1) MX2022002132A (es)
PT (1) PT4017871T (es)
RS (1) RS65421B1 (es)
TW (1) TW202120532A (es)
WO (1) WO2021035120A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4198046A1 (en) 2021-12-16 2023-06-21 Genethon Alpha-sarcoglycan gene transfer increase using modified itr sequences

Family Cites Families (23)

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US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
DK0733103T3 (da) 1993-11-09 2004-07-12 Univ Johns Hopkins Fremstilling af höje titre af rekombinante AAV vektorer
WO1995013392A1 (en) 1993-11-09 1995-05-18 Medical College Of Ohio Stable cell lines capable of expressing the adeno-associated virus replication gene
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
JPH10511264A (ja) 1994-12-06 1998-11-04 ターゲティッド ジェネティックス コーポレイション 高力価組換えａａｖベクターの生成のためのパッケージング細胞株
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
EP0847442A1 (en) 1995-08-30 1998-06-17 Genzyme Corporation Chromatographic purification of adenovirus and aav
JPH11514853A (ja) 1995-09-08 1999-12-21 ジエンザイム コーポレイション 遺伝子治療のための改良されたａａｖベクター
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ATE380037T1 (de) 1996-09-06 2007-12-15 Univ Pennsylvania Verfaheren zur durch rekombinante adeno- assoziierte virus-gerichtete gentherapie
CA2995542A1 (en) 1997-09-05 1999-03-11 Genzyme Corporation Methods for generating high titer helper-free preparations of recombinant aav vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
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AU2001255575B2 (en) 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
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DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
WO2015141521A1 (ja) 2014-03-21 2015-09-24 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及び記録媒体
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CN109121395B (zh) * 2016-04-15 2022-08-09 全国儿童医院研究所 腺相关病毒载体递送β-肌聚糖和微RNA-29以及肌营养不良症的治疗

Also Published As

Publication number Publication date
KR20220092489A (ko) 2022-07-01
CA3150964A1 (en) 2021-02-25
WO2021035120A1 (en) 2021-02-25
CO2022002864A2 (es) 2022-07-08
LT4017871T (lt) 2024-05-10
CN114945583A (zh) 2022-08-26
EP4017871A1 (en) 2022-06-29
AU2020331987A1 (en) 2022-03-31
RS65421B1 (sr) 2024-05-31
EP4374880A2 (en) 2024-05-29
JP2022544627A (ja) 2022-10-19
PT4017871T (pt) 2024-04-22
IL290734A (en) 2022-04-01
BR112022003142A2 (pt) 2022-05-17
US20220290180A1 (en) 2022-09-15
EP4017871B1 (en) 2024-01-17
TW202120532A (zh) 2021-06-01
FI4017871T3 (fi) 2024-04-22
DK4017871T3 (da) 2024-04-22

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