MX2023002569A - Administracion sistemica del vector de virus adenoasociado que expresa el sarcoglucano g y el tratamiento de la distrofia muscular. - Google Patents
Administracion sistemica del vector de virus adenoasociado que expresa el sarcoglucano g y el tratamiento de la distrofia muscular.Info
- Publication number
- MX2023002569A MX2023002569A MX2023002569A MX2023002569A MX2023002569A MX 2023002569 A MX2023002569 A MX 2023002569A MX 2023002569 A MX2023002569 A MX 2023002569A MX 2023002569 A MX2023002569 A MX 2023002569A MX 2023002569 A MX2023002569 A MX 2023002569A
- Authority
- MX
- Mexico
- Prior art keywords
- sarcoglycan
- muscular dystrophy
- adeno
- treatment
- associated virus
- Prior art date
Links
- 201000006938 muscular dystrophy Diseases 0.000 title abstract 2
- 241000702421 Dependoparvovirus Species 0.000 title 1
- 238000012385 systemic delivery Methods 0.000 title 1
- 108010083379 Sarcoglycans Proteins 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
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
- 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
- C07K14/4707—Muscular dystrophy
-
- 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
- A61K48/0016—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 wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
-
- 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
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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- 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
- 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
- C12N15/63—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
- 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
- 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
- C12N2750/14171—Demonstrated in vivo effect
-
- 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
Abstract
En la presente descripción se describen métodos para tratar la distrofia muscular que comprenden administrar un vector de AAV recombinante (rAAV) scAAVrh74.MHCK7.hSGCG, métodos para expresar el gen de sarcoglucano gamma en un paciente, composiciones farmacéuticas que comprenden el rAAV, y métodos para generar el rAAV.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063075697P | 2020-09-08 | 2020-09-08 | |
PCT/US2021/048957 WO2022055791A1 (en) | 2020-09-08 | 2021-09-03 | Systemic delivery of adeno-associated virus vector expressing g-sarcoglycan and the treatment of muscular dystrophy |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2023002569A true MX2023002569A (es) | 2023-03-22 |
Family
ID=78086895
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2023002569A MX2023002569A (es) | 2020-09-08 | 2021-09-03 | Administracion sistemica del vector de virus adenoasociado que expresa el sarcoglucano g y el tratamiento de la distrofia muscular. |
Country Status (14)
Country | Link |
---|---|
US (1) | US11925675B2 (es) |
EP (1) | EP4204088A1 (es) |
JP (1) | JP2023540095A (es) |
KR (1) | KR20230066021A (es) |
CN (1) | CN116134047A (es) |
AR (1) | AR123445A1 (es) |
AU (1) | AU2021340584A1 (es) |
BR (1) | BR112023003736A2 (es) |
CA (1) | CA3189647A1 (es) |
CO (1) | CO2023004031A2 (es) |
IL (1) | IL300986A (es) |
MX (1) | MX2023002569A (es) |
TW (1) | TW202227634A (es) |
WO (1) | WO2022055791A1 (es) |
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 |
JP3952312B2 (ja) | 1993-11-09 | 2007-08-01 | メディカル カレッジ オブ オハイオ | アデノ関連ウイルス複製遺伝子を発現可能な安定な細胞株 |
PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
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 |
US6143548A (en) | 1995-08-30 | 2000-11-07 | Genzyme Corporation | Chromatographic purification of adeno-associated virus (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 |
US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
EP0932418B1 (en) | 1996-09-06 | 2007-12-05 | The Trustees Of The University Of Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
CA2302992C (en) | 1997-09-05 | 2011-11-01 | 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 |
US7056502B2 (en) | 2000-04-28 | 2006-06-06 | 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 |
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 |
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 |
US20200360534A1 (en) * | 2018-01-31 | 2020-11-19 | Research Institute At Nationwide Children's Hospital | Gene therapy for limb-girdle muscular dystrophy type 2c |
KR20210133242A (ko) * | 2019-02-26 | 2021-11-05 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | B-사르코글리칸의 아데노 관련 바이러스 벡터 전달 및 근이영양증의 치료 |
WO2021126880A1 (en) * | 2019-12-16 | 2021-06-24 | Research Institute At Nationwide Children's Hospital | Compositions and methods for restoring and maintaining the dystrophin-associated protein complex (dapc) |
CN115996758A (zh) * | 2020-06-19 | 2023-04-21 | 吉尼松公司 | 使sgcg在肌肉和心脏中充分表达的基因治疗表达系统 |
-
2021
- 2021-08-30 TW TW110132144A patent/TW202227634A/zh unknown
- 2021-09-03 BR BR112023003736A patent/BR112023003736A2/pt unknown
- 2021-09-03 EP EP21790297.2A patent/EP4204088A1/en active Pending
- 2021-09-03 AU AU2021340584A patent/AU2021340584A1/en active Pending
- 2021-09-03 CN CN202180053937.3A patent/CN116134047A/zh active Pending
- 2021-09-03 CA CA3189647A patent/CA3189647A1/en active Pending
- 2021-09-03 MX MX2023002569A patent/MX2023002569A/es unknown
- 2021-09-03 JP JP2023514417A patent/JP2023540095A/ja active Pending
- 2021-09-03 KR KR1020237011162A patent/KR20230066021A/ko unknown
- 2021-09-03 WO PCT/US2021/048957 patent/WO2022055791A1/en active Application Filing
- 2021-09-03 IL IL300986A patent/IL300986A/en unknown
- 2021-09-07 US US17/468,086 patent/US11925675B2/en active Active
- 2021-09-07 AR ARP210102484A patent/AR123445A1/es unknown
-
2023
- 2023-03-29 CO CONC2023/0004031A patent/CO2023004031A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
KR20230066021A (ko) | 2023-05-12 |
CN116134047A (zh) | 2023-05-16 |
WO2022055791A1 (en) | 2022-03-17 |
US20220088123A1 (en) | 2022-03-24 |
US11925675B2 (en) | 2024-03-12 |
AU2021340584A1 (en) | 2023-05-04 |
CO2023004031A2 (es) | 2023-04-27 |
JP2023540095A (ja) | 2023-09-21 |
AR123445A1 (es) | 2022-11-30 |
BR112023003736A2 (pt) | 2023-03-28 |
EP4204088A1 (en) | 2023-07-05 |
CA3189647A1 (en) | 2022-03-17 |
IL300986A (en) | 2023-04-01 |
TW202227634A (zh) | 2022-07-16 |
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