JP2023538519A - Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療 - Google Patents

Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療 Download PDF

Info

Publication number
JP2023538519A
JP2023538519A JP2023507876A JP2023507876A JP2023538519A JP 2023538519 A JP2023538519 A JP 2023538519A JP 2023507876 A JP2023507876 A JP 2023507876A JP 2023507876 A JP2023507876 A JP 2023507876A JP 2023538519 A JP2023538519 A JP 2023538519A
Authority
JP
Japan
Prior art keywords
vector
pkp2
polynucleotide
promoter
seq
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
JP2023507876A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023538519A5 (OSRAM
Inventor
クリストファー ディーン ハーツォグ
チェスター ビッテンコート サクラメント
ラジ プラバカール
デイビッド リックス
Original Assignee
スペースクラフト セブン リミテッド ライアビリティ カンパニー
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 スペースクラフト セブン リミテッド ライアビリティ カンパニー filed Critical スペースクラフト セブン リミテッド ライアビリティ カンパニー
Publication of JP2023538519A publication Critical patent/JP2023538519A/ja
Publication of JP2023538519A5 publication Critical patent/JP2023538519A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • 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
    • 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
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • 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
    • 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
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0375—Animal model for cardiovascular diseases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023507876A 2020-08-07 2021-08-09 Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療 Pending JP2023538519A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063063032P 2020-08-07 2020-08-07
US63/063,032 2020-08-07
PCT/US2021/045220 WO2022032226A1 (en) 2020-08-07 2021-08-09 Plakophilin-2 (pkp2) gene therapy using aav vector

Publications (2)

Publication Number Publication Date
JP2023538519A true JP2023538519A (ja) 2023-09-08
JP2023538519A5 JP2023538519A5 (OSRAM) 2024-08-16

Family

ID=80118582

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023507876A Pending JP2023538519A (ja) 2020-08-07 2021-08-09 Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療

Country Status (11)

Country Link
US (4) US20220168447A1 (OSRAM)
EP (1) EP4192487A4 (OSRAM)
JP (1) JP2023538519A (OSRAM)
KR (1) KR20230043869A (OSRAM)
CN (1) CN116194154A (OSRAM)
AU (1) AU2021320902A1 (OSRAM)
BR (1) BR112023001852A2 (OSRAM)
CA (1) CA3185267A1 (OSRAM)
IL (1) IL300263A (OSRAM)
MX (1) MX2023001615A (OSRAM)
WO (1) WO2022032226A1 (OSRAM)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024512483A (ja) * 2021-03-19 2024-03-19 ユーシーエル ビジネス リミテッド 遺伝子治療組成物及び不整脈原性右室心筋症の治療

