SG11201706755SA - Methods and compositions for treating genetic eye diseases - Google Patents
Methods and compositions for treating genetic eye diseasesInfo
- Publication number
- SG11201706755SA SG11201706755SA SG11201706755SA SG11201706755SA SG11201706755SA SG 11201706755S A SG11201706755S A SG 11201706755SA SG 11201706755S A SG11201706755S A SG 11201706755SA SG 11201706755S A SG11201706755S A SG 11201706755SA SG 11201706755S A SG11201706755S A SG 11201706755SA
- Authority
- SG
- Singapore
- Prior art keywords
- compositions
- methods
- eye diseases
- treating genetic
- genetic eye
- Prior art date
Links
Classifications
-
- 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
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/861—Adenoviral vectors
- C12N15/8616—Special methods for targeting systems
-
- 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
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- C12N2750/14145—Special targeting system for viral vectors
-
- 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
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562118934P | 2015-02-20 | 2015-02-20 | |
| PCT/US2016/018970 WO2016134375A1 (en) | 2015-02-20 | 2016-02-22 | Methods and compositions for treating genetic eye diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11201706755SA true SG11201706755SA (en) | 2017-09-28 |
Family
ID=56689466
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11201706755SA SG11201706755SA (en) | 2015-02-20 | 2016-02-22 | Methods and compositions for treating genetic eye diseases |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US10472650B2 (en) |
| EP (1) | EP3261440B1 (en) |
| JP (1) | JP6873907B2 (en) |
| KR (1) | KR102655319B1 (en) |
| CN (1) | CN108347932B (en) |
| AU (1) | AU2016219789B2 (en) |
| CA (1) | CA2977355A1 (en) |
| ES (1) | ES2920850T3 (en) |
| SA (1) | SA517382162B1 (en) |
| SG (1) | SG11201706755SA (en) |
| WO (1) | WO2016134375A1 (en) |
| ZA (1) | ZA201705628B (en) |
Families Citing this family (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN105247044B (en) | 2013-05-31 | 2021-05-07 | 加利福尼亚大学董事会 | Adeno-associated virus variants and methods of use thereof |
| MA44873A (en) | 2016-04-15 | 2019-03-13 | Univ Pennsylvania | COMPOSITION FOR THE TREATMENT OF MACULAR DEGENERATION RELATED TO EXSUDATIVE AGE |
| CN114672516A (en) | 2016-07-29 | 2022-06-28 | 加利福尼亚大学董事会 | Adeno-associated virus virions with variant capsids and methods of use thereof |
| MX2019002518A (en) * | 2016-09-02 | 2019-07-18 | Spark Therapeutics Inc | Methods and vectors for treating cns disorders. |
| WO2019006182A1 (en) * | 2017-06-30 | 2019-01-03 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsids and methods of use thereof |
| EP3662060A2 (en) | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| BR112020003571A2 (en) | 2017-08-28 | 2020-08-25 | The Regents Of The University Of California | Capsid variants of adeno-associated virus and methods of using them |
| CA3078277A1 (en) * | 2017-10-16 | 2019-04-25 | Vigeneron Gmbh | Aav vectors |
| WO2019094904A1 (en) * | 2017-11-13 | 2019-05-16 | The Regents Of The University Of California | Compositions and methods for enhancing visual function |
| EP3714055A1 (en) | 2017-11-21 | 2020-09-30 | CRISPR Therapeutics AG | Materials and methods for treatment of autosomal dominant retinitis pigmentosa |
| AU2019220368B2 (en) * | 2018-02-15 | 2024-06-13 | Brave Bioscience Ab | Modified viral capsids |
| TW202015742A (en) | 2018-05-15 | 2020-05-01 | 美商航海家醫療公司 | Compositions and methods for delivery of aav |
| MX2020012077A (en) | 2018-05-15 | 2021-03-09 | Voyager Therapeutics Inc | Compositions and methods for the treatment of parkinson's disease. |
