PL3265568T3 - Terapie augmentacji genów w przypadku wrodzonego zwyrodnienia siatkówki spowodowanego mutacjami genu prpf31 - Google Patents
Terapie augmentacji genów w przypadku wrodzonego zwyrodnienia siatkówki spowodowanego mutacjami genu prpf31Info
- Publication number
- PL3265568T3 PL3265568T3 PL16762307T PL16762307T PL3265568T3 PL 3265568 T3 PL3265568 T3 PL 3265568T3 PL 16762307 T PL16762307 T PL 16762307T PL 16762307 T PL16762307 T PL 16762307T PL 3265568 T3 PL3265568 T3 PL 3265568T3
- Authority
- PL
- Poland
- Prior art keywords
- gene
- mutations
- prpf31
- retinal degeneration
- degeneration caused
- Prior art date
Links
Classifications
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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
- 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
- A61P27/02—Ophthalmic agents
-
- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
-
- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
-
- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- 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
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Ophthalmology & Optometry (AREA)
- Mycology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562129638P | 2015-03-06 | 2015-03-06 | |
| US201562147307P | 2015-04-14 | 2015-04-14 | |
| PCT/US2016/021226 WO2016144892A1 (en) | 2015-03-06 | 2016-03-07 | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
| EP16762307.3A EP3265568B1 (en) | 2015-03-06 | 2016-03-07 | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL3265568T3 true PL3265568T3 (pl) | 2020-11-30 |
Family
ID=56879585
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL16762307T PL3265568T3 (pl) | 2015-03-06 | 2016-03-07 | Terapie augmentacji genów w przypadku wrodzonego zwyrodnienia siatkówki spowodowanego mutacjami genu prpf31 |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US20180043034A1 (pl) |
| EP (2) | EP3265568B1 (pl) |
| JP (3) | JP6812368B2 (pl) |
| CY (1) | CY1123158T1 (pl) |
| DK (1) | DK3265568T3 (pl) |
| ES (1) | ES2806054T3 (pl) |
| HK (1) | HK1247637A1 (pl) |
| HR (1) | HRP20201225T1 (pl) |
| HU (1) | HUE051491T2 (pl) |
| LT (1) | LT3265568T (pl) |
| PL (1) | PL3265568T3 (pl) |
| PT (1) | PT3265568T (pl) |
| RS (1) | RS60668B1 (pl) |
| SI (1) | SI3265568T1 (pl) |
| WO (1) | WO2016144892A1 (pl) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| RS60207B1 (sr) | 2011-04-22 | 2020-06-30 | Univ California | Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu |
| US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| SG10201810150UA (en) | 2014-03-17 | 2018-12-28 | Adverum Biotechnologies Inc | Compositions and methods for enhanced gene expression in cone cells |
| AU2016226289B2 (en) | 2015-03-02 | 2021-04-29 | Adverum Biotechnologies, Inc. | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| US20180043034A1 (en) * | 2015-03-06 | 2018-02-15 | Massachusetts Eye And Ear Infirmary | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
| ES2860712T3 (es) | 2015-03-24 | 2021-10-05 | Univ California | Variantes de virus adenoasociados y métodos de uso de las mismas |
| JP7071332B2 (ja) | 2016-07-29 | 2022-05-18 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 変異カプシドを有するアデノ随伴ウイルスビリオン及びその使用方法 |
| CA3034527A1 (en) | 2016-08-23 | 2018-03-01 | Emmanuel John Simons | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| WO2018075798A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| CA3061968A1 (en) * | 2017-05-10 | 2018-11-15 | Massachusetts Eye And Ear Infirmary | Methods and compositions for modifying assembly-activating protein (aap)-dependence of viruses |
| BR112020003571A2 (pt) | 2017-08-28 | 2020-08-25 | The Regents Of The University Of California | variantes capsidiais de vírus adenoassociado e métodos de uso das mesmas |
| EP3802836A4 (en) * | 2018-06-01 | 2022-10-19 | University of Florida Research Foundation, Incorporated | COMPOSITIONS AND METHODS FOR THE TREATMENT OF DOMINANT RETINITIS PIGMENTOSA |
| WO2020102740A2 (en) * | 2018-11-16 | 2020-05-22 | Spark Therapeutics, Inc. | Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins |
| IL295747A (en) | 2020-02-21 | 2022-10-01 | Akouos Inc | Preparations and methods for the treatment of hearing impairment that is not related to age in humans |
| 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 |
Family Cites Families (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE3023787A1 (de) | 1980-06-25 | 1982-01-21 | Studiengesellschaft Kohle mbH, 4330 Mülheim | Verfahren zur erhoehung der inkorporation und der expression von genetischem material in die kerne von intakten zellen mit hilfe von liposomen |
| DE3681787D1 (de) | 1985-07-05 | 1991-11-07 | Whitehead Biomedical Inst | Expression von fremdem genetischem material in epithelzellen. |
