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)

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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
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BR
Brazil
Prior art keywords
opmd
compositions
aav
methods
muscular dystrophy
Prior art date
Application number
BR112021017054A
Other languages
English (en)
Portuguese (pt)
Inventor
David Suhy
W Roelvink Petrus
Shih-Chu Kao
Vanessa Strings-Ufombah
Original Assignee
Benitec Ip Holdings Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Benitec Ip Holdings Inc filed Critical Benitec Ip Holdings Inc
Publication of BR112021017054A2 publication Critical patent/BR112021017054A2/pt

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    • 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
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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
    • 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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    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
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    • 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
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    • 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
    • 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
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    • C12N2750/14011—Parvoviridae
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BR112021017054A 2019-02-28 2020-02-28 Composições e métodos para o tratamento da distrofia muscular oculofaríngea (opmd) BR112021017054A2 (pt)

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)

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BR112021017054A2 true BR112021017054A2 (pt) 2021-11-09

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Country Link
US (1) US20220098614A1 (https=)
EP (1) EP3931317A4 (https=)
JP (1) JP2022522166A (https=)
CN (2) CN121160804A (https=)
AU (1) AU2020229886B2 (https=)
BR (1) BR112021017054A2 (https=)
CA (1) CA3130221A1 (https=)
IL (1) IL285841A (https=)
MX (1) MX2021010412A (https=)
SG (1) SG11202108469XA (https=)
WO (1) WO2020172720A1 (https=)

Families Citing this family (3)

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Publication number Priority date Publication date Assignee Title
CN112867511A (zh) * 2018-10-17 2021-05-28 贝尼泰克生物制药有限公司 用于治疗眼咽肌营养不良(opmd)的方法
KR20260030147A (ko) * 2023-06-28 2026-03-05 베니텍 아이피 홀딩스 아이엔씨. 인두 근육에 치료 조성물을 투여하기 위한 장치 및 방법
WO2026011009A1 (en) * 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders

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EP2017338A1 (en) 2001-05-24 2009-01-21 Genzyme Corporation Muscle-specific expression vectors
WO2003042361A2 (en) 2001-11-09 2003-05-22 Government Of The United States Of America, 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
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WO2007120533A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Minigene expression cassette
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
KR101589259B1 (ko) 2006-06-21 2016-02-01 유니큐어 아이피 비.브이. 곤충세포 내 ａａｖ의 생산에 유용한 ａａｖ-ｒｅｐ78의 번역을 위한 변형된 개시 코돈을 갖는 벡터
DK2242840T3 (da) 2008-01-29 2019-10-21 Applied Genetic Tech Corporation Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension
EP3024497B1 (en) * 2013-07-26 2021-01-13 University of Iowa Research Foundation Methods and compositions for treating brain diseases
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
KR20230079511A (ko) 2016-04-21 2023-06-07 바이로베크 인코포레이티드 곤충 세포에서의 aav 생산, 및 이를 위한 방법 및 조성물
EP3555294A4 (en) * 2016-12-14 2020-08-26 Benitec Biopharma Limited REAGENTS FOR THE TREATMENT OF OCULOPHARYNGEAL MUSCLE DYSTROPHY (PDD) AND THEIR USE
WO2018139634A1 (ja) * 2017-01-30 2018-08-02 学校法人日本医科大学 アデノ随伴ウイルス（ａａｖ）キャプシドタンパク質の変異体
UY37863A (es) 2017-08-31 2019-03-29 Benitec Biopharma Ltd Virus adenoasociado (aav) con dominio de fosfolipasa modificado

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CA3130221A1 (en) 2020-09-03
AU2020229886B2 (en) 2025-09-18
KR20210138030A (ko) 2021-11-18
IL285841A (en) 2021-10-31
CN113631706A (zh) 2021-11-09
EP3931317A4 (en) 2023-01-04
JP2022522166A (ja) 2022-04-14
AU2020229886A1 (en) 2021-09-09
WO2020172720A1 (en) 2020-09-03
US20220098614A1 (en) 2022-03-31
SG11202108469XA (en) 2021-09-29
CN121160804A (zh) 2025-12-19
MX2021010412A (es) 2022-01-24
EP3931317A1 (en) 2022-01-05

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