BR112016019432A8 - composição terapêutica, kit e seus usos na terapia gênica contra degeneração da retina - Google Patents

composição terapêutica, kit e seus usos na terapia gênica contra degeneração da retina

Info

Publication number
BR112016019432A8
BR112016019432A8 BR112016019432A BR112016019432A BR112016019432A8 BR 112016019432 A8 BR112016019432 A8 BR 112016019432A8 BR 112016019432 A BR112016019432 A BR 112016019432A BR 112016019432 A BR112016019432 A BR 112016019432A BR 112016019432 A8 BR112016019432 A8 BR 112016019432A8
Authority
BR
Brazil
Prior art keywords
retinal degeneration
gene therapy
kit
therapeutic composition
therapy against
Prior art date
Application number
BR112016019432A
Other languages
English (en)
Other versions
BR112016019432B1 (pt
BR112016019432A2 (pt
Inventor
Robert Lucas
Paul Bishop
Jasmina Cehajic-Kapetanovic
Original Assignee
Univ Manchester
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=50482737&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=BR112016019432(A8) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Univ Manchester filed Critical Univ Manchester
Publication of BR112016019432A2 publication Critical patent/BR112016019432A2/pt
Publication of BR112016019432A8 publication Critical patent/BR112016019432A8/pt
Publication of BR112016019432B1 publication Critical patent/BR112016019432B1/pt

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    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/13—Amines
    • A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
    • A61K31/137—Arylalkylamines, e.g. amphetamine, epinephrine, salbutamol, ephedrine or methadone
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/4409—Non condensed pyridines; Hydrogenated derivatives thereof only substituted in position 4, e.g. isoniazid, iproniazid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/51—Lyases (4)
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • 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
    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • 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
    • 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
    • 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
    • A61P27/10—Ophthalmic agents for accommodation disorders, e.g. myopia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/72—Receptors; Cell surface antigens; Cell surface determinants for hormones
    • C07K14/723—G protein coupled receptor, e.g. TSHR-thyrotropin-receptor, LH/hCG receptor, FSH receptor
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/88—Lyases (4.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y402/00—Carbon-oxygen lyases (4.2)
    • C12Y402/02—Carbon-oxygen lyases (4.2) acting on polysaccharides (4.2.2)
    • C12Y402/02001—Hyaluronate lyase (4.2.2.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y402/00—Carbon-oxygen lyases (4.2)
    • C12Y402/02—Carbon-oxygen lyases (4.2) acting on polysaccharides (4.2.2)
    • C12Y402/02008—Heparin-sulfate lyase (4.2.2.8)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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

Abstract

tratamento da degeneração da retina usando terapia genética. a presente invenção refere-se a um método melhorado de proporcionar a função de fotorreceptores a uma célula, por exemplo, para utilização no tratamento de degeneração da retina. a presente invenção também refere-se às composições e kits, em particular para utilização em tais métodos.
BR112016019432-2A 2014-02-25 2015-02-24 Composição terapêutica, kit e seus usos na terapia gênica contra degeneração da retina BR112016019432B1 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB201403260A GB201403260D0 (en) 2014-02-25 2014-02-25 Treatment of retinal degeneration using gene therapy
GB1403260.1 2014-02-25
PCT/GB2015/050516 WO2015128624A1 (en) 2014-02-25 2015-02-24 Treatment of retinal degeneration using gene therapy

Publications (3)

Publication Number Publication Date
BR112016019432A2 BR112016019432A2 (pt) 2017-08-15
BR112016019432A8 true BR112016019432A8 (pt) 2021-07-06
BR112016019432B1 BR112016019432B1 (pt) 2023-09-26

Family

ID=50482737

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112016019432-2A BR112016019432B1 (pt) 2014-02-25 2015-02-24 Composição terapêutica, kit e seus usos na terapia gênica contra degeneração da retina

Country Status (8)

