TWI908746B - 微小肌縮蛋白基因療法之構築體及其用途 - Google Patents

微小肌縮蛋白基因療法之構築體及其用途

Info

Publication number
TWI908746B
TWI908746B TW109141932A TW109141932A TWI908746B TW I908746 B TWI908746 B TW I908746B TW 109141932 A TW109141932 A TW 109141932A TW 109141932 A TW109141932 A TW 109141932A TW I908746 B TWI908746 B TW I908746B
Authority
TW
Taiwan
Prior art keywords
seq
aav
nucleic acid
acid sequence
protein
Prior art date
Application number
TW109141932A
Other languages
English (en)
Chinese (zh)
Other versions
TW202134260A (zh
Inventor
春平 喬
迪文 麥道加
曄 劉
奧利斐爾 達諾斯
Original Assignee
美商銳進科斯生物股份有限公司
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 美商銳進科斯生物股份有限公司 filed Critical 美商銳進科斯生物股份有限公司
Publication of TW202134260A publication Critical patent/TW202134260A/zh
Application granted granted Critical
Publication of TWI908746B publication Critical patent/TWI908746B/zh

Links

Classifications

    • 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
    • C07K14/4707—Muscular dystrophy
    • 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
    • C12N15/62—DNA sequences coding for fusion proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular 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
    • 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
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • 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
    • 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
    • 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
    • A01K2267/035—Animal model for multifactorial diseases
    • 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
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • 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
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

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)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
TW109141932A 2019-11-28 2020-11-27 微小肌縮蛋白基因療法之構築體及其用途 TWI908746B (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962941719P 2019-11-28 2019-11-28
US62/941,719 2019-11-28
US202063024933P 2020-05-14 2020-05-14
US63/024,933 2020-05-14

Publications (2)

Publication Number Publication Date
TW202134260A TW202134260A (zh) 2021-09-16
TWI908746B true TWI908746B (zh) 2025-12-21

Family

ID=74206146

Family Applications (1)

Application Number Title Priority Date Filing Date
TW109141932A TWI908746B (zh) 2019-11-28 2020-11-27 微小肌縮蛋白基因療法之構築體及其用途

Country Status (11)

Country Link
US (4) US12697399B2 (enExample)
EP (1) EP4065596A2 (enExample)
JP (2) JP7822933B2 (enExample)
KR (1) KR20220107222A (enExample)
AU (1) AU2020392243A1 (enExample)
BR (1) BR112022009895A2 (enExample)
CA (1) CA3159516A1 (enExample)
CL (1) CL2022001372A1 (enExample)
MX (1) MX2022006427A (enExample)
TW (1) TWI908746B (enExample)
WO (1) WO2021108755A2 (enExample)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11535868B2 (en) 2020-04-29 2022-12-27 Bristol-Myers Squibb Company Miniaturized dystrophins having spectrin fusion domains and uses thereof
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
IL307902A (en) * 2021-04-26 2023-12-01 Regenxbio Inc Microdystrophin gene therapy administration for treatment of dystrophinopathies
US20240269329A1 (en) * 2021-06-07 2024-08-15 University Of Florida Research Foundation, Incorporated Gene therapy for duchenne muscular dystrophy
CA3221707A1 (en) * 2021-06-23 2022-12-29 Jorge Omar YANEZ-CUNA Regulatory nucleic acid sequences
AU2022313258A1 (en) * 2021-07-19 2024-02-08 New York University Auf1 combination therapies for treatment of muscle degenerative disease
JP2024531138A (ja) * 2021-08-11 2024-08-29 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 筋ジストロフィーを処置するための組成物および方法
CA3228365A1 (en) * 2021-08-11 2023-02-16 Solid Biosciences Inc. Treatment of muscular dystrophy
US20240342314A1 (en) * 2021-09-30 2024-10-17 The Board Of Regents Of The University Of Texas System Slc13a5 gene therapy vectors and uses thereof
EP4413018A1 (en) 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for targeted delivery
CA3234162A1 (en) * 2021-10-15 2023-04-20 Michele Fiscella Antibodies and methods of using thereof
CA3248878A1 (en) * 2022-01-17 2023-07-20 Dinaqor Ag GENE THERAPY COMPOSITION AND TREATMENT OF DYSTROPHIN-RELATED CARDIOMYOPATHY
WO2023141582A1 (en) 2022-01-21 2023-07-27 Regenxbio Inc. Engineered promoters
EP4230196A1 (en) 2022-02-21 2023-08-23 Som Innovation Biotech, S.A. Compounds for use in the treatment of dystrophinopathies
US20250213727A1 (en) 2022-03-25 2025-07-03 Regenxbio Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
WO2023248251A1 (en) * 2022-06-24 2023-12-28 Indian Institute Of Technology Kanpur An optimized aav vector for gene therapy of muscular dystrophy
WO2024044725A2 (en) 2022-08-24 2024-02-29 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
JP2025534666A (ja) 2022-10-11 2025-10-17 リジェネックスバイオ インコーポレイテッド 操作された核酸調節エレメントならびにその使用方法
WO2024091495A1 (en) * 2022-10-24 2024-05-02 Biogen Ma Inc. Compositions and methods for treating retinitis pigmentosa
WO2024138158A2 (en) 2022-12-23 2024-06-27 Ultragenyx Pharmaceutical Inc. Method of producing soluble microdystrophin protein and uses of the same
CN115838725B (zh) * 2022-12-30 2023-09-08 广州派真生物技术有限公司 在哺乳动物心脏中特异性启动基因的启动子序列及其应用
EP4694904A2 (en) 2023-04-13 2026-02-18 REGENXBIO Inc. Targeting aav capsids, methods of manufacturing and using same
AR133921A1 (es) 2023-09-25 2025-11-12 Sarepta Therapeutics Inc Distrofinas humanas adaptadas con mayor afinidad de unión a la actina para el tratamiento de las distrofias musculares
TW202613313A (zh) 2024-05-31 2026-04-01 美商薩羅塔治療公司 肌向性ｒａａｖ
WO2026075999A2 (en) * 2024-10-01 2026-04-09 Sarepta Therapeutics, Inc. Dystrophin and micro-dystrophin antibodies and fragments thereof
WO2026080823A1 (en) 2024-10-10 2026-04-16 Research Institute At Nationwide Children's Hospital Methods for diagnosing and treating muscular dystrophy diseases