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109641063B (zh) 2016-04-20 2022-11-15 能源环境和技术研究中心O.A., M.P. 用于增强pklr的基因表达的组合物和方法
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
JP7576754B2 (ja) 2018-04-11 2024-11-01 スペースクラフト セブン リミテッド ライアビリティ カンパニー 幹細胞移植のための組成物および方法
AU2019261646A1 (en) 2018-04-27 2020-11-12 Spacecraft Seven, Llc Gene therapy for CNS degeneration
WO2020205889A1 (en) 2019-04-01 2020-10-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
US20220389452A1 (en) * 2019-09-20 2022-12-08 Ucl Business Ltd Gene therapy composition and treatment of right ventricular arrythmogenic cardiomyopathy
EP4087489B1 (en) 2020-01-08 2025-05-14 Vitruvia Holdings Inc. Methods and computing system for processing ultrasound image to determine health of subdermal tissue
MX2022009883A (es) 2020-02-13 2022-10-03 Tenaya Therapeutics Inc Vectores de genoterapia para tratar enfermedades cardíacas.
IL300263A (en) 2020-08-07 2023-03-01 Spacecraft Seven Llc Plakophilin-2 (PKP2) gene therapy using an AAV vector
US11781156B2 (en) * 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
JP7415042B2 (ja) 2020-10-09 2024-01-16 テナヤ セラピューティクス， インコーポレイテッド プラコフィリン２遺伝子治療の方法および組成物
AU2022258329A1 (en) * 2021-04-12 2023-10-12 The Regents Of The University Of California Gene therapy for arrhythmogenic right ventricular cardiomyopathy
MX2024000175A (es) * 2021-07-08 2024-03-05 Tenaya Therapeutics Inc Casetes de expresión optimizada para genoterapia.
TW202404992A (zh) * 2022-04-11 2024-02-01 美商特納亞治療股份有限公司 用於斑菲素蛋白—２（ｐｌａｋｏｐｈｉｌｌｉｎ—２）基因療法之蛋白殼
JP2025512405A (ja) * 2022-04-11 2025-04-17 テナヤ セラピューティクス， インコーポレイテッド プラコフィリン２遺伝子治療の処置の方法
TW202421788A (zh) * 2022-09-22 2024-06-01 美商拜奧馬林製藥公司 用ａａｖ基因療法載體治療致心律不整性心肌病
TW202432835A (zh) * 2023-01-06 2024-08-16 美商航天七號有限責任公司 使用aav載體之斑菲素蛋白-2(pkp2)基因療法
EP4684025A1 (en) * 2023-03-22 2026-01-28 Skyline Therapeutics Limited Recombinant aav for the gene therapy of wilson disease

