ZA201701881B - Adeno-associated viral vectors for treating myocilin (myoc) glaucoma - Google Patents

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

Info

Publication number
ZA201701881B
ZA201701881B ZA2017/01881A ZA201701881A ZA201701881B ZA 201701881 B ZA201701881 B ZA 201701881B ZA 2017/01881 A ZA2017/01881 A ZA 2017/01881A ZA 201701881 A ZA201701881 A ZA 201701881A ZA 201701881 B ZA201701881 B ZA 201701881B
Authority
ZA
South Africa
Prior art keywords
myoc
spondin
adeno
glaucoma
myocilin
Prior art date
Application number
ZA2017/01881A
Other languages
English (en)
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
Priority claimed from PCT/US2015/050515 external-priority patent/WO2016044478A1/en
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of ZA201701881B publication Critical patent/ZA201701881B/en

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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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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
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • 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
    • AHUMAN NECESSITIES
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
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    • A61P27/02Ophthalmic agents
    • A61P27/06Antiglaucoma agents or miotics
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    • 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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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
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    • 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
    • C12N2750/14171Demonstrated in vivo effect

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
ZA2017/01881A 2014-09-16 2017-03-16 Adeno-associated viral vectors for treating myocilin (myoc) glaucoma ZA201701881B (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462051299P 2014-09-16 2014-09-16
PCT/US2015/050515 WO2016044478A1 (en) 2014-09-16 2015-09-16 Adeno-associated viral vectors for treating myocilin (myoc) glaucoma

Publications (1)

Publication Number Publication Date
ZA201701881B true ZA201701881B (en) 2023-09-27

Family

ID=58640637

Family Applications (1)

Application Number Title Priority Date Filing Date
ZA2017/01881A ZA201701881B (en) 2014-09-16 2017-03-16 Adeno-associated viral vectors for treating myocilin (myoc) glaucoma

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Country Link
US (1) US10821193B2 (enExample)
EP (1) EP3194601B1 (enExample)
JP (2) JP6673923B2 (enExample)
KR (2) KR102612871B1 (enExample)
CN (2) CN107001436B (enExample)
AU (2) AU2015317756B2 (enExample)
BR (1) BR112017005235B1 (enExample)
IL (1) IL251178B (enExample)
MX (2) MX2017003426A (enExample)
RU (2) RU2746991C2 (enExample)
SG (3) SG11201702055PA (enExample)
ZA (1) ZA201701881B (enExample)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201702055PA (en) 2014-09-16 2017-04-27 Genzyme Corp Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
WO2019155833A1 (ja) * 2018-02-07 2019-08-15 学校法人日本医科大学 改良型アデノ随伴ウイルスベクター
WO2019165241A1 (en) * 2018-02-22 2019-08-29 University Of South Florida Grp94 inhibitors to treat steroid-induced ocular hypertensions and glaucomas
CN113557010A (zh) 2019-02-28 2021-10-26 瑞泽恩制药公司 用于递送治疗剂的腺相关病毒载体
KR20230117177A (ko) * 2020-12-03 2023-08-07 유니버시티 오브 매사추세츠 진행성 골화성 섬유이형성증을 위한 신규 유전자 치료제의개발
CN113032010B (zh) * 2021-03-12 2022-09-20 歌尔科技有限公司 命令的传输控制方法、终端及计算机可读存储介质
MX2023014976A (es) * 2021-06-28 2024-02-09 Alnylam Pharmaceuticals Inc Composiciones y metodos para silenciar la expresion de miocilina (myoc).
WO2023114446A2 (en) * 2021-12-16 2023-06-22 Peter Koulen A machine learning-based framework using electroretinography for detecting early-stage glaucoma
CN117343152B (zh) * 2023-04-18 2024-06-04 上海本导基因技术有限公司 一种用于治疗青光眼疾病的慢病毒样颗粒
CN118546217B (zh) * 2024-07-25 2024-10-29 广州译码基因科技有限公司 靶向小梁网、角膜缘及rpe的aav衣壳蛋白及其应用

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AU2002342010A1 (en) 2001-10-03 2003-04-14 Incyte Genomics Inc. Secreted proteins
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
WO2008075796A1 (ja) * 2006-12-21 2008-06-26 Kyowa Hakko Kirin Co., Ltd. 血球回復促進剤
JP2010540564A (ja) * 2007-10-01 2010-12-24 アルコン リサーチ, リミテッド 自己相補的なaavが媒介する眼の障害を処置または予防するための干渉rna分子の送達
GB201106395D0 (en) * 2011-04-14 2011-06-01 Hubrecht Inst Compounds
KR20220056884A (ko) * 2011-04-22 2022-05-06 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 지니는 아데노-관련 바이러스 및 이의 사용 방법
EP2554662A1 (en) * 2011-08-05 2013-02-06 M Maria Pia Cosma Methods of treatment of retinal degeneration diseases
US20130209473A1 (en) * 2012-02-11 2013-08-15 Genentech, Inc. R-spondin translocations and methods using the same
JP6385920B2 (ja) * 2012-05-09 2018-09-05 オレゴン ヘルス アンド サイエンス ユニバーシティー アデノ随伴ウイルスプラスミド及びベクター
SG11201702055PA (en) 2014-09-16 2017-04-27 Genzyme Corp Adeno-associated viral vectors for treating myocilin (myoc) glaucoma

Also Published As

Publication number Publication date
MX2023005998A (es) 2023-06-08
JP2020105194A (ja) 2020-07-09
SG10201913430VA (en) 2020-03-30
US10821193B2 (en) 2020-11-03
IL251178A0 (en) 2017-05-29
EP3194601A1 (en) 2017-07-26
RU2718047C2 (ru) 2020-03-30
JP2017529395A (ja) 2017-10-05
KR102612871B1 (ko) 2023-12-13
EP3194601B1 (en) 2021-10-27
RU2020111038A (ru) 2020-04-29
AU2022202413A1 (en) 2022-05-05
RU2017112972A (ru) 2018-10-17
AU2015317756B2 (en) 2022-01-13
RU2746991C2 (ru) 2021-04-23
KR20230172610A (ko) 2023-12-22
IL251178B (en) 2022-07-01
JP6673923B2 (ja) 2020-03-25
MX2017003426A (es) 2017-07-28
CN107001436A (zh) 2017-08-01
KR20170054493A (ko) 2017-05-17
BR112017005235B1 (pt) 2023-10-31
RU2020111038A3 (enExample) 2020-10-15
SG11201702055PA (en) 2017-04-27
SG10201902285SA (en) 2019-04-29
AU2015317756A1 (en) 2017-04-13
RU2017112972A3 (enExample) 2019-04-16
CN116585492A (zh) 2023-08-15
US20170304465A1 (en) 2017-10-26
JP7097398B2 (ja) 2022-07-07
BR112017005235A2 (pt) 2017-12-12
CN107001436B (zh) 2023-01-06

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