JP2011511090A - 治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法 - Google Patents

治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法 Download PDF

Info

Publication number
JP2011511090A
JP2011511090A JP2010546007A JP2010546007A JP2011511090A JP 2011511090 A JP2011511090 A JP 2011511090A JP 2010546007 A JP2010546007 A JP 2010546007A JP 2010546007 A JP2010546007 A JP 2010546007A JP 2011511090 A JP2011511090 A JP 2011511090A
Authority
JP
Japan
Prior art keywords
cells
aav
vector
retinal
eye
Prior art date
Legal status (The legal status 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 status listed.)
Ceased
Application number
JP2010546007A
Other languages
English (en)
Japanese (ja)
Other versions
JP2011511090A5 (enExample
Inventor
レイモンド ティー. バータス
キャシー エム. ビショップ
メディ ガスミ
Original Assignee
セレジーン インコーポレイテッド
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 セレジーン インコーポレイテッド filed Critical セレジーン インコーポレイテッド
Publication of JP2011511090A publication Critical patent/JP2011511090A/ja
Publication of JP2011511090A5 publication Critical patent/JP2011511090A5/ja
Ceased legal-status Critical Current

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • 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
    • A61K38/18Growth factors; Growth regulators
    • A61K38/185Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • 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
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • A61K9/0051Ocular inserts, ocular implants
    • 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
    • C12N2799/025Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2010546007A 2008-02-07 2009-02-05 治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法 Ceased JP2011511090A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US2699008P 2008-02-07 2008-02-07
US9322808P 2008-08-29 2008-08-29
PCT/US2009/033275 WO2009100253A1 (en) 2008-02-07 2009-02-05 Rescue of photoreceptors by intravitreal administation of an expression vector encoding a therapeutic protein

Publications (2)

Publication Number Publication Date
JP2011511090A true JP2011511090A (ja) 2011-04-07
JP2011511090A5 JP2011511090A5 (enExample) 2013-03-28

Family

ID=40939054

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2010546007A Ceased JP2011511090A (ja) 2008-02-07 2009-02-05 治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法

Country Status (7)

Country Link
US (2) US8242093B2 (enExample)
EP (1) EP2247258A4 (enExample)
JP (1) JP2011511090A (enExample)
CN (1) CN101980668A (enExample)
AU (1) AU2009212309B2 (enExample)
CA (1) CA2713755A1 (enExample)
WO (1) WO2009100253A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2009212309B2 (en) * 2008-02-07 2014-04-03 Ceregene, Inc. Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein
GB201103062D0 (en) * 2011-02-22 2011-04-06 Isis Innovation Method
CN103459593A (zh) * 2011-02-25 2013-12-18 独立行政法人理化学研究所 视网膜色素上皮细胞片层的生产方法
CA2878171C (en) * 2012-07-11 2021-04-27 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
US20160256571A1 (en) * 2013-09-30 2016-09-08 Sanofi Invention
WO2015082690A1 (en) * 2013-12-06 2015-06-11 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject
US10293059B2 (en) * 2015-04-09 2019-05-21 Cornell University Gene therapy to prevent reactions to allergens
US11390866B2 (en) * 2015-12-04 2022-07-19 Sorbonne Université Promoters and uses thereof
EP3448411A1 (en) * 2016-04-29 2019-03-06 Gensight Biologics SA Optogenetic visual restoration using chrimson
PL239642B1 (pl) * 2018-08-16 2021-12-20 Pomorski Univ Medyczny W Szczecinie Kompozycja do doszklistkowego podawania białka leczniczego
CN116916937A (zh) * 2020-10-22 2023-10-20 维斯基因股份有限公司 用于人类基因疗法的elovl2构建体
WO2024123461A1 (en) * 2022-12-05 2024-06-13 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Pigment epithelium-derived factor peptides and use for treating retinal degeneration

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002512627A (ja) * 1997-06-11 2002-04-23 アコーダ セラピーティックス Cns神経再生組成物及びその使用方法
JP2002539176A (ja) * 1999-03-15 2002-11-19 カイロン コーポレイション 眼の疾患を処置または予防するための組換え遺伝子送達ベクターの使用