Family Cites Families (92)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US4861719A (en) 1986-04-25 1989-08-29 Fred Hutchinson Cancer Research Center DNA constructs for retrovirus packaging cell lines
EP0377676B1 (en) 1987-09-17 1995-05-24 Massachusetts Institute Of Technology Human erythroid-specific transcriptional enhancer
US5278056A (en) 1988-02-05 1994-01-11 The Trustees Of Columbia University In The City Of New York Retroviral packaging cell lines and process of using same
US5670488A (en) 1992-12-03 1997-09-23 Genzyme Corporation Adenovirus vector for gene therapy
AU8200191A (en) 1990-07-09 1992-02-04 United States of America, as represented by the Secretary, U.S. Department of Commerce, The High efficiency packaging of mutant adeno-associated virus using amber suppressions
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
WO1993003769A1 (en) 1991-08-20 1993-03-04 THE UNITED STATES OF AMERICA, represented by THE SECRETARY, DEPARTEMENT OF HEALTH AND HUMAN SERVICES Adenovirus mediated transfer of genes to the gastrointestinal tract
DE69434860T2 (de) 1993-02-22 2007-03-15 The Rockefeller University Herstellung von helfer-freien retroviren mit hohem titer mittels transienter transfektion
FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
FR2712812B1 (fr) 1993-11-23 1996-02-09 Centre Nat Rech Scient Composition pour la production de produits thérapeutiques in vivo.
IL116816A (en) 1995-01-20 2003-05-29 Rhone Poulenc Rorer Sa Cell for the production of a defective recombinant adenovirus or an adeno-associated virus and the various uses thereof
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6004797A (en) 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
US6027721A (en) 1996-05-20 2000-02-22 Cytotherapeutics, Inc. Device and method for encapsulated gene therapy
US6136597A (en) 1997-09-18 2000-10-24 The Salk Institute For Biological Studies RNA export element
US20020065236A1 (en) 1998-09-09 2002-05-30 Yew Nelson S. CpG reduced plasmids and viral vectors
US7994278B1 (en) 1999-08-06 2011-08-09 Nobel Biosciences Llc Biologically active polypeptides derived from a novel early stage pregnancy factor designated maternin (MA)
WO2001027304A2 (en) 1999-10-12 2001-04-19 Institut Pasteur Lentiviral triplex dna, and vectors and recombinant cells containing lentiviral triplex dna
AU2001255575B2 (en) 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US6627442B1 (en) 2000-08-31 2003-09-30 Virxsys Corporation Methods for stable transduction of cells with hiv-derived viral vectors
US20070037165A1 (en) * 2000-09-08 2007-02-15 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
GB0024550D0 (OSRAM) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US7575924B2 (en) 2000-11-13 2009-08-18 Research Development Foundation Methods and compositions relating to improved lentiviral vectors and their applications
WO2003012054A2 (en) 2001-08-02 2003-02-13 Institut Clayton De La Recherche Methods and compositions relating to improved lentiviral vector production systems
CA2462628C (en) 2001-10-02 2019-08-20 Institut Clayton De La Recherche Restricted expression lentiviral vectors
SG10202108118RA (en) 2001-11-13 2021-08-30 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
CA2483915A1 (en) 2002-05-02 2003-11-13 University Of Rochester Vectors having both isoforms of b-hexosaminidase
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
WO2004015106A1 (en) 2002-08-12 2004-02-19 UNIVERSITé DE SHERBROOKE Methods to reprogram splice site selection in pre-messenger rnas
DK1616012T3 (da) 2003-04-24 2008-04-21 San Raffaele Centro Fond Lentivirale vektorer bærende syntetiske bidirektionelle promotorer og anvendelser deraf
US7960100B1 (en) * 2004-04-30 2011-06-14 Celera Corporation Colon cancer targets and uses thereof
JP2007054069A (ja) 2005-08-24 2007-03-08 Medizinische Hochschule Hannover 自己不活性化レトロウイルスベクター
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
US8324367B2 (en) 2006-11-03 2012-12-04 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
EP2333091B1 (en) 2007-05-29 2017-06-21 Nature Technology Corporation Vectors and methods for genetic immunization
US8299215B2 (en) 2007-07-14 2012-10-30 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
WO2009126894A2 (en) * 2008-04-11 2009-10-15 Massachusetts Eye And Ear Infirmary Methods and compositions for the diagnosis and treatment of angiogenic disorders
AU2010241864B2 (en) 2009-04-30 2014-02-20 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Inducible interleukin-12
DE102009021592A1 (de) 2009-05-15 2010-11-18 Medizinische Hochschule Hannover ASLV-Vektorsystem
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
KR102057540B1 (ko) 2012-02-17 2019-12-19 더 칠드런스 호스피탈 오브 필라델피아 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법
JP2015527889A (ja) 2012-07-25 2015-09-24 ザ ブロード インスティテュート， インコーポレイテッド 誘導可能なｄｎａ結合タンパク質およびゲノム撹乱ツール、ならびにそれらの適用
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
GB201318347D0 (en) 2013-10-16 2013-11-27 Ucl Business Plc Retroviral vectors
WO2015060722A1 (en) 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
JP2017514476A (ja) 2014-05-01 2017-06-08 ユニバーシティ・オブ・ワシントン アデノウイルスベクターを用いたインビボでの遺伝子操作
