AU2020330108A1 - Combined transgene and intron-derived miRNA therapy for treatment of SCA1 - Google Patents

Combined transgene and intron-derived miRNA therapy for treatment of SCA1 Download PDF

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Publication number
AU2020330108A1
AU2020330108A1 AU2020330108A AU2020330108A AU2020330108A1 AU 2020330108 A1 AU2020330108 A1 AU 2020330108A1 AU 2020330108 A AU2020330108 A AU 2020330108A AU 2020330108 A AU2020330108 A AU 2020330108A AU 2020330108 A1 AU2020330108 A1 AU 2020330108A1
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AU
Australia
Prior art keywords
aav
promoter
sequence
nucleic acid
expression cassette
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
AU2020330108A
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English (en)
Inventor
Ellie CARRELL
Beverly L. Davidson
Megan S. KEISER
Alejandro Mas Monteys
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Children's Hospital of Philadelphia
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Children's Hospital of Philadelphia
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Publication date
Application filed by Children's Hospital of Philadelphia filed Critical Children's Hospital of Philadelphia
Publication of AU2020330108A1 publication Critical patent/AU2020330108A1/en
Pending legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous 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
    • C07K14/46—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
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • 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
    • C12N15/11—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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2020330108A 2019-08-15 2020-08-14 Combined transgene and intron-derived miRNA therapy for treatment of SCA1 Pending AU2020330108A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962887209P 2019-08-15 2019-08-15
US62/887,209 2019-08-15
PCT/US2020/046499 WO2021030745A1 (en) 2019-08-15 2020-08-14 Combined transgene and intron-derived mirna therapy for treatment of sca1

Publications (1)

Publication Number Publication Date
AU2020330108A1 true AU2020330108A1 (en) 2022-03-17

Family

ID=74570777

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020330108A Pending AU2020330108A1 (en) 2019-08-15 2020-08-14 Combined transgene and intron-derived miRNA therapy for treatment of SCA1

Country Status (9)

Country Link
US (1) US20220288101A1 (enExample)
EP (1) EP4013414A4 (enExample)
JP (1) JP2022545378A (enExample)
CN (1) CN114555084A (enExample)
AU (1) AU2020330108A1 (enExample)
BR (1) BR112022002794A2 (enExample)
CA (1) CA3151122A1 (enExample)
MX (1) MX2022001984A (enExample)
WO (1) WO2021030745A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10820847B1 (en) 2019-08-15 2020-11-03 Talis Biomedical Corporation Diagnostic system
US20260043024A1 (en) * 2022-08-26 2026-02-12 The Children's Hospital Of Philadelphia Mirnas targeting atnx2 for the treatment of als and sca2
CN120603600A (zh) * 2023-01-19 2025-09-05 优尼科生物制药有限公司 基因递送媒介物的药物配制品

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7001733B1 (en) * 1998-05-12 2006-02-21 Rigel Pharmaceuticals, Inc. Methods and compositions for screening for modulations of IgE synthesis, secretion and switch rearrangement
US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US7605249B2 (en) * 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
DK2164967T3 (en) * 2007-05-31 2015-10-19 Univ Iowa Res Found Reduction of off-target rna interferenstoksicitet
JP6091435B2 (ja) * 2011-02-22 2017-03-08 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス（ａａｖ）ベクターを用いたタンパク質の送達
PT3628334T (pt) * 2014-03-21 2023-09-26 Genzyme Corp Terapia genética para retinopatia pigmentar
AU2015264263B2 (en) * 2014-05-20 2021-08-05 University Of Iowa Research Foundation Huntington's disease therapeutic compounds
MA40782A (fr) * 2014-10-03 2017-08-08 Hocuslocus Llc Procédés et compositions utilisant un arn non codant en vue de la culture et de la sélection de cellules
CA2977607A1 (en) * 2015-02-26 2016-09-01 Jacob Schneiderman Methods and compositions relating to leptin antagonists
WO2016196507A1 (en) * 2015-05-29 2016-12-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
IL302455A (en) * 2016-03-01 2023-06-01 Univ Florida Adeno-associated virus vectors for the treatment of dominant retinitis pigmentosa
CA3075168A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. Modified closed-ended dna (cedna)
KR20200119782A (ko) * 2017-12-06 2020-10-20 오비드 테라퓨틱스 인크. 발작 장애 치료 시 mir101 또는 mir128의 용도
US20210355509A1 (en) * 2018-08-07 2021-11-18 The Children's Hospital Of Philadelphia Alternative splicing regulation of gene expression and therapeutic methods

Also Published As

Publication number Publication date
JP2022545378A (ja) 2022-10-27
CA3151122A1 (en) 2021-02-18
US20220288101A1 (en) 2022-09-15
WO2021030745A1 (en) 2021-02-18
BR112022002794A2 (pt) 2022-08-09
CN114555084A (zh) 2022-05-27
EP4013414A4 (en) 2023-09-27
EP4013414A1 (en) 2022-06-22
MX2022001984A (es) 2022-05-19

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