JP2021534755A - 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法 - Google Patents

筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法 Download PDF

Info

Publication number
JP2021534755A
JP2021534755A JP2021509821A JP2021509821A JP2021534755A JP 2021534755 A JP2021534755 A JP 2021534755A JP 2021509821 A JP2021509821 A JP 2021509821A JP 2021509821 A JP2021509821 A JP 2021509821A JP 2021534755 A JP2021534755 A JP 2021534755A
Authority
JP
Japan
Prior art keywords
aav
dmpk
seq
gene
expression
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.)
Withdrawn
Application number
JP2021509821A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021534755A5 (https=
JPWO2020041634A5 (https=
Inventor
ニコラス セバスティアン ウェイン，
スコット クエントン ハーパー，
ケリー リード クラーク，
Original Assignee
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
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 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 filed Critical 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
Publication of JP2021534755A publication Critical patent/JP2021534755A/ja
Publication of JP2021534755A5 publication Critical patent/JP2021534755A5/ja
Publication of JPWO2020041634A5 publication Critical patent/JPWO2020041634A5/ja
Priority to JP2024083610A priority Critical patent/JP2024105681A/ja
Priority to JP2025132404A priority patent/JP2025166094A/ja
Withdrawn legal-status Critical Current

Links

Images

Classifications

    • 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
    • C12N15/1137—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 against enzymes
    • 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
    • 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
    • C12N2320/33—Alteration of splicing
    • 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

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021509821A 2018-08-22 2019-08-22 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法 Withdrawn JP2021534755A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2024083610A JP2024105681A (ja) 2018-08-22 2024-05-22 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法
JP2025132404A JP2025166094A (ja) 2018-08-22 2025-08-07 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862721105P 2018-08-22 2018-08-22
US62/721,105 2018-08-22
PCT/US2019/047779 WO2020041634A1 (en) 2018-08-22 2019-08-22 Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the dmpk gene

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024083610A Division JP2024105681A (ja) 2018-08-22 2024-05-22 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法

Publications (3)

Publication Number Publication Date
JP2021534755A true JP2021534755A (ja) 2021-12-16
JP2021534755A5 JP2021534755A5 (https=) 2022-08-19
JPWO2020041634A5 JPWO2020041634A5 (https=) 2022-08-19

Family

ID=69591460

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2021509821A Withdrawn JP2021534755A (ja) 2018-08-22 2019-08-22 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法
JP2024083610A Pending JP2024105681A (ja) 2018-08-22 2024-05-22 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法
JP2025132404A Pending JP2025166094A (ja) 2018-08-22 2025-08-07 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2024083610A Pending JP2024105681A (ja) 2018-08-22 2024-05-22 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法
JP2025132404A Pending JP2025166094A (ja) 2018-08-22 2025-08-07 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法

Country Status (6)

Country Link
US (2) US12492402B2 (https=)
EP (1) EP3841209A4 (https=)
JP (3) JP2021534755A (https=)
AU (2) AU2019326551A1 (https=)
CA (1) CA3110466A1 (https=)
WO (1) WO2020041634A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025507958A (ja) 2022-03-04 2025-03-21 ロックアネイビオ， インコーポレイテッド 操作された核内低分子ＲＮＡ（ｓｎＲＮＡ）を含む組成物および方法
CA3247417A1 (en) * 2022-04-06 2023-10-12 Genzyme Corporation TARGETED GENE THERAPY FOR DM-1 MYOTONIC DYSTROPHY
WO2024006770A1 (en) * 2022-06-27 2024-01-04 Astellas Gene Therapies, Inc. Compositions and methods for the treatment of myotonic dystrophies
EP4604970A1 (en) * 2022-10-19 2025-08-27 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of dm1 protein kinase (dmpk) compositions thereof, and methods of use
AR131170A1 (es) * 2022-12-02 2025-02-19 Humble Bee Ltd Polipéptido recombinante
JP2026511976A (ja) * 2023-04-05 2026-04-14 ジェンザイム・コーポレーション Ｄｍ－１筋強直性ジストロフィーの標的遺伝子療法
WO2025137219A1 (en) * 2023-12-21 2025-06-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
EP4692348A1 (en) * 2024-08-07 2026-02-11 Nocturna Therapeutics SL Artificial rnas for treating repeat expansion disorders