EP3137497B2 (en) 2014-05-02 2025-03-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
US11030531B2 (en) 2014-06-06 2021-06-08 Trustees Of Boston University DNA recombinase circuits for logical control of gene expression
WO2015196179A1 (en) 2014-06-20 2015-12-23 University Of Florida Research Foundation, Inc. Methods of packaging multiple adeno-associated virus vectors
MA41346A (fr) 2015-01-12 2017-11-21 Juno Therapeutics Inc Eléments régulateurs post-transcriptionnels d'hépatite modifiée
JP6734283B2 (ja) 2015-01-21 2020-08-05 フレッド ハッチンソン キャンサー リサーチ センター 遺伝子治療用ポイントオブケア及び／又はポータブルプラットフォーム
ES2836258T3 (es) 2015-03-10 2021-06-24 Univ Columbia Construcciones de vectores virales adenoasociados de GLUT1 recombinante y métodos relacionados para restaurar la expresión de GLUT1
EA201792500A1 (ru) 2015-05-13 2018-04-30 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Aav-опосредованная экспрессия антител против гриппа и способы их использования
SG11201803934YA (en) * 2015-11-11 2018-06-28 Intrexon Corp Compositions and methods for expression of multiple biologically active polypeptides from a single vector for treatment of cardiac conditions and other pathologies
EP3875593A1 (en) 2015-12-11 2021-09-08 California Institute of Technology Targeting peptides for directing adeno-associated viruses (aavs)
PL3405215T3 (pl) 2016-01-19 2022-10-31 The Regents Of The University Of California Sposoby leczenia choroby danona i innych zaburzeń autofagii
WO2017136536A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
CN109641063B (zh) 2016-04-20 2022-11-15 能源环境和技术研究中心O.A., M.P. 用于增强pklr的基因表达的组合物和方法
MX2019002699A (es) 2016-09-08 2019-12-16 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Terapia genica para pacientes con anemia de fanconi.
WO2018060097A1 (en) 2016-09-30 2018-04-05 Laboratorios Del Dr. Esteve, S. A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
US20180326022A1 (en) 2017-04-21 2018-11-15 The Trustees Of The University Of Pennsylvania Compositions and methods for improving heart function and treating heart failure
JP2020517720A (ja) 2017-04-27 2020-06-18 フレッド ハッチンソン キャンサー リサーチ センター ネガティブ選択により誘導されるｃｄ３４＋幹細胞を含む治療用製剤
CA3062595A1 (en) 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
EP3684422B1 (en) * 2017-09-20 2023-12-20 The Regents of the University of California Connexin-43 gene therapy to restore electrical and cardiac function, and cardiac structure, in arrhythmogenic right ventricular cardiomyopathy
WO2019073058A1 (en) * 2017-10-12 2019-04-18 Universität Heidelberg GENE THERAPY OF CARDIAC ARRHYTHMIA BASED ON KCNK3
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
JP7576754B2 (ja) 2018-04-11 2024-11-01 スペースクラフト セブン リミテッド ライアビリティ カンパニー 幹細胞移植のための組成物および方法
AU2019261646A1 (en) * 2018-04-27 2020-11-12 Spacecraft Seven, Llc Gene therapy for CNS degeneration
CA3097375A1 (en) 2018-04-27 2019-10-31 Universitat Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
WO2020014523A1 (en) 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
MX2021001292A (es) 2018-07-30 2021-07-15 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Metodos para la modificacion genica de celulas hematopoyeticas.
US12612644B2 (en) 2018-08-10 2026-04-28 Regenxbio Inc. Scalable method for recombinant AAV production
CN112770768A (zh) 2018-08-16 2021-05-07 太空飞船七有限责任公司 用于病毒载体的生产方法
US20220088052A1 (en) 2019-01-22 2022-03-24 Koninklijke Nederlandse Akademie Van Wetenschappen Tfap2 inhibition for treating cardiac disease involving fibro-fatty replacement
CN120324641A (zh) * 2019-02-12 2025-07-18 太空飞船七有限责任公司 用于治疗达农病的基因治疗载体
CN113994008A (zh) 2019-05-23 2022-01-28 太空飞船七有限责任公司 用于婴儿恶性骨硬化病的基因治疗载体
US20220389452A1 (en) * 2019-09-20 2022-12-08 Ucl Business Ltd Gene therapy composition and treatment of right ventricular arrythmogenic cardiomyopathy
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
WO2021187380A1 (ja) 2020-03-16 2021-09-23 国立大学法人大阪大学 不整脈源性心筋症患者由来の多能性幹細胞およびその利用ならびに不整脈源性心筋症治療用医薬
KR20230004719A (ko) 2020-04-20 2023-01-06 테나야 테라퓨틱스, 인코포레이티드 조작된 캡시드를 갖는 아데노-연관 바이러스
US20230174994A1 (en) 2020-05-20 2023-06-08 Spacecraft Seven, Llc Engineered parkin and uses thereof
WO2022017630A1 (en) 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
WO2022031756A1 (en) 2020-08-05 2022-02-10 Spacecraft Seven, Llc Csrp3 (cysteine and glycine rich protein 3) gene therapy
JP7853952B2 (ja) 2020-08-05 2026-04-30 スペースクラフト セブン リミテッド ライアビリティ カンパニー Ｇｌｕｔ１発現のためのアデノ随伴ウイルスベクターおよびその使用
IL300263A (en) 2020-08-07 2023-03-01 Spacecraft Seven Llc Plakophilin-2 (PKP2) gene therapy using an AAV vector
JP7415042B2 (ja) 2020-10-09 2024-01-16 テナヤ セラピューティクス， インコーポレイテッド プラコフィリン２遺伝子治療の方法および組成物
KR20230129431A (ko) 2020-12-07 2023-09-08 스페이스크래프트 세븐, 엘엘씨 다농병의 치료
EP4308172A2 (en) 2021-03-19 2024-01-24 UCL Business Ltd Gene therapy composition and treatment of right ventricular arrhythmogenic cardiomyopathy
AU2022258329A1 (en) 2021-04-12 2023-10-12 The Regents Of The University Of California Gene therapy for arrhythmogenic right ventricular cardiomyopathy
MX2024000175A (es) 2021-07-08 2024-03-05 Tenaya Therapeutics Inc Casetes de expresión optimizada para genoterapia.

