PT2271369E - Novas ferramentas terapêuticas e métodos para tratar a cegueira - Google Patents

Novas ferramentas terapêuticas e métodos para tratar a cegueira Download PDF

Info

Publication number
PT2271369E
PT2271369E PT97315691T PT09731569T PT2271369E PT 2271369 E PT2271369 E PT 2271369E PT 97315691 T PT97315691 T PT 97315691T PT 09731569 T PT09731569 T PT 09731569T PT 2271369 E PT2271369 E PT 2271369E
Authority
PT
Portugal
Prior art keywords
methods
treating blindness
tools
novel therapeutical
therapeutical tools
Prior art date
Application number
PT97315691T
Other languages
English (en)
Inventor
David Balya
Volker Busskamp
Pamela Lagali
Botond Roska
Original Assignee
Novartis Forschungsstiftung
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 Novartis Forschungsstiftung filed Critical Novartis Forschungsstiftung
Publication of PT2271369E publication Critical patent/PT2271369E/pt

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/164—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • 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
    • 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
    • A61K48/0075—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C07K14/215—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria from Halobacteriaceae (F)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
PT97315691T 2008-04-18 2009-04-16 Novas ferramentas terapêuticas e métodos para tratar a cegueira PT2271369E (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP08154828 2008-04-18
EP08155942 2008-05-09

Publications (1)

Publication Number Publication Date
PT2271369E true PT2271369E (pt) 2014-07-10

Family

ID=40626866

Family Applications (1)

Application Number Title Priority Date Filing Date
PT97315691T PT2271369E (pt) 2008-04-18 2009-04-16 Novas ferramentas terapêuticas e métodos para tratar a cegueira

Country Status (14)

Country Link
US (4) US20120258530A1 (pt)
EP (2) EP2679246A3 (pt)
JP (2) JP2011516091A (pt)
KR (1) KR20100136550A (pt)
CN (1) CN101970013B (pt)
AU (1) AU2009237585C1 (pt)
BR (1) BRPI0911426A2 (pt)
CA (1) CA2721635A1 (pt)
EA (1) EA201001621A1 (pt)
ES (1) ES2474721T3 (pt)
MX (1) MX2010011467A (pt)
PL (1) PL2271369T3 (pt)
PT (1) PT2271369E (pt)
WO (1) WO2009127705A1 (pt)

Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20100136550A (ko) 2008-04-18 2010-12-28 노파르티스 포르슝스티프퉁 쯔바이크니덜라쑹 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 실명을 치료하기 위한 신규의 치료 도구 및 방법
EP2315833B8 (en) 2008-05-20 2015-05-27 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
EP2483394A1 (en) * 2009-09-29 2012-08-08 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research Functionalized organotypic systems
US9545422B2 (en) * 2010-03-01 2017-01-17 The Regents Of The University Of California Methods of inducing photosensitivity by targeting channelrhodopsin-2 and halorhodopsin to subcellular regions of retinal ganglion cells
GB2492719A (en) 2010-04-05 2013-01-09 Eos Neuroscience Inc Methods and compositions for decreasing chronic pain
EP2383286A1 (en) 2010-04-30 2011-11-02 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and compositions for treatment of retinal degenerative diseases
US8868351B2 (en) 2011-10-13 2014-10-21 The Cleveland Clinic Foundation Estimation of neural response for optical stimulation
ES2660114T3 (es) * 2011-11-12 2018-03-20 Massachusetts Institute Of Technology Canalrodopsinas para el control óptico de células
US20150038557A1 (en) * 2012-02-24 2015-02-05 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods and compositions for treatment of retinal degenerative diseases
EP2888368A1 (en) 2012-08-27 2015-07-01 Friedrich Miescher Institute for Biomedical Research Retinal off circuit-specific promoter
WO2014199299A1 (en) * 2013-06-11 2014-12-18 Friedrich Miescher Institute For Biomedical Research Retinal on bipolar cells-specific artificial promoter
JP2017501834A (ja) * 2014-01-08 2017-01-19 サーキット セラピューティクス， インコーポレイテッド 高眼圧症の治療管理のための方法
GB201403260D0 (en) * 2014-02-25 2014-04-09 Univ Manchester Treatment of retinal degeneration using gene therapy
GB201503008D0 (en) * 2015-02-23 2015-04-08 Ucl Business Plc Treatment
ES2747433T3 (es) 2015-04-30 2020-03-10 Friedrich Miescher Institute For Biomedical Res Promotor para la expresión específica de genes en células de Müller
EP3350330B1 (en) * 2015-09-15 2022-06-22 Friedrich Miescher Institute for Biomedical Research Novel therapeutical tools and methods for treating blindness by targeting photoreceptors
WO2017064642A1 (en) 2015-10-14 2017-04-20 Fredrich Miescher Institute For Biomedical Research Promoter for the specific expression of genes in retinal endothelial cells
US10995344B2 (en) 2015-12-03 2021-05-04 Friedrich Miescher Institute For Biomedical Research SYNP159, a promoter for the specific expression of genes in rod photoreceptors
WO2017093936A1 (en) 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp162, a promoter for the specific expression of genes in rod photoreceptors
EP3384033B1 (en) * 2015-12-03 2021-09-08 Friedrich Miescher Institute for Biomedical Research Synp160, a promoter for the specific expression of genes in rod photoreceptors
BR112018011059A2 (pt) * 2015-12-03 2018-11-21 Friedrich Miescher Institute For Biomedical Research synp161, um promotor para a expressão específica de genes em fotoreceptores de haste
CA3025975A1 (en) 2016-04-29 2017-11-02 Gensight Biologics Sa Optogenetic visual restoration using chrimson
KR20190020702A (ko) * 2016-06-03 2019-03-04 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. 채널로돕신의 돌연변이 광-유도성 이온 채널
EP3516046B1 (en) * 2016-09-22 2022-07-13 Sorbonne Université Optogenetically transformed photoreceptor precursor cells for the use in the treatment of retinal degenerative diseases
KR20190077471A (ko) 2016-11-02 2019-07-03 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 방향 선택적인 망막 신경절 세포에서의 유전자의 특이적 발현을 위한 프로모터, synp198
EP3548094A1 (en) * 2016-12-01 2019-10-09 Friedrich Miescher Institute for Biomedical Research Synpi, a promoter for the specific expression of genes in interneurons
JP7112414B2 (ja) 2017-02-08 2022-08-03 フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ 網膜神経節細胞中の遺伝子の特異的発現のためのプロモーターｓｙｎｐ８８
US20200115419A1 (en) * 2017-04-12 2020-04-16 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. New optogenetic tool
EP3422065A1 (en) 2017-06-28 2019-01-02 Gensight Biologics Objective, camera and system adapted for optogenetics comprising such objective
AU2018369975B2 (en) 2017-11-15 2022-05-19 Friedrich Miescher Institute For Biomedical Research Primate retinal pigment epithelium cell-specific promoter
RU2020121408A (ru) 2017-11-30 2021-12-30 Фридрих Мишер Инститьют Фор Байомедикал Рисерч ПРОМОТОР SynPIII ДЛЯ СПЕЦИФИЧЕСКОЙ ЭКСПРЕССИИ ГЕНОВ В ПИГМЕНТНОМ ЭПИТЕЛИИ СЕТЧАТКИ
WO2019137973A1 (en) 2018-01-11 2019-07-18 Gensight Biologics Method and device for processing asynchronous signals generated by an event-based light sensor
EP3693061A1 (en) 2019-02-05 2020-08-12 Gensight Biologics Method for controlling an optogenetic device using filtering and associated devices
EP3693060A1 (en) 2019-02-05 2020-08-12 Gensight Biologics Method for controlling an optogenetic device using a command law for the radiant power of a light source and associated devices
EP3733139A1 (en) 2019-05-02 2020-11-04 Gensight Biologics Viewing apparatus and method for projecting a light signal
GB202002073D0 (en) * 2020-02-14 2020-04-01 Ucl Business Ltd Horizontal cells

