AU2021391643A1 - Rna-targeting compositions and methods for treating cag repeat diseases - Google Patents

Rna-targeting compositions and methods for treating cag repeat diseases Download PDF

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Publication number
AU2021391643A1
AU2021391643A1 AU2021391643A AU2021391643A AU2021391643A1 AU 2021391643 A1 AU2021391643 A1 AU 2021391643A1 AU 2021391643 A AU2021391643 A AU 2021391643A AU 2021391643 A AU2021391643 A AU 2021391643A AU 2021391643 A1 AU2021391643 A1 AU 2021391643A1
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Australia
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sequence
seq
rna
cag
protein
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AU2021391643A
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AU2021391643A9 (en
Inventor
Ranjan BATRA
David A. Nelles
Daniela ROTH
Angeline TA
Dimitrios ZISOULIS
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Astellas Gene Therapies Inc
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Locanabio Inc
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Publication of AU2021391643A9 publication Critical patent/AU2021391643A9/en
Assigned to ASTELLAS GENE THERAPIES, INC. reassignment ASTELLAS GENE THERAPIES, INC. Request for Assignment Assignors: Locanabio, Inc.
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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
    • C12N15/62—DNA sequences coding for fusion proteins
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • 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
    • 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
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    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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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
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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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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/095—Fusion polypeptide containing a localisation/targetting motif containing a nuclear export signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/85—Fusion polypeptide containing an RNA binding domain
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Pharmacology & Pharmacy (AREA)
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  • Biophysics (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Physical Education & Sports Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2021391643A 2020-12-01 2021-12-01 Rna-targeting compositions and methods for treating cag repeat diseases Pending AU2021391643A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063119977P 2020-12-01 2020-12-01
US63/119,977 2020-12-01
US202063130060P 2020-12-23 2020-12-23
US63/130,060 2020-12-23
PCT/US2021/061482 WO2022119974A1 (en) 2020-12-01 2021-12-01 Rna-targeting compositions and methods for treating cag repeat diseases

Publications (2)

Publication Number Publication Date
AU2021391643A1 true AU2021391643A1 (en) 2023-06-29
AU2021391643A9 AU2021391643A9 (en) 2023-08-17

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AU2021391643A Pending AU2021391643A1 (en) 2020-12-01 2021-12-01 Rna-targeting compositions and methods for treating cag repeat diseases

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US (1) US20240000972A1 (https=)
EP (1) EP4255470A1 (https=)
JP (1) JP2023551873A (https=)
KR (1) KR20230127221A9 (https=)
AU (1) AU2021391643A1 (https=)
CA (1) CA3200453A1 (https=)
WO (1) WO2022119974A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
IL301675A (en) 2020-10-09 2023-05-01 Tenaya Therapeutics Inc Methods and preparations for placophilin-2 gene therapy
US20240108751A1 (en) * 2020-12-01 2024-04-04 Locanabio, Inc. Rna-targeting compositions and methods for treating myotonic dystrophy type 1
WO2022221278A1 (en) * 2021-04-12 2022-10-20 Locanabio, Inc. Compositions and methods comprising hybrid promoters
JP2025525378A (ja) * 2022-06-21 2025-08-05 リーゲル セラピューティクス，インコーポレイティッド 遺伝子調節エレメント及びその使用
CN119320798A (zh) * 2023-07-17 2025-01-17 中国科学院上海营养与健康研究所 一种可用于筛选针对rna重复序列药物的报告系统
WO2025155722A1 (en) * 2024-01-16 2025-07-24 Astellas Gene Therapies, Inc. Muscle selective hybrid regulatory combinations and methods of use thereof for the treatment of myotonic dystrophy type 1

Family Cites Families (11)

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Publication number Priority date Publication date Assignee Title
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
DK2191001T3 (en) 2007-04-09 2016-09-19 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
CN101895633A (zh) 2010-07-14 2010-11-24 中兴通讯股份有限公司 一种移动终端及其解锁方法
WO2012068627A1 (en) 2010-11-24 2012-05-31 The University Of Western Australia Peptides for the specific binding of rna targets
WO2013058404A1 (ja) 2011-10-21 2013-04-25 国立大学法人九州大学 Pprモチーフを利用したrna結合性蛋白質の設計方法及びその利用
US10330674B2 (en) 2015-01-13 2019-06-25 Massachusetts Institute Of Technology Pumilio domain-based modular protein architecture for RNA binding
US10876101B2 (en) 2017-03-28 2020-12-29 Locanabio, Inc. CRISPR-associated (Cas) protein
US11168322B2 (en) 2017-06-30 2021-11-09 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
US10476825B2 (en) 2017-08-22 2019-11-12 Salk Institue for Biological Studies RNA targeting methods and compositions
JP2021526858A (ja) * 2018-06-08 2021-10-11 ロックアネイビオ， インコーポレイテッド Ｒｎａ標的化融合タンパク質組成物および使用方法
US20240108751A1 (en) * 2020-12-01 2024-04-04 Locanabio, Inc. Rna-targeting compositions and methods for treating myotonic dystrophy type 1

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Publication number Publication date
JP2023551873A (ja) 2023-12-13
EP4255470A1 (en) 2023-10-11
KR20230127221A (ko) 2023-08-31
CA3200453A1 (en) 2022-06-09
US20240000972A1 (en) 2024-01-04
WO2022119974A1 (en) 2022-06-09
KR20230127221A9 (ko) 2024-12-06
AU2021391643A9 (en) 2023-08-17

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