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016177911A1 (en) * 2015-05-07 2016-11-10 Royal Holloway & Bedford New College Production of large-sized microdystrophins in an aav-based vector configuration
CN107250364A (zh) * 2015-01-16 2017-10-13 华盛顿大学 新的微小肌养蛋白及相关使用方法

Family Cites Families (81)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6596535B1 (en) 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
WO2001083695A2 (en) 2000-04-28 2001-11-08 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
EP1366160B1 (en) 2000-10-06 2008-07-09 The Regents Of The University Of Michigan Mini-dystrophin nucleic acid and peptide sequences
CN103589692B (zh) * 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US7183969B2 (en) 2004-12-22 2007-02-27 Raytheon Company System and technique for calibrating radar arrays
DE23193606T1 (de) 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
JP4495210B2 (ja) 2005-06-09 2010-06-30 パナソニック株式会社 振幅誤差補償装置及び直交度誤差補償装置
WO2008088895A2 (en) 2007-01-18 2008-07-24 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma
AU2009215987B2 (en) 2008-02-19 2015-01-22 Uniqure Ip B.V. Optimisation of expression of parvoviral rep and cap proteins in insect cells
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
JP2013516389A (ja) 2009-01-06 2013-05-13 ダイアックス コーポレーション カリクレイン阻害剤による粘膜炎治療
DK2425000T3 (da) 2009-04-30 2019-05-13 Univ Pennsylvania Sammensætninger rettet mod ledende luftvejsceller omfattende konstrukter af adeno-associeret virus
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
US10738326B2 (en) 2010-10-27 2020-08-11 Jichi Medical University Adeno-associated virus vector for gene transfer to nervous system cells
KR102011156B1 (ko) 2011-01-06 2019-08-16 다이액스 코포레이션 혈장 칼리크레인 결합 단백질
CA2826273C (en) 2011-02-10 2021-11-02 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
EP3795581A3 (en) 2011-08-24 2021-06-09 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
US9624282B2 (en) 2012-11-26 2017-04-18 The Curators Of The University Of Missouri Microdystrophin peptides and methods for treating muscular dystrophy using the same
EP4234583A3 (en) 2013-01-20 2024-01-24 Takeda Pharmaceutical Company Limited Evaluation and treatment of bradykinin-mediated disorders
DK2946206T3 (en) 2013-01-20 2019-04-15 Dyax Corp EVALUATION, ANALYSIS AND TREATMENT OF PKAL-MEDIATED DISORDERS
EP2970946A4 (en) 2013-03-13 2016-09-07 Philadelphia Children Hospital ADENOASSOZED VIRUS VECTORS AND METHOD FOR USE THEREOF
JP2016513682A (ja) 2013-03-15 2016-05-16 ダイアックス コーポレーション 抗血漿カリクレイン抗体
US9862945B2 (en) 2013-04-20 2018-01-09 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs
NZ716102A (en) 2013-07-22 2021-12-24 The Children’S Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
HUE052676T2 (hu) 2013-10-11 2021-05-28 Massachusetts Eye & Ear Infirmary Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik
RS61993B1 (sr) 2013-12-03 2021-07-30 Hookipa Biotech Gmbh Cmv vakcine
ES3065185T3 (en) 2014-01-21 2026-05-04 Takeda Pharmaceuticals Co Plasma kallikrein binding proteins and uses thereof in treating hereditary angioedema
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
IL247942B (en) 2014-03-27 2022-09-01 Dyax Corp Compositions and methods for drops in macular edema as a result of diabetes
US10746742B2 (en) 2014-04-25 2020-08-18 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
WO2016049230A1 (en) 2014-09-24 2016-03-31 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
JP6754361B2 (ja) * 2014-12-16 2020-09-09 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
CA2972800A1 (en) 2015-01-02 2016-07-07 Dyax Corp. Bispecific antibodies against plasma kallikrein and factor xii
KR102554850B1 (ko) * 2015-02-06 2023-07-13 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도
CA3290174A1 (en) 2015-03-30 2026-03-02 Takeda Pharmaceutical Company Limited Plasma kallikrein inhibitors and uses thereof for preventing hereditary angioedema attack
JP6665466B2 (ja) 2015-09-26 2020-03-13 日亜化学工業株式会社 半導体発光素子及びその製造方法
