HUE070278T2 - Adeno-associated viral vectors for treating myocilin (myoc) glaucoma - Google Patents

Adeno-associated viral vectors for treating myocilin (myoc) glaucoma

Info

Publication number
HUE070278T2
HUE070278T2 HUE21204257A HUE21204257A HUE070278T2 HU E070278 T2 HUE070278 T2 HU E070278T2 HU E21204257 A HUE21204257 A HU E21204257A HU E21204257 A HUE21204257 A HU E21204257A HU E070278 T2 HUE070278 T2 HU E070278T2
Authority
HU
Hungary
Prior art keywords
myoc
adeno
glaucoma
viral vectors
associated viral
Prior art date
Application number
HUE21204257A
Other languages
Hungarian (hu)
Inventor
Peter Pechan
Abraham Scaria
Jeffery Ardinger
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Publication of HUE070278T2 publication Critical patent/HUE070278T2/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
HUE21204257A 2014-09-16 2015-09-16 Adeno-associated viral vectors for treating myocilin (myoc) glaucoma HUE070278T2 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201462051229P 2014-09-16 2014-09-16

Publications (1)

Publication Number Publication Date
HUE070278T2 true HUE070278T2 (en) 2025-05-28

Family

ID=54238590

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE21204257A HUE070278T2 (en) 2014-09-16 2015-09-16 Adeno-associated viral vectors for treating myocilin (myoc) glaucoma

Country Status (5)

Country Link
ES (1) ES3011359T3 (en)
HU (1) HUE070278T2 (en)
PL (1) PL4012035T3 (en)
PT (1) PT4012035T (en)
WO (1) WO2016044478A1 (en)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190262373A1 (en) * 2016-08-16 2019-08-29 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
EP4133078A1 (en) * 2020-04-06 2023-02-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
US20230381341A1 (en) * 2020-10-07 2023-11-30 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
CA3220800A1 (en) * 2021-06-03 2022-12-08 Sylvain LAPAN Capsid variants and methods of using the same
JP2024526234A (en) * 2021-06-28 2024-07-17 アルナイラム ファーマシューティカルズ， インコーポレイテッド Compositions and methods for silencing myoc expression

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6225291B1 (en) 1997-04-21 2001-05-01 University Of Florida Rod opsin mRNA-specific ribozyme compositions and methods for the treatment of retinal diseases
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
PT1916258E (en) 1999-08-09 2014-07-29 Genzyme Corp Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
NZ522840A (en) 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
GB0403600D0 (en) * 2004-02-18 2004-03-24 Trinity College Dublin Methods and reagents for treating disease
WO2005117938A2 (en) * 2004-04-13 2005-12-15 Regents Of The University Of Minnesota Methods of treating ocular conditions
US7765583B2 (en) 2005-02-28 2010-07-27 France Telecom System and method for managing virtual user domains
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
RU2010117178A (en) * 2007-10-01 2011-11-10 Алькон Рисерч, Лтд. (Us) SELF-COMPLETE AAV DELIVERED DELIVERY OF MOLECULES INTERFERING RNA FOR TREATMENT OR PREVENTION OF EYE DISEASES
US20100081707A1 (en) 2008-02-21 2010-04-01 Ali Robin R Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
DK2675902T3 (en) 2011-02-17 2019-06-03 Univ Pennsylvania COMPOSITIONS AND METHODS OF CHANGING TISSUE SPECIFICITY AND IMPROVING AAV9 MEDIUM TRANSFER

Also Published As

Publication number Publication date
WO2016044478A1 (en) 2016-03-24
PT4012035T (en) 2025-02-11
PL4012035T3 (en) 2025-03-24
ES3011359T3 (en) 2025-04-07

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