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SG11202100022SA - Gene therapy vectors for treatment of danon disease - Google Patents

Gene therapy vectors for treatment of danon disease

Info

Publication number
SG11202100022SA
SG11202100022SA SG11202100022SA SG11202100022SA SG11202100022SA SG 11202100022S A SG11202100022S A SG 11202100022SA SG 11202100022S A SG11202100022S A SG 11202100022SA SG 11202100022S A SG11202100022S A SG 11202100022SA SG 11202100022S A SG11202100022S A SG 11202100022SA
Authority
SG
Singapore
Prior art keywords
treatment
gene therapy
therapy vectors
danon disease
danon
Prior art date
Application number
SG11202100022SA
Inventor
Annahita Keravala
Simon Moore
David Ricks
Original Assignee
Rocket Pharmaceuticals Ltd
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 Rocket Pharmaceuticals Ltd filed Critical Rocket Pharmaceuticals Ltd
Publication of SG11202100022SA publication Critical patent/SG11202100022SA/en

Links

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70596Molecules with a "CD"-designation not provided for elsewhere
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/021Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/022Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from an adenovirus
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/04Uses of viruses as vector in vivo
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/80Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
    • C12N2810/85Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/001Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cardiology (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Urology & Nephrology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
SG11202100022SA 2018-07-12 2019-07-11 Gene therapy vectors for treatment of danon disease SG11202100022SA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862697302P 2018-07-12 2018-07-12
PCT/US2019/041465 WO2020014523A1 (en) 2018-07-12 2019-07-11 Gene therapy vectors for treatment of danon disease

Publications (1)

Publication Number Publication Date
SG11202100022SA true SG11202100022SA (en) 2021-01-28

Family

ID=69141704

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202100022SA SG11202100022SA (en) 2018-07-12 2019-07-11 Gene therapy vectors for treatment of danon disease

Country Status (12)

Country Link
US (2) US10703797B2 (en)
EP (1) EP3820537A4 (en)
JP (1) JP2024150588A (en)
KR (1) KR20210030965A (en)
CN (1) CN112512596A (en)
AU (1) AU2019299970A1 (en)
BR (1) BR112021000509A2 (en)
CA (1) CA3106010A1 (en)
IL (1) IL279918A (en)
MX (1) MX2021000443A (en)
SG (1) SG11202100022SA (en)
WO (1) WO2020014523A1 (en)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020167996A1 (en) * 2019-02-12 2020-08-20 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
WO2022026516A1 (en) * 2020-07-29 2022-02-03 The Board Of Regents Of The University Of Texas System Transgene cassettes, aav vectors, and aav viral vectors for expression of human codon-optimized cstb
MX2023001615A (en) 2020-08-07 2023-03-08 Spacecraft Seven Llc Plakophilin-2 (pkp2) gene therapy using aav vector.
WO2022067228A1 (en) * 2020-09-28 2022-03-31 U1 Bio, Inc. Trans-splicing system for tissue-specific replacement of rna sequences
EP4228610A1 (en) * 2020-10-13 2023-08-23 Kriya Therapeutics, Inc. Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer
CN116887868A (en) * 2020-12-07 2023-10-13 太空飞船七有限责任公司 Treatment of darinopathies
CA3224488A1 (en) * 2021-07-08 2023-01-12 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
EP4400590A1 (en) * 2021-09-08 2024-07-17 Kkoomlab Inc. Plasmid platform for stable expression and delivery of biomolecules
AU2022342997A1 (en) * 2021-09-08 2024-03-21 Kkoomlab Inc. Plasmid platform for stable expression and delivery of biomolecules

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US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
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
ATE237694T1 (en) 1991-08-20 2003-05-15 Us Gov Health & Human Serv ADENOVIRUS-MEDIATED GENE TRANSFER INTO THE GASTROINTESTINAL TRACT
US20020065236A1 (en) * 1998-09-09 2002-05-30 Yew Nelson S. CpG reduced plasmids and viral vectors
AU2003295881A1 (en) * 2002-11-25 2004-06-18 Exelixis, Inc. Lamps as modifiers of the p53 pathway and methods of use
WO2007091250A2 (en) 2006-02-06 2007-08-16 The Medical Research And Infrastructure Fund Of The Tel-Aviv Sourasky Medical Center Enzyme replacement therapy for treating lysosomal storage diseases
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
PL2158322T3 (en) 2007-06-06 2017-10-31 Genzyme Corp Gene therapy for lysosomal storage diseases
EP2571530A4 (en) * 2010-05-20 2014-03-05 Univ Rochester Methods and compositions related to modulating autophagy
ES2613691T3 (en) 2012-07-11 2017-05-25 Sangamo Biosciences, Inc. Methods and compositions for the treatment of lysosomal storage diseases
US10648001B2 (en) * 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
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JP7197466B2 (en) * 2016-09-08 2022-12-27 セントロ デ インベスティガシオンス エネルジェチカス メディオアンビエンタゥス イェ テクノロジカス オー.エイ. エム.ピー. Gene therapy for patients with Fanconi anemia
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WO2020167996A1 (en) * 2019-02-12 2020-08-20 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease

Also Published As

Publication number Publication date
MX2021000443A (en) 2021-05-28
BR112021000509A2 (en) 2021-04-06
CN112512596A (en) 2021-03-16
KR20210030965A (en) 2021-03-18
AU2019299970A1 (en) 2021-01-28
WO2020014523A1 (en) 2020-01-16
EP3820537A1 (en) 2021-05-19
US20210381003A1 (en) 2021-12-09
US10703797B2 (en) 2020-07-07
JP2021531752A (en) 2021-11-25
EP3820537A4 (en) 2022-04-13
CA3106010A1 (en) 2020-01-16
US20200148745A1 (en) 2020-05-14
IL279918A (en) 2021-03-01
JP2024150588A (en) 2024-10-23

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