| US12060567B2 (en) | 2018-06-13 | 2024-08-13 | Voyager Therapeutics, Inc. | Engineered untranslated regions (UTR) for AAV production |
| WO2020023612A1 (en) | 2018-07-24 | 2020-01-30 | Voyager Therapeutics, Inc. | Systems and methods for producing gene therapy formulations |
| CA3114621A1 (en) | 2018-09-28 | 2020-04-02 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| US20210348242A1 (en) | 2018-10-04 | 2021-11-11 | Voyager Therapeutics, Inc. | Methods for measuring the titer and potency of viral vector particles |
| US20210348194A1 (en) | 2018-10-05 | 2021-11-11 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding aav production proteins |
| WO2020077165A1 (en) | 2018-10-12 | 2020-04-16 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| CN113166781A (en) | 2018-10-15 | 2021-07-23 | 沃雅戈治疗公司 | Large-scale production of rAAV expression vectors in the baculovirus/Sf9system |
| JP2022522995A (en) | 2019-01-18 | 2022-04-21 | ボイジャー セラピューティクス インコーポレイテッド | Methods and systems for producing AAV particles |
| WO2020206098A1 (en) * | 2019-04-03 | 2020-10-08 | Regenxbio Inc. | Gene therapy for eye pathologies |
| AU2020271052A1 (en) * | 2019-04-08 | 2021-10-28 | The Children's Hospital Of Philadelphia | Treatment of lysosomal storage disease in the eye through administration of AAVs expressing TPP1 |
| EP3962536A1 (en) | 2019-04-29 | 2022-03-09 | Voyager Therapeutics, Inc. | Systems and methods for producing baculoviral infected insect cells (biics) in bioreactors |
| EP3966227A1 (en) | 2019-05-07 | 2022-03-16 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
| US20220290182A1 (en) | 2019-08-09 | 2022-09-15 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| TW202122582A (en) | 2019-08-26 | 2021-06-16 | 美商航海家醫療公司 | Controlled expression of viral proteins |
| WO2021046155A1 (en) | 2019-09-03 | 2021-03-11 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
| CA3157464A1 (en) * | 2019-10-31 | 2021-05-06 | Universitat Bern | Aav vector variants for ocular gene delivery |
| CN113025618B (en) * | 2019-12-24 | 2024-02-06 | 朗信启昇(苏州)生物制药有限公司 | Gene therapy scheme and application of X-linked hereditary retinal splitting disease |
| WO2021207500A2 (en) * | 2020-04-08 | 2021-10-14 | President And Fellows Of Harvard College | TGFβ THERAPY FOR OCULAR AND NEURODEGENERATIVE DISEASES |
| AU2021273447A1 (en) | 2020-05-13 | 2022-12-08 | Voyager Therapeutics, Inc. | Redirection of tropism of AAV capsids |
| WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
| CN116323949A (en) * | 2020-07-21 | 2023-06-23 | 方拓生物科技公司 | Compositions and methods for treating ocular disorders |
| WO2022026410A2 (en) | 2020-07-27 | 2022-02-03 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
| IL299928A (en) | 2020-07-27 | 2023-03-01 | Voyager Therapeutics Inc | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| EP4192514A1 (en) | 2020-08-06 | 2023-06-14 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| EP4301768A2 (en) | 2021-03-03 | 2024-01-10 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| WO2022187548A1 (en) | 2021-03-03 | 2022-09-09 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| CN113121655B (en) * | 2021-04-19 | 2021-11-19 | 上海信致医药科技有限公司 | Ocular and muscle specific targeting type adeno-associated virus vector and application thereof |
| WO2023049710A1 (en) * | 2021-09-21 | 2023-03-30 | California Institute Of Technology | Viral compositions for targeting the nervous system and the lung |
| AU2022397707A1 (en) | 2021-11-29 | 2024-06-13 | Shanghai Regenelead Therapies Co., Ltd. | Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease |