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
FRANCISCO M. CRUZ ET AL., JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY, vol. 65, no. 14, JPN6025031851, 2015, pages 1438 - 1450, ISSN: 0005655298 *
MAJA Z SALVA ET AL., MOLECULAR THERAPY, vol. 15, no. 2, JPN6025031852, 2007, pages 320 - 329, ISSN: 0005655299 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024512483A (ja) * 2021-03-19 2024-03-19 ユーシーエル ビジネス リミテッド 遺伝子治療組成物及び不整脈原性右室心筋症の治療

Also Published As

Publication number Publication date
US20240408233A1 (en) 2024-12-12
CA3185267A1 (en) 2022-02-10
KR20230043869A (ko) 2023-03-31
EP4192487A1 (en) 2023-06-14
BR112023001852A2 (pt) 2023-02-23
US11883506B2 (en) 2024-01-30
WO2022032226A1 (en) 2022-02-10
AU2021320902A1 (en) 2023-04-06
CN116194154A (zh) 2023-05-30
MX2023001615A (es) 2023-03-08
US20240390519A1 (en) 2024-11-28
US20220168446A1 (en) 2022-06-02
IL300263A (en) 2023-03-01
US20220168447A1 (en) 2022-06-02
EP4192487A4 (en) 2024-10-02

Similar Documents

Publication Publication Date Title
US11883506B2 (en) Plakophilin-2 (PKP2) gene therapy using AAV vector
US12257317B2 (en) Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
TWI856016B (zh) 腺相關病毒載體遞送肌肉特異性微小肌縮蛋白以治療肌肉萎縮症
KR102502091B1 (ko) β-사르코글리칸 및 마이크로RNA-29의 아데노-관련 바이러스 벡터 전달 및 근이영양증의 처리
US20230257431A1 (en) Csrp3 (cysteine and glycine rich protein 3) gene therapy
TW201840850A (zh) 腺相關病毒載體遞送肌肉特異性微小肌縮蛋白以治療肌肉萎縮症
JP2019513393A (ja) 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達
TW202409285A (zh) 使用ａａｖ載體之ｂ細胞淋巴瘤２相關抗凋亡３（ｂａｇ３）基因療法
US20240335566A1 (en) Troponin c (tnnc1) gene therapy using aav vector
JP2024546103A (ja) Ａａｖベクターを用いたジャンクトフィリン－２（ｊｐｈ２）遺伝子治療
KR20250133681A (ko) Aav 벡터를 이용한 플라코필린-2 (pkp2) 유전자 치료
US20250161483A1 (en) Methods and pharmaceutical compositions for the treatment and the prevention of cardiomyopathy associated with friedreich ataxia

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230425

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20230522

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240227

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240807

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240807

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250804

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20251031

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260120

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20260508