Country Link
US (2) US10912845B2 (pt)
EP (2) EP3110433B2 (pt)
JP (2) JP6511066B2 (pt)
CN (1) CN106456711A (pt)
BR (1) BR112016019432B1 (pt)
ES (1) ES2742499T5 (pt)
GB (1) GB201403260D0 (pt)
WO (1) WO2015128624A1 (pt)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201403260D0 (en) 2014-02-25 2014-04-09 Univ Manchester Treatment of retinal degeneration using gene therapy
JP6898920B2 (ja) * 2015-09-15 2021-07-07 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 光受容器のターゲッティングにより失明を治療するための新規な治療用ツールおよび方法
EP3210632B1 (en) * 2016-02-23 2022-09-28 EyeServ GmbH Gene therapy for the treatment of a retinal degeneration disease
JP6779546B2 (ja) * 2016-11-06 2020-11-04 ナノスコープ テクノロジーズ エルエルシーＮａｎｏｓｃｏｐｅ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｌｌｃ 視力回復のための多特性オプシンによる光遺伝学的調節及びその別の用途
US20210130413A1 (en) * 2017-02-28 2021-05-06 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
CN112501208A (zh) * 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
WO2019094904A1 (en) 2017-11-13 2019-05-16 The Regents Of The University Of California Compositions and methods for enhancing visual function
US20200399656A1 (en) * 2017-12-05 2020-12-24 University Of Washington Compositions and methods for enhancing functional expression of therapeutic genes in photoreceptors
US20220031864A1 (en) 2018-09-24 2022-02-03 The University Of Manchester Quantitative regulation of a g protein signalling pathway
WO2020236352A1 (en) * 2019-05-17 2020-11-26 Research Institute At Nationwide Children's Hospital Optimized gene therapy targeting retinal cells
GB201914826D0 (en) * 2019-10-14 2019-11-27 Univ Manchester Modulating OPSIN signaling lifetime for optogenetic applications
CR20220287A (es) * 2019-11-18 2022-09-30 Univ Bern Promotores específicos para neuronas bipolares activadas para el suministro ocular de genes
WO2023116720A1 (en) * 2021-12-20 2023-06-29 National Institute Of Biological Sciences, Beijing ULTRA LIGHT-SENSITIVE NEUROPSIN-BASED OPTOGENETIC TOOL FOR ACTIVATING G q-COUPLED SIGNALING AND/OR ACTIVATING CELLS
WO2023116729A1 (en) * 2021-12-20 2023-06-29 Genans Biotechnology Co., Ltd Optogenetic visual restoration using light-sensitive gq-coupled neuropsin (opsin 5)
WO2023196746A1 (en) * 2022-04-04 2023-10-12 The Regents Of The University Of California Compositions and methods for enhancing visual function

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WO2004009022A2 (en) 2002-07-18 2004-01-29 The General Hospital Corp. Method for augmenting vision in persons suffering from photoreceptor cell degeneration
GB0426196D0 (en) † 2004-11-30 2004-12-29 Imp College Innovations Ltd Methods of treatment
EP2019588A4 (en) 2006-05-04 2010-11-24 Univ Wayne State RECOVERY OF VISUAL REACTIONS BY IN VIVO ADMINISTRATION OF RHODOPSIN NUCLEIC ACIDS
PT2271369E (pt) * 2008-04-18 2014-07-10 Novartis Forschungsstiftung Novas ferramentas terapêuticas e métodos para tratar a cegueira
EP2910637A1 (en) 2008-05-20 2015-08-26 University of Florida Research Foundation, Inc. Vectors for delivery of light-sensitive proteins and methods of use
US20130071373A1 (en) * 2010-02-02 2013-03-21 Christina Zeitz Methods for the diagnosis and therapy of retinitis pigmentosa
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
BR122022007044B1 (pt) 2011-06-24 2023-11-14 Novartis Ag Composições farmacêuticas que compreendem uma proteína de gpcr quimérica, proteína de gpcr quimérica, molécula de ácido nucleico, e vetor
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GB201403260D0 (en) 2014-02-25 2014-04-09 Univ Manchester Treatment of retinal degeneration using gene therapy

Also Published As

Publication number Publication date
ES2742499T3 (es) 2020-02-14
ES2742499T5 (es) 2023-04-27
BR112016019432B1 (pt) 2023-09-26
GB201403260D0 (en) 2014-04-09
WO2015128624A1 (en) 2015-09-03
JP2019131590A (ja) 2019-08-08
EP3578197A1 (en) 2019-12-11
US20160361437A1 (en) 2016-12-15
JP6511066B2 (ja) 2019-05-15
US10912845B2 (en) 2021-02-09
CN106456711A (zh) 2017-02-22
EP3110433A1 (en) 2017-01-04
US20210330812A1 (en) 2021-10-28
EP3110433B1 (en) 2019-07-10
EP3578197B1 (en) 2022-06-08
BR112016019432A2 (pt) 2017-08-15
EP3110433B2 (en) 2023-02-22
JP2017507942A (ja) 2017-03-23

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