| CN114195859B (en) * | 2021-12-10 | 2022-11-18 | 和元生物技术（上海）股份有限公司 | Adeno-associated virus mutant suitable for specifically infecting U251 cells |
| KR20240152351A (en) | 2022-02-17 | 2024-10-21 | 스카이라인 테라퓨틱스 리미티드 | Recombinant adeno-associated virus having a modified AAV capsid polypeptide |
| WO2023155918A1 (en) | 2022-02-21 | 2023-08-24 | 上海瑞宏迪医药有限公司 | Vegf-binding molecule and pharmaceutical use thereof |
| WO2024017387A1 (en) * | 2022-07-22 | 2024-01-25 | Shanghai Vitalgen Biopharma Co., Ltd. | Novel aav capsids for targeting nervous system and uses thereof |
| WO2024054983A1 (en) | 2022-09-08 | 2024-03-14 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6329501B1 (en) | 1997-05-29 | 2001-12-11 | Auburn University | Methods and compositions for targeting compounds to muscle |
| US6399575B1 (en) * | 1998-11-10 | 2002-06-04 | Auburn University | Methods and compositions for targeting compounds to the central nervous system |
| 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 |
| AU2002248297A1 (en) * | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
| US7588772B2 (en) * | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US20100081707A1 (en) * | 2008-02-21 | 2010-04-01 | Ali Robin R | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| WO2009108274A2 (en) * | 2008-02-26 | 2009-09-03 | The University Of North Carolina At Chapel Hill | Methods and compositions for adeno-associated virus (aav) with hi loop mutations |
| KR101154374B1 (en) * | 2009-02-18 | 2012-07-09 | 재단법인 아산사회복지재단 | Composition Comprising Adeno-Associated Virus Serotype 5 Vector for Targeted Gene Delivery |
| US20110052666A1 (en) * | 2009-09-03 | 2011-03-03 | Medtronic, Inc. | Compositions, Methods, and Systems for SIRNA Delivery |
| LT3254703T (en) * | 2011-04-22 | 2020-05-25 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| WO2013159036A1 (en) * | 2012-04-19 | 2013-10-24 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| DK2954051T3 (en) * | 2013-02-08 | 2019-07-08 | Univ Pennsylvania | MODIFIED KAPSID FOR TRANSFER FOR TREATMENT OF THE NETWORK |
-
2016
- 2016-02-22 US US15/551,869 patent/US10472650B2/en active Active
- 2016-02-22 EP EP16753229.0A patent/EP3261440B1/en active Active
- 2016-02-22 ES ES16753229T patent/ES2920850T3/en active Active
- 2016-02-22 CA CA2977355A patent/CA2977355A1/en active Pending
- 2016-02-22 CN CN201680022560.4A patent/CN108347932B/en active Active
- 2016-02-22 KR KR1020177026037A patent/KR102655319B1/en active IP Right Grant
- 2016-02-22 AU AU2016219789A patent/AU2016219789B2/en active Active
- 2016-02-22 JP JP2017543944A patent/JP6873907B2/en active Active
- 2016-02-22 SG SG11201706755SA patent/SG11201706755SA/en unknown
- 2016-02-22 WO PCT/US2016/018970 patent/WO2016134375A1/en active Application Filing
-
2017
- 2017-08-18 ZA ZA2017/05628A patent/ZA201705628B/en unknown
- 2017-08-20 SA SA517382162A patent/SA517382162B1/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP3261440B1 (en) | 2022-04-06 |
| CN108347932B (en) | 2022-09-13 |
| CN108347932A (en) | 2018-07-31 |
| JP2018506980A (en) | 2018-03-15 |
| JP6873907B2 (en) | 2021-05-19 |
| CA2977355A1 (en) | 2016-08-25 |
| EP3261440A1 (en) | 2018-01-03 |
| WO2016134375A1 (en) | 2016-08-25 |
| EP3261440A4 (en) | 2018-10-31 |
| SA517382162B1 (en) | 2021-11-02 |
| AU2016219789A1 (en) | 2017-09-28 |
| ES2920850T3 (en) | 2022-08-10 |
| KR20180012734A (en) | 2018-02-06 |
| US10472650B2 (en) | 2019-11-12 |
| ZA201705628B (en) | 2024-08-28 |
| KR102655319B1 (en) | 2024-04-04 |
| US20180142259A1 (en) | 2018-05-24 |
| AU2016219789B2 (en) | 2021-03-25 |
| BR112017017867A2 (en) | 2018-04-10 |
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