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003000918A2 (en) * 2001-06-21 2003-01-03 Curagen Corporation Nucleic acids, polypeptides, single nucleotide polymorphisms and methods of use thereof
CA2451919A1 (en) * 2001-08-09 2003-02-20 Curagen Corporation Nucleic acids, polypeptides, single nucleotide polymorphisms and methods of use thereof
DE10216005A1 (de) 2002-04-11 2003-10-30 Max Planck Gesellschaft Verwendung von biologischen Photorezeptoren als direkt lichtgesteuerte Ionenkanäle
CN101484005A (zh) * 2006-05-04 2009-07-15 韦恩州立大学 通过向体内递送视紫红质核酸恢复视觉响应
EP1891976A1 (en) 2006-08-23 2008-02-27 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute for Biomedical Research Use of light sensitive genes
WO2008137066A1 (en) * 2007-05-02 2008-11-13 The Board Of Regents Of The University Of Oklahoma Use of compacted nucleic acid nanoparticles in non-viral treatments of ocular diseases
KR20100136550A (ko) 2008-04-18 2010-12-28 노파르티스 포르슝스티프퉁 쯔바이크니덜라쑹 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 실명을 치료하기 위한 신규의 치료 도구 및 방법
EP2888368A1 (en) 2012-08-27 2015-07-01 Friedrich Miescher Institute for Biomedical Research Retinal off circuit-specific promoter
US9999685B2 (en) 2014-02-10 2018-06-19 Friedrich Miescher Institute For Biomedical Research AII retinal amacrine cell-specific promoter
ES2748282T3 (es) 2014-02-11 2020-03-16 Friedrich Miescher Institute For Biomedical Res Promotor específico de células de Müller
ES2747433T3 (es) 2015-04-30 2020-03-10 Friedrich Miescher Institute For Biomedical Res Promotor para la expresión específica de genes en células de Müller
EP3350330B1 (en) 2015-09-15 2022-06-22 Friedrich Miescher Institute for Biomedical Research Novel therapeutical tools and methods for treating blindness by targeting photoreceptors
WO2017064642A1 (en) 2015-10-14 2017-04-20 Fredrich Miescher Institute For Biomedical Research Promoter for the specific expression of genes in retinal endothelial cells
WO2017093936A1 (en) 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp162, a promoter for the specific expression of genes in rod photoreceptors
BR112018011059A2 (pt) 2015-12-03 2018-11-21 Friedrich Miescher Institute For Biomedical Research synp161, um promotor para a expressão específica de genes em fotoreceptores de haste
US10995344B2 (en) 2015-12-03 2021-05-04 Friedrich Miescher Institute For Biomedical Research SYNP159, a promoter for the specific expression of genes in rod photoreceptors
EP3384033B1 (en) 2015-12-03 2021-09-08 Friedrich Miescher Institute for Biomedical Research Synp160, a promoter for the specific expression of genes in rod photoreceptors
EP3384036B1 (en) 2015-12-04 2021-02-24 Sorbonne Université Promoters and uses thereof
US20190209708A1 (en) 2016-05-17 2019-07-11 Friedrich Miescher Institute For Biomedical Research Novel therapeutic tools and methods for treating blindness
KR20190077471A (ko) 2016-11-02 2019-07-03 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 방향 선택적인 망막 신경절 세포에서의 유전자의 특이적 발현을 위한 프로모터, synp198
EP3548093A1 (en) 2016-12-01 2019-10-09 Friedrich Miescher Institute for Biomedical Research Synp107, a promoter for the specific expression of genes in interneurons
EP3548094A1 (en) 2016-12-01 2019-10-09 Friedrich Miescher Institute for Biomedical Research Synpi, a promoter for the specific expression of genes in interneurons
JP7112414B2 (ja) 2017-02-08 2022-08-03 フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ 網膜神経節細胞中の遺伝子の特異的発現のためのプロモーターｓｙｎｐ８８
AU2018369975B2 (en) 2017-11-15 2022-05-19 Friedrich Miescher Institute For Biomedical Research Primate retinal pigment epithelium cell-specific promoter
US20230193314A1 (en) 2017-11-30 2023-06-22 Friedrich Miescher Institute For Biomedical Research SynP61, A PRIMATE RETINAL PIGMENT EPITHELIUM CELL-SPECIFIC PROMOTER
RU2020121408A (ru) 2017-11-30 2021-12-30 Фридрих Мишер Инститьют Фор Байомедикал Рисерч ПРОМОТОР SynPIII ДЛЯ СПЕЦИФИЧЕСКОЙ ЭКСПРЕССИИ ГЕНОВ В ПИГМЕНТНОМ ЭПИТЕЛИИ СЕТЧАТКИ
WO2020084542A1 (en) 2018-10-25 2020-04-30 Friedrich Miescher Institute For Biomedical Research Synp194 (prob15), a promoter for the specific expression of genes in retinal ganglion cells
WO2020084540A1 (en) 2018-10-25 2020-04-30 Friedrich Miescher Institute For Biomedical Research Synp78 (proa27), a promoter for the specific expression of genes in retinal ganglion cells
US20210395750A1 (en) 2018-10-25 2021-12-23 Friedrich Miescher Institute For Biomedical Research Synp17 (prob1), a promoter for the specific expression of genes in retinal ganglion cells
WO2020084538A1 (en) 2018-10-25 2020-04-30 Friedrich Miescher Institute For Biomedical Research Synp27 (prob12), a promoter for the specific expression of genes in protoplasmic astrocytes
WO2020084539A1 (en) 2018-10-25 2020-04-30 Friedrich Miescher Institute For Biomedical Research Synp57 (proa14), a promoter for the specific expression of genes in photoreceptors
US20210388387A1 (en) 2018-10-25 2021-12-16 Friedrich Miescher Institute For Biomedical Research Synp151 (proc29), a promoter for the specific expression of genes in retinal ganglion cells

