SG11201705324UA - Novel micro-dystrophins and related methods of use - Google Patents

Novel micro-dystrophins and related methods of use

Info

Publication number
SG11201705324UA
SG11201705324UA SG11201705324UA SG11201705324UA SG11201705324UA SG 11201705324U A SG11201705324U A SG 11201705324UA SG 11201705324U A SG11201705324U A SG 11201705324UA SG 11201705324U A SG11201705324U A SG 11201705324UA SG 11201705324U A SG11201705324U A SG 11201705324UA
Authority
SG
Singapore
Prior art keywords
dystrophins
related methods
novel micro
micro
novel
Prior art date
Application number
SG11201705324UA
Inventor
Jeffrey S Chamberlain
Julian Ramos
Stephen D Hauschka
Original Assignee
Univ Washington
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 Univ Washington filed Critical Univ Washington
Publication of SG11201705324UA publication Critical patent/SG11201705324UA/en

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
    • C07K14/4708—Duchenne dystrophy
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • 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
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • 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
SG11201705324UA 2015-01-16 2016-01-15 Novel micro-dystrophins and related methods of use SG11201705324UA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562104537P 2015-01-16 2015-01-16
PCT/US2016/013733 WO2016115543A2 (en) 2015-01-16 2016-01-15 Novel micro-dystrophins and related methods of use

Publications (1)

Publication Number Publication Date
SG11201705324UA true SG11201705324UA (en) 2017-07-28

Family

ID=56406571

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201705324UA SG11201705324UA (en) 2015-01-16 2016-01-15 Novel micro-dystrophins and related methods of use

Country Status (11)

Country Link
US (3) US10479821B2 (en)
EP (1) EP3245291A4 (en)
JP (2) JP6832280B2 (en)
KR (1) KR20170107481A (en)
CN (1) CN107250364A (en)
AU (2) AU2016206518B2 (en)
CA (1) CA2972811A1 (en)
IL (3) IL297239A (en)
NZ (1) NZ734019A (en)
SG (1) SG11201705324UA (en)
WO (1) WO2016115543A2 (en)

Families Citing this family (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10479821B2 (en) 2015-01-16 2019-11-19 University Of Washington Micro-dystrophins and related methods of use
AU2016344609B2 (en) 2015-10-28 2022-05-12 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of duchenne muscular dystrophy
MA45477A (en) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital ADENOASSOCIATED VIRUS VECTOR VECTOR MICROARN-29 AND MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY
US20170362635A1 (en) * 2016-06-20 2017-12-21 University Of Washington Muscle-specific crispr/cas9 editing of genes
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
JOP20190166A1 (en) * 2017-01-05 2019-07-02 Univ Texas Optimized strategy for exon skipping modifications using crispr/cas9 with triple guide sequences
CA3050910A1 (en) 2017-01-23 2018-07-26 Rutgers, The State University Of New Jersey Compositions for reducing sarcolipin expression and preventing and treating muscular dystrophy and cardiomyopathy and methods of use
US10687520B2 (en) 2017-03-07 2020-06-23 The Board Of Regents Of The University Of Texas System Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44
EP3596112A2 (en) * 2017-03-17 2020-01-22 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
JP7213238B2 (en) 2017-10-18 2023-01-26 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 Adeno-associated viral vector delivery of muscle-specific microdystrophins to treat muscular dystrophy
TW201936201A (en) 2017-12-14 2019-09-16 美商堅固生物科技公司 Non-viral production and delivery of genes
CA3099958A1 (en) * 2018-04-16 2019-10-24 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
JP7450945B2 (en) 2018-08-30 2024-03-18 テナヤ セラピューティクス， インコーポレイテッド Cardiac cell reprogramming using myocardin and ASCL1
EA202191123A1 (en) * 2018-10-24 2022-03-29 Бристол-Майерс Сквибб Компани MINIATURIZED DYSTROPHINS AND THEIR APPLICATIONS
CN113646004A (en) * 2018-12-12 2021-11-12 坚固生物科技公司 Combination therapy for the treatment of muscular dystrophy
EP3976806A1 (en) 2019-05-30 2022-04-06 Solidus Biosciences, Inc. Recombinant herpesvirales vector
US20200405824A1 (en) * 2019-06-26 2020-12-31 University Of Washington Use of ribonucleotide reductase alone or in combination with micro-dystrophin to treat duchenne muscular dystrophy striated muscle disease
US20230270886A1 (en) * 2019-11-28 2023-08-31 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
CA3171166A1 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
WO2021163357A2 (en) * 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
WO2021222332A1 (en) 2020-04-29 2021-11-04 Bristol-Myers Squibb Company Miniaturized dystrophins having spectrin fusion domains and uses thereof
EP4164668A1 (en) 2020-06-15 2023-04-19 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery for muscular dystrophies
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
US20240003898A1 (en) 2020-10-30 2024-01-04 Pfizer Inc. Methods for measuring dystrophin in tissue samples
RU2767335C1 (en) * 2021-03-02 2022-03-17 Общество с ограниченной ответственностью «Марлин Биотех» Chimeric proteins based on human utrophin and dystrophin and use thereof for treating duchenne muscular dystrophy
CA3218333A1 (en) 2021-04-05 2022-10-13 Solid Biosciences Inc. Recombinant herpesvirales vector
JP2024513907A (en) * 2021-04-09 2024-03-27 ユニバーシティ オブ ワシントン Artificial regulatory cassettes for muscle-specific gene expression
BR112023021999A2 (en) * 2021-04-26 2023-12-26 Regenxbio Inc ADMINISTRATION OF MICRODYSTROPHIN GENE THERAPY FOR THE TREATMENT OF DYSTROPHINOPATHIES
EP4337267A1 (en) * 2021-05-11 2024-03-20 RegenxBio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
EP4108263A3 (en) 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
WO2022261143A2 (en) * 2021-06-07 2022-12-15 University Of Florida Research Foundation, Incorporated Gene therapy for duchenne muscular dystrophy
WO2023018854A2 (en) 2021-08-11 2023-02-16 Solid Biosciences Inc. Treatment of muscular dystrophy
WO2023019168A1 (en) 2021-08-11 2023-02-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
WO2023060269A1 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
AU2022380841A1 (en) 2021-11-04 2024-05-02 Regeneron Pharmaceuticals, Inc. Viral particles retargeted to skeletal muscle
CN114316070B (en) * 2021-12-29 2022-11-15 上海勉亦生物科技有限公司 Transgenic expression cassettes for the treatment of muscular dystrophy
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
WO2023212193A1 (en) * 2022-04-27 2023-11-02 The Curators Of The University Of Missouri Micro-dystrophin for heart protection