| US4980286A (en) | 1985-07-05 | 1990-12-25 | Whitehead Institute For Biomedical Research | In vivo introduction and expression of foreign genetic material in epithelial cells |
| EP0378576B1 (en) | 1987-09-11 | 1995-01-18 | Whitehead Institute For Biomedical Research | Transduced fibroblasts and uses therefor |
| ATE110108T1 (de) | 1987-12-11 | 1994-09-15 | Whitehead Biomedical Inst | Genetische modifizierung von endothelialen zellen. |
| JP2917998B2 (ja) | 1988-02-05 | 1999-07-12 | ホワイトヘッド・インスティチュート・フォー・バイオメディカル・リサーチ | 修飾された肝細胞およびその用途 |
| ATE157012T1 (de) | 1989-11-03 | 1997-09-15 | Univ Vanderbilt | Verfahren zur in vivo-verabreichung von funktionsfähigen fremden genen |
| JP3249516B2 (ja) | 1990-10-31 | 2002-01-21 | ソマティクス セラピー コーポレイション | 遺伝子治療のためのレトロウイルスのベクター |
| ES2150832B1 (es) | 1996-06-12 | 2001-06-16 | Fichtel & Sachs Ag | Dispositivo de maniobra para la maniobra, en particular maniobra neumatica, de un embrague de friccion. |
| US6569662B1 (en) * | 2000-01-21 | 2003-05-27 | Hyseq, Inc. | Nucleic acids and polypeptides |
| WO2002082904A2 (en) * | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
| US20040208847A1 (en) * | 2003-03-28 | 2004-10-21 | Fabienne Rolling | Method and vectors for selectively transducing retinal pigment epithelium cells |
| WO2007095186A2 (en) * | 2006-02-14 | 2007-08-23 | Dana-Farber Cancer Institute, Inc. | Compositions, kits, and methods for identification, assessment, prevention, and therapy of cancer |
| ES2907452T3 (es) * | 2007-04-12 | 2022-04-25 | The Provost Fellows Found Scholars & The Other Members Of Board Of The College Of The Holy & Undiv T | Supresión y reemplazo genético |
| 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 |
| WO2009134681A2 (en) * | 2008-04-30 | 2009-11-05 | The Trustees Of The University Of Pennsylvania | Aav7 viral vectors for targeted delivery of rpe cells |
| CA2729605A1 (en) * | 2008-06-30 | 2010-01-14 | The Johns Hopkins University | Compositions and methods for the treatment of ocular oxidative stress and retinitis pigmentosa |
| GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| TWI775096B (zh) * | 2012-05-15 | 2022-08-21 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| 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 |
| CN106232618A (zh) | 2013-10-11 | 2016-12-14 | 马萨诸塞眼科耳科诊所 | 预测祖先病毒序列的方法及其用途 |
| EP3154573A1 (en) * | 2014-06-13 | 2017-04-19 | Universidade De Santiago De Compostela | Nanoparticulate systems for use in gene transfer or gene delivery |
| US20180043034A1 (en) | 2015-03-06 | 2018-02-15 | Massachusetts Eye And Ear Infirmary | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
-
2016
- 2016-03-07 US US15/555,915 patent/US20180043034A1/en not_active Abandoned
- 2016-03-07 SI SI201630873T patent/SI3265568T1/sl unknown
- 2016-03-07 PT PT167623073T patent/PT3265568T/pt unknown
- 2016-03-07 EP EP16762307.3A patent/EP3265568B1/en active Active
- 2016-03-07 JP JP2017564778A patent/JP6812368B2/ja active Active
- 2016-03-07 WO PCT/US2016/021226 patent/WO2016144892A1/en active Application Filing
- 2016-03-07 DK DK16762307.3T patent/DK3265568T3/da active
- 2016-03-07 RS RS20200945A patent/RS60668B1/sr unknown
- 2016-03-07 ES ES16762307T patent/ES2806054T3/es active Active
- 2016-03-07 PL PL16762307T patent/PL3265568T3/pl unknown
- 2016-03-07 EP EP20171646.1A patent/EP3748007B1/en active Active
- 2016-03-07 HU HUE16762307A patent/HUE051491T2/hu unknown
- 2016-03-07 LT LTEP16762307.3T patent/LT3265568T/lt unknown
-
2018
- 2018-05-25 HK HK18106900.6A patent/HK1247637A1/zh unknown
-
2020
- 2020-08-04 HR HRP20201225TT patent/HRP20201225T1/hr unknown
- 2020-08-04 CY CY20201100718T patent/CY1123158T1/el unknown
- 2020-08-13 US US16/992,689 patent/US20210069347A1/en active Pending
- 2020-12-16 JP JP2020208068A patent/JP7239550B2/ja active Active
-
2023
- 2023-03-02 JP JP2023031931A patent/JP2023071867A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| HK1247637A1 (zh) | 2018-09-28 |
| CY1123158T1 (el) | 2021-10-29 |
| HRP20201225T1 (hr) | 2020-11-13 |
| WO2016144892A1 (en) | 2016-09-15 |
| EP3265568A4 (en) | 2018-08-08 |
| DK3265568T3 (da) | 2020-08-10 |
| JP2018511652A (ja) | 2018-04-26 |
| HUE051491T2 (hu) | 2021-03-01 |
| JP2021059561A (ja) | 2021-04-15 |
| RS60668B1 (sr) | 2020-09-30 |
| PT3265568T (pt) | 2020-08-20 |
| US20210069347A1 (en) | 2021-03-11 |
| JP6812368B2 (ja) | 2021-01-13 |
| EP3265568A1 (en) | 2018-01-10 |
| EP3265568B1 (en) | 2020-05-06 |
| LT3265568T (lt) | 2020-08-25 |
| EP3748007A1 (en) | 2020-12-09 |
| EP3748007B1 (en) | 2024-06-26 |
| JP2023071867A (ja) | 2023-05-23 |
| US20180043034A1 (en) | 2018-02-15 |
| SI3265568T1 (sl) | 2020-10-30 |
| JP7239550B2 (ja) | 2023-03-14 |
| ES2806054T3 (es) | 2021-02-16 |
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