CO2022002864A2 - Administración de vectores de virus adenoasociado de alfa-sarcoglicano y el tratamiento de la distrofia muscular - Google Patents
Administración de vectores de virus adenoasociado de alfa-sarcoglicano y el tratamiento de la distrofia muscularInfo
- Publication number
- CO2022002864A2 CO2022002864A2 CONC2022/0002864A CO2022002864A CO2022002864A2 CO 2022002864 A2 CO2022002864 A2 CO 2022002864A2 CO 2022002864 A CO2022002864 A CO 2022002864A CO 2022002864 A2 CO2022002864 A2 CO 2022002864A2
- Authority
- CO
- Colombia
- Prior art keywords
- sarcoglycan
- alpha
- administration
- muscular dystrophy
- adeno
- Prior art date
Links
- 108010083379 Sarcoglycans Proteins 0.000 title abstract 3
- 102000016579 Alpha-sarcoglycan Human genes 0.000 title abstract 2
- 201000006938 muscular dystrophy Diseases 0.000 title abstract 2
- 241000702421 Dependoparvovirus Species 0.000 title 1
- 239000013598 vector Substances 0.000 title 1
- 239000013607 AAV vector Substances 0.000 abstract 1
- 239000000835 fiber Substances 0.000 abstract 1
- 210000003205 muscle Anatomy 0.000 abstract 1
- 210000002966 serum Anatomy 0.000 abstract 1
- 230000009885 systemic effect Effects 0.000 abstract 1
Classifications
-
- 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
- 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
-
- 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
- C07K14/46—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
-
- 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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Cell Biology (AREA)
- Immunology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (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 alfa-sarcoglicano en el tejido muscular de un sujeto.
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 |
---|---|
CO2022002864A2 true CO2022002864A2 (es) | 2022-07-08 |
Family
ID=72340432
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2022/0002864A CO2022002864A2 (es) | 2019-08-21 | 2022-03-11 | Administración de vectores de virus adenoasociado de alfa-sarcoglicano y el tratamiento de la distrofia muscular |
Country Status (20)
Country | Link |
---|---|
US (1) | US20220290180A1 (es) |
EP (2) | EP4017871B1 (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) |
HR (1) | HRP20240505T1 (es) |
IL (1) | IL290734A (es) |
LT (1) | LT4017871T (es) |
MX (1) | MX2022002132A (es) |
PT (1) | PT4017871T (es) |
RS (1) | RS65421B1 (es) |
SI (1) | SI4017871T1 (es) |
TW (1) | TW202120532A (es) |
WO (1) | WO2021035120A1 (es) |
Families Citing this family (1)
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)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
EP0733103B1 (en) | 1993-11-09 | 2004-03-03 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
US5837484A (en) | 1993-11-09 | 1998-11-17 | 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 |
WO1996017947A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
AU722196B2 (en) | 1995-08-30 | 2000-07-27 | Genzyme Corporation | Chromatographic purification of adenovirus and AAV |
AU715543B2 (en) | 1995-09-08 | 2000-02-03 | Genzyme Corporation | Improved AAV vectors for gene therapy |
US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
EP1696036B1 (en) | 1996-09-06 | 2010-04-21 | The Trustees of The University of Pennsylvania | Use of recombinant adeno-associated virus in the manufacture of a medicament for gene therapy via muscle cells |
EP2325299A3 (en) | 1997-09-05 | 2011-10-05 | Targeted Genetics 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 |
US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
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 |
US6962815B2 (en) | 2001-01-05 | 2005-11-08 | Children's Hopital Inc. | AAV2 vectors and methods |
WO2009137006A2 (en) * | 2008-04-30 | 2009-11-12 | The University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
WO2013078316A1 (en) | 2011-11-23 | 2013-05-30 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
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 |
JP6202701B2 (ja) | 2014-03-21 | 2017-09-27 | 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プログラム |
JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
RS65490B1 (sr) * | 2016-04-15 | 2024-05-31 | Res Inst Nationwide Childrens Hospital | Vektor adeno-asociranog virusa za isporuku β-sarkoglikana i mikrorna-29 i tretman mišićne distrofije |
-
2020
- 2020-08-21 CA CA3150964A patent/CA3150964A1/en active Pending
- 2020-08-21 US US17/635,978 patent/US20220290180A1/en active Pending
- 2020-08-21 DK DK20765424.5T patent/DK4017871T3/da active
- 2020-08-21 WO PCT/US2020/047339 patent/WO2021035120A1/en active Application Filing
- 2020-08-21 AU AU2020331987A patent/AU2020331987A1/en active Pending