EA201891002A1 (ru) 2015-10-19 2018-11-30 Дайэкс Корп. Иммунологический анализ для определения расщепленного высокомолекулярного кининогена
WO2017070491A1 (en) 2015-10-23 2017-04-27 Applied Genetic Technologies Corporation Ophthalmic formulations
US11286307B2 (en) 2015-12-11 2022-03-29 Takeda Pharmaceutical Company Limited Plasma kallikrein inhibitors and uses thereof for treating hereditary angioedema attack
EP4085934A1 (en) * 2015-12-11 2022-11-09 The Trustees of The University of Pennsylvania Gene therapy for treating familial hypercholesterolemia
WO2017181011A1 (en) 2016-04-15 2017-10-19 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of microrna-29 to treat muscular dystrophy
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
US11596699B2 (en) 2016-04-29 2023-03-07 CureVac SE RNA encoding an antibody
WO2017223128A1 (en) 2016-06-21 2017-12-28 The Curators Of The University Of Missouri Modified dystrophin proteins
CA2971303C (en) 2016-06-21 2026-03-03 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
JP7186400B2 (ja) 2016-08-30 2022-12-09 ボード オブ リージェンツ，ザ ユニバーシティ オブ テキサス システム ゲノム的に再コードした生物におけるセレノ－生物製剤の製造
WO2018053244A1 (en) 2016-09-16 2018-03-22 Dyax Corp. Protein biomarkers for diseases associated with the contact activation system
KR102569559B1 (ko) 2016-09-16 2023-08-23 다케다 파머수티컬 컴패니 리미티드 유전성 혈관부종에 대한 rna 바이오마커
IL314752A (en) 2016-09-16 2024-10-01 Takeda Pharmaceuticals Co Metabolite biomarkers for diseases associated with the contact activation system
CN110997923B (zh) 2017-03-17 2024-01-02 全国儿童医院研究所 腺相关病毒载体递送肌肉特异性微肌营养不良蛋白以治疗肌营养不良症
US11338045B2 (en) 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
US11447546B2 (en) 2017-07-20 2022-09-20 Nbe-Therapeutics Ag Human antibodies binding to ROR2
KR20200067195A (ko) 2017-10-18 2020-06-11 리젠엑스바이오 인크. 완전-인간 번역후 변형된 항체 치료제
CA3086942A1 (en) 2018-02-07 2019-08-15 Genethon Hybrid regulatory elements
AU2019221642A1 (en) 2018-02-14 2020-07-09 Generation Bio Co. Non-viral DNA vectors and uses thereof for antibody and fusion protein production
WO2019195362A1 (en) 2018-04-03 2019-10-10 Curators Of The University Of Missouri Hinges 1 and/or 4 modified dystrophins for dystrophinopathy therapy
CA3099958A1 (en) 2018-04-16 2019-10-24 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
IL324506A (en) 2018-06-18 2026-01-01 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector for delivery of muscle-specific microdystrophin for the treatment of muscular dystrophy
EP3843840A1 (en) 2018-08-30 2021-07-07 Dyax Corp. Plasma kallikrein inhibitors and uses thereof for treating hereditary angioedema attack
CN113613668A (zh) 2018-10-24 2021-11-05 百时美施贵宝公司 小型化肌营养不良蛋白及其用途
JP2022510218A (ja) 2018-11-30 2022-01-26 メモリアル スローン ケタリング キャンサー センター ヘテロ二量体四価特異性抗体およびこれらの使用
KR20210142006A (ko) 2019-03-14 2021-11-23 다케다 파머수티컬 컴패니 리미티드 유전성 혈관부종 발작의 치료를 위한 혈장 칼리크레인 저해제 및 이의 용도
EP3947427A1 (en) 2019-03-25 2022-02-09 Genethon Production of large-sized quasidystrophins using overlapping aav vectors
GB201905552D0 (en) 2019-04-18 2019-06-05 Kymab Ltd Antagonists
US20220195462A1 (en) 2019-04-24 2022-06-23 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
EP3966227A1 (en) 2019-05-07 2022-03-16 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
US20230038502A1 (en) 2019-06-11 2023-02-09 Shire Human Genetic Therapies, Inc. Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein
WO2020261178A1 (en) 2019-06-27 2020-12-30 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
EP4004214A1 (en) 2019-07-26 2022-06-01 RegenxBio Inc. Engineered nucleic acid regulatory element and methods of uses thereof
IL307902A (en) 2021-04-26 2023-12-01 Regenxbio Inc Microdystrophin gene therapy administration for treatment of dystrophinopathies
US20240358857A1 (en) 2021-05-11 2024-10-31 Regenxbio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
AU2022313258A1 (en) 2021-07-19 2024-02-08 New York University Auf1 combination therapies for treatment of muscle degenerative disease

