ZA201606763B - Aav vectors for retinal and cns gene therapy - Google Patents

Aav vectors for retinal and cns gene therapy

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Publication number
ZA201606763B
ZA201606763B ZA2016/06763A ZA201606763A ZA201606763B ZA 201606763 B ZA201606763 B ZA 201606763B ZA 2016/06763 A ZA2016/06763 A ZA 2016/06763A ZA 201606763 A ZA201606763 A ZA 201606763A ZA 201606763 B ZA201606763 B ZA 201606763B
Authority
ZA
South Africa
Prior art keywords
retinal
gene therapy
aav vectors
cns gene
cns
Prior art date
Application number
ZA2016/06763A
Other languages
English (en)
Inventor
Abraham Scaria
Jennifer Sullivan
Lisa M Stanek
Lamya Shihabuddin
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
Family has litigation
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=ZA201606763(B) "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 ZA201606763B publication Critical patent/ZA201606763B/en

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    • 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
    • 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
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    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
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    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs 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
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
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    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • C12N15/113—Non-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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors

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ZA2016/06763A 2014-05-02 2016-09-30 Aav vectors for retinal and cns gene therapy ZA201606763B (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461988131P 2014-05-02 2014-05-02
US201562114575P 2015-02-10 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
ZA201606763B true ZA201606763B (en) 2023-05-31

Family

ID=53189202

Family Applications (1)

Application Number Title Priority Date Filing Date
ZA2016/06763A ZA201606763B (en) 2014-05-02 2016-09-30 Aav vectors for retinal and cns gene therapy

Country Status (32)

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US (3) US10982228B2 (OSRAM)
EP (3) EP4600255A3 (OSRAM)
JP (4) JP6741591B2 (OSRAM)
KR (3) KR102863726B1 (OSRAM)
CN (2) CN114231563B (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) IL248102B (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) SG11201607991WA (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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