- 2020-08-21 KR KR1020227007040A patent/KR20220092489A/ko unknown
- 2020-08-21 HR HRP20240505TT patent/HRP20240505T1/hr unknown
- 2020-08-21 PT PT207654245T patent/PT4017871T/pt unknown
- 2020-08-21 CN CN202080073755.8A patent/CN114945583A/zh active Pending
- 2020-08-21 BR BR112022003142A patent/BR112022003142A2/pt unknown
- 2020-08-21 MX MX2022002132A patent/MX2022002132A/es unknown
- 2020-08-21 SI SI202030411T patent/SI4017871T1/sl unknown
- 2020-08-21 TW TW109128653A patent/TW202120532A/zh unknown
- 2020-08-21 EP EP20765424.5A patent/EP4017871B1/en active Active
- 2020-08-21 LT LTEPPCT/US2020/047339T patent/LT4017871T/lt unknown
- 2020-08-21 RS RS20240449A patent/RS65421B1/sr unknown
- 2020-08-21 FI FIEP20765424.5T patent/FI4017871T3/fi active
- 2020-08-21 JP JP2022510976A patent/JP2022544627A/ja active Pending
- 2020-08-21 EP EP24152241.6A patent/EP4374880A2/en active Pending
-
2022
- 2022-02-20 IL IL290734A patent/IL290734A/en unknown
- 2022-03-11 CO CONC2022/0002864A patent/CO2022002864A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
JP2022544627A (ja) | 2022-10-19 |
CN114945583A (zh) | 2022-08-26 |
RS65421B1 (sr) | 2024-05-31 |
EP4374880A2 (en) | 2024-05-29 |
PT4017871T (pt) | 2024-04-22 |
LT4017871T (lt) | 2024-05-10 |
IL290734A (en) | 2022-04-01 |
BR112022003142A2 (pt) | 2022-05-17 |
FI4017871T3 (fi) | 2024-04-22 |
AU2020331987A1 (en) | 2022-03-31 |
KR20220092489A (ko) | 2022-07-01 |
DK4017871T3 (da) | 2024-04-22 |
EP4017871A1 (en) | 2022-06-29 |
EP4017871B1 (en) | 2024-01-17 |
CA3150964A1 (en) | 2021-02-25 |
TW202120532A (zh) | 2021-06-01 |
WO2021035120A1 (en) | 2021-02-25 |
HRP20240505T1 (hr) | 2024-07-05 |
SI4017871T1 (sl) | 2024-06-28 |
MX2022002132A (es) | 2022-05-18 |
US20220290180A1 (en) | 2022-09-15 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
CY1126080T1 (el) | Χορηγηση αδενο-σχετιζομενου ιικου διαβιβαστη μικρο-δυστροφινης με εξειδικευση στους μυς για τη θεραπευτικη αντιμετωπιση της μυïκης δυστροφιας | |
CO2018012084A2 (es) | Administración de vectores del virus adenoasociado de microdistrofina para tratar la distrofia muscular | |
CO2018012082A2 (es) | Administración de vectores del virus adenoasociado de β-sarcoglicano y microarn-29 y el tratamiento de la distrofia muscular | |
BR112021021156A2 (pt) | Terapêuticos anticorpos totalmente humanos pós-tradução modificados | |
CO2020002283A2 (es) | Métodos de terapia génica del factor viii (fviii) | |
MX2019007873A (es) | Terapia génica para tratar la enfermedad de wilson. | |
CL2021002236A1 (es) | Inserción de vectores de virus adeno-asociados de b-sarcoglicano y el tratamiento de distrofia muscular | |
JOP20200156A1 (ar) | إيسكيتامين لعلاج الاكتئاب | |
EA202190569A1 (ru) | Способы лечения болезни фабри у пациентов, имеющих мутацию в гене gla | |
CO2022002864A2 (es) | Administración de vectores de virus adenoasociado de alfa-sarcoglicano y el tratamiento de la distrofia muscular | |
BR112021015751A2 (pt) | Vetores de terapia genética para tratamento da doença de danon | |
BR112021009856A8 (pt) | Terapia para tratamento do câncer com uma administração intratumoral e / ou intravenosa de um mva recombinante que codifica 4-1bbl (cd137l) e / ou cd40l | |
CO2022012917A2 (es) | Vectores de genoterapia para tratar enfermedades cardíacas | |
CO2021000914A2 (es) | Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a | |
MX2021002349A (es) | Inhibidores de calicreína en plasmá y usos de los mismos para tratar ataque de angioedema hereditario. | |
CO2023000156A2 (es) | Administración de vectores de virus adenoasociado para distrofias musculares | |
MX2022004352A (es) | Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c. | |
CL2021003474A1 (es) | Producto farmacéutico para terapia enzimática para tratamiento de homocistinuria | |
AR121175A1 (es) | ADMINISTRACIÓN DE VECTORES DE VIRUS ADENOASOCIADO DE a-SARCOGLICANO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR | |
CL2023001650A1 (es) | Tratamiento de la enfermedad de danon | |
AR118192A1 (es) | ADMINISTRACIÓN DE b-SARCOGLICANO CON UN VECTOR DE VIRUS ADENOASOCIADO Y TRATAMIENTO DE LA DISTROFIA MUSCULAR | |
CO2023004031A2 (es) | Administración sistémica del vector de virus adenoasociado que expresa el sarcoglucano g y el tratamiento de la distrofia muscular | |
EA202192346A1 (ru) | Доставка -саркогликана с помощью вектора на основе аденоассоциированного вируса и лечение мышечной дистрофии | |
AR115563A1 (es) | Suministro de microdistrofina específica de músculo mediante vectores de virus adenoasociados para tratar la distrofia muscular | |
AR115647A1 (es) | Productos y métodos con virus adenoasociados recombinantes para el tratamiento de la distrofia muscular de extremidades y cintura de tipo 2a |