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5932479A (en) 1988-09-26 1999-08-03 Auburn University Genetic engineering of plant chloroplasts
JP2002534139A (ja) * 1999-01-05 2002-10-15 マサチューセッツ・アイ・アンド・イア・インファーマリー 網膜と脈絡膜への経強膜徐放性薬剤標的送達
US6943153B1 (en) * 1999-03-15 2005-09-13 The Regents Of The University Of California Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
CA2401855A1 (en) 2000-02-29 2001-09-07 Auburn University Genetic engineering of drought tolerance via a plastid genome
CA2402066C (en) 2000-03-01 2015-09-29 Auburn University Plastid transformation vectors for expressing human proteins in plants
US7354760B2 (en) 2001-12-26 2008-04-08 University Of Central Florida Research Foundation, Inc. Expression of protective antigens in transgenic chloroplasts
AU2003253781A1 (en) 2002-07-03 2004-01-23 University Of Central Florida Expression of human interferon in transgenic chloroplasts
US20100172871A1 (en) * 2005-02-17 2010-07-08 Flannery John G Muller Cell Specific Gene Therapy
US20060188481A1 (en) * 2005-02-22 2006-08-24 Saitama Medical School Methods for prophylactically or therapeutically treating an animal for an ocular-related disorder
JP2008537478A (ja) * 2005-02-24 2008-09-18 ザ・スクリプス・リサーチ・インステイチユート 単離骨髄球様骨髄細胞集団及びそれを用いた治療方法
AU2006309281B2 (en) 2005-05-27 2011-11-03 University Of Central Florida Chloroplasts engineered to express pharmaceutical proteins
AU2009212309B2 (en) * 2008-02-07 2014-04-03 Ceregene, Inc. Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002512627A (ja) * 1997-06-11 2002-04-23 アコーダ セラピーティックス Cns神経再生組成物及びその使用方法
JP2002539176A (ja) * 1999-03-15 2002-11-19 カイロン コーポレイション 眼の疾患を処置または予防するための組換え遺伝子送達ベクターの使用

Also Published As

Publication number Publication date
CN101980668A (zh) 2011-02-23
WO2009100253A8 (en) 2010-08-19
AU2009212309B2 (en) 2014-04-03
WO2009100253A1 (en) 2009-08-13
US8420619B2 (en) 2013-04-16
AU2009212309A1 (en) 2009-08-13
US8242093B2 (en) 2012-08-14
US20090202505A1 (en) 2009-08-13
EP2247258A1 (en) 2010-11-10
US20120316112A1 (en) 2012-12-13
CA2713755A1 (en) 2009-08-13
EP2247258A4 (en) 2012-01-18

Similar Documents

Publication Publication Date Title
JP2011511090A (ja) 治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法
Lau et al. Retinal degeneration is slowed in transgenic rats by AAV-mediated delivery of FGF-2
JP7190352B2 (ja) 認知保護を提供しつつ疾患の発症及び進行を遅らせるための遺伝子治療を用いた神経変性疾患の治療方法
CN107405507B (zh) 用于将多核苷酸玻璃体内递送到视网膜视锥的组合物和方法
JP7766393B2 (ja) 眼疾患のための遺伝子療法
JP7671790B2 (ja) 眼疾患の遺伝子治療
JP7227765B2 (ja) 網膜変性疾患を治療するための遺伝子治療
JP7618287B2 (ja) 医薬の製造におけるCYP4V2およびRdCVFの使用
JP2001527399A (ja) 網膜疾患の処置のための材料および方法
US11845952B2 (en) Adeno-associated virus vector
JP2024105661A (ja) 眼疾患に対するadeno随伴ウイルス連ウイルスベクター媒介遺伝子治療
JP2025503495A (ja) 錐体光受容体細胞を産生するためのプロセス
Chekuri et al. AAV2-mediated intravitreal delivery of exon-specific U1 snRNA rescues optic neuropathy in a mouse model of familial dysautonomia
CN113795279B (zh) 靶向akt通路的神经保护性基因疗法
WO2020163250A1 (en) Cx3cl1 compositions and methods for the treatment of degenerative ocular diseases
JP2025500979A (ja) 光受容体細胞のインビボリプログラミング

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20120123

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20120123

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20130116

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20130520

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20130816

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20130823

A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20140519

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20140606

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20140611

A045 Written measure of dismissal of application [lapsed due to lack of payment]

Free format text: JAPANESE INTERMEDIATE CODE: A045

Effective date: 20140925