BR112021017054A2 - Composições e métodos para o tratamento da distrofia muscular oculofaríngea (opmd) - Google Patents
Composições e métodos para o tratamento da distrofia muscular oculofaríngea (opmd)Info
- Publication number
- BR112021017054A2 BR112021017054A2 BR112021017054A BR112021017054A BR112021017054A2 BR 112021017054 A2 BR112021017054 A2 BR 112021017054A2 BR 112021017054 A BR112021017054 A BR 112021017054A BR 112021017054 A BR112021017054 A BR 112021017054A BR 112021017054 A2 BR112021017054 A2 BR 112021017054A2
- Authority
- BR
- Brazil
- Prior art keywords
- opmd
- compositions
- aav
- methods
- muscular dystrophy
- Prior art date
Links
- 201000009110 Oculopharyngeal muscular dystrophy Diseases 0.000 title abstract 4
- 239000000203 mixture Substances 0.000 title abstract 4
- 101150003773 pabpn1 gene Proteins 0.000 abstract 3
- 241000702421 Dependoparvovirus Species 0.000 abstract 2
- 108090000565 Capsid Proteins Proteins 0.000 abstract 1
- 102100023321 Ceruloplasmin Human genes 0.000 abstract 1
- 108091030071 RNAI Proteins 0.000 abstract 1
- 108091070501 miRNA Proteins 0.000 abstract 1
- 210000001184 pharyngeal muscle Anatomy 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
Classifications
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- 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
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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
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
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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
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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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- 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
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
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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
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
- C07K14/01—DNA viruses
- C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
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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
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- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- C12N2710/00011—Details
- C12N2710/14011—Baculoviridae
- C12N2710/14041—Use of virus, viral particle or viral elements as a vector
- C12N2710/14045—Special targeting system for viral vectors
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- C12N2710/00011—Details
- C12N2710/14011—Baculoviridae
- C12N2710/14051—Methods of production or purification of viral material
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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- 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
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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Abstract
composições e métodos para o tratamento da distrofia muscular oculofaríngea (opmd).a presente divulgação se refere a vetores de entrega de vírus adenoassociado modificado (aav) compreendendo construções de dna "silenciar e substituir", composições compreendendo os mesmos, e o uso do aav modificado e composições para tratar a distrofia muscular oculofaríngea (opmd) em indivíduos que sofrem de opmd ou que sejam predispostos à mesma. em particular, a divulgação se refere ao aav contendo uma proteína de capsídeo com uma subunidade 1 modificada (vp1) e compreendendo uma construção de dna "silenciar e substituir", em que a construção de dna "silenciar e substituir" compreende (i) uma construção de rnai dirigida por dna (ddrnai) que codifica microrna curto em grampo (shmir) direcionado a pabpn1 causadora da opmd e (ii) uma construção de pabpn1 que codifica a proteína pabpn1 funcional tendo um transcrito de rnam que não é alvo dos shmirs em (i). a presente divulgação também se refere aos métodos para o tratamento da opmd que compreende a injeção direta de um aav da divulgação nos músculos faríngeos de um indivíduo.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962812187P | 2019-02-28 | 2019-02-28 | |
PCT/AU2020/050182 WO2020172720A1 (en) | 2019-02-28 | 2020-02-28 | Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd) |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112021017054A2 true BR112021017054A2 (pt) | 2021-11-09 |
Family