Also Published As

Publication number Publication date
US10987404B2 (en) 2021-04-27
WO2009127705A1 (en) 2009-10-22
JP2011516091A (ja) 2011-05-26
EP2679246A2 (en) 2014-01-01
AU2009237585C1 (en) 2013-09-26
EP2271369A1 (en) 2011-01-12
KR20100136550A (ko) 2010-12-28
CN101970013A (zh) 2011-02-09
BRPI0911426A2 (pt) 2015-09-29
AU2009237585A1 (en) 2009-10-22
PL2271369T3 (pl) 2014-09-30
US20130059374A1 (en) 2013-03-07
AU2009237585B2 (en) 2013-03-14
US20180125925A1 (en) 2018-05-10
US20160250282A1 (en) 2016-09-01
CA2721635A1 (en) 2009-10-22
EA201001621A1 (ru) 2011-06-30
US9844579B2 (en) 2017-12-19
EP2679246A3 (en) 2014-02-26
CN101970013B (zh) 2014-11-05
EP2271369B1 (en) 2014-03-26
ES2474721T3 (es) 2014-07-09
MX2010011467A (es) 2011-02-15
US20120258530A1 (en) 2012-10-11
JP2015128440A (ja) 2015-07-16

Similar Documents

Publication Publication Date Title
PT2271369E (pt) Novas ferramentas terapêuticas e métodos para tratar a cegueira
IL257418A (en) Methods for treating addiction
EP2176283A4 (en) METHOD AND COMPOSITIONS FOR TREATING BRAIN DISEASES
HK1156631A1 (en) Novel compounds and methods for therapy
IL212348A0 (en) Treatment method
EP2083857A4 (en) METHOD FOR TREATING INTERFERENCE IN CONNECTION WITH MICA
EP2066246A4 (en) SKIN TREATMENT TOOL
HK1203516A1 (en) Methods for treating psoriasis
IL204728A0 (en) Methods and compounds for treating retinol-related diseases
HK1203305A1 (en) Methods for treating psoriasis
EP2341936A4 (en) METHOD FOR TREATING OR PREVENTING IL-1ß-CONTAINED ILLNESSES
IL238394A0 (en) Cancer treatment method
EP2144888A4 (en) METHODS OF TREATING CANCER
AU2013204721A1 (en) Methods for treating cachexia
GB0718684D0 (en) Treatment method
EP2086560A4 (en) METHODS OF TREATING EPIPHORA
EP2088862A4 (en) METHOD OF TREATING CANCER
EP2068911A4 (en) METHOD FOR THE TREATMENT OF CANCER
EP2002840A4 (en) METHOD FOR THE TREATMENT OF ONCOLOGICAL ILLNESSES
GB2450219B (en) Treatment tool
EP2203432A4 (en) PROCESSING METHOD
GB0711228D0 (en) Treatment method
ZA201000867B (en) Methods for treating dependence
EP2173334A4 (en) METHOD OF TREATING AMBLYOPIA
AU2013207600A1 (en) Methods and apparatus for treating glaucoma