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
- volleile
- prpf
- congenital
- degeneration
- 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 [CRISPR]
-
- 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)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Ophthalmology & Optometry (AREA)
- Mycology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (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)
- Medicinal Preparation (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (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 | |
| EP16762307.3A EP3265568B1 (en) | 2015-03-06 | 2016-03-07 | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
| 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 |
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 (3) | US20180043034A1 (pl) |
| EP (2) | EP3748007B1 (pl) |
| JP (3) | JP6812368B2 (pl) |
| CY (1) | CY1123158T1 (pl) |
| DK (1) | DK3265568T3 (pl) |
| ES (1) | ES2806054T3 (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) |
| SM (1) | SMT202000423T1 (pl) |
| WO (1) | WO2016144892A1 (pl) |
Families Citing this family (21)
| 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 |
| HRP20171334T1 (hr) | 2011-04-22 | 2017-11-17 | The Regents Of The University Of California | Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe |
| US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| KR102537394B1 (ko) | 2014-03-17 | 2023-05-30 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법 |
| MY187898A (en) | 2015-03-02 | 2021-10-27 | Adverum Biotechnologies Inc | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| PL3265568T3 (pl) * | 2015-03-06 | 2020-11-30 | Massachusetts Eye & Ear Infirmary | Terapie augmentacji genów w przypadku wrodzonego zwyrodnienia siatkówki spowodowanego mutacjami genu prpf31 |
| JP6836999B2 (ja) * | 2015-03-24 | 2021-03-03 | 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Ｔｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ | アデノ随伴ウイルス変異体及びその使用方法 |
| EP3827812B1 (en) | 2016-07-29 | 2025-10-29 | The Regents of the University of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| JP2019530737A (ja) | 2016-08-23 | 2019-10-24 | アコーオス インコーポレイテッド | ヒト対象において非加齢性聴力障害を治療するための組成物および方法 |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| JP7330899B2 (ja) | 2017-05-10 | 2023-08-22 | マサチューセッツ アイ アンド イヤー インファーマリー | ウイルスのアセンブリ活性化タンパク質（ａａｐ）依存性を改変する方法および組成物 |
| WO2019006182A1 (en) | 2017-06-30 | 2019-01-03 | The Regents Of The University Of California | VIRIONS OF ADENO-ASSOCIATED VIRUSES OF CAPSID VARIANTS AND METHODS OF USE THEREOF |
| JP2020534788A (ja) | 2017-08-28 | 2020-12-03 | 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 | アデノ随伴ウイルスカプシド変異体及びその使用方法 |
| CA3102095A1 (en) * | 2018-06-01 | 2019-12-05 | University Of Florida Research Foundation, Incorporated | Compositions and methods for 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 |
| JP2022525193A (ja) * | 2019-03-15 | 2022-05-11 | ユニヴァーシティ オブ ワシントン | Ｐｒｐｆ３１遺伝子発現ノックダウンによる、インビトロで分化したヒト細胞の生存の改善 |
| JP7807380B2 (ja) | 2020-02-21 | 2026-01-27 | アコーオス インコーポレイテッド | ヒト対象において非加齢関連聴力障害を処置するための組成物および方法 |
| US12257320B2 (en) * | 2020-03-11 | 2025-03-25 | Massachusetts Eye And Ear Infirmary | Gene therapy for NMNAT1-associated retinal degeneration |
| WO2022262756A1 (zh) * | 2021-06-16 | 2022-12-22 | 北京中因科技有限公司 | Prpf31变体及其用途 |
| AU2023365490A1 (en) * | 2022-10-21 | 2025-05-01 | Biogen Ma Inc. | Compositions and methods for treating retinitis pigmentosa |
| AU2023390868A1 (en) * | 2022-12-05 | 2025-06-19 | 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 |
| 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 |
| WO1987000201A1 (en) | 1985-07-05 | 1987-01-15 | Whitehead Institute For Biomedical Research | Epithelial cells expressing foreign genetic material |
