BR112021016570A2 - Entrega de vetor de vírus adenoassociado de beta-sarcoglicano e tratamento de distrofia muscular - Google Patents
Entrega de vetor de vírus adenoassociado de beta-sarcoglicano e tratamento de distrofia muscularInfo
- Publication number
- BR112021016570A2 BR112021016570A2 BR112021016570A BR112021016570A BR112021016570A2 BR 112021016570 A2 BR112021016570 A2 BR 112021016570A2 BR 112021016570 A BR112021016570 A BR 112021016570A BR 112021016570 A BR112021016570 A BR 112021016570A BR 112021016570 A2 BR112021016570 A2 BR 112021016570A2
- Authority
- BR
- Brazil
- Prior art keywords
- beta
- sarcoglycan
- muscular dystrophy
- adeno
- treatment
- Prior art date
Links
- 108010083379 Sarcoglycans Proteins 0.000 title abstract 3
- 201000006938 muscular dystrophy Diseases 0.000 title abstract 3
- 102000019370 Beta-sarcoglycan Human genes 0.000 title abstract 2
- 241000702421 Dependoparvovirus Species 0.000 title abstract 2
- 238000000034 method Methods 0.000 abstract 3
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
Classifications
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- 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
-
- 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
-
- 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
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- A61K38/1719—Muscle proteins, e.g. myosin or actin
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
- A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
-
- 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
- A61K48/0025—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 non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—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 non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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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
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- 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
- 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
- A61K48/0083—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 administration regime
-
- 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
- C12N2750/14041—Use of virus, viral particle or viral elements as a vector
- C12N2750/14042—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
-
- 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
- C12N2750/14071—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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biophysics (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physical Education & Sports Medicine (AREA)
- Wood Science & Technology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Neurology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Marine Sciences & Fisheries (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Inorganic Chemistry (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
entrega de vetor de vírus adenoassociado de beta-sarcoglicano e tratamento de distrofia muscular. a presente invenção trata se de métodos de tratamento de distrofia muscular que compreende administrar um vetor de aav recombinante (raav) scaavrh74.mhck7.hsgcb, métodos de expressão do gene beta sarcoglicano em um paciente, composições farmacêuticas que compreendem o raav e métodos de geração do raav.
Applications Claiming Priority (7)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962810917P | 2019-02-26 | 2019-02-26 | |
US201962834012P | 2019-04-15 | 2019-04-15 | |
US201962858644P | 2019-06-07 | 2019-06-07 | |
US201962881901P | 2019-08-01 | 2019-08-01 | |
US201962909564P | 2019-10-02 | 2019-10-02 | |
US201962910779P | 2019-10-04 | 2019-10-04 | |
PCT/US2020/019892 WO2020176614A1 (en) | 2019-02-26 | 2020-02-26 | Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112021016570A2 true BR112021016570A2 (pt) | 2021-11-03 |
Family
ID=70005777
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112021016570A BR112021016570A2 (pt) | 2019-02-26 | 2020-02-26 | Entrega de vetor de vírus adenoassociado de beta-sarcoglicano e tratamento de distrofia muscular |
Country Status (15)
Country | Link |
---|---|
US (1) | US20230241252A1 (pt) |
EP (1) | EP3930765A1 (pt) |
JP (1) | JP2022521776A (pt) |
KR (1) | KR20210133242A (pt) |
CN (1) | CN113766935A (pt) |
AU (1) | AU2020229340A1 (pt) |
BR (1) | BR112021016570A2 (pt) |
CA (1) | CA3131390A1 (pt) |
CL (1) | CL2021002236A1 (pt) |
CO (1) | CO2021012074A2 (pt) |
IL (1) | IL285849A (pt) |
MX (1) | MX2021010356A (pt) |
SG (1) | SG11202109113TA (pt) |
TW (1) | TW202045531A (pt) |
WO (1) | WO2020176614A1 (pt) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
IL299094A (en) | 2020-06-15 | 2023-02-01 | Res Inst Nationwide Childrens Hospital | Administration of an adeno-associated virus vector for muscular dystrophies |
TW202227634A (zh) | 2020-09-08 | 2022-07-16 | 美商薩羅塔治療公司 | 表現γ—肌聚醣之腺相關病毒載體之全身性遞送及肌肉失養症之治療 |
EP4198048A1 (en) | 2021-12-16 | 2023-06-21 | Genethon | Calpain-3 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 |
AU678867B2 (en) | 1993-11-09 | 1997-06-12 | Medical College Of Ohio, The | Stable cell lines capable of expressing the adeno-associated virus replication gene |
JPH09509564A (ja) | 1993-11-09 | 1997-09-30 | ターゲテッド 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 |
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 |
AU707866B2 (en) | 1994-12-06 | 1999-07-22 | 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 |
EP0847442A1 (en) | 1995-08-30 | 1998-06-17 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
ES2317646T3 (es) | 1995-09-08 | 2009-04-16 | Genzyme Corporation | Vectores aav mejorados para terapia genica. |
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 |
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 |
CA2406743A1 (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 |
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 |
JP7079733B2 (ja) * | 2016-04-15 | 2022-06-02 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | β-サルコグリカン及びマイクロRNA-29のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療 |
MX2018012537A (es) * | 2016-04-15 | 2019-02-25 | Univ Pennsylvania | Terapia de genes para tratar hemofilia a. |
-
2020
- 2020-02-26 MX MX2021010356A patent/MX2021010356A/es unknown
- 2020-02-26 TW TW109106292A patent/TW202045531A/zh unknown
- 2020-02-26 CN CN202080031591.2A patent/CN113766935A/zh active Pending
- 2020-02-26 CA CA3131390A patent/CA3131390A1/en active Pending
- 2020-02-26 EP EP20714395.9A patent/EP3930765A1/en active Pending
- 2020-02-26 KR KR1020217030293A patent/KR20210133242A/ko unknown
- 2020-02-26 WO PCT/US2020/019892 patent/WO2020176614A1/en unknown
- 2020-02-26 US US17/432,417 patent/US20230241252A1/en active Pending
- 2020-02-26 JP JP2021549782A patent/JP2022521776A/ja active Pending
- 2020-02-26 SG SG11202109113TA patent/SG11202109113TA/en unknown
- 2020-02-26 AU AU2020229340A patent/AU2020229340A1/en active Pending
- 2020-02-26 BR BR112021016570A patent/BR112021016570A2/pt unknown
-
2021
- 2021-08-24 CL CL2021002236A patent/CL2021002236A1/es unknown
- 2021-08-25 IL IL285849A patent/IL285849A/en unknown
- 2021-09-15 CO CONC2021/0012074A patent/CO2021012074A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
CN113766935A (zh) | 2021-12-07 |
TW202045531A (zh) | 2020-12-16 |
AU2020229340A1 (en) | 2021-09-16 |
CA3131390A1 (en) | 2020-09-03 |
JP2022521776A (ja) | 2022-04-12 |
CL2021002236A1 (es) | 2022-04-29 |
KR20210133242A (ko) | 2021-11-05 |
EP3930765A1 (en) | 2022-01-05 |
US20230241252A1 (en) | 2023-08-03 |
WO2020176614A1 (en) | 2020-09-03 |
MX2021010356A (es) | 2022-03-04 |
SG11202109113TA (en) | 2021-09-29 |
IL285849A (en) | 2021-10-31 |
CO2021012074A2 (es) | 2021-09-30 |
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