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2001296600A1 (en) * 2000-10-06 2002-04-15 Regents Of The University Of Michigan Mini-dystrophin nucleic acid and peptide sequences
US20070202587A1 (en) 2002-09-23 2007-08-30 Applied Genetic Technologies Corporation Recombinant AAV production in mammalian cells
PT3211085T (en) 2003-09-30 2021-06-17 Univ Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US20080044393A1 (en) * 2004-07-16 2008-02-21 White Robert L Retinal dystrophin transgene and methods of use thereof
US7436622B2 (en) * 2006-07-31 2008-10-14 Imation Corp. Concurrent servo and data track writing
US7892824B2 (en) * 2007-01-18 2011-02-22 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma
AU2009209408B2 (en) 2008-01-29 2015-06-11 Beacon Therapeutics Limited Recombinant virus production using mammalian cells in suspension
KR101555083B1 (en) * 2008-11-11 2015-09-22 인터내셔널 비지네스 머신즈 코포레이션 Data providing device, system, server device, program, and method
AU2012258525B2 (en) * 2011-05-26 2017-02-23 University Of Washington Cell and gene based methods to improve cardiac function
US20130136729A1 (en) 2011-11-11 2013-05-30 University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus 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
US20150329609A1 (en) * 2014-03-03 2015-11-19 Sujata Acharjee Chimeric dystrophin proteins to treat dystrophinopathies
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
US10479821B2 (en) 2015-01-16 2019-11-19 University Of Washington Micro-dystrophins and related methods of use

Also Published As

Publication number Publication date
AU2016206518B2 (en) 2020-03-05
JP6832280B2 (en) 2021-02-24
IL286316A (en) 2021-10-31
IL286316B (en) 2022-11-01
US11958888B2 (en) 2024-04-16
KR20170107481A (en) 2017-09-25
JP2022191404A (en) 2022-12-27
EP3245291A4 (en) 2018-09-19
US10479821B2 (en) 2019-11-19
US20200095298A1 (en) 2020-03-26
IL253246B (en) 2021-09-30
WO2016115543A2 (en) 2016-07-21
NZ734019A (en) 2023-01-27
AU2020203358A1 (en) 2020-06-11
IL253246A0 (en) 2017-08-31
JP2018503374A (en) 2018-02-08
IL286316B2 (en) 2023-03-01
US20200031890A1 (en) 2020-01-30
AU2020203358B2 (en) 2022-03-31
AU2016206518A1 (en) 2017-08-10
CA2972811A1 (en) 2016-07-21
JP2021072839A (en) 2021-05-13
CN107250364A (en) 2017-10-13
IL297239A (en) 2022-12-01
EP3245291A2 (en) 2017-11-22
US20180148488A1 (en) 2018-05-31
JP7164896B2 (en) 2022-11-02
WO2016115543A3 (en) 2016-09-29

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