SG11201811244SA - Dual overlapping adeno-associated viral vector system for expressing abc4a - Google Patents
Dual overlapping adeno-associated viral vector system for expressing abc4aInfo
- Publication number
- SG11201811244SA SG11201811244SA SG11201811244SA SG11201811244SA SG11201811244SA SG 11201811244S A SG11201811244S A SG 11201811244SA SG 11201811244S A SG11201811244S A SG 11201811244SA SG 11201811244S A SG11201811244S A SG 11201811244SA SG 11201811244S A SG11201811244S A SG 11201811244SA
- Authority
- SG
- Singapore
- Prior art keywords
- nucleic acid
- acid sequence
- international
- abca4
- sequence
- Prior art date
Links
- 230000009977 dual effect Effects 0.000 title abstract 2
- 239000013603 viral vector Substances 0.000 title abstract 2
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 9
- 150000007523 nucleic acids Chemical group 0.000 abstract 9
- 108091026890 Coding region Proteins 0.000 abstract 8
- 101000801643 Homo sapiens Retinal-specific phospholipid-transporting ATPase ABCA4 Proteins 0.000 abstract 8
- 102100033617 Retinal-specific phospholipid-transporting ATPase ABCA4 Human genes 0.000 abstract 7
- 239000002773 nucleotide Substances 0.000 abstract 6
- 125000003729 nucleotide group Chemical group 0.000 abstract 6
- 239000013607 AAV vector Substances 0.000 abstract 4
- 108700019146 Transgenes Proteins 0.000 abstract 4
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 abstract 2
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 238000011144 upstream manufacturing Methods 0.000 abstract 2
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 102000051503 human ABCA4 Human genes 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 1
- 230000002265 prevention Effects 0.000 abstract 1
- 230000006798 recombination Effects 0.000 abstract 1
- 238000005215 recombination Methods 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
- 230000003612 virological effect Effects 0.000 abstract 1
Classifications
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- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
-
- 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
- A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
-
- 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
- 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
-
- 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
-
- 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
- C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
-
- 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
-
- 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
- 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
- C12N2750/14171—Demonstrated in vivo effect
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Ophthalmology & Optometry (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Cell Biology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physics & Mathematics (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Mycology (AREA)
- Toxicology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 21 December 2017 (21.12.2017) WIP0 I PCT Iiiimmomoiollmolommiolominiloolionv oimIE (10) International Publication Number WO 2017/216560 Al (51) International Patent Classification: Cl 2N 15/86 (2006.01) (21) International Application Number: PCT/GB2017/051741 (22) International Filing Date: 14 June 2017 (14.06.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 1610448.1 15 June 2016 (15.06.2016) GB 1707261.2 05 May 2017 (05.05.2017) GB (71) Applicant: OXFORD UNIVERSITY INNOVATION LIMITED [GB/GB]; Buxton Court, 3 West Way, Botley Oxford OX2 OJB (GB). (72) Inventors: MACLAREN, Robert; Nuffield Laboratory of Ophthalmology, Levels 5 & 6, West Wing, John Rad- cliffe Hospital, Headley Way, Headington Oxford OX3 9DU (GB). MCCLEMENTS, Michelle; Nuffield Labora- tory of Ophthalmology, Levels 5 & 6, West Wing, John Radcliffe Hospital, Headley Way, Headington Oxford OX3 9DU (GB). (74) Agent: D YOUNG & CO LLP; 120 Holborn, London EC1N 2DY (GB). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, (54) Title: DUAL OVERLAPPING ADENO-ASSOCIATED VIRAL VECTOR SYSTEM FOR EXPRESSING ABC4A ITR Promoter ABCA4 CDS ITR Upstream transgene Region of over ap Downstream transgene ITR ABCA4 CDS WPRE pA ITR Region of overlap enables recombination of the upstream and downstream transgenes to form a single, larger transgene with the complete ABCA4 CDS TR Promoter ABCA4 CDS WPRE pA 1-1 O 1-1 N _ N O 1-1 N C FIG. 10 (57) : The present invention provides an adeno-associated viral (AAV) vector system for expressing a human ABCA4 protein in a target cell, the AAV vector system comprising a first AAV vector comprising a first nucleic acid sequence and a second AAV vector comprising a second nucleic acid sequence; wherein the first nucleic acid sequence comprises a 5' end portion of an ABCA4 coding sequence (CDS) and the second nucleic acid sequence comprises a 3' end portion of an ABCA4 CDS, and the 5' end portion and the 3' end portion together encompass the entire ABCA4 CDS; wherein the first nucleic acid sequence comprises a sequence of contiguous nucleotides corresponding to nucleotides 105 to 3597 of SEQ ID NO: 1; wherein the second nucleic acid sequence comprises a sequence of contiguous nucleotides corresponding to nucleotides 3806 to 6926 of SEQ ID NO: 1; wherein the first nucleic acid sequence and the second nucleic acid sequence each comprise a region of sequence overlap with the other; and wherein the region of sequence overlap comprises at least about 20 contiguous nucleotides of a nucleic acid sequence corresponding to nucleotides 3598 to 3805 of SEQ ID NO: 1. Also provided are uses of AAV vector systems in the prevention or treatment of disease. [Continued on next page] WO 2017/216560 Al MIDEDIM011111 11111111OFIEHIEMI111111111111111101111111111111111111 TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Published: with international search report (Art. 21(3)) with sequence listing part of description (Rule 5.2(a))
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1610448.1A GB201610448D0 (en) | 2016-06-15 | 2016-06-15 | Adeno-associated viral vector system |
| GBGB1707261.2A GB201707261D0 (en) | 2017-05-05 | 2017-05-05 | Adeno-associated viral vector system |
| PCT/GB2017/051741 WO2017216560A1 (en) | 2016-06-15 | 2017-06-14 | Dual overlapping adeno-associated viral vector system for expressing abc4a |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11201811244SA true SG11201811244SA (en) | 2019-01-30 |
Family
ID=59101505
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11201811244SA SG11201811244SA (en) | 2016-06-15 | 2017-06-14 | Dual overlapping adeno-associated viral vector system for expressing abc4a |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20190309326A1 (en) |
| EP (1) | EP3472328A1 (en) |
| JP (1) | JP2019523648A (en) |
| KR (1) | KR20190020745A (en) |
| CN (1) | CN109642242A (en) |
| AU (1) | AU2017286623A1 (en) |
| BR (1) | BR112018075855A2 (en) |
| CA (1) | CA3025445A1 (en) |
| IL (1) | IL263523A (en) |
| MX (1) | MX2018015629A (en) |
| RU (1) | RU2765826C2 (en) |
| SG (1) | SG11201811244SA (en) |
| WO (1) | WO2017216560A1 (en) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2014170480A1 (en) | 2013-04-18 | 2014-10-23 | Fondazione Telethon | Effective delivery of large genes by dual aav vectors |
| AU2018261769B2 (en) | 2017-05-05 | 2024-03-14 | The Regents Of The University Of California | Compositions and methods for expressing Otoferlin |