ID=72238736
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112021017054A BR112021017054A2 (pt) | 2019-02-28 | 2020-02-28 | Composições e métodos para o tratamento da distrofia muscular oculofaríngea (opmd) |
Country Status (12)
Country | Link |
---|---|
US (1) | US20220098614A1 (pt) |
EP (1) | EP3931317A4 (pt) |
JP (1) | JP2022522166A (pt) |
KR (1) | KR20210138030A (pt) |
CN (1) | CN113631706A (pt) |
AU (1) | AU2020229886A1 (pt) |
BR (1) | BR112021017054A2 (pt) |
CA (1) | CA3130221A1 (pt) |
IL (1) | IL285841A (pt) |
MX (1) | MX2021010412A (pt) |
SG (1) | SG11202108469XA (pt) |
WO (1) | WO2020172720A1 (pt) |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2020077412A1 (en) * | 2018-10-17 | 2020-04-23 | Benitec Biopharma Limited | Methods for treating oculopharyngeal muscular dystrophy (opmd) |
Family Cites Families (20)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
AU715543B2 (en) | 1995-09-08 | 2000-02-03 | Genzyme Corporation | Improved AAV vectors for gene therapy |
US7208315B2 (en) | 2000-01-05 | 2007-04-24 | Fred Hutchinson Cancer Research Center | Compositions and methods for efficient AAV vector production |
EP1390490B1 (en) | 2001-05-24 | 2009-04-15 | Genzyme Corporation | Muscle-specific expression vectors |
US7271002B2 (en) | 2001-11-09 | 2007-09-18 | United States Of America, Represented By The Secretary, Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
WO2003074714A1 (en) | 2002-03-05 | 2003-09-12 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
WO2005072364A2 (en) | 2004-01-27 | 2005-08-11 | University Of Florida | A modified baculovirus expression system for production of pseudotyped raav vector |
US8008468B2 (en) | 2005-02-16 | 2011-08-30 | Benitec, Inc. | RNAi expression constructs with liver-specific enhancer/promoter |
US8163543B2 (en) | 2005-10-20 | 2012-04-24 | Amsterdam Molecular Therapeutics B.V. | AAV vectors produced in insect cells |
WO2007120533A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Minigene expression cassette |
CN101528916B (zh) | 2006-04-28 | 2013-09-04 | 宾夕法尼亚大学托管会 | 规模可调的aav生产方法 |
AU2007261806B2 (en) | 2006-06-21 | 2013-08-15 | Uniqure Ip B.V. | Vectors with modified initiation codon for the translation of AAV-Rep78 useful for production of AAV in insect cells |
AU2009209408B2 (en) | 2008-01-29 | 2015-06-11 | Beacon Therapeutics Limited | Recombinant virus production using mammalian cells in suspension |
KR20220106852A (ko) * | 2013-07-26 | 2022-07-29 | 유니버시티 오브 아이오와 리써치 파운데이션 | 뇌 질환을 치료하기 위한 방법 및 조성물 |
EP3039129B1 (en) | 2013-08-30 | 2018-06-27 | Amgen Inc. | High titer recombinant aav vector production in adherent and suspension cells |
CN104815338A (zh) * | 2015-05-01 | 2015-08-05 | 许瑞安 | 一种携带kal基因重组腺相关病毒载体的基因治疗药物 |
WO2017184879A1 (en) | 2016-04-21 | 2017-10-26 | Virovek, Inc | Aav production in insect cells, methods and compositions therefor |
CN118421624A (zh) | 2016-12-14 | 2024-08-02 | 贝尼泰克知识产权控股股份有限公司 | 用于治疗眼咽肌营养不良症(opmd)的试剂及其用途 |
AU2018323049A1 (en) | 2017-08-31 | 2020-04-16 | Benitec IP Holdings Inc. | Adeno-associated virus (AAV) with modified phospholipase domain |
-
2020
- 2020-02-28 US US17/434,666 patent/US20220098614A1/en active Pending
- 2020-02-28 MX MX2021010412A patent/MX2021010412A/es unknown
- 2020-02-28 EP EP20762817.3A patent/EP3931317A4/en active Pending
- 2020-02-28 SG SG11202108469XA patent/SG11202108469XA/en unknown
- 2020-02-28 BR BR112021017054A patent/BR112021017054A2/pt unknown
- 2020-02-28 AU AU2020229886A patent/AU2020229886A1/en active Pending
- 2020-02-28 CN CN202080017400.7A patent/CN113631706A/zh active Pending
- 2020-02-28 KR KR1020217031046A patent/KR20210138030A/ko unknown
- 2020-02-28 WO PCT/AU2020/050182 patent/WO2020172720A1/en unknown
- 2020-02-28 CA CA3130221A patent/CA3130221A1/en active Pending
- 2020-02-28 JP JP2021549908A patent/JP2022522166A/ja active Pending
-
2021
- 2021-08-24 IL IL285841A patent/IL285841A/en unknown
Also Published As
Publication number | Publication date |
---|---|
EP3931317A4 (en) | 2023-01-04 |
CA3130221A1 (en) | 2020-09-03 |
WO2020172720A1 (en) | 2020-09-03 |
KR20210138030A (ko) | 2021-11-18 |
MX2021010412A (es) | 2022-01-24 |
AU2020229886A1 (en) | 2021-09-09 |
EP3931317A1 (en) | 2022-01-05 |
SG11202108469XA (en) | 2021-09-29 |
IL285841A (en) | 2021-10-31 |
US20220098614A1 (en) | 2022-03-31 |
JP2022522166A (ja) | 2022-04-14 |
CN113631706A (zh) | 2021-11-09 |
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