| EP0378576B1 (en) | 1987-09-11 | 1995-01-18 | Whitehead Institute For Biomedical Research | Transduced fibroblasts and uses therefor |
| WO1989005345A1 (en) | 1987-12-11 | 1989-06-15 | Whitehead Institute For Biomedical Research | Genetic modification of endothelial cells |
| 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 |
| CA2442670A1 (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 |
| EP1991701A4 (en) * | 2006-02-14 | 2010-03-17 | Dana Farber Cancer Inst Inc | COMPOSITIONS, KITS AND METHODS OF IDENTIFICATION, ASSESSMENT, PREVENTION AND THERAPY OF CANCER |
| US8257969B2 (en) * | 2007-04-12 | 2012-09-04 | The Provost Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth | Genetic suppression and replacement |
| 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 |
| WO2010005533A2 (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) |
| EP2872183B1 (en) * | 2012-07-11 | 2018-09-26 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
| HUE052676T2 (hu) | 2013-10-11 | 2021-05-28 | Massachusetts Eye & Ear Infirmary | Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik |
| EP3154573A1 (en) * | 2014-06-13 | 2017-04-19 | Universidade De Santiago De Compostela | Nanoparticulate systems for use in gene transfer or gene delivery |
| PL3265568T3 (pl) | 2015-03-06 | 2020-11-30 | Massachusetts Eye & Ear Infirmary | Terapie augmentacji genów w przypadku wrodzonego zwyrodnienia siatkówki spowodowanego mutacjami genu prpf31 |
-
2016
- 2016-03-07 PL PL16762307T patent/PL3265568T3/pl unknown
- 2016-03-07 HR HRP20201225TT patent/HRP20201225T1/hr unknown
- 2016-03-07 RS RS20200945A patent/RS60668B1/sr unknown
- 2016-03-07 WO PCT/US2016/021226 patent/WO2016144892A1/en not_active Ceased
- 2016-03-07 SI SI201630873T patent/SI3265568T1/sl unknown
- 2016-03-07 ES ES16762307T patent/ES2806054T3/es active Active
- 2016-03-07 HU HUE16762307A patent/HUE051491T2/hu unknown
- 2016-03-07 EP EP20171646.1A patent/EP3748007B1/en active Active
- 2016-03-07 EP EP16762307.3A patent/EP3265568B1/en active Active
- 2016-03-07 LT LTEP16762307.3T patent/LT3265568T/lt unknown
- 2016-03-07 PT PT167623073T patent/PT3265568T/pt unknown
- 2016-03-07 DK DK16762307.3T patent/DK3265568T3/da active
- 2016-03-07 US US15/555,915 patent/US20180043034A1/en not_active Abandoned
- 2016-03-07 SM SM20200423T patent/SMT202000423T1/it unknown
- 2016-03-07 JP JP2017564778A patent/JP6812368B2/ja active Active
-
2020
- 2020-08-04 CY CY20201100718T patent/CY1123158T1/el unknown
- 2020-08-13 US US16/992,689 patent/US20210069347A1/en not_active Abandoned
- 2020-12-16 JP JP2020208068A patent/JP7239550B2/ja active Active
-
2023
- 2023-03-02 JP JP2023031931A patent/JP2023071867A/ja active Pending
-
2024
- 2024-10-25 US US18/927,466 patent/US20250195691A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US20250195691A1 (en) | 2025-06-19 |
| EP3748007B1 (en) | 2024-06-26 |
| HK1247637A1 (en) | 2018-09-28 |
| HRP20201225T1 (hr) | 2020-11-13 |
| SMT202000423T1 (it) | 2020-09-10 |
| CY1123158T1 (el) | 2021-10-29 |
| HUE051491T2 (hu) | 2021-03-01 |
| EP3265568A4 (en) | 2018-08-08 |
| ES2806054T3 (es) | 2021-02-16 |
| JP7239550B2 (ja) | 2023-03-14 |
| SI3265568T1 (sl) | 2020-10-30 |
| LT3265568T (lt) | 2020-08-25 |
| EP3748007C0 (en) | 2024-06-26 |
| RS60668B1 (sr) | 2020-09-30 |
| EP3748007A1 (en) | 2020-12-09 |
| EP3265568A1 (en) | 2018-01-10 |
| US20180043034A1 (en) | 2018-02-15 |
| PT3265568T (pt) | 2020-08-20 |
| JP2018511652A (ja) | 2018-04-26 |
| JP2023071867A (ja) | 2023-05-23 |
| EP3265568B1 (en) | 2020-05-06 |
| WO2016144892A1 (en) | 2016-09-15 |
| US20210069347A1 (en) | 2021-03-11 |
| JP6812368B2 (ja) | 2021-01-13 |
| DK3265568T3 (da) | 2020-08-10 |
| JP2021059561A (ja) | 2021-04-15 |
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