CA2946593A1 - Aav vectors for retinal and cns gene therapy - Google Patents

Aav vectors for retinal and cns gene therapy Download PDF

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Publication number
CA2946593A1
CA2946593A1 CA2946593A CA2946593A CA2946593A1 CA 2946593 A1 CA2946593 A1 CA 2946593A1 CA 2946593 A CA2946593 A CA 2946593A CA 2946593 A CA2946593 A CA 2946593A CA 2946593 A1 CA2946593 A1 CA 2946593A1
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Prior art keywords
amino acid
numbering
raav
nucleic acid
acid substitutions
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Pending
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CA2946593A
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English (en)
French (fr)
Inventor
Abraham Scaria
Jennifer Sullivan
Lisa M. Stanek
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Genzyme Corp
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Genzyme Corp
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=53189202&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CA2946593(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of CA2946593A1 publication Critical patent/CA2946593A1/en
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    • 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
    • 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
    • A61K48/0075Medicinal 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 delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/08Antiepileptics; Anticonvulsants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
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    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
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    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • A61P27/06Antiglaucoma agents or miotics
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
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    • AHUMAN NECESSITIES
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    • A61P9/00Drugs for disorders of the cardiovascular system
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01DNA viruses
    • C07K14/015Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
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    • 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
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    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14022New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
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  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
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  • Immunology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Epidemiology (AREA)
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  • Pain & Pain Management (AREA)
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CA2946593A 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy Pending CA2946593A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201461988131P 2014-05-02 2014-05-02
US61/988,131 2014-05-02
US201562114575P 2015-02-10 2015-02-10
US62/114,575 2015-02-10
PCT/US2015/028966 WO2015168666A2 (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy

Publications (1)

Publication Number Publication Date
CA2946593A1 true CA2946593A1 (en) 2015-11-05

Family

ID=53189202

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2946593A Pending CA2946593A1 (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy

Country Status (32)

Country Link
US (3) US10982228B2 (OSRAM)
EP (3) EP3913061A1 (OSRAM)
JP (4) JP6741591B2 (OSRAM)
KR (3) KR20160147974A (OSRAM)
CN (2) CN106661591B (OSRAM)
AU (3) AU2015252797C1 (OSRAM)
CA (1) CA2946593A1 (OSRAM)
CL (2) CL2016002713A1 (OSRAM)
CR (1) CR20160555A (OSRAM)
DK (1) DK3137497T4 (OSRAM)
DO (1) DOP2016000280A (OSRAM)
EA (1) EA034355B1 (OSRAM)
ES (1) ES2879636T5 (OSRAM)
FI (1) FI3137497T4 (OSRAM)
GT (1) GT201600227A (OSRAM)
HR (1) HRP20211024T4 (OSRAM)
HU (1) HUE054768T2 (OSRAM)
IL (2) IL292951B2 (OSRAM)
LT (1) LT3137497T (OSRAM)
MX (2) MX2016014220A (OSRAM)
MY (1) MY188987A (OSRAM)
PE (1) PE20170260A1 (OSRAM)
PH (1) PH12016502046A1 (OSRAM)
PL (1) PL3137497T5 (OSRAM)
PT (1) PT3137497T (OSRAM)
RS (1) RS62078B2 (OSRAM)
SG (3) SG10201912977RA (OSRAM)
SI (1) SI3137497T2 (OSRAM)
TW (1) TWI686476B (OSRAM)
UY (1) UY36106A (OSRAM)
WO (1) WO2015168666A2 (OSRAM)
ZA (1) ZA201606763B (OSRAM)

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