CO2021000914A2 - Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a - Google Patents
Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2aInfo
- Publication number
- CO2021000914A2 CO2021000914A2 CONC2021/0000914A CO2021000914A CO2021000914A2 CO 2021000914 A2 CO2021000914 A2 CO 2021000914A2 CO 2021000914 A CO2021000914 A CO 2021000914A CO 2021000914 A2 CO2021000914 A2 CO 2021000914A2
- Authority
- CO
- Colombia
- Prior art keywords
- methods
- muscular dystrophy
- treatment
- recombinant adeno
- associated virus
- Prior art date
Links
Classifications
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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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification 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
- A61K48/005—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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6472—Cysteine endopeptidases (3.4.22)
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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
- 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
- C12N2750/14131—Uses of virus other than therapeutic or vaccine, e.g. disinfectant
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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
- 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
Abstract
Se proporcionan productos y métodos para tratar la distrofia muscular de cinturas 2A. En los métodos, los virus adenoasociados recombinantes administran ADN que codifica una proteína con actividad de calpaína 3.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862691934P | 2018-06-29 | 2018-06-29 | |
US201962865081P | 2019-06-21 | 2019-06-21 | |
PCT/US2019/039893 WO2020006458A1 (en) | 2018-06-29 | 2019-06-28 | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2021000914A2 true CO2021000914A2 (es) | 2021-02-08 |
Family
ID=67544319
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2021/0000914A CO2021000914A2 (es) | 2018-06-29 | 2021-01-28 | Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a |
Country Status (15)
Country | Link |
---|---|
US (1) | US20210277362A1 (es) |
EP (1) | EP3814514A1 (es) |
JP (1) | JP2021528983A (es) |
KR (1) | KR20210028162A (es) |
CN (1) | CN112543810A (es) |
AU (1) | AU2019295779A1 (es) |
BR (1) | BR112020026853A2 (es) |
CA (1) | CA3105216A1 (es) |
CL (1) | CL2020003384A1 (es) |
CO (1) | CO2021000914A2 (es) |
IL (1) | IL279789A (es) |
MX (1) | MX2020014119A (es) |
SG (1) | SG11202012715UA (es) |
TW (1) | TW202020161A (es) |
WO (1) | WO2020006458A1 (es) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2021257595A1 (en) | 2020-06-15 | 2021-12-23 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery for muscular dystrophies |
EP4108263A3 (en) | 2021-06-02 | 2023-03-22 | Research Institute at Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
USD1008419S1 (en) * | 2021-06-14 | 2023-12-19 | Jong Won Kim | Vent valve of a wastewater drainage pipe |
EP4198048A1 (en) | 2021-12-16 | 2023-06-21 | Genethon | Calpain-3 gene transfer increase using modified itr sequences |
Family Cites Families (24)
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 |
PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
AU688428B2 (en) | 1993-11-09 | 1998-03-12 | Johns Hopkins University, The | Generation of high titers of recombinant AAV vectors |
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 |
CA2625279A1 (en) | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
EP1983057A3 (en) | 1995-09-08 | 2009-01-07 | 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 |
KR20000068501A (ko) | 1996-09-06 | 2000-11-25 | 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 재조합 아데노-관련 바이러스 지정 유전자 요법을 위한 방법 |
US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
EP1009808B1 (en) | 1997-09-05 | 2012-12-05 | Genzyme Corporation | Methods for generating high titer helper-free preparations of 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 |
WO2001083692A2 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
AU2002248297A1 (en) | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
US9434928B2 (en) | 2011-11-23 | 2016-09-06 | 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 |
FR3004463A1 (fr) * | 2013-04-11 | 2014-10-17 | Genethon | Systeme d'expression pour une therapie genique selective |
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 |
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 |
BR112018009954A2 (pt) * | 2015-11-16 | 2018-11-13 | Res Inst Nationwide Childrens Hospital | materiais e métodos para o tratamento de miopatias baseadas em titinas e outras titinopatias |
CA3021057A1 (en) * | 2016-04-15 | 2017-10-19 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy |
-
2019
- 2019-06-28 CN CN201980044250.6A patent/CN112543810A/zh active Pending
- 2019-06-28 SG SG11202012715UA patent/SG11202012715UA/en unknown
- 2019-06-28 AU AU2019295779A patent/AU2019295779A1/en active Pending
- 2019-06-28 US US17/255,488 patent/US20210277362A1/en active Pending
- 2019-06-28 CA CA3105216A patent/CA3105216A1/en active Pending
- 2019-06-28 KR KR1020207037223A patent/KR20210028162A/ko unknown
- 2019-06-28 TW TW108123006A patent/TW202020161A/zh unknown
- 2019-06-28 WO PCT/US2019/039893 patent/WO2020006458A1/en active Application Filing
- 2019-06-28 MX MX2020014119A patent/MX2020014119A/es unknown
- 2019-06-28 EP EP19749867.8A patent/EP3814514A1/en active Pending
- 2019-06-28 BR BR112020026853-4A patent/BR112020026853A2/pt unknown
- 2019-06-28 JP JP2020572953A patent/JP2021528983A/ja active Pending
-
2020
- 2020-12-24 CL CL2020003384A patent/CL2020003384A1/es unknown
- 2020-12-27 IL IL279789A patent/IL279789A/en unknown
-
2021
- 2021-01-28 CO CONC2021/0000914A patent/CO2021000914A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
MX2020014119A (es) | 2021-06-18 |
IL279789A (en) | 2021-03-01 |
EP3814514A1 (en) | 2021-05-05 |
AU2019295779A1 (en) | 2021-01-28 |
CL2020003384A1 (es) | 2021-07-23 |
TW202020161A (zh) | 2020-06-01 |
SG11202012715UA (en) | 2021-01-28 |
KR20210028162A (ko) | 2021-03-11 |
CN112543810A (zh) | 2021-03-23 |
CA3105216A1 (en) | 2020-01-02 |
US20210277362A1 (en) | 2021-09-09 |
JP2021528983A (ja) | 2021-10-28 |
BR112020026853A2 (pt) | 2021-04-20 |
WO2020006458A1 (en) | 2020-01-02 |
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