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107250364A (zh) * 2015-01-16 2017-10-13 华盛顿大学 新的微小肌养蛋白及相关使用方法
WO2016177911A1 (en) * 2015-05-07 2016-11-10 Royal Holloway & Bedford New College Production of large-sized microdystrophins in an aav-based vector configuration

Also Published As

Publication number Publication date
KR20220107222A (ko) 2022-08-02
US20250367328A1 (en) 2025-12-04
US12697399B2 (en) 2026-08-04
JP2026027271A (ja) 2026-02-18
US20250367326A1 (en) 2025-12-04
US20250367327A1 (en) 2025-12-04
TW202134260A (zh) 2021-09-16
AU2020392243A1 (en) 2022-06-16
US20230270886A1 (en) 2023-08-31
EP4065596A2 (en) 2022-10-05
MX2022006427A (es) 2022-09-07
JP2023503637A (ja) 2023-01-31
CA3159516A1 (en) 2021-06-03
BR112022009895A2 (pt) 2022-08-09
CL2022001372A1 (es) 2023-01-13
JP7822933B2 (ja) 2026-03-03
WO2021108755A3 (en) 2021-07-08
WO2021108755A2 (en) 2021-06-03

Similar Documents

Publication Publication Date Title
US20250367327A1 (en) Treatment of dystrophinopathies with microdystrophin gene therapy constructs
US10301367B2 (en) Compositions and methods for treatment of muscular dystrophy
KR102427379B1 (ko) 헌팅톤 질환을 치료하기 위한 조성물 및 방법
US10286085B2 (en) Compositions and methods for treatment of muscular dystrophy
US20240409955A1 (en) Auf1 combination therapies for treatment of muscle degenerative disease
US20240216543A1 (en) Microdystrophin gene therapy administration for treatment of dystrophinopathies
JP2010516252A (ja) 筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子
CN114127296B (zh) Ube3a基因和表达盒及其应用
KR20240000542A (ko) 근이영양증의 유전자 요법을 위한 AAVrh74 벡터
JP2023521090A (ja) ＡＡＶ遺伝子治療のためのＣｐＧフリーＩＴＲ
US20240358857A1 (en) Treatment of duchenne muscular dystrophy and combinations thereof
WO2024020574A2 (en) Auf1 gene therapy for limb girdle muscular dystrophy
CN115485291A (zh) 具血影蛋白融合结构域的微型化抗肌萎缩蛋白及其用途
IL293334A (en) Microdystrophin gene therapy constructs and uses thereof
US20250312398A1 (en) Method for expressing a muscle-specific gene and cassettes for same
EP4680741A2 (en) Exon skipping gene therapy constructs, vectors and uses thereof