Family Cites Families (28)

* Cited by examiner, † Cited by third party
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
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
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
AU722196B2 (en) 1995-08-30 2000-07-27 Genzyme Corporation Chromatographic purification of adenovirus and AAV
EP1983057A3 (en) 1995-09-08 2009-01-07 Genzyme Corporation Improved AAV vectors for gene therapy
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
JP2001514845A (ja) 1997-09-05 2001-09-18 ターゲティッド ジェネティクス コーポレイション 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法
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
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
FR2874384B1 (fr) * 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
PL381824A1 (pl) * 2007-02-22 2008-09-01 Instytut Chemii Bioorganicznej Pan W Poznaniu Sekwencja siRNA, wektor, cel molekularny dla reagentów siRNA i wektorów wprowadzanych do komórek i tkanek, sposób oceny specyficzności wyciszania zmutowanego transkryptu, sposób badania oddziaływań enzymów szlaku interferencji RNA z transkryptami oraz zastosowanie sekwencji siRNA i wektora
CA2792696C (en) 2010-03-17 2020-01-07 Association Institut De Myologie Modified u7 snrnas for treatment of neuromuscular diseases
WO2012012467A2 (en) * 2010-07-19 2012-01-26 Isis Pharmaceuticals, Inc. Modulation of nuclear-retained rna
ES2739804T3 (es) 2011-02-12 2020-02-04 Univ Iowa Res Found Compuestos terapéuticos
US9610362B2 (en) * 2012-03-16 2017-04-04 Valerion Therapeutics, Llc Antisense conjugates for decreasing expression of DMPK
TW201536329A (zh) * 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
US20180344817A1 (en) * 2015-05-01 2018-12-06 Precision Biosciences, Inc. Precise deletion of chromosomal sequences in vivo and treatment of nucleotide repeat expansion disorders using engineered nucleases
WO2017048732A1 (en) 2015-09-14 2017-03-23 Research Institute At Nationwide Children's Hospital Recombinant virus products and methods for inhibition of expression of myotilin
FR3044926B1 (fr) * 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
BR112018070249A2 (pt) 2016-04-02 2019-01-29 Research Institute At Nationwide Children's Hospital sistema promotor de u6 modificada para expressão específica de tecido

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
MOL. THER., vol. Vol. 20, Suppl. 1, JPN6023027316, 2012, pages 171 - 172, ISSN: 0005241986 *

Also Published As

Publication number Publication date
US20260049320A1 (en) 2026-02-19
CA3110466A1 (en) 2020-02-27
EP3841209A1 (en) 2021-06-30
WO2020041634A1 (en) 2020-02-27
JP2025166094A (ja) 2025-11-05
JP2024105681A (ja) 2024-08-06
AU2019326551A1 (en) 2021-03-18
EP3841209A4 (en) 2023-01-04
US12492402B2 (en) 2025-12-09
AU2025283617A1 (en) 2026-02-19
US20220033823A1 (en) 2022-02-03

Similar Documents

Publication Publication Date Title
US12545909B2 (en) DUX4 RNA silencing using RNA targeting CRISPR-CAS13b
US20260049320A1 (en) Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the dmpk gene
US10301649B2 (en) Recombinant virus products and methods for inhibition of expression of DUX4
EP3495472B1 (en) Recombinant virus products and methods for inhibition of expression of myotilin
US12325856B2 (en) Recombinant virus products and methods for inhibition of expression of myotilin
US20240318172A1 (en) Products and methods for inhibition of expression of peripheral myelin protein-22
JP2024537167A (ja) ミエリンタンパク質ゼロサイレンシング及びｃｍｔ１ｂ疾患を治療するための生成物及び方法
EP3350319B1 (en) Recombinant virus products and methods for inhibition of expression of myotilin
HK40009865A (en) Recombinant virus products and methods for inhibition of expression of myotilin
HK1258752B (en) Recombinant virus products and methods for inhibition of expression of myotilin

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220810

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220810

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230630

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230929

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231030

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20240122

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240522

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20240531

A761 Written withdrawal of application

Free format text: JAPANESE INTERMEDIATE CODE: A761

Effective date: 20240605