| US20210147870A1 (en) * | 2018-04-05 | 2021-05-20 | Nightstarx Limited | Compositions and methods for the treatment of stargardt disease |
| WO2020079034A2 (en) * | 2018-10-15 | 2020-04-23 | Fondazione Telethon | Intein proteins and uses thereof |
| GB201817469D0 (en) * | 2018-10-26 | 2018-12-12 | Univ Oxford Innovation Ltd | Gene therapy for retinal disease |
| WO2020093018A1 (en) | 2018-11-01 | 2020-05-07 | University Of Florida Research Foundation, Incorporated | A codon optimized otoferlin aav dual vector gene therapy |
| EP3921032A4 (en) | 2019-02-08 | 2022-11-30 | Decibel Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR TREATING SENSORY-NEURO DEAFNESS USING TWO-VECTOR SYSTEMS FOR OTOFERLIN |
| WO2021015997A1 (en) * | 2019-07-15 | 2021-01-28 | President And Fellows Of Harvard College | Methods and compositions for gene delivery |
| CN115666658A (en) * | 2020-04-01 | 2023-01-31 | 佛罗里达州大学研究基金会 | Dual AAV-MYO7A vectors with improved safety for treatment of USH1B |
| EP4196140A4 (en) * | 2020-08-14 | 2024-11-27 | Case Western Reserve University | PLASMID VECTORS AND NANOPARTICLES FOR THE TREATMENT OF EYE DISEASES |
| CA3218195A1 (en) * | 2021-05-07 | 2022-11-10 | Robin Ali | Abca4 genome editing |
| US20240425876A1 (en) * | 2022-02-25 | 2024-12-26 | Chigenovo Co., Ltd. | Expression cassette combinations and uses thereof |
| WO2024257061A1 (en) * | 2023-06-16 | 2024-12-19 | Arkasubhra Ghosh | A hybrid dual aav vector system with splice enhancer elements for expression of large genes |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1945779B1 (en) * | 2005-10-20 | 2013-03-06 | UniQure IP B.V. | Improved aav vectors produced in insect cells |
| US20090214478A1 (en) * | 2008-02-21 | 2009-08-27 | Alberto Auricchio | Method of treating ocular diseases by gene therapy |
| SG178254A1 (en) * | 2009-08-07 | 2012-03-29 | Transgene Sa | Composition for treating hbv infection |
| WO2013075008A1 (en) * | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| WO2014170480A1 (en) * | 2013-04-18 | 2014-10-23 | Fondazione Telethon | Effective delivery of large genes by dual aav vectors |
-
2017
- 2017-06-14 US US16/309,428 patent/US20190309326A1/en not_active Abandoned
- 2017-06-14 WO PCT/GB2017/051741 patent/WO2017216560A1/en not_active Ceased
- 2017-06-14 AU AU2017286623A patent/AU2017286623A1/en not_active Abandoned
- 2017-06-14 EP EP17732175.9A patent/EP3472328A1/en not_active Withdrawn
- 2017-06-14 BR BR112018075855-8A patent/BR112018075855A2/en not_active Application Discontinuation
- 2017-06-14 CN CN201780037639.9A patent/CN109642242A/en active Pending
- 2017-06-14 CA CA3025445A patent/CA3025445A1/en not_active Abandoned
- 2017-06-14 JP JP2018565324A patent/JP2019523648A/en active Pending
- 2017-06-14 MX MX2018015629A patent/MX2018015629A/en unknown
- 2017-06-14 KR KR1020197001281A patent/KR20190020745A/en not_active Ceased
- 2017-06-14 RU RU2019100525A patent/RU2765826C2/en active
- 2017-06-14 SG SG11201811244SA patent/SG11201811244SA/en unknown
-
2018
- 2018-12-05 IL IL263523A patent/IL263523A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| MX2018015629A (en) | 2019-09-26 |
| CN109642242A (en) | 2019-04-16 |
| RU2019100525A (en) | 2020-07-15 |
| KR20190020745A (en) | 2019-03-04 |
| JP2019523648A (en) | 2019-08-29 |
| AU2017286623A1 (en) | 2018-12-20 |
| IL263523A (en) | 2019-01-31 |
| EP3472328A1 (en) | 2019-04-24 |
| US20190309326A1 (en) | 2019-10-10 |
| BR112018075855A2 (en) | 2019-04-02 |
| CA3025445A1 (en) | 2017-12-21 |
| WO2017216560A1 (en) | 2017-12-21 |
| RU2765826C2 (en) | 2022-02-03 |
| RU2019100525A3 (en) | 2020-07-15 |
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